New drug for Cystic Fibrosis

Scientists at Queen's University in Belfast have developed a new drug for Cystic Fibrosis suffers by targeting the so-called Celtic gene, which is common in Ireland.

Ireland has the highest rate of Cystic Fibrosis in the world. The Queen's-led researchers believe the breakthrough will have significant implications for all Cystic Fibrosis sufferers.

The Queen's team were joined by scientists from the University of Ulster and the Belfast Health and Social Care Trust, as well as teams of researchers from Europe, the U.S. and Australia.

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They found significant improvement in lung function, quality of life and reduction in disease fare-ups for those receiving the new treatment. Stuart Elborn from Queen's University, Belfast, co-led the team.

"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," he said.

"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."

Dr. Judy Bradley, from 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."

Dr. Damien Downey, from the Belfast Health and Social Care Trust, said the success of the study illustrated the benefits that come from collaborative work in Northern Ireland.

"Not only will this breakthrough help patients in Ireland and the UK but it has the potential to change the lives for those with Cystic Fibrosis around the world."

The new drug will be submitted for licensing in the fall of this year and is expected to be available to patients by as early as next year.