[Senate Hearing 115-469]
[From the U.S. Government Publishing Office]
S. Hrg. 115-469
PROGRESS TOWARD A CURE FOR TYPE I
DIABETES: RESEARCH AND THE
ARTIFICIAL PANCREAS
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HEARING
BEFORE THE
SPECIAL COMMITTEE ON AGING
UNITED STATES SENATE
ONE HUNDRED FIFTEENTH CONGRESS
FIRST SESSION
__________
WASHINGTON, DC
__________
JULY 26, 2017
__________
Serial No. 115-8
Printed for the use of the Special Committee on Aging
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Available via the World Wide Web: http://www.govinfo.gov
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SPECIAL COMMITTEE ON AGING
SUSAN M. COLLINS, Maine, Chairman
ORRIN G. HATCH, Utah ROBERT P. CASEY, JR., Pennsylvania
JEFF FLAKE, Arizona BILL NELSON, Florida
TIM SCOTT, South Carolina SHELDON WHITEHOUSE, Rhode Island
THOM TILLIS, North Carolina KIRSTEN E. GILLIBRAND, New York
BOB CORKER, Tennessee RICHARD BLUMENTHAL, Connecticut
RICHARD BURR, North Carolina JOE DONNELLY, Indiana
MARCO RUBIO, Florida ELIZABETH WARREN, Massachusetts
DEB FISCHER, Nebraska CATHERINE CORTEZ MASTO, Nevada
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Kevin Kelley, Majority Staff Director
Kate Mevis, Minority Staff Director
CONTENTS
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Page
Opening Statement of Senator Susan M. Collins, Chairman.......... 1
Statement of Senator Robert P. Casey, Jr., Ranking Member........ 3
PANEL OF WITNESSES
Paul Sparks, Actor, New York, New York........................... 5
Griffin P. Rodgers, M.D., M.A.C.P., Director of the National
Institute of Diabetes and Digestive and Kidney Diseases,
National Institutes of Health, U.S. Department of Health And
Human Services................................................. 7
Charlie Albair, JDRF 2017 Children's Congress Delegate, Gray,
Maine.......................................................... 10
Lorynn Watt, JDRF 2017 Children's Congress Delegate, Stroudsburg,
Pennsylvania................................................... 11
Angie Platt, Chair Mom of the JDRF 2017 Children's Congress,
accompanied by her son, Jonathan Platt, Encino, California..... 12
APPENDIX
Prepared Witness Statements
Paul Sparks, Actor, New York, New York........................... 28
Griffin P. Rodgers, M.D., M.A.C.P., Director of the National
Institute of Diabetes and Digestive and Kidney Diseases,
National Institutes of Health, U.S. Department of Health And
Human Services................................................. 29
Charlie Albair, JDRF 2017 Children's Congress Delegate, Gray,
Maine.......................................................... 42
Lorynn Watt, JDRF 2017 Children's Congress Delegate, Stroudsburg,
Pennsylvania................................................... 42
Angie Platt, Chair Mom of the JDRF 2017 Children's Congress,
accompanied by her son, Jonathan Platt, Encino, California..... 43
PROGRESS TOWARD A CURE FOR
TYPE I DIABETES: RESEARCH AND
THE ARTIFICIAL PANCREAS
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JULY 26, 2017
U.S. Senate,
Special Committee on Aging,
Washington, DC.
The Committee met, pursuant to notice, at 9:30 a.m., in
room SD-106, Dirksen Senate Office Building, Hon. Susan M.
Collins (Chairman of the Committee) presiding.
Present: Senators Collins, Scott, Fischer, Casey,
Gillibrand, Donnelly, Warren, and Cortez Masto.
Also present: Senator Shaheen.
OPENING STATEMENT OF SENATOR SUSAN M. COLLINS, CHAIRMAN
The Chairman. Good morning. What a wonderful sea of blue I
see out there. Welcome.
I am delighted to convene this biennial hearing in
conjunction with the JDRF 2017 Children's Congress to examine
how Type 1 diabetes affects Americans of all ages. This is the
tenth Children's Congress that the JDRF has held in Washington,
DC, and it is the ninth that I have chaired. It is such a
privilege to work with JDRF, whose commitment to finding a cure
is unwavering.
Let me welcome our distinguished witnesses and the more
than 160 delegates who have traveled here from every state in
the Nation and from around the world to this year's Children's
Congress. We are really glad to have you here.
[Applause.]
The Chairman. Thank you for coming to share your personal
stories and to tell Members of Congress what it is like to live
with diabetes, just how serious it is, and why it is so
important that Congress fund the research necessary to discover
better treatments and, ultimately, a cure.
I want to give a special shout-out to the two delegates
from the great State of Maine: Charlie Albair of Gray, who we
will hear from later, and Brady Chappell of Naples. I am so
proud that you are here representing our state.
We also will be joined shortly by our colleague Senator
Jeanne Shaheen, who joins me as the Co-Chair of the Senate
Diabetes Caucus. She has been a strong advocate for those
living with the disease, including her own granddaughter.
Since founding the Senate Diabetes Caucus 20 years ago, I
have learned a lot about the difficulties and, at times,
heartbreak that this disease causes for so many American
families as we await a cure.
Diabetes is a lifelong condition. It does not discriminate.
It affects people of every age, race, and nationality. It also
takes a major financial toll. The devastating disease costs our
country an estimated $240 billion a year--a cost that is
skyrocketing and is projected to reach more than $490 billion
by the year 2020. Treatment of diabetes accounts for one out of
three Medicare dollars. In fact, medical costs for Americans
with diabetes are more than double those incurred by
individuals without diabetes.
The statistics are pretty overwhelming. But the burden is
particularly heavy for individuals with Type 1 diabetes.
Usually diagnosed in childhood or adolescence, Type 1 diabetes
is a lifelong disease that, to date, one can never outgrow.
Thankfully, there is good news for people with diabetes.
Since I first started the Diabetes Caucus, funding for diabetes
research has more than tripled. It is now about $1 billion a
year. As a result, we have seen encouraging developments in the
management, treatment, and potential cures for Type 1 diabetes.
One program in particular at the National Institutes of
Health has led to phenomenal discoveries, and that is the
Special Diabetes Program. This critical program provides an
extra $150 million a year for Type 1 diabetes research, and
that is in addition to the regular NIH appropriation for
diabetes research.
Last year, the Special Diabetes Program led to the
development of the first-ever, FDA-approved artificial
pancreas, which can control blood glucose levels automatically
and will revolutionize diabetes care. So the Special Diabetes
Program is changing the future of diabetes.
You know, when I first held these hearings, there was not
even a very usable pump. Now we have a really small pump. How
many of you wear a pump? Look at that. Wow.
Now, are any of you involved in the clinical trials for the
artificial pancreas? All right. That is wonderful. Well, I
think that that holds such great promise.
And today you will hear from some of our witnesses about
how truly life-changing these innovative technologies can be.
Other advances in technology, like continuous glucose monitors,
are helping patients better control their blood glucose levels,
and you know that that is key to preventing diabetes
complications.
So what is our task? This year, we must pass legislation to
extend the Special Diabetes Program because, otherwise, it will
expire in September. Are you all on board with that?
[Applause.]
The Chairman. So the great news is due to the efforts that
all of you have been making. Seventy-five senators signed a
letter that I led in September urging that this federal
investment continue. Seventy-five senators out of 100. You
hardly get 75 senators to agree on anything nowadays.
[Applause.]
The Chairman. So with the continuation of this investment,
we can see a future in which all the children who are here
today can look forward to a better and brighter tomorrow.
It is so inspiring for both Senator Casey and me and for
all those who will be joining us to look out and see that wave
of Carolina Blue--each of you personally affected by diabetes.
But let me just end by taking a moment to remember a personal
hero--and we have put up her picture on the screen--who worked
so passionately on behalf of all of you and testified year
after year at the Children's Congresses in the past.
Mary Tyler Moore endured the ups and down of Type 1
diabetes for many years, but she did not complain. She focused
her energy on finding better treatments and a cure to improve
the lives of all of you. So we will honor Mary's legacy by
continuing the work to which she committed so much of her life.
Again, thank you all for joining us from all over the
country, and I am now very pleased to turn to our Ranking
Member, Senator Casey, for his statement. Thank you.
[Applause.]
OPENING STATEMENT OF SENATOR ROBERT P. CASEY, JR., RANKING
MEMBER
Senator Casey. Let us hear it for Senator Collins, our
Chairman.
[Applause.]
Senator Casey. We do not get a chance to do that very
often. I am grateful you participated in that. We do not do it
enough, but we do want to thank Chairman Collins for having the
hearing and also for her years and years of work on these
issues. And we are grateful that you all are here to help us
this year.
We are also pleased that we will have the Co-Chair of the
Diabetes Caucus, Senator Shaheen, to participate in our hearing
today. That is unusual, when you are not a member of a
committee to be part of another committee hearing. So we are
grateful that she is willing to spend that time and to do that
work.
