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Statement on Juvenile Diabetes by Phillip Gorden, M.D.
Director, National Institiute of Diabetes and Digestive and Kidney Diseases
National Institutes of Health
U.S. Department of Health and Human Services

Before the Senate Appropriations Committee, Subcommittee on Labor, HHS and Education
June 22, 1999


Mr. Chairman, I am Phillip Gorden, the Director of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which has lead responsibility for diabetes research at the National Institutes of Health (NIH), within the Department of Health and Human Services. I appreciate the opportunity to testify before this Subcommittee about NIH research to understand, treat, prevent, and ultimately cure diabetes.

In both human and economic terms, diabetes is an extremely costly disease. It affects an estimated 16 million Americans, including both genders, the young and the old, all races and ethnic groups, the rich and the poor. Consistent with the focus of today's hearing, I will address my testimony toward diabetes in children, who, in many ways, suffer most from the disease. They have the disease from an early age and must endure lifelong treatment. They must carefully adjust what they eat and everything they do--from schoolwork to sports--in order to manage their disease. Even with a continuous struggle to follow such rigorous regimens, they may still develop serious, long-term complications of diabetes.

Children with type 1 diabetes must have daily insulin injections to survive. They and their families must monitor their blood glucose levels throughout the day. While the value of maintaining blood glucose control is clear, this therapy is extremely difficult, and must be made better and easier for everyone.

Based on case reports and clinic-based studies, new information is emerging about children with type 2 diabetes. It appears that these children are not dependent upon insulin injections; however, their cells and tissues are resistant to insulin. Some may require insulin to maintain control of their blood glucose. The number of children with type 2 diabetes is increasing in our population and the age of onset is now occurring earlier. This is a special problem in racial and ethnic minority populations, who suffer disproportionately from diabetes, as well as from obesity--a major risk factor for type 2 diabetes. In most cases, type 2 diabetes in children appears as a complex, polygenic disease similar to that seen in adults. The NIDDK will be bringing together pediatric endocrinologists from across the U.S. to share information on the emerging problem of type 2 diabetes in children.

HIGHLIGHTS OF NIH-WIDE RESEARCH EFFORTS AND STRATEGIES

I would like to emphasize several points that are central to present and future efforts to treat diabetes effectively and ultimately cure it. The many institutes and centers of the NIH have a broad and multifaceted research agenda to treat, prevent and cure diabetes. The trans-NIH diabetes effort has led to major clinical advances in diabetes, and clues in the search for a real cure. We are witnessing rapid advances in genetics and genomics that underlie our goal to find and eliminate the cause of many diseases, including diabetes. Discoveries of mechanisms to manipulate the immune system have significant implications for diabetes prevention and treatment. Major new understandings of cell communication are critical to diabetes. Key advances in clinical research are showing how the control of blood pressure and serum cholesterol, as well as other lipids, is important in the comprehensive care of patients with diabetes.

The Diabetes Control and Complications Trial (DCCT) demonstrated that the complications of diabetes affecting the eye, kidney and nerves can be ameliorated or prevented. These results have been extended by the clinical progress achieved in treating diabetic eye disease with photocoagulation, and the use of drugs--such as ACE inhibitors--for the kidney disease of diabetes. Together, these advances represent major steps forward in our continued quest for a cure. However, they also further emphasize the compelling need to develop better technologies both to manage blood glucose levels and to treat complications more effectively and directly.

In the past decade, investigators supported by the NIDDK, the National Institute of Child Health and Human Development (NICHD), and the National Institute of Allergy and Infectious Diseases (NIAID) have been able to establish immune, metabolic, and genetic screening tests to identify individuals at high risk for developing type 1 diabetes. In animal models and in preliminary human trials, researchers have also shown that low-dose insulin therapy may prevent or delay the onset of the clinical manifestation of type 1 diabetes. Thus, scientists can identify individuals at high risk for type 1 diabetes and to intervene with a safe and possibly effective therapy. These tools are now being applied in a major multi-center clinical trial cosponsored by the NIDDK, NIAID, NICHD, the Juvenile Diabetes Foundation International (JDF) and the American Diabetes Association to prevent or delay the onset of type 1 diabetes. This trial is called the Diabetes Prevention Trial-1, or DPT-1. Another clinical trial, "Prevention of Cardiovascular Disease in Diabetes," will generate knowledge that will be important for preventing heart and vascular disease in early onset type 1, as well as type 2 patients. The National Heart, Lung, and Blood Institute (NHLBI) is sponsoring this trial, in conjunction with the NIDDK.

We at the NIH join the children and families touched by diabetes in a shared effort to support vigorous, promising research aimed at the prevention, treatment and cure of this disease and have been pleased to collaborate with voluntary organizations, such as the JDF, in fruitful research partnerships.

To guide our diabetes research programs, we have sought the best advice possible from the scientific and voluntary diabetes community. For example, we have accelerated and enhanced our efforts based on a special trans-NIH workshop, entitled "Diabetes Mellitus: Challenges and Opportunities" -- complemented by the Strategic Plan of the Diabetes Research Working Group. We have already initiated many recommendations from these processes and will continue to build upon them in the future. I am pleased to share with you today some of our most recent efforts, which relate to diabetes in children. A number of these have been undertaken in partnership with other NIH Institutes, other agencies, and voluntary organizations.

SPECIAL INITIATIVE ON TYPE 1 DIABETES

I am pleased to report progress on a special, NIH-wide initiative for innovative, clinically relevant and multidisciplinary research aimed at the treatment and cure of type 1 diabetes. This initiative is relevant to all of the scientific opportunities in type 1 diabetes research today, but especially to the development of more effective therapies, which can be easily administered and followed. Through this initiative, we are seeking the best research talent from diverse fields, the most promising research ideas, and the most technologically advanced research tools for combating type 1 diabetes. We are exploiting the fruits of the biotechnology revolution, with special attention to clinical issues.

