Two little boys in my extended family have Cystic Fibrosis, the debilitating, life-shortening lung disease, so every time there’s news on potential new drugs or treatments, we get a flurry of hopeful email.
This week, Vertex Phamaceuticals, the Cambridge-based biotech, announced “promising results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ and VX-809, a CF drug in development. The results showed a significant improvement in lung function in people with two copies of the most common CF mutation who received the two drugs in combination.”
My relatives, a pair of 11-year-old boys, both have two copies of this mutation, the Delta 508 gene. Needless to say, their mother is psyched.
In a letter to supporters, Robert Beall, president and CEO of the Cystic Fibrosis Foundation explains the details and calls the findings “a significant step.” Continue reading