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New drug treats cystic fibrosis 'at gene stage'
Mark Prigg, Science Correspondent9 Sep 2008
A radical new drug treatment for cystic fibrosis is being developed, scientists were told today.
The disease, which affects around 8,000 Britons, is the UK's most common life-threatening inherited disease.
However, existing treatments only alleviate symptoms, for example, physiotherapy to clear the air passages, antibiotics for lung infections and enzymes to aid the digestion of food.
The new drug, VX-770, promises to be able to treat the genetic defect which causes the disease.
Dr David Sheppard, of the University of Bristol, presented his work at the British Association Festival of Science in Liverpool. He said early results with VX-770 suggest it has "the potential to improve greatly the quality of life of CF patients".
Cystic fibrosis causes ducts and tubes in the body to become blocked by thick, sticky mucus. This mucus affects the lungs, pancreas, the intestines, liver and reproductive organs.
US tests of the new drug therapy saw lung function improve by 10 per cent. "Early results are very encouraging," said the researchers.
Planning is currently under way for European trials. VX-770 is being developed by Vertex Pharmaceuticals.
More than two million people in the UK carry the faulty gene that causes cystic fibrosis - around one in 25 of the population. If two carriers have a child, the baby has a one in four chance of having cystic fibrosis.
The average life expectancy is around 31, although improvements in treatments mean a baby born nowadays can expect to live for longer.
Gordon Brown's son Fraser was diagnosed with cystic fibrosis months after his birth in 2006.
Reader views (1)
I think it is important to point out that this drug is targeted at those with the G551D mutation which is about 5% of CF sufferers in the UK. The majority of those with CF have the mutation deltaF508. The article does not make this clear.
- Caroline Egan, London, 10/09/2008 14:24
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