Target Discovery to Diabetes Therapy - TXNIP From Bench to Bedside with NIDDK.

Abstract

Diabetes is the most expensive chronic disease in the U.S. with over $400 billion in annual costs and it affects over 38 million Americans. While major advances in drug treatment have been made for type 2 diabetes (T2D) and the often-associated obesity, there are still no approved and effective medications targeting beta cell loss or islet dysfunction, which is one of the major underlying causes of both, type 1 diabetes (T1D) and T2D. In addition, there are no oral medications for T1D approved in the U.S. more than a hundred years after the discovery of insulin and attractive therapeutic targets are only starting to emerge. As we celebrate the 75th anniversary of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), progress is finally being made in this area with NIDDK support. This mini-review follows the discovery of thioredoxin-interacting protein inhibitors as an example of a methodical approach to identify and develop an oral beta cell treatment for T1D. It further discusses how the initial molecular findings were translated into novel clinical treatment approaches that promote the patient's own islet health and beta cell function using drug repurposing as well as new drug discovery.