Study into new treatment for Cystic Fibrosis | Action Medical Research

Study into new treatment for Cystic Fibrosis

19 November 2014

Cystic fibrosis: developing a new type of inhaled therapy

The children’s charity Action Medical Research and the Cystic Fibrosis Trust have joined forces to fund a laboratory study to develop a new, once-monthly, inhaled treatment for children with cystic fibrosis (CF).

The total amount awarded for the new study, being led by Professor Steve Hart, of University College London, is £149,062. The Cystic Fibrosis Trust is contributing one third.

Today in the UK, over 10,000 people have the inherited condition.Many are children and young people. Symptoms include a persistent cough, shortness of breath and recurrent chest infections. Although life expectancy has been increasing for people with CF, many are still losing their lives too young.

There is no cure. A medicine that targets the underlying cause of symptoms became available recently, but it only works for around four per cent of people with cystic fibrosis.

The ultimate aim of the new inhaled treatment is to protect the lungs from dehydration, believed to be a major cause of the build-up of sticky mucus which allows harmful bacteria to flourish. This is hoped to benefit most children with CF, easing their breathing difficulties and reducing the number of chest infections they get.

Professor Steve Hart says: “Each week in the UK, five people – mostly babies and young children – are diagnosed with cystic fibrosis, while two lives are lost to the condition.”

“We hope the medicine will one day help children to breathe more easily and stop them from getting so many bacterial infections – something which could dramatically change children’s lives for the better.” 

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Cystic Fibrosis Trust. About cystic fibrosis. Website accessed 12 October 2014.
UK Cystic Fibrosis Registry. Annual data report 2013. Website accessed 20 October 2014.
NHS Commissioning Board. Clinical Commissioning Policy: Ivacaftor for Cystic Fibrosis. March 2012. Reference : NHSCB/A01/P/b. Website accessed 20 October 2014.


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Action Medical Research is a UK-wide charity saving and changing children’s lives through medical research.

We want to make a difference in:

  • tackling premature birth and treating sick and vulnerable babies
  • helping children affected by disability, disabling conditions and infections
  • targeting rare diseases that together severely affect many forgotten children.

Just one breakthrough, however small, can mean the world.

Charity reg.nos 208701 and SC039284.


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