We are also pleased to have so many delegates here of the
JDRF Children's Congress to help us in the Senate to get this
message out and to work with us. It is so important that you
have joined us today because finding a cure for Type 1 diabetes
requires a combined effort from people of all ages and all
backgrounds. Thanks to the delegates for advocating for the
Special Diabetes Program.
Each of you, each delegate here will be more persuasive
than I will be in making the case. That is why it is so
important that you are here today. Please continue to tell
elected officials, not only here in Washington but around the
country in your own communities and your own states, why
research funding matters.
Investing in research has led to the development of the
artificial pancreas. Investing in research means a lot of you
do not have to use needles to track your blood sugar levels.
Investing in research also means that your diabetes has not
stopped you from going to camp or playing outside at recess or
riding a bike or doing so many other things. These are just a
couple of examples of the success of the Special Diabetes
Program and what can be accomplished when Congress fully funds
our promise to fight Type 1 diabetes.
You are also here during an important debate about health
care. So, in addition to telling those of us on the Committee
and in Congress about the importance of this program, it is
important to explain why health insurance matters, why Medicaid
and CHIP are so important to you and your families.
We must ensure that people who have Type 1 diabetes are not
denied care or asked to pay more for their care. That used to
happen once in a while around here when people would make those
arguments, but too often lately those arguments have not been
made. We want to ensure that when you get older you can access
care that is affordable as well.
The Senate health care bill, in my judgment, could lead to
higher costs for individuals and families in this room.
I am pleased that our witnesses are here today with us.
In my case, because I represent the State of Pennsylvania,
I am grateful to welcome Lorynn to the witness table and
grateful for her testimony and also grateful that she is
willing to share not only why research is so important, but why
affordable care is as well. Max and Ryan--if you can put your
hands up--are also Pennsylvania delegates. Thank you to Max and
Ryan for being here as well.
So we look forward to hearing from Lorynn and the other
witnesses. We are grateful that each of you is here to provide
testimony. And I look forward to working with Chairman Collins
and others to ensure that the Special Diabetes Program
continues in 2018 and beyond. And I am convinced that together
we can turn Type 1 into type none.
Thank you.
[Applause.]
The Chairman. Thank you very much, Senator Casey.
I also want to acknowledge the presence of Senator Fischer
from Nebraska. Senator Warren and Senator Gillibrand from
Massachusetts and New York will be back shortly.
And we now get to hear from the stars of the show, and that
is our panel of witnesses. And it is appropriate that I use the
word ``star'' because our first witness is actor Paul Sparks.
Paul is known for his roles in the HBO series ``Boardwalk
Empire'' as well as the Netflix series ``House of Cards.'' And
no--Washington really is not like that.
[Laughter.]
Mr. Sparks. I did not think so.
The Chairman. I want to go on record.
[Laughter.]
The Chairman. Paul was diagnosed with Type 1 diabetes at
the age of 28, and he has since been a powerful advocate for
JDRF and for those with Type 1. And I think that is the
starring role that he is probably most proud of.
Second, we will hear from Dr. Griffin Rodgers, the Director
of the National Institute of Diabetes and Digestive and Kidney
Diseases at the National Institutes of Health. Dr. Rodgers has
testified before us so many times, and I always look forward to
hearing his update. I know how talented and committed that he
is.
And, of course, I take special pleasure in welcoming our
witness from the great State of Maine, Charlie Albair. Charlie
is 10 years old and was diagnosed with Type 1 at age 6.
Nevertheless, be careful because he has a red belt in the
martial arts. He also plays football, baseball, and basketball.
He has been active in his community, raising awareness about
what it is like to live with Type 1 diabetes.
I am now going to turn to our Ranking Member to introduce
our witness from Pennsylvania.
Senator Casey. Thanks very much, Chairman Collins. I was
mentioning before that one of our witnesses is from
Pennsylvania: Lorynn Watt. Lorynn is 17. She is a rising senior
from Stroudsburg, Pennsylvania, up in northeastern Pennsylvania
near where I live. And despite living with Type 1 diabetes
since the time she was 9, Lorynn, like all of the delegates
before us, has not let the disease slow her down or stop her
from pursuing her goals.
Along with her regular academic work as a high school
student, she is very involved in extracurricular activities.
For example, in the upcoming school year, she will be directing
the school play, participating in the Community Service Club,
and serving on the Prom Committee. So that is a pretty busy
schedule coming up.
Lorynn, thank you for making the time in your schedule to
become active in JDRF and advocating here in Washington on
behalf of your peers. We look forward to your testimony. Thank
you.
The Chairman. Finally, we will hear today from Angie Platt.
Angie serves on the board of the Los Angeles Chapter of JDRF as
well as JDRF's International Board of Directors. But her
impressive work does not end there. She is also the mother of
Jonathan, who accompanies her as a witness today, and we
welcome you back, Jonathan.
He was diagnosed with Type 1 at age 6, but that has not
held him down at all. In fact, I am told you are six-five--is
there any truth to that?--at age 14. Outside of being an
advocate, Jonathan is an impressive athlete and was a
participant in the artificial pancreas trial. So we are really
looking forward to your testimony.
And I also want to acknowledge that we have been joined by
our colleague Senator Donnelly from Indiana, who is here today.
You will see senators in and out because of their
schedules, but believe me, they care a lot about this issue.
So we will now start with Mr. Sparks.
STATEMENT OF PAUL SPARKS, ACTOR, NEW YORK, NEW YORK
Mr. Sparks. Thank you, Chairman Collins, and thank you,
Ranking Member Casey, and the members of the Committee for
inviting me to testify here today. I am totally nervous, but it
is a really big honor for me to be here.
I will just start by saying that I know how important the
research supported by the Special Diabetes Program is because
in my own lifetime personally, as a person with Type 1
diabetes, I have seen and I have benefited from the advances
discovered in our labs and clinical trials and brought to
market.
I was diagnosed with Type 1 diabetes when I was 28 years
old. I was living in New York, working as an actor--which is
code for I was living in New York working as a construction
worker trying to be an actor.
Over the course of about 7 or 8 months, I lost close to 40
pounds. I had to go to the bathroom all the time. I had cramps
constantly. I was thirsty, hungry, starving. I could not see
very clearly. I mean, my body was completely falling apart.
Luckily, I went home to Oklahoma to visit my parents, but I
was so thin, so grim, looked so unhealthy that my mother and
father almost had a heart attack when they saw me.
Luckily, my brother was a medical resident at OU, Oklahoma
University, and over the phone I talked about symptoms. And he
said, ``Well, get to a doctor because it sounds like you may
have Type 1 diabetes or diabetes.'' I went the next day, and he
was right. Thanks, David.
I spent the next few months or years trying to learn about
T1D, trying to learn and figure out how to get care for myself,
learn how to care for myself.
As the kids in this room and the parents know, this is a
very anxiety-producing disease. You are the patient, but you
are also sort of the caregiver. You have to do it yourself. You
take care of yourself. You listen to yourself. You are
responsible for keeping yourself healthy in many ways. And you
have to stay on top of it because if you do not, you will get
very sick, as you guys know, or worse.
That is why the research and the advances in care are so
important. Today, nearly 20 years after my diagnosis, I use an
inhaled insulin that quickly and safely brings my blood glucose
back into range. I wear a continuous glucose monitor, which is
awesome, and it allows me to know at all times what my glucose
levels are so I do not go too high and I do not go too low.
These advances have transformed my life.
I used to have to stash sugar when I would do a play on the
stage somewhere in case I needed to stabilize my blood glucose.
I can tell you that it is a pretty strange moment when an 18th
century period drama character pulls a bottle of Tropicana
orange juice out of a sofa cushion and starts drinking it mid-
dialog.
But probably the most demonstrative example that I have
experienced of how important these advances are: About 3 years
ago, I turned my CGM off at night before I went to bed so that
my very pregnant wife at that time, Annie--who is sitting right
there with that little kid who was inside of her--could sleep
peacefully through the night. She was struggling to do that
because she was about 8 months pregnant, because the glucose
monitor beeps when your blood sugar goes low. Well, because it
was off, it did not beep when I went low, and I had a really
severe low. And in the morning, they could not wake me up. And
when I finally did wake up, I was standing in the presence of a
very terrified pregnant woman and a crying 4-year-old boy, who
has green hair right over there, and seven New York City EMTs.
These technologies, like the CGM, when they are turned on,
and other research advances literally save my life every day.
They save the lives of all these delegates. We are on the cusp
of a new generation of therapies and devices and, hopefully, a
cure.
That is why we cannot let up on this research. We need more
advances. We need to cure and prevent Type 1 diabetes so all of
us, like Charlie and Lorynn and Jonathan who are sitting here,
and all these kids, can live a life without thinking about the
disease all the time.
We need to keep the momentum going. We need to renew the
Special Diabetes Program before it expires at the end of
September.
