[Senate Hearing 108-151]
[From the U.S. Government Publishing Office]
S. Hrg. 108-151
JUVENILE DIABETES: EXAMINING THE PERSONAL TOLL ON FAMILIES, FINANCIAL
COSTS TO THE FEDERAL HEALTH CARE SYSTEM, AND RESEARCH PROGRESS TOWARD A
CURE
=======================================================================
HEARING
before the
COMMITTEE ON
GOVERNMENTAL AFFAIRS
UNITED STATES SENATE
ONE HUNDRED EIGHTH CONGRESS
FIRST SESSION
__________
JUNE 24, 2003
__________
Printed for the use of the Committee on Governmental Affairs
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COMMITTEE ON GOVERNMENTAL AFFAIRS
SUSAN M. COLLINS, Maine, Chairman
TED STEVENS, Alaska JOSEPH I. LIEBERMAN, Connecticut
GEORGE V. VOINOVICH, Ohio CARL LEVIN, Michigan
NORM COLEMAN, Minnesota DANIEL K. AKAKA, Hawaii
ARLEN SPECTER, Pennsylvania RICHARD J. DURBIN, Illinois
ROBERT F. BENNETT, Utah THOMAS R. CARPER, Delaware
PETER G. FITZGERALD, Illinois MARK DAYTON, Minnesota
JOHN E. SUNUNU, New Hampshire FRANK LAUTENBERG, New Jersey
RICHARD C. SHELBY, Alabama MARK PRYOR, Arkansas
Michael D. Bopp, Staff Director and Counsel
Priscilla Hobson Hanley, Professional Staff Member
Joyce A. Rechtschaffen, Minority Staff Director and Counsel
Jason M. Yanussi, Minority Professional Staff Member
Amy B. Newhouse, Chief Clerk
C O N T E N T S
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Opening statements:
Page
Senator Collins.............................................. 1
Senator Pryor................................................ 3
Senator Lautenberg........................................... 4
Senator Bennett.............................................. 5
Senator Specter.............................................. 13
Senator Durbin............................................... 13
Senator Coleman.............................................. 24
Senator Fitzgerald........................................... 25
Prepared statement:
Senator Lieberman............................................ 41
WITNESSES
Tuesday, June 24, 2003
Hon. E. Benjamin Nelson, a U.S. Senator from the State of
Nebraska....................................................... 6
Mary Tyler Moore, International Chairman, Juvenile Diabetes
Research Foundation (JDRF)..................................... 7
Katie Halasz, Delegate, age 16, JDRF Children's Congress, Wells,
Maine.......................................................... 17
Colleen Rea, on behalf of her son, Dylan Rea, Delegate, age 4,
JDRF Children's Congress, Stamford, Connecticut................ 18
Sophia Cygnarowicz, Delegate, age 7, JDRF Children's Congress,
Columbia, Illinois............................................. 19
Eric Bonness, Delegate, age 18, JDRF Children's Congress, Omaha,
Nebraska....................................................... 20
Alex Bonness, Delegate, age 15, JDRF Children's Congress, Omaha,
Nebraska....................................................... 21
LaNiece Evans-Scott, Delegate, age 10, JDRF Children's Congress,
Backlick, Ohio................................................. 22
Allen M. Spiegel, M.D., Director, National Institute of Diabetes
and Digestive and Kidney Diseases, National Institutes of
Health......................................................... 28
Bernhard J. Hering, M.D., Associate Professor of Surgery,
Director of Islet Transplantation, University of Minnesota,
Minneapolis, MN................................................ 30
Anne Seidel, Islet Cell Transplant Recipient, Dallas, Texas...... 33
Alphabetical List of Witnesses
Bonness, Alex:
Testimony.................................................... 21
Prepared statement........................................... 56
Bonness, Eric:
Testimony.................................................... 20
Prepared statement........................................... 54
Cygnarowicz, Sophia:
Testimony.................................................... 19
Prepared statement........................................... 52
Evans-Scott, LaNiece:
Testimony.................................................... 22
Prepared statement........................................... 58
Halasz, Katie:
Testimony.................................................... 17
Prepared statement........................................... 48
Hering, Bernhard J., M.D.:
Testimony.................................................... 30
Prepared statement........................................... 66
Moore, Mary Tyler:
Testimony.................................................... 7
Prepared statement........................................... 42
Nelson, Hon. E. Benjamin:
Testimony.................................................... 6
Rea, Colleen:
Testimony.................................................... 18
Prepared statement........................................... 50
Seidel, Anne:
Testimony.................................................... 33
Prepared statement........................................... 77
Spiegel, Allen M., M.D.:
Testimony.................................................... 28
Prepared statement........................................... 60
Appendix
Prepared statement from Alessio Fasano, M.D., Director, Center
for Celiac Research, University of Maryland, Karoly Horvath,
M.D./Ph.D., Co-Medical Director Center for Celiac Research,
University of Maryland, Carlo Catassi, M.D., M.P.H., Co-Medical
Director Center for Celiac Research, University of Maryland,
Stefano Guandalini, M.D., Director, Celiac Disease Program,
University of Chicago, and Peter H.R. Green, M.D., Director,
Celiac Disease Center, Columbia University with an attachment.. 79
JUVENILE DIABETES: EXAMINING THE PERSONAL TOLL ON FAMILIES, FINANCIAL
COSTS TO THE FEDERAL HEALTH CARE SYSTEM, AND RESEARCH PROGRESS TOWARD A
CURE
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TUESDAY, JUNE 24, 2003
U.S. Senate,
Committee on Governmental Affairs,
Washington, DC.
The Committee met, pursuant to notice, at 10:05 a.m., in
room SH-216, Hart Senate Office Building, Hon. Susan M.
Collins, Chairman of the Committee, presiding.
Present: Senators Collins, Coleman, Specter, Bennett,
Fitzgerald, Durbin, Lautenberg, and Pryor.
Also Present: Senator Nelson.
OPENING STATEMENT OF CHAIRMAN COLLINS
Chairman Collins. The Committee will come to order.
Good morning. Good morning particularly to all the children
who have joined us from around the country today. We are so
glad to have you here.
As one of the co-Chairs of the Juvenile Diabetes Research
Foundation's 2003 Children's Congress, I am very pleased this
morning to hold this hearing to examine the devastating impact
that juvenile diabetes has had on American children and their
families. In addition to hearing about the personal toll that
this disease imposes, we will also take a look at the
tremendous economic costs to our Federal health programs of
caring for people with diabetes. And, finally, we will discuss
the promising breakthroughs in juvenile diabetes research that
holds such hope for a cure. Particularly, we will examine
pancreatic islet cell transplantation.
The work that I have done in the Senate on behalf of the 17
million Americans with diabetes has been truly rewarding. It
has been a privilege to work with the families, with the
Juvenile Diabetes Research Foundation, whose commitment to
finding a cure for this serious disease is truly inspiring. I
want to welcome our distinguished witnesses today, but
especially the 188 delegates to the Children's Congress. They
have traveled to Washington from every State in the Union to
tell us what it is like to have diabetes, just how serious the
disease is, and how important it is that we fund the research
necessary to find a cure.
I particularly want to welcome the delegate from my home
State of Maine, 16-year-old Katie Halasz of Wells. Katie will
be testifying on our second panel this morning, and I am
looking forward to her testimony.
As the founder and the co-Chair of the Senate Diabetes
Caucus, I have learned a great deal about this disease and the
difficulties and heartbreak that it causes for so many American
families as they await a cure. Diabetes is a devastating,
lifelong condition that affects people of every race, every
age, and every nationality. It is the leading cause of kidney
failure, blindness in adults, and amputations not related to
injury.
A study released by the American Diabetes Association
earlier this year estimates that diabetes cost the Nation $132
billion in health care costs last year and that health spending
for people with diabetes is almost double what it would be if
they did not have diabetes.
When I met with Katie yesterday, she gave me a nice
photograph album telling about her family and her battle with
diabetes. And I was amazed when she showed me this picture of
just 1 month's supply of the medical devices and syringes and
insulin that she needs to keep healthy. And she told me that it
costs her family $2,000 a month for those supplies. That really
hit home to me, the cost of treating people with diabetes and
keeping children like Katie healthy.
The statistics are truly overwhelming, but what really
prompted me to begin working on this issue was meeting more and
more people like our delegates and their families today whose
lives have been forever changed by diabetes. And that is why I
want to say to all the children, it is so important that you
traveled here to Washington today to tell your personal
stories. You put the human faces on all of these statistics.
You are more effective than any adult lobbyist could ever be,
with the exception of, of course, Mary Tyler Moore. [Laughter.]
You help us focus on what Congress can do to better
understand and ultimately conquer this terrible disease. And I
know that the burden of diabetes is particularly heavy for
children and young adults with type 1 or juvenile diabetes.
Juvenile diabetes is the second most common chronic disease
affecting children. Moreover, it is a disease that you never
outgrow.
In individuals with juvenile diabetes, the body's immune
system attacks the pancreas and destroys the islet cells that
produce insulin. While the discovery of insulin was a landmark
breakthrough in the treatment of people with diabetes, it
unfortunately is not a cure. And people with juvenile diabetes
face the constant threat of developing life-threatening
complications as well as a dramatic reduction in their quality
of life.
But thankfully, there is good news for people with
diabetes. We have seen some tremendous breakthroughs in recent
years in diabetes research, and I am convinced that diabetes is
a disease that can be cured and will be cured.
[Applause.]
All of us have been encouraged by the development of the
Edmonton Protocol, an experimental treatment developed at the
University of Alberta involving the transplantation of insulin-
producing pancreatic islet cells. This protocol has been hailed
as the most important advance in diabetes research since the
discovery of insulin in 1921.
Of the 257 patients who have been treated, all have seen a
reversal of their life-disabling hypoglycemia and 80 percent
have maintained normal glucose levels without insulin shots for
more than a year. Amazingly, many of the transplant recipients
have even reported a reversal in some of their complications,
such as improved vision and less pain from neuropathy.
Earlier this year, I joined my colleague from Washington
State, Senator Patty Murray, as well as my colleague and co-
Chair of the Senate Diabetes Caucus, Senator Breaux, in
introducing the Pancreatic Islet Cell Transplantation Act of
2003. This will help to advance the significant research that
holds the promise of a cure for more than 1 million Americans
with juvenile diabetes. We now have 39 Senate cosponsors, and
you can help me get even more cosponsors in your visits to Hill
offices today.
Before turning to my colleagues for their opening
statements, I want to welcome Representative Diana DeGette of
Colorado, who I believe is here today with her daughter. Diana
serves as the co-Chair, along with George Nethercutt, of the
House Diabetes Caucus, and it was the House Diabetes Caucus
that motivated Senator Breaux and myself to establish the
Senate Diabetes Caucus. So I want to welcome the Congresswoman
and her daughter, Frannie, who is a delegate to the Children's
Congress, to the Committee hearing today.
Again, it is wonderful to have all the children here today.
I know that your slogan is ``Promise to Remember Me.'' I want
to tell you that when I see your faces, I know all of us do
promise to remember you and to fight for the research funding
that will produce a cure.
I would now like to call on my colleague, Senator Pryor,
for any opening remarks that he might have, and thank you for
joining us today.
OPENING STATEMENT OF SENATOR PRYOR
Senator Pryor. Thank you, Madam Chairman.
I want everybody in this room to know what a great national
leader we have here for your cause. She is doing great work.
Fantastic.
[Applause.]
I know that the Chairman and I share the sense that this is
what being in the Senate is all about: Trying to help people
out there around this country every day face their unique
challenges. And certainly juvenile diabetes is one of those,
and our thoughts and our prayers are with you.
One thing that I want you to know is that we on this
Committee are very sensitive to your needs and very attentive
to what you need. And, in fact, as the Chairman set this
meeting up, she wants to talk about examining the personal toll
on families; she wants to talk about financial costs to the
Federal health care system; and she wants to talk about
research progress toward a cure. And I tell you, she wants to
continue her great leadership on this, and she is doing a
fantastic job. And I just want to welcome everyone here to the
Senate and to this Committee. And I want to ask the children:
Has this been a good experience for you so far in Washington?
Has this been good? Good.
Well, I hope it is. And it is meaningful and it is
important, and you all are making a real difference for people
that have juvenile diabetes all over this country.
Now, the last thing I need to say before I step aside here
is: Who is here from Arkansas? That is what I need to know. All
right. Good. Great. Well, listen, maybe we will get to visit
afterwards.
But thank you for coming to Washington. Your cause is very
important. Mary Tyler Moore, thank you for being here. It is
always an honor to see you, and thank you for your leadership
on this. You do a great job to bring awareness to this and show
great courage on this.
Thank you very much.
[Applause.]
Chairman Collins. Thank you, Senator Pryor.
Senator Lautenberg, it is a pleasure to welcome you here
this morning as well.
OPENING STATEMENT OF SENATOR LAUTENBERG
Senator Lautenberg. Thank you very much, Madam Chairman,
and I commend you for holding this hearing today on juvenile
diabetes, and I welcome Mary Tyler Moore. We have seen you in
so many places, but we never get tired of seeing you, and we
are particularly pleased to have you here today with this
group.
As the children I am sure know, when they look at me, they
see a grandfather. Right? OK. Well, I have nine grandchildren,
and the oldest is 9 and the youngest is 2 months. And I love
them dearly, and I am always looking for some reassurance about
how they feel and how they are doing.
