[Senate Hearing 113-807]
[From the U.S. Government Publishing Office]
S. Hrg. 113-807
DIABETES RESEARCH: REDUCING THE BURDEN OF DIABETES AT ALL AGES AND
STAGES
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HEARING
BEFORE THE
SPECIAL COMMITTEE ON AGING
UNITED STATES SENATE
ONE HUNDRED THIRTEENTH CONGRESS
FIRST SESSION
__________
WASHINGTON, DC
__________
WEDNESDAY, JULY 10, 2013
__________
Serial No. 113-8
Printed for the use of the Special Committee on Aging
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
Available via the World Wide Web: http://www.fdsys.gov
______
U.S. GOVERNMENT PUBLISHING OFFICE
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SPECIAL COMMITTEE ON AGING
BILL NELSON, Florida, Chairman
RON WYDEN, Oregon SUSAN M. COLLINS, Maine
ROBERT P. CASEY JR, Pennsylvania BOB CORKER, Tennessee
CLAIRE McCASKILL, Missouri ORRIN HATCH, Utah
SHELDON WHITEHOUSE, Rhode Island MARK KIRK, Illinois
KIRSTEN E. GILLIBRAND, New York DEAN HELLER, Nevada
JOE MANCHIN III, West Virginia JEFF FLAKE, Arizona
RICHARD BLUMENTHAL, Connecticut KELLY AYOTTE, New Hampshire
TAMMY BALDWIN, Wisconsin TIM SCOTT, South Carolina
JOE DONNELLY Indiana TED CRUZ, Texas
ELIZABETH WARREN, Massachusetts
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Kim Lipsky, Majority Staff Director
Priscilla Hanley, Minority Staff Director
CONTENTS
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Page
Opening Statement of Chairman Bill Nelson........................ 1
Prepared Statement........................................... 68
Statement of Ranking Member Susan M. Collins..................... 1
Prepared Statement........................................... 69
PANEL OF WITNESSES
Jean Smart, Actress.............................................. 4
Ray Allen, Miami Heat Player and Father of JDRF Children's
Congress Delegate, Walker Allen................................ 12
Quinn Ferguson, Juvenile Diabetes Research Foundation, Children's
Congress Delegate.............................................. 19
Griffin Rodgers, MD, Director, National Institute of Diabetes and
Digestive and Kidney Disorders, National Institutes of Health.. 24
Jeffrey Brewer, President and CEO, Juvenile Diabetes Research
Foundation..................................................... 46
APPENDIX
Prepared Witness Statements
Jean Smart, Actress.............................................. 7
Ray Allen, Miami Heat Player and Father of JDRF Children's
Congress Delegate, Walker Allen................................ 15
Quinn Ferguson, Juvenile Diabetes Research Foundation, Children's
Congress Delegate.............................................. 21
Griffin Rodgers, MD, Director, National Institute of Diabetes and
Digestive and Kidney Disorders, National Institutes of Health.. 27
Jeffrey Brewer, President and CEO, Juvenile Diabetes Research
Foundation..................................................... 48
ADDITIONAL STATEMENTS FOR THE RECORD
Juvenile Diabetes Research Foundation............................ 71
Robert J. Shapiro and Nam D. Pham, Sonecon....................... 73
DIABETES RESEARCH: REDUCING THE
BURDEN OF DIABETES AT ALL AGES
AND STAGES
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WEDNESDAY, JULY 10, 2013
U.S. Senate,
Special Committee on Aging,
Washington, DC.
The Committee met, pursuant to notice, at 2:10 p.m., in
Room G-50, Dirksen Senate Office Building, Hon. Bill Nelson,
Chairman of the Committee, presiding.
Present: Senators Nelson, Blumenthal, Donnelly, Warren,
Collins, and Ayotte.
Also Present: Senator Shaheen.
OPENING STATEMENT OF SENATOR BILL NELSON, CHAIRMAN
The Chairman. The meeting will come to order.
For all of you, this is a Congressional hearing in the
Senate. We have these kinds of meetings, and as you can see,
this is a meeting room that is one of the large ones, and, of
course, we needed a large room today because of our special
guests, all in the blue shirts, and your parents, and this very
fine panel of witnesses that we are going to have.
I am Bill Nelson. I am from Florida. Susan Collins is from
Maine. We are in opposite parties, but that makes no
difference. We work together, and that is the way it ought to
be, and that is in a lot of contrast to what you see going on
around here with regard to a lot of other stuff.
I am very pleased that I have a colleague who works
together, and when we have differences, then what we do is we
respect each other's opinion and then we work out the
differences and we build consensus. And that is the essence of
trying to govern in a democracy that represents a big, large,
beautiful, diverse, and complicated country such as ours.
So, we want to welcome all of you, and this is a very
special topic today of which you are intimately familiar.
[The prepared statement of Chairman Nelson appears in the
Appendix on p. 68.]
The Chairman. And so I want to turn to our Ranking Member,
Senator Collins, for her statement.
OPENING STATEMENT OF SENATOR SUSAN M. COLLINS
Senator Collins. Thank you very much, Mr. Chairman. You put
it so well.
And when it comes to the issue of diabetes, it does not
matter whether you are a Democrat or a Republican or an
Independent or a Green Party member. What matters is that we
all work together to focus on the estimated three million
Americans with Type 1 diabetes and their families.
This is my seventh consecutive Children's Congress hearing,
and I am very grateful to Chairman Nelson for allowing me to
continue this tradition. I want to join him in welcoming our
distinguished witnesses and the more than 160 delegates to the
Children's Congress who have traveled to Washington to tell us
what it is like to have diabetes, just how serious it is, and
why it is so important that we fund the research that is
necessary to find a cure.
I particularly want to give a special welcome to the
delegate from Maine, 14-year-old Quinn Ferguson of Poland
Spring, who will be speaking on our panel.
As the founder and Co-Chair with Senator Jeanne Shaheen of
New Hampshire of the Senate Diabetes Caucus, I have learned a
lot about this disease and the heartbreak that it causes for so
many American families as they await a cure.
Diabetes is a lifelong condition that does not
discriminate. It affects people of every age, race, and
nationality. It is an extremely serious disease and it costs
the United States an estimated $245 billion a year and accounts
for one out of every three Medicare dollars. Because of the
serious complications associated with diabetes, medical costs
for Americans with the disease are 2.3 times higher than those
incurred by individuals without diabetes.
Those statistics alone are overwhelming, but what motivates
me to devote so much energy to this cause is meeting the
children, the family, whose lives have been forever changed by
diabetes. That is why it is so important that you have traveled
here to Washington to tell your stories. You put a human face
on the statistics.
When I was meeting with Quinn and he showed me his
scrapbook, he said that he felt that the day he was diagnosed
at age eight with diabetes was his last full day of freedom,
and that really touched me. So you help us focus on what
Congress can do to understand and ultimately conquer this
disease.
While often associated with children, the fact is that 85
percent of those living with Type 1 diabetes are adults and
many of them are seniors. An average individual with Type 1
will have to take more than 50,000 insulin shots or infusions
over his or her lifetime. The discovery of insulin was a
landmark breakthrough. However, it is not a cure for diabetes,
as you well know.
Thankfully, there is some good news. Since I founded the
Senate Diabetes Caucus, we have been successful in working
together in a bipartisan manner to triple the funding for
diabetes research. As a consequence, we have seen some
encouraging breakthroughs and we are on the threshold of a
number of exciting discoveries. Advances in technology, like
continuous glucose monitors, are helping patients control their
blood glucose levels, which is key to preventing the
complications from diabetes.
We are also moving closer and closer to the goal of an
artificial pancreas, which would revolutionize diabetes care.
Recent advances also include the development of new treatments
that can stop or even reverse certain complications, such as
some nerve damage and diabetic eye disease.
I am pleased to tell you that there is strong support in
Congress for diabetes research funding, thanks in no small
measure to the grassroots support provided by the JDRF
volunteers. Earlier this year, we passed legislation to extend
the Special Diabetes Program which provides $150 million a year
above and over the regular appropriations for Type 1 diabetes
research. This important program represents more than a third
of our Federal commitment to diabetes research. As such, it is
critical to our efforts to find better treatments, a means of
prevention, and ultimately what we are all seeking, and that is
a cure for this devastating disease.
Again, I thank the Chairman for holding this important
hearing and it is wonderful to see you all here. You really
inspire us to work even harder. Thank you.
[The prepared statement of Senator Collins appears in the
Appendix on p. 69.]