I am going to let the other people talk about the science
and the policies that support it. But let me just say this:
This research has made a difference in my life. It has made a
difference in the lives of all these people in this room, and
millions of people all over the United States.
So thank you, seriously, thank you, Chairman Collins, for
your outstanding leadership, and thank you, Ranking Member
Casey, for your support of T1D research and coverage for
technologies like the continuous glucose monitor. It is great
that people on Medicare can get it just like the rest of us.
Thank you and your colleagues for their bipartisan support
of the Special Diabetes Program. It is doing great work for
everybody.
Thank you.
[Applause.]
The Chairman. Thank you so much for your testimony, Mr.
Sparks, and for your willingness to take the time out of your
schedule to be with us today.
You mentioned the continuous glucose monitor, and Senator
Shaheen, who has joined us and, as I mentioned earlier, is Co-
Chair of the Diabetes Caucus, and I had to push so hard for CMS
to cover that for people on Medicare. We are still having a
problem with the Omnipod, but we are not giving up on that
either.
Dr. Rodgers, thank you for being here.
STATEMENT OF GRIFFIN P. RODGERS, M.D., M.A.C.P., DIRECTOR OF
THE NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY
DISEASES, NATIONAL INSTITUTES OF HEALTH, U.S. DEPARTMENT OF
HEALTH AND HUMAN SERVICES
Dr. Rodgers. Thanks so much. Chairman Collins, Senator
Casey, and members of the Committee, thank you for your
invitation to testify today.
Type 1 diabetes is a lifelong disease that affects
Americans of all ages, including seniors. And on behalf of the
National Institutes of Health, I am pleased to report that our
research investments continue to improve the lives of people
with Type 1 diabetes. Through coordinated efforts with research
partners, such as the JDRF, the ADA, the Helmsley Charitable
Trust, as well as the support of the special statutory funding
program for Type 1 diabetes, we are helping the children here
today and all people with Type 1 diabetes live longer,
healthier lives.
Since I last testified before this Committee two years ago,
significant scientific progress has been made that is putting
us closer to the goal of preventing, treating, and ultimately
curing Type 1 diabetes and its complications. These advances
are really the fruits of the long-term sustained investment of
the Special Diabetes Program in Type 1 diabetes research and a
preview of what we hope will come as a result of those efforts.
First, I would like to just acknowledge the important
contribution of my fellow witnesses.
Paul Sparks, you are a tireless advocate not only for a
future cure but for improving people's lives today.
Charlie Albair and Lorynn Watt, your commitment to
educating others and raising awareness about Type 1 diabetes is
truly outstanding.
And, Jonathan and Angie Platt, I am happy to be able to
share the table again with you. Without participation of you
and others in clinical research, we would not be where we are
today.
I would also like to thank those here today representing
Americans of all ages. This is so important for people with
Type 1 diabetes.
I would say that the outlook for people with Type 1
diabetes is better than ever, and one of the main reasons is
the evolution of the artificial or bionic pancreas technology.
This device measures blood sugar levels and automatically
administers insulin and can help people with Type 1 diabetes
achieve better blood sugar control and avoid the episodes of
hypoglycemia, as Mr. Sparks just talked about, or dangerously
low blood sugar levels.
As you have heard, a significant milestone was achieved
last fall when the FDA approved the first commercial hybrid
artificial pancreas device, and NIDDK supported early research
that contributed to the development of the approved device and
continues to vigorously support research at all stages to
advance this technology.
The FDA-approved device is a first-generation device for
people age 14 and older. It partially automates blood sugar
sensing and insulin administration, but still requires users to
count and enter mealtime carbohydrates.
Numerous improvements are on the horizon to more fully
automate artificial pancreas technology, to make the devices
simpler and more user friendly, to personalize care, and to
bring this technology to all.
For example, the first at-home study of a bihormonal
pancreas system--one which delivers not only insulin but
glucagon, without the need for a mealtime carbohydrate
counting--showed it improved blood sugar control better than
the conventional insulin pump. And the success of this and
other studies lay the groundwork for four new Special Diabetes
Program-supported clinical trials to generate data toward FDA
approval of other devices. These trials will bring us closer to
the goal of having multiple artificial pancreas devices that
are FDA-approved for all ages, allowing people with Type 1
diabetes, the caregivers, and their health care providers to
choose the technology that is best suited to their needs.
And although this technology holds great promise as a near-
term approach to helping people manage Type 1 diabetes, it is
not a cure. Replacing the destroyed beta cells, which would be
a biological cure, is another area of vigorous investigation.
And one way to do this is through islet transplantation, and I
am happy to report progress in this field. The NIDDK and the
NIAID co-led the Clinical Islet Transplantation Consortium,
completed a study of islet transplantation without accompanying
kidney transplantation in people with difficult-to-control Type
1 diabetes despite expert care. This study found two years
after the transplantation that more than 70 percent of the
participants were free of severe hypoglycemia events and had
established near-normal control of their blood sugar levels and
had restored hypoglycemia awareness.
These findings indicate that islet transplantation is an
effective treatment for people whose Type 1 diabetes cannot be
controlled by other means and for whom hypoglycemia events are
life-threatening. These results will be the basis of an
application to the FDA for licensure of a pancreatic islet
product for transplantation.
Now, a current barrier to islet transplantation is the
scarcity of donor islet transplants, and so the NIDDK's Human
Islet Research Network, known as HIRN--everything in government
has an acronym--is pursuing other strategies for replacing the
lost beta cells.
Small molecules can be made into the form of drugs and hold
promise for inducing beta cell replication in a patient's
remaining beta cells or regenerating beta cells from related
cells in the body, and HIRN scientists are pursuing these
possibilities.
HIRN has also made exciting progress in identifying
biomarkers of beta cell loss and developing means to measure
these markers in people with Type 1 diabetes. These advances
can lead to an approach to monitor people at risk for the
disease and to diagnose Type 1 diabetes much earlier, perhaps
when the beta cells are still present, and, therefore, they can
be prevented from being completely destroyed.
I am also pleased to report important strides in combating
diabetic eye disease. Nearly 35 years after the NIDDK's
landmark Diabetes Control and Complication Trial, or DCCT,
began, this study continues to provide critical information and
insights about reducing the devastating risks of this
complication. The study recently reported that people who
intensively controlled their blood sugar early in life are
nearly 50 percent less likely to need eye surgery. The DCCT
scientists also determined that people with Type 1 diabetes
should get eye exams to detect diabetic eye disease based upon
their risk rather than on an automated schedule. So adjusting
the frequency of eye examination and screening to a more
personalized approach will result in fewer eye exams and lower
costs and quicker diagnosis.
The Special Diabetes Program also provides significant
funds to our sister institute, the National Eye Institute, its
Diabetic Retinopathy Clinical Research Network, which also
continues to report significant advances in treating diabetic
eye disease. Building on the network's finding that sanative
drugs are more effective than laser treatment, they compared
three of these drugs with widely differing costs for treating
different people with a certain type of diabetic eye disease.
The results showed that in people with mild vision loss, all
three drugs were equally effective after 2 years. These results
can inform clinical decisions and lead to a more personalized
treatment for diabetic eye disease, while having significant
cost implications. Importantly, the drugs were found to improve
vision, which could be the difference between whether a person
can drive or not, greatly affecting the person's quality of
life.
The network also showed that the sanative is more effective
than laser treatment for treating patients with a more severe
diabetic eye disease, so-called proliferate diabetic
retinopathy. This result gave people with diabetes and their
providers the first new option for treating this disease since
the 1970's, four decades. In April, based upon these results,
the FDA has now approved this drug for all forms of diabetic
eye disease, extending their previous approval.
Chairman Collins, Senator Casey, and members of the
Committee, thank you for this opportunity to testify before you
today. The NIH is grateful for the continued support of
Congress, for our public and private research partners, and for
the unwavering efforts of our clinical study volunteers. With
the remarkable progress already achieved through the support of
the Special Diabetes Program and the progress of future
biomedical research, it is possible to imagine that people will
lives free of the burden of Type 1 diabetes and its
complications.
Thank you for your attention, and I am pleased to answer
any questions you may have. Thanks so much.
The Chairman. Thank you very much, Doctor.
[Applause.]
The Chairman. Charlie, you are up.
STATEMENT OF CHARLIE ALBAIR, JDRF 2017 CHILDREN'S CONGRESS
DELEGATE, GRAY, MAINE
Mr. Albair. Chairman Collins, Ranking Member Casey,
Senators, thank you for inviting me to speak before you today.
My name is Charlie Albair from Gray, Maine. I am 10 years
old, and I will be entering the fifth grade at Gray New
Gloucester Middle School.
I am just like a lot of other kids. I love sports,
especially basketball and baseball. And when I grow up, I hope
to play in the Major Leagues--for the Boston Red Sox.
[Applause/laughter.]
