Testimony
By
Mary Tyler Moore
New York, NY

On Behalf of the
Juvenile Diabetes Research Foundation International

"Juvenile Diabetes - Examining the Personal Toll on Families, Financial Costs to the Federal Health Care System, and Research Progress Towards a Cure"

Before the
Senate Governmental Affairs Committee
216 Hart Senate Office Building

June 24, 2003
10:00 a.m.

Good Morning. Before my formal remarks, Senator Collins, I'd like to personally thank you for your leadership and all your efforts on behalf of people with diabetes, including conducting these hearings and Co-Chairing (with Senator Breaux) the Senate Diabetes Caucus. With your continued support, I know we will, together, find a cure. Now, please allow me to offer this testimony:

Chairman Collins, Senator Lieberman, committee members. I am Mary Tyler Moore, and I'm here today as International Chairman of the Juvenile Diabetes Research Foundation. I wish I could say that I am happy to be here. But meaning no disrespect, I'm not. You see, I've had juvenile diabetes for more than 35 years now, and I'm tired of it. I sincerely wish that I didn't have to come back, year after year, to seek your help - but I do. I have to, for myself, for everyone with diabetes, and most especially for the beautiful and courageous children who are here with me today.

Be certain, at JDRF we have never been more dedicated to finding a cure for diabetes and its complications -- and the evidence suggests we are truly close. Much of the progress that has led us to what we hope and believe are the final stages of our fight against diabetes is the result of your support and effort. We greatly appreciate the good partnership we have always had with Congress and every Administration since our founding. We are incredibly grateful for the extraordinary support you all showed last year in passing the Special Juvenile Diabetes Program-the most significant legislation ever enacted relating to juvenile diabetes research. But, as you can see, I'm here again. Despite all our accomplishments together, we must face the fact that there are still significant hurdles we need to overcome to take the last necessary steps along our path to a cure.

Senators, some of you have already met many of the 200 child delegates to JDRF's 3rd Children's Congress who are here in Washington this week; you'll be hearing from some, and meeting others, later today. They are passionate and eloquent spokespeople for the need for a cure. But don't be misled by their drive, their energy, and their unwavering commitment. They - and I - struggle every minute of every day to do what happens naturally for people who don't have diabetes: achieve a balance between what I eat, what I do, and how I feel. For most of you, blood sugar balance is as automatic as breathing. But for people with juvenile diabetes, like me, it requires vigilance 24 hours per day, seven days per week, 365 days per year.

Each of these children and I need to be a mathematician, a physician, a personal trainer, and a dietician all rolled into one. We need to be constantly factoring and adjusting, making frequent finger sticks to check blood sugars, and giving ourselves multiple daily insulin injections just to stay alive. Not to live life to its fullest, mind you; just to stay alive. And God knows, it isn't easy. Even with the greatest of care and the closest of personal scrutiny, like many children and adults with juvenile diabetes, I find I am often unable to achieve good balance. My blood sugars can go dangerously low, or frighteningly high. Yes, dangerous and frightening - because serious lows can lead to seizures, coma, and death. And high blood sugars can, over time, result in disabling and life-shortening complications. Some of you may already know the startling toll diabetes takes. For example, diabetes causes over 40% of kidney failures in our country that require dialysis or transplantation. It is responsible for more than half of amputations not associated with traumatic injuries. Is the leading cause of blindness in adults - the leading cause. Moreover, I've seen studies that say that virtually everyone with juvenile diabetes shows evidence of heart disease by age 40 - age 40! - and that pre-menopausal women with juvenile diabetes have a more than 30 times greater risk of death from heart attack. Beyond the incomprehensible personal costs, consider the economic burden our country must bear as a result of this disease: diabetes costs this nation over $130 billion per year. This includes one out of every four Medicare dollars spent. Taken altogether, it should be crystal clear why it is urgent that we find a cure, and find it as soon as possible.

Chairman Collins, you and your colleagues, from both sides of the aisle, have always welcomed us warmly during our past Children's Congresses. In our prior meetings, you've promised to remember us when making decisions about funding for juvenile diabetes research. Today, we are thrilled to acknowledge to all who might listen that you are, indeed, keeping your promise.

We thank you not just as your constituents, but also as your partners in a shared mission to find a cure. Reflecting our long-term commitment to partnering with government for this purpose, JDRF is working closely with the NIDDK - the lead agency responsible for investing the supplemental research dollars called for in the Special Diabetes Program --- to ensure that this funding is truly responsive to the needs of people affected by juvenile diabetes. From our discussions with Dr. Alan Spiegel, NIDDK's director and a good friend of JDRF, we are confident that these "special" dollars will be used to fund projects that otherwise could not be undertaken through the usual NIH appropriation. And we expect that these projects will focus on what's needed to rapidly acquire mission-critical knowledge, provide essential research resources, and speed the application of scientific advances for the benefit of people with juvenile diabetes.

Madame Chairman, Senators, it's vital to remember that because children and adults with juvenile diabetes and their families never get a rest from their disease, we never rest in our efforts to find and deliver them a cure. And because JDRF is truly a global cure enterprise, we are always looking beyond the horizon, to anticipate what we must do next to achieve our ultimate goal - a world without juvenile diabetes. Our approach has consistently focused on "what's next." Let me give you an example, looking at four steps we have taken to get to the point where we are today in islet transplantation:

Step one: In the mid-90s, JDRF created a map of all that is known about diabetes, and identified the knowledge gaps and obstacles to progress along the various paths to a cure. We use this continuously updated map to guide us in efforts to push scientific advances from the laboratory bench quickly to the patient's bedside.

