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Diabetes Research: Joslin Director C. Ronald Kahn, M.D. Gives Testimony on Need for Increased Diabetes Research Funding

WASHINGTON — In early October, Joslin Diabetes Center Director C. Ronald Kahn M.D. presented testimony before a Senate Subcommittee on the need for greatly increased funding for diabetes research. His testimony calls for a five-year step up in the research budget for diabetes from the current level of $443 million to $827 million in the year 2000, rising to $1.6 billion by the year 2004. Dr. Kahn was the chairman of a Congressionally established committee to review and make recommendations on how federal dollars for diabetes research can be spent most effectively to help reverse the diabetes epidemic. His testimony reflects the recommendations of that Congressionally established Diabetes Research Working Group which he chaired.

Below is a transcript of his testimony.

TESTIMONY FOR THE SENATE PERMANENT SUBCOMMITTEE ON INVESTIGATIONS

C. RONALD KAHN, M.D.
October 14, 1999

First, I would like to thank Chairman Collins and Senator Levin for giving me a chance to present the report of the Diabetes Research Working Group to the Senate Subcommittee on Investigations. As you are aware, the DRWG was created in response to House Report (105-205 and 105-635) and Senate Report 105-98 to evaluate the current state of diabetes research in the US and to develop a strategic plan for the nation as to how best to proceed and make progress against this disease. In the next few minutes, I would like to briefly summarize what the Working Group found and indicate what some of our recommendations are.

So, "What is diabetes mellitus and why is it so important?" As I am sure most of you are aware, diabetes mellitus is not just a single disease, but a group of diseases, which have in common the presence of high blood sugar and many other metabolic abnormalities.

There are two major forms of diabetes

Type 1 - the insulin-dependent form of diabetes, often with juvenile-onset
Type 2 - the adult-onset form, often called non-insulin dependent diabetes, even though at least one-third of Type 2 diabetic patients take insulin.

Together, these two forms of diabetes currently affect an estimated 16 million Americans.

Over 700,000 children and young adults have Type 1 diabetes.
As people age, diabetes becomes even more common. In fact, if we all live to be 80 (and we all hope to live to be 80), 17% of the people in this room will have diabetes.
As a result of these two forces, about 800,000 new cases of diabetes are diagnosed each year.

In addition, there are many other forms of diabetes, some of which are rare, but others are not. For example, gestational diabetes, that is, diabetes that occurs specifically during pregnancy, affects over 5% of all women in the US who become pregnant.

Diabetes spares no one — from Avi Robbins, the 16 year-old son of my neighbor, to Nicole Johnson, the Miss America for 1999, from our grandchildren to our grandparents, from Italian Americans in Boston to Siberian Yupiks in Alaska, — all are at risk of diabetes.

Indeed, the minority populations of the US, that is the African-Americans, Hispanic-Americans, Native Americans and Asian Americans, represent some of the fasting growing segments of the population and are particularly vulnerable to diabetes and its complications. For example, among the Pima Indians of Arizona, over 50% of the adults have diabetes.

Not only does living with diabetes present many day-to-day challenges, diabetes also affects virtually every tissue of the body with long-term and severe damage.

Diabetic eye disease is the most common cause of blindness in adults.
Diabetic kidney disease accounts for 42% of cases of end-stage renal disease and is the fastest growing cause of patients requiring kidney dialysis and transplantation.
Stroke and heart disease are 2-4 times higher in people with diabetes, and especially increased in women.
Diabetes affects the nervous system leading to impaired sensation, pain, slowed digestion, impotence, and other problems.
The rate of congenital malformations in offspring of diabetic women is increased 3-4 times.
More than half of lower limb amputations in the U.S. are secondary to diabetes.

As a result, diabetes is the sixth leading cause of death in the U.S., and the third in some minorities.

One of the surprising findings, even to members of the DRWG, is that since 1980, the age-adjusted death rate due to diabetes has increased by 30%, while the death rate for other common diseases, such as stroke and cardiovascular disease, has fallen.

And least you think that these are just impersonal statistics, or that diabetes is not really that serious, I would like to point out that there five personal profiles of people with diabetes in this book which I present today. I am saddened to have to tell you that even in the few months that have passed since we began to assemble the final report, one of the individuals with diabetes highlighted in this report, Mr. Jerrold Weinberg of Detroit, has died at the age of 39 from complications of this terrible disease.

The economic impact of diabetes is staggering.

The cost of diabetes to the nation is conservatively estimated at $105 billion annually, and some estimates are as high as $130 billion.
Between 10% and 14% of all U.S. healthcare dollars are spent for diabetes.
One of every four Medicare dollar pays for health care of people with diabetes.

Another striking finding of the DRWG was that while healthcare costs for each person in the US affected with diabetes average $6,560 annually, the current investment in diabetes research is only about $30 per person affected per year. That is, less than 1/2 of one percent of the cost of this disease is invested in R & D in attempt to reduce the burden created by diabetes. This is a small investment for a disease that affects 6 to 7% of the population and accounts for about 10 to 14% of all health care dollars.

With these facts in mind, the Strategic Plan created by the DRWG had multiple goals.

