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Major breakthrough in cystic fibrosis treatment

Ireland has the largest proportion of cystic fibrosis sufferers in the world and the news has been largely welcomed.

Queen's University in Belfast.
Queen's University in Belfast.
Image: Paul Faith/PA Archive/Press Association Images

Updated 2.20pm

TRIALS OF A new drug to treat cystic fibrosis have found it can significantly improve lung function, reduce disease flare-ups, and improve the overall quality of life.

An international research team, led by scientists at Queen’s University in Belfast, have made the discovery, RTÉ reports.

Now, a licence for the drug – known as VX-770 – will be sought in the autumn by the American pharmaceutical company Vertex.

The chief executive of the Cystic Fibrosis Association of Ireland (CFAI) welcomed the news. In a statement Philip Watt said:

This is exciting news. It is the first time that a drug will impact on Cystic Fibrosis itself. It’s the most significant breakthrough since the so called ‘CF gene’ was discovered in 1989.

Cystic fibrosis is an inherited disease that causes thick, sticky mucus to build up in the lungs and digestive tract. It is most common in children and young adults, and may result in early death.

The researchers say that the drug will particularly benefit sufferers who have the so-called ‘Celtic gene’, a genetic mutation which is particularly common in Ireland, according to the Irish Times.

Ireland has the highest proportion of people suffering from cystic fibrosis in the world with an estimated 1,100 sufferers here because one in 19 Irish people carries the ‘Celtic’ gene.

There have previously been concerns about the number of beds available to cystic fibrosis sufferers in Ireland.

The drug is the first to treat the basic defect caused by the gene mutation in patients, according to the the researchers.

It opens the defective channel in the lung cells of sufferers allowing proper clearance of bacteria on the lungs. All going to plan, the drug could be available to patients by next year with sufferers taking two tablets day, according to BBC News.

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.

About the author:

Hugh O'Connell

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