Mr. Albair. The one big difference is that I have Type 1
diabetes, or T1D. I was diagnosed with T1D way back when I was
6 years old. I was in the first grade. I started not feeling
like myself. I kept asking the teacher to go to the bathroom
because I really, really had to. She got angry at me because
she thought I just was trying to skip class.
She felt bad when she found out the real reason.
At first I was kind of confused when I was diagnosed. I did
not know what it was; ``diabetes'' was a big word for a first-
grader.
In the beginning, we treated my diabetes with syringes. And
a half a year later, I got the Omnipod pump and then a CGM to
monitor my sugar levels. I love it.
I do not have to be constantly stabbing myself with a
needle five or ten times a day.
What does this mean for me?
When I first found out I had diabetes, I remember thinking
that this would change my whole life. I thought that I would
not realize my dream of being a sports star.
Now I can realize I can do whatever I want.
Sometimes my Omnipod or CGM beeps in class, and the other
kids say, ``Charlie, stop making noise.'' I just tell them that
that is my natural ``robot'' noise.
[Laughter.]
Mr. Albair. The pump and CGM are so much a part of me. But
I do wish that they did not have to be.
I want my disease to go away--for me and all the other kids
who suffer from it. I want us all to be able to live without
thinking about it.
That is why I am here.
We need money for research.
We need money so scientists can invent new pumps and
monitors better than what we have now and so they can come up
with a cure for T1D.
You have supported kids like me for so many years, and all
I ask is that you continue to do so. And if you do, I will
invite you to a game when I am on the Red Sox.
[Laughter.]
Thank you.
The Chairman. Thank you, Charlie.
[Applause.]
The Chairman. I cannot wait to see you playing at Fenway
Park. I will be there.
Lorynn, welcome.
STATEMENT OF LORYNN WATT, JDRF 2017 CHILDREN'S CONGRESS
DELEGATE, STROUDSBURG, PENNSYLVANIA
Ms. Watt. Hi. Chairman Collins, Ranking Member Casey,
Senators, thank you so much for inviting me to talk with you
today.
My name is Lorynn Watt, as we have established. I am 17
years old, and this fall I will be a senior at Evergreen
Community Charter School in Cresco, Pennsylvania.
When I was 9, I was diagnosed with Type 1 diabetes. I
remember sitting on my parents' bed, just about a week before
Halloween, as my Mom and step-dad told me that I had T1D. That
day they loaded me into the car and took me to the hospital,
where I would learn to care for myself.
At that point, I only knew about diabetes from an episode
of ``Hannah Montana'' I saw just before I was diagnosed. That
was it. What I did know was that I was scared and felt awful,
and all of a sudden I was living a life where I had to inject
myself with insulin multiple times a day, even though I was
horrified of needles.
Then at 14, I got a continuous glucose monitor, or CGM, and
an insulin pump. It made my life so much easier. Now, all I
have to do is look at my phone--which kids my age do anyways--
and I can see my blood sugar. It has been life-changing, and I
hope this, the artificial pancreas, and other advances are
small steps toward a cure.
I have heard every year since I was diagnosed that in 5
years there will be a cure. I have had this disease for almost
8 years, and you know, I am no mathematician, but I think we
are behind schedule.
I believe that with your help we can have a cure. After
all, we have come so far.
I know this because my biological father also had T1D. He
was not in my life much, and he did not have great care--no
pump or CGM or even the ability to check his blood sugar every
day.
So he lost his foot and then his eyesight and then the use
of his kidneys. He had to get a stent in his heart. And he died
less than a year after my own T1D diagnosis, at just 38 years
old.
I am here today asking you for more support and more
funding for more research because no one should have to suffer
and lose their life because of T1D.
I am here, inspired by his memory, and determined that none
of the kids here today or sitting in a hospital room--I am
sorry----
[Applause.]
Ms. Watt [continuing]. Or sitting in a hospital room right
now--scared--as they get their diagnosis, will have the same
fate as my father.
With your help, I know we can do it. I know we can find a
cure.
Thank you.
The Chairman. Thank you, Lorynn.
[Applause.]
The Chairman. Thank you so much. I know that was really
hard, but your story really inspires us to work even harder.
Mrs. Platt, I know you are speaking on behalf of yourself
and your son. We would love to hear your testimony.
STATEMENT OF ANGIE PLATT, CHAIR MOM OF THE JDRF 2017 CHILDREN'S
CONGRESS, ACCOMPANIED BY HER SON, JONATHAN PLATT, ENCINO,
CALIFORNIA
Ms. Platt. Chairman Collins, Ranking Member Casey,
Senators, thank you for inviting me to speak with you today.
I am Angie Platt from Encino, California. My husband, Jon,
and I have three children, all boys: twin 4-year-olds, and this
is our oldest son, Jonathan, who is 14 years old.
It is hard for me to believe that this, like you said, six-
foot-five young man sitting next to me is the same boy who was
here for JDRF's 2011 Children's Congress. He was only 7 years
old at the time, just two years into his diagnosis.
If you watch Jonathan play in his competitive basketball
tournaments, it would be very hard to believe that he is a
child living with Type 1 diabetes.
I am here today to tell you and your colleagues that
Jonathan is living proof that your leadership and actions have
made a real difference in the lives of Jonathan and the lives
of all the people with Type 1 diabetes.
In 2011, when Jonathan was here, the Type 1 diabetes
community was asking for your help, and, Senators, you gave it.
The Special Diabetes Program has provided hundreds of millions
of dollars of crucial funding for a range of therapies and
investigations, including the artificial pancreas.
In April 2016, Jonathan was enrolled in the pediatric trial
for the Medtronic Hybrid Closed Loop 670G--otherwise known as
the ``artificial pancreas.'' We felt as if we had won the
diabetes lottery when Jonathan got a spot on that trial.
This device has given Jonathan better blood sugar control
than he has ever had in his diabetic life, and it gives our
family some desperately needed peace of mind. In the past, we
would wake up and check Jonathan's blood sugar at minimum three
times a night, similar to the parents behind me and the parents
at home caring for children with Type 1 diabetes. Jonathan used
to have to stop practicing with his team or even sit out in
games because his blood sugar levels were too erratic. Now he
gets to play right through crunch time, and we are so thankful
for that.
As you know, last fall the FDA approved this artificial
pancreas system. And, Senators, let me be clear: This would not
have happened without your help and without the support of the
Special Diabetes Program.
Diabetes is relentless. We all work so hard. We are so
responsible. We are playing by all the rules. Jonathan is on
the latest and the most advanced technology, and he doing
everything right.
But the ugly reality of diabetes is that, as hard as we
work, our kids are still vulnerable. This past June at
Jonathan's eye exam, it was discovered that Jonathan has three
dot hemorrhages. Children with Type 1 diabetes at the age of 14
should not be diagnosed with diabetes complications. And, quite
frankly, there are kids who are a lot worse off than Jonathan,
and those of us who are sitting here are among the lucky ones.
I know that we have made progress, and my son is wearing
the very first artificial pancreas system approved in the
world. But this disease does not stop, and so we cannot stop.
We need the next generation of devices that can fully
automate insulin delivery.
We need to ensure that progress continues in the area of
diabetes complications.
We also need to prevent others from ever developing Type 1
diabetes, including my twin sons, Jonathan's brothers, who are
at a higher risk of developing Type 1. They are enrolled in
TrialNet, a Special Diabetes Program-funded prevention program.
The Special Diabetes Program has done so much, but as you
said, it will expire on September 30th. The SDP gives me so
much hope, but it needs a hero. We need you Senators and your
Senate colleagues to renew it for another three years so
researchers can continue their great work.
Senators, I want to thank you for all that you have done to
make my family's life and the lives of all of us here today,
and the lives of all of those back at home better. Senator
Collins, I particularly want to thank you for your steadfast
commitment and outstanding leadership to advance Type 1
diabetes research and to help people gain access to new
technologies. You have been a champion of us for a long time.
Look at Jonathan. He will not lose to Type 1 diabetes.
We will fight alongside him. We will fight alongside all
these kids before you, and we will fight alongside all the kids
at home.
I ask that you please continue to fight alongside with us.
Thank you.
[Applause.]
The Chairman. Thank you all for such inspiring testimony. I
know that it is hard to share your personal stories, and it
makes such a difference because then it puts a human face on
the disease, and that is what we need as we advocate for more
money.
I want to start with Dr. Rodgers because I know that you
will remember that 10 years ago, in September 2006, I chaired a
hearing, the Children's Congress, and the topic was the
artificial pancreas. And it was like something way in the
future, but what a difference it would make. And it is so
exciting when I learned of the FDA's decision last fall to see
the progress that we have made. So I know that it seems hard
for those of you, particularly those of you who are newly
diagnosed, but the amount of progress in the 20 years that I
have co-chaired the Diabetes Caucus really is encouraging. And
we are going to keep working on that.
