The Cystic Fibrosis Foundation (CFF) has announced positive results from ongoing Phase 2a trials for a new oral drug in patients who carry the G551D mutation of cystic fibrosis (CF). The drug, VX-770, targets a basic defect in CF, and is the first potential therapy ever to improve the abnormal sweat chloride levels in a person with CF.
Cystic fibrosis is caused by a missing or malfunctioning protein, which results in an imbalance of water and salt. This causes mucous to build up, causing infection and inflammation in the lungs and other organs.
20 CF patients were included in the trial, which lasted 14 days. Patients who took the drug for the 14 days showed great improvement in several key indicators of CF, including lung function and sweat chloride levels. Sweat chloride levels are often the key to a diagnosis of CF.
VX-770 is being developed by Vertex Pharmaceuticals Inc as a part of collaboration between the CFF and Vertex, which began in 1998. The CFF has since invested $79 million into the collaboration, for use of new technology known as high throughput screening, which is utilized to find compounds to attack the core defect in CF.
Part Two of the study is expected to take place in later this year.