The Chairman. As a courtesy to all of you, overflow crowd,
standing, we will suspend any of our opening statements,
including the Chairman's. We will insert our opening statements
as a part of the official Congressional record.
And as a courtesy to you all standing, I want to invite you
to come up and have a seat here at the dais and at the staff
chairs, where available. I would not like any lady standing in
the audience. So, gentlemen----
[Laughter and applause.]
The Chairman. As Senator Donnelly says, if there is any guy
sitting down with a lady standing, the guy is going to get some
dirty looks.
[Laughter.]
Okay. First, we are going to hear from Jean Smart. Ms.
Smart is an Emmy winning television, film, stage actress who
was diagnosed with Type 1 diabetes at the age of 13.
And then we are going to hear from Ray Allen. He is a ten-
year NBA All-Star who currently plays, by the way----
[Laughter.]
And made the critical shot in Game Six----
[Applause.]
For the NBA Champions, the Miami Heat. He and his wife,
Shannon----
Senator Donnelly. Being from Indiana, can I object to that
shot?
[Laughter.]
The Chairman. Well, Indiana suffered, but the ones who
really suffered was San Antonio.
Ray's wife, Shannon, who serves on the JDRF's International
Board, they are parents to Walker, who was diagnosed with Type
1 diabetes at 17 months, and Walker is right there with us.
They will share the difficulties in managing Type 1 diabetes in
very young children.
And then we will hear from Quinn Ferguson, a JDRF
Children's Congress Delegate from Senator Collins's State of
Maine.
And then we are going to hear from Griffin Rodgers, the
Director of the National Institute of Diabetes and Digestive
and Kidney Disorders at NIH, the National Institutes of Health,
which is one of the most fantastic research centers, all trying
to do good in finding cures for the diseases that afflict us.
And then we will hear from Jeffrey Brewer. Mr. Brewer is
President and CEO of JDRF.
And so let us start with you, Ms. Smart.
STATEMENT OF JEAN SMART, ACTRESS
Ms. Smart. Senator Nelson, Senator Collins, and members of
the committee, thank you so much for allowing me the
opportunity to appear here today and talk about an issue that
is important to all these great kids and their families and
their guardians and I, myself, and that is the issue of living
life with Type 1 diabetes.
I was actually here in Washington, D.C. in 1965. I was 13
years old. Go ahead, do the math.
[Laughter.]
And I was waiting with my family to see the Tomb of the
Unknown Soldier and I was showing all the telltale signs of
Type 1 diabetes. I was so tired. I was so parched. I could not
drink enough liquid to slake my thirst. It was over 100 degrees
on that hot day in August, so my parents put it down to the
heat. But later, after stopping, I think, at almost every gas
station restroom on the drive home from D.C. to Seattle,
Washington--a long drive--and after losing almost 20 pounds in
a matter of weeks, my parents knew there was something
seriously wrong.
So shortly after returning home, I went to the doctor and I
was told I had Type 1 diabetes and I cried. I started to cry. I
remember the nurse--well, it was very shocking for my parents,
too, as you can imagine. I remember the nurse very quietly
offering smelling salts to my poor Daddy. He did not look too
good. Nobody in our family had diabetes. No one can remember of
anybody in the family ever having diabetes. And all I knew
about it was, at that age, that you had to take injections and
you could not eat sugar, which is quite a sentence to give to a
scared child.
But by the end of that day, I was actually giving myself
injections, and five days later, I was released from the
hospital, put on a very strict no-sugar diet. I was also told
that I should not consider having children, ever, and that was
a statement that would come back years later to haunt me.
But 48 years ago, there were no blood glucose meters. There
were no continuous glucose monitors. If you had diabetes, you
had to assess your blood sugar level by doing a urine test,
which was pretty inaccurate, but it was the best we had for
home testing at the time. The syringes were large. The needles
were big and heavy and they had to be reused, which made for
some pretty painful injections in my skinny little 13-year-old
thighs. Also, you had to boil them in a pot of water before
every use. Now, luckily, of course, the needles are much
smaller and finer.
But even with the advent of devices like the insulin pump,
it really is far from where we hope to be. My friend, Beverly,
has realized much more stable blood sugars on the pump, but she
still has some really frightening highs and lows if she is not
ever, ever vigilant.
I have never let diabetes define me. I never really thought
of myself as a sick person. But, ironically, probably one of
the main reasons I am a working actress today is because of
diabetes. I actually had wanted to go to college on the other
side of the State, to a college that did not offer a degree in
drama, but my mother had other thoughts. She really wanted me
to go to the University of Washington because it was very close
to home. And so I went to UW for five years. I got my BFA in
acting, which kind of launched my career.
So, I was busy working as an actress and in 1989, I
discovered I was pregnant. And my husband and I were quite
devastated when we were advised to terminate the pregnancy
because my blood sugars were not in good control, and we were
reminded of the harm that Type 1 diabetes could do to myself
and my baby. But I found a specialist who took me by the hand
and guided me through the next eight months. I tested my blood
sugar 12 times a day, with a meter at that point, fortunately.
I was on the phone daily with the specialist to adjust my
insulin dosages. And I was lucky enough, also, to have a
husband who was very supportive and turned himself into a
pregnancy diabetic specialist. And it was very, very hard work,
but thankfully for myself and my strong, healthy son, Connor,
it paid off.
I am sorry. I did not want to forget anything here.
But, unfortunately for a lot of people in my generation who
were diagnosed as kids with diabetes, they have had a high rate
of complications--heart disease, heart attacks, strokes,
blindness, kidney failure, nerve damage, very complicated
pregnancies.
I have had some very scary low blood sugars, and I am sure
the kids will identify what I am talking about. If you have
never had one, it is hard to describe. It feels to me a little
bit like drowning, actually. One close call I had, I was
actually on stage in front of about 900 people on Broadway,
opposite Mr. Nathan Lane--name dropping--and I knew I was in
trouble. It was very scary. And my mouth somehow kept saying
the words, but my brain was screaming, you are in big trouble,
and fortunately, that scene ended just in time. And ever since
then, I have stashed jelly beans all over every set I have ever
worked on. Years from now, they will find petrified jelly beans
in anyplace that I have ever worked.
[Laughter.]
You know, it is hard for me to remember what life was like
before I had diabetes, and sometimes, selfishly, of course, I
wish that I could just remember what that was like, to feel
what that was like, to not be diabetic, to go maybe a day
without being diabetic, to go more than just a couple of hours
without having to think about my blood sugar. But what I really
pray for is that the next generation of beautiful children like
these do not ever have to go through the uncertainty and the
fears of being diabetic, or the physical tolls that it can take
on their bodies.
You know, I never made it to the Tomb of the Unknown
Soldier that day. I did not have the strength. But because I
was lucky enough to be born in the second half of the 20th
century, and because of the advances in diabetes care, and
because Kay Smart was afraid to let me go away to college, I am
able to appear here before you and to thank you and to thank
Congress and to thank the JDRF for all you have done to promote
Type 1 diabetes research, and I ask that you please, please
continue your efforts so that one day, we can actually all
say--everybody in this room can say, remember that day that we
cured diabetes?
Thank you.
[Applause.]
[The prepared statement of Ms. Smart follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
The Chairman. By the way, you all may be wondering, why is
the Aging Committee having a hearing with so many young people
here?
Ms. Smart. It is because of me.
[Laughter.]
The Chairman. It is simply because the physical, the
economic, and the emotional toll of diabetes lasts throughout a
lifetime, and when one becomes a senior citizen, you really
start to see a lot of the impacts on the individual and
society.
Thank you, Ms. Smart.
Next, Mr. Allen.
STATEMENT OF RAY ALLEN, PLAYER, MIAMI HEAT, NATIONAL BASKETBALL
ASSOCIATION, AND FATHER OF WALKER ALLEN, JDRF CHILDREN'S
CONGRESS DELEGATE FROM FLORIDA
Mr. Allen. Thank you. Good afternoon, everybody. It is an
honor to be invited to appear before you here today to give you
an understanding of what it is like for a family to live with
Type 1 diabetes.
As you listen to our story, please know there is nothing
special about our family, my wife, Shannon, our son, Walker, or
me. Hundreds of thousands of other families across the United
States and the world could tell you a story similar to ours.
So, here is our story.
In 2008, I was a member of the Boston Celtics and we had a
great run that year and we found ourselves in the middle of
playing in the NBA Finals against the L.A. Lakers, the moment I
had dreamed of since I was a little boy. There I was, competing
for an NBA Championship. It was everything that I could ever
ask for. There were a lot of special things about that series.
