A Novel Treatment for Cystic Fibrosis

A new treatment directly targets the defective cystic fibrosis transmembrane conductance regulator.

Current treatments for cystic fibrosis (CF) focus on alleviation of symptoms. Now, researchers are attempting to treat the actual cause of CF — a defect in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. In an industry-supported, double-blind, randomized clinical trial, 39 adults with CF received oral VX-770 (a CFTR potentiator that increases the activity of CFTR protein) or placebo every 12 hours for 28 days. All patients had a specific CFTR mutation called G551D.

After 28 days, CFTR activity, as measured by nasal potential difference (an electrophysiologic assay of CFTR channels), increased significantly in the VX-770 group compared with the placebo group, and the level of sweat chloride decreased. Lung functio…

Reviewing Author

Howard Bauchner, MD

Disclosures

Grant / research supportHuman Resources and Services Administration; NIH; NIH-NHLBI; USDA; GlaxoSmithKline

Editorial boardsArchives of Disease in Childhood (Editor-in-Chief)

Howard Bauchner, MD

Disclosures

Grant / research supportHuman Resources and Services Administration; NIH; NIH-NHLBI; USDA; GlaxoSmithKline

Editorial boardsArchives of Disease in Childhood (Editor-in-Chief)