Cystic fibrosis (CF) is a genetic condition affecting over 11,300 people in the UK, including 3,800 children and babies.1 It is a serious, inherited condition that affects many parts of the body, including the lungs and the gastrointestinal tract. Symptoms can include recurrent chest infections and tummy pain and bloating. There is no cure for the condition.
Recently, groundbreaking new medicines – called CFTR modulators – have been developed that can address the underlying cause of CF for many people with the condition, helping to reduce symptoms and make the condition easier to live with. But these medicines don’t work for everyone and are not a cure. Treatments continue to take many hours a day, which can dramatically decrease children’s quality of life. Although life expectancy has been increasing for people with cystic fibrosis, it is still much lower than the general population.
Charities Action Medical Research and Cystic Fibrosis Trust are continuing their joint efforts to drive change and help improve the quality of life of babies and children with CF, by funding much-needed research.
Since 2014 the two charities have invested over £1.2m into seven research projects to further understanding in this area, improve treatments and diagnosis, and to help protect more children with cystic fibrosis.
Current jointly funded projects underway to help tackle cystic fibrosis in children are:
Cystic fibrosis: predicting long-term health outcomes for children with an inconclusive diagnosis led by Dr Rebecca Dobra at the National Heart and Lung Institute, Imperial College London.
Newborn screening helps detect CF early, but some babies receive inconclusive results, creating uncertainty about their future health. The research team is aiming to identify ways to predict which of these children will remain healthy and which may later develop CF or a related condition. The long-term goal is to enable doctors to provide tailored care for each child, reducing parental anxiety, avoiding unnecessary monitoring of healthy children, and enabling early intervention for those most at risk of developing disease.
Developing new drug treatments to help improve the lives of children with cystic fibrosis led by Professor Guy Moss at the Department of Neuroscience, Physiology and Pharmacology, UCL.
Existing medicines (CFTR modulators) used to treat CF can be life-changing for many children, but they do not fully restore health and they do not help some children. The research team will be working to develop effective new drug treatments for CF – helping more children to live longer, healthier lives.
Director of Research at Action Medical Research, Sarah Wagstaffe-Jones says: “Investing together in this important area can really help move us closer to developing much-needed new approaches and treatments to help tackle cystic fibrosis in children.”
Dr Lucy Allen, Director of Research and Healthcare Data at Cystic Fibrosis Trust explains: “Though there have been many improvements in medicines and treatments over the last few years, CF remains an incurable, life-limiting condition. Charities like the Trust and Action Medical Research play a vital role in driving forward the breakthroughs that will lead to the new treatments and support that families and children affected by CF urgently need. We won’t stop until everyone can live without limits imposed by CF.”
References
1. UK Cystic Fibrosis Registry 2023 Annual Data Report (2024), Cystic Fibrosis Trust. London https://www.cysticfibrosis.org.uk/sites/default/files/2024-11/CFT_2023_Annual_Data_Report_Oct2024%201.pdf