I also love, Dr. Rodgers, that you called it, instead of
the artificial pancreas, the ``bionic pancreas.'' I think I
will adopt that term from now on. But I want to point out that
the Special Diabetes Program has been critical to achieving
many of the advances you described and was the program that
spurred the development of the artificial pancreas technology.
What additional advancements is the Special Diabetes
Program supporting that are in the pipeline now? Is the islet
transplantation part of that?
Dr. Rodgers. Absolutely, Senator. In fact, I remember
vividly. That is about 10 years ago, and that was actually the
first year I was the Director of the Institute, and you really
challenged us to move forward with this artificial pancreas.
And it seemed at that time to sort of be a dream, but here we
are.
What I can tell you is that we are actually even moving
much further along in that regard with the bionic artificial
pancreas. With the current funds that are remaining in the
Special Diabetes Program, we are actually just launching four
new clinical trials toward FDA approval of the artificial
pancreas device, and one of these trials, I have already begun
recruiting patients. These trials will be testing, for example,
the combination of insulin and glucagon, as I mentioned, a
bihormonal therapy led by researchers at Boston University.
There is a trial that is underway at the University of
Virginia which is developing an intraoperative system. We
understand there are going to be advances in the infusion
technology as well as the sensing technology. This system will
be a plug and play where it does not matter which of these
devices really moves forward. This system will test that
interoperability, and that is at the University of Virginia.
There is a third, which is the trial that is currently
recruiting patients. That is led by investigators at the
University of Cambridge in England, and they will test an
artificial pancreas specifically in youth. As I said, the
current device is for kids 14 and older. This is looking at a
much younger pediatric group to determine its efficacy.
Then the fourth trial that we are funding is led by an
international diabetes center in Minneapolis and the Schneider
Children's Medical Center in Israel, and they will be testing
the next generation of this currently approved device to
determine its efficacy in a broader group.
The HIRN that I mentioned has also been spurred on by the
Special Diabetes Program, and being able to develop a way to
take the patient's cells from their skin and to induce them to
actually become glucose-responsive beta-type cells and to
expand them for potential transplantation is really going to be
a great advance. And we are well along the way with that with
the additional and continuous funding of this Special Diabetes
Program.
Having these cells available--and I do not want to go on
very long, but it will also give us another advantage, because
one can take these cells and culture them in a dish and put
them on a chip, and this chip now, with the patient's own
cells, can actually determine the effectiveness of certain
drugs, for example. In this way, you can envision making
therapies even more personalized.
So those are just a few of the many things that have been
spurred on and we hope will continue with the Special Diabetes
Program support. Thank you.
The Chairman. Thank you.
Charlie, how does having Type 1 affect you when you are
playing sports, when you are on the court or in the field?
Mr. Albair. Well, sometimes if my Dexcom says I am low, I
have to just run out onto the sidelines, take sugar, and then
wait however long I need to. And then when I go up, I can go
back out and play. But the normal blood sugar that my parents
want it at is 160 to 170 because it is a lot of running around,
so it could make me go low, and there is a lot of adrenalin, so
I could go high. And it is just hard because we do not know
what way I am going to go, down or up.
The Chairman. That must be hard to constantly monitor it,
and yet pay attention to the game, too. But it sounds like you
do a great job doing both.
My time has expired, so Senator Casey. Thank you, Charlie.
Senator Casey. Thanks, Chairman Collins.
I want to thank all the witnesses for being here. Your
testimony is very compelling, and that is the kind of testimony
that will help us win some of these legislative battles. So we
are grateful you are willing to do that, and I extend that to
all of the delegates. In your own ways, you will all be
witnesses to bring testimony to individual Members of Congress.
Lorynn, I am especially grateful that you are here
representing our state. I do have one brief suggestion. Just a
thought. You do not have to answer. I know you are busy with
the Prom Committee and all those other activities, but you are
going to finish high school with great grades. You are going to
go to college. My suggestion is become a lawyer, then become a
sports agent, and get Charlie and Jonathan to play for the
`76ers. OK?
[Laughter/applause.]
Senator Casey. We could use the help.
[Laughter.]
Senator Casey. But, Lorynn, thank you for sharing your
story about your dad and about your own life. It is those
stories that move the needle on these public debates. All of us
will continue to make arguments. We will make speeches and all
of that. If you are Senator Collins, they usually work out
better than the rest of us. We know we are going to need you.
I guess the one question I had is: You have got, obviously,
the benefit, and so many do, of new technology, and that is
something to be positive about. And I guess part of the benefit
of that is it has helped you to manage your own condition. But
tell me a little bit more about how it has made your life
better both at school and at home. If you can just kind of walk
us through part of your day or give us a sense of what it is
like every day.
Ms. Watt. Well, a major part of it is sleep. I like to
sleep, and I did not really get to do that a lot before,
because my Mom would come in three times a night, four times a
night, depending on how that day is going, and check my blood
sugar for me, to the point where I would just sleep through it
because it just became like clockwork.
But, you know, it is really cool to be in class, and all of
my teachers know that I have Type 1, and so I am allowed to
have my phone on the desk. And so I can look at my phone, and,
you know, if I see that I am high, I can take my insulin with
my pump without having to run to the nurse and do a shot. I do
not really need to do a finger stick right away. I have time.
And if I see I am low or if I am going down, I have the ability
to treat it before it becomes a real problem for me. And so it
has been really helpful. I am really grateful.
Senator Casey. Well, that helps us as well to be able to
give folks a sense of the challenge that you face every day.
Another issue that I wanted to raise is part of the debate
we are having right now about programs like Medicaid. We know
that living with T1D--and it is nice to have an acronym for
that, too. I guess as we said, we have lots of acronyms. But
living with T1D presents lots of challenges. One of the
challenges, of course, is making sure that the services are
there. Whether it is insulin or doctors' visits, to technology,
technology like the continuous glucose monitor, those are
significant costs. And I know that a lot of families here
benefit not only from coverage, but I guess, Lorynn, in your
case, in terms of what your Mom gets from work, that impacts
you as well. Can you talk about that in terms of the coverage?
Ms. Watt. I do not know a lot about that, just because I am
a minor, so I am not allowed to deal with a lot of that.
Senator Casey. Right.
Ms. Watt. I do know that the ability to afford the
technology I have is really great because a lot of people
cannot afford $14,000 out-of-pocket for an insulin pump and a
CGM. So the fact that we have the insurance, you know, even
like a vial of insulin is about $300, and I am using about
three a month. We do not have $900 a month to put toward
insulin. So having the ability to know that that is going to be
there and we are not going to have to worry is really relaxing
to know.
Senator Casey. Well, we will have to bring you back here
when we are debating the budget with regard to NIH research
dollars. So thanks for your help.
Ms. Watt. Thank you.
The Chairman. Thank you.
Senator Donnelly?
Senator Donnelly. Thank you, Madam Chair. And I want to
thank all of you for coming to the Congress today. To my
Hoosier friends, Becca and Katie and everyone else, we are so
excited that you are here. It is awesome to see all of you.
Charlie, my dream in life was to be a Major League baseball
player, but I could not hit a curve ball, so my recommendation
to you is spend a lot of time in the cage. And, actually living
not too far from Chicago, it is difficult to root for the Red
Sox. However, when you get to Fenway, I will come and watch you
play, and it will be awesome to see that.
And to Jonathan, I am a Notre Dame graduate and would be
happy to put you in touch with the basketball coach.
[Laughter.]
Senator Donnelly. It could be a win-win situation, my
friend.
Dr. Rodgers, you talked a bit about the successful clinical
trials on artificial pancreas technology, and we have all been
very interested in this. Could you tell us about the future and
how you think this is going to go from clinical to products in
the market and where we are headed?
Dr. Rodgers. Sure. Thank you, Senator, and, again, thank
you for all your support. I think, you know, again, this next
generation, our goal is really to provide patients and their
families with options. And while this has really been quite
successful, getting this first artificial pancreas approved by
the FDA, as you have heard, it still has limitations. And so
really the purpose of these four trials that I just outlined a
moment ago is to provide people with even more options,
something that is more----
Senator Donnelly. You are still in effect flooding the
zone, so to speak, to put as many lines out there as you can.
Dr. Rodgers. Exactly right. And as I said in my opening
remarks, you know, even having these available, it is still not
a cure. And so we really have to make sure that we look at ways
of replacing those cells that have been damaged. Ideally,
actually determining the disease so early in people who are
genetically at risk that we can halt the progression of the
autoimmune disease and then get their own cells to repopulate
and regenerate, those islet cells produce cells.
Senator Donnelly. Now, when you do that, the islet
transplantation and the work that is being done on that, would
that be like a patch that would be used? Or how would it be
delivered? And, you know, the magic thing that Lorynn was
talking about, we are always told it is five years every year.
Dr. Rodgers. Right.
Senator Donnelly. Where are we in terms of that? Because I
know up in Harvard with Dr. Melton they are doing work, and
elsewhere they are doing work.
