First drug to tackle cause of cystic fibrosis ‘on sale as soon as next year’

The first drug to tackle the root cause of cystic fibrosis, rather than just the symptoms, could go on sale next year.

In trials, the twice-a-day pill dramatically improved the lung health of men and women with the debilitating condition.

They also put on weight and needed fewer antibiotics.

Hope: In trials, the twice-a-day pill dramatically improved the lung health of men and women with the debilitating condition

If trials on children are as successful, manufacturers Vertex Pharmaceuticals could apply later this year for permission to market the drug in Europe. Clearance is likely in 2012.

Britain's 8,000 adults and children with cystic fibrosis include Gordon Brown's four and a half-year-old son Fraser.

Cystic fibrosis is the UK’s most common life-threatening inherited condition and occurs when a genetic flaw produces a defective version of a protein key to the health of the lungs and digestive system.

Thick, sticky mucus clogs these organs, leading to recurrent chest infections and poor growth. Other symptoms include diabetes and infertility.

Although treatments have improved greatly in recent years, average life expectancy is under 40 and there is no cure.

In the trial, 161 people with cystic fibrosis were given either the new drug, which is known only as VX-770, or a dummy drug two times a day for a year.

At the outset, most of those taking part had just 60 per cent of the lung function of a healthy person - a figure that improved by almost 20 per cent by giving VX-770.

Life-threatening: Britain's 8,000 adults and children with cystic fibrosis include Gordon Brown's four and a half-year-old son Fraser

No other drug has produced such dramatic improvements, this week’s New Scientist reports, probably because they have tackled symptoms rather than the underlying cause, a defective protein in lung cells.

Patients also put on an average of half a stone in weight, suggesting the disease’s effects on digestion were eased, and were half as likely to need antibiotics for flare-ups.

Peter Mueller, Vertex’s chief scientific officer, said: ‘Treating the underlying cause of cystic fibrosis with VX-770 led to clinical improvements that were far beyond our expectations, providing support for an entirely new approach to the treatment of this disease.’



The Cystic Fibrosis Trust described the results as ‘very big news’ but cautioned that VX-770 would only be suitable for about 5 per cent of sufferers.

But a second drug, that is at an earlier stage in development, offers hope to another 75 per cent of patients.

Vertex is already testing a combination of the two drugs on patients and the first results are expected within months.





