Cystic Fibrosis Related Diabetes (CFRD): Advancing Pathophysiologic Understanding and Clinical Treatment

May 2022 Council

Lead Division/Office

DEM

Point(s) of Contact

Tom Eggerman, M.D., Ph.D.

Executive Summary

Cystic Fibrosis (CF) is one of the most common, life-limiting genetic diseases. Cystic Fibrosis Related Diabetes (CFRD) is a poorly understood and clinically significant complication that affects about half of adult CF patients.  The CF Foundation and NIDDK sponsored a CFRD Scientific Workshop June 23–25, 2021 with the goal of sharing basic and clinical research data regarding CFRD disease mechanisms and treatment and to identify key research questions and knowledge gaps that can be used to inform research priorities. A wide variety of topics were covered including mechanisms of disease, sites where cystic fibrosis transmembrane conductance regulator gene (CFTR) functions and the interaction between the exocrine and endocrine pancreas, prevention and treatment of CFRD and the effect of CFTR modulator therapy on glucose and insulin homeostasis. Some of the major CFRD research questions/gaps raised included biologic basis, development of better model systems, ascertaining genetic risk factors, identifying biomarkers and clinical markers and optimizing nutritional aspects. In addition, determining the effects of highly effective modulator treatment (HEMT), utilization of novel treatment approaches and how best to optimize healthcare delivery and patient education were additional research questions. The goal of this initiative is to stimulate both clinical and basic CFRD research to address the key research questions and knowledge gaps identified in this workshop.