S. HRG. 103-694
SICKLE DISEASE RESEARCH: AN UPDATE
Y4.L 11/4: S. HRG. 103-694
Sickle Disease Research: An Update, ... RING
OF THE
COMMITTEE ON
LABOR AND HUMAN RESOURCES
UNITED STATES SENATE
ONE HUNDRED THIRD CONGRESS
SECOND SESSION
ON
TO AWARD A GRANT TO THE LOUISIANA DEPARTMENT OF HEALTH
AND HOSPITALS TO ESTABLISH AND CONSTRUCT THE NATIONAL
CENTER FOR SICKLE CELL DISEASE RESEARCH AT SOUTHERN UNI-
VERSITY IN BATON ROUGE, LA, AND FOR RELATED FACILITIES AND
EQUIPMENT AT SUCH CENTER
JULY 28, 1994
Printed for the use of the Committee on Labor and Human Resources
U.S. GOVERNMENT PRINTING OFFICE
82-663 CC WASHINGTON : 1994
°" 26 1994
For sale by the U.S. Government Printing Office
Superintendent of Documents, Congressional Sales Office, Washington, DC 20402
ISBN 0-16-044846-8
S. HRG. 103-694
SICKLE DISEASE RESEARCH: AN UPDATE
Y4.L 11/4: S. HRG. 103-694
Sickle Disease Research: An Update, . . . RING
OF THE
COMMITTEE ON
LABOR AND HUMAN RESOURCES
UNITED STATES SENATE
ONE HUNDRED THIRD CONGRESS
SECOND SESSION
ON
TO AWARD A GRANT TO THE LOUISIANA DEPARTMENT OF HEALTH
AND HOSPITALS TO ESTABLISH AND CONSTRUCT THE NATIONAL
CENTER FOR SICKLE CELL DISEASE RESEARCH AT SOUTHERN UNI-
VERSITY IN BATON ROUGE, LA, AND FOR RELATED FACILITIES AND
EQUIPMENT AT SUCH CENTER
JULY 28, 1994
Printed for the use of the Committee on Labor and Human Resources
U.S. GOVERNMENT PRINTING OFFICE
82-553 CC WASHINGTON : 1994
For sale by the U.S. Government Printing Office
Superintendent of Documents, Congressional Sales Office, Washington, DC 20402
ISBN 0-16-044846-8
0Cr26fi94
COMMITTEE ON LABOR AND HUMAN RESOURCES
EDWARD M. KENNEDY, Massachusetts, Chairman
CLAIBORNE PELL, Rhode Island
HOWARD M. METZENBAUM, Ohio.
CHRISTOPHER J. DODD, Connecticut
PAUL SIMON, Illinois
TOM HARKIN, Iowa
BARBARA A. MIKULSKI, Maryland
JEFF BINGAMAN, New Mexico
PAUL D. WELLSTONE, Minnesota
HARRIS WOFFORD, Pennsylvania
NICK LTTTLEHELD, Staff Director and Chief Counsel
SUSAN K. HattaN, Minority Staff Director
NANCY LANDON KASSEBAUM, Kansas
JAMES M. JEFFORDS, Vermont
DAN COATS, Indiana
JUDD GREGG, New Hampshire
STROM THURMOND, South Carolina
ORRIN G. HATCH, Utah
DAVE DURENBERGER, Minnesota
01)
CONTENTS
STATEMENTS
Thursday, July 28, 1994
Page
Johnston, Hon. J. Bennett, a U.S. Senator from the State of Louisiana;
and Hon. William Jefferson, a Representative in Congress from the State
of Louisiana ■£
Prepared statement of Senator Johnston <*
Lenfant, Claude, M.D., Director, National Heart, Lung, and Blood Institute,
National Institutes of Health ?
Prepared statement ••■••• ••• •••• 9
Kennedy, Hon. Edward M., a U.S. Senator from the State of Massachusetts,
prepared statement ■»•■ ■»•• •— •» •■•"•••'••••••:••" *°
Agnew, Shawnita, honor student, English High School Boston, MA; Lillian
McMahon, M.D., Director, Comprehensive Sickle Cell Center, Boston City
Hospital, Boston, MA; Ernest A. Turner, M.D., Director, Comprehensive
Sickle Cell Center, Meharry Medical School, Nashville, TN; William E.
Moore, M.D., vice chancellor for academic affairs, Southern University,
Baton Rouge, LA; Kwaku Ohene-Frempong, M.D., Director, Comprehensive
Sickle Cell Center, The Children's Hospital of Philadelphia, Philadelphia,
PA; and Hon. Charles D. Jones, Louisiana State Senator 17
Prepared statements of:
Ms. Agnew j-°
Dr. McMahon 21
Dr. Turner 45
Dr. Moore 29
Dr. Ohene-Frempong 39
Mr. Jones 53
an>
SICKLE DISEASE RESEARCH: AN UPDATE
THURSDAY, JULY 28, 1994
U.S. Senate,
Committee on Labor and Human Resources,
Washington, DC.
The committee met, pursuant to notice, at 1:34 p.m., in room
SD-430, Dirksen Senate Office Building, Senator Edward M. Ken-
nedy (chairman of the committee) presiding.
Present: Senators Kennedy, Simon, and Wellstone.
Opening Statement of Senator Simon
Senator Simon [presiding]. The hearing will come to order.
This is a hearing requested specifically by Senator Bennett John-
ston, and we are pleased to have it. I am pinch-hitting temporarily
for Senator Kennedy, who will be along before very long to join us
for the hearing.
The hearing is about sickle cell anemia, a very major problem in
our country, and the question is whether we are doing enough re-
search in this field. In the whole field of science, there is no one
in the U.S. Senate who is as knowledgeable as Senator Bennett
Johnston. I have to say in candor, I do not know if that applies to
sickle cell anemia, but I have worked with him on a variety of
other scientific projects and have always been amazed at the depth
of his knowledge.
One of the questions is whether we should be earmarking funds,
and how do we make sure that we do the kind of research job that
needs to be done.
So we are pleased to call on you, Senator Johnston, to open this
up.
STATEMENTS OF HON. J. BENNETT JOHNSTON, A U.S. SEN-
ATOR FROM THE STATE OF LOUISIANA; AND HON. WILLIAM
JEFFERSON, A REPRESENTATIVE IN CONGRESS FROM THE
STATE OF LOUISIANA
Senator Johnston. Mr. Chairman, thank you very much, and
thank you for your very kind comments, which I appreciate.
Mr. Chairman, this bill would establish a sickle cell anemia re-
search center at Southern University in Baton Rouge.
I have a written statement, which I would like to submit for the
record.
Senator Simon. It will be entered in the record.
Senator Johnston. I think the members of this committee gen-
erally are familiar with the scourge of sickle cell anemia, which is
(1)
really the scourge of the African American community in this coun-
try.
The trait in the hemoglobin affects 10 percent of the African
American population. Not that many are afflicted, but among that
10 percent, you never know where it is going to strike and when
it is going to strike, and when it does, it is terribly severe, and it
has just been a terrible thing for the African American community
and one that cries out for research; there is not enough researcn
being done.
The real focus, I think, of S. 1724 is not whether or not sickle
cell anemia is serious enough to warrant the kind of Federal effort
that this envisages. I think that question answers itself, and I
think it is overwhelmingly in favor of the affirmative answer. Nor
is it arguable that we know enough about it. I think we clearly
need to do that.
The question is why Southern University. That, I think, is really
the crux of this, and that was the question that submitted itself to
the President who, on two occasions, endorsed and committed him-
self to a sickle cell anemia research center at Southern University
in Baton Rouge. And it is a question that addressed itself to the
Black Caucus in Congress, and the Black Caucus addressed the
question, and every, single member of that Caucus — and I would
like to submit for the record letter to Donna Shalala endorsing this
research center, signed by each and every member of the Black
Caucus, some of whom have in their districts institutions that do
researcn.
So at least insofar as this administration and all of the members
of the Black Caucus, they have made the determination that
Southern is the appropriate spot.
So the question for this committee is why Southern. Well, we
begin with the fact that Southern University is the largest histori-
cally black college or university in the country. It has 16,000 stu-
dents, among whom are some distinguished graduates, some less
distinguished — I am only kidding — my colleague, Congressman
William Jefferson, seated beside me, is a graduate of Southern Uni-
versity undergrad and some other, mucn less distinguished, law
school — Harvard, I think it was.
Thirty percent of the population of my State of Louisiana is Afri-
can American, among which 10 percent have this sickle cell trait
in their blood. That means 130,000 Louisianians have this trait.
More to the point, there has been a commitment by Southern
University to fund this center. You will be hearing later from the
president and the chancellor from Southern University, who will
tell you more about that commitment, but suffice it to say that the
State of Louisiana, through our Governor, and Southern University
have committed to fund this institution, to keep its research funded
as it goes on, and the legislature has voted $7 million which is now
available in a fund for the construction. So that the commitment
of the State of Louisiana, of Southern University, and of the legis-
lature is complete and is not matched anywhere else in the coun-
try.
Now, what is their capability of doing this? Well, the legislation
calls for, and there has been collaboration with Louisiana State
University Medical School and Tulane University Medical School,
along with the other sickle cell anemia research institutions in the
country, including NIH, Duke, and Children's Hospital of Philadel-
phia, which have all given resources and have, in the case of
Tulane and LSU, committed to enter into cooperative agreements
in setting up this institution, which in turn would draw upon the
very best in research, the best experts in the country. The full-time
faculty would be given tenure status at Southern University, to
which Southern has committed.
So that what we have in effect is a total commitment from the
administration, from the Black Caucus, from Southern University,
from our Governor, from our legislature, and from the State of Lou-
isiana to ensure that this institution would be the best and do the
best kind of research that is possible to do.
Mr. Chairman, I do not think — I know — that there is no other in-
stitution that can make that statement. There are others that per-
haps have, along with a lot of other research — NIH, for example,
does very good research — but the African American community
wants a place that they can identify as the clearinghouse where
this is the principle endeavor, the principle focus, and the principal
reason and raison d'etre is sickle cell research — not hundreds of
other research endeavors, worthy though they may be, but one
committed to sickle cell that they can claim as their own and that
the State of Louisiana will fully support.
Mr. Chairman, I strongly urge this committee to act favorably
upon this bill.
Senator Simon. Thank you very much.
[The prepared statement of Senator Johnston follows:]
Prepared Statement of Senator J. Bennet Johnston
If you are an African American, and particularly if you are an African American
parent, S. 1724 may be one of the most important pieces of legislation considered
by the Congress this year.
S. 1724 establishes a national center for research on Sickle Cell Disease, a pain-
ful, life-threatening, inherited illness which affects African Americans almost exclu-
sively. Approximately one in every twelve black Americans is born with the sickle
cell trait; about one in every 400 has the disease.
Because it is genetic in origin, parents pass the disease to their children. If both
parents carry the trait, one in four of their children, on a statistical basis, will have
the disease. For thousands of African-American parents, the joy of childbirth is
marred by fear, because in our present condition of medical ignorance, doctors do
not know how to prevent the disease or cure it. The best they can do is treat pa-
tients for the excruciating pain which accompanies a disease crisis. Children af-
flicted with severe forms of the disease — about one case in four — do not usually live
through adolescence.
No group of Americans should have to watch its children live and die in pain if
there is anything the Congress can do about it. In this case, I believe that we can
do something which will help those with the disease, and which will offer hope to
those carrying the trait. We can establish a national center to serve as a resource
for those affected by the disease and those trying to eliminate it: patients, trait-
bearers, doctors, psychologists, counselors and researchers. This center would have
three missions.
First, it would perform, or collaborate in the performance of research into the na-
ture of the disease: a hemoglobin mutation which causes the distortion of red blood
cells. Research will also be conducted in the areas of molecular biology and genetic
engineering: including DNA manipulation.
The tragedy of sickle cell disease is that parents suffer as much as their afflicted
children. The second function of a national center would be the examination of the
psychosocial aspects of the disease, including the effectiveness of various counseling
and education methods. Data from broad-based studies would be gathered to illu-
minate public policy issues such as the mandatory genetic testing of susceptible
newborns.
Finally, a national center would function as a clearinghouse for all available infor-
mation, both biomedical and psychosocial, about the disease. A national center is in
a position to track ongoing research projects at universities and hospitals across the
country, to organize their findings and disseminate this information, in an acces-
sible form, to research scientists worldwide.
S. 1724 directs that this national center be located at Southern University in
Baton Rouge, Louisiana. Founded 112 years ago, Southern is a land grant college
with a distinguished teaching faculty. Its administration is committed to scholarship
and the preparation of minority students for the professions, including medicine and
the sciences. Southern is the largest predominantly African American university in
the United States with over 16,000 students enrolled. Because its administra-
tion,faculty, students and alumni are all affected, directly or indirectly, by sickle cell
disease, research performed at Southern will have an urgency and involvement not
necessarily found at other institutions.
Location of the Center at Southern is supported by the State of Louisiana. Sickle
cell disease is a serious public health problem for Louisiana's 30 96 minority popu-
lation: one of every 10 African Americans in the state — 130,000 people — carries the
sickle cell trait. The Louisiana Legislature has indicated the strength of its commit-
ment by appropriating $7 million for the project, contingent upon a three to one
match from Federal funds.
Southern University is prepared, without reservation, to commit its resources and
personnel to this project. The University will offer faculty appointments and a ten-
ure track in order to attract qualified research faculty to the Center, and will en-
courage undergraduate students to work as research assistants. Realizing that there
might be concern about establishing a biomedical research center at what is pri-
marily a teaching institution, planners for the Center have made every effort to
draw upon existing medical resources in Louisiana and in the sickle cell research
community.
A team of eminent scientists with expertise in sickle cell research will act as a
Technical Advisory Council to the director of the Center. A number of these experts
including representatives of Sickle Cell Centers at NIH, Duke University and the
Childrens Hospital of Philadelphia have already participated in a planning session
and submitted suggestions for projects to be undertaken by the Center.
Louisiana medical centers, including the Earl R. Long Memorial Hospital in Baton
Rouge, the Louisiana State University centers at New Orleans and Shreveport and
the Tulane University Medical Center, have indicated their support for the Center.
They are willing to enter into collaborative arrangements to conduct clinical trials
of the Center's research findings. Southern University's plan for a National Center
is fully supported by the Louisiana community and, nationwide, by the sickle cell
disease research community. It represents a systematic and well-thought-out attack
on an important public health problem. Its research offers the possibility of finding
a cure or, at the very least, a more effective treatment for an agonizing disease
which targets a vulnerable segment of our population. It deserves the support of the
Congress.
Senator Simon. We are pleased to have Congressman Jefferson,
who by now is a veteran in the House.
Mr. Jefferson. Thank you, Mr. Chairman. Good afternoon to
you and to others who make up this committee.
I reluctantly admit that my Senator has covered just about ev-
erything that is important to be said here today and has covered
it, I think, with a great deal of compassion and sincerity, which
shows his strong commitment to this issue.
Those of us in Louisiana see him as a guy who works a lot of
mundane little things, moving around the issues, and putting bills
together. He exhibits his passion very carefully. But today, Mr.
Chairman, I think we saw a dose of it, and I think that when he
shows it, he really means it, and I am glad that he is working hard
on this bill, which I think is worthy of all of our best efforts.
So I do appreciate the opportunity to appear before you today to
give a brief statement in support of his bill, S. 1724, a bill to estab-
lish the sickle cell disease research center at Southern University.
5
I am in strong support of this legislation, and I hope that the Sen-
ate will pass this bill this year.
I want to commend you, Mr. Chairman, for your leadership in
the area of medical research. You have been in the forefront in au-
thorizing important research programs that will 1 day — and some
have already done it — find" cures for many of the diseases that are
affecting millions of people around the world.
As a graduate of Southern University, as Senator Johnston has
already informed you, which is the largest predominantly African
American university in the country, I am proud that this center
will be cited if this legislation passes — and we are hopeful that it
will — at Southern University. Southern University has committed
itself, its resources, and its personnel to seeing that this project is
successful and that it produces a cure for this dread disease.
As Senator Johnston described in general, the sickle cell disease
is a serious illness that disproportionately affects African Amer-
ican. One of every 12 African Americans is born with the sickle cell
trait, and about one in every 600 develop the sickle cell disease. In
my State, that means 130,000 people have the trait out of 1.3 mil-
lion people, and approximately 4,000 people currently suffer from
the disease in Louisiana.
Although the impact of this disease has been greatest in the Afri-
can American community, it also occurs in other populations, in-
cluding those of Puerto Rican, Cuban, Southern Italian ancestry,
and more recently, other population groups.
Despite this well-documented data on this disease, despite its
high incidence of occurrence and its spread to broader populations,
there is no national research center seeking a cure for this deadly
disease.
Mr. Chairman, sickle cell disease is a very painful disease caused
by inadequate transportation of oxygen due to an abnormal type of
hemoglobin molecule in the red blood cells. Even with medication,
this disease will cause long-term suffering. Until recently, a person
with this disease usually died before the age of 20. Victims of this
disease have strokes, heart attacks, and other fatal illness due to
oxygen loss.
We think it is time the Congress and the Nation make important
steps to address this tragic oversight. The legislation before us
today creates an excellent partnership between the Federal Gov-
ernment, State, and teaching universities in Louisiana. The State
of Louisiana has already appropriated $7 million to match our re-
quest for a $21 million Federal grant from the Department of
Health and Human Services.
Southern University has devoted significant resources toward the
development of this research center, as Senator Johnston has
talked about, and there will be a collaborative arrangement for
joint research programs and projects between LSU, Tulane, and
other medical ana research institutions in our State and in our
city.
Mr. Chairman, today, you will hear from a number of people in-
volved in this project who are committed to making this national
research center the best research facility on this issue in the world.
I hope that you and the members of this committee will support
this important bill.
6
Thank you very much for giving me this chance to appear before
you.
Senator Simon. I thank you both.
Let me just ask a question, and I am speaking for myself now
and not for the chairman of the committee. How did you arrive at
the $21 million figure? Ordinarily, in NIH grants, we have the peer
review process. How do you handle that aspect of it to make sure
that we really are spending our money wisely?
Senator Johnston. First of all, they have formed a coordinating
committee made up of researchers from all around the country —
NIH, Children's Hospital of Philadelphia, LSU, Tulane, and South-
ern University has committed personnel to this — who have done
the preliminary design upon which the $21 million — actually, it is
$28 million, because the State of Louisiana has put up $5 million,
and I think they have just appropriated an additional $500,000 —
so that comprises the $28 million figure.
Now, this will be a national canter, drawing on the finest re-
searchers from around the country. We do not intend to have this
be an in-house research center, but rather a national center, fund-
ed, to be sure, by the State of Louisiana through the Southern Uni-
versity budget. But the researchers who are available from around
the country will be those who do the work at Southern University.
Now, in terms of the peer review, Mr. Chairman, of course, it
would be possible to say, well, let us do this research at NIH. But
the legislature of NIH has not put up $7 million; the President has
not committed to build this at NIH. The Black Caucus has not en-
dorsed NIH. And I think it is because there is a particular feeling
of— is the word "ownership" of this disease. I mean, this is a dis-
ease that afflicts African Americans, and Southern University is
their institution, and there is that proprietary interest at Southern
University. It is not just a Louisiana deal because it is endorsed
by every, single member of the Black Caucus.
So I think that is the reason, rather than peer review. We expect
the peers to be those who run the institution.
Mr. Jefferson. I would agree with what Senator Johnston has
said, and in addition, I would say that there is nothing inconsistent
about what we are doing here and the idea of peer review. There
can be peer review of this proposal, and there can be done peer dis-
cussion about the funding levels. If someone wants to quibble about
whether it should be lower or higher. So this does not prevent a
real examination of whether this proposal ought to go forward as
it is presented or whether it ought to be rearranged. But it does
set a framework within which this discussion can take place, and
that is really all that we are asking to have done here.
Senator Simon. All right. I was not suggesting that it ought to
be NIH, but simply that we ought to have some mechanism so that
we make sure — and I do not mean this disrespectfully to Southern
University — that we are really putting this where we ought to be
putting the money.
Senator Johnston. Oh, yes; we solicit that, we seek that, be-
cause indeed, we do want to make it a national institute of the very
highest quality. So, yes, on oversight, during the construction, dur-
ing the formulation of the research projects, we very much seek
that, and if the legislation should more explicitly say that, we
would welcome that.
Senator Simon. And real candidly, I was drafted at the last
minute to preside here, as both of you understand, although I have
been interested in this subject for a long time. And we appreciate
your testimony. It is a worthy cause, and it sounds like something
that we ought to be looking at very, very carefully.
I appreciate your being here. Thank you.
Senator Johnston. Thank you, Mr. Chairman.
Mr. Jefferson. Thank you, Senator.
Senator Simon. Our next witness is Dr. Claude Lenfant, Director
of the National Heart, Lung, and Blood Institute at NIH. We are
pleased to have you here, Dr. Lenfant, speaking in behalf of your
institute.
Please proceed.
STATEMENT OF CLAUDE LENFANT, M.D„ DIRECTOR, NA-
TIONAL HEART, LUNG, AND BLOOD INSTITUTE, NATIONAL
INSTITUTES OF HEALTH
Dr. Lenfant. Thank you very much, Mr. Chairman.
I am very pleased indeed to be here. As you just noted, I am
Claude Lenfant, and I am director of the National Heart, Lung,
and Blood Institute, which is one of the institutes at the NIH
which has just been discussed.
My institute is responsible for the sickle cell disease program
that we are supporting, and the invitation which I received from
Senator Kennedy was to discuss with you the sickle cell disease
center that the National Institutes of Health supports.
I cannot think of a better way to present to you the national net-
work of sickle cell disease centers that we are supporting than by
telling you about the research accomplishments which have been
realized since the creation of this network in 1972.
With your permission, I would like to take you very briefly
through two charts which I have here. I hope you can see my little
pointer here, with the red dots.
Senator Simon. Yes.
Dr. Lenfant. OK. At the bottom, we have a span of three dec-
ades, from 1970 to the year 2000. Here, what you have is the re-
search progress which has been accomplished during that period of
time since we have been supporting the center and where we ex-
pect to go. You can see for each decade some very significant
achievements which have been accomplished which have led us to
where we are today.
Senator Simon. Is the red line dollars spent?
