Cystic Fibrosis breakthrough
Professor Stuart Elborn, Centre for Infection and Immunity at Queen's
An international research team led by Queen’s has developed a ground breaking treatment for Cystic Fibrosis sufferers. The new drug will benefit sufferers who have the ‘Celtic Gene’, a genetic mutation which is particularly common in Ireland.
The study, which was carried out by scientists at Queen’s, the University of Ulster, the Belfast Health and Social Care Trust and teams of researchers in Europe, USA 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 a significant breakthrough not only for those with the ‘Celtic Gene’, known as G551D, but also for all other Cystic Fibrosis sufferers as it indicates that the basic defect in Cystic Fibrosis can be treated. This is the first drug aimed at the basic defect in Cystic Fibrosis to show an effect. It is still too early to determine whether this treatment will improve life expectancy but the improvements in the breathing tests and the reduction in flare-ups would suggest survival will be better.
Stuart Elborn, Centre Director in the Centre for Infection and Immunity at Queen’s 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 Cystic Fibrosis 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.”
The new drug will be submitted for licensing in the Autumn of this year and is expected to be available to patients by as early next year.