Initiatives have been launched to develop therapies to achieve normal glucose levels in people with type 1 diabetes and to develop improved glucose sensors for regulating blood glucose. They also include expanded programs to understand the mechanisms by which high glucose levels result in the late complications of diabetes; to apply this information to the development of ways to prevent, limit or reverse complications associated with diabetes; and to understand the role of factors important in disease development.

This year special funds for type 1 diabetes research appropriated in the Balanced Budget Act of 1997 are being used to focus on the mechanisms by which the disease results in painful and disabling neuropathies and other neurological complications; identification of stem cells and factors that regulate development and differentiation of pancreatic beta cells through the establishment of a functional genomics resource in diabetes; and pilot studies for new therapies for type 1 diabetes and its complications.

INITIATIVE ON CELL-BASED THERAPIES FOR TYPE 1 DIABETES

We are embarking on a new and exciting initiative to restore insulin-producing capacity through transplantation of the whole pancreas, or of islets from the pancreas. This research is being propelled by an impressive series of advances. Recent studies in primates have shown that both insulin-producing islet cells and kidneys can be transplanted using a highly selective method to control for immune rejection of the transplant. This new technology involves what is referred to as "blockade of the co-stimulatory pathway." It allows for a selective form of immune tolerance and does not require suppression of the overall immune system, as is required by conventional therapy for organ transplantation.

Paralleling this initiative is a major, new, collaborative effort on immune tolerance within the intramural program of NIH. The research partnership involves the NIDDK, the Warren Grant Magnuson Clinical Center, the Department of Defense, and the Diabetes Research Institute of the University of Miami. The strategy under study is relevant to both the treatment of type 1 diabetes and kidney transplantation. Furthermore, there is a major, additional joint effort involving NIDDK, NIAID and the JDF to broaden this program into a network of collaborating institutions.

INITIATIVE TO ENHANCE MODALITIES OF TREATMENT

We are working diligently to develop a wide range of new and more effective therapies for avoiding the consequences of low blood glucose levels and for improving the treatment of diabetes. For example, the NICHD is supporting two comprehensive studies in adolescents on how low blood glucose levels affect learning skills and how the undesirable effects of multiple daily insulin injections affect compliance. We are also pioneering the development of glucose sensors and mechanical systems to facilitate insulin administration and thus ease the burden of this therapy for children and adults who are insulin-dependent.

One exciting recent advance may well have important therapeutic implications with respect to the immune system's attack on its own insulin-producing cells in type 1 diabetes. Researchers have shown that a protein called GAD, which is expressed by beta cells, controls the development of diabetes in an animal model of human type 1 diabetes. The demonstration that this protein initiates autoimmune diabetes builds on an earlier NIH-supported advance, which showed that cells specifically reactive against GAD directly produced beta cell injury in a mouse model. This avenue of research could have important consequences for the development of new therapies to prevent type 1 diabetes, provided these findings can be extended to human disease.

We also hope that new clinical advances will emerge from other NIH-supported investigations of pancreas and islet transplantation in animals. This work includes studies on ways to regenerate the pancreas; to develop methods to enable the protection and survival of implanted insulin-producing cells; and to discover innovative approaches to prevent graft rejection by induction of immune tolerance.

INITIATIVE ON PREVENTION AND TREATMENT OF COMPLICATIONS

Multiple NIH institutes are participating in a major initiative to combat the eye, nerve, kidney and vascular system complications of diabetes. These efforts include the search for genes that make individuals with diabetes particularly susceptible to developing one or more of these complications. Also featured is a new emphasis on understanding and treating diabetic nerve disease, and the inauguration of a major clinical trial aimed at reducing the cardiovascular complications of diabetes. These research areas have been identified as having high priority by the Diabetes Research Working Group.

INITIATIVE FOR DIABETES CLINICAL TRIAL NETWORK

One of the recommendations of the Diabetes Research Working Group is to establish a Diabetes TrialNet to foster clinical studies. In response, the NIDDK recently provided administrative extensions to the centers involved in the ongoing DPT-1, while a task force considers approaches for establishing such a network for future studies of prevention and treatment of type 1 diabetes. A component of this initiative will probably include support for the infrastructure for clinical researchers and nurses to conduct research; a central data coordinating center; central laboratories; mechanisms to review and prioritize research projects; and, databases of research volunteers, investigators and projects. Such a diabetes clinical research network would provide the necessary infrastructure for the efficient, rapid evaluation of promising new therapeutic approaches.

RESEARCH PARTNERSHIPS

In closing, I would like to mention just two examples of the many beneficial research collaborations between the NIH and voluntary health organizations, such as the JDF. The NIDDK-JDF centers of excellence program represents a productive research partnership and a model that has been adapted by several other NIH institutes. Also, the JDF and the NICHD are now co-funding a study of 12,000 infants who are at various levels of genetic risk for type 1 diabetes in order to detect the earliest evidence of immune attack on the insulin producing cells of the pancreas. These examples reflect the strong relationships the NIH and voluntary health organizations have formed to accelerate research progress.

Mr. Chairman, I am grateful for the opportunity to share with you recent and exciting NIH efforts focusing on diabetes in children. I have tried to emphasize today that we at the NIH truly understand the great burden diabetes places on families. At the same time, I want to share my feelings of great encouragement and hope because of the pace at which diabetes research is moving. I believe that our strong national research programs hold the essential key to curing this disease for the benefit of all children and their families. I am pleased to answer any questions you may have.


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