The outcomes for people with cystic fibrosis (CF) have greatly improved thanks to the development of new drugs (called CFTR modulators) to treat the condition. Before these treatments were introduced we could only treat the symptoms of cystic fibrosis, but now we can treat the genetic causes, prolonging people's lives and transforming their quality of life.
A team at Imperial College London played a key role in introducing these drugs. Imperial’s Professor Jane Davies led a clinical trial programme evaluating potential treatments to target the genetic causes of cystic fibrosis. This led to the introduction of various drugs, including one called ivacaftor. Professor Davies led the UK arms of clinical trials of ivacaftor, which found the drug to be highly effective for patients with particular genetic mutations. She has since been involved in trials of the newer drugs, which are suitable for the majority of people with CF.
Once proven to be safe and effective in adults, she also helped design the clinical trials of these treatments in children.
CFTR modulators restore the faulty processes that cause cystic fibrosis symptoms, prolonging people’s lives and reducing their need for lung transplants. The drugs can also return patients’ lung function, as well as slowing the rate of lung function loss, reducing lung infections, improving patients’ nutrition and reducing the amount of time people with cystic fibrosis spend in hospital.
