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Cystic fibrosis - developing a new type of inhaled treatment

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What did the project achieve?

“We’re delighted with the results from our laboratory research, which bring us one step closer to our goal of getting our new medicine for children with cystic fibrosis into the clinic,” says Professor Steve Hart of the UCL Great Ormond Street Institute of Child Health.

Around 10,400 people in the UK – including over 4,000 children – are living with an inherited lung condition called cystic fibrosis.1

The symptoms, which are caused by a build-up of sticky mucus in the lungs and other organs, can impact heavily on a child’s quality of life. Sadly, in the UK, two people with cystic fibrosis will lose their lives every week, with lung damage caused by regular chest infections a big factor.2

The team aims to help protect the lungs of children with cystic fibrosis by developing a new inhaled treatment.

“We think a lot of the damage is caused because the surface of their lungs becomes dehydrated – our new medicine is designed to combat this,” says Professor Hart. “By keeping the surfaces moist, we hope this will reduce the thickness of the mucus – leading to fewer chest infections, improved breathing and reduced coughing.”

But a big challenge is getting their new medicine to penetrate through the sticky mucus and into the child’s lung cells where it can get to work.

“During this study, we have developed a formulation that can deliver the medicine to where it’s needed,” says Professor Hart. “And we are now planning more laboratory research to find out if it can help reduce the thickness of the mucus.”

“We’re excited about the progress we’re making with this new medicine, which we hope will one day dramatically improve the lives of children with cystic fibrosis,” says Professor Hart.


Cystic Fibrosis Trust. UK Cystic Fibrosis Registry. 2016 Annual Data Report. Published August 2017. https://www.cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/reporting-and-resources [Website accessed 28 November 2017.

British Lung Foundation. Cystic Fibrosis statistics: https://statistics.blf.org.uk/cystic-fibrosis [Website accessed 28 November 2017]

This research was completed on

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.”



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.



Project Leader Professor S L Hart PhD
Project Team Professor C L O'Callaghan FRCPCH FRCP PhD DM BM BS B Med
Project Location Molecular Immunology Unit and Child Health Portex Anaesthesia Unit, Institute of Child Health,University College London
Project duration 2 years
Date awarded 29 August 2014
Project start date 12 September 2014
Project end date 11 September 2016
Grant amount £149,062
Grant code GN2299


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