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QUB-led team in drug breakthrough for Cystic Fibrosis

By Claire McNeilly

Cystic Fibrosis sufferers are to benefit from a new, groundbreaking treatment developed by scientists in Northern Ireland, it has emerged.



An international research team led by Queen’s University discovered the drug which will aid those who have the ‘Celtic Gene’, a genetic mutation which is particularly common in Ireland.

But the medication — known as VX-770 — will also help other Cystic Fibrosis sufferers, as it indicates that the basic defect in the illness can be treated.

The study was carried out by scientists at Queen’s University Belfast, the University of Ulster and the Belfast Health and Social Care Trust, as well as teams of researchers in Europe, the USA and Australia.

They found significant improvement in lung function, quality of life and a reduction in disease flare-ups for those receiving the new treatment.

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

Stuart Elborn, Director of the Centre for Infection and Immunity at QUB and co-leader of the study, said the development was significant.

“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 University of Ulster’s Dr Judy Bradley said it was a groundbreaking treatment.

“This drug opens the defective channel in the lung cells of people with Cystic Fibrosis and allows proper lung clearance of bacteria,” she said.

“Correcting the cells with this mutation shows that treatments aimed at the basic mutation can work, leading to improvements in lung function and symptoms.”

Background

Cystic Fibrosis is a common recessive genetic disease which affects the entire body, causing progressive disability and often early death. It is caused by a single faulty gene that controls the movement of salt in the body. Symptoms of CF can include a troublesome cough, repeated chest infections, prolonged diarrhoea and poor weight gain.

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