Continue to Overview - Part 1

Diabetes Mellitus: Challenges and Opportunities
Final Report and Recommendations

Introduction

Report and Recommendations of the Trans-NIH Symposium: "Diabetes Mellitus: Challenges and Opportunities" September 4-5, 1997

On September 4-5, 1997, the Director of the National Institutes of Health (NIH) and the Director of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) sponsored-with the co-sponsorship of eight other institutes and centers of NIH- a unique, landmark symposium on "Diabetes Mellitus: Challenges and Opportunities." The NIH components that co-sponsored this symposium were the National Center for Research Resources; the National Eye Institute; the National Heart, Lung, and Blood Institute; the National Human Genome Research Institute; the National Institute on Aging; the National Institute of Allergy and Infectious Diseases; the National Institute of Child Health and Human Development; and the National Institute of Neurological Disorders and Stroke.

The NIH-wide scope of this symposium reflects the multifaceted and complex nature of diabetes and its complications, which attack many organ systems of the body, including the kidneys, eyes, nerves, and heart. Diabetes and its complications affect an estimated 16 million Americans at an annual cost of approximately $92 billion, which includes direct medical costs and indirect costs such as disability, work loss, and premature mortality.

The major conclusion of the symposium was that diabetes research needs to be intensified in order to close research gaps, take advantage of new technologies, and capitalize on highly promising research leads and advances.

The symposium participants recognized that science has produced or contributed to major progress in the understanding, diagnosis, and treatment of diabetes. Examples include the following:

Research Advances Providing Key Insights into the Causes and Disease Processes of Diabetes

• Recognition of type 1 diabetes as an autoimmune disease.
• Recognition of type 2 diabetes as a disease related to the body's resistance to insulin and to the body's impaired ability to secrete insulin.
• Achievement of major inroads into understanding the autoimmune process in type 1 diabetes and the mechanisms through which cellular signals are produced and transmitted in this process.
• Demonstration in epidemiologic studies that lifestyle changes can modify the occurrence of type 2 diabetes.
• Epidemiologic evidence that type 2 diabetes disproportionately affects minority subpopulations, including African Americans, Hispanics, Native Americans, and Alaska Natives.

Research Advances Achieved through the Application of the New Techniques of Molecular and Cellular Biology and Molecular Genetics to Diabetes

• Development of genetic markers and other biochemical indicators to predict risk for developing either type 1 or type 2 diabetes, thus paving the way for clinical trials aimed at preventing or delaying the onset of the disease in such populations.
• Discovery of the insulin receptor and its contribution to the insulin transduction system.
• Discovery and use of radioimmunoassay and other sophisticated laboratory procedures that have permitted precise measurements essential to understanding critical events in the onset and progression of both type 1 and type 2 diabetes.
• Successful pancreas transplantation in humans undergoing kidney transplants, and development of experimental bioengineering and surgical approaches to improve islet cell transplantation.
• Development of implantable insulin pumps, glycosylated hemoglobin tests, home glucose monitoring and other tools to permit near normalization of blood glucose levels for prolonged periods of time.
• Discovery of genes associated with certain forms of diabetes and identification of candidate genes for intense study.
• Discovery of genes for obesity, a major risk factor for type 2 diabetes.

Research Advances with Clinical Significance for the Treatment and Prevention of Diabetes

• Discovery of insulin as a treatment for type 1 diabetes, production of pure insulin derived from animals and subsequent development of a process for producing human insulin through genetic engineering.
• Development of several oral drugs effective in the treatment of type 2 diabetes.
• Demonstration that close control of blood glucose levels can significantly prevent or delay the eye, kidney, and nerve complications of diabetes.
• Reduction in infant mortality and morbidity associated with diabetic pregnancies through close control of blood glucose levels in pregnant diabetics.
• Discovery that laser treatments can significantly reduce the risk of blindness from proliferative diabetic eye disease.
• Discovery that a class of blood-pressure-lowering drugs can ameliorate the kidney disease of diabetes.

Yet, despite this remarkable progress, symposium participants concluded that major deficiencies still exist in our knowledge base about the underlying causes and disease processes of diabetes and its complications. Even though diabetes research has appropriated and harnessed many of the new tools of molecular and genetic technology, compelling research questions remain unanswered and many promising scientific opportunities and needs remain to be pursued. Perhaps most important, deficiencies exist in the development and application in medical practice of efficient, effective, and practical models for treating diabetes. As with many other chronic diseases, diabetes has many complex features that make the development of new therapies especially difficult.

The purpose of the September symposium was to bring together leading experts in the areas of diabetes and related research to examine the state of the science and identify research gaps and opportunities that could be pursued across NIH. This one-and-one-half day meeting began with a plenary session, which outlined central themes related to diabetes research. The participants then divided into five work groups for more in-depth discussions. The work groups were organized around the following topics:

• Type 1 Diabetes-Etiology and Pathophysiology
• Type 2 Diabetes-Etiology and Pathophysiology
• Therapy
• Microvascular Complications
• Macrovascular Complications

At the end of this symposium, the leaders of the plenary and work group sessions developed a detailed scientific report, with recommendations concerning the future directions diabetes research should take to close the gaps in our knowledge and capitalize on scientific opportunities. This report, provided in Section IV, provides specific suggestions that can be pursued through research initiatives involving multiple components of NIH. Preceding the full technical report generated by the leaders of the symposium is a two-part overview of the report. The overview summarizes (1) the scientific accomplishments, gaps, and opportunities identified and (2) the research recommendations made.

The report and recommendations emerging from this important symposium are expected to provide the scientific framework for the development of future trans-NIH diabetes research efforts.

Continue to Overview - Part 1