It was the first time since 1986 that the Celtics won the NBA
Title. It was the most watched NBA telecast in NBA history.
And, personally, it was my first NBA Championship.
All of those things are very memorable, but what I remember
it for is for another reason. While our family was in L.A. for
Game 5, our son, Walker, who was 17 months old at the time,
became very ill. My wife, Shannon, rushed him to the hospital
where he was diagnosed with Type 1 diabetes. It was as if a rug
was pulled from underneath us. Life as we knew it was over. You
see, even though just a few days later we finished our journey,
winning Game 6 and being crowned an NBA Champion, another
journey began that we are still on today.
As you can see, Walker, he is six now, but he is still too
young to manage diabetes on his own. No disease is easy, but
managing Type 1 diabetes may be one of the most complicated and
complex responsibilities facing any caregiver or person living
with diabetes, or any disease, for that matter.
From the moment Walker wakes up in the morning until the
moment he goes to sleep, we have to monitor everything he
drinks. We have to test his blood sugar with finger pricks ten
times a day. And we have to count the amount of carbohydrates
he consumes. Then, do the mathematical equation required to
decide how much insulin to inject. This can be as many as seven
shots a day just so the blood glucose levels remain in the safe
range. Any miscalculation on our part could be life-threatening
for Walker.
You might think we finally can catch our breath when Walker
goes to bed. Not so. Type 1 diabetes can be at its most
dangerous at night, as you all know. Blood sugars that seem
fine at bedtime could suddenly come crashing down in the middle
of the night. Without juice or food to restore the balance,
Walker could drift into a coma and we would never know it until
we tried to wake him in the morning, and that might just be too
late. So we wake up every two hours throughout the night, on
the clock, to check Walker's blood sugars. Shannon and I often
joke that we are vampires. Neither one of us has slept through
the night in five years since his diagnosis. That is a reality
for all families and not just ours.
The Miami Heat, we played 106 games this past season. We
had game days, practice days, travel days, even a few off days,
days to rest, days to heal, days to rejuvenate, recharge. But
for Walker, here, and I know a lot of the kids here in this
room and a lot of the people that live with diabetes, there are
no off days.
As he gets older, Walker will gradually take more charge of
his Type 1 diabetes, but it will not get any easier because
more and more factors will impact the blood sugar levels--
exercise, stress, even normal changes in adolescent hormonal
activity, changes utterly beyond Walker's control. They will
all cause his blood sugars to gyrate wildly.
Shannon and I know from talking to other parents that
children and young adults with Type 1 diabetes show tremendous
courage, resolve, and steadfast determination not to let this
disease define them. But we also know that no matter how old
Walker is, no matter where he is or what he is doing every day,
he has to live a new day with Type 1 diabetes, and what works
one day may not work the next day, and the risks are always
ever present. I doubt Shannon and I will ever go a day without
just a little bit of fear and worry in the back of our minds,
fear for our son's safety and his overall health that reaches
his potential and is able to live his dreams.
It is not his fault that he has diabetes. There is nothing
he or we did to cause it. It is not diet or lifestyle related,
as is the case with Type 2 diabetes. Genetics is a part of it,
but there is no history of Type 1 diabetes in our family. We do
know this. Type 1 diabetes and autoimmune disease, the body
launches an attack on the cells that produce insulin,
eventually destroying them and leaving people with Type 1
diabetes dependent on synthetic insulin to survive. But insulin
is not a cure. It is a lifeline, and there is a big difference.
That is why Shannon and I got involved with the JDRF.
Shannon now serves on the Board of Directors, and we know that
JDRF is working on therapies that may reduce the tremendous
daily burden of living with Type 1 diabetes and that is working
towards a cure. As we know, JDRF is working with Congress to
make sure the government does its share and funds important
programs like the Special Diabetes Program.
On the basketball court, a lot of your success comes from
experience, hard work, repetition, practice, instinct, and, of
course, a little luck. A rebound careens off the rim, a
teammate grabs it and passes you the ball in the corner.
Without thinking, you catch it, step back, and you hit a
``three.'' Sometimes it happens that way.
[Laughter.]
It keeps your championship hopes alive. We will need more
than hard work, repetition, instinct, luck, practice, and
experience, though, to beat Type 1 diabetes.
I know Type 1 diabetes will never hold Walker back, but we
dream of a day when Walker can leave this disease behind with
the continued support of Congress for the Special Diabetes
Program, with the investment of JDRF and the private sector,
and the dedication and commitment all the families in this room
and all around America, all around the world, today, and
hundreds of thousands of other people in other countries, that
we will create a world without Type 1 diabetes. We have to,
because together, we are a winning team.
Thank you.
[Applause.]
[The prepared statement of Mr. Allen follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
The Chairman. And, Walker, we are glad that you are here,
too. Thank you for bringing your Dad here today.
Mr. Allen. I am glad to be here.
The Chairman. Okay. Quinn Ferguson. Now, you are a
representative of all of these blue shirts out here, so tell us
your story.
STATEMENT OF QUINN FERGUSON, JDRF CHILDREN'S CONGRESS DELEGATE
FROM POLAND SPRINGS, ME
Mr. Ferguson. Thank you, Senator Collins, Senator Nelson,
and members of the Aging Committee, for inviting me to testify.
My name is Quinn Ferguson. I am 14 years old and I am from
historic Poland Spring, Maine. I have been living with Type 1
diabetes since I was diagnosed a week before my ninth birthday.
I will never forget the day I was diagnosed. Actually, I
was misdiagnosed. I was at my grandparent's house when I passed
out and had fallen on the ground, so my Mom drove me
immediately to the hospital. On the way, I started throwing up,
but my doctor said it was a concussion and sent me home.
A week later, I was still not feeling better. After looking
online to find out what was causing my symptoms, my Nana
brought over my Grandpa's diabetes test kit. He has Type 2
diabetes. My blood sugar was over 600, more than four times the
normal range. After that, I was diagnosed with Type 1 diabetes,
or T1D.
Voices have been silenced and lives have been cut short
because of this disease. I am here today to speak for them as
well as myself, and I am not alone in my story. People are
misdiagnosed every day or not diagnosed at all, suffering the
consequences and sometimes paying the ultimate price. I am here
today because they cannot be and because we need to do more
about this disease.
Every day since I was diagnosed, I have had to check my
blood sugar at least ten times a day, even in the middle of the
night. I measure everything I eat and drink and I think about
my blood sugars constantly, when I am in school, on the
football field, or in a chess match. If I do not, I could
experience a seizure or a coma or suffer from long-term
complications like eye disease, kidney failure, and heart
problems.
I am fully responsible for my diabetes, from caring for
myself to taking charge of my attitude on T1D. While there are
times I get down, I am not out. I am a strong person for living
with this disease, but every day is a trial by fire. I will
never have a day off, and even as a teenager, I will never
outgrow T1D.
Thanks to medical research, life will get better. It has
to. I am not giving up on a cure and hope Congress will not,
either, and will continue to support the Special Diabetes
Program, my hope for a cure. While we wait for a cure, I am
doing my part by educating others about T1D and enrolled in a
TrialNet study that tests the drug Abatacept to stop or slow
down destruction of insulin-producing beta cells. Those newly
diagnosed with T1D who got the drug produced insulin longer
than people who did not for almost one year. The study is now
testing the drug in people at risk, but not yet diagnosed, to
see if it can help delay or prevent T1D. Having a relative
greatly increases the chance of being diagnosed. Without the
SDP, TrialNet studies of almost 20,000 patients will not see
results.
The SDP led us to the artificial pancreas technology now
being used in--tested in T1D patients. These patients are
living my dream. They do not have to worry constantly about
blood sugars and can sleep through the night. The artificial
pancreas is taking the guess work out of managing the disease
by automatically testing blood sugars and giving insulin as
needed. It will help keep people with T1D safe and healthy
until a cure is found.
My Great-Grandfather Alfred Clark Ferguson came to the New
World on an orphan ship when he was five years old. At that
age, he could only dream of a future where the sugars would
never take parents away from their children again. He lived to
see science find a treatment for diabetes. Whether my
generation lives to see a cure depends on research and funding.
We need your help.
Today, I am surrounded by 160 delegates and their parents
who represent the millions of people doing everything possible
for a cure. I am grateful that Senator Collins and so many in
Congress have been so supportive and I urge you to keep it up.
Our hope for a full life depends on it. While the worry never
goes away, we know better days are ahead. That is why we need
your support for the Special Diabetes Program, so that maybe
one day we can say that the world is free from T1D.
Thank you.