Step two: This process made clear that islet cell transplantation is a potential cure. So we invested in creating a global network of research centers to prepare and distribute human islets for research.

Step three: As it became clear that with JDRF support, research Centers throughout the world were able to test islet cell transplantation, JDRF moved its focus ahead. We substantially boosted our investments in research aimed at the induction of immune tolerance, concentrating on finding the means to replace the need for transplant patients to take toxic immunosuppressive drugs for the rest of their lives.

Step four: In 2000, when Dr. James Shapiro's group in Edmonton reported the first major clinical success in islet transplantation, JDRF jumped to the next obstacle confronting transplant research: the minute supply of islets for transplantation. To create an unlimited supply of islet cells for transplantation, we led a global effort to support stem cell research. This is particularly necessary because each year, there are fewer than 2,000 cadaver pancreases donated. Yet well over 1 million Americans with juvenile diabetes could potentially benefit from islet transplantation.

Overall, we've made terrific progress, Madame Chairman, but we continue to look into the future and ask ourselves, "What's Next?" Here is what we believe must be next, to speed a cure for juvenile diabetes:

First, we need policies and regulations that encourage organ donations and promote the retrieval of additional human pancreases. As I said, supply is a major obstacle to making islet transplantation a cure for all those with diabetes who might benefit. I am thrilled that you have shown real leadership and vision by introducing "The Pancreatic Islet Cell Transplantation Act of 2003." This bill, which JDRF strongly supports, will provide regulatory incentives for organ procurement organizations to retrieve additional pancreases. It will also create a demonstration project within Medicare to test insurance coverage for islet transplants for people with juvenile diabetes and kidney failure. I am hopeful that Congress will pass the legislation this year, and we are eager to work with you on this.

Second, we need to work with the Senate and the President to make the policy adjustments required to be certain we can accomplish our goal and that of the President: empowering stem cell researchers to find ways to relieve the suffering of millions of Americans. Even with increased organ procurement, we will - ultimately - still face a critical shortage of islet cells for transplantation. As a result, we need to ensure that the public and private sectors have the scientific freedom and support required for the U.S. to take its proper place as the world leader in promoting stem cell research and the development of human stem cell-derived therapies.

As you know, Madame Chairman, right now, due to the restrictions of current Administration policy, this just won't happen. I know the President worked hard to find balance in this policy. He clearly recognized the great potential of stem cell-derived therapies to reduce pain and suffering of millions of people, with a host of diseases, including juvenile diabetes. And, in my heart, I know it was his intent to make it easier, not more difficult, to create therapies to treat human disease. But now, nearly two years after his August 9th, 2001 decision, researchers tell us that the progress being made in stem cell research is not as far along as it should be. The number of cell lines everyone had hoped for - more than 60 were supposed to be available - turns out to be closer to 10. Of these lines, only a few are widely available for research. Perhaps most important, of those "approved lines" none - that's right none - can be used to develop human therapies. The problem is that each of those cell lines were established using mouse feeder cells or other non-human products; the threat of contamination makes them unusable for human therapies.

For these reasons and others, the best and the brightest of young researchers in the U.S. are shying away from the field of human stem cell research. So not only do we have an insufficient number of cell lines to conduct the necessary research, we have a potentially more devastating deficiency of brainpower.

Again, I have no doubt this was not the outcome the President intended, since if unaddressed, it certainly does not well serve the interests of the American people. Similarly, I should acknowledge that the White House, Secretary Thompson, and Dr. Zerhouni, the NIH director -- as well as other NIH directors working on the NIH stem cell task force - are all doing what they can, within the current constraints. We are working closely with them, and we are all the same page in terms of our need to focus on finding a cure as soon as possible.

Third, we need to continue to invest in developing methods to replace human islets without immunosuppression. It is nothing short of miraculous that today, just three years after the Edmonton Protocol, more than 250 people have undergone successful islet transplantation and no longer need insulin injections to survive. But the procedure is not yet safe for children and most people with diabetes. So we must sharpen our research focus on immune tolerance, as well as redouble our efforts to prevent diabetes and reverse or prevent its devastating complications.

Finally, because we want a cure for everyone with juvenile diabetes, including the children here today, we need to establish a framework for research oversight which properly balances the need to protect children from harm with the need to support the conduct of the research required to find the cure on their behalf.

Madame Chairman, diabetes is an all-too-personal time bomb, which can go off today, tomorrow, next year, or ten years from now. A time bomb that affects millions, including me and the children here today. It needs to be defused. But to find a cure for diabetes and its complications, and then make these cures available to everyone who might benefit, will require that we remain vigilant in our purpose and continuously committed to asking, answering, and acting upon the "What Next?" question.

So "What's next?" for you. Please listen to the children this morning, who'll tell you how they struggle with juvenile diabetes. Learn about how they bravely face its daily challenges - challenges no 15-year-old, or eight-year-old, or two-year-old should have to endure. Feel their longing to know a day without diabetes and live the normal, care-free life of a child. Listen to the researchers, as they highlight the progress made to date and the exciting opportunities we can now capitalize on because of JDRF and NIH leadership - and the wonderful investment Congress has made in medical research. And hear first hand from an islet transplant recipient about how spectacularly her life has changed since her procedure - and how she won't rest until her young son who also suffers from juvenile diabetes can be cured.

Finally, please join me and JDRF in making a promise to remember what we have learned today, and as a result ask ourselves: What Next?

Thank you, once again, for all you have done, and for the opportunity to speak with you this morning.