Dr. Lenfant. The red line represents the progress which has
been made and the accumulation of knowledge which is going to
eventually bring us to a cure of this condition.
Senator Simon. How do you measure that? When you put it on
a graph, it looks very precise.
Dr. Lenfant. It is not that precise. It is a symbolic representa-
tion of the progress. What I am attempting to show you is that in
the early 1970 s, when the center network program was created,
our knowledge was very low, very rudimentary, and over the years,
with very significant support from the Congress, our knowledge has
8
increased to bring us close to what we believe is a cure for this dis-
ease. It is in sight. I cannot tell you whether it is going to be in
3 years or in 4 years, but I can tell you without any hesitation that
it is in sight.
And basically, what you see on these charts is the history of the
basic research that has been done — very basic research at the cel-
lular, at the molecular level. And in fact, I am sure, Senator, you
hear in Congress and elsewhere the term, "molecular medicine."
Today, medicine is seen at the molecular level. And it may be of
interest to you that the term, "molecular medicine," was invented
in relation to all the discoveries and the advances which have
taken place relative to sickle cell disease. In fact, the gene which
controls this disease and which controls the hemoglobin, which we
all have in our red cells, is really the gene about which we have
the most knowledge today.
The second chart brings you the clinical research and the ad-
vances which have taken place during that period of time. Here,
during the first decade, you can see great advances in comprehen-
sive care, treatment of eye abnormality — in fact, I should tell you,
Senator, that a great deal of the work concerning the eye abnor-
mality comes from investigators from Chicago, from your State; at
this point in time, we are no longer supporting them, but when
they were supported by the Institute, we were extremely proud of
the very remarkable work which came from Chicago.
I would like to underscore susceptibility to infection. So many of
these children who are born with sickle cell disease are at very
high risk of pneumococcal infection, which is a respiratory system
infection, and would die from that. But through the research that
has been done, we have found ways to prevent this complication,
and we can see, as we move along from the 1970's to now, all the
advances which have been made in prenatal diagnosis, in the pre-
vention of stroke, which is a complication of this condition, the de-
velopment of prophylactic penicillin to treat this infection that I
was mentioning, the development of methods for newborn screen-
ing so we can identify babies born with the sickle cell trait and pre-
vent occurrence of complications, and so forth.
Now, to finish my remarks, I would like to return to the first
chart and tell you that the basic research that is ongoing today is
bringing us to, if you will, the eve of finding a cure for this condi-
tion. And I am focusing most specifically on bone marrow trans-
plantation and gene therapy, two therapies, two approaches, that
the Congress hears a lot about from the research community.
Bone marrow transplantation is not yet used in many children.
In fact, only 14 cases have been completed in the whole world — I
believe, 6 in Europe and 8 in this country — with very, very inter-
esting results. Gene therapy is also something which is getting to
be in our possibilities, and n fact, within a couple of weeks, the Na-
tional Heart, Lung, and Blood Institute is going to initiate a na-
tional program of gene therapy research for the cure of sickle cell
disease.
We think that the prospects from these two therapeutic ap-
proaches are just enormous, and we are very confident that within
some period of time— but again, I have to tell you I cannot say
whether it will be in 2 years, 5 years, or 10 years — but we know
where to go, and we have the opportunity to do it, we have the
basic science which permits us to reach the goals that we want to
reach.
So when I was asked to come here and tell you about this na-
tional network of sickle cell research centers that has been sup-
ported by the Congress since 1972, I thought the best way to
present that to you would be to say here are all of the things wnich
nave been accomplished, and this is where we are today and what
we expect to accomplish within an attainable period of time.
I would be glad to answer questions, Mr. Chairman.
[The prepared statement of Dr. Lenfant follows:]
Prepared Statement of Claude Lenfant, M.D.
Mr. Chairman and members of the Committee, I am Dr. Claude Lenfant, Director
of the National Heart, Lung, and Blood Institute (NHLBI), a component of the Na-
tional Institutes of Health (Nffl). I am pleased to appear before you today to present
testimony about our research program in sickle cell disease and, in particular, about
the Comprehensive Sickle Cell Centers grant program.
BACKGROUND
To place my testimony in context, let me review some basic facts about sickle cell
disease, the most common serious inherited blood disorder seen in this country. It
is characterized by recurrent bouts of pain called "crises," chronic anemia related
to accelerated destruction of red blood cells, increased susceptibility to certain infec-
tions, and acute or chronic damage to various organs. Sickle cell disease results
when the gene for defective ("sickle *0 hemoglobin is inherited from both parents.
In the United States, it occurs predominantly, but not exclusively, in persons of Af-
rican ancestry; about 50,000 to 60,000 African Americans are affected. Medical costs
for patients with sickle cell disease can be extremely high, quality of life is im-
paired, and loss of time from school or employment is common. Thus, sickle cell dis-
ease is a problem of significant psychological, social, and economic importance.
President Nixon's health message to Congress in 1972 identified sickle cell ane-
mia as a major neglected health problem. The National Sickle Cell Anemia Control
Act, subsequently passed by Congress, provided authorization to establish programs
of research, research training, and demonstration service activities related to the di-
agnosis, treatment, and control of sickle cell anemia. The NIH was designated as
the lead agency for the National Sickle Cell Disease Program, and the NHLBI was
assigned responsibility for research and development activities and for coordination
across federal agencies. The National Sickle Cell Disease Advisory Committee was
appointed by the Secretary, Department of Health, Education, and Welfare, and
charged with making recommendations on program direction and policy. The com-
mittee articulated the overall goals of the program as reduction of the frequency,
morbidity, and mortality of sickle cell anemia through a program of research and
development and demonstration activities in education, testing, counseling, patient
referral, and rehabilitation.
The Comprehensive Sickle Cell Centers (CSCC) Program
HISTORY AND ACCOMPLISHMENTS
Among the advisory committee's first recommendations was a program of com-
prehensive centers focusing on the range of problems associated with sickle cell dis-
ease. The Comprehensive Sickle Cell Center (CSCC) program was established in fis-
cal year 1972 as a direct result of this recommendation. The program began oper-
ation with a total of 10 centers and increased to 15 centers in fiscal year 1973. The
Institute has consistently supported 10 centers since fiscal year 1977, each for a
five-year award period. Continued funding for the CSCC program was ensured in
1983 by the passage of Public Law 97-414, which directed the Secretary of Health
and Human Services to "provide for the development and support of not less than
10 comprehensive centers for sickle cell disease.
The goals of the CSCC program are to conduct basic and clinical research to im-
prove the diagnosis and treatment of sickle cell disease and prevent its complica-
tions. The program encompasses both scientific investigation and service so that ad-
vances in research on sickle cell disease can be translated readily into practice and
10
incorporated into the health-care delivery system. Each sickle cell center is com-
prehensive in that it integrates and coordinates fundamental and applied research
with clinical applications and trials to assess various modes of therapy, with dem-
onstration projects that address methods of disease diagnosis and approaches to pa-
tient counseling and education, and with training programs for health-care profes-
sionals and allied personnel that focus on the specific problems associated with sick-
le cell disease. By requiring each center to be comprehensive, the NHLBI ensures
that a variety of models based on this approach are developed. Furthermore, by al-
lowing each center to select its own area or areas of primary emphasis, the Institute
encourages development of specialized expertise in individual centers that can serve
as a shared resource for the entire program.
This unique program concept or interrelated investigational and service compo-
nents has been noteworthy for accomplishments made possible by the synergistic
interaction of scientific investigators and a well-informed patient population.
Through this support mechanism, the scope and quality of research and clinical
studies have increased markedly. Highly eflective,innovative programs in education,
diagnosis, counseling, and patient care have been created where none existed before.
The program has proven successful in attracting talented scientists into studies re-
lated to sickle cell disease by providing challenging environments conducive to cross-
stimulation and synergism between specialties. Collaborative efforts among center
projects-and between centers enhance research productivity, facilitate technology
transfer between research and clinical components, and ensure a knowledgeable
study population through education and counseling.
The CSCC program has contributed to many important advances in understand-
ing molecular mechanisms and the pathogenesis of sickle cell disease. Investigations
conducted in the centers have led to: progress in basic sickle cell research and in
management of sickle-cell-related complications; better understanding of gene ex-
pression and regulation of fetal hemoglobin synthesis; a scientific foundation for
clinical trials of hydroxyurea as a therapeutic approach; modern DNA techniques for
prenatal diagnosis of sickle cell disease; identification and classification of over 300
abnormal hemoglobins; and guidelines for ocular sequelae of sickle cell retinopathy.
Clinical investigations have shed light on the natural history of sickle cell disease
and provided a basis for the ongoing collaborative national study on the clinical
course of sickle cell disease. The current centers have implemented additional col-
laborative clinical research protocols in preoperative transfusion therapy, pain man-
agement, acute chest syndrome, and use of magnetic resonance imaging to diagnose
and follow the progression of cerebral and skeletal events.
REVIEW PROCEDURES
The continued quality of the Institute's CSCC program is ensured by a careful
and thorough process that includes review and approval of the program concept by
the National Heart, Lung, and Blood Advisory Council, peer review of individual
grant applications, and internal review within the CSCCs. Grant applications in re-
sponse to announcements for CSCC support are subjected to a multistage review
process that parallels that for review of other large program grant applications. For
example, in the most recent competition for CSCC grants (for funding in fiscal years
1993 through 1997), 20 applications were received and given an initial review by
the parent peer review committee, a group of 18 expert scientists and clinicians con-
vened by the NHLBI. Seventeen applications were recommended for further consid-
eration. All were site visited by teams that included at least two representatives
from the parent committee, as well as scientists, clinicians, educators, and other
consultants with expertise in areas proposed by the applicants. Reports and rec-
ommendations of the site visit teams were then referred back to the parent commit-
tee for consideration at a second review meeting. Applications were evaluated ac-
cording to a number of criteria, including the qualifications, experience, and commit-
ment of the center director and senior personnel; the scientific merit and quality of
the proposed projects; the adequacy of laboratory and health-care facilities and ac-
cess to patients; the overall structure and management of the center, including sci-
entific and fiscal management, integration of the parts, and quality control; the in-
stitutional commitment to the program; and the appropriateness of the budget. The
parent committee voted a numerical priority score for each application, reflecting its
overall merit. Reports and recommendations of the parent committee were then con-
veyed to the National Heart, Lung, and Blood Advisory Council for final review and
consideration.
As with all NHLBI-supported programs, the review process does not terminate
with award of the grant. Each of the CSCCs is required to submit an annual
progress report for review by NHLBI program staff. NHLBI staff members also visit
11
each of the centers during the middle period of the grant cycle to provide construc-
tive guidance and work with center staff to determine what improvements might be
possible in their programs.
NHLBI Research in Sickle Cell Disease: Past, Present, and Future
The CSCC program has been and will continue to be an important element in our
national strategy to reduce the burden of sickle cell disease. However, I want to em-
phasize that it is but one part of a much larger effort. For the past 22 years, the
NHLBI has initiated and supported a broad-based program of basic and clinical re-
search in sickle cell disease with the goal of reducing deaths and improving the
quality of life for patients and their families. We believe our approach to achieving
this goal has been logical and stepwise. It began in the laboratory, with studies at
the most fundamental level of the red blood cell, moved to research on globin gene
expression and regulation, progressed to epidemiologic studies of the natural history
of sickle cell disease and, today, also encompasses clinical trials of potential thera-
peutic agents.
Let me give you a brief history of sickle cell disease research and summarize its
most important advances. This information provides a context in which to view the
contributions of the CSCC program, and offers important insights into where we
have been, where we are now, and where we will be in the future as we move rap-
idly toward the twenty-first century.
La the 1970s, basic research supported in scientific laboratories throughout the
country brought a tremendous revolution in our understanding of sickle cell disease
at the molecular level. One of our earliest Institute initiatives focused on research
to determine the mechanisms that regulate the 'switch" from fetal to adult hemo-
globin during infancy. It had been recognized for some time that sickle cell patients
who were fortunate enough to have inherited a tendency to continue producing fetal
hemoglobin beyond the first year of life had relatively benign disease. There fore, it
seemed logical to pursue therapeutic modalities that would enable patients produc-
ing adult sickle hemoglobin to "switch back" to producing normal fetal hemoglobin.
This research catalyzed the field of molecular biology, and became the cornerstone
for development of therapeutic approaches based on increasing the level of fetal he-
moglobin in patients with sickle cell disease- -work that continues to this day.
In 1977, sickle cell anemia became the first human disease to be described at the
level of DNA and RNA. Breakthroughs that rapidly followed introduced gene map-
ping into prenatal diagnosis, and made it possible to use placental tissue rather
than fetal Dlood samples. This substantially increased the safety of prenatal diag-
nosis for sickle cell disease, and rapidly led to the application of molecular genetics
for prenatal diagnosis of other inherited diseases.
Very early on, it became apparent that, although much was known about the mo-
lecular basis of sickle cell disease, little was known about its natural history or clini-
cal course. Only the sickest patients were described in the medical literature, and
most clinical reports of patient outcomes were anecdotal and retrospective. There
was, consequently, a poor understanding of the variable severity of the disease. In
1979, the NHLBI responded to the need for epidemiologic and clinical information
with the Cooperative Study of Sickle Cell Disease (CSSCD).
The CSSCD— a large scale, multi-institutional study — recruited over 4,000 pa-
tients of all ages, from newborns to people in their sixth decade. The study clarified
issues of growth and maturation patterns among children with sickle cell disease;
defined causes of death in the pediatric population, showing longer survival rates
than had been reported previously, described the epidemiology of painful episodes
and documented, for the first time, that the frequency with which such crises"
occur is a predictor of premature death in adult patients; and pointed out the risks
of alloimmunization for sickle cell patients receiving repeated blood transfusions.
Most recently, exciting new data from this study showed that average survival of
fiatients with sickle cell anemia has increased to 48 years of age for women and 42
or men. Previously, it was thought that the average patient rarely lived beyond 20
years of age. These findings, recently reported in the New England Journal of Medi-
cine dated June 9, have provided renewed impetus for the search for long-term
therapeutic agents to improve the quality of life lor sickle cell disease patients.
As mentioned earlier, the CSCC program has had a major impact on the manage-
ment of sickle cell disease. The centers have served as models for a revised manage-
ment approach that places the central focus on the patient. Care that was pre-
viously fragmented, impersonal, and episodic has been replaced with a team ap-
proach, involving a cadre of trained personnel that include not only the clinician,
but the nurse, social worker, psychologist, nutritionist, counselors, and allied health
professionals.
12
In the mid-1980s, an NHLBI-supported clinical trial demonstrated the value of
grophylactic penicillin in preventing major infections in infants and young children,
efore that tune, approximately 30 percent of sickle cell deaths occurred before five
years of age, most in children under the age of two. Pneumococcal infection was the
major cause of mortality in these early -years. Prophylactic penicillin has thus been
among the most dramatic contributions to saving lives. This trial also provided the
impetus for recommending that all newborns be screened for sickle cell disease. In-
fants at risk could then be referred for comprehensive care, and prophylactic penicil-
lin therapy could be given by three months of age. Sickle cell disease is now in-
cluded in the screening programs of more than 40 states.
Unfortunately, many children with sickle cell disease are faced with crippling
central nervous system (CNS) complications, including strokes. Chronic transfusion
therapy to maintain the level of sickle hemoglobin below 30 percent is, however, ex-
tremely effective in preventing the recurrence of strokes in such children. Current
and future emphasis is on preventing the initial strokes by early detection of nar-
rowed cerebral vessels and implementing therapy prior to CNS damage.
We see a new era of optimism for treating sickle cell patients. We are on the
threshold of moving molecular medicine even closer to the bedside. The NHLBI is
currently supporting a multicenter clinical trial using hydroxyurea in 21 centers
around the country. Approximately 300 severely affected adult patients are enrolled.
Now entering its thira year, this trial is investigating one of many agents identified
as able to "turn on" the production of fetal hemoglobin in patients with sickle cell
anemia. If efficacy and safety are proven, hydroxyurea will be the first agent to be-
come available as an effective therapy for sickle cell disease. Erythropoietin, a hor-
mone, also significantly increases fetal hemoglobin synthesis. The recent availability
of recombinant erythropoietin, a major scientific advance, made it possible to use
this drug in combination with hydroxyurea. This combination approach offers the
possibility that lower doses of hydroxyurea can be-used to achieve the required
therapeutic levels of fetal hemoglobin.
Because we recognize that this approach will not achieve therapeutic levels of
fetal hemoglobin in all patients, parallel research efforts to seek other strategies are
continuing. Looking into the future, gene therapy and bone marrow transplantation
appear to offer the best hopes for a cure of sickle cell disease. Bone marrow trans-
plantation has been successfully used by several investigators in Europe, as well as
a small number in the United States. Although early reports are promising, patient
selection, donor availability, and complications of the procedure continue to be po-
tential problems that prevent widespread use of this therapeutic modality today.
Gene therapy research is advancing, with the possibility of inserting normal genes
for hemoglobin production into the Done marrow precursor or stem cells, thus lead-
ing to the production of normal hemoglobin. This approach is receiving active atten-
tion by many researchers around the country ana is the subject of a targeted re-
search program recently initiated by the NHLBI.
A number of opportunities exist for further advances in sickle cell disease re-
search. What needs to be done was best stated by the Sickle Cell Task Force on
Investigator-Initiated Research. Its report defined areas of research that currently
hold the greatest promise for achieving a cure for sickle cell disease, including: de-
velopment of mice that serve as models of sickle cell disease, improved understand-
ing of factors important in the switch from fetal hemoglobin to adult hemoglobin,
targeted studies of methods for gene insertion into primitive blood cell precursors,
and fundamental studies of red cell structure and metabolism. Also recommended
were epidemiologic investigations of the role of clotting and clot dissolution in sickle
cell crises and additional studies on the natural history of sickle cell disease. The
NHLBI is strongly committed to a balanced and innovative approach to achieve our
national goal of reducing the personal and public health burden of sickle cell dis-
ease.
I would be pleased to answer any questions the Committee may have.
13
History of
Basic Sickle Cell
Research
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a>
Membrane
Proteins
Rheology
Polymer Formation
Cell Sickling
Kinetics of Delay
Bone Marrow
Transplantation
Hemoglobin
"Switching" Agents
Gene Therapy
Globin Gene
Regulation
'Haplotypes and
Population Studies
Sequence of the (3-globin
Gene
Globin Gene
Expression
1970 1980 1990 ?
Enlightenment Renaissance Therapeutic
Triumph
Period
2000 ?
Cure?
History of
Clinical Sickle Cell
<n
v
O)
o
sz
o
ca
0)
in
CD
DC
Comprehensive Care
Eye Abnormalities
Susceptibility to
Infections
Prenatal Diagnosis
"Natural History
Study-
Prevention
of Stroke
Research
Longer Lifespan
Painful Episodes -
Influence on Mortality
Leg Ulcers
Prophylactic
Penicillin
Aseptic Necrosis
Newborn Screening
CNS - Early Detection
and Prevention
Clinical Severity Index
1970 1980 1990 ? 2000 ?
Enlightenment Renaissance Therapeutic Cure?
Period
Triumph
82-553 0-94-2
14
Senator Simon. Thank you.
We now have 10 sickle cell centers.
Dr. Lenfant. That is correct.
Senator Simon. How would the center that is now being proposed
for Southern University differ from those centers?
Dr. Lenfant. Well, I should say to you, Mr. Chairman, that we
have not seen a detailed description of the proposal that would be
presented by the university. I have heard the two previous wit-
nesses, and many of the things which they have discussed, describ-
ing research that would span from very basic to clinical, describing
supports to the community and to the patients, treatment, care,
counseling, and all of these things, there is a very remarkable simi-
larity between what I think I have heard here and what our cen-
ters are now supporting.
Senator Simon. We will have to make a decision on this, obvi-
ously. If you were a member of the U.S. Senate, would you vote for
it? Is this a wise way to invest the money in terms of achieving
the breakthrough?
Dr. Lenfant. That is a very difficult question. Let me answer it
this way, if I may. The more research we support, the faster we
are going to get where we want to be. Just a few months ago, I
testified before the Appropriations Committee of the Senate for my
budget, and Mr. Harkin asked us, if you had so much more money,
what would you do? And my answer is basically the answer that
I am giving you today, that we know where to go, and we know
we are going to reach it; if I have more money, I will reach it fast-
er. I think that is a decision which is not within my purview.
Senator Simon. I understand. We thank you very, very much.
Senator Kennedv and other members of the committee may have
some questions for you.
Dr. Lenfant. I will be here, Senator, for the duration of the
hearing.
Senator Simon. Thank you. And they may be submitting some in
writing. Senator Kennedy had planned to be here by now.
My difficulty right now, frankly, is that I am supposed to be at
another meeting. I think what we had better do is recess tempo-
rarily until Senator Kennedy gets here, or another member of the
committee can get here. So we will take a temporary recess at this
point.
[Recess.]
Opening Statement of Senator Kennedy
The Chairman. We will come to order.
I am enormously grateful for the understanding and patience of
our witnesses this afternoon on a subject which is of enormous im-
portance to all of us on this committee and something which I have
been involved in for a very extensive period of time.
As the Senate schedule has indicated, we are caught in this situ-
ation of advancing the hearing and sharing the leadership in
chairing the hearing, so we are very, very grateful to all of those
who have testified and for the testimony we will receive.
I will put my full and complete statement in the record, and we
will utilize the time we do have to hear from some very special peo-
ple.
15
[The prepared statement of Senator Kennedy follows:]
Prepared Statement of Senator Kennedy
Despite the unprecedented growth in scientific knowledge and
the ability of medical science to diagnose, prevent, treat, and cure
many illnesses, sickle cell disease continues to devastate the lives
of tens of thousands of Americans.
Sickle cell disease affects an estimated 50,000 African Ameri-
cans. It is a hereditary illness that occurs in 1 out of every 625 Af-
rican American infants born in the United States. The death rate
for the disease during infancy can be as high as 30 to 35 percent
if treatment is delayed or inadequate. Those living with the disease
own have frequent attacks of severe pain. They suffer strokes at an
early age and endure debilitating joint and bone disease.
The severity of the symptoms and life expectancy vary consider-
ably. Some patients survive beyond middle age. But many die in
childhood, and many others die in infancy. Treatment for this
chronic illness is expensive and the disease places a heavy psycho-
logical burden on patients and caretakers alike.