And when I look at you, I feel similarly a love and
affection for you, and I want you to be healthy, and I want to
do whatever we can to make sure of that. And our Chairman,
Susan Collins from Maine, deserves a vote of thanks for the
courage that she has displayed, and she and Senator Murray from
the State of Washington have put together a program to try and
get more money to fight this disease, to fight juvenile
diabetes, to see if we cannot find, if not a cure, some
significant relief in the process. And it is shocking to learn
that as many as a million Americans suffer from this
devastating disease, and roughly 35 children will be diagnosed
with juvenile diabetes each and every day.
The incidence of diabetes, type 1 and type 2, is on the
rise with enormous consequences for our society. We talk about
the cost, but the cost, no matter how many billions, is
insignificant compared to the discomfort and the danger, the
risk that children have when they get diabetes. And that is
what we are about. That is what we are talking about here
today. And that is why we are so glad to see each and every one
of you.
Madam Chairman, I ask that my full statement be put in the
record, and I would close with this: That those here, the young
people who are going to tell us their story, lend encouragement
for our government to spend more on research and try to find
out what causes it and what we can do about it. If I would pick
one of the wonderful assets, the values of this job of ours, it
is that we can sometimes do absolutely the right thing and try
to help people who have contracted a disease or who are sick.
Now we want to do what we can to make sure that we reduce that
possibility as much as possible.
And so we send our love and encouragement to all of you,
and we thank you for being here and for telling us how it is
that you live your lives. It might help us get more courage and
more energy about finding enough money to help you. Thank you
very much.
[Applause.]
Chairman Collins. Thank you, Senator. Your full statement
will be placed in the record.
[The prepared statement of Senator Lautenberg follows:]
PREPARED STATEMENT OF SENATOR LAUTENBERG
Madam Chairman, I commend you for holding today's hearing on
Juvenile Diabetes. As many as 1 million Americans suffer from this
devastating disease; roughly 35 children will be diagnosed with
Juvenile diabetes each and every day.
The incidence of Diabetes--type 1 and type 2--is on the rise, with
enormous consequences for our society. Diabetes already accounts for
more than $132 billion in health care costs each year. That number will
continue to rise until we can stem the tide of new cases and find
better treatments.
I commend Senators Collins and Murray for introducing S. 518, a
bill to promote pancreatic islet transplants so that the procedure can
become standard therapy covered by insurance. Clearly, there is good
reason for the people who suffer from Juvenile Diabetes and their
families to have hope.
I would make one observation: This year, Congress is completing the
5-year mission to double funding for the National Institutes of Health
(NIH). I was an enthusiastic supporter of that mission before I took my
``sabbatical'' from the Senate and am happy to support it now that I am
back.
The research community has called for an 8-10 percent increase in
NIH's budget for next year. But the President just wants a2.7 percent
increase--that's barely enough to cover inflation.
I don't know how we can tell the witnesses gathered here for this
hearing that we will have to slow down research.
But we will have to slow down research. Why? Because the
President's tax cuts are causing the budget deficit to explode.
What NIH, the National Institute for Diabetes and Digestive and
Kidney Diseases (NIDDK), the National Cancer Institute, and similar
groups do is a pretty good example of tax dollars at work finding new
treatments, cures, and preventions for all types of illnesses and
diseases. But we are told that it's more important to cut taxes.
President Kennedy said, ``To govern is to choose.'' I think we
ought to choose to invest in the research that will help our witnesses
live longer and better lives.
I want to welcome our witnesses, especially Mary Tyler Moore, who
needs no introduction, and the brave boys and girls who are going to
share their stories about what it is like to have Juvenile Diabetes.
Thank you, Madam Chairman.
Chairman Collins. Senator Bennett, a pleasure to welcome
you today.
OPENING STATEMENT OF SENATOR BENNETT
Senator Bennett. Thank you very much, Madam Chairman.
I recall a hearing in this same room with Connie Mack in
the chair as we began the determination on the part of the
Congress to double the amount of funding for NIH over a 5-year
period. And I was part of that effort. There were some in my
party who, after we had done it for the first year, then the
second year said to us, ``Well, why do we continue to give NIH
double-digit increases? Look how well they did last year, and
we have got serious budget problems. Let's cut it back to a
single-digit increase this year. Look how well they have
done.''
And we said no, we have to continue on the pattern of
double-digit increases for NIH if we are going to get to the
goal of doubling medical research over a 5-year period.
I am happy to say we stayed the course that was set in the
hearing in this room under Senator Mack. And we did, in fact,
double the amount of money going to medical research over that
5-year period.
So it is appropriate, Madam Chairman, that you are carrying
on in that tradition that Senator Mack set by now addressing
the question of the appropriate amount of research focused
particularly on diabetes. I am happy to report that in the
State of Utah, if I might get parochial--but that is what we do
around here, we have at the University of Utah some of the
finest research on the issue of diabetes going on anywhere in
the country. It is tied to the genealogical information that
the Church of Jesus Christ of Latter Day Saints maintains in
Utah, because diabetes has been determined to have genetic
ties. There is more information in the genealogical library
that the church maintains than anyplace else in the world. So
the people at the University of Utah, some of them have said to
me, ``I could go anywhere, I have the credentials that I would
be hired by any research institution anywhere in the world, and
I have chosen to come to Utah because of the genealogical
information that is available, and the leg up that this gives
us as we deal with diabetes.''
I have a particular interest in it. My grandfather died of
diabetes. So I appreciate very much the leadership that you are
showing with respect to this hearing.
[Applause.]
Chairman Collins. Thank you very much, Senator.
We are also pleased to have with us this morning as an
honorary member of the Committee for the day Senator Ben Nelson
of Nebraska. He is going to be introducing two of our delegates
today who will be testifying, but I also want to give him the
opportunity if he would like to make any opening comments.
OPENING STATEMENT OF HON. E. BENJAMIN NELSON, A U.S. SENATOR
FROM THE STATE OF NEBRASKA
Senator Nelson. Well, thank you very much, Madam Chairman,
and it truly is an honor to be here today under these
circumstances, recognizing the importance of the work that is
about to be done and the work that will have to be done in the
future.
It is my pleasure to be able to introduce the Bonness
family on the second panel, and so I will have more comments at
that time. But thank you very much for convening this hearing.
The cause is important, and the work is equally important.
Thank you very much.
[Applause.]
Chairman Collins. Thank you, Senator.
Our first witness this morning is Mary Tyler Moore. I have
a feeling that she needs very little introduction. Most
Americans know her well from her work, her distinguished career
on both television and in films and on the stage. But she also
plays a very special role for everyone in this room because of
her remarkably effective advocacy on behalf of individuals with
juvenile diabetes.
Ms. Moore serves as the International Chairman of the
Juvenile Diabetes Research Foundation, and it has been my great
pleasure to work very closely with her during the last few
years. Together, we have worked to almost triple funding for
diabetes research since 1996. I know that is making a big
difference. It would not have been possible without the
effective advocacy of Mary Tyler Moore and the JDRF.
So it is a great pleasure to welcome you back to testify
today, and please proceed with your statement. Welcome.
[Applause.]
TESTIMONY OF MARY TYLER MOORE,\1\ INTERNATIONAL CHAIRMAN,
JUVENILE DIABETES RESEARCH FOUNDATION (JDRF)
Ms. Moore. Thank you. Thank you all for that wonderful
welcome. Good morning. Before I begin my formal remarks,
Senator Collins, I would like to personally thank you for your
leadership and all your efforts on behalf of people with
diabetes throughout our country, including conducting these
hearings and co-Chairing with Senator Breaux the Senate
Diabetes Caucus. With your continued support, I know we will
together find a cure.
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\1\ The prepared statement of Ms. Moore appears in the Appendix on
page 42.
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Now, please let me offer the following testimony:
Chairman Collins and Committee Members and all, I am here
today as International Chairman of the Juvenile Diabetes
Research Foundation. I wish I could say that I am happy to be
here. But meaning no disrespect, I am definitely not. You see,
I have had juvenile diabetes for more than 35 years now, and I
am tired of it. I sincerely wish that I did not have to come
back, year after year, to seek your help. And I do. I have to
for myself, for everyone with diabetes, and most especially for
the beautiful and courageous children who are here with me
today.
Be certain, at JDRF we have never been more dedicated to
finding a cure for diabetes and its complications, and the
evidence suggests that we are truly close. Much of the progress
that has led us to what we hope and believe are the final
stages of our fight against diabetes is the result of your
support and effort. We greatly appreciate the good partnership
we have always had with Congress and every administration since
our founding. We are deeply grateful for the extraordinary
support you showed last year in passing the Special Juvenile
Diabetes Program. But as you can see, I am here again. Despite
all our accomplishments together, we must face the fact that
there are still significant hurdles that we need to overcome to
take the last necessary steps along our path to a cure.
Senators some of you have already met many of the 200
children, the delegates to JDRF's Third Children's Congress who
are here in Washington this week. And you will be hearing from
some and meeting others. You will do that later today. They are
passionate and eloquent spokespeople for the need for a cure.
But don't be misled by their drive, their energy, and their
unwavering commitment. They--and I--struggle every minute of
every day to do what happens naturally for people who don't
have diabetes--that is, to achieve a balance between what I
eat, the energy I expend, the amount of insulin I inject. For
most of you, blood sugar balance is automatic, as automatic as
breathing. But for people with juvenile diabetes, like me, it
requires vigilance 24 hours a day, 7 days a week, 365 days a
year.
Each of these children and I need to be a mathematician, a
physician, a personal trainer, and a dietitian all rolled into
one. We need to be constantly factoring and adjusting, making
frequent finger sticks to check blood sugar levels, and giving
ourselves multiple daily insulin injections just to stay alive.
Not to live life to its fullest, mind you; just to stay alive.
And it isn't easy. Even with the greatest of care and the
closest of personal scrutiny, like many children and adults
with juvenile diabetes, I find I am often unable to achieve
good balance. My blood sugars can go dangerously low or
frighteningly high. Yes, dangerous and frightening, because
serious lows can lead to seizures, coma, and death. And high
blood sugars can, over time, result in disabling and life-
shortening complications.
Some of you may already know the startling toll diabetes
takes. For example, diabetes causes over 40 percent of kidney
failures in our country that require dialysis or
transplantation. It is responsible for more than half of
amputations not associated with trauma. It is the leading cause
of blindness in adults--the leading cause. Moreover, I have
seen studies that say that virtually everyone with juvenile
diabetes shows evidence of heart disease by age 40--and,
further, that the pre-menopausal women with juvenile diabetes
have a more than 30 times greater risk of death from heart
attack.
Beyond the incomprehensible personal costs, consider the
economic burden that our country must bear as a result of this
disease. Diabetes costs this Nation over $130 billion every
year. This includes one out of every four Medicare dollars
spent. Taken altogether, it should be crystal clear why it is
urgent that we find a cure and find it as soon as possible.
Chairman Collins, you and your colleagues from both sides
of the aisle have always welcomed us warmly during the past
Children's Congresses. In our prior meetings, you have promised
to remember us when making decisions about funding for juvenile
diabetes research. Today, we are happy to acknowledge to all
who might listen that you are indeed keeping your promise. And
we thank you, not just as your constituents, but also as your
partners in a shared mission to find a cure.
JDRF is working closely with the NIDDK and its Director,
Dr. Allen Spiegel. Our joint task is to ensure that the dollars
provided by the Special Juvenile Diabetes Program are used to
fund projects that otherwise could not be done. And we are
confident that these projects will focus on what is needed to
rapidly acquire mission-critical knowledge, provide essential
research resources, and speed the application of advances for
the benefit of people with juvenile diabetes.
Madam Chairman, Senators, it is vital to remember that
because children and adults with juvenile diabetes and their
families never get a rest from their disease, we never rest in
our efforts to find and deliver a cure. And because JDRF is a
global cure enterprise, we are always looking beyond the
horizon to anticipate what must be done next to achieve our
ultimate goal--a world without juvenile diabetes. Our approach
has consistently focused on what is next. And let me give you
an example, looking at our four steps we have taken to get to
the point where we are today in islet transplantation.
Step one: In the mid-1990's, JDRF created a map of all that
is known about diabetes and identified the knowledge gaps and
obstacles to progress along the various paths to a cure. We use
this continuously updated map to guide us in efforts to push
scientific advances from the laboratory bench to the patient's
bedside.
Step two: This research mapping made it clear that islet
cell transplantation was a potential cure. So we are invested
in creating a global network of research centers to prepare and
distribute human islets for basic and clinical research.
Step three: As it became clear that research centers
throughout the world were now able to test islet cell
transplantation, JDRF substantially boosted our investments in
research to find a way to transplant these islets without the
need for toxic immunosuppressive drugs.
Step four: In 2000, when Dr. James Shapiro's group in
Edmonton reported the first major critical success in islet
transplantation, JDRF jumped to overcome the next obstacle
confronting transplant research, and that is the inadequate
supply of islets. So to create an unlimited supply of islet
cells for transplantation, we are leading a global effort to
support stem cell research. This is particularly necessary
because each year, there are fewer than 2,000 cadaver
pancreases donated. Yet well over 1 million Americans with
juvenile diabetes could potentially benefit from islet cell
transplantation.
Overall, we have made terrific progress. But we do continue
to look into the future and ask ourselves, ``What is next?''
And here is what we believe must be next, to speed a cure for
diabetes.
First, we need policies and regulations that encourage
organ donations and promote the retrieval of additional human
pancreases. As I said, supply is a major obstacle to making
islet transplantation a cure for all those with diabetes who
might benefit. I am grateful for the real leadership and vision
that you have shown by introducing the Pancreatic Islet Cell
Transplantation Act of 2003. This bill, which JDRF supports,
will provide regulatory incentives for organ procurement
organizations to retrieve additional pancreases. It will also
test, within Medicare, insurance coverage for islet transplants
for people with juvenile diabetes and kidney failure. I am
hopeful that Congress will pass the legislation this year, and
we are eager to work with you in achieving this goal.
