Lessons from Prediction and Prevention of Type 1 Diabetes

Abstract

Two major accomplishments in the field of prediction and prevention have had important implications for type 1 diabetes (T1D), as well as other immune-mediated diseases such as inflammatory bowel disease (IBD). First, individuals destined to develop T1D can now be identified by testing for the presence of autoantibodies, long before signs or symptoms of clinical disease. Second, there is now an FDA-approved therapy to delay disease progression. These accomplishments were made possible by more than 4 decades of multicenter research, sustained commitments from funders, a culture of collaboration that enabled international standardization of autoantibody measurements, and common entry criteria and outcome measures for clinical trials. Robust data find that essentially everybody with 2 or more autoantibodies will eventually progress to clinical disease. This concept is now codified as stages of T1D, whereby those with multiple autoantibodies are considered to have early-stage disease. This provided a pathway for regulatory approval by clarifying that therapy was aimed at treating people with disease in contrast to preventing disease in healthy individuals and has helped antibody-positive individuals understand risk. Translating these scientific accomplishments into clinical practice remains challenging, but the availability of an approved therapy to delay T1D onset suggests that such translation is on the horizon for T1D and ultimately for other immune-mediated diseases.