The disease was first diagnosed in the United States in 1910 by
Dr. James Herrick. The discovery was made on a dental student
from Grenada, rather than on the thousands of African Americans
with the disease. It was called sickle cell anemia because of the
characteristic sickle shape of the red blood cells in patients with
the disease.
Ih 1949, Dr. Linus Pauling discovered the association of sickle
cell anemia with an abnormal form of hemoglobin, and later won
the Nobel prize for his work in this area. Eight years later, the mo-
lecular basis of the disease was discovered in one of the landmark
breakthroughs in the field of human genetics. Dr. Vernon at Mas-
sachusetts Institute of Technology demonstrated that the hemo-
globin in patients with sickle cell anemia differed from normal he-
moglobin by a single amino acid substitution on the beta chain of
the protein. In 1977, Dr. Bernard Forget identified the particular
gene responsible for the disease.
Patients with the disease inherit a sickle cell gene from both par-
ents. If each parent has a sickle cell gene, one-quarter of the chil-
dren on the average will inherit both genes and will have the dis-
ease.
Half of the children will inherit one of the genes. They will be
carriers of the genes, and can pass it on to their own children.
Their condition is called sickle cell trait, and 8 percent of African
Americans have it. The sickle cell gene has persisted in operation
even though a double dose of the gene is so lethal, because the red
cells of individuals sickle cell trait are less vulnerable to the ma-
laria parasite.
In spite of the remarkable knowledge that science has gained as
a result of sickle cell research, there has been a continuing percep-
tion over many decades that U.S public health policy has not given
enough priority to prevention of the disease and treating those who
suffer from it. The perception is especially troubling because of the
racial nature of the disease.
In 1971, in response to these concerns, I joined Senator John
Tunney, Senator Ed Brooke, and other Senators in sponsoring the
16
National Sickle Cell Anemia Act, which was signed into law by
President Nixon the following year. I still regard it as one of the
most important health bills I have ever introduced.
The Act contained numerous provisions on screening, counseling,
research and training. It included programs to educate the public
about the disease and to facilitate early diagnosis, and effective
treatment.
In 1972, the National Institutes of Health established the Sickle
Cell Disease Branch of the National Heart, Lung and Blood Insti-
tute. In the years since then, Congress has enacted legislation to
strengthen basic and applied research, to provide more extensive
testing, counseling, and treatment and to expand public informa-
tion and education programs.
An important provision in the Orphan Drug Act of 1983 provided
support for the establishment of 10 Comprehensive Sickle Cell Cen-
ters around the country. These national centers are now well-estab-
lished. They are located in Alabama, Georgia, Massachusetts,
North Carolina, Pennsylvania, and Tennessee, and two centers are
located in New York and California these centers are at the cutting
edge of research on the disease, and there each receive Federal
support of $1 to $3 million a year. Since 1972, the Federal grant
has spent $610 million on sickle cell research, and the amount for
the current year in $46 million.
The developments over the past two decades have seen signifi-
cant improvements in the treatment of patients with the disease.
Thirty years ago, 50 percent of patients with the disease died be-
fore the age of 20. Today, 50 percent live past the age of 50. Much
of the recent progress has come from research conducted by the ten
Comprehensive Sickle Cell Centers around the country and from
research supported by the Sickle Cell Disease Branch of the Na-
tional Heart, Lung and Blood Institute. These Centers have suc-
cessfully brought together experts in basic science, clinical re-
search, and psychosocial research to address the multitude of prob-
lems faced by those with the disease.
In the important effort to find a cure for the disease, promising
research is Deing carried out in bone marrow transplantation and
gene therapy. In addition, promising new anti-sickling drugs are
being tested.
In spite of these advances, much remains to be done. Our hear-
ing this afternoon will assess the effectiveness of all aspects of our
current efforts, identify unmet needs, and try to develop a strategy
to achieve more rapid progress in the coming years.
I look forward to the testimony from our witnesses today, and to
working closely with my colleagues on the committee and in the
Congress to achieve this goal.
The Chairman. Shawnita Agnew is an honor student at English
High School in Boston. In September, she will be a freshman at
Fisner College. She is a team leader in sickle cell support for chil-
dren, and she will share her experience with sickle cell disease.
Her mother is here as well, Ms. Sharon Agnew Stewart, who is a
sickle cell counselor.
We also welcome Lillian McMahon, who is director of the Com-
prehensive Sickle Cell Center at Boston City Hospital and has suc-
cessfully brought together experts in basic science research, clinical
17
research, and psychological research, to address the many problems
faced by those who have the sickle cell disease.
We also welcome Dr. Ernest Turner, who is director of the Com-
prehensive Sickle Cell Center at Meharry Medical School in Nash-
ville, TN. Clinical studies at Meharry have vast improved the un-
derstanding of sickle cell disease and its treatment; and Dr. Wil-
liam Moore is vice chancellor for Academic Affairs at Southern Uni-
versity, and is the principal investigator at two research centers,
minority institution projects funded by NIH.
Shawnita, we are glad to hear from you. We know that you have
endured a good deal of personal discomfort in joining us here today,
and we are also very mindful that it is difficult to share one's
health experiences and needs publicly, so we are certainly grateful
to you for your willingness to be here and for all the good things
that you do. We want you to know that the best way we can thank
you is to redouble our energy and commitment in terms of both the
research and, hopefully, solutions, to some of these challenges.
We are thankful to you for being here today, and we look forward
to hearing your comments. So if you would like to make a state-
ment, we would be glad to hear from you now.
STATEMENTS OF SHAWNITA AGNEW, HONOR STUDENT, ENG-
LISH HIGH SCHOOL, BOSTON, MA; LILLIAN McMAHON, M.D.,
DIRECTOR, COMPREHENSIVE SICKLE CELL CENTER, BOS-
TON CITY HOSPITAL, BOSTON, MA; ERNEST A. TURNER, M.D.,
DIRECTOR, COMPREHENSIVE SICKLE CELL CENTER,
MEHARRY MEDICAL SCHOOL, NASHVILLE, TN; WHXIAM E.
MOORE, MX)., VICE CHANCELLOR FOR ACADEMIC AFFAIRS,
SOUTHERN UNIVERSITY, BATON ROUGE, LA; KWAKU OHENE-
FREMPONG, MJ)., DIRECTOR, COMPREHENSIVE SICKLE
CELL CENTER, THE CHILDREN'S HOSPITAL OF PHILADEL-
PHIA, PHILADELPHIA, PA; AND HON. CHARLES D. JONES,
LOUISIANA STATE SENATOR
Ms. agnew. Hi. My name is Shawnita Agnew, and I am a young
lady who lives her life every day with a disease called sickle cell
anemia.
As a child, living with this disease was very hard for me. I really
did not understand it as much as I do now. Many times, I would
have to be admitted to the hospital with terrible pain in my legs,
my back, my arms, and sometimes my chest. I would need to stay
many days, weeks, sometimes months, with i.v. medication, oxygen,
and sometimes, blood transfusions, until the pain was bearable
enough to go home and later return to school.
As a child, I did not really understand why I could not partici-
pate with other children when they would swim, play running
games, and so on. I could not get too hot or too cold, excited, or
sad. It really confused me.
As I get older, I started to understand the disease more, but I
still did not participate as much. But I started to learn to live with
it. Together with other kids from Boston City Hospital, we formed
a teen group for support with each other.
I will be working with this teen group and recruiting younger
kids and helping them to understand and deal with the disease.
18
Now, being 18 years of age, I have really learned how to take
care of my sickle cell anemia. When I swim, I know to keep warm.
When I go out in the winter, I know to dress warm. When it is hot,
keep cool, and drink a lot.
In September, I will be moving on to Fisher Junior College to
study early childhood education, and I hope to become a teacher.
I think I have improved 90 percent from what I used to be.
The model for our teen support group is "A Cure in My Lifetime."
Please help us get there.
Thank you.
The Chairman. Thank you very much. We will come back to you
for some questions.
[The prepared statement of Ms. Agnew follows:]
Prepared Statement of Shawn^a Agnew
Senator Kennedy and members of the committee, hi, my name is Shawnita Agnew
and I am a young lady who lives her life every day with sickle cell disease, which
doesn't bother me any more.
As a child living with this disease was very hard for me. I really didn't under-
stand it as much as I do now. Many times I would have to be admitted to the hos-
pital with terrible pains in my body and needed to stay for days with I.V.'s, some-
times blood transfusions, and medications until the pain was bearable enough for
me to go home and later on return to school.
As a child I really didn't understand why I couldn't participate with the other
children when they would swim, when they would play running games, and etc. I
couldn't get cold or hot, excited or sad, it really confused me. Any of these things
could bring on a sickle cell crisis and I would end up in the hospital again.
As I got older I started to understand this disease more, but I still couldn't par-
ticipate as much. But I started to learn to live with it. Together with other kids
from Boston City Hospital and two other hospitals we formed a mutual support
team for teens with sickle cell disease at the Boston Sickle Cell Center. Together
we shared our experiences and learned more about the disease. Together we wrote
a booklet for teens by teens on what it is like to have sickle cell disease. We worked
on many art projects and Teens for Peace. I will be working with the Teen group
this year in recruiting younger kids and help them understand and deal with their
disease.
Now as me being 18 years of age I have really learned how to take cars of my
sickle cell disease myself. When I swim I know to keep warm. When I go out in
the winter time I know to dress warm. When it's hot I know to keep cool and drink
too much. So basically living with sickle cell disease doesn't bother me now. I live
my life as a regular life. It seems like I'm a normal person now. My mind has ac-
cepted the fact.
In September I will be moving on to Fisher Junior College to study Early Child-
hood Education and I hope to become a teacher. I myself think have improved 90
percent of what I used to be.
The motto of our Teen Group is "A Cure In My Lifetime". Please help us get
there. Thank you.
The Chairman. Dr. McMahon.
Dr. McMahon. Thank you, Senator Kennedy.
I have to say that Shawnita did not mention that she is right
now in the middle of a painful crisis. I offered to read her testi-
mony here before the committee, but she was determined to come
in spite of it to speak to groups of people who are important in
maintaining research and treatment of sickle cell disease. Thanks,
Shawnita.
We have travelled a long way since the age of 3, when she was
diagnosed with sickle cell disease, and there have been many,
many, many admissions for pain, for surgery, for infection, and a
couple of near misses. So she has come a long way, and she did not
19
mention that she did receive the headmaster's award in her school,
and many scholarships which will allow her to go to Fisher.
I want to thank you, Senator Kennedy, and the committee for in-
viting me here to speak about the Boston Comprehensive Center.
The Boston Center is one of 10 national comprehensive centers in
the United States, all of which work in an academic environment
within a university and medical center, which is actually essential
to bring both researchers and clinicians together so that research
does not happen in a vacuum, in a laboratory, and clinicians are
not delayed in delivering the most important findings of research
to their patients.
So Boston Sickle Cell Center is particularly important that it has
affiliated four medical schools — Harvard, Tufts, and Boston Univer-
sity— and our six research projects take place in six university-af-
filiated hospitals and one graduate school.
The Boston Center has really concentrated their research in red
cell membrane, endothelial adhesion and hemoglobin switching. It
is really impossible in 5 minutes to tell you the accomplishments
of the center, but I will try to highlight them.
In red cell membrane, our investigators have focused on under-
standing the alterations of the red cell membrane which contribute
to the disease. For example, one group of our researchers under
Jiri Palek has discovered sites in the red cell membrane where the
polymers of hemoglobin will penetrate, disrupt the membrane,
damage it, and cause cell damage. This then causes sickling, causes
the red cells to stick inside the lining of the blood vessels and occa-
sions obstructions to blood flow and produces pain or organ dam-
age. This is vital knowledge that has increased our overall under-
standing of the disease.
Another group of our researchers under Dr. Carla Brugnara has
developed a novel approach in attempting to prevent the dehydra-
tion of the red blood cell which occurs in sickle cell disease. They
began with the premise that a certain channel within the red cell
called the Gardos channel, which is calcium-dependent, has a pri-
mary role in the potassium loss and dehydration of the red cells.
They demonstrated in vitro that inhibitors of this channel actually
would block the water loss from the red blood cells. And one such
inhibitor, a drug called clotrimazole, which is a very common drug
used in treating fungal infections in the general population and ac-
tually had concentrations lower than what is routinely used, is ac-
tually effective in doing this. They studied this in transgenic mouse
model of sickle cell disease and demonstrated that indeed it is ef-
fective in preventing red cell damage. And on the basis of this very
exciting and promising work, we are now embarking on phase one
trials with patients with sickle cell disease, and Shawnita is one
that is looking at joining one of these trials.
Another approach in research of our group has been in hemo-
globin switching, one in which researchers look for drugs that will
promote the bone marrow to increase production of fetal hemo-
globin instead of sickle in patients with sickle cell disease. This
prevents sickling.
Well, the studv of hydroxyurea originated in Boston by sickle cell
researchers, and this drug does indeed promote the bone marrow
to produce cells with a greater amount of fetal hemoglobin and, in
20
selected patients who have been put on clinical trial, it decreases
the tendency to sickle and ameliorates their disease. It actually di-
minishes the frequency and the intensity of the crisis.
There are further trials that are now taking place in a
multicenter fashion.
Another drug that also promotes the increase of fetal hemoglobin
is butyrate. Dr. Perrine, a researcher at the California center, has
now transferred to our center and has been very important in the
investigation of this drug, and clinical trials are now in progress
with this drug in sickle cell disease and thalassemia.
Sickle cell disease is a disease that affects persons from birth as
long as they will live. We are increasingly happy to see how much
longer patients are living as a result of better medical care and
translating research results into clinical care.
So as a result of the penicillin prophylaxis study which Dr.
Lenfant mentioned, this was a landmark, multicenter study which
demonstrated that administering penicillin twice a day to children
prevents the mortality, which was as h;gh as 30 percent, in the
first 3 years of life and decreased the morbidity of infection. As a
consequence of that, newborn screening has been instituted in a
majority of the States in this country, so that our center was in-
strumental in establishing newborn screening in Massachusetts.
Babies, as soon as they are identified, are placed on penicillin,
which will prevent that tremendous human loss which we saw be-
fore this study was published. In addition, in Massachusetts, in
conjunction with the department of public health, we have ar-
ranged so that any family that does not have insurance or a way
of buying penicillin will receive free penicillin for the first 7 years
of life.
The Boston Center serves also as a resource to the New England
area. When Rhode Island initiated newborn screening, it was the
Boston Center that did newborn screening for Rhode Island until
the New England Regional Newborn Screening Program took over
this task. We are also a resource to New England States for test-
ing, counseling and consultation.
In Rhode Island, we helped establish a sickle cell clinic in
Women and Infants Hospital, and we continue to do testing for
Maine, New Hampshire and Vermont. Outside the United States,
we have served as consultation for the establishment of two clinics
in England, and in Brazil, we continue to share our experience in
basic and clinical research and educational materials, which I have
helped to translate into Portuguese, in their efforts to establish
screening and start sickle cell clinics.
The physicians of the Boston Sickle Cell Center are responsible
for over 400 patients in the greater Boston area and serve as con-
sultants for the entire State, and again, for the region. Our popu-
lation is mostly African American, African, Haitian, Cape Verdean.
Puerto Rican, and other countries from the Caribbean, Central
America and South America. To better serve this population, we
try to hire Spanish-speaking, Haitian-Creole-speaking, and myself,
as both Puerto Rican and Portuguese, also help to address many
of our patients needs and anxieties in their own language.
Recently, as you are well aware, we have a very significant num-
ber of refugees from Somalia, wnich are increasing in numbers,
21
whom we are now testing and will be counseling and enrolling any
patient that we discover in comprehensive care.
Finally, because the ultimate goal of the combined efforts of all
researchers in the 10 national centers is to find a cure, we are tre-
mendously pleased to know that the National Institutes of Health
will be funaing three projects in gene therapy this year. We, along
with other centers, are also participating in bone marrow trans-
plantation studies for young patients with sickle cell disease.
There is so much more that I would like to tell you about my cen-
ter, but I know there are other colleagues here who are equally
eager to tell you about the accomplishments in their centers. Again,
I thank you for inviting me here to tell you about this.
The Chairman. Thank you very much.
[The prepared statement of Dr. McMahon follows:]
Prepared Statement of Lillian Caldeira McMahon, M.D.
Dear Senator Kennedy, thank you for inviting me to testify on the Senate Com-
mittee on Labor and Human Resources hearing on Sickle Cell Research next Thurs-
day^ July 28, 1994.
The Boston Comprehensive Sickle Cell Center is one of ten national comprehen-
sive centers funded through the Sickle Cell Branch of the National Heart, Lung and
Blood Institute. Each of the ten Sickle Cell Centers operate within the academic en-
vironment of a university and medical center which allows tar a productive collabo-
ration between researchers and clinicians and for continuity of care of patients.
The Boston Sickle Cell Center is especially fortunate to have three medical
schools, Tufts, Harvard, and Boston University, affiliated with our program. Our six
research projects in red cell membrane, endothelial adhesion and hemoglobin
switching are conducted in six university-affiliated hospitals and a graduate school,
Northeastern University. This academic and clinical environment brings together a
community of scholars, researchers, clinicians and other health professionals, which
are vital to progress of sickle cell research and treatment of patients with sickle cell
disease.
It is almost an impossibility to report to your committee the accomplishments of
the Boston Sickle Cell Center in only five minutes allowed for this presentation.
However I will attempt to highlight some of our achievements.
Investigators in the Boston Sickle Cell Center have focused on understanding how
alterations in the red cell membrane contribute to the pathogenesis of sickle cell dis-
ease. Such knowledge could lead to the development of therapeutic strategies that
prevent the dehydration that is such a critical determinant in the formation of
intracellular sickle hemoglobin polymers. Even a modest reduction of the hemo-
f;lobin concentration inside the red cell could have a marked effect in inhibiting sick-
ing.
Dr. Carlo Brugnara and co-investigators in the Boston Sickle Cell Center have de-
veloped a novel approach based on these principles. They began with the premise
that the calcium dependent Gardos channel inside the red cell assumes a primary
role in the loss of potassium and cell dehydration that accompanies sickling. They
have demonstrated that, in vitro, inhibitors of the Gardos channel could block water
loss from sickle red cells. One of these inhibitors, clotrimazole, a commonly used
antifungal agent, was fully effective on sickle red cells at concentrations lower than
that used in patients routinely treated with the drug. They then tested this drug
in a transgenic mouse model of sickle cell disease and demonstrated the efficacy of
the drug in vivo in preventing the dehydration of sickle red cells. On the basis of
these exciting and promising findings, these investigators, in collaboration with an-
other Boston Center investigator, Dr. Orah Piatt, are embarking on a phase I clini-
cal trial in patients with sickle cell disease.
Also in red cell membrane research Dr. Jiri Palek and collaborators discovered
sites in the red cell where polymers of hemoglobin penetrate disrupting the mem-
brane, causing breakage and cell damage, which in turn leads to stickiness of the
cell inside the blood vessels and obstruction of blood flow all this resulting in pain
to the patient and organ damage. This important work was published in the journal
Science.
Research using the drug hydroxyurea originated in Boston by sickle cell research-
ers. Hydroxyurea promotes the bone marrow to produce greater quantity of red cells
containing fetal hemoglobin and decreases the tendency of the. red blood cells to
22
sickle. The use of hydroxyurea in selected patients with sickle cell disease resulted
in diminishing the frequency and intensity of their sickle cell crises. Further clinical
trials continue in Boston and other Centers with careful monitoring of patients by
sickle cell researchers and clinicians.
With the transfer of Dr. Perrine and her research project from the California Sick-
le Cell Center to our Center in Boston we are continuing the investigation of other
drugs, such as butyrate, which also promotes the bone marrow to switch from pro-
ducing sickle hemoglobin to producing more fetal hemoglobin. Clinical trials are now
in progress with patients with sickle cell disease and with thalasseaia.
The Boston Center participated in the landmark multicenter study of penicillin
prophylaxis in children with sickle cell anemia which demonstrated that administer-
ing penicillin twice daily to children with thin disease prevents the mortality which
was as high as 30Q in the first 3 years of life and diminishes the morbidity caused
by certain bacterial infections in children.
As a result of the findings of this study screening for sickle cell disease in
newborns was instituted in most states in the U.S. As soon as babies are identified
as having sickle cell disease they are started on penicillin prophylaxis, preventing
the tremendous human loss we experienced before this study. The Boston Center
was responsible for establishing universal newborn screening for sickle cell disease
and other hemoglobinopathies in Massachusetts in 1988 and performed sickle cell
screening of newborns for the state of Rhode Island as part of a Special Program
of National and Regional Significance grant. We now serve as consultants to the
Now England Regional Newborn Screening Program and are responsible for the
medical care of babies identified through this program in Massachusetts. Once iden-
tified, newborns with Sickle call disease are started on penicillin prophylaxis and
enrolled in a comprehensive medical care program. We continue to screen for the
states of Vermont, Maine, and New Hampshire.
We serve as a resource for the New England states for testing, education and
counseling, and for consultation concerning sickle cell patients. The Boston Sickle
Cell Center participated in the establishment of the Sickle Cell Clinic in Women
and Infants' Hospital in Rhode Island. Outside the country we assisted with con-
sultation in the creation of two programs for screening and clinical management of
patients with sickle cell disease in England, and share our experience in basic and
clinical research, as well as educational materials with our colleagues in Brazil in
their efforts to establish screening programs and start sickle cell clinics.
Boston Sickle Cell Center physicians deliver comprehensive medical care to over
400 patients with sickle cell disease. Our population is diverse and comprised of Af-
rican Americans, Africans, Cape Verdeans, Haitians, Puerto Ricans and persons
from other Caribbean, Central and South American countries. To best meet the
needs of our patients the Boston Center has staff who speak Spanish and Haitian
Creole. Myself, as being both Puerto Rican and Portuguese, am able to address
many of our patients in their native language, which facilitates communication and
helps ease patients' anxieties. There is presently a significant and increasing num-
ber of refugees from Somalia in' Massachusetts which the Boston Center will test
and counsel concerning sickle cell disease, and enroll in comprehensive medical care.
