Alan S. Verkman
Director, University of California San Francisco Novel Small-Molecule Therapies for Cystic Fibrosis
Alan S. Verkman, MD, PhD
specializes in drug discovery and lung disease mechanisms in Cystic Fibrosis. After identification of the Cystic Fibrosis (CF) gene in 1989, there has been dramatic progress in Cystic Fibrosis research.
The Cystic Fibrosis gene product is a large protein (Cystic Fibrosis Transmembrane conductance Regulator, CFTR) that functions as a cAMP-stimulated chloride channel and possibly as a regulator of multiple
intracellular processes. Active work in the Verkman laboratory is directed at the identification of cellular abnormalities in CF that could account for the lung pathology, including studies of the airway
surface fluid layer and submucosal glands.
A major focus of research is discovery of new drugs for CFTR inhibition (as antidiarrheals and to create CF animal models) and activation of mutant CFTRs (including ΔF508-CFTR) that cause CF. Resources
for drug discovery include high-throughput screening, a large collection of small drug-like molecules, rodent pharmacology and efficacy testing, and synthetic/medicinal chemistry.