Dr. Rodgers. That is right. They are supported by the
Special Diabetes Program, and expanding these cells was really
a remarkable achievement. I hate to use the word
``breakthrough,'' but that was truly a breakthrough. Being able
to take the patient's own cells and expand them in such a
fashion is totally outstanding.
Of course, even if you expand those cells and transplant
them back in through a variety of ways, you still have to
overcome the autoimmune disease, and so you could either
encapsulate these or in some way modulate the autoimmune
disease to make sure that those cells remain healthy and
survive.
The one aspect I do want to sort of go back to this
artificial pancreas is even if we expand this technology and
really achieve a lot of benefits, it really will not be to
anyone's benefit if people do not use them. And so we have to
make sure that these are readily available for a large number
of people and for whatever barriers that there may be to using
them that we can overcome that. And one of the major things
that SDP is doing, we are able to bring people into this field
of research who had never envisioned going into this--chemical
engineers, bioinformaticians, computer scientists, people who
are involved in psychology to try to determine how one might
effectively influence behaviors in terms of using these
technologies. So it really does take a large number of people
that we are just envisioning.
And then, finally, I just would quickly say that, as you
heard, even as these technologies emerge, there are still
people with the disease at risk for developing complications,
so we have to put efforts into making sure that the eye
disease, the kidney disease, the foot ulcers, the heart disease
is appropriately managed. And we have activities underway to--
--
Senator Donnelly. Well, I can tell you that on the Special
Diabetes Program you have got--what is the term? ``A steely
eyed missile commander'' in Susan Collins and Jeanne Shaheen
and Bob Casey, and everybody is all in to make sure that is
protected.
And to all of you young people, one of the folks who
visited my office today is a fellow named Charlie Kimball.
Charlie is a young man who is an IndyCar driver. Charlie has
won IndyCar races. Charlie drives around the track at 230 miles
an hour. And Charlie has Type 1 diabetes and has driven entire
races, was ahead in the Indy 500 with about 10 laps to go a few
years ago. And so any dream you have, anything you are hoping
for, anything you want to accomplish, I think Dr. Rodgers and
all of you would say--Dr. Rodgers, there is no reason they
cannot achieve anything they want, is there?
Dr. Rodgers. Absolutely.
Senator Donnelly. Thank you.
The Chairman. Thank you very much, Senator.
Senator Scott?
Senator Scott. Thank you, Chairwoman. And thank you for
holding such an important hearing this morning. I will always
remember growing up in South Carolina with my best friend at
the time, at 13 years old was diagnosed as a child with
juvenile diabetes. And the good news is that at 51 years old--I
am dating myself at this point. At 51 years old now, Billy is
still very healthy. So the longevity and the good health is
prayerfully in the future of so many kids who have juvenile
diabetes.
I do know that we have at least three delegates from South
Carolina. If you are here, please stand up. Cameron. Is Cameron
here? Or Julian?
The Chairman. Right here.
Senator Scott. A tall youngster. And Riley? All right.
[Laughter.]
Senator Scott. Excellent. Are there any other South
Carolina delegates here? Would you stand up for me, please?
Excellent. Well, thank you very much for being here. In my
previous life before Congress, I served on our local Juvenile
Diabetes Research Foundation for a short period of time, and I
am so thankful to see this hearing and hear about the progress
being made.
I thank the panelists for being here to discuss this very
important issue as well.
The Chairman. Thank you very much, Senator.
I am now very pleased to call upon the Co-Chair of the
Senate Diabetes Caucus, who has been a wonderful partner and
leader in the fight against Type 1 diabetes and in helping us
get research dollars, and that is my neighbor from New
Hampshire, Senator Jeanne Shaheen.
Senator Shaheen. Well, thank you very much, Madam Chair,
and I am honored to serve with you and appreciate the
leadership that you have provided for so many years in the
Senate. We would not be here where we are today without your
leadership, so thank you very much for that.
I also want to recognize Anna Cook, who is here from New
Hampshire, and her mother, Amy. Is Anna here in front
someplace? Thank you, Anna, for being here.
And I really appreciate all of you being here because you
do not know yet how much difference you have made and the
Children's Congress has made in advancing research in diabetes.
You are the best advocates there could be as you fan out and go
meet with senators and members of the House to talk about why
this is so important, and the stories that you have to tell are
what has moved this debate.
I have a granddaughter, Elle, who has Type 1 diabetes. She
was diagnosed a little after her eighth birthday. She is headed
off to college this fall with her diabetes service dog, Coach,
who went to high school with her. He graduated with his own cap
and gown.
[Laughter.]
Senator Shaheen. And he is headed off with her, and he has
made a huge difference for her. But even Coach has not been
able to address the kind of issue that you all know about and
that Mr. Sparks spoke to, where she has on occasion--in fact,
just last week, she also turned off her CGM, and her mother was
not able to wake her. And, fortunately, she did not have to go
to the hospital. She was finally able to get her awake after a
lot of carbohydrates.
So I appreciate and have seen how important the Special
Diabetes Fund and the research it promotes is in her life.
And I hope that as you all go to offices that you will talk
about this not just in the context of how important it is for
you individually and what your stories are like, but you will
also let people know--oh, we have a diabetes service dog in the
audience. All right. What is the dog's name? Gigi? All right.
Gigi is great, I bet.
But that you will let people know that this is also
important to the policy of this country and to addressing a
chronic illness that is not going to get any better unless we
actually do the research.
Now, Dr. Rodgers, you talked about the importance of better
understanding and diagnosing people who might have a proclivity
to diabetes so we can address it before it becomes full-blown.
Can you talk about the connection between gestational diabetes
and whether that will then lead to diabetes later in life?
Dr. Rodgers. Thank you, Senator. Gestational diabetes,
which is, as the Senator indicated, the type of diabetes that
occurs during pregnancy, affects some five to seven percent of
all pregnancies. In certain ethnic groups, it may be as high as
17 percent. As a result of NIH funding for this research, we
know that the infant born during that pregnancy has a higher
risk--not only does the mother have a higher risk of developing
diabetes, but the infant born of that pregnancy compared to
their siblings in which the mother did not have gestational
diabetes has an increased risk of developing obesity and
diabetes. In this particular case, it is Type 2 diabetes.
Senator Shaheen. Right. Thank you. So it would behoove us
to screen for gestational diabetes in every pregnant mother,
right?
Dr. Rodgers. Absolutely.
Senator Shaheen. Mr. Sparks, you talked about inhaled
insulin. I have never heard of inhaled insulin. Can you talk
about how long you have had that and how that is different?
Mr. Sparks. Yes, I can. I have been on inhaled insulin. It
is called ``Afrezza.'' It has been on the market, I think, for
a little over 2 years. Aaron Kowalski, who is the chief mission
officer for JDRF, turned me on to it. He said this is something
that was going on. I know it is not a lot out there, but it is
really simple. There are no needles. You just have these little
pods, and there is a little inhaler device that goes in. I find
it to be--look, I do not know the science. I am an actor. I do
not even play a doctor on TV.
[Laughter.]
Mr. Sparks. But I know that my experience of it is that it
is very quick acting. It leaves the body very quickly, maybe an
hour and a half after you use it. It does not store and then
sneak up on you like later for some unknown reason. And it has
been a big change for me. I love it. It does not seem to have
the same low quality that I have had before when I was using
vial insulin. So, yes, I love it. I sing its praises whenever I
talk to anybody.
Senator Shaheen. Thank you. My time is up. Thank you, Madam
Chair.
The Chairman. Thank you.
Senator Warren?
Senator Warren. Thank you, Madam Chair. Thank you once
again for having this hearing, and thank you, Ranking Member
Casey.
I am going to meet with some Massachusetts folks this
afternoon, but I do not know if there are any here today
already. Well, there they are. OK, good. Good. I thought you
would be louder than that. All right. It is good to see you. I
hope I did not wake you up over there. OK, good. Good.
But I am really glad that all of you are here, and I am
glad that we are getting a chance to talk about Type 1 diabetes
and to meet some of the people who are speaking out for the
one-and-a-quarter million Americans who have Type 1 diabetes.
I know that researchers all across the country are working
hard to try to understand Type 1 diabetes and to develop better
treatments on how to deal with it. There is even some talk of
cure. And we have made a lot of progress with medicines and
innovative devices that I think we have been talking about this
morning, like insulin pumps and an artificial pancreas and
inhalable insulin. That all happens because of our investments
in biomedical research. But we know there is a lot more to do.
So, Dr. Rodgers, I know you have already talked some about
the artificial pancreas and how it has been a game changer for
many of the people in this room. And you mentioned that NIH
funding has been critical for several trials that have been
able to get you in a position to push the envelope on what the
artificial pancreas can do, including one led by researchers at
Boston University and Massachusetts General Hospital. And I
just wondered if you could just say one more word about the
work that is being done, there in Massachusetts or anywhere
else in the country, about how we are pushing out on the edges
of research for diabetes.
