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Belfast scientist makes major cystic fibrosis breakthrough

A doctor from Queen's University in Belfast has made a major breakthrough in the treatment for cy...
Newstalk
Newstalk

12.28 25 Jun 2014


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Belfast scientist makes major...

Belfast scientist makes major cystic fibrosis breakthrough

Newstalk
Newstalk

12.28 25 Jun 2014


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A doctor from Queen's University in Belfast has made a major breakthrough in the treatment for cystic fibrosis.

Professor Stuart Elborn, along with colleagues from the United States and Australia, has led pivotal studies of a new treatment for people with the disease.

The combination therapy, developed by Vertex, is said to improve lung function and reduces hospitalisations for patients with the most common type of cystic fibrosis.

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Two studies of the drugs 'Ivacaftor' and 'lumacaftor' built on previous studies. Ivacaftor is the first drug to treat the underlying causes of cystic fibrosis rather than just its symptoms.

It is currently approved for patients with the 'Celtic gene' mutation carried by about 4% of all patients and 10-15% of patients in Ireland.

This recent trial found that a combination of Ivacaftor with lumicaftor improved lung function between 2.6 and 4%.

Cystic fibrosis is a fatal lung disease affecting 75,000 children and adults world-wide, and is caused by inherited genetic mutations that vary among different patient groups.

Professor Stuart Elborn said "This is a very significant breakthrough for people with cystic fibrosis. While we had previously found an effective treatment for those with the 'Celtic gene' this new combination treatment has the potential to help roughly half of those with cystic fibrosis, those who have two copies the F508DEL mutation".

12 patients from the Northern Ireland Adult and Paediatric Cystic Fibrosis Centres participated in the study.


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