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Cystic Fibrosis breakthrough!

A GROUND-breaking treatment for people with Cystic Fibrosis has been developed by an international research team led by Queen’s University Belfast.

The new drug will benefit those who have the ‘Celtic Gene’, a genetic mutation which is particularly common in Ireland.

The study 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 Cystic Fibrosis can be treated.

This is the first drug aimed at the basic defect in Cystic Fibrosis to show an effect.

It is still too early to determine whether this treatment will improve life expectancy but the improvements in the breathing tests and the reduction in flare-ups would suggest survival will be better.

Stuart Elborn, 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 CF.”

Dr Judy Bradley, from the University of Ulster, said: “This drug opens the defective channel in the lung cells of people with Cystic Fibrosis and allows proper lung clearance of bacteria. 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 this study illustrates the benefits that come from collaborative work here 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 autumn and is expected to be available to patients by as early next year.

 

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