Cystic Fibrosis Drug Treats 'Celtic Gene'

Cystic fibrosis sufferers will soon have a new drug which takes aim at the 'Celtic gene''

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) New research has shown that the basic defect in those with cystic fibrosis (CF) can be treated with a new drug which can lead to better lung function and alleviation of symptoms.

This new study, carried out in the US, Great Britain, Europe and Australia found significant improvement in lung function, a reduction in disease flare ups for those receiving the new treatment and enhanced quality of life.

The drug (VX-770) is an exciting breakthrough not only for those with the 'Celtic Gene', known as G551D (a common genetic mutation in Ireland), but also for all other cystic fibrosis sufferers as it indicates that the basic defect in cystic fibrosis can be treated.

"Ask your doctor when VX-770 will be available."

Professor Stuart Elborn, Centre Director in the Centre for Infection and Immunity at Queen's University and co-leader of the study said that this is the first time the underlying cause of 'Celtic gene' CF has been targeted. It could prove to have enormous impact on the symptoms of the disease even among patients who have suffered from CF for decades.

He notes that the reductions in sweat chloride when patients are taking this drug support the idea that VX-770 improves protein function which addresses the fundamental defect that leads to CF.

Judy Bradley, PhD from the University of Ulster explained that the drug works by opening the defective channel in the lung cells of people with CF, and this permits proper lung clearance of bacteria. 

Damien Downey, PhD from the Belfast Health and Social Care Trust said that the success of this study shows the benefits that come from collaborative work involving Northern Ireland. 

VX-770 has been submitted for licensing approval and is expected to be available to patients during 2012.

Reviewed by: 
Review Date: 
June 21, 2011
Last Updated:
June 22, 2011