There is so much more that I would like to relate to you and your committee but
I understand that my colleagues from two other Sickle Cell Centers are equally ex-
cited and eager to report the accomplishments of their Centers.
Finally, because the ultimate goal of our combined endeavors is to find a cure,
we are tremendously pleased to hear that the Sickle Cell Branch will fund 3 projects
in gene therapy this year.
Again I thank you for this opportunity to report some of the achievements of the
Boston Sickle Cell Center to your Committee.
The Chairman. Dr. Turner.
Dr. Turner. Thank you, Senator Kennedy, for the opportunity to
speak before you and your committee this afternoon.
We have given you some written information, and I would like
to focus my comments around three of the slides that I have pre-
pared for you to try to give you an overview about our center. This
also, I think, gives a better overview of all the centers in the Unit-
ed States that are currently funded by the NIH. This slide is the
last one in my packet that gives you an overview of the program
components that all of the centers currently have.
We are all involved in research activities, which includes both
basic and clinical activities. We all serve as resources for our sur-
23
rounding communities and catchment areas. I am at Meharry Med-
ical College in Nashville, TN, and I serve the 21 counties as my
catchment area of Tennessee, and in addition to that, I serve as the
consultant for the State of Kentucky, especially southern Kentucky,
where we are responsible for that patient population.
In addition, all the centers have a major role in making sure that
information is disseminated to the lay public and to our own col-
leagues, who many times are not aware of new developments that
may happen in our centers and other research activities.
We have major psychological support in our institution in terms
of research activities. We have a joint partnership with Vanderbilt
University, where many of our psychological projects are conducted
with their staff at Peabody Institution, which is a major institution
that has been involved in research and psychological activities in
the United States for many, many years.
We are involved in counseling, and we also have the State con-
tract for making sure that individuals who are diagnosed with sick-
le cell trait in the State of Tennessee get information that is appro-
priate for the type of hemoglobin variant that they have.
We have not talkea about yet in this hearing that there are over
700 known hemoglobins right now in the United States that are
documented, recognized, and known. Thus, there is a wealth of in-
formation that needs to be translated to a variety of individuals.
As was mentioned earlier, we have State newborn screening pro-
grams, and on a daily basis in our laboratory — I am the reference
laboratory for the State of Tennessee — we are always getting this
"alphabet soup," as I call it, and I am always getting calls to help
someone understand what hemoglobin-D or J or H, or hemoglobin-
Bart represents.
So we have a tremendous responsibility to make sure that the in-
formation that is there is translated to other individuals who may
not be as familiar with this "alphabet soup," that I call the 700
types of variants that may be available.
What I would like to do in the next few minutes is talk about
some of the accomplishments that have occurred at our center. For
the past 20 years, Meharry Medical College has been involved in
sickle cell disease research. We have come a long way. We started
out as an infant, and I think we are now in the teenage stage of
our activities at Meharry Medical College in the sense that we
have taken our time to develop a center. There needs to be a criti-
cal mass that is there to carry on these kinds of activities, these
very sophisticated and time-consuming activities that occur in the
center itself. So we have a critical mass that has allowed us to de-
velop basic research.
For example, many of us in this room know we now believe that
the reason for the sickle cell gene mutation was protection against
malaria. And we have been working with one of the membrane re-
ceptors, glycophorin, over the past 10 years, to help solidify that
concept and also to look at how it can help us further develop strat-
egies and treatment plans for individuals who have sickle cell dis-
ease.
We have also been involved in looking at mechanisms and ma-
nipulating systems that would help us to ultimately get closer to
gene therapy — looking at the viruses that may possibly serve as
24
vectors that we could use to help us with a cure for sickle cell dis-
ease.
As I have already mentioned, we have a laboratory that is the
State reference laboratory. We are a recipient and participate in
the cap certification. In factx- we just recently sent out hemoglobin-
Zurich as one of the unknowns to all the State laboratories; that
came out of our laboratory, where we just cloned and sequenced the
gene that was responsible for that hemoglobin-Zurich. And this is
a very interesting hemoglobin that is not predominantly found in
African Americans, but is found in individuals from Middle Europe,
and individuals who may get exposed to certain kinds of oxidants,
specifically sulphur drugs, may have occasion to become severely
ill. So the family that we described is not an African American
family; it is a Caucasian family from middle Kentucky, where we
have now done pedigree studies and are looking at the extraction
of this family back to Central Europe. So we have done some very
interesting work in that area.
We are involved not only in the United States — as Dr. McMahon
has said, she is involved in Brazil — we are involved in Peru. We
have an ongoing relationship and have some AID grants in Peru.
We have also translated with my help into Spanish information
that is now being used in Peru from our center. So we are also in-
volved on the international scene and have been involved with a
number of other centers and countries in sickle cell disease.
I would really like for us to think of this as a global issue in
many ways. As I mentioned, there are 700 different types of hemo-
globins. There are many people who are now getting involved in
this arena. And as the world gets smaller, we are starting in our
area to see other kinds of hemoglobins that we did not have infor-
mation on. For example, hemoglobin-E is now one of the most fre-
quently diagnosed hemoglobins in the United States. This is a he-
moglobin that has its predominance in Southeast Asia, and in mid-
dle Tennessee, we have a fairly large Southeast Asian population —
we have Cambodians and Vietnamese — and in our center, we have
an individual who speaks all of these languages. There is a rural
and cultural difference, and you have to be culturally sensitive to
talk about taking blood from those individuals. As you may or may
not know, individuals from Southeast Asia view blood differently
than we do. So we have a person in our center who is aware of
that, so when we talk about taking blood, we can use the right eth-
nic-sensitive approach in helping individuals from Southeast Asia.
So we are involved in a variety of things that I think have ex-
panded our horizons, and those systems that we have developed
have allowed other centers to use them, and we are in very close
collaboration with many of those centers.
So the support that we have received has been very important
for us to expand our horizons, but that did not occur overnight. It
took us a while to get to that point, and we are looking forward
to continuing to be able to do that with adequate support. I think
that is one of the issues that we need to be very careful about in
terms of recognizing that there is about $60 million to $70 million
coming of the NIH, but hat is not adequate to do the kinds of
things that we need to do.
25
And Dr. Lenfant did not get into it, but I will say it — the moneys
that have been coming out have not been adequate. They have
been flat. For the last years, we have taken a hit. I get appro-
priated a line item from NIH in terms of my grant, my P-60, and
yet I have had to take from 5 to 8 to 10 percent each year because
of the lack of resources that the NIH has.
So I think one of the things that I would like to see come out
of this is a recognition that there is ongoing quality, adequate re-
search, but despite our efforts, we are handicapped because many
times, we have to put it on the back burner, or we are not able to
follow up leads we have in terms of looking at those leads and
helping us move toward curing this disease, because of the shortfall
that is occurring in Congress.
I know I am not saying anything that you do not know, but what
we really need is more support, in a variety of ways, to accomplish
some things that many of us have ideas about but have inadequate
resources for. Out State institutions many times are not available
to provide adequate resources. So it would be very nice if one of the
things that happens is a movement to put more money in the pipe-
line to help us solve some of the problems that many of us have
on our burners.
I will stop here and let my other colleagues have the opportunity
to speak.
Tnank you.
[The prepared statement of Dr. Turner appears at the end of the
hearing record.]
The Chairman. As I said, I am managing the reauthorization of
the Elementary and Secondary Education Act on the floor, which
deals with disadvantaged students, and I am going to have to go
back over to the floor. But I would be interested if the researchers
could give us some insight as to the similarities, if any, in terms
of sickle cell, Tay-Sachs and Cooley's diseases. Are they basically
variations on a theme, or are they pretty disparate?
Dr. Turner. The biochemical differences are totally different.
But when we talk about a chronic illness, whether we are talking
about Tay-Sachs or sickle cell disease or hemophilia, we have some
common elements that are present. A chronic illness has some
things that need to be dealt with. There is the psychological im-
pact. There needs to be research put into those specific diseases.
So there are similarities in that respect. But in terms of the bio-
chemistry, the physiology, there are some differences.
Did I understand your
The Chairman. Yes, that is helpful.
Dr. Moore. Both diseases, sickle cell and Tay-Sachs, are genetic
in nature. Both seem to afflict different ethnic groups, have a pre-
dominance of influence on different ethnic groups. The incidence of
sickle cell disease appears to be much higher in the African Amer-
ican community than Tay-Sachs is in the Jewish community.
The Chairman. And wouldn't Cooley's be basically ethnic as
well?
Dr. Turner. If you are referring to Cooley's anemia, we are talk-
ing about the homozygous condition, and the predominance of that
disease would be in Italian descent. But we have beta-thalassemia,
and we can have the combination beta-thalassemia trait in the Af-
82-553 0-94-3
26
rican American population. So we have the S-beta-thalassemia syn-
dromes that we have to address also, which can be just as severe
in many instances as the homozygous SS condition in individuals
with sickle cell disease.
Dr. McMahon. Putting it very basically, on thalassemia and
sickle cell, in sickle cell, hemoglobin that is produced is abnormal,
and in thalassemia, there is not sufficient hemoglobin being pro-
duced, although biochemically, it is normal.
The Chairman. Thank you.
Just before calling on Dr. Moore, Shawnita, if I could, I would
just like to ask you a couple of questions. As far as your greatest
challenge in coping with the disease, what is the most difficult
thing about coping with the disease itself for you?
Ms. agnew. The pain, really.
The Chairman. The continuing pain.
Ms. agnew. Yes.
The Chairman. And that iust seems to go on and on. Has the
Boston City Hospital Center been really helpful to you?
Ms. agnew. Yes, a lot.
The Chairman. And do you remember which programs there
have been the most helpful?
Ms. agnew. The teen support group.
The Chairman. The support group. And have you thought about
the research a little bit and which areas you would like to see re-
searched, or just the whole thing?
Ms. AGNEW. Everything, the whole thing.
The Chairman. Everything. OK. Thank you.
The Chairman. Dr. Moore.
Dr. Moore. Thank you, Mr. Chairman.
I want to talk about Southern University, but in so doing, I want
to allow that to set the tone for what I believe are some of the con-
cerns that have been raised, especially from Senator Simon, a con-
cern that was raised about peer review, and of course, the degree
of medical school affiliation and whether that has anything to do
with an institution's capability.
I am William E. Moore, vice chancellor for academic affairs at
Southern University in Baton Rouge. I am pleased to have the op-
portunity to speak in behalf of our institution and to seek commit-
ted support for the establishment of a national sickle cell research
center on the Baton Rouge campus, a major constituent of the
Southern University system.
The four entities that constitute the Southern University system
are strategically located throughout the State of Louisiana. They
include a 2-year campus in Shreveport, a law center in Baton
Rouge, an urban commuter campus in New Orleans, and a Level-
3 doctoral institution in Baton Rouge.
The Southern University system, with almost 17,000 students, is
the only system of higher education within the historically black
college community in this country, or in the world, for that matter.
Dr. Delores Spikes, who is here today, is president of the South-
ern University system, and Dr. Marvin Yates, who is also here, is
chairman of the Baton Rouge campus.
I am also joined by Dr. Jonathan Roberts, who is the director of
Charity Hospitals of Louisiana. Before assuming that position, Dr.
27
Roberts served as coordinator of planning of our national sickle cell
disease center.
Our appearance here today provides a mechanism for us to share
with you, Senator Kennedy, what we believe to be indisputable doc-
umentation of the need for the proposed center and why it should
be at Southern University in Baton Rouge — for indeed, despite our
best efforts, some of the noteworthy accomplishments of our insti-
tution remain well-kept secrets, and we sense the tone of that
today, and I hope in your allowing me to tell you a little bit about
some of them, we will also address the issue of peer review, docu-
mentation, and of course, budgetary needs.
I should say that the warm hospitality we have already received
has enabled me to dispel the belief that the atmosphere near the
Potomac or inside the beltway would be intimidating at best. We
thank you for this reception.
By way of experience, I have spent my entire professional career
as a university professor and administrator having had the privi-
lege of full-time teaching appointments at four historically black in-
stitutions. I have also served as adjunct professor at four majority
institutions, the latter representing some of the finer examples of
tradition, elitism, research productivity, and academic standards. I
have been a visiting lecturer in Italy, Belgium and France, the
highlight being an invited lecturer at the Pasteur Institute in Paris
in 1978.
I have served as chairman of the general research support review
committee of the National Institutes of Health, and I am currently
serving a second 3-year term on the board of review of the National
League of Nursing.
I mention these affiliations not in the interest of self-indulgence,
but because I believe these experiences have given me an oppor-
tunity to observe and participate in a spectrum of scientific-related
activities, many of which have relevance to your committee, and
some of which I will refer to today in regard to sickle cell research
and the appropriateness at our university.
Although I am a part of the Southern University system, I will
confine my remaining comments to the Baton Rouge campus, the
oldest and largest unit within the system. Southern University at
Baton Rouge is an 1890 land grant institution, with enrollment on
the Baton Rouge campus in excess of 10,000 students, the history
of the university is punctuated with the achievements of its grad-
uates.
For example, the university claims among its graduates eight
generals in the United States Army; a significant percentage of the
minority lawyers in this country — and I should say that in less
than 10 years, our nursing program has achieved the status of
being one of the leading undergraduate programs in the United
States. Our graduates consistently score above 95 percent on board
examinations, and our recent self-study and its initial accreditation
was selected by the National League of Nursing as a model for
other institutions across this country to use as a guide.
Southern University has a long history, Mr. Chairman, and this
has some implications of peer review, as one of the leading HBCUs
to produce graduates who have earned the Ph.D. in political
28
science. In fact, we rank at the top of producing black political sci-
entists at the Ph.D. level.
What is little-known, however — and I point this out by under-
scoring it— is that between 1985 and 1989, 10 percent of all of the
blacks in this country who received a Ph.D. in physics were South-
ern University graduates. This is indeed a major accomplishment
given the large number of colleges and universities in this country.
Further, Southern University provides a wholesome intellectual
and cultural environment for the development of leaders among its
graduates. Three former Southern University student government
presidents now serve in the Louisiana legislature or have served
there, and as I speak to you today, I should remind this distin-
guished committee that two of those graduates, William Jefferson
and Cleo Fields, are the only African Americans represented in the
State of Louisiana in the United States Congress. And a third,
State Senator Charles Jones, is the architect of the sickle cell ane-
mia bill in Louisiana, and is also present today.
My point in mentioning these few examples is to underscore the
fact that our general university environment is geared to support
and to sustain excellence in teaching, research, and service.
I will not read to you the details of the background of the need
for tie sickle cell center, because you have heard about that from
several of the scientists and persons who have testified before you
today. I would like to point out, though, that the center we are pro-
posing will include a section which will focus on physical-chemical
studies, principally on in vitro studies of hemoglobin. We will draw
on the faculty in the departments of physics, chemistry, and biology
to investigate the effects of physical stress and chemical agents on
the solution properties of hemoglobin.
We will also have a division which will focus on anti-sickhng
agents, which are still important despite the progress that is being
made. This division will be devoted to the isolation, characteriza-
tion, and synthesis of anti-sickling agents, substances which enable
patients to lead a normal life by decreasing the chances of blood
cells to become sickled.
We will have a clinical division — and this is extremely important,
because we have heard questions raised about the need to be asso-
ciated with a hospital. We will devote primary attention to longitu-
dinal studies of patients who have sickle cell disease, and these in-
vestigations will be carried out in conjunction with the Earl K.
Long Hospital and with other clinical settings. The physicians pro-
viding immediate supervision for these studies will be hospital phy-
sicians who will hold adjunct appointments in biomedical sciences
at Southern University.
I have other information about the clinical studies in the written
text.
We will have a division of molecular biology and genetic engi-
neering, where we hope to do gene therapy research. We will also
have a laboratory on ultrastructural studies, in which we will en-
gage in electron microscopy as a major tool for investigating in
vitro and in vivo sickling.
Finally, there will be a laboratory on behavioral and epidemiolog-
ical studies. This division of the sickle cell center will have the role
of investigating disease patterns similar to what you have already
29
raised. Senator Kennedy, with a view toward using the knowledge
gained therefrom for education and prevention.
The Chairman. Dr. Moore. I am going to have to interrupt you
here. We have present Dr. Onene-Frempong, who will be testifying
as well. I have keen called over to the floor Dy the Majority Leader,
and Senator Wellstone will be here momentarily to chair.
Let me just ask you this. There is no question that the university
has been an extraordinary educational center. I suppose the ques-
tion is the reason why it is necessary to have a new center, or
whether the scarce resources should not be further targeted on the
existing research centers which have been working in these areas.
How would you respond to that?
Dr. Moore. I will try to answer that, Senator, by giving four bul-
lets, which was going to be my concluding statement, if I may, be-
cause I think that shows you where we are different from
The Chairman. If you can do it; I must leave in a couple of min-
utes because I am required to be on the floor of the Senate. We
could make it part of the record, and if you want to summarize in
a couple of minutes, I would be glad to hear you. If you need a
longer period of time, we can put it in the record.
Dr. Moore. Sure. It will not take 2 minutes to do it.
The Chairman. Fine. Go ahead.
Dr. Moore. First of all, the State of Louisiana has already com-
mitted substantial financial support to this effort. The proposal has
received the categorical endorsement of both State and Congres-
sional Black Caucuses.
We have a peer review at the Board of Regents in Louisiana, and
that board relies heavily on outside scientists to evaluate what we
have done.
We have the full endorsement of the hospital systems of Louisi-
ana, the two medical schools, Tulane University and Louisiana
State University, and as I said, we have the director of Charity
Hospital, who is providing support.
So when you look at the track record combined with the support
that has emanated from within our State, we believe that it is un-
matched in any State in this Nation, and for that reason, we are
seeking your support for the momentum that we have already gen-
erated to be carried out.
The Chairman. Thank you. I saw some references to those points
in the testimony, which I will study further at another time. We
appreciate very much your presence nere.
[The prepared statement of Dr. Moore follows:]
Prepared Statement of William E. Moore
Mr. Chairman and members of this committee, I am William E. Moore , Vice
Chancellor for Academic Affairs at Southern University in Baton Rouge. I am
pleased to have the opportunity to speak in behalf of our institution and to seek
the committee's support for the establishment of a National Sickle Cell Disease Re-
search Center on the Baton Rouge Campus a major constituent of the Southern Uni-
versity System. The four entities which constitute the Southern University System
are strategically located throughout the state of Louisiana. They include a two-year
campus in Shreveport, a Law Center in Baton Rouge, an urban commuter campus
in New Orleans, and a Level III Doctoral institution in Baton Rouge. The Southern
University System, with almost 17,000 students, is the only system of higher edu-
cation within the Historically Black College and University (HBCU) Community.
Dr. Dolores Spikes is President of the Southern University System and Dr.
Marvin Yates is Chancellor of the Baton Rouge campus. I am joined today by Chan-
30
cellor Yates and by Dr. Jonathan Roberts, Director of Charity Hospitals of Louisi-
ana. Before assuming his present position Dr. Roberts served as coordinator of plan-
ning for the National Sickle Cell Disease Center.
Our appearance here today provides a mechanism for us to share with you what
we believe to be indisputable documentation for the need for this proposed center
and why it should be at Southern University at Baton Rouge. For indeed, despite
our best efforts, some of the noteworthy accomplishments of our university have re-
mained well kept secrets, and it is through a hearing such as this that we are able
to tell you why this modest investment would serve the Nation well in scientific re-
search and improving human health.
The warm hospitality we have already received has enabled me to dispel the belief
that the atmosphere near the Potomac or inside the Beltway would be intimidating
at best. We thank you for this reception.
By way of experience, I have spent my entire professional career as a university
professor and administrator, having had the privilege of holding full-time teaching
or administrative appointments at four historically black institutions. I have also
served as visiting or adjunct professor at four other majority institutions — the latter
representing some of the finer examples of tradition, elitism, research productivity,
and academic standards. I have been a visiting lecturer in Italy, Belgium, and
France— the highlight being an invited lecturer to The Pasteur Institute in Paris in
1978. I have served as chairman of the General Research Support Review Commit-
tee of the National Institutes of Health and I am current serving a second three-
year term an the Board of Review of the National League of Nursing. I have written
for The New York Times, Saturday Review, The Houston Chronicle and Change
Magazine. In the case of Change, I was one of four essayists selected in 1977 to pub-
lish articles on how the educational needs of Blacks are being met.
I mention these things, not in the interest of self indulgence, but because I believe
these experiences have given me the opportunity to observe and participate in a
spectrum of scientific and health related programs, many of which have relevance
to the concerns of your committee, and some to which I will refer today in regard
to Sickle Cell Disease research and its appropriateness at our institution.
Although I am a part of the Southern University system I will confine most of
my comments to the Baton Rouge campus, the largest and oldest unit within the
system. Southern University Baton Rouge is an 1890 Land Grant institution with
an enrollment in excess of 10,000 students. The history of Southern University is
punctuated with achievement of its graduates. For example, the university claims
among its graduates eight generals in the United States Army which is more than
25 percent of all generals produced by 1890 Land Grant institutions. The prominent
role the University has played in graduating a significant percentage of minority
lawyers is no doubt well known to this committee. In less than ten years, we have
produced one of the leading undergraduate nursing Programs In the United States.
Our graduates consistently score above the 95 percent level on national Board ex-
aminations, and when our program achieved its initial accreditation, our self-study
was selected by the National League of Nursing as a model for others schools across
the nation to use as a guide.
Southern University also has a long and distinguished history as one of the lead-
ing HBCU's to produce graduates who have earned Ph.D. degrees in political
science, chemistry, and biological sciences. In fact the University has traditionally
led all schools in producing black political scientists and ranks among the top four
HBCU's in producing graduates who have obtained the Ph. D. in chemistry. What
is little known is that during the period of 1985 through 1989, 10 percent of all
blacks in the United States who received the Ph. D. In physics were Southern Uni-
versity graduates. This is indeed a major accomplishment, given the large number
of colleges and universities in this country. Further, Southern University provides
a wholesome intellectual and cultural environment for the development of leaders
among its graduates. In 1990, three former student government presidents from
Southern University served in the Louisiana Legislature, and today as I speak to
you, I should remind this distinguished committee that two of those graduates (Rep-
resentatives William Jefferson and Cleo Fields) are the only African Americans rep-
resenting the State of Louisiana in the the United States Congress, and a third
former SGA president (the Honorable State Senator Charles Jones) is the architect
of the Bill to establish a Sickle Cell Disease Center at Southern university. My point
for mentioning these few examples is to underscore the fact that our general univer-
sity environment is geared to support and sustain excellence in teaching, research
and service.
