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Cystic Fibrosis Trust

A rare chance!

Study investigates extending the use of existing treatments for people with rare cystic fibrosis (CF) mutations.

A team led by Dr Paola Vergani at University College London is exploring how existing treatments for CF could help people with rare mutations, funded by the Cystic Fibrosis Trust’s Venture Innovation Awards and a grant from Sparks, the children’s medical research charity.

In cells affected by cystic fibrosis, the CFTR channels that move salt and water in and out of the cells do not function. This research aims to identify cells with rare mutations that recover this function when exposed to existing treatments. 

Drugs that address this root cause of cystic fibrosis symptoms can dramatically alleviate symptoms, improving quality of life and extending life expectancy, but people with rare mutations can wait longer for such treatments to become available to them owing to the costs and strict rules involved in clinical trials. Research into the cross-mutation use of drugs that have already been through the clinical trial process is important for widening the application of these existing medications.

Sparks’s CEO Rob Booker, said: “We are delighted to be funding our latest cystic fibrosis project in collaboration with the Trust. We understand the importance of working together to achieve the best possible results to improve life for children, and their families, living with this condition.

“This is a really exciting project that we hope will have a positive benefit for children affected by rare forms of cystic fibrosis.”

Venture Innovation Awards are just one example of the cutting-edge research the Trust is funding to help create a brighter future for everyone with cystic fibrosis.