[Applause.]
[The prepared statement of Mr. Ferguson follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
The Chairman. Thank you, Quinn.
I still see some ladies standing back there, one there, one
there, and I see one over here, and we have plenty of seats
here, so please avail yourselves of the opportunity.
Okay, Dr. Rodgers. Tell us what is happening at NIH.
STATEMENT OF GRIFFIN P. RODGERS, M.D., DIRECTOR, NATIONAL
INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISORDERS,
NATIONAL INSTITUTES OF HEALTH, U.S. DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Dr. Rodgers. Thank you. Chairman Nelson, Senator Collins,
members of the committee, thank you for giving me the
opportunity to testify today.
On behalf of the National Institutes of Health, I am
pleased to report that our investment in Type 1 diabetes
research continues to improve lives around the country and
around the world. Through coordinated efforts with research
partners such as the JDRF and the ADA, as well as continuous
support from the special statutory funding program for Type 1
diabetes research, we are helping people with Type 1 diabetes,
such as the children sitting here today, live longer and
healthier lives.
First, I would like to acknowledge the important
contribution of my fellow witnesses. Ray Allen, you are a
champion in basketball, but you and your family--your entire
family--are truly champions in promoting efforts toward curing
this disease.
I am also pleased to share the table with Jeffrey Brewer
and Jean Smart, who I know share our goals of preventing,
treating, and ultimately curing Type 1 diabetes.
I would also like to thank those here today representing
Americans of all ages with Type 1 diabetes. Quinn, the clinical
trial that you and other young people here today have
participated in could not occur without your support. Our
research is your research and we thank you for making it
possible.
The future for those with Type 1 diabetes is brighter than
ever. NIDDK-supported research has found that life expectancy
for people with diabetes has increased by 15 years, and this
increase is largely due to advances in technology and
understanding that early glucose control is critical to
reducing risk of long-term complications. This key insight was
provided by the NIDDK's landmark Diabetes Control and
Complication Trial, or DCCT, and its follow-up study called
EDIC, E-D-I-C. Everything in government has an acronym.
[Laughter.]
This year, DCCT/EDIC celebrated its 30th anniversary, and
we celebrate the great contributions to health of those with
Type 1 diabetes.
Controlling blood glucose, as you all know, is very
challenging, so it is critical that we continue the search for
ways to prevent, treat, and cure diabetes. And we are, thus,
focusing research on every stage of Type 1 diabetes
development.
The first stage is the onset of auto-immunity, a stage
where no symptoms are apparent, but where insulin-producing
beta cells are being slowly destroyed. We have long sought to
better understand what causes auto-immunity and now know that
both genetic and environmental factors play a role. We support
various efforts, including a major international study
involving thousands of children, to better understand these
factors and we look forward to translating our discoveries into
new treatments to prevent not only Type 1 diabetes, but other
auto-immune diseases.
Stopping the auto-immune attack once it has begun is
another focus of NIH research. The NIDDK's Type 1 Diabetes
TrialNet and the NIAID's Immune Tolerance Network have
identified several treatments to protect the beta cells, and
studies to extend the effects of those promising results are
ongoing.
Replacing or restoring the function of beta cells is
another area of great interest, and one strategy is to replace
the beta cells via islet transplantation, and recent progress
in this area was largely made possible by NIDDK and the NIAID
co-led Clinical Islet Transplantation Consortium. Today, islet
transplant recipients are more likely to not need insulin at
some point after their transplant. Their insulin independence
lasts much longer. And they are less likely to have severe side
effects than their peers receiving islet transplants just a
decade ago.
We also plan to build upon the success of the NIDDK's Beta
Cell Biology Consortium by creating the Human Islet Research
Network, which will focus on improving methods to create beta
cells and to extend their survival.
Now, we know that self-managing one's blood glucose levels
is critical to long-term health, but it is also a huge burden,
as you have heard from the witnesses already. A promising
approach to ease this burden is an artificial pancreas, a
device that can automatically sense blood glucose levels and
administer insulin accordingly, and there has been tremendous
progress in this area in recent years.
Preliminary studies indicate that these devices can keep
blood glucose levels within an optimal range and allow the
blood glucose to stay in control, much better than self-managed
insulin pumps. Ongoing trials, such as devices in the real
world setting, are also yielding extremely promising results.
With continued research, artificial pancreas technology has
tremendous potential to allow people with Type 1 diabetes to
live, eat, and exercise freely without the fear of
hypoglycemia.
The NIH also vigorously supports research to prevent and
treat diabetes complications. For example, progress has been
made by the Diabetic Retinopathy Clinical Research Network led
by the NEI with support from the Special Diabetes Program.
Research conducted through this network contributed to the
recent FDA approval of a treatment for a serious diabetic eye
complication, progress that has been hailed as the most
important advanced treatment for diabetes retinopathy in the
last 25 years.
Chairman Nelson, Senator Collins, members of the committee,
thank you for this opportunity to speak today, and we are
grateful for the continued support of Congress and for our
public and private research partners and for the inspiring
efforts of our clinical trial volunteers. Together, we strive
to allow those of all ages with Type 1 diabetes to live longer,
healthier lives, free of the burden of this disease.
Thank you for your attention. I will be pleased to answer
any questions that you might have.
[The prepared statement of Dr. Rodgers follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
The Chairman. Thank you, Dr. Rodgers.
[Applause.]
Okay, Mr. Brewer. You brought along a lot of your
colleagues today.
Mr. Brewer. I sure did.
The Chairman. Share with us, please.
STATEMENT OF JEFFREY BREWER, PRESIDENT AND CHIEF EXECUTIVE
OFFICER, JDRF
Mr. Brewer. Chairman Nelson, Senator Collins, members of
the committee, I greatly appreciate the opportunity to be here
today. My name is Jeffrey Brewer and I am the President and CEO
of JDRF. As an entrepreneur and the father of a son with Type 1
diabetes, it is my great honor to lead the world's largest
charitable funder of Type 1 diabetes research and to partner
with the public and private sectors to drive progress toward
our goal of a world without Type 1 diabetes.
I want to begin by thanking all the Senators who are here
today for your great leadership in diabetes research. All of us
at JDRF were extremely grateful in January when the Congress
included a one-year, $150 million extension of the Special
Diabetes Program. The SDP funds critical research upon which
the private sector relies to identify in advance new therapies
for Type 1 diabetes. Thanks to the SDP, scientists have
identified more than 50 genes that influence a person's risk of
developing Type 1 diabetes, and researchers are halfway through
a 15-year study to identify what factors trigger its onset in
the first place. At the same time, the SDP has helped advance
important new therapies, including one that is available to
patients today, one that can help reverse vision loss related
to diabetes.
Last year, 72 Senators signed a letter in support of the
Special Diabetes Program, and I would like to say a special
thanks to Senate Diabetes Caucus Co-Chairs Collins and Shaheen,
who are here today. Thank you very much for leading this
effort.
As many of you know, Type 1 diabetes, also known
increasingly as T1D, is a life-threatening disease that creates
constant challenges for Americans of all ages. At every meal
and many other times throughout the day and night, people with
T1D must test their blood sugar and try to give the right
amount of insulin, always at the right time. Too little insulin
and high blood sugar will cause devastating complications. Too
much insulin and low blood sugar will cause seizures, coma, and
sometimes death, unfortunately.
In my family, my wife had to watch in horror as paramedics
rushed into our home to rescue our son, who had lapsed into a
coma as a result of too much insulin. Firefighters had to break
down his door with an axe and rush him to the hospital, where
he spent 48 hours in the ICU before recovering full
consciousness. Living with T1D is a daily high-stakes
challenge.
Diabetes has taken a toll on my family, the Children's
Congress families here today, and many others around the world.
It has also taken a toll on our nation, costing $245 billion in
medical and economic expenses last year. Diabetes costs are
rising rapidly, and according to a new study released today,
the costs of diabetes are expected to more than double in the
next eight years. The overall costs are expected to rise from
$245 billion in 2012 to $512 billion in 2020. Costs to Medicare
due to diabetes will also double, rising from $104 billion to
$226 billion in 2020. As our population ages in the coming
years, what is currently a significant problem is going to
become a fiscal crisis.
Diabetes is a health crisis for patients and their families
and a financial crisis for every American. Therefore, all of
us, especially older Americans, have a serious stake in
diabetes research. Research investments offer the only real
hope for reversing these troubling trends.
