Drug boost in cystic fibrosis study
A new drug for people with cystic fibrosis will make it easier to clear the lungs of bacteria, researchers in Northern Ireland have said.
The treatment targets a basic defect caused by a genetic mutation known as the Celtic gene because it is so common in the region.
Dr Judy Bradley, of the University of Ulster, said: "This is a ground-breaking treatment because it treats the basic defect caused by the gene mutation in patients.
"Correcting the cells with this mutation shows that treatments aimed at the basic mutation can work, leading to improvements in lung function and symptoms."
The study, carried out by Queen's University Belfast, 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 the condition can be treated.
This is the first drug aimed at the basic defect to show an effect. It is still too early to determine whether the treatment will improve life expectancy but the improvements in the breathing tests and reduction in flare-ups would suggest survival rates will be better.
Stuart Elborn, director of 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 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 cystic fibrosis."
The drug will be submitted for licensing in the autumn of this year and is expected to be available to patients next year.