A message from the ECFS President and the Director of the ECFS Clinical Trials Network 

The recent trial of VX770 in patients with G551D mutations has reported its headline results.  As you will see from the below press release, the clinical impact of VX770 on the relevant outcome measures of lung function, sweat chloride concentration and quality of life have all shown statistically significant and clinically meaningful improvements.  The magnitude of the improvements is the biggest seen with any therapy in Cystic Fibrosis and is significantly greater than many of us anticipated.  The results of  this clinical trial are groundbreaking also because this is the first treatment of the basic defect in Cystic Fibrosis that has shown unequivocal clinical benefit to patients.  The study is proof of principle that mutant CFTR is modifiable in terms of function.  This is good news for patients with the G551D mutation but also extremely encouraging that other strategies that correct and subsequently potentiate other mutations such as F508DEL will be achievable. 

We are really grateful to all the PI’s in Europe and North America and their teams for participating in this study and all the people with Cystic Fibrosis who volunteered to participate. 

The results of this study really do give us great encouragement, and focus our research and care efforts to finding effective treatments that correct the underlying defect in patients with Cystic Fibrosis.

Vertex press release:
http://investors.vrtx.com/releasedetail.cfm?ReleaseID=551869 [1]

Stuart Elborn		Kris De Boeck
ECFS President		ECFS CTN Director