Today, due to public and private research partnerships,
there are new therapies in the pipeline that show great promise
in improving the health of people with diabetes and reducing
the costs to our nation. For example, in the last year, for the
first time, people with T1D in the United States have worn
artificial pancreas systems outside of the hospital as a part
of groundbreaking research studies. In these studies, people
with T1D have gone about daily life, going for walks, out to
eat to restaurants, and to work with experimental technologies
that automatically controlled their blood sugar. Less burden,
better glucose control--it is not a cure, but it is great
progress, progress made possible by JDRF, the National
Institutes of Health, the Food and Drug Administration, various
private sector companies, other research foundations, and all
of you here today.
And with great progress, there is great potential for
fiscal savings. For example, researchers have found that
intensive glucose control over six-and-a-half years can cut in
half the onset of kidney disease, and a 50 percent reduction in
end-stage renal disease could save Medicare $126 billion over a
term of 25 years.
With your leadership and the public-private partnership for
diabetes research, potential therapies could transform millions
of lives and improve the fiscal health of our nation. But we
must move forward with urgency and with a long-term commitment
to the investments and research that are needed to finish the
job.
JDRF is funding ourselves more than $100 million in
research this year and we currently have more than $500 million
deployed in research programs at work today around the world.
JDRF and our many volunteers will keep doing our part. But if
we are to continue to make progress to advance towards
desperately needed new therapies to cure, treat, and prevent
T1D, we need the Federal Government to continue to do its part
and that requires a long-term commitment to ensure that vital
multi-year research proceeds without interruption.
I urge the Congress to fund a three-year extension of the
Special Diabetes Program this year at the current funding
level. We must make the promise of life saving and cost saving
diabetes therapies a reality. We can make this future happen.
We need your help.
Thank you, and I would be happy to answer any questions you
may have.
[Applause.]
[The prepared statement of Mr. Brewer follows:]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
The Chairman. Thank you, Mr. Brewer.
Now, for our young friends, normally, what happens, we get
into the questions. The Chairman will ask questions, the
Ranking Member, and then according to the order in which the
members appeared, then they are recognized. Because of my
colleagues being so smart and so interested in all of this, I
will defer my questions and I will do a wrap-up. So I want to
call first on Senator Collins.
Senator Collins. Thank you once again, Mr. Chairman.
Dr. Rodgers, when Quinn was talking to me prior to the
hearing, he mentioned that when he was diagnosed at age eight,
he was the only person in his community with Type 1 diabetes.
Now, he tells me, there are 12 or 13 children. I am very
alarmed by a recent CDC study that shows that Type 1 diabetes
among young people rose by a troubling 23 percent between 2001
and 2009. Do researchers have any idea why this increase?
Dr. Rodgers. Thanks for that question, Senator Collins.
First, I would point out that the reason that we know that it
has increased over 20 percent in that period of time is
directly due to the Special Diabetes Program. The Special
Diabetes Program has enabled us through a program called SEARCH
in which the NIH works in conjunction with the CDC in several
cities around the United States to monitor the onset of
diabetes in youth. And in that group, there has been not only
an increase in Type 1 diabetes at a younger age, as you
indicated, but also Type 2 diabetes among adolescents and
youth.
We know, as I indicated, that diabetes, especially Type 1
diabetes, is a result of a combination of genetic and
environmental factors. It is quite likely that our genes have
not changed over this short period of time, strongly indicating
that it is quite likely some environmental factor that is
increasing that is associated with that increased incidence of
Type 1 diabetes.
And this long-term investment made possible by the Special
Diabetes Program, a study called TEDDY, The Environmental
Determinants of Diabetes of the Youth, in which we screen over
425,000 kids and we have enrolled 8,000 kids who are about
halfway through the study. The hope there is to actually learn
what are those environmental factors that trigger the
autoimmune response to this disease.
If it turns out that it is a virus or a collection of
viruses, then we would be prepared to develop vaccines to
prevent this in people at youth. If it turns out that it is a
dietary factor, then, obviously, avoidance of that would be
important. But it is with the funding of the Special Diabetes
Program that we have been able to both monitor the numbers of
kids as well as be in a position to now determine what those
environmental factors are.
Senator Collins. That is, indeed, absolutely critical
research.
I know that everyone in this room is very interested in
development of an artificial pancreas and much progress has
been made in moving the clinical trials from hospital settings
to diabetes camps and real world settings. Could you give us
some idea on when you think the artificial pancreas will be
more generally available.
Dr. Rodgers. Sure. Well, I would be pleased to comment on
the research progress in this area. Obviously, I will have to
defer to my colleagues at the FDA to provide information on the
regulatory aspects of this.
What I can say is that the FDA issued final guidance on the
low-glucose suspend system, actually, in June of 2011, just a
few weeks two years ago as a result of this meeting, as you
remember, and for the development of an artificial pancreas
system, they issued guidance in November of 2012.
What I would say is that there has been really strong
research findings recently in trials conducted in Europe, in
Australia, as well as in the United States that were recently
published in the last six to eight months in the prestigious
New England Journal of Medicine. These trials showed that this
system can reduce the episodes of hypoglycemia, especially at
night, which is especially worrisome, as you have heard from
Ray Allen and Jeff Brewer as well as from other parents in this
room.
This is an important finding, because we know that the
threat of hypoglycemia is oftentimes what prevents people from
very closely managing their blood sugar in tight control, which
we now know definitely prevents long-term complications of the
disease.
From a research perspective, I can say that the NIDDK
continues its vigorous support for this artificial pancreas,
both with the funds that we have in fiscal year 2013 and
because the committee allowed us to move forward in fiscal year
2014. We will continue the strong support for this artificial
pancreas in those years, as well.
Senator Collins. Thank you, Doctor, and I want to thank all
of the witnesses. You are absolutely terrific.
The Chairman. Thank you, Senator Collins.
Senator Shaheen.
Senator Shaheen. Thank you very much, Mr. Chairman and
Ranking Member Collins and Senator Warren, for allowing me to
go next.
I very much appreciate the inspiring and compelling
testimony from each of you on the panel, and I want to thank
all of the delegates who are here with the Children's Congress.
Your effort here today really makes a difference in educating
us in Congress about what it is like living with Type 1
diabetes and how important it is for us to look at the policy
level on things that we can do to help address Type 1 diabetes.
I have a personal window into what it is like to live with
Type 1 diabetes because my oldest granddaughter has Type 1. She
was diagnosed a little after she turned eight. So I have
watched very directly the challenges that you all face and so
appreciate your being here. Thank you very much.
Dr. Rodgers, you and Mr. Brewer and, really, everyone on
the panel has talked about the importance of making investments
that can help us find a cure for Type 1. Can you talk about
what the impact of sequestration has been on the efforts
currently underway at NIH with the Special Diabetes Fund and
other efforts to try and look for therapies and a cure for
diabetes.
Dr. Rodgers. What I can say is that, obviously, the more
funds that we have available, obviously, we can provide
progress much more rapidly. We attempt in a strategic manner to
bring together external experts to sort of tell us about what
it is that is critically important to move forward with. With
the possibility that funds will be limited, what would be the
first studies that one might consider amplifying and what
studies, of course, would either have to be scaled back or
stopped completely?
The efforts for clinical research, such as the ones
involving the artificial pancreas, or in the use of trials of
drugs that may either prevent or delay the destruction of the
beta cells, are the types of studies that we would be, from an
ethical standpoint, not good judgment to move forward with if
you are uncertain about having future funds. And so in
opportunities in which funds are cut back, one really has to
think, despite the fact that these are high priority areas,
think very strongly about committing families and patients to
getting involved in clinical trials that you may or may not be
able to complete.
Senator Shaheen. Well, let me ask the question a little
differently.
Dr. Rodgers. Sure.
Senator Shaheen. What would happen if we do not fund the
Special Diabetes Program this year, if we do not fund it for
another year, and if we do not fund it for the three years that
you are suggesting we should do?
Dr. Rodgers. Well, let me answer that question by telling
you about the progress that has been made when the committee
has been able to give us three years of funding. The TEDDY
program, The Environmental Determinants of Diabetes in the
Young, is a program that, because of the long-term nature of
it, we were able to begin it, and now with the renewal, we have
been able to continue it. And TEDDY has really put us in the
position now to determine what are those risk factors and how
we might intervene.
TrialNet, the trial that Quinn is on, is another one of
these long-term trials, and once we have an idea to move
forward, TrialNet has allowed us the infrastructure to begin to
address some of those questions about promising treatments.
Those types of trials would be in jeopardy, those that are sort
of next up, without a commitment for longer-term research
funding.
Senator Shaheen. Thank you.
Dr. Rodgers. Thank you.
