VX-770 Treatment Could Hold Promise in Cystic Fibrosis

Trial assesses safety, possible effects of CFTR potentiator in adults with cystic fibrosis

WEDNESDAY, Nov. 17 (HealthDay News) -- Among adults with cystic fibrosis, VX-770 -- a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator -- may be linked to improvements in CFTR and lung function, according to research published in the Nov. 18 issue of the New England Journal of Medicine.

Frank J. Accurso, M.D., of the University of Colorado Denver and Children's Hospital in Aurora, and colleagues analyzed data from 39 adults with cystic fibrosis and one or more G551D-CFTR alleles. In the first part of the study, participants were randomized to receive oral VX-770 at a variety of doses or placebo for 14 days, and in the second part, participants were randomized to receive the drug, mostly at higher doses, or placebo for 28 days.

The researchers found that the study groups had similar frequency of adverse events. The treatment was associated with within-subject improvements in lung function and CFTR ion-channel function in sweat glands and the nasal epithelium.

"Acquiring knowledge of the pathophysiology of cystic fibrosis and developing new assays with the use of that knowledge should be priorities. One hope is that studies of the use of agents such as VX-770 will advance these goals. The reaching of this milestone along the pathway of discovery leaves me optimistic for people who have cystic fibrosis," writes the author of an accompanying editorial.

The study was supported in part by Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics. A number of the authors disclosed financial relationships with a variety of medical device and pharmaceutical companies, including Vertex.

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