An international team of scientists has developed a new drug for cystic fibrosis sufferers.
VX-770 targets people with the ‘celtic gene’, a genetic mutation particularly common in patients with the condition.
The study, which was led by Queen’s University in Belfast and involved researchers in Europe, the US and Australia, found a significant improvement in lung function and a reduction in disease flare-ups in those receiving the treatment.
It is too early to say whether the drug will improve life expectancy.
VX-770 works by opening the defective channel in lungs cells of people with cystic fibrosis. It is the first treatment aimed at this basic defect to show an effect.
Stuart Elborn, director of the Centre for Infection and Immunity at Queen’s University, 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 fibroses.”
The drug will be submitted for licensing later this year.