Novel small-molecule therapies for Cystic Fibrosis

Investigator: Alan Verkman, MD, PhD
Sponsor: NIH National Institute of Diabetes and Digestive and Kidney Diseases

Location(s): United States


The focus of the Cystic Fibrosis Core Center is to develop novel therapies for cystic fibrosis and to enhance many research projects examining the mechanisms of cystic fibrosis lung disease.

This is a proposal to continue our Cystic Fibrosis (CF) Research and Translational Core Center at the University of California, San Francisco and collaborating institutions. The focus of our Core Center remains the discovery and evaluation of novel small-molecule therapies for CF. The original proposal followed from 5 years of work establishing a unique academic drug discovery program in the laboratories of Dr. Verkman and collaborators to identify and characterize small-molecule modulators of CFTR activity. The proposed Core Center will fund five Cores to support the activities of 2 Pilot Projects and 28 CF-related projects. The Core directors are experienced CF investigators with recognized expertise in their areas of investigation and a history of productive collaboration. The Cores include: High-Throughput Screening, Clinical Resources, Cell Models, Synthetic Chemistry, and Cell & Tissue Bioassays. Projects to utilize the cores include the discovery of modulators of epithelial ion transporters, epithelial cell mucin secretion, and Pseudomonas biofilm formation. Other projects to utilize the cores include research on mechanisms of lung disease in CF. The general goal of the research to be enhanced by the Core Center is to develop new small-molecule therapies for CF that can be translated into the clinic.