Dr. Rodgers. Absolutely, Senator. And that work that they
have done, particularly in Boston--and then I will talk about
the other places--they really have done some critical pivotal
trials sort of in the real world. So, for example, they led
trials looking at the Beacon Hill area and having people in the
community living as normal an existence as possible as they
were being monitored. They have had summer camps for young kids
using these, and a lot of these were precursors to their
current study that is on the way of this bihormonal pump, which
includes both insulin as well as glucagon investigators.
I had mentioned a little bit earlier but it does bear
repeating that other investigators are looking at ways with the
existing FDA-approved device to expand this in the pediatric
population because currently it is only approved for 14 years
of age. And we know that the better control of your blood sugar
that you can get early on will lead to better improvements or
risk reduction in complications later on in life.
Senator Warren. I appreciate that. I think there are a
bunch of people here under 14 who are rooting for you to get
this done as quickly as possible. Are you guys ready for that,
ready to do a little experimentation?
[Applause.]
Senator Warren. Yes, you have got to help here.
Now, I want to put in a small plug because this research
has been made possible by the National Institutes of Health,
and it is vitally important that it continue, important to
everyone in this room, important to all of your families, and
important to people all across the country.
We are grateful for your work, Dr. Rodgers, and grateful
for the work of other researchers in this area. But we are also
grateful to our fellow Americans whose tax dollars help make
that research possible. And it is the reason that many of us
oppose cuts to the National Institutes of Health. Even if the
Special Diabetes Program is reauthorized, right now there are
proposed cuts of over 20 percent in a single year, and that
would knock out the legs from underneath the kind of innovation
we are trying to build and the kind of innovation for new
treatments and even cures for diabetes.
So I just want to make the point that research takes money,
and we need to support it in a sustained way. I know it is
something that Senator Collins has been strong on, Senator
Casey has been strong on, Senator Shaheen, Senator Donnelly.
But we are going to need all of you to be out there and help
support the National Institutes of Health because they support
the research scientists who are going to make your lives a
whole lot better. Thank you.
Thank you, Madam Chair.
[Applause.]
The Chairman. Thank you, Senator.
Before I adjourn the hearing and let all those who are
sitting on the floor have an opportunity to get up and move
about, I just wanted to ask Jonathan if he had anything that he
would like us to know or to tell us about. I do not mean to put
you on the spot, but since you did not get a chance to talk--I
know that you are known for having a really positive attitude,
and maybe you could share that with the other children who are
here today. How do you keep such a great attitude? Sorry. I
think I did put you on the spot.
[Laughter.]
Ms. Platt. How has your device helped with basketball?
Mr. Platt. Well, my artificial pancreas has really helped
with my basketball games because now when I get pulled out of
basketball games, I do not always have to check my blood sugar
to make sure it is ready for me to get back in, because before
this device, when I was on the CGM and the pump, when I came
out of games, I would always have to check and make sure it was
right. If I was high, I would stay on the bench for an extended
amount of time to make sure I get my mind ready to get back in
the game. And if I was low, I would not be able to get back in
the game at all until my blood sugar raised up, which since I
was on the court a long time running up and down, it normally
took a really long time for my blood sugar to start getting up.
And I would have to wait until it got to like 150 in order to
get back out, because if I got back on at like 120, I would
just go low again in a matter of minutes.
The Chairman. Thank you. That is really helpful.
And as I am sure everybody here knows, but maybe not
everyone watching knows, the artificial pancreas is in the
midst of a clinical trial for younger children, so that it
would be more widely available, and I am really looking forward
to the results of that clinical trial as well. So thank you for
sharing.
Senator Shaheen. Madam Chair?
The Chairman. Yes?
Senator Shaheen. Could we ask Jonathan to maybe hold up his
artificial pancreas? I think a lot of people do not know what
we are talking about when we say that.
The Chairman. Good idea. Wow, look at how tiny it is. That
is amazing.
Senator Shaheen. Can everybody see that? It is really
small.
The Chairman. Hold it up high.
[Applause.]
The Chairman. There you go. Thank you. And, Jonathan, you
now just proved that you really are six-five.
[Laughter.]
The Chairman. So thank you for sharing your personal story,
and thank you, Senator Shaheen. That was an excellent idea.
I would like to thank each of our witnesses for being here
today. You each added so much to our understanding.
I want to end this hearing for my part by telling you how I
got involved in this issue. I do not have any family members or
close friends or relatives with Type 1 diabetes. I do have some
friends who have it, but what got me involved was 20 years ago,
when I was a brand-new Senator, and JDRF arranged for me to
meet with a family who had a 10-year-old son with diabetes. I
will never forget his looking up at me and saying that he
wished that he could just take 1 day off from having diabetes,
his birthday or Christmas. And that so touched me that when I
came back to Washington, I said to my assistant at the time,
``Is there a Diabetes Caucus in the Senate?'' And she said,
``No. Only in the House.'' And I said, ``Well, guess what?
There is going to be one now.''
[Applause.]
The Chairman. And the reason that I share that story with
you is I want you to know what a difference you make when you
come to Washington and meet with your Senators and your Members
of the House. When you tell them what it is like to live with
diabetes, it really helps us mobilize support for the research
dollars that have made the artificial pancreas, continuous
glucose monitors, and better pumps possible. So you and your
advocacy make such a difference, and I know that from my
personal experience. So please keep up the good work. It really
makes a difference.
Thank you all for traveling to Washington. Your commitment
to this cause is truly extraordinary and inspiring.
I would now like to turn to Senator Casey for any final
thoughts he might have.
Senator Casey. Madam Chair, thanks very much. We want to
thank all of the witnesses, to Lorynn, Charlie, and Jonathan,
Angie, Dr. Rodgers, Mr. Sparks, so many others who are here.
Another suggestion I have, you know, we are worried about
the budget cut to the National Institutes of Health, a big cut
of billions of dollars. I thought, because Mr. Sparks is
familiar with the White House, he could somehow get in there
and get that and just cross it out of the budget document.
[Laughter.]
Senator Casey. Just a suggestion.
But I am serious about the issue. In addition to the work
we have to do to reauthorize the Special Diabetes Program, we
have got to fight hard against the cuts to NIH. Fortunately,
the opposition to those cuts is bipartisan. I think we can do
that together. But all of our delegates will play a role in all
of these issues.
The stories that you tell about your own lives and your own
challenges are going to be critically important in this debate.
We are grateful you are with us today, and this is among the
more enjoyable hearings we will ever have, despite all the
challenges that you have and the stories that you told. Thank
you and God bless you.
The Chairman. Thank you.
[Applause.]
The Chairman. Committee members will have until Friday,
August 11th, to submit questions for the record. Again, my
thanks to JDRF, to all the delegates who inspire us, to our
terrific panel of witnesses. Thank you so much for being here.
This hearing is now adjourned.
[Applause.]
[Whereupon, at 10:55 a.m., the Committee was adjourned.]
=======================================================================
APPENDIX
=======================================================================
Prepared Witness Statements for the Record
=======================================================================
Prepared Statement of Paul Sparks, Actor, New York, New York
Thank you, Chairman Collins, thank you, Ranking Member Casey, and
the members of the Committee for inviting me to testify today. It's an
honor for me to be here.
I'll start by saying, I know how important the research supported
by the Special Diabetes Program is because in my own lifetime as a
person with type 1 diabetes, I have seen--and benefited--from the
advances discovered in our labs, tested in our clinical trials, and
brought to market.
I was diagnosed with T1D when I was 27, living in New York, working
as an actor--which also meant that I was working as a construction
worker in order to pay my bills.
Over the course of about seven or eight months, I lost about 45
pounds. I noticed that I was going to the bathroom a lot. I was having
muscle cramps all the time. I started not being able to see clearly. I
was very thirsty--and constantly starving.
Basically, my body was falling apart. Thankfully, I went home to
Oklahoma to see my parents for Thanksgiving. I looked so grim, so thin
and so unhealthy, that my mother almost had a heart attack when she saw
me.
Luckily, my brother was a medical resident at the time. Via a phone
call, he recommended that I see a doctor soon, because it sounded like
I had diabetes.
Well, I did see a doctor the next day, and my brother was right.
I spent the next few months trying to learn about T1D and figure
out how to get the care I needed.
As the kids and parents here know, this is an anxiety-producing
disease. You are the patient but you are also, in many ways, the
caregiver. You are responsible for keeping yourself healthy. And you
have to stay on top of it; because if you don't, you will get very
sick--or worse.
That's why the research and advances in care are so important.
Today, nearly 20 years after my diagnosis, I use inhaled insulin
that quickly and safely brings my blood glucose back in range. And I
wear a continuous glucose monitor, or CGM, that allows me to know at
all times whether my glucose is going too high or too low so I can take
action.
These advances have transformed the quality of my life.