Senator Shaheen. Mr. Allen and Mr. Brewer, too, as parents
of children with Type 1 diabetes, what would you like us as
policymakers to go away from this hearing understanding and
what action would you like us to take?
Mr. Allen. Well, first and foremost, we live with this
disease every day. You know, when we look at Walker every day
and he stands up and we have to give him a shot and every day
he says he wants this disease to stop, he wants to not have to
take a shot, and his brothers run outside and go play and he
has to stay behind and he has to take a shot, he looks at us
and he says, you know, ``When is this going to end? When can we
stop doing this?''
So we say we are going to do everything we can in our power
to make this disease cease, so we are here today because we
want you to understand that this is the face of our children,
you know, all these kids in here, and they all feel the same
way every single day, seven to ten times a day. So we are
advocating for them so you understand. I am sure you have
people living with diabetes every day. So when you walk away
from this, you understand that these are innocent children who
had nothing to do with getting diabetes, and we want you to
understand that as you make your decisions, knowing that these
are our children and they are the future of our universe and
future of our world.
Senator Shaheen. Thank you very much.
Mr. Brewer. I have a unique perspective as a father of a
child with the disease but also the CEO of this organization.
So I have the sense of the opportunities that we have before us
to solve this problem. There are concrete opportunities that
can improve the lives of people in a very substantial way, to
help them to live safer and easier lives and avoid some of the
adverse events that you heard about today. And they are really
lying right before us. They are lying right before us as a
result of previous research funding, JDRF having funded $1.7
billion of research over the last 43 years and the NIH having
funded untold billions dollars of research.
We have been brought to a point where we can translate
these scientific discoveries through to really meaningful
impact on people living with this disease--children, adults,
everybody living with the disease. It is going to take some
more research funding, but it is also going to take some very
close look at the regulatory challenges that are involved in
translating therapies, testing therapies for safety and
efficacy in order to deliver them through.
We need to take a look at the reimbursement paradigm for
how Type 1 technologies for treatment are paid for, because
there is a tremendous cost savings to prevent some of these
catastrophic events that we are talking about. Emergency room
visits are very expensive. The complications of Type 1 diabetes
later in life are very expensive and burdensome on the medical
system. And yet, we have such promise and opportunity to really
change those outcomes.
So there is a fiscal imperative here. There is an
imperative to look at how our system translates these
scientific discoveries into technologies that can be used here
and around the world where we have historically been a leader
and where, frankly, we are now falling behind because of some
structural challenges in this pipeline.
Senator Shaheen. Thank you very much. Thank you, Mr.
Chairman.
The Chairman. Dr. Rodgers, directly to Senator Shaheen's
question, the continued existence of this program, is it not
absolutely critical to continue your efforts to fight the
disease?
Dr. Rodgers. I could not say it better, Senator. Thanks for
the question. It is. It is critically important. We have a
range of areas not only in the course of the disease that I
have tried to outline during my oral statement, but also a
range from very basic research. We are learning quite a bit
about how one could actually tease cells that are in the
pancreas, that exist, and transfer them into beta-producing
cells, and these discoveries which have now been found in
animal models but also their equivalents exist in humans, would
not have been possible. And we are on the precipice of
beginning to translate these findings into clinical research,
translational research as well as clinical research. All of
this work over and above the regular appropriations that we get
would not be possible without the Special Diabetes Program.
The Chairman. Senator Warren.
Senator Warren. Thank you, Mr. Chairman, and I want to say
to all of you at the Children's Congress, thank you very much
for being here. You know, this is what democracy is about.
People here in Washington will decide how we spend money and
how much money goes into research for diabetes and for other
medical problems. And so your coming here and making the case
for why it is important that we put money into research for
diabetes is really important. I am really glad you are here. I
think you make a real difference.
I also want to say to Senator Collins and to Senator
Shaheen, thank you very much for your leadership in creating a
caucus that has made a real difference. I am a new Senator. I
am just getting started, so I am learning about this. But they
have shown great leadership in making sure that there is better
funding, better support, better awareness here in Washington
for the issues surrounding diabetes.
So, thanks for being here today. I also want to say to our
panel, a terrific panel. Thank you so much. I am grateful to
all of you for being here.
There are a couple of questions I want to follow up on. I
want to start with you, Dr. Rodgers. This is not just about the
level of funding, which I understand is critically important,
but it is also the long-term commitment on funding. And what I
have observed is that NIH funding, generally, and diabetes
funding, has been getting shorter and shorter and shorter. That
is, you cannot be sure the money is coming until just before
you get it and it is for a short period of time. I suspect that
has an impact on the research and on attracting more people to
do more research in this field. Could you speak about that just
a little bit, Dr. Rodgers.
Dr. Rodgers. Thank you, Senator. You are absolutely right
in your observations. I think when one--first of all, let me
start off by saying that the investments are already paying off
as clearly shown in these critical studies, the Diabetes
Control and Complication Trial. People are living longer and
healthier lives than even a decade ago. And so if future
promise--anything about past successes really point out that we
are really at that point in which we are about to make major
milestones and landmarks.
Now, specifically to your question about the duration of
funding, as one is managing programs, one has to think about
what are the feasible programs that one can begin. Clinical
trials are such that without having an assurance for long-term
funding, it would be, in a sense, unethical to put people into
trials with all that is involved in the trials without knowing
that the funding will continue through a period of time to see
it successfully completed. And that is why the three-year
funding that Mr. Brewer suggests, on the longer-term, makes
both planning and management of clinical trials and clinical
studies so vitally important.
When we get one year of funding, of course, we appreciate
it and we try to manage it in a way that we can achieve it. But
having longer-term trials, or longer-term funding horizons
allows us not only to complete ongoing trials or ancillary
trials to those trials to get really the best return on the
investment, but also to begin the planning of longer-term
trials as our external experts help us with prioritizing them.
Senator Warren. Thank you. That is very helpful, Dr.
Rodgers.
You know, as much as we talk about our funding through
NIH--and please count me as a supporter of substantially
increasing our funding--it is also important, though, to talk
about the investments that are made by the private sector in
new technologies and making living with diabetes easier. And I
am very proud, of course, to be from Massachusetts, where we
are not only making great progress on the research, the front-
end part with NIH and other support, but also that we have a
medical device, pharmacology, biotechnology that we are working
on in Massachusetts to try to help people who are currently
living with diabetes.
And you raised the question about the Special Diabetes
Program. I understand there are clinical trials going on, and I
understand, Mr. Ferguson, you have been a participant in the
clinical trials, is that right?
Mr. Ferguson. Yeah.
[Laughter.]
Senator Warren. Okay. He gets to the point.
[Laughter.]
So I have a question for you. Would you mind, just for a
minute, talking a little bit about what it is like to
participate in a clinical trial on diabetes?
Mr. Ferguson. Well, it is just a great experience. Even
though you are going down for a trial, it is, like, I have got
to go down every month to get the drug Abatacept tested, so
they would--like, I get an IV and then they put in water first
to make sure I was hydrated and then they would start putting
Abatacept in. And I have got to go down to Boston every time
with my father, so--and it is really cool. You get to see
really smart people, minds like no others, and--yeah.
Senator Warren. All right.
[Laughter.]
Thanks, Quinn. But, you know, you are out there making a
difference, and you are making a difference for everybody,
everybody who has got diabetes now and people who develop it in
the future.
But the question, then, that I want to ask Dr. Rodgers and
Mr. Brewer, is if you can just speak briefly to the issue about
barriers that are in the way of getting people into clinical
trials. Mr. Brewer, would you like to start.
Mr. Brewer. Well, there are barriers in the way of
information getting out to people about what clinical trials
there are. We probably do not have enough clinical trial sites
to be able to provide opportunities outside of concentrations
like we have in Massachusetts of these kinds of resources. And,
frankly, we just do not have enough things to test right now,
and that is why we need the research funding, because the
pipeline that is created by this research funding, the Special
Diabetes Program, other NIH funding, and the funding that JDRF
does, we are the feeder for those clinical trials.
Senator Warren. Dr. Rodgers, would you like to add anything
to that?
Dr. Rodgers. I would agree with that, and I would just
emphasize that we--just because of the unawareness of the
availability of participating in trial, we are trying to
explore other avenues, such as using social media, for example,
to have--instead of having doctors recommend patients, actually
going directly to the patient communities, and we are working
in conjunction with the JDRF to try to use social media to get
patients to participate, perhaps who live in areas that are not
heavily concentrated with researchers.