Second, we need to work with the Senate and the President
to do things necessary to speed progress in stem cell research
and to provide ample opportunity for this research to
accomplish our shared goal: Finding ways to relieve the
suffering of millions of Americans. Even with increased organ
procurement, we will ultimately still face a critical shortage
of islet cells for transplantation. To meet this demand, I
believe we need to ensure that the United States can take its
proper place in the world as a leader in stem cell research and
the development of human stem cell-derived therapies, including
the creation of human islets for transplantation.
But, Madam Chairman, right now, due to the restrictions of
the current administration policy, this is just not happening.
I know the President worked hard to find a balance in his
policy. He clearly recognized the great potential of stem cell-
derived therapies to reduce pain and suffering of millions of
people with many diseases, including juvenile diabetes. And in
my heart, I know that he intended to make it easier, not more
difficult, to create therapies to treat human disease. But now,
nearly 2 years after his August 9, 2001, decision, researchers
tell us that the progress being made in stem cell research is
not as far along as it could be. The number of cell lines
everyone had hoped for--there were supposed to be more than
60--turns out to be closer to 10. Of these lines, only a few
are widely available for research. Perhaps more important, of
those approved lines, none can be used to develop human
therapies. That is right--none. The problem is that each of
those cell lines were established using mouse feeder cells, and
the threat of contamination makes them unsuitable for human
therapies.
Further, because of the current circumstances, the best and
brightest of young researchers in the United States are shying
away from the field of human stem cell research. So not only do
we have an insufficient number of cell lines to conduct the
necessary research, we have a potentially more devastating
deficiency of brain power.
Again, I am confident this was not the outcome the
President intended. His staff, Secretary Thompson, and Dr.
Zerhouni are all doing what they can within the current
constraints. And we are pleased to be working closely with
them. But at the end of the day, without some change, we may
not be able to achieve our shared goals: Finding cures for
juvenile diabetes and other disabling diseases as soon as
possible.
Third, we need to continue to invest in developing methods
to replace human islets without immunosuppression. Just 3 years
after the reported success of the Edmonton Protocol, more than
250 people have undergone islet transplantation and no longer
need insulin injections to survive. But the procedure is not
yet safe for children and most people with diabetes. So we must
sharpen our research to focus on transplantation without toxic
immunosuppression, as well as redoubling our efforts to prevent
diabetes and reverse or prevent its devastating complications.
Finally, because we want a cure for everyone with juvenile
diabetes, including the children here today, we need to
establish a framework for research oversight which ensures that
the needed clinical studies are safe for the participation of
children.
Madam Chairman, diabetes is an all-too-personal time bomb
which can go off today, tomorrow, next year, or 10 years from
now. A time bomb that affects millions, including me and the
children here today. It needs to be defused. But to find a cure
for diabetes and its complications, and then make these cures
available to everyone who might benefit, will require that we
remain vigilant in our purpose and continuously committed to
asking, answering, and acting upon the ``What next?'' question.
So what is next for you? Please listen to the children this
morning, who will tell you how they struggle with juvenile
diabetes. Learn about how they bravely face its daily
challenges--challenges that no 15-year-old or 8-year-old or 2-
year-old should have to endure. Feel their longing to know a
day without diabetes and live the normal, carefree life of a
child. Listen to the researchers as they highlight the progress
made to date and the exciting opportunities we can now realize
because of JDRF and NIH leadership and the extraordinary
investment Congress has made in medical research. And hear
firsthand from an islet transplant recipient about how
spectacularly her life has changed since her procedure and how
she will not rest until her young son, who also suffers from
juvenile diabetes, can be cured.
Finally, please join me in making a personal promise to
remember what we have learned today. Ask ourselves: What must
we do next? And then commit together to do what we must to find
a cure.
Thank you once again for all you have done and for the
opportunity to speak with you this morning.
[Applause.]
I cannot tell you how touched I am by that reception. Thank
you.
Chairman Collins. Well, thank you for your testimony. I
think every member of this panel joins everyone in this room in
remarking on your eloquence in telling your own personal story
as well as your advocacy. Your willingness to share your
personal story as well as your leadership at the Juvenile
Diabetes Research Foundation makes a real difference. It
inspires each and every one of us.
As you spoke, I could not help but remember the first time
I met a family whose son had diabetes from my home State of
Maine, and the little 10-year-old boy looked up at me, and he
said, ``I just wish that I could take one day off from my
diabetes.'' And that is when I knew that I had to get involved.
So thank you for presenting your story.
I am going to limit questions on this round so that we can
hear from the children, so let me just ask you one question.
Two years ago, you came to Congress to testify at the
Children's Congress. Are you more hopeful today as a result of
the investment in research and the medical advances that we
have seen that we are on the verge of providing a cure and
better treatment for people with diabetes?
Ms. Moore. Senator Collins, I most definitely am more
enthusiastic and stronger in my belief that a cure is,
figuratively, around the corner. It cannot come soon enough for
all of us, I know that, but especially because of the promise
of stem cell research. I think that is the big hope. I think
that it will make a huge difference to the people who suffer
from all kinds of diseases. And won't it be lovely to have all
that money that we don't have to spend taking care of people
with horrible illnesses and can spend it on things that make us
a better, healthier, happier Nation.
Chairman Collins. Thank you.
[Applause.]
Senator Pryor.
Senator Pryor. Thank you. I know that you are more
encouraged today than you have ever been, but what do you see
as the largest obstacle to finding a cure today?
Ms. Moore. I think any roadblock that is thrown in the way
of the good scientists who are working to find the cure. I
think fear is a major obstacle. And from good guidance
sometimes we recognize fear and have to put it in its place and
take the chance and move forward.
Senator Pryor. Are you comfortable with the funding levels
that NIH and others are providing for the research going on
right now?
Ms. Moore. We are very grateful for all the advances made
possible by the government and by private donors who have done
whatever they can to bring this to the attention of government
and the people.
Senator Pryor. I am not as familiar with the islet cell
transplantation like the Chairman is here. Do I understand that
there are some people who are very good candidates for that and
others that maybe are not so good? Could you educate me on that
very briefly?
Ms. Moore. Well, mostly it is children who are not good
candidates for it right now. That may change, but in all, the
overwhelming lack of islet cells is the major problem. There
are over a million people who could benefit from a pancreatic
islet cell transplant, but they cannot because there are not
enough of them. There just are not enough, and it costs too
much. It is approximately $100,000 per operation. And the
immunosuppressants that one must take in order to receive and
make functioning the islet cells are a tough thing to live
with. Very tough.
Senator Pryor. Right. Thank you again for being here today.
Ms. Moore. Thank you.
Chairman Collins. Thank you, Senator. Senator Bennett.
Senator Bennett. Thank you, Madam Chairman. I have nothing
to add.
Chairman Collins. Thank you. Senator Lautenberg.
Senator Lautenberg. Thank you, Madam Chairman, for holding
this hearing. We can sense from the reaction of people here
whose lives are affected by juvenile diabetes a sense of
interest and encouragement and even gratitude for what has
happened. We thank Mary Tyler Moore for her eloquent statement
and for information that frankly those of us--and I include
myself in this--who have been spared contact, direct contact
with juvenile diabetes fail at times to understand the impact
of this disease. When you talk about a child in a lifetime
having 50,000 injections prospectively, it is a painful
prospect. And if we could wave a magic wand, we certainly would
do it.
I would ask you this, Ms. Moore: What do you think we can
do on a relatively immediate basis to speed up the process for
the development of islet and other technologies that we know
something about?
Ms. Moore. I think continue to fund the research, just get
in back of us 100 percent, support stem cell research in
particular.
Senator Lautenberg. How about the expansion of stem cell
availability? Would that make a big difference, do you think?
Ms. Moore. The expense of stem cell----
Senator Lautenberg. Expanse.
Ms. Moore. Expanse. It is possible. It is there to be done.
But we need the permission from our President to advance, to do
the things that they are doing in other countries.
Senator Lautenberg. That would give a lot of encouragement
to people, wouldn't it?
Ms. Moore. It certainly would.
Senator Lautenberg. Thank you very much.
Ms. Moore. Thank you.
[Applause.]
Chairman Collins. Senator Specter.
OPENING STATEMENT OF SENATOR SPECTER
Senator Specter. Thank you very much, Madam Chairman, and
thank you for convening this hearing. And thank you, Ms. Moore,
for your leadership for many years on this important subject.
You have appeared in this room on a number of occasions
testifying before the Subcommittee on Labor, Health and Human
Services, and Education. And as a result of your efforts and
the efforts of others, the funding in the National Institutes
of Health has been raised for the National Institutes on
Diabetes and Digestive and Kidney Diseases to $1.633 billion,
and the total diabetes funding is at $845 million. That is as a
result of the increases on NIH funding from $12 billion to
$27.5 billion in the past several years.
The issue of fear which you commented on and the question
raised by Senator Lautenberg on stem cell research is a very
important one, and the reality is that the hands of the
scientists are being tied or at least efforts are being made to
tie their hands, and there is a lot of concern and a lack of
initiative in the scientific community because of legislation
passed by the House of Representatives which criminalizes
research on stem cells, which some people have characterized as
``therapeutic cloning.''
That is a term which raises a lot of emotion, erroneously.
We are all against human cloning. But when you talk about
therapeutic cloning, it is really not true. The essential
medical procedure is that an egg is donated, the core is taken
out, and then if a woman, for example, has Parkinson's or
Alzheimer's or juvenile diabetes, a skin sampling is taken,
placed in the core. It is kept in a laboratory dish, and those
stem cells then are consistent to be used for that patient.
The entity dies within 14 days so there is nothing cloning
about it at all.
Ms. Moore. Right.
Senator Specter. And I believe that it is important to use
every opportunity to acquaint Americans that about 128 million
are suffering from one of these maladies which might be curable
by additional stem cell research. And juvenile diabetes is
among them.
So thank you for all that you have done, and I thank all of
you for coming to this proceeding today, especially the
children, and there are many others on Capitol Hill who are
dedicated to scientific research additional funding and to
finding a cure for juvenile diabetes and other dreaded
maladies.
Thank you, Madam Chairman.
Ms. Moore. Senator, we thank you.
[Applause.]
Chairman Collins. Senator Durbin.
OPENING STATEMENT OF SENATOR DURBIN
Senator Durbin. Thank you very much, Madam Chairman, and
thank you, Mary Tyler Moore, for what you have done and for
your leadership, and thanks to all of your young supporters
here who are starting to move a little bit here. They want the
Senators to get moving with the questions.
I want to refer to one aspect of this. Another one of my
``sheroes'' in life is a lady named Connie Payton from Chicago,
whose late husband, Walter Payton, really made a plea toward
the end of his life for organ donation. And she has continued
in that fine work, and we have engaged the National Football
League to make this part of their public service. And we have
focused on Thanksgiving, the great national holiday, to
encourage families when they come together to spend just a few
moments with one another and let other members of the family
know if you want to be an organ donor.
It seems to be one of the major obstacles for organ
donation, that the family members don't know what you feel in
your heart about organ donation. And as I read these amazing
statistics which you have brought to us about how--and, in
fact, you need really two donors, do you not?
Ms. Moore. Yes.
Senator Durbin. For the pancreata that is necessary for
this new approach. It really dramatizes the enormous demand and
need for organ donors in our country as part of this effort.
And if the Chairman will give me a moment here, I would
just like to do a little show of hands here, and the children
are exempt from this vote. They are used to raising their hands
in classrooms. But this is for all of the adults in the room,
Senators and staff and audience. How many people in this room
have communicated to members of their family that they would be
organ donors or have signed a organ donation card? Good. This
is an audience that is not typical of America because you
understand this. And I hope that this hearing, among other
things, spreads the word across America. We need more organ
donors for so many good reasons and for these children. And I
hope, Madam Chairman, that is a message that we send from this
hearing as well.
Thank you very much.
[Applause.]
Ms. Moore. Thank you so much, Senator.
Chairman Collins. I want to thank you very much for
testifying today. Senator Nelson is going to start off with the
introduction of our next panel when I bring them up here. Thank
you so much, Ms. Moore, for all that you are doing. Because of
your eloquent testimony and your leadership and advocacy year
after year, we have been able to make a real difference in
funding research for diabetes. And I know we are going to
continue our great partnership, so thank you so much for being
here today.
Ms. Moore. Thank you, Madam Chairman.
[Applause.]
Chairman Collins. I would now like to call forward the
children's panel. Our second panel of witnesses this morning
consists of children who know firsthand the burden of living
with diabetes. Our witnesses on this panel are Colleen Rea of
Stamford, Connecticut, accompanied by her son, Dylan; Sophia
Cygnarowicz of Columbia, Illinois, whom Senator Durbin may wish
to introduce; Katie Halasz of Wells, Maine; Eric and Alex
Bonness of Omaha, Nebraska; and LaNiece Evans-Scott of
Backlick, Ohio. And we are just delighted to have those
children with us. They are all delegates to the Children's
Congress, and we are going to start by having Senator Nelson
introduce the Bonness children.
Senator Nelson. Well, thank you very much, Madam Chairman.
It really is an honor for me to be here today to introduce some
fellow Nebraskans. I would like to thank you, Madam Chairman,
for not only this opportunity but for your leadership. It is
very clear that your commitment is making progress possible,
and we all thank you very much. I know all the families who
have been touched by this disease appreciate what you are
doing, and we hope that as a result of this hearing today we
will be able to advance the efforts toward a cure.
