'Just What We Dreamed.' New Vertex Drugs Show Dramatic Benefit Against Cystic Fibrosis


Three different three-drug regimens all provided dramatic results against cystic fibrosis, a fatal disease of the lungs and digestive system that afflicts 75,000 people worldwide. The results are a step forward both for the drugs’ maker, Vertex Pharmaceuticals of Boston, Mass., and a long-awaited victory in the quest to use genes to develop new drugs.

“This is just what we dreamed would someday happen,” says Francis Collins, the director of the National Institutes of Health, who led the discovery of the cystic fibrosis gene two decades ago. “This is a remarkably happy moment to see how far this has come and how incredibly encouraging this news is.”