New Cystic Fibrosis Treatment Offers Hope

20 June 2011

CFAI welcomes the announcement today that there has been a significant breakthrough in the treatment of Cystic Fibrosis but also cautions that we are still a long way from a cure. 

The new drug - VX770 is in the final stages of development and should become available in Ireland and other countries by mid 2012.Philip Watt, CEO of the CFAI said 'This is exciting news. It is the first time that a drug will impact on Cystic Fibrosis itself. It’s the most significant breakthrough since the so called ‘CF gene’ was discovered in 1989'.

However he continued 'It is important to caution that it is still not a cure and that at present it will impact on only about 10% of the Irish CF population. However the new drug points the way for future research and offers improved quality of life to those with the Celtic gene and hope to others with CF with another gene mutation.'

The new drug VX770 developed by the American company Vertex working with CF  centres around the world, including Queens University Belfast, St Vincent’s University Hospital, Beaumont Hospital and Cork University Hospital.The new drug will impact on the G551D gene mutation. Sometimes called the ‘Celtic Gene’ as it originated in Celtic countries, but is now present in the US and other countries, mainly because of historic emigration. The mutated gene affects about 4% of the world’s CF population, but affects about 10% of the CF population in Ireland and about 20% of the CF population in the greater Cork region.

 The drug tries to compensate for the genetic defect that causes think mucus to build up in the lungs and other problems facing people with CF. Among other outcomes, the new drug has been shown to:

•    Help ease breathing (lung function improvements of the order of 10%)

•    Ease exacerbations

•    Improve weight gain (poor weight gain is a problem for many with CF)

In short, the clinical trial has been shown to improve quality of life of those with the Celtic gene mutation and this is really welcome news. We are still at relatively early days and we will only know the full impact in time.