I would like to start off this morning, before I introduce
the two delegates, by introducing a gentleman who is well known
in Nebraska, not simply as a former football player, all-
American at Nebraska, and an NFL player, coached by one of our
colleagues in the House, former coach but now Representative
Tom Osborne, but a person who has taken this cause not only
seriously but to the far reaches of our country to make sure
that we are all aware. He has advanced the recognition of this
disease. He has advanced the efforts to make sure that we are
all working every way that we can to help this cause.
Today, he joins us as the Chair-dad of the Children's
Congress, and I could list all the accomplishments on the
gridiron and in the courtroom as a lawyer. But I just simply
want to say to Eric Bonness, thank you very much, and please be
recognized this morning.
[Applause.]
He is joined by a very able partner. Dr. Bonness, it is
good to have you here as well.
[Applause.]
As we all turn our attention to the two young
representatives, the two young delegates here, I think that I
can speak on behalf of their parents and all of us who would
say that with all the recognition and all the accomplishments
of their parents, their parents and we all hope that they will
be able to exceed those accomplishments in their lives ahead. I
speak, of course, today of Alex and Eric Bonness. They are two
of our Nebraska delegates and two of Nebraska's finest young
people. Both are extraordinary young men. They were diagnosed
with type 1 juvenile diabetes before they entered their teens.
Eric was diagnosed when he was 10, and Alex, although he is the
younger brother, he is the elder when it comes to living with
his disease because he was diagnosed when he was only 4.
But for every day since those fateful days that they were
diagnosed, these two have measured their blood sugar, taken
medication, and, in general, have had to monitor their health
in ways that most adults could not imagine, certainly with the
level of care that few could match. But they, like so many
others like them, have done this day in and day out because
they simply have to.
I will let them fill you in on what this entails for them,
but I am sure my words could never match theirs. I do want to
say, though, that the kind of courage that they have shown from
the time they were young children in dealing with this disease
has to be an inspiration for all of us, and it has to be
encouragement for all of us to do everything that we can, as
adults, as Members of the Congress, and as human beings.
That inspiration should lead us to work with the Juvenile
Diabetes Research Foundation in finding a cure for diabetes so
that Alex and Eric and all the young people in this room and
all the young people out there who live with this disease every
day will not have to live with it any longer. That is what our
hope is all about today. That is what our dedication must
bring. And I only hope that these young men who come to us
today, who have had to learn words like ``glaucoma,'' ``retinal
scans,'' and ``diagnostics,'' when they should have only had to
learn about their favorite action figures or video games, they
have met the challenges of juvenile diabetes, and we need to
join them in the challenge of finding a cure.
So it is an honor for me today to host Eric and Alex and
their family, as well as Nebraska's other two delegates who are
in town but will not be testifying, Megan Stewart and
Alyxandria Harter. It is my pleasure to present Eric and Alex
Bonness.
[Applause.]
Chairman Collins. Senator Durbin.
Senator Durbin. Thank you very much, Madam Chairman. There
are a number of Illinois children delegates here, and if they
would either raise their hand or stand up, and I am going to
introduce Sophia at the end here: Sean Bottorff from St.
Charles, there he is; Alexandra Case of Chicago; Kevin
Covarrubias of Maryville; Devan D'Silva, Arlington Heights;
Michael Johnson of Evanston; and Abigail Wolter of Alton. Thank
you.
And the person I would like to especially introduce has
just successfully completed the first grade. She is here today
from Columbia, Illinois. Sophia Cygnarowicz is here to tell us
a little about her experience with juvenile diabetes. She said
she would like to know what life is like without diabetes, and
she would like to eat Sno-Cones whenever she wants. [Laughter.]
We are looking forward to her testimony. Thank you, Madam
Chairman.
[Applause.]
Chairman Collins. Thank you. Senator Lautenberg.
Senator Lautenberg. Thank you, Madam Chairman. I want to
call attention to the wonderful young people who have come from
New Jersey. They don't have as long a trip geographically as
some of the others, but their trip to getting here is the same
perilous journey. And we want to say hello, and I will ask the
same thing, that a hand be raised for Jessica Barszcz--Jessica,
are you here? Yes, OK. Welcome. Kyle Gertner. Emily Greatrex.
Is Emily here? Oh, a beautiful child from New Jersey. Lindsey
Rosenthal. Hi, Lindsey. And Carey Towell. Carey, hi. Well, we
are happy to see these youngsters and tell them that we are
going to work hard to see that life gets better for them and
their friends as well.
Thank you.
Chairman Collins. Thank you.
We are going to start with Katie Halasz today because she
is from my home State and I am the Chairman. [Laughter.]
I got to meet Katie and her Mom yesterday in my office, and
she gave me a photograph album illustrating what it has been
like for her since she was diagnosed. She is 16 years old. She
is from Wells, Maine. This photograph album is just wonderful.
It tells about what she felt. It shows what she has to do each
day to monitor her blood and to give herself insulin. It was
very helpful, and I really treasure it.
So, Katie, please begin. Bring the mike up close to you so
we can hear you. Thank you.
TESTIMONY OF KATIE HALASZ,\1\ DELEGATE, AGE 16, JDRF CHILDREN'S
CONGRESS, WELLS, MAINE
Ms. Halasz. Unlike Dylan and Sophia, I do know what it is
like to live without diabetes. I dream of another day without
diabetes. My name is Katie Halasz. I am 16 years old and from
Wells, Maine. I want to tell you how my life changed on June
12, 1999, at 11 p.m. when I was diagnosed with juvenile
diabetes.
---------------------------------------------------------------------------
\1\ The prepared statement of Ms. Halasz appears in the Appendix on
page 48.
---------------------------------------------------------------------------
At first, I did not know what diabetes was. The doctor told
me I would have to take insulin shots the rest of my life. I
did not think that diabetes would change my life that much,
but, boy, was I wrong. I have to test my blood sugar and take
shots all the time. It affects everything I do, even being part
of the color guard for the school band is a challenge. My blood
sugars often go high and result in ketones, which is poison in
my body, and it can be really dangerous. When you get ketones,
you cannot do any exercise. It is very hard on my team and on
me when I cannot march.
School is a lot harder when you have diabetes. My school
does not allow me to test my blood in the classroom. My classes
are on the third floor, and the nurse's office is on the first
floor. When I feel low, I have to walk down three flights of
stairs, and this is very dangerous. Some of my teachers think
that I leave the classroom just to get out of class. Do they
think I enjoy sticking a needle in my finger? When my blood
sugars are high, I cannot concentrate on my work and my vision
gets blurred. My teachers do not understand. Some of them even
think it is my fault that my sugars go high because they think
that I eat things like candy bars. They do not understand that
my sugars can go up for no apparent reason. Educating my
teachers has been my biggest challenge since being diagnosed
with diabetes.
Having diabetes complicates more than just school. Last
year I had a cyst underneath my arm. It became infected. I went
to see a surgeon once a month to have the area opened so it
could be drained. My sugars were always high because of the
infection, and the area would not heal because of the high
sugars. Finally, I had to have surgery to remove the cyst. The
doctors had to remove half the tissue from my underarm.
Senator Collins and Members of this Committee, we are here
today to ask for your help in finding a cure for juvenile
diabetes. Each of us wants to be able to go to school, play in
the band or on sports teams, without worrying about going into
a coma because of low blood sugars. Each of us wants to grow up
without the daily stress of trying to treat this horrible
disease and the fear of complications. We will do our part to
educate policymakers about diabetes and raise money to support
research. But we need you to be our partner in this effort. We
cannot do it alone.
Senator Collins, I am glad and grateful you are my Senator.
You have shown incredible dedication and leadership in our
common quest for a cure. Thank you from the bottom of my heart.
[Applause.]
Chairman Collins. Thank you, Katie. You did a great job.
We are now going to hear from Dylan Rea, but we are
actually going to hear from his mother, Colleen, since Dylan is
age 4. So, Colleen, would you please proceed?
TESTIMONY OF COLLEEN REA, ON BEHALF OF HER SON, DYLAN REA,\1\
DELEGATE, AGE 4, JDRF CHILDREN'S CONGRESS, STAMFORD,
CONNECTICUT
Ms. Rea. Good morning. My name is Colleen Rea, and I am
from Stamford, Connecticut, and it is an honor to appear before
you today with my 4-year-old son Dylan. Dylan and I are here
today, along with all of these children, to tell you why
finding a cure for juvenile diabetes is so important. You see,
I know all too well about the devastation of diabetes. Diabetes
has followed me and haunted me my entire life for three
generations of my family.
---------------------------------------------------------------------------
\1\ The prepared statement of Ms. Rea appears in the Appendix on
page 50.
---------------------------------------------------------------------------
When I was a child, I found glass syringes in my house and
was told that my grandfather, who died before I was born, had
diabetes. When I was a young woman, my mother called and asked
me to meet her at a hospital emergency room. My mother was
being admitted for the first time because of her diabetes. When
I saw her, she was scared. Within 10 years and many hospital
stays and operations later, I was told that my mother was
dying. It took only 10 years for diabetes to kill my mother.
When my son Dylan was diagnosed with type 1 diabetes, or
juvenile diabetes, at the age of 14 months old--less than 2
years after my mother had died--I was devastated. It was almost
more than I could bear. I knew what diabetes had done to my
grandfather, and I had seen what diabetes did to my mother.
We are very diligent in trying to care for Dylan's
diabetes. But controlling blood sugar levels in a toddler is
impossible because there are so many variables. There are
always times when Dylan's blood sugar is dangerously high or
frighteningly low, sometimes in the same day. He wears an
insulin pump 24 hours a day, and we check his blood sugar at
least eight times a day. And there are precious few people who
are able to babysit him. My husband and I must be available 24
hours a day, 7 days a week. Diabetes never takes a break, and
neither can we. We are Dylan's pancreas.
While we are concerned about Dylan's physical health, we
also worry about his emotional health. Dylan may grow up to
live a long life, have a great and fulfilling career, and a
wife and children of his own someday. But we know that in our
hearts, despite our best efforts, he may be denied all of that.
Either way, he has this day and this childhood, and I want it
to be wonderful.
We walk a tightrope in caring for Dylan, to help our child
feel love and joy when we continually assault his body with
needles and lancets. We give our child hope and faith in the
face of fear and disease. We tell Dylan he is healthy but needs
medicine every day, all day, to survive.
I know that a cure is possible and within our reach. I hope
that it will be found soon so that Dylan and all of these
children will live a long and fulfilling life. This is my
greatest wish and why we are here today to ask for your help
and support in our quest for a cure.
Say thank you.
Master Rea. Thank you.
[Laughter/applause.]
Chairman Collins. Good job, Dylan. That was great.
Thank you very much, Colleen, for your very moving
testimony.
We are in the middle of two roll call votes, so I am going
to have Sophia testify, and then we are going to have a 15-
minute recess before we come back and hear from Eric and Alex
and LaNiece. Sorry about that. We cannot control the schedule.
Sophia, could you give your statement to us?
TESTIMONY OF SOPHIA CYGNAROWICZ,\1\ DELEGATE, AGE 7, JDRF
CHILDREN'S CONGRESS, COLUMBIA, ILLINOIS
Miss Cygnarowicz. Hi. My name is Sophia, and like my friend
Dylan, I don't know what life is like to have a day without
diabetes. I am 7 years old. I have had diabetes since I was
one.
---------------------------------------------------------------------------
\1\ The prepared statement of Miss Cygnarowicz appears in the
Appendix on page 52.
---------------------------------------------------------------------------
I have taken 4,380 shots of insulin and have pricked my
finger over 13,000 times to test my blood sugar. I don't like
it. It hurts. It is so hard to keep my blood sugar in a good
range. No matter how hard I try, I still go low and high.
Low blood sugars make me very tired and cranky. I need to
eat but a lot of times I don't want to. Sometimes I wake up in
the middle of the night because I go low. My Mom and Dad will
feed me and test my blood sugar to make sure I will be OK
before I go back to sleep.
I just finished first grade. It is hard going to school
when you have diabetes. We did cooking projects and had lots of
parties. I watched all the other kids eat cookies and cake. I
couldn't eat them, and that wasn't fair. When I feel low at
school, I can't think well. My teacher gives me sugar tablets,
and I walk down to the nurse's office to do a blood sugar test.
A friend comes with me to make sure I get there OK. Then I have
juice and crackers. It takes me a while before I get better. I
don't like to miss class.
Summer is lots of fun, but not when I go out and can't stay
outside and play with my friends. I have to eat at the same
time every day so my blood sugar won't go too low. I have to
eat even when I am not hungry.
I don't know what life is like without diabetes, but I sure
would like to find out. Finding a cure is important to me
because I won't have to take shots or do blood tests. Most of
all, I could eat a Sno-Cone whenever I wanted to. My friends in
this room and I aren't asking for much. We just want a life
without diabetes.
[Applause.]
Chairman Collins. Thank you, Sophia. You did a wonderful
job talking to us and helping us understand what it has been
like for you to live with diabetes, so thank you for a great
job.
We are now going to have to recess for 15 minutes, and then
we will come back and hear from the three remaining children.
Thank you.
[Applause.]
[Recess.]
Chairman Collins. The hearing will come to order.
I am very pleased now to recognize Eric Bonness for his
testimony. Eric and his brother Alex are going to be testifying
next. And then we will hear from LaNiece Evans-Scott, who has
been very patient at the end of the table. So, Eric, would you
please give your testimony.
TESTIMONY OF ERIC BONNESS,\2\ DELEGATE, AGE 18, JDRF CHILDREN'S
CONGRESS, OMAHA, NEBRASKA
Mr. Eric Bonness. Having diabetes is not something you
struggle with alone. It affects your whole family. If you have
diabetes, it affects everything you do. If you also have a
brother with diabetes, it affects everything--times two. Our
family has twice as many blood glucose kits and twice as many
insulin pumps, more than $10,000 worth. We do twice as many
blood sugar checks and change catheters twice as often. We have
twice as many blood sugar highs and lows. We have twice the
costs--economic, physical, and emotional.
