Type I diabetes is a major health problem, affecting more than 1.5 million Americans, and advances in therapy for this disease are of interest to both physicians and patients. In the past few years, significant advances have been made in our understanding of the pathogenesis, prevention, and potential cure of this disease. These advances formed the theme of a symposium held at Experimental Biology 2001 in Orlando, Fla, which was sponsored by the American Federation for Medical Research and the Juvenile Diabetes Research Foundation International (JDRF). The symposium opened with Dr Richard Furlanetto of the JDRF introducing the speakers and topics of the session. The session began with Dr George Eisenbarth discussing the pathophysiology of type I diabetes. Dr Dale Greiner then discussed immune tolerance, a potential target for both treatment and possible prevention of this autoimmune disorder. Finally, Drs Mark Atkinson and James Shapiro discussed ongoing trials in prevention and treatment of type 1 diabetes, respectively.
Type I diabetes is an autoimmune disorder. Both genetics and environmental components play a role in the pathogenesis of the disease. The emerging hypothesis about the pathophysiology of type 1 diabetes is that a heterogeneous genetic component and one or more autoantigens interact to trigger the autoimmune process. Ultimately, type 1 diabetes represents a heterogeneous and polygenic disorder, with a few rare examples of monogenic mutations leading to autoimmune diabetes.
Early studies in identical twins who develop type I diabetes indicate the heterogeneous nature of this disorder. If the first twin develops type I diabetes after the age of 25 years, then the risk of subsequent development of diabetes in the other twin is less than 5%. However, if the first twin develops type I diabetes before the age of 5 years, the risk for the second twin is very high, approximately 50% …