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New Drug Targets Underlying Cause of Cystic Fibrosis
Date:11/3/2011

By Jenifer Goodwin
HealthDay Reporter

WEDNESDAY, Nov. 2 (HealthDay News) -- A new drug that targets a faulty protein that causes cystic fibrosis led to improved lung function and fewer symptoms in people with the lung disease, researchers report.

The drug -- ivacaftor -- is the first to halt the underlying processes that cause the inherited disease, which causes thick, sticky mucus to build up in the lungs and the pancreas and can lead to life-threatening infections, experts said.

"It has a huge significance for the whole cystic fibrosis community," said study author Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research at Seattle Children's Hospital and a professor at the University of Washington School of Medicine. "It's the first time we have developed a therapy directed at the abnormal proteins and showing that it can be corrected."

Only 4 percent to 5 percent of cystic fibrosis patients have the particular genetic variant that the drug is being studied to treat, but for them, the results could mean a significant improvement in their health, said Robert Beall, president and CEO of the Cystic Fibrosis Foundation.

"We're talking about adding decades to these people's lives, that's how profound this drug is," Beall said.

But Beall and other experts say the drug may end up helping people with other cystic fibrosis genetic variants, including the most common one, D508, one copy of which is present in more than 90 percent of people with cystic fibrosis.

Though ivacaftor (previously known as VX-770) on its own didn't work all that well in these patients, a trial looking at using ivacaftor in conjunction with another drug is currently under way. Results of that trial are expected in the fall of 2012, said Beall, whose organization has provided funding for VX-770 research.

In the study reported in the Nov. 3 issue of the New England Journal of Medicine,
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