---------------------------------------------------------------------------
\2\ The prepared statement of Mr. Eric Bonness appears in the
Appendix on page 54.
---------------------------------------------------------------------------
My name is Eric Bonness. I am 18 years old and from Omaha,
Nebraska. I was diagnosed with type 1 diabetes when I was 10, 3
years after doctors diagnosed my little brother, Alex, with
diabetes. I did not have to go to the doctor's office to be
told I had diabetes. I found out one morning in my bedroom
after I had been up all night going to the bathroom and
guzzling water. Just before dawn, I opened my eyes as my
parents sat down on my bed holding my little brother's blood
glucose meter. For 3 years I had seen the fear--and the tears--
in the eyes of my little brother as he endured thousands of
finger pokes and insulin shots. I knew I was about to get my
first finger poke to test my blood sugar. I was terrified. I
had always been afraid for Alex. Now I was afraid for me. The
doctors said there was only a small chance I would ever get
diabetes. Suddenly, statistics did not matter.
My mom poked my finger. Then my parents and I watched in
silence as the seconds ticked down on the blood glucose meter
to reveal my blood sugar. The numbers seems to explode off the
screen at me--495. I asked my mom if I had diabetes, but I
already knew the answer.
Like any brother, Alex can be a real pain. [Laughter.]
But he has always helped me with the pain of diabetes. When
I was first diagnosed, Alex would get candy for me when I had a
low blood sugar. He also taught me how to give myself insulin
shots to bring down high blood sugars. Now that we are older,
we still help each other recover from insulin reactions. We
give each other test strips when one of us runs out. We even
borrow the other's insulin pump when ours stops working.
It is scary enough to have diabetes myself. But it is even
worse to watch my little brother suffer with diabetes. Alex
sometimes has a low blood sugar and migraine headache at the
same time. He becomes semiconscious, incoherent, and unable to
help himself. I watch my parents struggle as they try to raise
his blood sugar before he slips into a coma. We are lucky my
mom is a doctor. Other families would have to go to the
hospital. During these episodes, my parents give Alex an
emergency IV or shot of glucagon to raise his blood sugar
level. It is horrible to see diabetes make my brother so
vulnerable. If diabetes is doing this to him now, even with the
best available medical care, what is diabetes going to be doing
to him in the future?
I am not going to wait to find out. I start college next
year and I plan to go on to medical school. We have to find a
cure, and soon. Diabetes is not going to kill my brother, and
it is not going to kill me.
We need your vote to keep our hope alive. Thank you.
[Applause.]
Chairman Collins. Thank you very much. Eric, you should see
your parents' faces right now. They are so proud of you, and we
all are. Thank you for your testimony.
Alex, now you get to take your revenge on your brother for
that ``pain'' comment, right?
TESTIMONY OF ALEX BONNESS,\1\ DELEGATE, AGE 15, JDRF CHILDREN'S
CONGRESS, OMAHA, NEBRASKA
Mr. Alex Bonness. My name is Alex Bonness. I am 15 years
old and Eric's younger brother. I was diagnosed with type 1
diabetes when I was 4 years old. I don't remember the day I
found out I had diabetes, but I do remember being really scared
of the finger pokes and shots. When I was diagnosed, no one on
either side of my family had type 1 diabetes, so living with it
was a real big shock to us. I remember feeling lost, confused,
and alone. Some nights I would throw tantrums and cry when it
was time to go to bed. I never talked about why I did it. My
parents thought it was because of the monster in my closet.
Boy, were they wrong. I was afraid of a monster all right, but
it was not in my closet. It was diabetes.
---------------------------------------------------------------------------
\1\ The prepared statement of Mr. Alex Bonness appears in the
Appendix on page 56.
---------------------------------------------------------------------------
When I was 7, I felt like I was saved. That is when Eric
was also diagnosed with diabetes. I know it sounds terrible to
say I felt like I was saved because my brother was diagnosed
with diabetes. How could such a bad thing become good? I was
sad Eric had to go through so much pain, but somehow I no
longer felt alone. When Eric reached out to me, I made sure I
was there for him. Don't get me wrong. Eric can be a real pain.
[Laughter.]
But it was important for me to be there for him.
As Eric and I have grown up together, we have always told
each other we can beat diabetes. I know we can. But as I get
older, my fears grow, too. When Eric hit his teenage growth
spurt, I watched his uncontrollable blood sugars skyrocket to
high levels because of his changing hormones. It scared me to
death. I worried about him.
Now I am hitting my teenage growth spurt, and I worry about
me. I have cried myself to sleep. I have also cried with my mom
about what scares me most, like complications such as blindness
and having my legs amputated. Mom always tries to comfort me.
But she and I both know that complications could occur at any
time.
For example, I have to go to the eye doctor every year. At
my recent eye appointment, I sat on pins and needles with every
word my eye doctor said. She saw no signs of complications--
yet. On the way home, I thought about what I would have said
and done if she had seen blood vessels in my retina starting to
deteriorate.
I am not a little kid anymore. And I know my brother can't
save me from going blind. But I know research can. And you can
make that happen.
Thank you.
[Applause.]
Chairman Collins. Thank you.
Eric and Alex, thank you so much for your testimony. I
cannot imagine how hard it must be to have one child with
diabetes. To have two must be so difficult. But I am so glad
you have each other and that you can help each other.
LaNiece, you have been very patient today, and I am looking
forward to hearing what you have to tell us. So it is your turn
now.
TESTIMONY OF LANIECE EVANS-SCOTT,\1\ DELEGATE, AGE 10, JDRF
CHILDREN'S CONGRESS, BACKLICK, OHIO
Miss Evans-Scott. Many of the kids here today, like my
friend Alex, worry about getting complications from juvenile
diabetes when they get older. I am here to tell you that some
of us, like me, already have serious complications from
juvenile diabetes. I am LaNiece Evans-Scott from Backlick,
Ohio. I am 10 years old, and I have battled juvenile diabetes
my entire life.
---------------------------------------------------------------------------
\1\ The prepared statement of Miss Evans-Scott appears in the
Appendix on page 58.
---------------------------------------------------------------------------
I was diagnosed with juvenile diabetes after going into a
coma when I was only 16 months old. I spent the first 5 years
of my life in and out of hospitals. My mom had to learn how to
give me shots and take care of me. She has not been able to
work because she has to take care of me all the time. She
drives me 5 hours to see a special doctor who helps me care for
my diabetes.
I already have problems with my kidneys, and I take
medicine every day so that my kidneys won't fail. I worry about
what will happen to my kidneys and what other complications I
will face as I get older if a cure is not found soon. I am also
blind in one eye, not from diabetes, but I worry about what my
diabetes will do to my other eye.
I have a lot of trouble in school because the teachers send
me home when my blood sugar levels are bad. I am not bad. I am
good. It is the diabetes that is bad. I do the best I can, but
I am only a kid and diabetes is a horrible disease. I have
fallen behind in school because I miss so many classes. I like
school, and I also like bowling. I have a lot of friends, and I
am on the bowling team.
I used to be shy about having juvenile diabetes, but I have
learned that I must tell my story to important people like you
so you know how serious diabetes is and why a cure is
important. I want a cure so that I can be like my friends and
go to sleepovers and birthday parties. I want a cure so I can
go to school every day without worrying about being sent home.
I want a cure so my mom won't worry so much about me and can go
back to work.
I don't have time to wait. Please promise to remember me.
Please promise to remember us. Thank you.
[Applause.]
Chairman Collins. Thank you, LaNiece. You did a great job
helping us better understand what having diabetes has been like
for you.
I understand that each of you, in fact, all of the
delegates in this room to the Children's Congress were selected
from more than 800 applicants on the basis of a special letter
that you wrote on why getting a cure for diabetes is important
to you. So I want to congratulate not only those children who
testified, but every single child who is here today, because
you won the right to come to Washington to help us better
understand what having diabetes is like and why we need to work
really hard for a cure. So congratulations to each of the
children here today.
[Applause.]
Now, I am just going to ask you a couple of questions.
LaNiece, we will start with you. What is the hardest thing
about having diabetes for you?
Miss Evans-Scott. Taking shots.
Chairman Collins. Taking the shots. How about you, Alex?
Mr. Alex Bonness. I would say putting in catheters, and I
guess living with it every day, not being able to do what other
kids do.
Chairman Collins. Eric, how about you?
Mr. Eric Bonness. I would have to say that no matter how
careful you think you are, how much care you are taking with
your diabetes, your sugars are always off. There is always
something that--you can never have total control, which means
you are going to end up having complications. So just the fear
of not being able to fully be in control of this disease is
probably the most scary thing.
Chairman Collins. Katie, what is the hardest thing for you?
Ms. Halasz. I think taking shots and testing my blood all
the time.
Chairman Collins. Sophia, how about you?
Miss Cygnarowicz. Taking shots and doing blood tests.
Chairman Collins. And doing the blood tests.
And, Colleen, I have a little different question for you.
These other children, with the exception perhaps of Sophia, are
old enough that they can monitor their blood sugar so that they
know when they are getting into trouble to some extent,
although we have learned it is hard when you reach your teenage
years. How do you determine when Dylan may need your help since
obviously he is so young he cannot report to you other than
through the blood test?
Ms. Rea. Well, his eyes. His eyes tell a lot. And if he is
very high, he gets very aggressive with other children and with
myself. If he is very low, he gets sleepy. And if he is tired
during the day, which he doesn't normally nap, but we do a lot
of glucose testing, especially when he was first diagnosed and
could not even speak. He was 14 months old. I have tested up to
18 times in 1 day.
Chairman Collins. Oh, wow. That has got to be so hard.
I am going to turn to my colleagues for questions, and then
I am going to have one final question for you. Senator
Lautenberg.
Senator Lautenberg. Madam Chairman, my questions were
already answered by the stunning presentation made by these
young people, and when I say ``stunning,'' I am not talking
about--they are all good-looking, but that is not the stunning
part. The stunning part is the realization about how much more
difficult life is when juvenile diabetes is present.
I have got to say for everybody here that we are so
fortunate to have Susan Collins, the Senator that she is and
Chairman of this Committee--by the way, ``Chairman'' doesn't go
with female, but we know what we mean when we say ``Chairman.''
She is the Chairman, and she is so motivated by her experiences
with people who have had juvenile diabetes. And I feel
similarly because when I look at you, all of you, I see my own
grandchildren and what I wish for them in the years ahead. But
we are lucky that we have Susan Collins here because she is on
the Republican side of the ledger, I am on the Democratic side
of the ledger, and the two of us are determined--we talked
privately outside--to do whatever we can, and I know I include
my other colleagues here as well, do whatever we can to move
this process along, to make life just a little bit easier for
all of you. And we hope that one day you will come here and
visit and say, ``What a past I have had, but I have passed
it,'' and we thank you all.
[Applause.]
Chairman Collins. Thank you, Senator, for your support and
your kind comments.
Senator Coleman.
OPENING STATEMENT OF SENATOR COLEMAN
Senator Coleman. Thank you, Chairman Collins, and I join my
colleague, Senator Lautenberg, in applauding the Chairman for
her tremendous leadership, and not just because she has a great
heart, but she gets things done. And I am very hopeful. As we
listened to these small voices, by the way, these are little
voices, but they are very loud. They are very loud, and what
you are saying, the witnesses, by the presence of the other
kids is echoing very loudly in these halls. So you really are
making a difference. And, Chairman, I thank you for putting
this together and for your leadership.
I want to say to LaNiece, you are very good. And folks in
this room, the parents have got to be very proud. It is hard to
sit up here in front of U.S. Senators and present testimony,
and the parents should be very proud.
Eric, let me ask you, how do you describe to your pals, how
do you describe juvenile diabetes to kids, to your buddies?
Mr. Eric Bonness. I just say the only people that really
understand the disease are the people who are my close friends,
because in order to really understand the disease, you have to
be around it all day. So, actually, my close friends understand
it when they see--just by watching me test my sugar, seeing
what I do with the various readings that I get of my blood
sugar. As far as other kids, they think when I wear my insulin
pump that I have a pager or stuff like that.
So I can explain it to people, but in order to get a full
understanding of the disease, you pretty much have to live
around it.
Senator Coleman. Alex, the same question.
Mr. Alex Bonness. Yes, I agree. I think the awareness of it
is very hard to get out for people to understand really what it
is.
Senator Coleman. Do you take the time to talk to them a
little about it?
Mr. Alex Bonness. Yes, I try to, because I get jokes about
when my blood sugar is low, we are eating candy during the day.
And kids have actually said to me, ``Wow, I wish I had
diabetes.'' It is kind of weird to hear them say that because
they don't really understand how terrible it is.
Senator Coleman. LaNiece, the same question to you. How do
you tell your friends? What do you say to them, and how do they
react?
Miss Evans-Scott. Some people think the same thing he just
said. They just want diabetes because I get to eat in class.
And at the beginning of the school year, me and my mom always
go to the class that I am in and explain it to them. Some
people don't understand it, though.
Senator Coleman. Katie?
Ms. Halasz. I am actually really lucky because my friends
help me take care of the disease. They make sure I always have
food with me. They make me test my blood all the time and help
me take my shots and stuff. So I am very fortunate with that.
Senator Coleman. Sophia, do you talk to your friends at all
about this? And what do you tell them, and what do they say?
Miss Cygnarowicz. I do not really know.
Senator Coleman. OK. That is OK.
Colleen, in terms of other parents, do you have
conversations and do they get it?
Ms. Rea. Oh, they get it. I talk to every person who will
stand still long enough.
