Scientists develop ground breaking drug to treat cystic fibrosis


Targets the so-called Celtic gene

An international research team co-led by scientists at Queen’s University Belfast has developed a new treatment for Cystic Fibrosis (CF) sufferers. The drug specifically targets the so-called Celtic gene, which is common in Ireland.

Dr Judy Bradley, from the University of Ulster, said: ‘This drug opens the defective channel in the lung cells of people with CF and allows proper lung clearance of bacteria. This is a ground-breaking treatment because it treats the basic defect caused by the gene mutation in patients.’

The study, carried out by scientists at Queen’s University Belfast, the University of Ulster, the Belfast Health and Social Care Trust and teams of researchers in Europe, US and Australia found significant improvement in lung function, quality of life and a reduction in disease flare-ups for those receiving the new treatment.

The drug (VX-770) is said to be a significant breakthrough, not only for those with the Celtic gene, known as G551D, but also for other CF sufferers as it indicates that the basic defect in the disease can be treated.

CF affects around 70,000 people worldwide. More than 9,000 people in the UK have the disease and more than 1,100 in Ireland. Ireland has the highest proportion of CF people in the world.

The researchers say it is still too early to determine whether this treatment will improve life expectancy but the results of the breathing tests and the reduction in flare-ups suggest that survival will be better.

Stuart Elborn, director in the Centre for Infection and Immunity at Queen’s University and co-leader of the study, said: ‘The development of this drug is significant because it is the first to show that treating the underlying cause of CF may have profound effects on the disease, even among people who have been living with it for decades.

‘The remarkable reductions in sweat chloride observed in this study support the idea that VX-770 improves protein function thereby addressing the fundamental defect that leads to CF.’

As a result of this work researchers from Queen’s University, University of Ulster and clinicians from Belfast Health and Social Care Trust have been selected to join the European Cystic Fibrosis Society Clinical Trials Network.

This means Northern Ireland scientists will be collaborating with their European counterparts to work towards improved treatments for the disease on a global level.

The new drug will be submitted for licensing in autumn and is expected to be available to patients next year.