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news.Researchers at the Queen's University Belfast, the University of Ulster, the Belfast Health and Social Care Trust and teams of researchers in Europe, USA and Australia have developed a drug (VX-770) targeting cystic fibrosis.
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The study demonstrated that the drug improved lung function, quality of life and showed reduction in disease flare ups in patients who have the ‘Celtic Gene,’ (G551D).
The drug has also reduced sweat chloride and improved 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.
Queen’s University Centre for Infection and Immunity centre director 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.