31
Background and Focus of The Center
Sickle Cell Disease is a devastating, multifaceted blood disorder which has a high
incidence in African Americans. This hereditary disease can only be cured by a com-
plete bone marrow transplant or by some yet to be discovered method of DNA ma-
nipulation. The former approach has drawbacks in the difficulty of the procedure,
the problem of finding suitable marrow for transplantation, and the inability of the
patient receiving a transplant to produce non-sickle cell offspring. The genetic solu-
tion to the sickle cell problem must await further understanding of the human
gnome and the development of DNA procedures which can shut down the synthesis
of sickled hemoglobin and activate the manufacture of normal adult hemoglobin.
Because sickled blood cells do not bind nor transport oxygen effectively, the symp-
toms of the disease are numerous. These may include stroke, ulcers, heart attacks
and a variety of internal disorders. Until very recently most patients died before the
age of 25 and those who managed to survive experienced intense suffering. Hence
during the past 20 years much of the research has focused on developing methods
to alleviate pain, to minimize crises, and to enable patients to function without
much discomfort or suffering. Success in this area has come through the synthesis
and isolation of a variety of antisickling agents — drugs which when taken properly
will cause the blood cells to retain a normal shape and will minimize the likelihood
of a patient experiencing a crisis.
Southern University at Baton Rouge proposes to establish a comprehensive sickle
cell research center — one which will offer a unique opportunity for clinical research
affiliation with Earl K. Long Hospital and other medical centers. The Southern Uni-
versity facility will focus on five or six major research thrusts which will include
the following:
Physicochemical Studies
This facet of the program will focus principally on in vitro studies of hemoglobins.
Faculty members in the departments of physics, chemistry and biology will inves-
tigate the effects of physical stress and chemical agents on the solution properties
of hemoglobins. This program will provide further knowledge regarding conditions
under which sickling is most likely to occur. Such information will have national
significance and will prove particularly beneficial to other investigators in the
Southern University Center,
Anti-Sickling Agents
This division will be devoted to the isolation, characterization, and synthesis of
antisickling agents, substances which enable patients to lead a normal life by de-
creasing the chances for blood cells to become deformed.
Clinical Studies Division
In this section of the proposed center, We will devote primary attention to longitu-
dinal studies of patients who have sickle cell disease. These investigations will be
carried out in conjunction with the Earl K. Long Hospital and other clinical settings.
The physicians providing immediate supervision for these studies will be hospital
Ehysicians who will hold adjunct appointments in biomedical sciences at Southern
Fniversity. It is expected that some members of the nursing faculty at Southern
University will be involved in this program.
The clinical studies unit will have a strong interdisciplinary focus which will bring
together chemists, biologists, biophysicist, nurses and physicians. Results from from
the basic disciplines wul be tested within the boundaries of FDA guidelines and
other provisions which protect human subjects.
Division of Molecular Biology and Genetic Engineering
This phase of the program will focus on various genetic approaches to investigat-
ing sickle cell disease. In this regard we will devote particular attention to DNA ma-
nipulation and various techniques of modern biology which could cause a patient to
begin synthesizing normal adult hemoglobin.
Laboratory of Ultrastructural Studies
This section of the center will use electron microscopy as a major tool for inves-
tigating in vitro and in vivo sickling. Although many research studies will originate
in thislaboratory, this facility will provide an important service function to several
other parts of the center. This use of the electron microscope will provide an impor-
32
tant bridge between the basic physicochemical and biological studies and hospital
related clinical investigations.
Laboratory of Behavioral and Epidemiological Studies
This division of the Sickle Center will have the role of investigating disease pat-
terns, with a view toward using the knowledge gained therefrom for education and
prevention. This facet of the program will also investigate psychological and social
manifestations of sickle cell disease. In this regard, the new knowledge produced
will contribute to improved counseling approaches and better behavioral methods for
managing the disease.
Summary
By way of summary I wish to call the committee's attention to two evaluative
statements recently made about our institution. In the first case we were recently
evaluated by two different panels of the National Institutes of Health for the estab-
lishment of a center of Cellular and Molecular Biology at Southern University. At
both levels of review the panels rated our scientific leadership and administrative
capability as outstanding. For this review, we achieved a priority score of the high-
est order.
In the second instance, our university was invited to be featured in the fall 1994
Education, a 118 year old scholarly journal of which I have the privilege to be guest
editor. We were recently informed by the publisher that the Southern University
issue promises to be the best ever produced. And this is in the face of some of the
most prestigious universities and educational agencies in the country having been
featured in previous issues. Mr. Chairman, That is the standard of quality we con-
tinuously set at Southern University and that same spirit of competence and
achievement would carry over into the sickle cell disease center.
I wish to further summarize further by reminding the committee of the following
elements of uniqueness to the Southern University proposal:
The state of Louisiana has already committed substantial financial support to this
effort.
The proposal has received the categorical endorsement of both the state and con-
gressional black caucuses.
The university's track record in research and academic excellence are well docu-
mented.
Our outreach activities are well established. We have one of the more comprehen-
sive wellness programs you will find anywhere, and we are a national leader in
service learning at public institutions in the United States.
The uniqueness of the Southern University System makes it an appropriate set-
ting for the sickle cell disease research center.
Southern University is prepared to establish a first rate National Sickle Cell Dis-
ease Research Center. The disease itself is one that afflicts African Americans, and
it is fitting that a university which is predominantly African American has the vi-
sion, track record, technical capability and willingness to provide leadership in this
area. Through the collective efforts of the Health Research Center, the School of
Nursing, the Center for Social Research, and Center for Rehabilitation Counseling,
the University stands poised to make this Center become a reality— one for which
the Nation will be proud.
We appreciate this opportunity and would be pleased to answer any questions the
committee might have.
Southern University and AaM College
INSnTUnUONAL setting and research capability
During the past thirty years, Southern University has achieved an exemplary
record in science teaching and research — two activities which compliment one an-
other in the preparation of well qualified graduates. The following examples are rep-
resentative accomplishments in the college of Sciences and health related areas.
Student Achevements
-From 1967 through 1972 an average of three chemistry graduates per year ob-
tained the Ph. D. degree in chemistry.
From 1972 to 1985 the University was one of the leading producers of minority
undergraduates to obtain M. D. degrees.
33
From 1985 to 1989 the university produced 10 percent of all African Americans
in the United States who went on to obtain the Ph. D. degree in physics.
Although the School of Nursing is a relatively new academic unit it has gained
a nation reputation for the quality of its graduates as judged by performance on
board examinations and overall program quality revealed through the accreditation
process.
Success in graduate and professional school is matched by the accomplishments
of other science graduates who have risen to prominent positions in government and
the corporate world.
Faculty Accomplishments
In addition to a noteworthy record of general scholarship, the Southern University
Science faculty has been recognized for its accomplishments in the following ways:
In 1992-93, Dr. Fitzgerald Spencer of the Biology department was appointed a
Fulbright lecturer.
Dr. Gary Ross, a long-time biology faculty member who recently retired was recog-
nized as a world authority on butterflies.
During the past ten years two Southern University faculty member have ap-
Rointed to MH Study sections at the National Institutes of Health. Dr. William
loore chaired the General Research Support Review committee in 1982 and Dr.
Earl Doomes is presently in the second year of a four year term.
Faculty in the computer science department have developed software that is used
in major universities in the United States.
Dr. William Moore was invited lecturer at the Pasteur Institute in Paris, France
in 1978. His pioneering work on plasma albumins is closely related to a variety of
physicochemical studies of sickle cell hemoglobin.
In 1991 Dr. William Moore was elected to the Board of Review of the National
League of Nursing. He is one of two public members serving on the Board which
accredits all nursing programs in the United States.
In the applied sciences, Dr. James McNitt has become a leading authority on rab-
bitry and several investigators in the Center for Energy and environmental Studies
are becoming increasingly recognized for their work on pollution of the Mississippi
River.
PERSONNEL
The basic personnel in a sickle cell disease center will consist of a director, associ-
ate director, clerical staff, and faculty who will hold tenure or tenure track appoint-
ments in an appropriate department. A second level of personnel would include re-
search associates and assistants who may be employed on specific funded projects.
Some personnel will hold adjunct appointments in the biomedical sciences. This
group will consist largely of physicians (pediatricians and hematologists) affiliated
with one of the local hospitals or health care agencies.
Faculty members holding joint appointments in the sickle cell research center will
obtain these positions through released time. This mechanism will enable depart-
ment chairs to find suitable replacements to meet departmental needs.
Faculty Research investigators will be hired initially through support from exter-
nal funding. Each investigator employed will therefore come through an appropriate
departmental screening process. These persons would move into funded positions
over the next five years as such vacancies become available through anticipated re-
tirement or other forms of attrition. Hence by 1997, there should be at least one
faculty member in each section of the center.
The administrative staff and other core personnel would be supported through
special funding. However, our long-range plan would call for the raising of a five
million dollar endowment which would cover much of the basic operational costs.
Research assistants and associates would be employed mainly through special
grants. It is projected that by 1998 there would be at least one funded project in
each division of the center.
Southern University and A a M College
RESARCH PROGRAMS AND CENTERS
The University's demonstrated ability to develop and sustain research centers
over a long period of time provides the most compelling argument that Southern
University can develop and implement a successful sickle cell disease center. The
following three examples provide evidence of that success.
34
The NASA Industrial Applications Center
In 1987 Southern University was one of eleven Universities selected by the Na-
tional Aeronautics and Space Administration (NASA) to house an industrial applica-
tion center. When the agency discontinued this program, Southern University main-
tained its industrial applications initiatives through funding from the Department
of Defense and from other NASA programs. The continuation of this program with-
out State support underscores the University's commitment to industrial applica-
tions and to identifying funding sources to sustain this effort.
The Health Research Center
This center was constructed with funds form the Public Health Service in 1961
and expanded by funding from the National Institutes of Health in 1985. Although
the center has attracted limited external support since its inception, the health Re-
search Center as been an area of pivotal research activity at Southern University
since 1972. During the ensuing 20 years the University has attracted Federal sup-
gort from the National Institutes of Health, Eli Lily Foundation, and the United
tates Department of Agriculture. Only nominal state support has been required for
two staff positions and modest supplies and travel.
During its twenty-year history of substantial funding, the Health Research Center
has provided salaries, assistantships, equipment for teaching and research, and in-
direct costs. Since many of the faculty have done research on released time, the cen-
ter, through grant funding, has provided a mechanism for the university to look at
a long list of temporary faculty and determine which ones were suitable for proba-
tionary appointments.
On numerous occasions, we have learned that the large number of graduates who
went on to medical school and graduate school gained considerable advantage by
having had positive experiences in the Health Research Center. The point to be em-
phasized is that for more than 20 years the health research center has become an
indispensable component of scholarship and student preparation without reliance on
State funding..
The Center for Energy and Environmental Studies
This is the youngest comprehensive research center at Southern University. After
its inception in 1986 it has grown to become a thriving unit with a staff of more
than forty persons including students and faculty who hold part-time appointments.
The center has been designated as one of three minority centers of excellence by
the Department of Energy. Recently it completed a meritorious policy study of the
Mississippi River Corridor and the center ranks high among top candidates from the
McKnight Foundation to conduct a more comprehensive study of the integrity of the
Mississippi River. At present the center receive grants and contracts in excess of
two million dollars. The total cost of state funding is less than $100,000.
There are other examples of relatively self sufficient research centers at Southern
University. This track record over the past two decades indicates that with suffi-
cient start-up funds the proposed sickle cell disease center, within a six year period
after completion, would have little reliance on ongoing state support.
William E. Moore is Vice Chancellor for Academic Affairs at Southern University
in Baton Rouge, a position he has held since 1989. During his twenty-seven years
of work in higher education, Dr. Moore has held a series of professorial and admin-
istrative positions at several universities. He has published, more than 35 articles
as a research chemist, science educator, and more recently in the broad area of test-
ing and education of minorities. In 1976, he served as a NATO Fellow on Computers
in Science Education — a conference held in Brussels, Belgium. In 1977, Dr. Moore
was selected by Change Magazine as one of four essayists nationally to write on the
adequacy of education of minorities. In 1978, he served as invited lecturer at the
Pasteur Institute in Paris, France. There he gave talks on his work on the chem-
istry of blood proteins, with specific reference to the artificially induced aging of
glasma albumins and in 1980 he was appointed Chairman of the General Research
upport Review Committee of the National Institutes of Health.
During the 1980's, Dr. Moore assumed several administrative positions in higher
education and continued to publish in the areas of science education, testing, envi-
ronmental science, and race relations. In 1985, as a member of the NAFEO Science
and Technology Advisory Committee, he became one of the principal contributors to
the guidelines for the Research Centers at Minority institutions, known as the
RCMI program. Since that time he has been principal investigator of two RCMI
projects. In the first instance he led a $3.76 million project aimed at studying dis-
eases which show a high incidence in ethnic minorities, and more recently he as-
35
sumed the leadership of a $4.8 million NIH project to establish a research center
in cellular and molecular biology at Southern University. At the time of this award,
Southern was one of two such non-Ph. D. programs in science to be nationally
awarded a center by NIH.
In 1991 Dr. Moore was elected to the Board of Review of the National League of
Nursing as one of two public members on this 18 member accrediting council. In
1994, he was reelected to a three-year term on this body. During this period, he also
introduced several innovative programs aimed at improving the quality of under-
graduate education for minorities. These included a wellness program, a multimedia
learning environment, a mentoring program, a precollege enrichment project, and
a service learning requirement for all undergraduates. In 1991, he was designated
as a Commissioned Research Scholar for the NABSE Moody Roundtable and that
same year was invited by the NAACP to participate in the Daisy Bates Educational
Summit at Little Rock, Arkansas. In 1994, he was invited to serve as guest editor
of Education 115 year old International journal. Recently he was informed by the
Eublisher that the forthcoming issue which will feature Southern University is the
est issue ever produced. Dr/Moore holds a B. S. degree in Chemistry from South-
ern University and was the first African American to earn a Ph. O. in Chemistry
from Purdue University. He is married to the former Willa Warren, and they are
the parents of two children, Deirdra and Marcus.
The Chairman. We will recess just very briefly, and Senator
Wellstone will be here for the completion of the hearing. I want to
thank all of you very, very much. Shawnita, we thank you. We
know you have made a special effort to be with us. You are a very
courageous and brave individual and a real inspiration, and we
thank you very, very much for your presence.
We will stand in recess.
[Recess.]
Senator Wellstone [presiding]. The committee will come to
order.
First of all, let me apologize to the panelists for the delay. We
had the service for Hugh Scott, and a good many people on the
committee are at that service; and in addition, the Elementary and
Secondary Education Act is also up on the floor, and Senator Ken-
nedy had to go to manage that. So I really apologize from all of us.
Dr. Ohene-Frempong, would you proceed, please, and thank you
very much for being here.
Dr. Ohene-Frempong. And thank you very much, Senator, for
the opportunity.
Earlier, my colleagues at Boston Sickle Cell Center and at
Meharry Comprehensive Sickle Cell Center gave overviews that I
think pretty much describe what the comprehensive sickle cell cen-
ters are about. And I will briefly just go over some of the accom-
plishments of the center at Philadelphia where I am, and also fin-
ish with some recommendations that I have for consideration.
Our center serves the greater Philadelphia-Delaware Valley area
as a resource for families affected by sickle cell disease. Directly,
we care for 520 children up to 19 years of age who are affected by
this disease. We also serve as the referral center for all of Louisi-
ana, for the southern part of New Jersey, and also for Delaware,
for children with sickle cell disease who suffer very serious con-
sequences.
For the State of Pennsylvania, we also serve as a resource in de-
veloping and monitoring the State sickle cell programs. We devel-
oped the newborn screening program for the State and actually
conducted a pilot project in Philadelphia for 2 years before the
State picked up the program and went statewide with it.
36
Our sickle cell effort started in 1973, and we have had some sup-
port from the State to care for sickle cell patients continuously
since that time.
We became a comprehensive sickle cell center in the 1988-93
cycle, and we and our colleagues here went through the competitive
renewal applications in 1992 and were lucky to get funded from the
1993 to 1998 cycle.
Our research addresses all the major areas that the comprehen-
sive sickle cell centers deal with. We have basic in basic laboratory
areas, in clinical areas, in psychosocial areas, and in education,
counseling, and testing areas.
In the basic research arena, our attention has been focused on
studies that would lead to gene therapy and other treatment for
sickle cell disease. A team of our investigators studying the regula-
tion of the synthesis of fetal hemoglobin at the DNA level found
two previously undescribed changes in the DNA from Benin and
Bantu haplotypes of sickle cell disease. These are separate origins
of the sickle cell gene in Africa. The major Benin haplotype was as-
sociated with a genetic change which had not been previously de-
scribed, and another change was also found in the Banto haplotype.
We think that ultimately, this sort of knowledge promises to be
useful in developing ways to modify the expression of these fetal
hemoglobin genes, and hopefully, this will lead to a way of improv-
ing sickle cell disease as patients can be given treatment that will
increase the fetal hemoglobin production.
As we all know, sickle cells are abnormally shaped and stiff in
comparison with normal red blood cells. Effective treatment or cure
for sickle cell disease will have to alter the abnormal morphology
and the deformability of sickle cells.
Another team of our investigators has developed an accurate
technique to analyze the morphology of sickle cells, using a comput-
erized image analysis system. This technique allows the accurate
determination of morphological differences of sickle cells numeri-
cally, by calculating several differentiated factors. This system fa-
cilitates the investigation of the relationship between the morphol-
ogy of sickle cells and various environmental and drug conditions.
This technique is currently being applied to support the testing of
new drugs in sickle cell disease patients.
One of the drawbacks of the development of new treatments for
sickle cell disease has been the lack of animal models for this dis-
ease. All drug tests are either performed in vitro in the laboratory
or directly in humans. A group of our investigators together with
others in the country have developed transgenic sickle cell diseased
mice which produce sickle cells containing human sickle hemo-
globin. These mice offer the opportunity to study sickle cell disease
in the model that will permit the testing of a wide range of agents
and also for trials of gene therapy.
In clinical research, we have conducted studies aimed at under-
standing some of the most difficult problems encountered by sickle
cell disease patients, as well as to test new drugs for potential use
in treating trie disease. Stroke is one of the most devastating com-
plications of the disease, affecting about 5 percent of all patients,
including children less than 2 years of age. Our studies of the blood
circulation of the brain have aimed at discovering children who
37
may be at high risk for stroke so that preventive therapy can be
developed for them. Using magnetic resonance techniques, we and
others in the country have shown that as many as 20 percent of
children with sickle cell disease suffer silent stroke, and that these
children may be at the highest risk for overt strike. We are cur-
rently working with our collaborators at the national level to de-
velop methods of intervention to stop the occurrence of the first
stroke
Stroke and sickle cell disease again have a nasty tendency to
recur, and when they do recur, they lead to more severe brain dam-
age. The objective of current treatment of stroke, chronic trans-
fusion therapy, is to prevent this recurrence. One of the major haz-
ards of long-term chronic transfusion therapy is iron overload, a
problem which is potentially fatal if not aggressively treated. Inves-
tigators at our center have successful demonstrated that iron over-
load in chronically transfused patients can be safely reduced in two
ways. First, they demonstrated that in stroke patients who had
been transfused aggressively for at least 3 years and who have had
no neurological setback, the intensity of the transfusion can be
safely reduced without recurrence of stroke.
In addition, they have shown that when this modified trans-
fusion program is coupled with a red cell exchange instead of sim-
ple transfusion regimen, further reduction in transfusional iron
loading and in body iron stores can be achieved. These findings are
expected to have a major impact on management of the problem of
iron overload in sickle cell patients who are on chronic transfusion
therapy. In combination, these methods have the potential to post-
pone the need for iron chelation therapy for a number of years.
Hydroxyurea is perhaps the most promising drug ever tested in
sickle cell disease. This drug, as we heard earlier, is currently un-
dergoing control trials in adult sickle cell patients in an NIH-fund-
ed study. In anticipation of the potential impact of hydroxyurea on
sickle cell disease treatment, investigators at our center began pre-
liminary studies of hydroxyurea in children with sickle cell disease
2 years ago. This summer, we and others from three other institu-
tions are (beginning a limited trial of hydroxyurea in a larger group
of children in an NIH-funded study.
Have we made any progress at all in sickle cell disease? The an-
swer is a qualified yes. Certainly, sickle cell patients today are liv-
ing longer than they lived before. Thirty years ago, half of sickle
cell patients did not live beyond 20 years of age. In a recent publi-
cation which came out of the cooperative study of sickle cell dis-
ease, where the NIH organized a large group of centers that have
been collaborating in clinical studies since 1978, we have been able
to show that for patients at least living in the 1980's, at least half
of them lived beyond 45 years of age. This is a tremendous im-
provement from what it was 10 years ago. But that still means
that about half of these patients lose at least 30 years of useful life,
so we still have a long way to go, and sickle cell disease research
needs to be supported.
I have the following recommendations to make. First, that the 10
National Institute of Health approved comprehensive centers,
which have not had a real increase in their level of support for the
last 15 years, need to be funded at least at their approved levels.
38
Last year, these centers suffered an average of about 8.5 percent
reduction in their funding, and this year, they are operating at 5.6
percent below their recommended and approved budgets. We ask
that Federal funding for the centers be increased by at least $4
million to enable the approved research projects to be conducted as
recommended in the peer review process.
The centers should be specifically mentioned in the language of
any appropriation for increased funding for the National Institutes
of Health.
The well-organized cooperative study of sickle cell disease, a net-
work of medical centers established through the National Heart,
Lung, and Blood Institute, should be supported as the ideal set-up
for testing of new treatments in sickle cell disease. This network
demonstrated its value in the 1980's by the rapid conduct of the
penicillin prophylaxis study in children with sickle cell disease, and
I must say this study was the most important clinical study ever
conducted in this disease.
