Correcting CF with small molecules

So little has happened for cystic fibrosis patients over the past decade that it was notable last week when Vertex Pharmaceuticals Inc. said it has identified a group of small molecules that have shown the potential to help improve functioning of a defective protein responsible for CF pathogenesis. The company has used funding from the Cystic Fibrosis Foundation to discover four compounds - two correctors and two potentiators - that boost the availability of the critical membrane protein and improve ion channel activity in vitro.

Current treatments include drugs that treat the symptoms, such as bacterial infections and the inflammation that causes tissue damage. When combined with proper nutrition and exercise, these strategies have extended the life expectancy of CF patients past 30. However, they do not address the underlying problem. ...