12/03/2013

Cystic Fibrosis Drug To Help 'Celtic Gene'

A new drug to benefit sufferers of Cystic Fibrosis who possess the 'Celtic Gene' has been approved.

The drug Ivacaftor, or Kalydeco, will be made available to patients who have the G551D genetic mutation, which is known as the 'Celtic Gene' due to its prominence in Ireland.

Health Minister Edwin Poots said: "The drug Ivacaftor is a significant breakthrough, not only for those with the 'Celtic Gene', but also for all other CF sufferers, as it indicates that the basic defect in CF can be treated. This is the first drug aimed at the basic defect in CF to show an effect."

Cystic Fibrosis is one of the UK's most common life-threatening inherited diseases.

It affects over 9,000 people in the UK, of whom about 500 live in Northern Ireland.

The disease affects the internal organs, particularly the lungs and digestive system, by clogging them with thick sticky mucus.

Sufferers find it hard to breathe and digest food, and currently only half of those living with Cystic Fibrosis are likely to live past their late 30s.

About 7% of the 500 sufferers in Northern Ireland carry the G551D mutation.

"The study found significant improvement in lung function, quality of life and a reduction in disease flare-up for those receiving the new drug treatment," Minister Poots said.

"This is an extremely positive decision which means that eligible patients here will, in common with their counterparts elsewhere in the UK, be able to gain access to Ivacaftor."

Professor Stuart Elborn, Director of Queen’s University Belfast’s Centre for Infection and Immunity, led the original trial for the drug.

"We led an international team on this study which involved colleagues at Queen’s, the University of Ulster, the Belfast Health and Social Care Trust and researchers in Europe, USA and Australia, but what the success of this study also illustrates are the benefits that come from collaborative work here in Northern Ireland and the impact of Queen’s research," he said.

"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."

(IT)

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