Cystic fibrosis - developing a new type of inhaled treatment

Over 10,000 people in the UK – 70,000 worldwide – have cystic fibrosis.^1-3 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 cystic fibrosis, many are still losing their lives to the condition, often because of progressive damage to their lungs. Professor Steve Hart, of University College London, is developing a new treatment for cystic fibrosis with the ultimate aim of protecting the lungs, easing children’s breathing difficulties and reducing the number of chest infections they experience.

Action Medical Research and the Cystic Fibrosis Trust are together funding this study

How are children’s lives affected now?

“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,” says Professor Hart.^3,4 “Children with cystic fibrosis can experience persistent coughing, wheezing and recurring chest infections, which sometimes lead to pneumonia. Some people eventually need a lung transplant.”

There is no cure for cystic fibrosis. Treatment with medications and physiotherapy eases symptoms. Unfortunately though, this can be time-consuming and gruelling, sometimes taking as long as four hours per day, which can dramatically decrease children’s quality of life.

Cystic fibrosis is inherited and children’s symptoms all result from faults in a single gene. A medicine that targets this underlying cause of symptoms, rather than just the symptoms themselves, became available recently, but it only works for around four per cent of people with cystic fibrosis.⁵ New treatments that work like this, but help more people, are urgently needed.

How could this research help?

“We are developing a new medicine for cystic fibrosis that targets one of the underlying causes of children’s symptoms,” says Professor Hart.

“The lungs’ surface becomes dehydrated in children with cystic fibrosis,” continues Professor Hart. “We think this dehydration could be a major cause of symptoms – causing the production of thick sticky mucus in the lungs, which allows bacteria to flourish, for example, and damage the lungs. Our new medicine is designed to protect the lungs by stopping them from becoming dehydrated.” In theory, this approach could benefit most children with cystic fibrosis.

The team is testing how best to formulate the new medicine, and whether it is safe and effective, in the laboratory.

“We envisage that children would need to take the new medicine just once per month, by inhaling it,” says Professor Hart. “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.”

References

1. Cystic Fibrosis Trust. About cystic fibrosis. http://www.cysticfibrosis.org.uk/about-cf Website accessed 12 October 2014.

2. Cystic Fibrosis Foundation. About cystic fibrosis. http://www.cff.org/AboutCF/ Website accessed 12 October 2014.

3. UK Cystic Fibrosis Registry. Annual data report 2013. http://www.cysticfibrosis.org.uk/media/598466/annual-data-report-2013-ju... Website accessed 20 October 2014.

4. Cystic Fibrosis Trust. What is cystic fibrosis? http://www.cysticfibrosis.org.uk/about-cf/what-is-cystic-fibrosis Website accessed 12 October 2014.

5. NHS Commissioning Board. Clinical Commissioning Policy: Ivacaftor for Cystic Fibrosis. March 2012. Reference : NHSCB/A01/P/b. http://www.england.nhs.uk/wp-content/uploads/2013/04/a01-p-b.pdf Website accessed 20 October 2014.