Researchers in Northern Ireland are celebrating a major breakthrough in the search for new treatments for the lung disease cystic fibrosis.
Trials of a new drug aimed at the so-called "Celtic" gene have shown it can dramatically improve the quality of life of patients with the disease, it was announced today.
Researchers at the Queen’s University, Belfast, and the University of Ulster led an international team testing the drug VX-770.
The drug targets the mutation of the G551D, which is particularly common in Ireland. CF is the most common genetic disease in Ireland and affects some 70,000 globally.
The researchers say the drug may help most patients with the lung disease. It will be submitted for licensing for clinical use in the autumn.
Patients treated with the drug showed improvements in breathing and a reduction in the flare-ups that afflict people with the disease. Researchers say it is too early to say whether it will improve life expectancy.
Researcher Stuart Elborn 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."
And 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.”
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