A new drug for people with cystic fibrosis will make it easier to clear
the lungs of bacteria, researchers at the University of Ulster and QUB said today.
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 (pictured), 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
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
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.