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Drug Targets Faulty Process That Drives Cystic Fibrosis
Date:11/17/2010

By Jenifer Goodwin
HealthDay Reporter

WEDNESDAY, Nov. 17 (HealthDay News) -- A new drug focused on the underlying cause of cystic fibrosis is showing promise in Phase II clinical trials, new research shows.

If eventually approved by the U.S. Food and Drug Administration, the drug known as VX-770 would mark the first treatment that gets at what goes wrong in the lungs of people with cystic fibrosis, rather than just the symptoms.

Only 4 to 5 percent of cystic fibrosis patients have the particular genetic variant that the drug is being studied to treat, according to the study.

But Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said VX-770 is only the first in a new class of drugs, some of which are already in the pipeline, that may work in a similar way in people with other cystic fibrosis-linked gene variants.

"There has never been such a sense of hope and optimism in the cystic fibrosis community," Beall said. "This is the first time there's been a treatment for the basic defect in cystic fibrosis. If we can treat it early, maybe we won't have all the infections that destroy the lungs and eventually takes people's lives away."

The study appears in the Nov. 18 issue of the New England Journal of Medicine.

Cystic fibrosis is a progressive, inherited disease affecting about 30,000 U.S. children and adults. It is caused by a defect in the CF gene, which produces the CFTR (cystic fibrosis transmembrane conductance regulator) protein, which is important in the transport of salt and fluids in the cells of the lungs and digestive tract.

In healthy cells, when chloride moves out of cells, water follows, keeping the mucus around the cell hydrated.

However, in people with the faulty CFTR protein, the chloride channels don't work properly. Chloride and water in the cells of the lungs stay trapped inside the cell, causing the mucus to become thick, sticky and deh
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