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

s, dean of Case Western Reserve University School of Medicine, in Cleveland, said the drug holds great promise for cystic fibrosis sufferers.

"The exciting thing about this is a drug that interacts directly with the defective protein and changes its function," Davis said. "Admittedly this protein is only found in about 5 percent of the patients with CF but it does appear to restore normal function."

Though more testing is needed, it might be possible to give the drug to babies to head off the disease before it begins to damage the lungs, she added.

Millions of Americans carry a defective CF gene, but do not have any symptoms. To be symptomatic, a person with CF must inherit two defective CF genes -- one from each parent. About 30,000 people have CF, according to the Cystic Fibrosis Foundation.

More information

The Cystic Fibrosis Foundation has more on the disease.

SOURCES: Bonnie Ramsey, M.D., director, Center for Clinical and Translational Research, Seattle Children's Hospital, and professor, University of Washington School of Medicine; Pamela Davis, M.D., Ph.D, dean, Case Western Reserve University School of Medicine, Cleveland; Robert Beall, Ph.D, president and CEO, Cystic Fibrosis Foundation; Dawn Kalmar, director, product communications, Vertex Pharmaceuticals; Nov. 3, 2011, New England Journal of Medicine


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