[Laughter/applause.]
Senator Coleman. Thank you, Madam Chairman.
Chairman Collins. Thank you.
Senator Fitzgerald, your constituent, Sophia, has been
great in helping us understand the disease.
OPENING STATEMENT OF SENATOR FITZGERALD
Senator Fitzgerald. Well, thank you, Senator Collins. And,
Sophia, I want to welcome you here. I know where you are from
in Illinois. You are from Columbia, and I was there not too
long ago. I want to welcome you for coming all this way, and I
want to welcome the other Illinois constituents who are here. I
know Senator Durbin introduced them earlier.
And all of you kids, too, I was very impressed with your
statements, and you should be very proud of the good work that
you are doing helping to spread awareness of the difficulties
associated with diabetes.
Now, Senator Coleman asked you about what the other kids
were thinking, and I am just wondering. You did not quite get
into this, but are any kids at school maybe mean to you or do
they make fun of your diabetes? Does that happen to any of you?
LaNiece, are some of the kids mean?
Miss Evans-Scott. Sometimes.
Senator Fitzgerald. What do you say to them if they are
mean?
Miss Evans-Scott. I don't really say anything to them.
Senator Fitzgerald. Alex?
Mr. Alex Bonness. It is not really a disease that is--it is
not physical. You cannot see it. You couldn't look at me and
tell that I have diabetes. So I don't really get much trouble
from friends.
Senator Fitzgerald. Did you when you were younger, maybe in
second or third grade?
Mr. Alex Bonness. Sometimes people joke around about it,
bullies just trying to be mean, called me a diabetic and stuff.
I mean, it is rough, but you cannot do anything about it. So
you have got to move on.
Senator Fitzgerald. Katie, are the kids pretty
understanding?
Ms. Halasz. Some of them are. I know that the parents, they
don't really want me to go over to their houses because they
don't want to take the responsibility. But I don't--other than
that, they are fine with it.
Senator Fitzgerald. Now, if you were to stay at somebody's
house overnight, you would have to be checking your blood
sugar, and the parents of your friend might be worried about
liability having you stay overnight.
Ms. Halasz. Yes.
Senator Fitzgerald. How about the rest of you on the panel,
are you able to stay overnight at your friends' houses?
Mr. Alex Bonness. Pumps help, but I have actually had--my
brother talked about the migraine headaches, and at a sleepover
I did have one one time. So people kind of get worried about it
sometimes. I definitely don't do as many sleepovers as a lot of
my friends do.
Senator Fitzgerald. The others of you, have you ever slept
over at a friend's house?
Miss Cygnarowicz. Yes.
Senator Fitzgerald. Sophie, have you slept over at a
friend's house?
Miss Cygnarowicz. Yes.
Senator Fitzgerald. And do you check your own blood sugar?
Miss Cygnarowicz. Yes.
Senator Fitzgerald. You do. OK.
Do all of you have to wake up in the middle of the night to
check your blood sugar, or are you able to sleep through the
night?
Mr. Alex Bonness. I am lucky enough that my parents
actually wake up every night and check our blood sugars while
we are sleeping.
Senator Fitzgerald. Is that the same with all of you?
Ms. Halasz. I know when I am high or low, we have to wake
up every couple hours to make sure we are coming down or keep
going up. But, yes, there are a lot of nights that we have to
check our blood in the middle of the night.
Senator Fitzgerald. How many times does your blood sugar
have to be checked during the night?
Ms. Halasz. Anywhere from two to three.
Senator Fitzgerald. Two to three times every night.
Mr. Alex Bonness. If you are either high or low before you
go to bed. In most cases, you are usually good so you only
check like one time. But in some cases, yes, two or three
times.
Senator Fitzgerald. And do your parents get up and check
that?
Mr. Alex Bonness. Yes.
Miss Evans-Scott. Yes.
Senator Fitzgerald. For all of you, and do you sleep right
through it?
Mr. Alex Bonness. Well, mostly.
Senator Fitzgerald. OK. Well, Colleen, you are the one who
has to wake up then and check the blood sugar. It is a real
burden on the parents, isn't it?
Ms. Rea. Well, I go to bed late, and I test him before I go
to bed. And if his numbers are good, because he has the insulin
pump and he doesn't take a lot of insulin compared to these
other children yet, I can sometimes sleep through the night.
Occasionally I get to sleep through the night.
Senator Fitzgerald. Thank you so much for being here. We
will all be working together to try hopefully someday years
hence not to have these hearings in Washington because I am
hopeful that someday we will arrive at a cure or some
satisfactory solution that makes it easier to live life with
diabetes. But thank you all very much for what you have done to
bring awareness to people around our country on this issue. You
are making a great contribution and leaving a significant mark
on this country at a very early age.
So thank you all very much.
[Applause.]
Chairman Collins. I told you I was going to do one more
question, but I changed my mind in view of the hour. You are
the best panel of witnesses I have ever seen, so thank you so
much.
[Applause.]
And I know I speak on behalf of every Senator who has been
here today when I do promise to remember you. Thank you for
being here.
[Applause.]
We are now going to move to our final panel, but before we
do, the JDRF has asked me to announce that if any of the
children are feeling as if your blood sugar is low right now,
there is food right outside the hearing room if you need a
snack. So you can feel free to go get it if you think you need
a snack.
We are now going to call forth our final panel. Our final
panel of the day will testify about some of the promising
breakthroughs that have been made in juvenile diabetes research
as well as future opportunities for research. They will tell us
about the work that is being done in pancreatic islet cell
transplantation, which has been hailed as the most important
advance in diabetes research since the discovery of insulin.
Dr. Allen Spiegel is the Director of the National Institute
of Diabetes and Digestive and Kidney Diseases at the National
Institutes of Health.
Dr. Bernhard Hering is an Associate Professor of Surgery
and Director of Islet Transplantation at the University of
Minnesota.
And Anne Seidel of Dallas, Texas, is an actual islet cell
transplant recipient, and I am so eager to hear your story.
I want to turn to Senator Coleman, because there is a
distinguished witness from Minnesota here, for any additional
comments that he might have.
Senator Coleman. Thank you, Chairman Collins, and I would
just like to do a very brief introduction for Dr. Hering from
Minnesota.
Minnesota has established itself as the cluster for medical
research and technology, and in addition to making big
contributions to the State's economy, this activity also has
important benefits to our Nation's health. I was pleased to
form a Medical Technology Caucus with Senator Biden, again,
bipartisan. This is not a partisan issue. The health of our
kids and our families are not partisan issues. It is Democrats
and Republicans coming together.
I am proud to note that a large part of this activity has
always centered around the University of Minnesota,
particularly in dealing with the issue of islet transplantation
in our transplant facility. And Dr. Hering is one of the
leaders in islet transplantation. Again, as has been noted,
this technology represents a major source of hope for
diabetics, their families, and their friends. I share Senator
Fitzgerald's hope and passion that very quickly we move forward
with a cure. But the reality is that some of the advances that
Dr. Hering is involved with are very important, and I
appreciate, Madam Chairman, your inviting him here today to
discuss the current status of this technology and the steps we
next need to take.
I am proud to be a cosponsor with Chairman Collins on S.
518, the Pancreatic Islet Cell Transplantation Act. Voices are
being heard, and we will work hard to make sure that we make
progress in this area.
Thank you, Madam Chairman.
Chairman Collins. Thank you very much, Senator Coleman.
Dr. Spiegel, we will proceed with you.
TESTIMONY OF ALLEN M. SPIEGEL, M.D.,\1\ DIRECTOR, NATIONAL
INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES,
NATIONAL INSTITUTES OF HEALTH
Dr. Spiegel. Thank you. Chairman Collins, Senator Coleman,
delegates to the JDRF Children's Congress, family members, and
guests, I appreciate the opportunity to testify at this hearing
on type 1 diabetes. Nothing I could say would be more eloquent
than the testimony you just heard from Mary Tyler Moore, from
the children and teens who told their stories, nor the
testimony you will hear from Anne Seidel, an islet transplant
recipient. Nonetheless, I have much to tell you about what NIH
support has accomplished to date and how we plan to seize the
exciting opportunities before us in type 1 diabetes research.
---------------------------------------------------------------------------
\1\ The prepared statement of Dr. Spiegel appears in the Appendix
on page 60.
---------------------------------------------------------------------------
All this is detailed in a thick report on the special
statutory funding program for type 1 diabetes, released
yesterday by Secretary Thompson. The full report, as well as a
slimmed-down executive summary, are available in hard copy and
on the NIDDK website. This morning I will just offer a few
examples and highlights.
First, though, on behalf of NIH, I would like to thank you
and the other Members of Congress for your support of the NIH
and of the special statutory funding program which has
significantly augmented our regularly appropriated funds for
diabetes research.
The special program has enabled the formation of unique,
productive collaborations involving multiple institutes at NIH
and the Centers for Disease Control, and has brought new
researchers from multiple disciplines together to accelerate
research on type 1 diabetes.
I would also like to acknowledge the JDRF for its vigorous
support of type 1 diabetes research. The JDRF is an invaluable
collaborator as the NIH continues to define specific research
goals and needs in type 1 diabetes. The JDRF indeed has
partnered with NIH in diabetes initiatives and has brought its
international scope to bear in combating diabetes since this
disease knows no geographic boundaries.
When the special funding program was increased from $30
million to $100 million per year in fiscal year 2001 and
extended to 2003, we formulated an ambitious research plan with
input from expert scientists and key advocacy groups such as
the JDRF. Our type 1 diabetes research plan has six broad
goals: To identify the genetic and environmental causes; to
prevent or reverse the disease; to prevent or reduce
hypoglycemia, the low blood sugar you heard so much about; to
prevent or reduce complications; to attract new talent to type
1 diabetes research; and last, but certainly not least, to find
a cure.
In the 2 years since I last testified before you,
significant progress has been made in all these areas as
reflected in just five examples.
First, basic research on diabetes complications has shown
that a vitamin derivative can prevent complications in animal
models and has led to clinical trials of a drug to prevent
blindness. Second, a network of pediatric centers called
``DirecNet'' has been created with the special funds to test
new technology made by two different companies for continuous,
non-invasive glucose monitoring so we can develop alternatives
to the multiple finger sticks we heard about from the kids this
morning and that Senator Fitzgerald asked about. Third, a
biotechnology drug, a monoclonal antibody, has shown promise in
reversing recent onset type 1 diabetes and will be tested in a
larger clinical trial supported by the special funds. Fourth,
investigators have reported success in converting adult stem
cells--and both mouse and human embryonic stem cells--into
insulin-producing cells. Fifth, the Immune Tolerance Network,
with support from the special funding program, is completing a
multi-center trial of the Edmonton Protocol for islet
transplantation first reported in the year 2000. An interim
report released just 3 weeks ago indicated that several centers
are capable of rendering greater than 60 percent of their islet
transplant recipients insulin independent.
You will hear in much greater detail about islet
transplantation from one of the pioneers to my left, supported
by NIH, Dr. Bernhard Hering. Let me say that NIH is vigorously
addressing the two major scientific hurdles we must clear to
make islet transplantation a true cure: Developing an
inexhaustible supply of insulin-producing cells, and achieving
immune tolerance, a state of acceptance of a transplant that no
longer requires taking potentially toxic and costly
immunosuppressive drugs lifelong.
For the former hurdle, the Beta Cell Biology Consortium,
created with the special funds, is intensively studying all
aspects of islet development so we can learn how to grow
sufficient amounts for treatment. A significant portion of the
special funds, recently augmented--with your leadership--by the
Congress from $100 million to $150 million per year and
extended from 2004 to 2008, will be devoted to research needed
to make islet transplantation a widely available reality. We
will also be focusing attention on so-called translational
research that converts basic science advances into practical
diagnostic tests, treatments, and preventive measures.
I am grateful for this opportunity to give you some
examples of the progress and plans being made in type 1
diabetes research, particularly with the assistance of the
special statutory funding program. We are living in a new and
exciting era of scientific research that, with its rapid
advancement and immense opportunities, is unprecedented. We
intend to take full advantage of the new technologies and
information that have emerged in this new era to realize
greater progress in diabetes research.
We continue to be inspired by the dedicated efforts of
patients and their families and by organizations such as the
Juvenile Diabetes Research Foundation. We remain steadfast in
our fight against diabetes, helping kids such as Katie Halasz
right now with efforts such as our guide for school personnel,
``Helping the Student with Diabetes Succeed,'' while ever
focused on the ultimate goal--finding a cure.
We must help all the children in this room who represent
the children with type 1 diabetes in this entire country. They
are the reason for all of our efforts. Thank you.
[Applause.]
Chairman Collins. Dr. Hering.
TESTIMONY OF BERNHARD J. HERING, M.D.,\1\ ASSOCIATE PROFESSOR
OF SURGERY, DIRECTOR OF ISLET TRANSPLANTATION, UNIVERSITY OF
MINNESOTA, MINNEAPOLIS, MINNESOTA
Dr. Hering. Chairman Collins and Senator Coleman, my name
is Bernhard Hering, and I am addressing you today on behalf of
my physician-scientist colleagues. Our ability to transform the
landscape of juvenile diabetes in this country in the next
decade depends heavily on the Federal Government's efforts.
Chairman Collins, I want to applaud your efforts and those of
the more than 30 Senators who have cosponsored your legislation
entitled ``The Pancreatic Islet Cell Transplantation Act of
2003.'' I believe that, if passed, this bill will empower
clinical researchers to expedite the translation of islet
transplant research into unsurpassed, innovative treatments for
individuals with juvenile diabetes and accelerate the
integration of such treatments into health care delivery
systems.