Funding for centers for gene therapy for sickle cell disease
should be increased. Many brilliant molecular biologists and gene
therapy experts whose talents could be recruited into this area of
research are hesitant to join the effort because of concerns about
long-term commitment of support. Congress should make a com-
mitment to finding a cure for this genetic disease through major
funding for gene therapy for sickle cell disease. And this will be a
fitting response to the contributions made to medical science and
the understanding of genetic diseases in general by those affected
by sickle cell disease and its related disorders.
My final recommendation is that there is a severe under-rep-
resentation of African Americans and other minority groups in bio-
medical research. In order to increase the number of African Amer-
icans and members of other minority groups in biomedical re-
search, adequate funding should be provided to fully fund the Mi-
nority Supplement Program of the National Heart, Lung, and
Blood Institute.
In addition, in accord with a report of a National Heart, Lung,
and Blood Institute task for on the declining number of investiga-
tor-initiated research proposals in sickle cell disease, a Sickle Cell
Research Scholars Program should be established within the com-
prehensive sickle cell centers. Each center should be provided suffi-
cient resources to support at least one scholar drawn from the
ranks of young investigators on the medical school and hospital fac-
ulties where centers are funded.
I think that sickle cell centers have truly been much more than
what we expected from them, they have been worth much more
than the funding that they have received. The work that has been
done through the Sickle Cell Disease Branch of the NIH has ad-
vanced research in this work further than any of us expected. We
are very close, we think — and we promise this to our patients — we
are very close to a cure for this disease, and this is the time that
I think we should pull together all these resources to support the
scientists who have been working so hard in this area.
Thank you very much, Senator.
Senator Wellstone. Thank you, Doctor.
39
I think what we will have to do, since I will have to leave in a
short period of time, is we will submit written questions to you.
I did want to mention, though — and I really appreciate your tes-
timony—I was a little saddened by what you said— not by what you
said, because I was actually inspired by what you said — but when
you talked about the budgets, I think back to when my father, who
suffered from Parkinson s disease, was in the original L-DOPA
pilot group, when that was first used as a drug, at George Wash-
ington Hospital here, in the same room was a young African Amer-
ican man who was in a sickle cell crisis period, and I have just
never seen such agony, and I will never forget it.
The reason I mention that is I work with people who come here,
unfortunately, just in their 40's, with Parkinson s, and they too are
trying to see some expansion of the budget, and there is all sorts
of promise.
On the NIH budget, there is the Harkin-Hatfield initiative to try
to have a set-aside of one percent increase across-the-board. I just
do not want to see different people who are struggling with dif-
ferent illnesses pitted against one another, and it does seem to me
that we are making such a terrible mistake by not really commit-
ting the resources to the research, to save money in the short run,
we end up spending more in the long run. It is not just a question
of spending less; it is also a question of what you can do for people.
So I really appreciate what you say, and I thank you.
Dr. Ohene-Frempong. Thank you, Senator.
Senator Wellstone. And if it is okay, I would like to get some
of these questions to you to get a response in writing.
Dr. Ohene-Frempong. That will be fine, Senator.
Senator Wellstone. Thank you.
[The prepared statement of Dr. Ohene-Frempong follows:]
Prepared Statement of Kwaku Ohene-Frempong, M.D.
A. GENERAL COMMENTS
Sickle cell disease is an inherited disease of red blood cells. The red cells of people
with the disease have a tendency to become rigid ad abnormally shaped, causing
them to break up easily and lead to anemia, and also block the flow of blood through
blood vessels and lead to many other complications. The hallmark of the disease is
the episodic attack of severe pain in various parts of the body. Most people with
sickle cell disease eventually die from it. In the best circumstances, half of all sickle
cell patients die by 45 years of age, about 30 years earlier than expected in the gen-
eral population. There are approximately 60,000 sickle cell disease patients in the
United States of America with 2.5 million people with sickle cell trait, the healthy
carrier state of the sickle cell condition. Worldwide, hundreds of thousands of babies
are born each year with this disease, about 150,000 in Africa alone. Most of these
babies die by five years of age without the recognition of sickle cell disease as the
underlying cause of their deaths from malaria, respiratory and other diseases. Chil-
dren with sickle cell trait, not the disease, are protected from severe malaria. While
people of African descent are predominantly affected by sickle cell disease, the dis-
ease is also found in all the countries in the Mediteranean basin, especially Italy,
Turkey, and Greece, all the Middle Eastern countries, and in India. Throughout
these areas of the world and where descendants have immigrated, sickle cell condi-
tions are a major health problem.
Sickle cell disease was discovered by modern medical science in 1907 in Chicago.
From the early stages of research into the nature and effects of this disease, it was
also discovered that the disease in the United States of America affected predomi-
nantly Americans of African origin. The fact that the patient through whom the dis-
ease was revealed to medical science was a dental student from Grenada and not
one of the hundreds of American patients who must have suffered the pain and
other complications of the disease may reflect another facet of sickle cell disease.
40
Since the general public has become aware of the disease, there has been the im-
pression that the disease has not received much attention because it affects pri-
marily a minority African-American community.
The fact is that sickle cell disease has received plenty of attention from basic lab-
oratory research scientists in the U.S. and elsewhere. Sickle cell disease and its re-
lated disorders are some of the best studied and best understood diseases of man.
Sickle cell disease was the first "molecular disease". It was the first disease whose
cause was found to reside in dh abnormality in a molecule, hemoglobin. The simple
difference between "sickle" hemoglobin and its normal version was determined al-
most 50 years ago. When the genetic code was discovered, the genetic basis of sickle
cell disease was easily established as a simple singular mutation in one gene. The
gene for sickle cell disease was discovered in 1977, almost twenty years ago! With
all these "firsts", one would expect that research would have made dual progress
in the treatment of sickle cell disease. Unfortunately, medical science has gained a
lot more from the study of the blood of people affected by sickle cell conditions than
the people have received from medical science.
The treatment of sickle cell disease today is largely symptomatic and does not ad-
dress the fundamental cause of the disease. As the first molecular genetic disease,
it has always been expected that sickle cell disease would be one of the first diseases
for which gene therapy would be attempted. To date, this has not happened.
However, patients with sickle cell disease are better off today than they were 20
or 30 years ago. Thirty years ago, only half of children with sickle cell disease were
expected to live beyond 20 years of age. For patients living in the 1980's this had
improved to about 45 years of age. This improvement had come about because of
on-going research to understand the effects of sickle cell disease and to develop
treatment methods for its many complications.
Much of what we have learnt about sickle cell disease has come out of research
sponsored by the National Institutes of Health primarily through the Sickle Cell
Disease Branch of the National Heart, Lung and Blood Institute. A large part of
this NIH effort on behalf of sickle cell disease has been organized through the Com-
Erehensive Sickle Cell Centers. These Centers, funded on a competitive 5-year cycle,
ave provided a large body of new information on the basic, clinical and
psychosocial aspects ofsickle cell disease. The Centers have also provided opportuni-
ties for the development of demonstration projects on testing and counseling of indi-
viduals for sickle cell conditions. The lessons learnt from the testing and counseling
of large numbers of people for sickle cell disease are currently being applied to other
genetic disorders such as cystic fibrosis, as the discovery of the genes Tor other dis-
eases make such testing possible. For the past 15 years, there nave been 10 Com-
prehensive Sickle Centers. Even though the number 10 was established as a mini-
mum there have not been more than 10 Centers since that minimum was estab-
lished. The Comprehensive Sickle Cell Center at the Children's Hospital of Philadel-
phia is one of the current cycle of 10 Centers. Our Center and some 22 other Cen-
ters have participated in the Cooperative Study of Sickle Cell Disease (CSSCD). Per-
haps the greatest achievement of the CSSCD is the studies conducted on the risks
and prevention of bacterial infections in young children with sickle cell disease. The
famous Penicillin Prophylaxis Study (PROPS) conducted in the mid-1980's by the
CSSCD showed that the lives of young children with sickle cell disease could be
saved by giving them ordinary penicillin twice a day every day. Infection from
strains of the bacterium, pneumococcus, had been well established as the leading
cause of death in these children and the first 3 vears of life had been found to be
their riskiest for death. The eventual outcome of PROPS was the establishment of
newborn screening for sickle cell disease. Since young lives could be saved with pen-
icillin prophylaxis, it became important to discover babies with sickle cell disease
early so that the penicillin treatment can be started before they died of pneumo-
coccal infection. Today, some 43 states have added testing for sickle cell conditions
to their newborn screening programs. Many young lives are being saved and the
first three years are no longer the most dangerous for people with sickle cell disease.
While survival beyond childhood may not l>e as major a problem as it used to and,
while their overall life expectancy has improved, the lives of sickle cell disease pa-
tients are not without tremendous difficulty. They still lead lives frequently inter-
rupted by painful attacks ("crises "), paralyzing strokes, acute chest syndrome (lung
damage), acute splenic sequestration (lifethreatening sudden enlargement of the
spleen), aplastic crisis (transient cessation of red cell production), priapism (painful
sustained erections), and other acute manifestations of the disease. Over several
years, many patients suffer chronic damage to and failure of organs such as the
spleen, lungs, kidneys, liver, and heart. In the absence of treatment that prevents
the production of sickle cells, much of our efforts has been directed to understanding
and management of these acute and chronic complications.
41
B. RESEARCH AT THE COMPREHENSIVE SICKLE CELL CENTER, THE CHILDREN'S HOSPITAL
OF PHILADELPHIA
At the Comprehensive Sickle Cell Center at The Children's Hospital of Philadel-
phia, we have devoted our research efforts to address all the important aspects of
sic le cell disease, basic, clinical and psychosocial. The following is a brief summary
of our recent accomplishments, placed in the context of national developments in
sickle cell disease research.
1. Basic Research
Our attention has been focused on studies that will lead to gene therapy and
other treatments for sickle cell disease. A team of our investigators studying the
regulation of the synthesis of fetal hemoglobin at the DNA level found two pre-
viously undescribed changes in the Benin and Bantu haplotypes of the sickle gene.
The major Benin haplotype was associated with a Gy-309 A to G change and the
Central African Republic with an Ay-271 C to G change. Ultimately this knowledge
promises to be useful in developing ways to modify the expression of fetal genes and
therefore ameliorating the severity of sickle cell disease.
Sickle cells are abnormally shaped and stiff in comparison with normal red blood
cells. Effective treatment or cure for sickle cell disease will have to alter the abnor-
mal morphology and deformability of sickle cells. Another team of our investigators
has developed an accurate technique to analyze the morphology of sickled cells
using a computed image analysis system. This technique allows the accurate deter-
mination of morphological differences of sickled cells numerically by calculating sev-
eral different shape factors. This system facilitates investigation of the relationship
between the morphology of sickled cells and various environmental and drug condi-
tions. It is currently being applied to support the testing of new drugs in sickle cell
disease patients.
One of the drawbacks of the development of new treatment in sickle cell disease
has been the lack of a natural animal model for the disease. All drug tests are per-
formed either in vitro or directly in humans. A group of our investigators and others
in the country have developed transgenic sickle cell disease mice which produce
sickle cells containing the human sickle hemoglobin. These mice offer the oppor-
tunity to study sickle cell disease in a model that will permit the testing of a wide
range of agents and also for trials of gene therapy.
2. Clinical Research
In clinical research, we have conducted studies aimed at understanding some of
the most difficult problems encountered by sickle cell disease patients as well as test
new drugs for potential use in treating the disease.
Stroke is one of the most devastating complications of the disease, affecting about
5 percent of all patients including children less than 2 years of age. Our studies of
the blood circulation of the brain have aimed at discovering children who may be
at high risk for stroke so that preventive therapy can be developed for them using
magnetic resonance techniques, we and others in the country have shown that as
many as 20 percent of children with sickle cell disease suffer "silent" stroke and
that these children may be at the highest risk for overt stroke. We are currently
working with our collaborators at the national level to develop methods of interven-
tion to stop the occurrence of the first stroke.
Strokes in sickle cell disease have a tendency to recur and lead to more severe
brain damage. The objective of current treatment, chronic transfusion therapy, is to
prevent this recurrence. One of the major hazards of long term chronic transfusion
therapy is iron overload, a problem which is potentially fatal if not aggressively
treated. Investigators at our Center have successfully demonstrated that iron over-
load in chronically transfused patients can be safely reduced in two ways. First,
they demonstrated that in stroke patients who had been transfused aggressively for
at least three years, without any neurological set-back, the intensity of the trans-
fusion can be reduced without recurrence of stroke. In addition they have shown
that when this modified transfusion program is coupled with a red cell exchange in-
stead of simple transfusion regimen, further reduction in transfusional iron loading
and in body iron stores can be seen. These findings are expected to have a major
impact on the management of the problem of iron overload in sickle cell patients
on chronic transfusion therapy. In combination these methods have the potential to
postpone the need for iron chelation therapy for a number of years.
Hydroxyurea is perhaps the most promising drug ever tested in sickle cell disease.
This drug is currently undergoing a controlled trial in adult sickle cell disease pa-
tients in an NIH-funded study. In anticipation of the potential impact of
hydroxyurea on sickle cell disease treatment, investigators at our Center began pre-
liminary studies of hydroxyurea in children with sickle cell disease two years ago.
42
This summer, we and investigators at three other institutions are beginning a lim-
ited trial of hydroxyurea in a larger group of children in an NIH -funded study.
C. HAVE WE MADE SIGNIFICANT PROGRESS IN SICKLE CELL DISEASE RESEARCH?
The importance of sickle cell disease research in our understanding of human ge-
netic disease is well recognized. Recent developments finally are bringing some hope
of effective treatment and universal cure for this disease. Some of these important
developments are summarized below.
1. Treatment to increase the level of feted hemoglobin in red blood cells
Fetal hemoglobin, Hb F, is the predominant hemoglobin in the red cells of babies
during pregnancy. Normally, we switch off the production of Hb F toward the end
of pregnancy as we increase the production of Hb A, the normal "adult" hemoglobin.
Sickle cell disease is due to an abnormal form (Hb S) of Hb A. The most important
fact in sickle cell disease research is that Hb F in appreciable amounts can block
the abnormal behavior of Hb S and prevent the formation of sickle cells. Much re-
search attention has been devoted to understanding the natural switch for Hb F
production and to find ways to stimulate the production of large amounts of Hb F
beyond birth. Currently, two groups of medications hold promise toward this goal.
Hydroxyurea, alluded to above, has been the most tested, and Butyratess are begin-
ning intensive testing. This direction of research promises the simplest and most
cost effective way in which sickle cell disease can be treated worldwide.
2. Bone marrow transplantation for sickle cell disease
Sickle cell disease can be cured by transplantation of normal bone marrow. The
application of this treatment to sickle cell disease was first attempted, in Belgium
and has been gaining deliberately slow trial in the U.S. Because of the risks associ-
ated with marrow transplantation and the need for tissue matched donors, it is not
expected that large numbers of sickle cell disease patients will be treated with this
method in the near future. Lessons learnt from the few cases being performed will
be very useful in gene therapy for sickle cell disease.
3. Newborn testing for sickle cell disease
In the public health arena, the addition of sickle cell testing to the newborn
screening programs of most (43) states is the most important development in the
East twenty-five years. In 1986, the Sickle Cell Disease Branch of the National
[eart, Lung, and Blood Institute with the CSSCD published the results of the Peni-
cillin Prophylaxis Study. The implementation of newborn testing and penicillin pro-
phylaxis therapy at about two months of age is perhaps the most significant im-
f>rovement in the care of sickle cell disease patients to date. Implementation of simi-
ar programs around the world has the potential to save over a hundred thousand
lives a year.
4. Gene Therapy for sickle cell disease
In theory, this has been the expected cure for sickle cell disease ever since the
disease was found to be caused by a simple mutation in a single gene. Unfortu-
nately, as gene therapy was being aggressively pursued for other genetic disorders,
sickle cell disease, the prototypical genetic disease, was left to wait. Encouraging de-
velopment came this year as the NIH accepted applications for research projects in
gene therapy of sickle cell disease. It is hoped that this area of research will expand
into treatment trials in the not too distant future.
D. RESEARCH NEEDS IN SICKLE CELL DISEASE
At a time when there is excitement in sickle cell disease research for effective
treatment or universal cure, scientists are being discouraged by diminishing funds
for research. Since 1982, investigator initiated applications for funds for sickle cell
disease research has been dwindling. One of the reasons must be the perception of
unavailability of funding support. Research scientists are spending half of their
working hours writing and rewriting grant applications as they search for support.
On the patient and community side, there is increasing concern that when funds
become short in supply, conditions such as sickle cell disease, which affect a largely
minority population, receive even less attention.
Sickle cell disease has been a "natural" attraction for young African Americans
and members of other minority groups seeking careers in biomedical research. The
Comprehensive Sickle Cell Centers, with their large collection of senior and middle
level research scientists, have served as fertile breeding grounds for such budding
scientists. However, the Centers are no longer allowed to recruit students and young
physicians as part of the Minority Supplement Program of the NM. Each year, we
turn down several applications for research apprenticeship from students because
of lack of support.
I would like to make the following specific recommendations:
43
1. Overall the funding for research projects dealing directly with sickle cell dis-
ease has been declining in relationship to other conditions supported by the Na-
tional Heart, Lung, and Blood Institute of the National Institutes of Health. The
10 National Institutes of Health-approved Comprehensive Sickle Cell Centers have
not had a real increase in their level of support Tor the past 15 years. These Centers
are currently funded at 5.6 percent below their recommended and approved budgets;
they suffered 8.5 percent reduction in funding for 1993/94. We ask that federal fund-
ing for the Centers be increased by $4 million to enable approved research projects
tobe conducted as recommended in the peer review process. The success of the Cen-
ters concept in advancing research in sickle cell disease deserves to be recognized
by adequate funding. The Comprehensive Sickle Cell Centers should be specifically
mentioned in the language of any appropriation for increased funding for the Nffl.
2. The network of medical centers established through the National Heart, Lung,
and Blood Institute of the National Institutes of Health for clinical investigations
in sickle cell disease should be maintained as the best system for quickly testing
new treatments in sickle cell disease. In this regard, the well organized Cooperative
Study of Sickle Cell Disease should be supported as the i ideal network for clinical
trials in sickle cell disease. This network demonstrated its value in the rapid con-
duct of the Penicillin Prophylaxis Study. ,„,,«.
3. Funding for Centers for Gene Therapy for Sickle Cell Disease and Related Dis-
orders should be increased. At the moment only two such Centers are expected to
be established through the National Institutes of Health. Many brilliant molecular
biologists and gene therapy experts whose talents can be recruited into this area
of research areliesitant to come in because of concerns about long term commitment
of support. Congress should place its commitment to finding a cure for this genetic
disease through major funding for gene therapy for sickle cell disease. This will be
a fitting response to the contributions made to medical science and the understand-
ing oogenetic diseases by those affected by sickle cell disease and related disorders.
4. There is severe underrepresentation of African-Americans and other minority
groups in biomedical research. In order to increase the number of African Americans
and members of other minority groups in biomedical research, adequate funding
should be provided to fully fund the Minority Supplement Program of the National
Institutes of Health particularly in relationship with the Comprehensive Sickle Cell
Centers. In addition, in accord with a report of a National Heart, Lung, and Blood
Institute Task Force on the declining number of investigator-initiated research pro-
posals, a Sickle Cell Research Scholars Program should be established within the
Comprehensive Sickle Cell Centers. Each Center should be provided sufficient re-
sources to fund at least one such Scholar drawn from the ranks of young investiga-
tors on the medical school and hospital faculties where Centers are funded.
Senator Wellstone. Mr. Jones.
Mr. Jones. Thank you, Senator Wellstone.
It is indeed an honor and a distinct privilege for me to have an
opportunity to address the committee this afternoon on this very
important issue.
Southern University in Baton Rouge has, as a result of the 1992
legislative statute, created a vehicle for a national research center
for sickle cell anemia. Southern University is the largest predomi-
nantly black, historically black college or university in the United
States, with approximately 16,000 students in the Southern Uni-
versity system.
This research center not only has the support of our Governor
and our legislature, but also has the support of the National Bap-
tist Convention U.S.A., the National Rainbow Coalition, and the 40
members of the Congressional Black Caucus. So this research cen-
ter that is proposed at Southern University, as a national research
center, because there is no national research center — there are
comprehensive centers, and the basic difference between this pro-
posal and the comprehensive centers is that this center will be
dedicated to research and research only.
The practical effect of the disease was summed up by a young
woman with the disease who made this statement during the 23rd
Annual Congressional Black Caucus Week: "I have sickle cell ane-
44
mia, and I am not supposed to be here today. I usually wish that
I was not. I wished that I was dead. Sickle cell causes me a lot of
pain. The pain is so bad I cannot stand it. Sometimes, I start
breathing fast and can hardly catch my breath. I get weak. I start
to slur my speech and go in and out of consciousness. Sometimes,
I pass out in the street. People step over me or on me. They think
I am a drug addict. If I get to the hospital, everybody thinks I am
on drugs, too, and they treat me like some kind of criminal. I can-
not hold down a job because of the frequency of my crises. I do not
have no money, no nothing, because of sickle cell."
The role of the National Sickle Cell Research Center at Southern
University, again unlike the 10 comprehensive sickle cell service
and counseling centers which presently exist, this center will en-
gage exclusively in research, basic biomedical research and
psychosocial research.
Why establish the National Sickle Cell Research Center at
Southern University, some have asked. Even if Congress opts to
support a National Sickle Cell Research Center, why should it be
located at Southern University, and not in some other State or at
another institution?
The answer is three-fold. One, Southern University's location in
the State of greatest need, in a State that is approximately one-
third African American, with 3,248 persons with the disease, 25
percent of whom will have the acute form of the disease which is
usually fatal.
The second point is that Southern University is in the State of
Louisiana, and the State of Louisiana has made a commitment to
this project as evidenced by a $7 million appropriation to the na-
tional center for its establishment. No other State in the United
States has made such a commitment. In fact, in furtherance of the
State's effort, this year, just 2 months ago, the State of Louisiana
put an additional $700,000 into the State budget to create clinics
for sickle cell patients throughout the State of Louisiana. There is
not one urban area in the State of Louisiana that will not have a
sickle cell clinic where these patients will be able to receive treat-
ment, with a hematologist on staff.
