Two-year-old Isaac Byrne, who has cystic fibrosis, with Prof Paul McNally from RCSI. Image: Patrick Bolger
Prof Paul McNally, co-lead of the study, is ‘really excited’ about the potential for this new research to improve understanding and outcomes for people with cystic fibrosis.
A major new study into cystic fibrosis (CF) has been awarded funding of €5.6m to examine the early stages of the disease in children.
Co-led by Prof Paul McNally from the Royal College of Surgeons (RCSI) University of Medicine and Health Sciences, and by Prof Jane Davies from Imperial College London, the ENHANCE (Establishing Natural History in an Advanced New CF Care Era) research team at Children’s Health Ireland (CHI) will study newborn babies and children under six who are diagnosed with CF and children who do not have the disease for comparison. The aim is to deepen the understanding of how the disease progresses in early life in order to develop better treatments and improve the quality of life for children with CF.
“There is so much to learn about the changes that are happening in children with CF. We are really excited about this new study,” said McNally, who emphasised the importance of working closely with the CF community.
“We spent a long time working with parents of children with CF in Ireland and the UK to understand how best to design the study, as we wanted to ensure we are concentrating on the things that are most relevant to children with CF and their parents in this new era,” he said.
To minimise disruption to families, researchers will carry out tests only once each year in 13 paediatric centres in Ireland and the UK. The network of centres includes Crumlin, Tallaght and Temple Street Hospitals in Dublin, University Hospitals in Cork, Limerick and Galway, and the Royal Belfast Hospital for Sick Children.
‘The outlook for children with CF is considerably better than it was 15 years ago’
CF is an inherited condition that primarily affects the lungs and digestive system. Ireland has the highest incidence of the disease per population in the world, with three times the rate of the EU and the US. Approximately 1,400 people live with the condition in Ireland and there are typically about 30 new cases of CF diagnosed here each year.
Though a very serious condition, there have been huge advances in treatment, including new drug therapies to tackle the underlying causes of the disease.
“The ways that we can help children with CF have evolved considerably over the last 15 years, notably with new medicines becoming available, and many children will start these treatments from very early in life,” said McNally. “This means the outlook for children with CF is considerably better than it was 15 years ago, but there is much about this that we don’t yet understand.”
The study, which will be up and running by the end of the year, is currently planned to run for five years but the team hope to extend it long term.
“While we have made much progress as a community, more remains to be done, and it is through studies like ENHANCE that we can understand and focus on the next challenges faced by children with CF and their families,” said Prof Davies.
“By including children from underrepresented groups and those who cannot access some CF treatments, we will get a broad and comprehensive understanding of this new era of CF in childhood.”
The study was funded by the Cystic Fibrosis Foundation in the US, with a separate grant of £100,000 for equipment from the Cystic Fibrosis Trust in the UK.
A similar study is ongoing in the US and the ENHANCE team will collaborate with this research group on methods and findings to further their progress.
Philip Watt, CEO of Cystic Fibrosis Ireland, spoke in support of the study. “With many unknowns and uncertainties remaining, this research is both very welcome and timely from a patient perspective,” he said.
Microbiologists from Queen’s University Belfast recently discovered a protein action in a lung-favouring bacteria that has helped them understand its resistance to the body’s immune system, which they say could lead to new treatments for people with compromised immune systems, including people with CF.
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