---------------------------------------------------------------------------
\1\ The prepared statement of Dr. Hering appears in the Appendix on
page 66.
---------------------------------------------------------------------------
Although the research community has experimented with islet
cell transplants for decades, they are now at last becoming a
practical, successful, superior, and distinct therapy for
patients with juvenile diabetes. This disease continues to be
challenging, with significant morbidity and mortality. Diabetes
is a devastating burden to patients and their families,
especially when acute complications are frequent and chronic
complications are advanced. In the previous panel, you heard
about the daily challenges people face in living with juvenile
diabetes from the most effective messengers--children who
suffer each and every day with this disease.
Please allow me now to illustrate how islet transplants are
unsurpassed in their potential to transform the lives of people
with difficult-to-control juvenile diabetes. Lorna is a 35-
year-old mother of two boys who developed diabetes at age 9.
She completely lost her ability to sense low blood sugar
levels, also referred to as hypoglycemia. Brain function is
severely compromised during episodes of hypoglycemia, leading
to confusion, distorted perception of surroundings, dizziness,
odd behavior, seizure, coma, and possibly death. Lorna became
incapacitated by weekly episodes of severe hypoglycemia,
requiring the help of a third person and often paramedics. As a
result, Lorna lost her driver's license, was laid off at work,
was divorced, and was denied the right to see her boys.
In August 2000, Lorna was admitted to the General Clinical
Research Center at the University of Minnesota for an islet
transplant under a protocol supported by the Juvenile Diabetes
Research Foundation and the NIH. She was taken to a radiology
suite, where a local anesthetic was given and a catheter was
placed through her skin into the portal vein of her liver.
Islets isolated from a cadaver donor pancreas were resuspended
in buffer, transferred to a transfusion bag, and infused over
20 minutes through the catheter into her portal vein. Infused
islets were carried by her blood to smaller branches of the
portal vein, where they lodged, engrafted, and resumed tightly
controlled secretion of insulin in response to her blood sugar
levels. The catheter was then removed, and she was discharged
to go home 2 days later.
Lorna's blood sugar levels returned to normal. She was able
to completely discontinue insulin injections 1 month after her
islet transplant. Since her transplant, she has not experienced
a single episode of severe hypoglycemia. She learned to love
life again, rediscovered her ability to make contributions to
our world, regained control over her destiny, received her
driver's license back, found a new job, married the paramedic
who had repeatedly saved her life----
[Laughter/applause.]
Dr. Hering [continuing]. While she was diabetic, closed on
a new home, and was granted authorization to take care of her
boys again, all in the first year after her transplant. Now,
almost 3 years later, Lorna continues to be insulin independent
and diabetes free. She takes immunosuppressive medications to
prevent rejection of her transplanted islets. She has not
experienced any severe adverse events. You will hear another
remarkable success story from Anne Seidel following my
testimony. Equally impressive success stories have been
reported by a few other centers in the United States, including
the University of Miami, Baylor College in Houston, and the
University of Pennsylvania, to name the most active and
successful of centers.
For patients like Lorna, insulin is survival, but an islet
transplant is return to life. Islet transplants are now a
clinical reality, not just a topic for investigational
research.
Let us now assume for just one moment that your 35-year-old
daughter suffers from juvenile diabetes complicated by
recurrent episodes of severe hypoglycemia, that she meets all
accepted medical criteria for an islet transplant, that she has
good health insurance, and that she wants to proceed with an
islet transplant. Will she be able to undergo an islet
transplant?
Most likely no. There is a severe shortage of donor organs
for islet transplants. Funding in the area of clinical
translation of islet transplantation has not kept pace with the
science, and regulations must be changed to provide incentives
for additional procurement of donor organs for islet
transplantation and research. And insurance coverage of islet
transplantation is currently not available, thereby largely
restricting them to patients meeting the very stringent
eligibility criteria of research protocols.
This disconnect between the promise of basic science versus
the delivery of better health in the United States is of
significant concern. Islet transplants for the treatment of
diabetes are being covered by several provinces in Canada,
where a landmark pilot clinical trial called the Edmonton
Protocol was performed in 2000. The steadfast commitment to
basic biomedical research in the United States has provided the
basis for today's high success rate in reversing diabetes in
human patients. It has also provided an unprecedented supply of
information for further breakthroughs in clinical islet
transplants. Yet islet transplants remain largely unavailable 3
years after the demonstration of proof of principle. Failure to
use available science is costly and harmful. It leads to
overuse of inferior care. In contrast to Canada, we fail to
deliver the best care we could for patients with difficult-to-
control diabetes. Health insurance companies should be on board
but are not.
Both my learned opinion and my best bet are that one-third
of the gifted children with juvenile diabetes with us today in
this room will develop devastating, destructive, or deadly
diabetes complications before they are 50 years old--unless we
enhance our preclinical and clinical research agenda markedly
in the next 2 years in order to realize a sizable effect within
10 years. The potential short- to mid-term impact of islet
transplants on patients with juvenile diabetes prone to develop
devastating complications is unmatched by any other treatment
modality. The Pancreatic Islet Cell Transplantation Act of 2003
would help overcome the fragmentation and underfunding of
today's clinical islet transplant infrastructure and remove
major translational blocks in the implementation of islet
transplants. More specifically, it would be instrumental in
maximizing donor pancreas utilization, in documenting the
benefits of islet transplants, in shortening the time to FDA
approval of transplants of human islets, and, finally, in
developing an unlimited supply of tissue for transplantation
such as xenogeneic islets from animals or stem cell-derived
islets.
In closing, I believe the Pancreatic Islet Cell
Transplantation Act of 2003, if passed, will greatly enhance
the islet transplant translational infrastructure and help it
to operate much more efficiently. It will raise needed
awareness, create additional momentum, send a very strong
signal to all major stakeholders in health care delivery, and
facilitate the expedient delivery of today's science and
technology for the benefit of thousands of patients afflicted
with juvenile diabetes. The bill will also help prepare the
field to respond nimbly to the extraordinary advances that
surely will emerge from stem cell biology and other high-
impact, cell-based technologies of the future. Thus, this
legislation will have implications well beyond its primary
objectives.
As I look around the room at all these children who are
here today to take an active role in finding a cure for
juvenile diabetes, I know that the scientific community and
Congress must match their passion and dedication. This will not
be an easy task, but progress in science has been truly
remarkable and emerging opportunities are even more
extraordinary. Removing translation blocks will allow our
patients to benefit from scientific breakthroughs.
We were able to put a man on the moon more than 30 years
ago because we wanted it, because President Kennedy declared it
possible. I know we could cure diabetes if we declared it
possible.
Madam Chairman, Senator Coleman, thank you from the bottom
of my pancreas.
[Laughter/applause.]
Chairman Collins. Thank you for your testimony. Ms. Seidel.
TESTIMONY OF ANNE SEIDEL,\1\ ISLET CELL TRANSPLANT RECIPIENT,
DALLAS, TEXAS
Ms. Seidel. Senator Collins and Members of the Committee,
thank you for the opportunity to appear before you today as an
example of a true success story resulting from the public-
private partnership between the Juvenile Diabetes Research
Foundation and the Federal Government in supporting medical
research and the National Institutes of Health. I am Anne
Seidel, proud mother of Charlie, 6, and Lacey, 5. I have been
an active JDRF volunteer for 5 years in Dallas and nationally.
---------------------------------------------------------------------------
\1\ The prepared statement of Ms. Seidel appears in the Appendix on
page 77.
---------------------------------------------------------------------------
I was diagnosed with juvenile diabetes 35 years ago, at age
6. My son Charlie, now 6, was diagnosed 5 years ago. I cannot
describe to you the anguish I experienced when he was diagnosed
with juvenile diabetes. As a mother, your focus in life is
caring for and protecting your children, but I could not
protect him from this disease that was ravaging my body for
over 30 years at that point.
While I focused all of my energy on caring for Charlie when
he was diagnosed, diabetes was taking an enormous toll on my
body. Over the years of living with diabetes, I have won the
battle of near blindness and kept going. I have had nerve
damage in my feet and partial paralysis of my stomach and kept
going. My blood sugars would jump from being at a level of 16--
so low that I was in danger of becoming unconscious--to a level
of 600--which is so high that I could slip into a coma--in the
course of 2 hours and then back down and back up again. This
roller coaster defined many of my days. Most days, feeling
nauseated and exhausted, I had to keep going.
But the complication of my diabetes that prevented me from
keeping going was when I lost my ability to feel my low blood
sugars. Instead of being able to sense this and quickly take
some sugar and rectify the situation, my blood sugar often kept
dropping until I was unconscious, blacked out, totaling my car,
or so confused that I did not know who I was, who my children
were, and oftentimes not sure whose diabetes I was taking care
of. I came very close several times to accidentally giving
Charlie insulin that he didn't need, and the result of that
could have meant his death.
About 4 years ago, I began watching--with more than a
passing interest--the advancement of pancreatic islet cell
transplantation. Upon investigation, the state of my diabetes
matched the criteria for participating in these clinical
trials. When deciding to participate in the trials, I had to
come to terms with the fact that the procedure was not
available for children, and because of the immunosuppressive
drugs, this was not available for many. As a mother, your
instincts are to take care of your children first. And after
lots of introspection, I had to become OK with having the
procedure because I thought in an airplane the parent must put
the oxygen mask on themselves first so they can save their
child, and I had to do the same with my diabetes.
After speaking with every center in the world that was
conducting islet transplants, I was screened at several places
and I chose to be on one list. Because of the severe shortage
of pancreases in the United States and with my rare blood type,
I spent over 2 years on the waiting list, hoping for the call.
Every day that passed, I worried that my diabetes would worsen
and that while I waited I might be prevented from receiving a
transplant because of ensuing complications. I decided to
increase my chances for a transplant and listed at three
additional centers.
I received my transplant on February 5 and my second on
April 9 of this year. Most current protocols involve two
pancreases, two transplants to receive the appropriate number
of islet cells. I was called to come to the Methodist Hospital
in Houston, and the insulin-producing islet cells were
extracted from a donor pancreas, put into an IV bag. And while
this was being done, I changed into my gown and went into the
radiology suite where the radiologist fed a small needle into
my liver and dripped the cells into me. I was in the surgery
suite for only about 20 minutes and was conscious throughout
the procedure. Back in the recovery room, I looked down at my
side and I said the surgeon, ``You mean you just saved my life
and all I have to show for it is this Band-aid?'' Thirty hours
after the transplant, I was home.
Since my transplant, I no longer need to take insulin. To
give you a sense of how dramatically this has changed my life,
in the 35 years I had juvenile diabetes, I have taken 255,500
units of insulin and have pricked my fingers 56,210 times. For
the first 12 years of my diabetes, I tested my urine 21,900
times. People have asked me to describe how I feel after the
transplant, and the best analogy I can think of is that I felt
like I had not bathed in 35 years and somebody finally washed
me off.
An amazing result of the transplant is that many of the
complications that plagued my days before are now gone. The
nausea, exhaustion, confusion, unconsciousness, foot pain, and
a large helping of fear are now gone. Having clear thoughts,
energy, not feeling fearful of endangering myself or my
children has been nothing less than utopia. Diabetes doesn't
define me anymore. I truly have received the gift of a
lifetime.
As one of the approximately 250 people worldwide whose life
has been changed through an islet transplant, I thank you from
the bottom of my heart. Your commitment to the NIH, coupled
with JDRF's commitment, has truly saved my life. But there is
no time to sit back and congratulate ourselves because there
are many more who cannot take advantage of this procedure. And
I will not rest or be truly happy until Charlie and these kids
and all my friends with type 1 can be cured like myself. They
deserve no less. Call me demanding, but I do not like being one
of so few.
It may sound strange, but diabetes has given me many
opportunities in my lifetime, one of which is the privilege of
being here and talking to you. I want to thank you publicly and
JDRF for saving my life. A cure is within our grasp. Together,
our efforts and funds must make this happen sooner than later.
Thank you.
[Applause.]
Chairman Collins. Ms. Seidel, thank you so much for sharing
your miraculous story with us. I think your story gives hope to
every person in this room. And I, too, look forward to the day
when there are no shortages, when insurance and Medicare cover
this procedure--in fact, I am looking at introducing an
amendment to have a demonstration project under Medicare to pay
for the procedure. And we need to get my legislation passed to
overcome some of the other barriers that Dr. Hering also
outlined for us.
You have made the process sound relatively easy. But you do
have to take immunosuppressive drugs, don't you?
Ms. Seidel. Yes.
Chairman Collins. Have you had any negative reaction to
those or are you generally doing as well as you seem to be
doing?
Ms. Seidel. I am doing really well. I have had very few
complications from the drugs. I have experienced mouth sores,
which are common side effects of the immunosuppressants, but to
me, any bump in the road has been a very small price to pay for
what I have received.
Chairman Collins. It really is wonderful. I have followed
this procedure for the last couple of years and read a lot
about it, but you are the first recipient whom I have had the
honor to meet. It is just fabulous to hear your story today.
Ms. Seidel. Thank you for helping make it possible.
Chairman Collins. Thank you.
Dr. Hering, can you help us understand better why this
procedure is not appropriate for most children right now?
Dr. Hering. At this point in time, we have to limit this
procedure to a very small subgroup of patients in whom the
benefits outweigh the risks associated with the use of
immunosuppressive medication. And I think it would not be
appropriate to consider islet transplantation at this point in
time for the treatment of children because complications are
possibly very harmful.
But progress has been made recently in our ability to
prevent rejection, and as Dr. Spiegel pointed out, major
research networks have been established recently to evaluate
emerging breakthrough technologies in this direction.
