[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

                                 ------                                
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

                              ----------                              


                         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.
---------------------------------------------------------------------------
    \1\ The prepared statement of Ms. Moore appears in the Appendix on 
page 42.
---------------------------------------------------------------------------
    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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