Senator Warren. Well, that is very valuable. I just want to
say, it is so impressive to me when you meet young people who
are managing diabetes. They are not letting diabetes manage
them. They are managing it and taking responsibility. And then
taking that extra step to join in in part of how you find the
cure, being part of the research and the clinical trial for it,
good for you, Quinn. So, thank you all very much.
Thank you, Mr. Chairman.
The Chairman. Senator Donnelly.
Senator Donnelly. Thank you, Mr. Chairman.
It is an honor to have all of you here. I not only have the
opportunity to represent Indiana in the United States Senate,
but am part of a family that has been touched by Type 1
diabetes, and so I know every day what you deal with, as well--
not me personally, but a family member. My pledge to you, as I
know everybody is up here, is that we will beat this. We will
find a cure. We will take every step necessary along the way.
And I want you to all know that you have an incredible amount
of advocates here on this side who will fight for the funding
that you need to make sure that you have that three-year window
that you need and to--economically, it makes sense, as well,
that when we cure this, we not only make everybody have a
chance to be more productive, but we save so much funds, as
well.
But to all of you, what we want to do is make it so you
never have to think about this, so that as you live your life,
this is something that you can say we beat, we cured, and it is
over with, and that is the goal of what we are trying to
accomplish here.
And I want to thank, also, our three participants from the
Children's Congress who are from Indiana, Rebecca Moody, Claire
Dunagan, and Gwen Wahler [phonetic], and just for all of you to
know, another Indiana resident who is here, my friend Charlie,
is an Indy car driver, and----
[Applause.]
Charlie actually almost won the Pocono Race this past
weekend. Charlie has Type 1 diabetes and is an Indy car driver
and almost won the Indianapolis 500. If it was for the amount
of cheers given, he would have won the Indy 500.
But what I want to also do is ask you, Dr. Rodgers, in
terms of an artificial pancreas, how far are we from--and,
obviously, not an exact date--but how far are we from a time
when we will have this available to all?
Dr. Rodgers. Again, as I--trying to answer this, I can tell
you really more about the research and the great progress, and
I would have to say that--again, I would leave it to my
colleagues at the FDA to discuss the regulatory aspects of it,
but there really have been great advances within the last--and
published within the last few months and presented at the
American Diabetes Meeting just last month--about these devices.
They are much smaller. They allow for ways of actually
suspending insulin injection when the glucose level reaches a
critical point. Less frequent episodes of hypoglycemia at
night, which is really the critical time that people worry
about. There was concern, for example, from the FDA's
perspective not only that the threshold was not reached and
people would get seriously hypoglycemic, which was not shown,
but as you suspend the insulin, of course, the glucose will
begin to rise. And what these studies and presentation clearly
show, that those episodes of hyperglycemia and its associated
ketone production did not occur.
So from a safety perspective, trials here in the U.S. as
well as in Europe and Australia are clearly pointing to a point
where we are about to make major breakthroughs and allow this
for more children to be able to utilize this----
The Chairman. If the Senator would yield, would you
describe for the committee what the device looks like?
Dr. Rodgers. Sure. An artificial pancreas puts together two
pieces of instrumentation, one that can continuously measure
blood glucose levels, so a continuous glucose monitor. That
glucose monitor sends signals to an insulin pump, just as your
pancreas would. Your pancreas senses when there is too much
sugar. It puts out more insulin. When the sugar drops too low,
it puts out less insulin, and in certain instances, glucagon.
And the other aspect is the pump. These are now connected
by a computerized system which now the technology is available
that even on a smart phone, one can link these two together.
So this particular aspect, this insulin suspend, is a
situation in which a glucose monitor indicates that the glucose
levels are trending too low and, therefore, the insulin
infusion in the pump is suspended, and as I said, keeps you in
that critical window so that the blood sugar does not drop too
low or too high.
There has been a recent report, actually, from--and that is
done because it actually monitors what the glucose is. There is
a second major advance in which one uses mathematical
algorithms to actually predict, based upon the rate at which
the glucose is falling, when it is likely to reach that
critical threshold, and this is why you have to bring engineers
and mathematicians into this, as well. And this has actually
shown great promise, as well.
So, to answer your question, I cannot predict precisely,
but I think that we are really on the precipice of this.
Senator Donnelly. Just outside of--obviously, the FDA is
critical and has an important role, but outside of their role,
would you say the technology is basically just about there at
this point? Obviously, we have safety tests to do, but the
technology itself is there?
Dr. Rodgers. I think the components for the technology are
certainly there. We can always do better. For example, I
mentioned that the current artificial pancreas is actually
infusing insulin. Now, to completely replicate the pancreas,
one might do better with both insulin and glucagon, which is
what the pancreas does, so a so-called bi-hormonal pump. And,
obviously, one would have to, in a clinical setting, do trials
to indicate the superiority of that over others.
There are certainly areas in which we can improve upon, but
generally, the technologies have greatly advanced within the
last few years, and so I think we are certainly close to being
there.
Senator Donnelly. I just want to ask one other question,
and that is in regards to islet, that you discussed. There are
efforts on islet transplants that are being done. Have you
looked at the area of epigenetics and their possible assistance
in regards to making the islets work better?
Dr. Rodgers. Epigenetics refers to changes in the DNA
structure, either by the addition of methyl groups or others,
that can critically influence the expression of genes leading
to certain proteins, and that is certainly an area in which we
are critically--we are actively involved in research findings.
One example of the importance of epigenetics is actually
potentially in the development of diabetes. We talk about this
gene environment interaction, but what we know, for example, is
that the first environment that a developing infant is exposed
to is actually the interuterine environment, and it is quite
likely, and we certainly now know through a number of studies,
that a woman who has diabetes, who develops in this case Type 2
diabetes, the likelihood of that infant born from that
pregnancy has a greater risk of developing diabetes sometimes
later in life than a sibling born to that mother under the
situation in which she did not have diabetes. And it is thought
that epigenetics plays an important role.
Epigenetics is a way in which the environment makes marks
upon your existing genes that can change the development of
cells. So that is an important area that we are looking at.
Senator Donnelly. I want to thank all of you for being
here, and to all of you young people, we promise we will be
never ending in our efforts to cure this and to solve this so
you never have to worry about it. And when one of you is the
future President of the United States, I want to come visit you
in the White House.
[Applause.]
The Chairman. Thank you, Senator Donnelly.
Senator Blumenthal.
Senator Blumenthal. Thank you, Chairman Nelson, and thank
you for holding this hearing. I want to join in expressing my
gratitude to you for giving us this wonderful opportunity to
express our determination, as my colleague, Senator Donnelly,
has so eloquently and powerfully, that we will conquer
diabetes. No question. We will conquer diabetes, and it will be
due to the courage and strength of a lot of folks who are here
today, particularly young people who have joined us, and I
cannot imagine a better use of this space than for their to be
visiting and sitting here. And I want to join Senator Donnelly
in expressing the view that some of you, I am willing to bet a
lot of money, will be sitting up here one of these days in the
not too distant future.
Let me say, at the risk of offending the Chairman, that I
know he has referred to Ray Allen's distinguished career as an
NBA star, but I just want to remind the Chairman that before he
was a winner for the Miami Heat, he was a Husky, a UConn Husky
and a champion there.
Mr. Allen. I am always a Husky.
[Laughter.]
Senator Blumenthal. And I was going to say----
Mr. Allen. Yes, always.
[Applause.]
Senator Blumenthal. And we can applaud that, yes.
[Laughter.]
It is like being a Marine. You are always a UConn Husky.
Mr. Allen. Always.
Senator Blumenthal. I just want to say, Couch Calhoun would
be very proud of you today, and most especially proud of your
son, Walker. Thank you for being here today, Walker.
Mr. Allen. Thank you, Senator.
[Applause.]
Senator Blumenthal. Let me also thank Ms. Smart. Your work
personally on this has been so valuable, and your sharing with
us your story of your battle with diabetes from the age of 13,
your pregnancy in 1989, your nearly succumbing on the stage.
All of it shows, really, the human consequences and how even
the most illustrious struggle equally with this dreaded
disease.
I have been a longtime advocate of NIH funding. The costs
of this disease in Connecticut and elsewhere are staggering.
Two-hundred-and-sixteen thousand people in Connecticut alone
have diabetes, which costs a total of $2.92 billion. That is
billion with a ``b.'' Two-point-nine billion dollars,
Connecticut alone. So if we are really mindful of the costs
here, we will do more and do it more effectively.