The limiting factor has been to move the wonderful progress
that has been made from the mouse level to the point that it
can be safely evaluated in people. So we need to make this
translational step and need to provide compelling evidence
obtained in non-human primate studies that would justify
testing in people. If we could do this and if we could really
reduce the time it takes to take these steps, we could clearly
capitalize on all the breakthroughs that have been reported
recently.
Chairman Collins. Thank you for the research that you are
doing.
Dr. Spiegel, thank you for giving us the broader overview
of all of the promising developments in research. Every time
you appear before me, I always put you on the spot, and I
always feel bad about it, but I am going to do it again this
year. That is, we have heard from Dr. Hering and we have heard
from the Juvenile Diabetes Research Foundation that the
legislation that I have introduced to try to remove some of the
barriers to the procurement of more pancreases and to otherwise
encourage the development of more procedures to facilitate the
kind of transplant that has made such a difference to Ms.
Seidel. I have introduced the Pancreatic Islet Cell
Translantation Act and would really like to have the support of
NIH. Can we count on the support of NIH for my legislation?
Dr. Spiegel. Given what a strong supporter you have been of
type 1 diabetes research and of the NIH, I, of course, would
like to say yes, so I hesitate to really inject this note in
this wonderful hearing. You know, though, very well that in my
position I really can't comment on pending legislation.
What I will say is I am happy to address any aspect of the
science associated with this, and I would say that this is
clearly being very carefully evaluated in the Department and
elsewhere. We are obviously very motivated by the advances Dr.
Hering has talked about, and we are trying with a collaborative
islet transplant registry in a variety of ways to meet these
goals. But I really can't comment further.
Chairman Collins. Well, I thought I would try.
Dr. Spiegel, in addition to the very promising developments
with islet transplantation, what do you view as the single
other most promising therapy on the horizon for juvenile
diabetes?
Dr. Spiegel. Just sticking with the issue of a cure, we
have the wonderful opportunity with the resources you have put
at our disposal to explore at least two other alternative
strategies, and these need to be explored in parallel. One is
an artificial pancreas. Many of the kids you have seen--and
heard from--have these pumps, and these are an improvement in
terms of insulin delivery. If we could hook those up with a
continuous sugar-monitoring device that is non-invasive--and
these are being studied in this collaborative trial that I
mentioned--we might have what is called a closed-loop system.
Again, such an artificial pancreas would never mimic
perfectly what we see with the actual islets, but it would be
pretty close and could be pretty good. And if it were
miniaturized, it could make a huge difference. So that is being
pushed, and that is really an example also of public-private
partnership, basic research done by NIH-supported
investigators, including the small business set-aside--which we
support, as you know--which is then picked up by industry. Two
major companies are advancing this technology. They claim they
are close, but we have to see the evidence.
The other issue is a complicated issue that Dr. Hering
referred to--xenotransplantation, the idea, for example, of pig
islets. People have taken pig insulin for years, and it works
very well. It works just as well in many respects as human
insulin. So, why can't we just use pig islets? There are issues
about viruses that are present and, of course, transplant
rejection.
There, too, we are supporting research. I want to make a
point here with regard to both the immune tolerance we heard
about and xenotransplantation. As important as this is for type
1 diabetes, it transcends one disease. It is important for all
transplantation. There is a shortage of all these organs in
terms of kidney and liver, etc. So this is very important
research.
Knock-out technology, so-called, has created pigs that lack
the sugar that causes hyper-acute rejection. And that is a step
in the direction of making this a reality.
Chairman Collins. Thank you. Senator Coleman.
Senator Coleman. Thank you, Madam Chairman.
I was mentioning the Minnesota connection. First, I want to
thank Mary Tyler Moore for her leadership, and I would be
remiss in not mentioning the Minnesota connection there because
she lived and worked there for many years. So we are very
proud. But thank you for your strong voice here.
Another Minnesota connection are folks from Medtronic, and
I don't want to pick out one company, but there is some great
work going on in the biotechnology side.
Dr. Spiegel, let me ask you first, you just touched briefly
upon stem cell, and, in fact, another area where the University
of Minnesota is doing some breakthrough work on adult stem
cells. Give me your best sense of how far down the road, if you
can do that, we are from using stem cell research to make any
significant advances here, either for adult or for juvenile
diabetes?
Dr. Spiegel. Thank you, Senator Coleman. I will focus my
remarks exclusively on stem cells as applied to diabetes,
particularly type 1 diabetes.
Indeed, at the University of Minnesota, is a colleague of
Dr. Hering's, Dr. Catherine Verfaille. She just addressed our
National Advisory Council, and she is a member of our Beta Cell
Biology Consortium. She is doing extraordinary work on what are
called multi-potent adult progenitor cells. While she hasn't
yet had definitive success in turning these into insulin-
producing cells, they have been turned into liver cells and
other relevant cells, and we are looking for further
developments.
There have been reports of adult bone marrow-type stem
cells in mouse models curing diabetes. At the same time, there
have been substantial advances, as I said in my testimony, both
in terms of turning mouse embryonic and human embryonic stem
cells into insulin-producing cells. They don't produce as much
insulin as we would want. They are not perfect in terms of
being like the real beta cells in the islets. But this is a
very important step in the right direction that we are
supporting heavily within the guidelines of the President's
decision of August 9, 2001. We at NIH are totally committed to
making this not only an acceptable but a priority area of
research. And that is the message that I and Dr. James Battey,
who heads the NIH Stem Cell Task Force, are putting out across
the country to encourage our investigators.
Senator Coleman. I am familiar with Catherine's work and
working with some technology companies to do the next step, and
translate the research into a reality. Is the FDA process of
looking at that stuff, is that set up in a way to allow us to
move quickly enough to take advantage of some of these research
opportunities?
Dr. Spiegel. To answer this would really be beyond my
purview, so I hesitate to do so. What I would say is that the
company, Athersys, which you are referring to, is one that has
had an opportunity to present to the Stem Cell Task Force. We
are really interested in public-private partnerships under Dr.
Zerhouni's leadership. He has been creating a vision for the
future of NIH called a roadmap, and there are many aspects of
that vision which we discussed at a retreat last Friday that
are relevant to this audience: Re-engineering the clinical
research enterprise, facilitating harmonization with the NIH
and the FDA, and public-private partnerships. These are very
relevant themes.
Senator Coleman. Thank you very much.
Ms. Seidel, just one question. How were you chosen? You
said in your testimony you fit the criteria, but how did you
take advantage of or get picked for this opportunity?
Ms. Seidel. When the criteria was outlined by most of the
centers that were doing it, Minnesota being one, the criteria
included patients that were healthy otherwise, having primarily
one complication which is asymptomatic, low blood sugars,
meaning low blood sugars that you cannot feel to the point
where that can paralyze your life from being normal in any way.
And that was something that I suffered from. Otherwise, I was
healthy.
Dr. Hering can tell us more, but, really, the patients that
were being selected were patients that could then be quickly
rendered free of those low blood sugars once they were
transplanted, but whose bodies could withstand the
transplantation and the immunosuppressive drugs.
Senator Coleman. What was your biggest fear when you
decided to take advantage of the islet transplant?
Ms. Seidel. How long it was going to take. That was my
biggest fear. I knew that we had a severe shortage of organs in
the United States, and with a rare blood type, I hoped that my
complications of diabetes didn't worsen while I waited.
Senator Coleman. How long after the surgery did you first
get the sense that, wow, something has changed?
Ms. Seidel. Almost immediately. I felt better within a
couple hours of the anesthesia wearing off.
Senator Coleman. Dr. Hering, how many potential patients
like Ms. Seidel do you kind of wade through before you get to
choose? Which has to be, by the way, a pretty daunting feeling
about making those kinds of choices. I would hate to be in that
position. But what is your universe? And then ultimately how
large is the patient group that benefits from this opportunity?
Dr. Hering. Thank you, Senator Coleman, for your question.
When we announced our program, within a matter of a few weeks
we received 2,500 applications, and it was the first challenge
how to deal with those applications and how to be able to get
back to patients and explain in sufficient detail why we can do
it or why we cannot do it.
First and foremost, we have to protect the safety of
participants. We have a long list of 40 exclusion criteria. We
need to continue considering those criteria very seriously to
protect the safety of participants.
I believe that of those 2,500 that contacted us initially,
only 30 to 40 were identified as eligible candidates. If you
ask the question how many patients in the United States at this
point in time would meet criteria like Anne Seidel, I think you
are talking about at least 5 percent of our population that has
been diagnosed with type 1 diabetes. So you are talking about
50,000 people at this point in time.
When I look at the field of islet transplantation and cell
replacement therapies today, what we do is we show the
feasibility of cells to reverse diabetes. This is a completely
new area in diabetes therapy that has not been available
before. Everybody who wants to see can see what the
implications are and future technologies are waiting to be
implemented. Thus, what we do today is just a first step. We
show the feasibility of all therapy, a technology that is
fundamentally new and different. I think we need to keep the
momentum going. Of particular importance is that we maximize
donor pancreas utilization. We are not facing a true shortage.
We are facing logistical problems. Within 1 year we could
procure 2,000 additional donor pancreases. These organs are
available, but are currently not recovered. I think your bill
clearly addresses this point very directly. This example
indicates that it is not only science that is limiting our
ability to deliver the new technology of all therapy. We really
have to identify non-scientific roadblocks and address these
problems.
Senator Coleman. Thank you, Doctor. I again want to applaud
both of you for your work and also what you are doing today to
help us, I think simply better educate some folks so that in
the end we can pass Chairman Collins' legislation and make a
real difference in a lot of lives. So thank you very much.
Thank you, Madam Chairman.
Chairman Collins. Thank you, Senator.
I want to thank our panel for your expert testimony today.
It was fascinating and a fitting conclusion to what has been a
very moving experience for me in chairing this hearing. Thank
you all so much for being with us.
I want to thank all of the witnesses who appeared today,
and most of all, I want to thank the children because they are
the ones who really motivate all of us to work harder in the
search for the cure.
I also want to pay tribute to Mary Tyler Moore. You could
not have a better adult spokesperson for this cause.
And I want to thank the Juvenile Diabetes Research
Foundation. They have worked so hard to put this Children's
Congress together. I am very proud to have been the co-Chair
this year, and it has been a real privilege to work with a
group of people who are so caring and so committed. Together, I
am positive that we are making a difference every day. Thank
you for being here.
The hearing record will remain open for 15 days for the
submission of some additional questions that we ran out of time
for and any statements. Thank you very much.
This hearing is adjourned.
[Applause.]
[Whereupon, at 1:02 p.m., the Committee was adjourned.]
A P P E N D I X
----------
PREPARED STATEMENT OF SENATOR LIEBERMAN
Thank you, Madam Chairman for calling this hearing today so that we
can focus on a disease that affects the daily lives of up to a million
children and their parents across the country and on an organization--
the Juvenile Diabetes Research Foundation--that deserves enormous
credit for its global leadership in working to find a cure for this
misunderstood disease.
It's such a pleasure to have this group of young, lively
representatives here today who are all doing their part to raise
awareness about the importance of juvenile diabetes research. Three of
these young lobbyists are from my home State of Connecticut. If you'll
indulge me for a moment, Madam Chairman, they are Matt Ruby, a 17-year-
old from Avon, Amanda Updyke, a 14-year-old from East Lyme, and Dylan
Rae, from my home town of Stamford, who will join his mom and dad in
testifying today. Dylan, I know I'm a little late, but I understand you
had a big day over the weekend, so, on behalf of my colleagues here
today, I want to wish you a belated but hearty Happy Fourth Birthday.
As you all know too well, Type 1 diabetes affects anywhere from
500,000 to 1 million Americans, most of them children and young adults.
In act, the risk of developing Type 1 diabetes is higher than the risk
of developing virtually any other severe, chronic childhood disease.
That's why the search for a cure is so important.
What most people don't realize is that insulin is not a cure. Type
1 diabetes needs constant attention and is often difficult to manage.
And because of the threat of complications, it can reduce the quality
of life and shorten the life span of those who suffer from it.
Diabetes is also one of the most expensive diseases to treat.
Finding a cure not only would improve the lives of these young people
with us today, it would save taxpayers millions of dollars in Medicare,
Medicaid and State Children's Health Insurance Program expenses.
That's why I have proposed an American Center for Cures, an
institute dedicated to finding the next generation of medicines that
will enable this generation of Americans to live healthier, stronger,
and longer lives. We must take some of the miraculous developments that
are occurring in laboratories around American today--the Human Genome
Project, stem cell research, all sorts of other pharmaceutical
breakthroughs--and focus them in one place with the goal of curing some
of the chronic diseases--including diabetes--that plague 100 million
Americans and cost $750 billion a year to treat.
We can do this, but we can only do it with an investment of public
money, attractions for private money, and a center to make this a real
goal. The experts I talk to say that with research we are within reach
of cures for a variety of diseases including diabetes, Alzheimers,
Parkinson's, cancer and AIDS, if we really work at it.
In the meantime, we must lend our strong support to Chairman
Collins' bill, the Pancreatic Islet Cell Transplantation Act, which I
am pleased to co-sponsor. Among other things, this bill directs the
Secretary of Health and Human Services to study and assess the value of
a promising new therapy--pancreatic islet cell transplantation--whereby
healthy pancreatic cells are injected into diabetics who's own
pancreases have failed. It is a simple procedure, very similar to a
blood transfusion, and the early results have given a lot of people
hope.
When all is said and done, what we need is national leadership on
this issue, national leadership to commit resources and marshal our
resolve to cure this disease. This is America. In America we don't just
manage problems; we solve them. That's what our country is all about.
All of you have committed to a goal of curing diabetes. Today, I join
you in that commitment--to banish diabetes from our lives and the lives
of our loved ones forever.
Thank you, Madam Chairman.
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