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

161 patients aged 12 and older were randomly divided into two groups. One received the drug every 12 hours and the other received a placebo. All patients had at least one copy of the G551D mutation.

Researchers could tell the drug was working two weeks after people started taking it and the concentration of chloride in their sweat dropped, for some to levels seen in people without the disease. Very salty sweat is a telltale sign of the disease.

Patients also showed improved lung function, as measured by FEV1, or how much air they could blow out in one second.

"It's not surprising you would see an effect in two weeks. By changing the hydration of the mucus, you can clear it out better and open up the airways," Ramsey said. "We saw the improvement across all illness severities ... That was very encouraging. We had been very worried once you had the lung damage or the infections you wouldn't be able to reverse it. That's not saying the lungs would return to normal, but there was more reversibility than we thought there would be."

Patients also experienced an average relative change in their lung function of 17 percent. Relative change means relative to where they started. The absolute change was about a 10 percent improvement.

At 48 weeks, patients on the drug were 55 percent less likely to have experienced an exacerbation, or an infection that left them ill and unable to work or hospitalized.

Patients on the drug also gained an average of 7 pounds, a huge feat for someone with cystic fibrosis, experts said. The weight gain brought people who were nutritionally deficient and underweight closer to a normal body weight, Ramsey said.

The results stayed consistent through 48 weeks, and there were few side effects, according to the study.

Best of all, patients reported feeling better, Beall said. "We had a lot of people call us and say it was incredible how much better they feel."

Cystic fibrosis is a progressive, i
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