Senator Wellstone, as I sit here today, a little more than 1 year
after the national center's launching meeting at Southern Univer-
sity, in addition to the $7 million committed to the effort by the
State of Louisiana, Southern is obtaining financial commitments
for the center from alumni, corporations and foundations. Southern
has faculty, research staff commitments, and collaboration between
LSU and the Earl K. Long Hospital, which are located less than
5 miles from Southern.
I am here today to a:k the Federal Government to join us in this
collaborative public-private venture, to help us turn the lives of
tens of thousands of Americans who are impacted by sickle cell
anemia. Join in our partnership to find a cure for sickle cell anemia
tomorrow by investing the 3-to-l match of $21 million today.
The investment will help the State of Louisiana and the region
to develop a cure for the United States and for all persons who are
affected by sickle cell anemia.
I would like to again thank you for this opportunity of addressing
the committee and to say that we are committed to finding a cure
45
for sickle cell anemia. We need your help, and we would ask you
to vote favorably and support this support this initiative.
[The prepared statement of Mr. Jones appears at the end of the
hearing record.]
Senator Wellstone. Thank you, Mr. Jones.
Certainly, there is no question about the reputation of Southern
University, and I think we will probably want to put some ques-
tions to you as to what the State commitment might be, not just
this year, but the following year and the year after that, what kind
of annual appropriation would it be over a period of time. But let
us put those questions to you and get a written response.
I thank botn of you, and I do apologize for the committee. It is
very, very unusual the way this happened today. It is the worst
possible timing for everyone. I personally think that all too often,
sickle cell and those who suffer and struggle with it are out of
sight, out of mind, put in parentheses and put into brackets. There
should be, must be, has to be more of a commitment.
I only came in at the very end because of other commitments I
made a long time ago, but I think your testimony is very impor-
tant, and I thank everybody who was here today, and I apologize
for my colleagues for the conflicts that they had.
[Additional statements and material submitted for the record fol-
low:]
THE ACCOMPLISHMENTS OF THE COMPREHENSIVE SICKLE CELL
CENTER AT MEHARRY MEDICAL COLLEGE
JULY 26, 1994
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HTBttCTOR. ttlMPREHENSiyg STCXL2 CKT.l CSUTSR
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INTRODUCTION
Sickle cell disease (3CD) refers to a group of chronic,
hereditary blood disorders that primarily affects African-
Americans. One in every 400 black babies born in the United
States is affected by SCD. The disease is characterized by
diverse symptoms , and numerous complications ; throughout their
lives affected individuals are plagued by recurrent bouts of
pain secondary to vaso-occlusion which is the hallmark of the
disease. These recurrent episodes of pain and vaso-occlusion
often result in incapacitation. Other problems include
increased risk of infections aseptic necrosis, increased risk
of infections, and delays in growth and development just to
name a few of its associated complications.
The Physical Aspects of. SC3
Sickle Cell Disease is an autosomal recessive red blood cell
disorder characterized by sickling of erythrocytes,
obstruction of the microcirculation and a chronic hemolytic
anemia. The disease is seen predominantly among the Afericain-
46
American population in the United States, but is also
prevalent among populations of Africa, the Mediterranean,
Caribbean, Middle East, and southern India. Thus because the
United States is composed of many individuals from all parts
of the world its is also diagnosed in other ethnic groups
within the US. On the African continent SCD is thought to
provide a natural immunity to malaria, but it has no such
usefulness to its carriers in the rest of the world.
Pathogenesis
The first documentation of SCD was made in 1910, by a
Chicago cardiologist, James Herrick who discovered it in the
bloodstream of a west Indian student. Almost four decades
later, Pauling and colleagues demonstrated that the sickling
phenomenon was due to a structural defect in the hemoglobin
molecules and this work resulted in reward of the Nobel prize
for medicine.
Hemoglobin is composed of heme, the red-colored
substance, and globin, a protein component. The function of
hemoglobin is to transport oxygen from the lungs to the
tissues. The abnormality in siclcle cell disease lies in the
globin portion of hemoglobin. Globin consists of two pairs of
polypeptide chains which include alpha chains and non-alpha
chains. The alpha chain consists of 141 amino acids, while the
non-alpha chains consist of 146 amino acids.
A decade after Pauling's discovery, Ingram demonstrated
that the abnormality in the hemoglobin molecule was due to a
biochemical alteration, which was the resuit of the
substitution of a single amino acid by another. With classic
sickle cell disease, sickle cell anemia, (HbSS), valine has
be*n substituted Cor glutamic aeid at the sixth position or
the beta chain. Currently over 700 abnormal hemoglobin are
Known. Some of the more common variants are describe are found
in Table 1.
Table 1
syndrome
HbS
Substitution
Site
Glu-> Valine
36
HOC
Glu-> Lysine
fl6
HbD
Glu-> Glna
3121
HbE
Glu-> Lysine
326
Ordinarily, red blood cells with normal hemoglobin are able
to maneuver through the microcirculatory system with relative
ease. In sickle cell anemia, because of the single amino acid
substitution the hemoglobin takes on different properties. It
continues to have normal oxygen carrying capacity. Upon
deoxygenation, however, there i3 a change in the solubility of
the protein so that the HbS molecules clump together to form
tactoid. The cell membrane becomes distorted, and assumes a
sickle shape, these cells are inflexible, sticky, and survive
about 7-20 days. Sickled cells are often described as having
a "log- jamming effect" in the smaller passage ways, which
result in a broad range of clinical manifestations, sickled
cells can resume their rounded shape upon re-oxygenation but
only for a limited number of times. After a period of time,
they become irreversibly 3ickled and are removed from
circulation via the reticuloendothelial system.
Hfoqloftjn Variants
Currently more than 700 abnormal types of hemoglobin have
been identified in man. Three hemoglobin form our total
hemoglobin picture: Ha, Ha,, and HbF. Hemoglobin A is the most
abundant of the three. It is composed of two alpha chains and
two beta chains. The other two hemoglobin are found in lesser
amounts, but must be oresent for a normal hemoglobin pattern.
Ha, is composed of two alpha chains and two delta chains. HbF
is composed of two alpha chains and two gamma chains. HbF is
47
also known as fecal hemoglobin and is the major type of
Hemoglobin produced before birth. After birth, production of
gamma chains decreases as production of beta chains begin,
thus one would see a decreased amount of HbP and an increased
amount of Ha. Host abnormal hemoglobin result from point
mutations in the structural genes that code for the amino
acid 3eguence of the globin chains of the molecule.
Hemoglobin AS is sickle cell trait. Sickle cell trait
occurs in one out of every 10 Black Americans. In fact, there
are over two million Black Americans with sickle cell trait.
Sickle cell trait is not a disease, it is a benign carrier
state that rarely produces health problems. The concern of the
3ickle cell trait carrier is that of prospective parenthood.
If both partners have the trait, there is a 2S p«r cant chanco
that with each pregnancy the couple will have a child with
sickle cell anemia. Except in a few unusual circumstances,
individuals with sickle cell trait are asymptomatic.
The carrier state AS is benign because there is a
preponderance of hemoglobin A in each red blood cell of the
hemoglobin AS type. Usual proportions in each cell are
approximately 60 per cent hemoglobin A and 40 per cent
hemoglobin S. There is therefore not enough hemoglobin S to
cause the symptoms of the homozygous condition, HESS.
Vai-^ntB at SCD
After sickle cell anemia, the most common forms of sickle
cell disease in decreasing order of frequency are sickle
hemoglobin C disease (HbSC), and sickle-beta thalassemia of
which there are two subtypes ( S-6° and S-fl* disease ) . In each
form of sickle cell disease, at least one 3-globin structural
gene B* codes for the synthesis of a sickle hemoglobin. The
allelic fl-globin gene may code also for a sickle hemoglobin
(HbSS) , another structural abnormal hemoglobin (HbSC), or may
produce either a partial defect in hemoglobin A synthesis (S-
B* thalassemia), or a complete defect in hemoglobin A
synthesis (S-B° thalassemia) .
sickle Hemoglobin C Disease
It has been estimated that 2 per cent of the Black
population carry the gene for hemoglobin C. The incidence is
1 per 1000 live births. The carrier state as in the case of
sickle cell trait is asymptomatic.
Sickle hemoglobin C disease has been described as a
milder form of classic sickle cell disease, that is sickle cell
anemia. Individuals with the disease have a higher hemoglobin
level and less frequent episodes of pain. Growth and
development may not be affected to the same degree as in
hemoglobin ss disease. As in the case of sickle cell anemia,
HbSC patients have an increased risk of developing certain
complications that are related to their disease. For example,
retinopathy can result in retinal detachment, and necrosis of
the femoral head can be seen (Lin-Fu, 1979). Individuals with
HbSC have bouts of transient functional hyposplenia. This puts
them at risk for not being able to combat infections
adequately. Many individuals with the disease are Less
symptomatic than the homozygous HbSS patient and may not
obtain adequate medical supervision.
TTgnoglobin Sickle 3>f»-f hn1w«5«wi>i
Sickle beta-thalassemia occurs in about 0.4 per cent of
the Black population in the United States. Individuals with
sickle beta-thalassemia have symptoms that vary from mild to
those as marked as hemoglobin SS. Clinically, there is less
anemia, and less sickling was found in vivo. The vaso-
occiusive phenomenon that causes painful episodes still
occurs .
48
As was mentioned earlier, there are two types of beta-
thalassemia, 3* and 3*. In sicXia beta"-tnalassen\ia some normal
hemoglobin A is produced, but far more of the abnormal
hemoglobin S is produced. In sickle beta" -thalassemia , only
abnormal beta chains are made, producing hemoglobin s, and no
hemoglobin A is found. Often different clinical pictures are
seen with these two hemoglobinopathies. Variability is an
important factor, some experts report that Sickle beta-
"thalassemia produces milder clinical symptoms than sicXle
beta'-thalassemia. Nevertheless, sicXle beta-thalassemia is a
sickling disorder and must be managed in the same way as other
sickling syndromes.
Table 2
Incidence of SicXle Cell Disease
in the United States
Condition
Hb Types
% of BlacXs
SicXle Cell Trait AS
SicXle Cell Anemia SS
Hemoglobin C Trait AC
Homozygous C Disease CC
SicXle Hemoglobin
C Disease SC
Hemoglobin D Trait AD
Homozygous D Trait DD
Hemoglobin £ Trait AZ
SicXle Call Thalassemia S-Thal
Beta-Thalassemia Trait Thai Minor
8-14%
0.3-1.3%
2.3%
.016%
0. 1-0. 25%
0.08-0.4%
MA
HA
0.04%
1%
* KA =» Hot Available
Diagnosis
The diagnosis of sickle cell disease is aade by
performing a hemoglobin electrophoresis. The basic concept of
electrophoresis is that protein molecules are electrically
charged and will migrate in an electric field. The way a
particular hemoglobin migrates depends upon the pH and allows
for identification of different proteins.
Clinical Manifestations
The complications of sickle cell disease are quite
varied. In some cases individuals are entirely asymptomatic
and are detected only during population screening, wneraas
others are constantly plagued by painful episodes.
The major complications of sickle cell disease can be
placed into three categories: acute events, chronic events, or
health related events that require added medical attention.
Acute events include painful episodes, acute chest syndrome,
right upper quadrant syndrome, skeletal and joint events,
hand-foot syndrome, cerebrovascular accidents, aplastic
episodes, sequestration crisis, acute febrile event, and
priapism. Chronic events include leg ulcers, renal
49
complications, aseptic necrosis, and ocular complications.
Health related events include delayed growth and development,
delayed sexual maturation, pregnancy, surgery, and anesthesia
(Walters, 1983).
For the first few months of life there ia usually no
indication of the disease upon physical examination. Because
there is more production of sickle hemoglobin and less HbF, by
six months of age, more symptoms begin to develop. These
include dactylitis, hepatomegaly, and splenomegaly. In the
older child, during non-acute phases varying degrees of
pallor, scleral icterus, hepatomegaly and cardiomegaly can be
seen. In homozygous HbSS disease, the spleen is enlarged in
early childhood as a result of its function in removing
sickled cells from circulation. Functional activity is lost,
however, in the first few years of life. After about 6-3 years
of age, autosplenectomy or the self-destruction of the spleen
occurs as a result of repeated splenic infarcts. Delayed
growth and delayed sexual maturation, frontal bossing, and
sometimes gnatopathy are characteristic of sickle cell
disease.
The physical aspects of sickle cell disease constitutes
only one facet of the health problem. A number of psychosocial
issues, including the psychological concomitant of extended
hospitalization and isolation, long tern adjustment to
survival, and an uncertain disease course have also surfaced.
Mary care-givers feel that addressing these problems is
equally as important as addressing the physical problems that
arise as a result of the disease.
Despite the complexities of SCO, the health prognosis has
improved over the past decade. Prior to 1974, SCD was
considered a terminal illness. Although still considered a
chronic disease, SCD is no longer considered a fatal disease
prior to adulthood. In fact, individuals with 33 have been
reported as living well into their 70s. Deaths occurring
before adulthood were largely related to severe infections,
e.g., pneumonia and organ failure. Fortunately, treatment and
management, which include early detection, proper medication,
and counseling, have improved the overall physical status.
In summary, SCD is a non-contagious, inherited blood
disorder. There are many variants of SCD, the S3, and the SC
states are the most prominent in the United States. Although
many physical discomforts are associated with the disease,
affected individuals can lead useful and productive lives ,
given proper medical treatment and management.
The improvement in the overall survival of individuals
with SCD has occurred since 1972 when President Nixon in 1972
enacted the National Sickle Call Disease Act. This is directly
related to the establishment of Comprehensive Sickle Cell
Centers which have provided for:
1. an environment for research
a. basic
b. clinical
2. Laboratory diagnosis
3 . genetic counseling
4. universal slckla cell education
At the Meharry Comprehensive Sickle Cell Center the
development of our center has in addition to the above
activities being accomplished the following have also bean
achieved :
1. the achievement of a referral Center status
2. Stimulation of Research
a. intramural
b. extramural
3 . becoming a resource center locally and especially for
region IV
4 . the development of a strong community outreach
program
50
STIHUIATIOK OF RESEARCH
without the support of the NIH and tha sickle cell disease
branch it would have been difficult to establish the
environment where limited resources, facilities and manpower
existed to coordinated and stimulate research. The actual
development process took a number of years to develop. This
support has also allowed us interact with vanderbilt
University School of Medicine to further develop and apply
application of new knowledge in the diagnosis and treatment
of sickle call disease. In the development of these overall
activities a multidiscipline team approach from many
individuals involved with health care delivery was required.
And to maintained the activities requires that this team
approach be maintained.
EDUCATION
TESTING
COUNSELING
PATIENT CARE
RESEARCH
51
COMPREHENSIVE SICKLE CELL CENTERS
Program Components
EDUCATION
Pallenl
Famly
General Pubttc
Professionals1
PSYCHOSOCIAL
SERVICES
RESEARCH
Basic
CCm'cai
Appled
PATIENT
COUNSELNG
Carriers
Disease
Oilier
Abnormal
I leinogloblns
DIAGNOSIS
Pallenl
Family
Prenalal
Newborn
COMPREHENSIVE SICKLE CELL CENTER
COMPONENTS
PATIENT CARE
COUNSELING -
RESEARCH
COMMUNITY
- OUTREACH
DIAGNOSIS
EDUCATION
52
COMPREHENSIVE SICKLE CELL CENTER
COMPONENTS
INFANTS
MTiENT
DARE" m
ADULTS
ADOLESCENTS
COMPREHENSIVE SICKLE CELL CENTER
CASE MANAGEMENT
Genetics,
Science Emergency Medicine
^-— * — ^ JJursing
X. Legal Services
Health Education*
X
Radiology* ►/ —*.-—■-- ^ --^T , Social Services
PA II E N llY^» Pharmaceutics
PATI £ ^^ [^Ophthalmology
J ► Hematology
/— ♦Ob/Gyn
/■-*. Family Medicine
Surgery
Vocational Rehab
Pediatrics, —
Home Health Services
/
/ ■
\
Private NIH
Agencies DePt- of
Family Support Systems
TN.
Health and Environment
53
TESTIMONY OF CHARLES D. JONES
LOUISiANA STATE LEGISLATOR
CHAIRMAN, {LAW & JUSTICE COMMITTEE,
NATIONAL BLACK CAUCUS OF STATE LEGISLATORS
&
CONVENOR. CHAIRMAN, DELfTA PILOT ECONOMIC DEVELOPMENT PROJECT
DELTA ECONOMIC ENERGY DISTRICT
BEFORE THE UNITED STATE SENATE
COMMITTEE Ot\ LABOR AND HUMAN RESOURCES
JULY 28, 1994
INTRODUCTION
Chairman Kennedy , Senator i assenbaum, other distinguished members of the Committee,
I am Louisiana State Senator Charges Dean Jones, representing Louisiana's 34th Senatorial
District. I also appear before you ttlis morning as Chairman of the Law & Justice Commiaee
of the National Blade Caucus of Stale Legislators (NBCSL) and as the Convener and Chairman
of the Delta Pilot Economic Deyeioj ment Project and its Delta Economic Energy District, Inc. ,
(DEED). My remarks before this body today are also embraced by the National Baptist
Convention, USA, Inc., and the Naional Rainbow Coalition.1
Before discussing the background of. the need for and the utility of federal support for
the National Sickle Cell Research 'Center at Southern University, I want to thank Senator J.
Bennett Johnston for his unstinting support and his championship in the United States Senate of
legislation authorizing the Secretary bf the Health and Human Services to award a grant for me
establishment of the National Center) for Sickle Cell Disease Research and Southern. I want to
thank Senator Johnston also for his iresence here today.
I take this opportunity to eitend my gratitude to my long-time friend Congressman
William Jefferson (D-2-LA) who is >roviding the leadership necessary to advance the measure
through the United States House of R spresentanyes. Congressman Jefferson also joins us today.
Finally, I take this opportunity to ack xrwledge the efforts of Congressman Clco Fields (D-4-LA)
to gamer support for House legisation that wILL establish on the campus of Southern
University- located in Congressman Field's District- the National Sickle Cell Research Center.
The establishment of this Center is ; , matter of paramount concern not only to the residents of
Congressman Fields' District and to others in the State of Louisiana and the Delta Region, but
to African Americans across the nation.
As evidence of the broad national support for the National Sickle Cell Research Center,
I note that the unanimous 40-membc Congressional Black Caucus of the United States Congress
is in support of the National Center. In March of 1993, every member of the Congressional
Black Caucus joined in sending a later to Secretary of Health and Human Services. Donna
Shalala, urging her support for tbe Rational Research Center at Southern. (Letter attached.)
1 The National Baptist Convention, USA, Inc., (NBC, USA) is the nation's oldest and
largest association of Black Baptis, rninisters, representing tens of thousands of active and
involved registered voters across A merica, as well as residents of Africa and the Caribbean.
The national president of NBC, US \ is Reverend Dr. T.J. Jemison, who hati« from the State
of Louisiana. Last year at its annuil convention in New York City, NBC, USA, adopted by
unanimous consent "A Concurrent Resolution" urging and requesting that its members support
in any manner of means, the establishment of a national Sickle Cell Disease Research Center
at Southern University. (Resolution! attached.)
The National Rainbow Coalition is a national multi-racial, multi-cultural political and
economic empowerment association, founded and chaired by The Reverend Jesse L. Jackson.
Earlier this year. The Rainbow Coalition's Health Task Force recommended to Congressman
Louis Stokes (D-OH), Chairman of the Congressional Black Caucus Health Braintrost and
Convener of the African American Leadership Health Care Reform Work Group, that the CBC
and the Work Group support and urge the appropriation of adequate federal funds to compliment
(in a three to one match) the state ftnds appropriated by the State of Louisiana to establish and
rnainiain at Southern University, this nation's only Sickle Ceil Research Center.
54
The National Blade Caucus <k State Legislators, too, is in support of the Center. During
its 17th Annual Conference in Deiver Colorado last year, NBCSL adopted a resolution in
support of the National Centex for Sickle Cell Research , by unanimous consent. NBCSL is the
educational, research and training association of the nation's 540 African American state
legislators who primarily represent
and/or work in rural communities,
many who exist in poor America
Americans, NBCSL members are
social empowerment of African
institutions, is central to the coi
too. are united in the belief that
icons Americans across the country— some of whom reside
in urban areas. Some who exist in affluent America,
Whether representing urban or rural, affluent or poor
.ted in their belief that the continued political, economic and
and the enhancement of their communities and
growth and development of this nation. NBCSL members,
equality in this country can only be achieved when each
American is guaranteed equality or life chances; and that a crucial prerequisite for likely
achievement of equal life chancre e accessibility of affordable, quality health care beginning
before birth. This access must include necessary research to find cures for diseases peculiar to
either gender; diseases peculiar to ijfants, children, and seniors; and people of every race and
ethnicity, including African Americans. Access must also include necessary research to find
cures for those whose diseases are behavioral in origin and those whose diseases are hereditary.
BACKGROUND AND NEED FOR THE NATIONAL SICKLE CELL RESEARCH CENTER
It is axiomatic that acccssibi ity of affordable, quality health care, proper nutrition, safe
and sanitary home and cmploynent conditions and a clean environment are necessary
components of an overall quality health care system. It is a fact that quality health care, proper
nutrition, safe and sanitary home and employment conditions, and a clean environment are
necessary to begin to equalize life chances among the haves and the have-nots. As a child who
grew up m the poorest region in -unerica. in the nation's poorest city, Lake Providence,
Louisiana, where 82% of the population is on public assistance; and as a state legislator for
nearly two decades, representing ax only Lake Providence, but also the city of Monroe.
