Since March 2025, she has been involved in a two-year study investigating a new medication for younger children with CF, helping researchers better understand its long-term safety, how well it is tolerated and how effective it is.
Cystic fibrosis is a rare inherited genetic condition that causes breathing and digestive problems. There is currently no cure, but medicines and supportive treatments can help. By taking part in research with Alder Hey, Pearl is helping researchers develop more effective ways to treat CF.
Away from her hospital appointments, Pearl is a very happy toddler who is football mad. She proudly supports Everton, Manchester United and Tranmere Rovers. She loves watching England play and, when there’s no football on, Pearl usually enjoys episodes of Peppa Pig. She also loves spending time with her family, especially her nana and grandad. Her nana accompanies Pearl and her mum, Carmell, to every hospital appointment.
The two-year study is looking at a new medication, its long-term safety, how well children tolerate it and how effective it is in children aged 12 months and over. As part of the research, Pearl regularly attends Alder Hey for appointments, including blood tests, sweat tests, ECGs, and measurements of her height and weight. At home, mum Carmell manages her treatment as granules each morning and evening. This is on top of her usual CF regime including other medicines and chest physiotherapy.
Before becoming a mum, Carmell had felt nervous about medication, but Pearl’s diagnosis changed her perspective.
Carmell said:
“Once Pearl was born and we received her diagnosis, she needed several different types of medication, including having a nebuliser and needing physiotherapy to help keep her healthy. That changed how I felt. I have seen how important they are and the difference they can make to Pearl’s life and experiences.”
“Taking part in a research trial seemed scary at first. However, what reassured us was how closely Pearl would be monitored. She has regular tests and hospital visits, and the doctors and nurses always look carefully for any changes in her health. As a parent, you have to put your trust in the healthcare team, and we have always felt supported at Alder Hey.”
For the family, the opportunity to contribute to research that could improve care for other young children with CF was an important part of their decision.
She added:
“Taking part in this research trial has meant that Pearl has had the opportunity to trial new medication, whilst allowing healthcare professionals to learn more about how the treatment could help others. It has been a really positive experience, and I would encourage other families who are offered the opportunity to ask questions and consider taking part.”
Research is an important part of care at Alder Hey, helping clinical teams develop and evaluate treatments designed specifically for children and young people. The involvement of children such as Pearl, and the support of their families, helps build the evidence needed to improve care both now and in the future.
Professor Rebecca Thursfield, Consultant in Paediatric Respiratory Medicine, said:
“Research involving very young children is essential to make sure treatments are safe, well tolerated and effective for their age group.”
“By taking part in this study, Pearl and her family are helping us build the evidence needed to guide future cystic fibrosis care. We are very grateful for their time and commitment, and for the contribution they are making for other children and families.”
Pearl is doing well, returning to Alder Hey regularly for her research appointments. Over the summer holidays she has been having lots of fun singing and dancing as well as going on camping trips with her mum. Pearl has continued to watch football and spend time with her friends and family.
Find out more about Research at Alder Hey here