More than 11,300 people in the UK – including 3,800 children and babies – are living with cystic fibrosis (CF).[1] It’s a serious, lifelong condition that can make it hard for children to breathe and digest food. Sadly, there is no cure, but new medicines have dramatically improved the outlook for many children. However, these treatments don’t work for everyone, leaving around one in 10 children in desperate need of other options.[2] Dr Guy Moss of University College London is leading laboratory research to develop effective new drug treatments, ensuring every child with CF has the chance to live a long and full life.
This project is jointly funded by Action Medical Research and Cystic Fibrosis Trust.
How are children’s lives affected now?
CF is a genetic condition that disrupts the movement of salt and water in and out of cells, leading to a build-up of thick, sticky mucus in the lungs and digestive system. Children born with CF experience a range of challenging symptoms that significantly impact their quality of life and can shorten their life expectancy.
“Children with CF get frequent, long-lasting chest infections that are often difficult to treat and are usually the main cause of ill health,” says Dr Moss.
In recent years, groundbreaking new medicines – called CFTR modulators – have been developed that can address the underlying cause of CF, helping to reduce symptoms and make the condition easier to live with.
“These medicines are life-changing for many children with CF – but they do not fully restore health, leaving room for improvement,” says Dr Moss. “Crucially, some children do not benefit from existing CFTR modulators, highlighting the urgent need for new treatment options to ensure no child with CF is left behind.”
How could this research help?
Our goal is to develop effective new drug treatments for CF – helping more children to live longer, healthier lives.
The researchers have already identified several drugs, proven safe in early clinical trials for other conditions, which could further improve the effectiveness of the current gold-standard CFTR modulator treatment for children with CF.
“We now plan to test these drugs on lung cells donated by people with CF and grown in the laboratory – to see if they could be repurposed for CF,” says Dr Moss.
The team will also look for potential new drugs that act in a completely different way to existing CFTR modulators, which could help children who do not benefit from these treatments.
“We hope our laboratory work will ultimately lead to new, effective drug treatments that can help improve the quality of life for children with CF – reducing infections and lung damage, improving breathing and extending their lives,” says Dr Moss.
Research table
Project details
| Project Leader | Dr Guy W J Moss PhD |
| Location | Department of Neuroscience, Physiology and Pharmacology, University College London |
| Project Team |
Professor Paola Vergani PhD
Professor David N Sheppard PhD (Cantab) |
| Other Locations | School of Physiology, Pharmacology & Neuroscience, University of Bristol |
| Grant Awarded | |
| Grant Amount | £198,134 |
| Start Date | |
| End Date | |
| Duration | 24 months |
| Grant Code (GN number) | GN3079 |
References
- Cystic Fibrosis Trust, UK Cystic Fibrosis Registry 2023 Annual Data Report (October 2024). https://www.cysticfibrosis.org.uk/sites/default/files/2024-11/CFT_2023_Annual_Data_Report_Oct2024%201.pdf [Accessed March 2025].
- Kramer-Golinkoff et al. A survey: Understanding the health and perspectives of people with CF not benefiting from CTFR modulators. 2022; 57(5): 1253-1261.
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