Cystic Fibrosis

Cystic Fibrosis, In one of those rare cases of good science translating directly into good medicine, Vertex Pharmaceuticals yesterday unveiled positive results from a Phase III trial of VX-770, a small molecule that treats the underlying defect of cystic fibrosis.
Vertex’s drug is generating excitement because it addresses the underlying cause of the disease. Think of CFTR as a gate that allows chloride ions out of the cell. VX-770 is being tested in people with a mutation akin to the former, and works to increase the flow of chloride ions through the gate, thereby restoring the right level of hydration in the lungs.

Now there are some promising results from a study in 161 people with a specific genetic mutation that affects about four percent of cystic fibrosis patients.

That much improvement is "a big deal for CF patients," Robert Beall, president of the Cystic Fibrosis Foundation told Shots.

The gene responsible for cystic fibrosis was discovered in 1989. Vertex Pharmaceuticals announced Wednesday morning that the drug, VX-770, improved lung function in people with cystic fibrosis in a late-stage clinical trial. The drug also reduced the frequency of disease exacerbations that required treatment with antibiotics.

source

http://prescriptions.blogs.nytimes.com/2011/02/23/vertex-says-cystic-fibrosis-drug-helped-patients-breathe-easier/