I used to have to stash sugar on the set of plays I was in, in case
I needed to stabilize my blood glucose. And I can tell you it's kind of
strange when a character in a 18th century period drama pulls a bottle
of orange juice out of a sofa cushion and starts chugging mid-dialog!
Probably the most demonstrative example of how important these
advances are: Three years ago, I turned off my GCM so that my very
pregnant wife, Annie, could sleep peacefully through the night without
any beeping, which occurs when my glucose level goes low. Because I'd
switched it off, it did not alert me, I suffered a severe low blood
sugar while asleep, and woke up to a frightened pregnant wife, a crying
4-year old, and seven New York City EMTs standing over me.
These new technologies, when they are turned on, and other research
advances literally save my life every day and they save the lives of
every one of these delegates. And we are at the cusp of a whole new
generation of therapies, devices, and dare I say, a cure.
That's why we can't let up on research. We need more advances--so
we can cure and prevent T1D--so all of us, like Charlie, Lorynn and
Jonathan who are here on the panel with me, can live life without
thinking about this disease at all.
We need to keep the momentum going by renewing the Special Diabetes
Program before it expires at the end of September.
I'll let others today go on about the science and the policies that
support it. But let me just say this: this research has made a
difference in my life--it has made a difference in the lives of
everyone in this room--and millions more.
So thank you, Chairman Collins, for your outstanding leadership,
and thank you Ranking member Casey for your strong support of T1D
research and coverage for technologies like the Continuous Glucose
Monitor. It's great that people on Medicare now have access to CGMs
just like the rest of us.
And thank you and your colleagues for the bipartisan support of the
Special Diabetes Program. It's doing great work for the millions of
Americans living with this disease--like me.
Thank you.
[GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
Prepared Statement of Charlie Albair, JDRF 2017 Children's Congress
Delegate, Gray, Maine
Chairman Collins, Ranking Member Casey, and Senators, thank you for
inviting me to speak before you today.
My name is Charlie Albair from Gray, Maine. I am 10-years old, and
will be entering the 5th grade at Gray New Gloucester Middle School.
I am just like a lot of other kids. I love sports, especially
basketball and baseball. And when I grow up, I hope to play in the
Major Leagues--for the Boston Red Sox.
The one big difference is that I have type 1 diabetes or T1D.
I was diagnosed with T1D way back when I was 6-years-old. I was in
the first grade. I started not feeling like myself. I kept asking the
teacher to go to the bathroom because I really, really had to. She got
angry at me because she thought I just was trying to skip class.
She felt bad when she found out the real reason.
At first I was kind of confused when I was diagnosed. I didn't know
what it was; ``diabetes'' was a big word for a first-grader.
In the beginning, we treated my diabetes with syringes. And a half
a year later, I got the Omnipod pump and then a CGM to monitor my sugar
levels.
I love it.
I don't have to be constantly stabbing myself with a needle--like
five or 10 times a day.
What does this mean for me?
When I first found out I had diabetes, I remember thinking that
this would change my whole life. I thought that I wouldn't realize my
dream of being a sports star.
Now I can realize I can do whatever I want.
Sometimes my Omnipod or CGM beeps in class, and the other kids say,
``Charlie, stop making noise.'' I just tell them that that's my natural
``robot'' noise.
The pump and CGM are so much a part of me. But I do wish that they
didn't have to be.
I want my disease to go away--for me and all the other kids who
suffer from it. I want us all to be able to live without thinking about
it.
That's why I am here.
We need money for research.
We need money so scientists can invent new pumps and monitors
better than what we have now--and so they can come up with a cure for
T1D.
You have supported kids like me for so many years, and all I ask is
that you continue to do so. And if you do, I will invite you to a game
when I am on the Red Sox.
Thank you.
__________
Prepared Statement of Lorynn Watt, JDRF 2017 Children's Congress
Delegate, Stroudsburg, Pennsylvania
Chairman Collins, Ranking Member Casey, and Senators--thank
you for inviting me to talk with you today.
My name is Lorynn Watt, I am 17 years old, and this fall I
will be a senior at Evergreen Community Charter School in
Cresco, Pennsylvania.
When I was nine, I was diagnosed with type 1 diabetes. I
remember sitting on my parents' bed, just about a week before
Halloween, as my mom and step-dad told me that I had T1D. That
day they loaded me into the car and took me to the hospital,
where I'd learn how to care for myself.
At that point, I only knew about diabetes from an episode
of Hannah Montana I saw just before I was diagnosed. That was
it. What I did know was that I was scared, felt awful, and all
of a sudden was living a life where I had to inject myself with
insulin multiple times a day, even though I was horrified of
needles.
Then at 14, I got a continuous glucose monitor (or CGM) and
an insulin pump. It made my life so much easier. Now, all I
need to do is look at my phone--which most kids my age do all
the time--and I can see my blood sugar. It has been life-
changing, and I hope this, the artificial pancreas, and other
advances are small steps toward a cure.
I've heard every year since I was diagnosed that in 5 years
there will be a cure. I've had this disease for almost 8 years.
Now, I'm no mathematician, but you can see that we are a bit
behind schedule.
I believe that with your help we can have a cure.
After all, we already have come so far.
I know this because my biological father also had T1D. He
wasn't in my life much. He didn't have great care--no pump or
CGM or even ability to check his blood sugar every day.
So, he lost his foot, then his eyesight, and the use of his
kidneys. He had to get a stent in his heart. And he died less
than a year after my own T1D diagnosis, at just 38 years old.
I am here today asking you to support more funding for more
research because no one should have to suffer and lose their
life because of T1D.
I am here, inspired by his memory, and determined that none
of the kids here today or sitting in a hospital room right
now--scared--as they get their diagnosis, will have the same
fate as my father.
With your help, I know we can do it. I know we can find a
cure.
Thank you.
----------
Prepared Statement of Angie Platt, Chair Mom, JDRF 2017 Children's
Congress, Accompanied by her Son Jonathan Platt, Encino, California
Chairman Collins, Ranking Member Casey, and Senators--thank you for
inviting me to speak with you today.
I am Angie Platt from Encino, California. My husband Jon and I have
three children, all boys--twin 4 year olds and meet our oldest son
Jonathan who is 14 years old.
It's hard for me to believe that the 6'5'' young man sitting next
to me is the same boy who was here for JDRF's 2011 Children's Congress.
He was only 7 years old at the time, just 2 years into his diagnosis.
If you watch him play in his competitive basketball tournaments, it
would be very hard to believe that he is a child living with type 1
diabetes.
I am here today to tell you and your colleagues that Jonathan is
living proof that your leadership and actions have made a real
difference in our lives and the lives of all people with T1D.
In 2011, the T1D community asked you for help, and Senators, you
gave it. The Special Diabetes Program has provided hundreds of millions
of dollars of crucial funding for a range of therapies and
investigations--including the artificial pancreas.
In April 2016, Jonathan was enrolled in the pediatric trial for the
Medtronic Hybrid Closed Loop 670G--otherwise known as the ``artificial
pancreas.'' We felt as if we won the lottery.
This device has given Jonathan better blood sugar control than he
has ever had--and it gives our family some desperately needed peace of
mind. In the past, we would wake up at minimum 3 times a night to check
my son's blood sugar. Jonathan used to have to stop practicing with his
team or even sit out in a game because his blood sugars were too
erratic. Now, he gets to play right through crunch time.
As you know, last fall the FDA approved this artificial pancreas
system.
Senators, let me be clear: this would not have happened without
your support of the Special Diabetes Program.
Diabetes is relentless. We work so hard, we are so responsible, we
are playing by all the rules. Jonathan is on the latest most advanced
technology. Jonathan is doing everything right.
But the ugly reality of diabetes is that as hard as we work, our
kids are still vulnerable. This past June at Jonathan's eye exam it was
discovered that Jonathan has three dot hemorrhages. Quite frankly,
there are kids who are a lot worse off than Jonathan. Those of us here
are among the lucky ones.
I know that we have made progress; my son is wearing the first
artificial pancreas system approved in the world! But this disease
doesn't stop, so neither can we.
We need the next generation devices that can fully automate insulin
delivery.
We need to ensure that progress continues in the area of diabetes
complication treatments.
We also need to prevent others from ever developing T1D--including
my twin sons, Jonathan's brothers, who are at a higher risk of
developing it. They are enrolled in TrialNet, an SDP-funded prevention
program.
SDP has done so much, but it will expire on September 30th. The SDP
gives me so much hope, but it needs a hero. We need you and your Senate
colleagues to renew it for another three years so researchers can
continue their great work.
Senators, I want to thank you for all that you have done to make my
family's life and the lives of all of us here today better. Senator
Collins, I particularly want to thank you for your steadfast commitment
and outstanding leadership to advance type 1 diabetes research and to
help people gain access to new technologies. You have been a champion
for us for a long time.
Look at Jonathan--he will not lose to diabetes.
We will fight alongside Jonathan.
We will fight for all of these kids.
I ask you to continue to fight along with us.
Thank you.
[all]