Drug targets defective protein that causes cystic fibrosis

LONDON - A new small study has shown that an investigational drug targeting a defective protein that causes cystic fibrosis (CF) can improve lung function.

The investigational drug, VX-770, appeared to improve function of what is known as CFTR-the faulty protein responsible for CF and is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis.

Patients who took VX-770 for 28 days showed improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels.

“Patients with CF have a defective protein in chloride channels in lung cells that, in effect, causes a door to shut too tightly, ultimately leading to severe infections in the lung” said Steven M. Rowe, assistant professor of medicine at the University of Alabama at Birmingham and lead investigator at the UAB site.

“The data suggest that the drug seems to improve the function of the protein, so that the door opens and closes more properly,” he added.

Investigators studied 39 patients with CF in the trial who had a specific mutation in the gene known to cause CF with the primary objective of demonstrating the safety and tolerability of VX-770. All patients in the trial completed use of the study drug, and adverse events were similar to placebo.

VX-770 is currently in Phase 3 clinical trials for cystic fibrosis, and, pending data from these studies, Vertex anticipates submitting a new drug application to the FDA in the second half of 2011.

CF is a fatal genetic disease that affects 70,000 people worldwide. It is caused by a genetic mutation that causes life-threatening lung infections and premature death.

Findings are published in the New England Journal of Medicine. (ANI)