We must -

Understand the causes of diabetes and its complications
Develop methods to prevent and treat diabetes and its complications
Reduce the impact of diabetes in minority populations
Develop a research infrastructure and train investigators to do the necessary research
Translate theses findings into clinical practice

In developing an approach to this plan, I took some advice from Lee Iacocca, another strong supporter of diabetes research. When we first met, I told the group, "Imagine we are in the year 2010, and would like to say what we have accomplished for diabetes. Then given these goals, what would you have to do over the next decade to have at least a chance of accomplishing some of them?" Ultimately, what came out of this approach was the 140 page Research Strategic Plan that you have before you today.

There are three major components of the DRWG Strategic Plan:

Extraordinary Opportunities:

These represent rapidly expanding and important areas of research in which increased investment or development of new approaches will significantly speed research.
Special Needs for Special Problems:

These are equally important, but more focused research areas usually targeted to specific populations, complications, and methodological approaches.
Resources and Infrastructural Needs:

A plan for increasing research manpower, technology and other infrastructure elements for diabetes research.

In all, the Plan contains a total of 88 recommendations in 16 different categories.

Let us briefly look at a few of these:

Perhaps one of the most exciting areas in all of research today is the "Genetics of Diabetes and Its Complications." If we identify the genes for diabetes and its complications, we may some day be able to predict and prevent the disease. Understanding the genetics also will give us the opportunity to develop new therapies that are directed at the true central problem of the disease.

The DRWG proposes several initiatives in the area of genetics, including creation of a new National Consortium for the Study of the Genetics of Diabetes. One of the goals of this Consortium would be the development of a diabetes DNA chip. This is a DNA chip that we are using in my own laboratory to begin to identify some of the genes which might be altered in diabetes. With the proper investment in research, within a decade a diabetes chip could be developed that, when exposed to DNA from a few drops of blood, will tell us who is likely to develop diabetes, which of our diabetic patients are most likely to develop complications, and who will respond to each specific treatment approach.

A second area is "Autoimmunity and the Beta Cell." This holds the key to Type 1 Diabetes, since type 1 diabetes is an autoimmune disease that destroys insulin-producing b-cells. Important progress has been made in this area over the past several years, including

Identifying some of the major genes predisposing to Type 1 diabetes and the components of the beta cell that are attacked
Developing markers for detection of pre-diabetes
Demonstrating the critical importance of "tight" control of blood glucose for reducing complications (DCCT)

But there remain many challenges which are critical if we are to conquer this disease. The DRWG report, therefore, describes a program to

Intensify research to understand the immunological basis of Type 1 diabetes
Develop optimal strategies for blocking immune destruction of beta cells, including both pre-clinical and clinical studies
Expand research on Transplantation as therapy for Type 1 diabetes. This requires solving critical issues, such as
- How are we going to get enough islet cells to treat hundreds of thousands or millions of patients who could benefit from this treatment?
- How can we protect the transplanted cells from immunologic rejection?

Other Extraordinary Opportunities include research in "Cell Signaling and Cell Communication," which we believe hold the key for Type 2 diabetes; "Obesity"; and the area of "Clinical Research and Clinical Trials" to develop proper evidence-based approaches to this disease.

The DRWG has also made special recommendations regarding research into the "Eye, Kidney and Nerve Complications," as well as the "Cardiovascular Complications" which are the major killer of people with diabetes. In these areas, we must:

Define why and how diabetes enhances the atherosclerotic process and other diabetic complications
Determine why women with diabetes lose their vascular protection
Identify factors in the heart that lead to increased mortality after a heart attack and develop new therapies to enhance survival
Develop specific, noninvasive techniques to identify the presence of diabetic complications, predict their progression and assess the response to therapy.

We also describe research programs to better understand the "Impact of Diabetes in the Young and Old, in Women, and in Minority Populations" where there is a need for more basic research, as well as culturally sensitive approaches to applied clinical research.

BUDGET RECOMMENDATIONS

As I have already indicated, the current investment in research is small, and indeed far too small to provide the resources needed to begin to address the research plan.

As requested by Congress, the DRWG has developed budgetary recommendations to accompany this plan. You will see that these recommendations call for a five-year step up in the research budget for diabetes from the current level of $443 million to $827 million for the year 2000, rising to $1.6 billion by the year 2004.

Let me point out to this Committee that these numbers are not arbitrary. They are based on a detailed planning process and what is realistically needed to bring diabetes research to a point where there would at least be a chance that we could accomplish some of the goals we have set forth over the next ten years.

In the full report, this budget is detailed with project-by-project and Institute by Institute recommendations.

I would also like to point out that the members of the DRWG do not believe that diabetes research should be funded by taking resources from other important biomedical research needs. The DRWG is strongly supportive of the NIH, the Director of NIH, Dr. Harold Varmus, and the Director of NIDDK, Dr. Philip Gorden, and recognize the many challenges which must be faced in utilizing these precious resources. Like the military, however, which must be prepared to fight a battle on at least two fronts, the NIH and the biomedical research community must be prepared to fight the battle of human disease, not just on two fronts, but on many fronts at once — and this will require a significant investment, both immediately and in the long term.

The DRWG is convinced that there is both great urgency and unprecedented opportunities in diabetes research. The DRWG is also convinced that taking action now will save thousands of men, women and children from the severe consequences of diabetes, and save the nation billions of dollars in medical care and lost productivity. We have done the first part of our job by developing the comprehensive plan for diabetes research that you requested. We present this report to you and ask now for your support in going to the next phase — by allowing us to do the research that will be required to conquer this dreadful disease.