New drug may treat cystic fibrosis

Scientists in Northern Ireland have helped to develop a new drug that may benefit many people with cystic fibrosis.

The inherited disease affects more than 8,500 children and young adults in the UK, causing their internal organs to become clogged with thick, sticky mucus.

The new drug, VX-770, is designed to treat people with the so-called 'Celtic' gene - a genetic fault that is present in many people with cystic fibrosis.


An international team, co-led by Queen's University Belfast, have shown that people who receive the new drug typically benefit from a significant improvement in their lung function and quality of life.

Stuart Elborn, director of the Centre for Infection and Immunity at Queen's, said: 'The development of this drug is significant because it is the first to show that treating the underlying cause of cystic fibrosis may have profound effects on the disease, even among people who have been living with it for decades.'

People with cystic fibrosis have faulty channels in their lung cells, causing too much salt and not enough water to pass into the cells.

VX-770 works by opening these channels so that the lungs are able to clear bacteria more effectively, preventing infection.

It is hoped that the drug will be available to patients next year.ADNFCR-554-ID-800587922-ADNFCR

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