This diabetes has a face and a voice. You are here to tell
us about it. I also want to thank, by the way, Sophie Brown,
Harrison Zuckerberg, and Robert Miles [phonetic], who are from
Connecticut. Thank you for being here. I visited with you
earlier, very proud that you are here today.
[Applause.]
But I want to ask about the meaning of this NIH funding.
Again, if you could explain to us--I know you have alluded to
it before--the importance of sufficient funding to do the work
and do it effectively and what this money actually goes to do.
What is the use of this money that makes it so valuable?
Perhaps you could talk about that, Dr. Rodgers.
Dr. Rodgers. Sure. Let me mention that there are a range of
not only studies in terms of their design, both basic studies
to understand how the beta cell works, why you lose it over
time, translational studies, now understanding, for example,
that there are hormones that are produced as these cells start
to die that could allow for them to what is called
differentiate or de-differentiate into other cells, what can
allow them to proliferate, research that is currently ongoing
to try to develop therapies to reverse the autoimmune attack
once it has actually begun in patients or in people who are at
risk, actually preventing it from ever occurring, studies that
will allow us to more closely treat patients who have the
disease for some time in terms of their complications--heart
disease, stroke, blindness, amputations, for example.
We have made great progress, and, in fact, to a first
approximation, critically, we know that just controlling the
blood glucose makes major difference. But now we want to--I
think everyone agrees that it is better to prevent a disease
than to actually treat long-term complications, and obviously,
we have to continue efforts in all of these areas. But
preventing the disease or reversing it once it is established
very early on, as well as ultimately curing disease, is really
what the Special Diabetes Program funds allow us to do.
Some of the studies, one can do in the short term in terms
of basic studies. But the longer-term studies, the clinical
studies and the clinical trials, really require long-term
sustained funding in order to achieve successfully.
Senator Blumenthal. So we cannot do it just by one year, by
one shot. It has got to be sustained and committed.
Dr. Rodgers. Ultimately, optimally, that is the best way to
approach this problem.
Senator Blumenthal. Thank you. My time has expired,
regretfully, but again, my thanks to this very, very
distinguished panel. My thanks to the Chairman. And, again,
thank you to everyone for being here today. I know you are not
going away. We are not going away. We are going to win this.
Thank you.
[Applause.]
The Chairman. Thank you, Senator Blumenthal. Well said,
Senator Blumenthal.
Ms. Smart, tell us your lessons learned on how to stay
healthy from your experiences over the years.
Ms. Smart. Oh, gosh. I think, and I do not want to make too
much of this, but I do think that there is a great value in not
looking upon yourself as a sick person, not thinking about
being sick as a part of your identity, because we--obviously,
our brain sends messages to our body. So I think that that is
really, really important and I hope--and it seems like most of
the kids here are pretty--incredibly sharp and have that
attitude. I was reading all of your stories last night and it
is impressive, to say the least. I wish that I had had that
kind of gumption when I was 13.
And, again, it is just trying to do what you want to do. I
mean, I never felt like there was anything I could not do. I
was a cheerleader in high school and all that, and, of course,
sometimes I would use my diabetes as an excuse if I did not
want to do something sometimes. You know, if I wanted to take a
break, I would say, oh, I need to go get a Coke. You guys go
ahead without me. You know, things like that, which you have to
not try to do. But, again, it is just----
[Laughter.]
Do not copy what I did. Do as I say, not as I do.
But, no, it is follow your dreams, whatever they are, but
just be smart. Be wise. Do not ever feel self-conscious about
letting people know what is going on with you, and never be
afraid to ask for help if you need it, and just be smart. And I
do not think I need to tell this group that, but maybe they can
tell other kids they meet some of those messages.
And I would just like to add one little thing, too, just
from what I have heard here today and what little I know. I
hope and pray that the insurance business and our government
and everything will not be penny wise and pound foolish,
because it is, for all diseases but certainly for this one,
better to try to find a cure and a prevention than treat
complications 50 years down the road. I hope that we can
convince those in power that that is a huge thing.
And I think the work on beta cells and also this issue of
whatever environmental causes, to me, that has got to be huge,
because once we figure that out, that is going to be, it seems
like, half the battle. Thank you.
The Chairman. Speaking of the cost, Mr. Brewer, Medicare in
and of itself, the Medicare costs are going to double by 2020.
We are estimating $226 billion. How much of that is due to Type
1?
Mr. Brewer. I am not sure I have that specific estimate to
give to you today, but I can tell you that Type 1 is a
disproportionate cost of the expense of diabetes because the
complications tend to occur at a higher rate than in Type 2.
The costs of treating the disease over a lifetime are
increasingly burdensome to the system, as well. So I have seen
estimates that even though Type 1 may only account for five to
ten percent of the population of people who have diabetes,
upwards of 30 to 40 percent of the costs that actually are
absorbed by the system are driven by Type 1, which, I think,
makes the argument even greater that focus on this area is
going to have a very significant implication for Medicare
spending.
The Chairman. Absolutely.
Mr. Allen, tell us about the resources and services that
were available to you and Shannon as parents when Walker was
diagnosed and how you were able to use that resource and
services to manage the physical and emotional aspects of the
disease.
Mr. Allen. Well, when Walker was diagnosed in 2008, just, I
think, we talked about the environment. That was the one thing
we wanted to figure out, because Walker was pre-screened when
he was just one year old, or one day old. And we talked about
so many things that we can do that can help him benefit, or
help us help him benefit from this disease.
I think being in Boston was probably one of the best places
that we possibly could have been because of the care, the
hospitals there. The Joslin Center took us in immediately. They
had such great doctors there and we had a huge connection with
the Celtic ownership. So the minute we flew back from L.A., we
had doctors waiting for us that took care of us immediately. I
do not think--I can honestly say that just because I was a
Boston Celtic at the time, it was not because of that. You
know, they take care of all people that way. The Joslin Center
there in Boston has been incredible. They continue to be
incredible. Dr. Lori Laffel, who was Walker's doctor
permanently, regardless of where we live, she always sees him
and takes care of him and fields the phone calls from my wife.
So we feel so fortunate that we continue to learn and stay
on the cutting edge because the Joslin Center is right there in
Boston, and we live in Connecticut, as well, so we are a very
close distance, and we have people that continually stay in
contact with myself and my wife to make sure that we see and
know everything diabetes-related so we can help his care over a
long period of time.
The Chairman. Dr. Rodgers, how many types of diabetes are
there?
Dr. Rodgers. Largely, there are two types, but there are
perhaps others. Type 1 diabetes, the reason we are having this
Congress, as Mr. Brewer indicated, probably accounts for
somewhere between five to ten percent of the diabetes.
The overwhelming majority is Type 2 diabetes, what we used
to call adult-onset, certainly when I was in medical school.
But, as I said, as a result of this SEARCH program that we were
able to set up, increasingly, we are seeing more of this in
children and adolescents. And Type 2 diabetes in children is a
much more complicated disease to treat than even Type 1
diabetes. And because of the long nature of having
complications, this really could be a major challenge for this
country, and those numbers that you quoted in terms of
expenditures could really increase exponentially if we do not
keep an eye on this, as well.
There are rarer forms of diabetes which account for one or
two percent, maturity-onset diabetes, certain genetic diseases
in which insulin is not secreted well, et cetera. But the vast
majority is Type 1 and Type 2 diabetes.
The Chairman. Colleagues, any further wrap-up, mindful that
our young guests are still sitting horizontal?
[Laughter.]
I mean, still sitting vertical instead of horizontal.
Senator Collins. Or both.
[Laughter.]
Mr. Chairman, I just want to thank you so much for holding
this hearing. As I mentioned in my opening statement, this is
my seventh hearing of the Children's Congress and it is always
so inspiring to see the young people here, to hear their
stories, and to realize the progress that has been made, but
also the work that must be done.
So I particularly want to thank our delegates, my
constituent Quinn, who did such a great job, Walker, who puts a
human face on it, as well, and did a great job sitting next to
Dad for a very long time, and all of the wonderful young people
that we have here today.
When I first met families whose children had Type 1, I knew
that I had to get involved, and we are making a difference and
I just wanted to pledge my continued support and leadership,
and again, thank you, Mr. Chairman.
The Chairman. Senator Warren.
Senator Warren. Thank you, Mr. Chairman, for doing this.
Thank you.
The Chairman. Senator Donnelly.
Senator Donnelly. It was a great honor to be part of this.
Thank you, and we are going to win.
The Chairman. Amen to that.
[Applause.]
And with that statement, the meeting is adjourned.
[Applause.]
[Whereupon, at 3:41 p.m., the committee was adjourned.]
APPENDIX
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