Louisiana, where I presently reside-- which is the nation's third poorest city- I see daily and
have witnessed firsthand for more han four decades, the fact that children cannot study to
capacity and cannot grow to be sturc y, productive members of our communities without sound
nutrition, safe housing and health maintenance based on prevention. I have seen firsthand the
deleterious and debilitating effect uit poverty and the concomitant lack of health and human
needs services; absence of proper nutjrition; lack of safe and sanitary and employment conditions;
and a hazardous environment have,
entire region.
not only on a people, a dry, parish or a state, but on an
It was in part my witnessing these things, that lead me in 1993, to organize the Delta
Pilot Economic Development Project, and to launch as its initial project the Rural Community
Health Service Project. The Delu| Pilot Economic Development Project is an Arkansas/
Louisiana/ Mississippi Tri-State rtgiobal economic and human development corporation launched
at my urging with broad and diversfe support from the Tn-State region, including that of the
governors of the three states; many members of the congressional delegations of the three
states; state and local elected officials from the Tri-state Region: business persons, labor,
religious leaders, community leaders] and with the support of thousands of the suffering residents
of this beleaguered region. Our goal is to formulate and implement collaborative regional
solutions to the myriad problems wrfich plague this region, not the least of which are that it has
the nation's highest illiteracy rate, highest poverty rate, rural poverty and minonty poverty rate,
highest infant mortality rate, highestjmfinnity rate, and lowest educational attainment rate in the
nation. The group set as goals, Inz'tr alia, the establishment of an economic development and
heaith corridor defined by the Mississippi River. In so doing, we were building upon and
organizing to implement the recoramendations of the Lower Mississippi Delta Commission,
chaired by then Governor Bill Clini an.
With regard to the cstabllsl ment of the Health Corridor, the Delta Pilot Economic
Development Project noted that the ArkLaMlss Region has the highest percentage of medically
under-served residents in the nation , the highest infant mortality rate in the nation, the highest
low birth weight births, the highest inridwm of births to teen mothers, among the lowest
primary care physician-to-patient arid registered nurse-to-patient ratio in the nation. Collective
remedial goals were established to iring the Tri -State region at least up to par with the rest of
the nation. Each of yon has been provided with a copy of the Delta Pilot Economic
Development health care findings and goals.
55
The Sate of Louisiana took the lead in following up on the Tri-State Health Care
recommendations. The Northeast Louisiana Council on Black Economic Development conducted
a surrey of the rural health care pmblems in all 45 rural parishes in the state. A copy of the
survey and a copy of the recommendations and actions taken pursuant to the survey—including
the passage of state legislation to address some of the findings— have been provided to each of
you this morning. While all of the findings are troubling, most were anticipated. We were
alarmed, however, by the »w»rfingi y high number of African Americans across the state with
the Sickle Cell Anemia disease or Q ait. We found that Louisiana has the highest number of
citizens per capita afflicted with die £ ickle Cell disease in the moon. Of Louisiana's 1,299,281
minorities. 3,248 have Sickle Cell .'mania and 129.928 have the trait. In the rural parishes
alone, 990 residents have the disease while 37,838 possess the trait. Nationally, the sickle cell
disease is estimated to affect more tr. in 50,000 Americans. Approximately one in every twelve
(12) black Americans is born with th : Sickle Ceil trait, and about one in four hundred (400) has
Sickle Cell Anemia.
For those of you who are "nfamiliar with the disease, it is not a silent killer, like high
blood pressure, it takes its victims lacking and screaming. It causes excruciating pain. It causes
diseases, infections, mal-functioning of crucial organs like the spleen, then it kills! It is an
inherited disorder of the red blood cells, that disproportionately affects persons of African
ancestry. To a far lesser degree, pei sons of Arabian, Caribbean, Centrai and South American,
East Indian and Mediterranean ancestries are affected by the disease, also. As you know, red
blood ceils carry oxygen to all parts of the body through a protein called hemoglobin. Normal
red blood cells contain normal hemoglobin and are donut shaped. They move easily through
blood vessels. For those with sickle cell disease, the red blood ceils contain sickle hemoglobin
which causes them to have a curved or sickle shape— much like the end of a tuning fork— after
oxygen is released. Sickied cells become stuck in blood vessels, thus blocking the blood flow
and causing damage to the tissue. A major effect of the disease is the sickle cell "crisis" which
occurs when sickle cells plug blood vessels and block the flow of blood. The blockage typically
lasts several days, usually affecting ihe arms, legs, hands, feet, abdomen or another local part
of the body. Tissue damage, often i tvolymg major organs, occurs with each successive episode
of oxygen deprivation. The cumulative effects of the disease are debilitating and life
threatening. Those affljrtrd with severe forms of the disease usually do not live through their
teens.
The practical effect of the Usease was summed up by a young woman in her early
twenties, from Senator Mikulski's sate, who appeared on a panel Congressman Louis Stokes
convened on this deadly disease, last September during the 23rd annual Congressional Black
Caucus Week. Said the woman:
"I have Sickle Cell
today
dead.
taemia and I am not supposed to be here
1 usually wist that I wasn't [sic] here. I wish that I was
sickle cell cstses me a lot of pain. The pain is so bad
[sic] catch my breath
I can't stand it.... Sometime I start breathing fast and can't hardly
... I get weak, start to slur my speech and go
in and out of consciousness. Sometime I pass out In die street.
People step over me or on me. They think I'm a drug addict. If
I get to the hospital, ( Yerybody there think [sic] I'm on drugs, too,
and they treat me like some kind of criminal I can't hold down
a job for long because of the frequency of my crises. I don't have
oo [sic] money, no nothing [sic] because of Sickle Cell."
Despite the fact that the cause
very rninor structural variation in tte
toward developing a cure or suitable
ncatmem for the painful crisis is med can
symptoms. It does not cure the malady
sickle cell.
of the sickle cell disease has been known for years— the
mutant hemoglobin-very little progress has been made
treatment for the disease. At the present dine, the major
ion for relief of pain, which simply treats the immediate
or enhance the quality of the lives of those who have
In recent yean, the federal government has been responsive to this situation. The
Department of Health and Human Services has a nanonal program to reduce the morbidity and
mortality from sickle cell disease. The Department's National Heart, Lung and Blood Institute
(NHLBI) of the National Institutes ojf Health (NIH) has the lead responsibility for carrying cut
56
gfffm ?nf
the government's sickle ceil ah
Sickle Cell centers across 'he
screening, diagnosis, management,
center dedicated exclusively to
discovery of a cure for this deadly
federal authorization legislation
ihe-art Sickle Cell Research Center
There is a need for such a center,
that affects more than 50,000
program. There are currently ten (10) Comprehensive
nation which engage in demonstration education projects,
c muscling and clinical studies. No where is there a national
cot ducting multi-disciplinary research that will lead to the
disease. And, neither the current NIH appropriations nor
ide for the expenditure of federal funds to erect a stete-of-
No present monies can be used for structural development.
foster the expeditious discovery of a cure for this disease
provide
tc
Amercans
THE ROLE OF THE NATIONAL
SICKLE CELL RESEARCH CENTER AT SOUTHERN
Unlike the ten (10)
presently exist, the National Sickle
basic biomedical research, and
comprehensive
Sickle Cell service and counseling centers which
Cell Research Center will engage exclusively in research:
research.
BASIC BIOMEDICAL RESEARCH
The Center will undertake
formation of sickle ceils, the behavior
the characteristics of sickle cells al Der
isolation, characterization, and synthesis
of the disease to lead more normal
deformed. These areas of investigation
prevent, inhibit, or reverse the sickl jog
is an area of basic investigation tha :
Collaborative arrangements
have the interest in and expertise to
psychosocial
i lvestigations such as gaining a better understanding of the
of sickle ceils when they flow through blood vessels, and
they sickle. Considerable study will be devoted to the
of anti-sickling agents- substances that enable victims
'. ives by decreasing the '•frrr** of red blood ceils becoming
are required in order to determine the types of drugs that
process. A search of current literature suggests that this
leaves many questions unanswered.
will be undertaken with other universities In the state that
do basic biomedical research related to anti-sickling agents
as well as sickle cell disease and oha hemoglobin abnormalities.
The Center will conduct studies as the ultrastructural levels in the areas of molecular
biology and genetic engineering. These investigations will focus on various generic approaches
to the sickling process. Specific ittention will be given to DNA manipulation and other
techniques of modern biology which could cause panents to begin synthesizing normal adult or
fetal hemoglobin. Electron microscopy is one of the tools that will be used in investigating in
varo and in vivo sickling. The cor wpt of genetic engineering is one of the new and rapidly
developing disciplines io science. S Miches of the literature suggest that not enough attention
is being given genetic engineering with sickle cell disease at the target.
PSYCHOSOCIAL RESEARCH
The Center will conduct a wide variety of human behavioral studies designed to shed new
knowledge on such issues as the effectiveness of various counseling and education methods,
coping drills on the part of patiems and families, and counselor education and evaluation.
Qualification standards do not currently exist for sickle cell generics counselors. Research
questions need to be answered as to What rnimmum body of knowledge needs to be mastered in
order to become effective in the rol«
A broad range of national
related to Sickle Ceil Anemia will
of which some will be descriptive w
types of research questions they
studies on questions related to ma
testing for the presence of S (si<
registries are all areas of public po
es intended to shed new light on public policy issues
undertaken. Many of these will be observational studies
e others will be analytical in approach, depending on the
Cross-sectional, group comparison, and longitudinal
atory genetic testing of susceptible newborns, premarital
;) genes and the need for state and national sickle cell
that lack hard data.
Clinical Research Collaborative arrangements win be made with other Louisiana higher
education institutions, such as the sireYeport and Tulane University Medical Center to conduct
clinical trials on anti-sickling agents, some of which may have been discovered during basic
laboratory investigations at this CerJcer and elsewhere. Because of the close proximity of Earl
K. Long Memorial Hospital to Southern University, basic biomedical investigators at the Center
and LSU faculty at Earl X. Long, yul be able to conduct a number of joint studies.
57
The Center will not engage ib
of its research objectives. Howevei
Sickle Oil Foundadoo ind other
these organizations to do so.
Sfckle
WHY ESTABLISH THE
AT
sickl* cell services to patients except as negrirrl in support
, die Center will work cooperatively with the Baton Rouge
Cell service organizations in the state, when asked by
NATIONAL SICKLE CELL RESEARCH CENTER
SOUTHERN UNIVERSITY
:ven if Congress opts to support a National Sickle Cell
located at Southern University and not in another state or
Some of you have aslced,
Research Center, why should it be
at another institution. The answer tj> this question is twofold
Southern's Location in the State of Greatest Need,
Its Capability and Its ' Commitment to the National Center; and
The State's Cornmrcmmt to the Project as Evidenced by its
S7 million Appropnat on for the National Center.
SOUTHERN'S LOCATION IN TIE STATE OF GREATEST NEED, ITS CAPABILITY
& ITS COMMITMENT TO THE JNATIONAL CENTER
Southern University is located in Baton Rouge, Louisiana. As noted previously, the
state of Louisiana has the highest mimber of citizens per capita afflicted with the Sickle Cell
disease in the nation. Of Louisianais nearly 1.3 million minorities, more than 133,000
have either the disease or the trait. NCore than 10% of the state's minority populauon is direcdy
affected by this killer disease. Nationally, approximately one of every twelve (12) persons of
African descent is affected. Southern University, one of the state's premier institutions of higher
education, and the nation's largest 1 istorically and predominantly black land grant insotunon,
wants to do something immediately to find a cure for this disease. Southern has begun taking
steps to identify a remedy for this nalady.
Located in Baton Rouge,
has been graduating scholars and
for 113 years. It has a long track
some of whom go on to attend
the sciences, including chemistry
morfe than 16.663 students attend Southern University. Southern
sen ling them out into the national and international workforces
re cord of graduating students with degrees in basic sciences,
medic al school. Others receive advanced degrees in research and
aqd biology.
aid
The National Aeronautics
acknowledged Southern's research
American scholars. Senator Mikulfcki
Administration and the Department
grant to develop an aerospace eh]
Engineering. This is just one examal
and, the type of federal recognition]the
Space Administration and Department of Energy have
capability and its important role in educating African
just last year, the National Aerospace and Science
of Energy awarded the institution a five-year, S5 million
>gineering undergraduate option in the Department of
e of the type of federal support the institution has received
institution receives for academic excellence.
As the nation's largest HBCTJ and one that is located in the state with the largest
percentage of sickle cell victims, 5 outbern has •^tr"nittr*i its resources to finding a cure for
Sickle Cell disease. Toward this « ad, the University assembled on its campus for a two-day
work session, the foremost experts n the fields of biomedical research, psychosocial research,
pediatric hematology, oncology, ar d related fields. (The attendees and notes from the work
sessions are contained in the back c f the Rural Community Health Service Project, which you
have been provided.) The group de reloped a proposed curriculum for the Center, a set of goals
and a timetable for launching the N ational Research Center. It was resolved that the National
Sickle Cell Research Center at Sjuthern would conduct collaborative research with other
Louisiana universities, agencies am I medical schools. The group resolved that the Center will
later collaborate with other universities and agencies in the region, and across the nation. The
work group also discussed the ways at* means of budding a state-of-the-art Sickle Cell Research
center. Southern alumnae, loundadons, corporanons, state and federal funds were targeted for
support of the project.
Southern is taking affirmative steps to prepare its faculty for the coming of the Center
Faculty internships have been conducted on Southern's campus in the area of Sickle Ceil
58
Anemia. For one week, faculty
the sickle cell disease.
attend
i lectures conducted by the nation's foremost experts on
Evidencing its support for th* National Cenicr, the Louisiana State Legislature passed a
bill which I authored, committing S{1 million to the effort: an initial SI. 5 million in start up
funds; and S5.5 million for capitol butlay to be used toward building a research center. This
latter appropriation is contingent upoi 1 Southern' s receiving a three-to-one match from the federal
government.
CONCLUSION
Mr. Chairman, as I sit here tpday, a little more than one year after the National Center's
launching meeting at Southern, in addition to the S7 million committed to the effort by the state
of Louisiana, Southern is obtaini
corporations and foundations,
collaboration agreements with LSU
miles from Southern. I'm here
collaborative public/private venture
of Americans who are impacted by
.ig financial commitments for the Center from alumnae.
Southern has faculty, research staff commitments and
ind Earl K. Long Hospital, which are located less than five
loday to ask the federal government to join us in the
:o help us turn the lives around of the tens of thousands
Sickle Ceil Anemia. Join in our partnership to find a cure
for Sickle Cell Anemia tomorrow, >y investing $21 million today
This investment will help the State of Louisiana and. the Delta Region take the first step
toward launching its regional health ind economic development corridor-both of which are sorely
oeeded to bring relief to this beleaguered region. For, as long as the Delta is suffering, America
will suffer. As long as there is poverty in the Delta, the region of New Pngiand won't be rich.
As long as disease is rampant and thousands of people in the Delta cannot expect to live to be
twenty or thirty years, the people cf Minneapolis can never be totally healthy even if they get
a good check up at the Mayo Clinic Senator Durenburger. For we can never be what we ought
to be until the "least of these" are 'that they ought to be. We are interdependent No man or
woman stands alone. Senator Kennedy, Members of this Commioee, please stand with Southern
University, the State of Louisiana, the Delta Economic Energy District, the Congressional Black
Caucus, the National Black Caucus of State Legislators, the National Baptist Convention, USA,
the National Rainbow Coalition an|l the 50,000 Americans whose lives are adversely affected
by Sickle cell Anemia, on the isaic of funding a National Sickle Cell Research Center at
Southern University.
I thank you; and I will be nappy to entertain any questions you may have.
59
2^m.mii'a* a— Kunmn — ui ^.ttCAt -v~-^ui
JON 0. JOHNSON
CAo/rmon
MA«TY J. CHAiHT
Gr*oon> J. Sorro
Jim Cax
Willi* C.-oin
Oswald 0*cvir
Marc H. Mortal
CkiI Pieoro1
John Saunaan
77m Honorable Donna Shahia
Secretary, Department of Hearth
and Human Services
200 Independence Avenue, SW
Washington, D.C. 20201
Dear Secretary Shaiata:
:i0*t U2-:o*i
LAJi i. arvjcm
MfeVC/l JO, 1993
J*N<2 MUCHt5
During the 1992 Louisiana Legisiative Regular Session, 7.5 million was
appropriated lor start-up funding, planning and designing the first National
Research Center for SickleiCell Anemia in the United States,
As Chairman of the Revenue and Fiscal Affairs Committee for the Louisiana State
Senate, please know mat m plan to amend the Capital Outlay bill this session to
Include 5 J million dollars tor the First National Sickle Cell Anemia Research Center
at Southern University • Baton Rouge, Louisiana.
is natidr,
As you know, there Is national support for the Research Center and Its purpose,
which is to research, to develop and to conduct a nationally recognized short and
long range program that will delve into the causes of Sickle cell disease and its
cure. The Research Center will be domiciled at Southern University In Baton
Rouge and establish a cooperative relationship with the Department of Health and
Hospitals, other Universities, National and International Associations and Agencies
interested In the Research center.
Thank you tor your conspiration of this matter.
Very Truly Yours,
Jon D. Johnson
Chairman
60
3ouc-n«m ■JnJ.v«rsity, latan .i.ouq«, Ju.Uirj
Maclaaal sicx_L« Call An—la iueirc". Cantar
JOtn District, /Taxas
V1U1M Clay /
lac an triers, Ulsaouxi j
rXon*\ld 7. 3»lluasT
3«n District, Caliiorala
5th District, itissouri
Sth 3l»erlct. laor^l*
Craig a. waaninetan.
L8tn District. ?«u«
/Luclsn 31actw«il
Sad district, Pennsylvania
I
carol :^o»«iaY/6fc*un
U.S. Sanata, fliiaoia
;arriao 3rswn/
3rd District y Tlorida
Smrl Milliard
7th District, Alabama
ilcs« Hasting" y
lara^Dlstrict. ?lprtda
/Sax&ara-Ross Coilins^"^
■^i <«y c. Dixon
j5Btf"Diatrirt, Calixorai*
ai-pftaj Towns
.Ota District, *«v Tor*
Donald M. ?ayn«
icth District, Maw jaravy
5tS ilstrict.
lonnsctirut
tlaanor Holaaa-Horton
Siatrict ai Caluaoia
Sva X. Clayton y
1st District, .ions Carolina
? &
a r> > ;?■ ,. i
Sanxoro D. 3i*aop I / /
2aflF-oifctrict, SsorgU /
»s S. Cly&nm /
5th District, south- C*roli.T*
Carrie ?. xm*
17ts«»3istrict. riojjios
xsirta
2nd District, 'Illinois
61
waller ?ocJe»r, 1X2
AU»rt '<*ym» "
*«» Otstzicc, Maryland
Rob*^*^— Scott
3rd 3i«*rict, vtrtjiaia
.lelvin w«tc
:2th Di*tricc, NortA Carolina
(SENATE
STA71E OF LOUISIANA
CHARLES 0. JONES
MarwiM
FandMtal-
! «Uand. Em, Coral. TcnMl.
Ul DCSIMO STREET JUITtlli
MONROt. lOUBUNA 71 201
COMMITTEES:
S*noM> aW Gi'ihiipwhj AHam.
vk» ChaMinm
MfM
r t
A CONCURRENT RESOLUTION
r M I'
:o w Ouaor
Hid
To urge and reguestj the members or the National Baptist
Convention, U.S.A., Inc. to take the necessary steps to support the
National Sickle Call Disease Research Center located at Southern
University and Agricultural and Mechanical College in Baton Rouge,
Louisiana .
FURTHER, to set aside one Sunday annually for ail churches
belonging to the National Baptist Convention, U.S.A., Inc. to
recognize and financially contribute to the research program of the
"National Sickle Ceil Disiease Research Center." On this Sunday,
member churches will take part in a "One Great Hour of Sharing"
special offering to be donated to the Research Center in its vorfc
toward eradicating 3icklej cell disease.
WHEREAS, there is no national center exclusively devoted to
sickle cell disease research currently existing in the United
States; and
WHEREAS, the Congressional Black Caucus, consisting of 4a
members of the United States Congress unanimously is in support of
•etablishing a national sickle cell disease research center on the
campus of Southern Univerjsity; and
WHEREAS, during its f 1992 Legislative Session, the Louisiana
Legislature created a National Sickle Cell Anemia Research Center
under the leadership of Senator Charles D. Jones, and funded it
with $1.5 million for planning and development and a $5 . 5 million
outlay. commitment to begin construction; and
jia
WHEREAS, President-elect Bill Clinton has committed his
support for the establishment of a national sickle cell research
center on the campus of southern University and has committed to
appropriate a three to dne match of the State of Louisiana's $7
million, in the amount o± $21 million; and
WHEREAS, the National Black Caucus of State Legislators has
commended the Louisiana Legislative 31ack Caucus, the State of
Louisiana and Senator Charles D. Jones, lead author, for passing
legislation and for funding the creation of the nation's first
National Sickle Cell Aneaia Research Center; and
BOSTON PUBLIC LIBRARY
62 3 9999 05982 600 6
WHEREAS, the National 31ack Caucus of Stare Legislators
supports the efforts of the Louisiana State Black Caucus to
establish thia research center at Southern University at 3aton
Rouge, Louisiana; and
WHEREAS, only the sra^ll amount of $15 million is contributed
annually by the federal government to research sickle cell disease;
and
WHEREAS, one in ten African Americans carries a gene for
sickle hemoglobin, and one in every five hundred black nevroorns in
the United States has sickle cell; and
WHEREAS, all newborns at risk should be screened for 3ickle
cell disease to permit iarlier diagnosis and treatment and to
significantly reduce illness and the death rate among those
children with, the disease t .and
WHEREAS, there existjs a need to emphasize the importance of
early detection and educate the primary medical care providers ,
parents and community jsn the importance of timely medical
management of those patients with this inherited diseaoe; and
WHEREAS, sickle celij disease is one of the least understood
diseases of our time, vhLch presently has no cure and continued
research into its causes and cure is vital; and
I
WHEREAS, the establishment of a research center for such
purposes would certainly enhance the programs currently in place by
broadening the resource
committed to finding relisf for the unfortunate victims who suffer
base for research using those experts
serious and life- threatening disease.
resolved that the National Baptist
hereby urges and requests its members to
the consequences of this
THEREFORE , BE IT
Convention, U.S.A., Inc. nereoy urges ana requests its meraoers to
take the necessary steps to support the national sickle cell
disease research center at Southern University and Agricultural and
Mechanical College in Bation Rouge, Louisiana.
BE IT RESOLVED that a copy of this Resolution be transmitted
_to sach_ member church, leach member of the Congressional 31ack
"Caucus" of the United Stages Congress , each member of the National
Association of Black State Legislators and the President of the
United States .
LtbtK) £> tf/iZrusr^
Senator Wellstone. Thank you very much. The committee is ad-
journed.
[Whereupon, at 3:36 p.m., the committee was adjourned.]
82-553 (68)
ISBN 0-16-044846-8
9 780160"448461
90000