Campaigners hail CF breakthrough

A new drug which targets the genetic mutation associated with cystic fibrosis has been heralded as a "groundbreaking" development by campaigners.

The treatment is the first of its kind to work directly on the genetic defect which causes thick mucus to build up in the lungs and digestive tract of CF patients.

Clinical trials of the “VX-770” drug were found to significantly improve lung function and quality of life for sufferers.

The drug, which was developed by an international team of researchers led by scientists from Queen’s University Belfast, and US pharmaceutical firm Vertex, is expected to be available in Ireland by the middle of next year.

Cystic fibrosis campaigner Orla Tinsley today described the discovery as a “groundbreaking step” in the treatment of CF.

“We haven’t seen anything like this before because it gets right to the core of where the disease originates,” Ms Tinsley said.

“For scientists to be able to target those cells which cause the inflammation and, essentially, stall the progression of the illness brings us closer to finding a more successful treatment for the disease. It’s early days but from what I understand it significantly decreases the level of inflammation which should, in turn, lessen the amount of infections and disease flare-ups.

“This should decrease the requirement for intravenous treatment and slow down the rate of lung damage perhaps prolonging life,” she said.

The Cystic Fibrosis Association of Ireland (CFAI) also welcomed the development but cautioned “we are still a long way from finding a cure” for the disease.

The association’s chief executive Philip Watt said it was "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," he said.

“It is important to caution that it is still not a cure and that at present it will impact on only about 10 per cent 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,” he said.

Cystic fibrosis is the most common life-threatening inherited disease in Ireland, affecting around 1,100 people.

The mutated “Celtic gene” affects about 4 per cent of the world’s CF population, but up to 10 per cent of the CF population in Ireland and about 20 per cent of the CF population in the greater Cork region.

While clinical trials show the drug reduces disease symptoms, it is too soon to say if it will help extend life expectancy.