Paul is fundraising for the children’s charity Action Medical Research.
Paul from Lincolnshire is delighted that after eight years of trying he will be running in the 2024 London Marathon for the first time. Paul will be running to raise funds for the children’s charity Action Medical research, to help support rare disease research. He is doing this in memory of his son, Oliver, who died in 2012 due to a rare condition called X-linked lymphoproliferative disease (XLP), when he was just two years old.
XLP is an extremely rare and life-limiting, inherited disease that affects boys and causes the immune system to overact to infections. There are some medicines which help boys with XLP and bone marrow transplants can cure the condition, but this means finding a donor who is a good match. For transplants to work well, it’s best to carry them out early in the disease process and sadly, if transplants come too late or donors cannot be found, boys with XLP remain at risk of losing their lives. Without treatment, around seven in every 10 boys with XLP will die by the age of 10. XLP can also cause other complications, such as haemophagocytic lymphohistiocytosis (HLH) – an extreme immune reaction that can be triggered by a common human virus. Paul’s son Oliver sadly passed away due to HLH.
Before 2012, Paul had never been a runner, but the devastating loss of Oliver propelled him and his wife Andrea to raise awareness and funds for research into HLH. Paul entered his first run, a 20-mile run with 200 obstacles in 2013 to raise money for charity. This inspired a love of running. Paul says, “Because of Oliver, this is a cause very close to my heart. Even writing this has made me cry as the underlying pain never goes away. The whole journey of training for and running the marathon will be emotional. But if it helps raise money to prevent other parents from going through the same pain we have it will be worth it. No parent should every have to go through the pain of losing a child which is why, since 2013, we have raised more than £25,000 into research into finding a cure and are committed to doing as much as we can.”
Over the past 10 years, Action Medical Research has funded research into XLP, investigating new treatments and ultimately working towards a cure for boys with this condition. This funding has enabled the project team to modify the faulty gene in T-cells, from the child’s own immune system, and has shown that these modified cells improve the symptoms of XLP in the laboratory. Professor Claire Booth, Professor in Gene Therapy and Paediatric Immunology at Great Ormond Street Hospital, says: “This would not have happened without Action funding – we have shown this approach can work and is worth progressing to the clinic.” The next step is a clinical trial, due to happen early 2025, with the hope of developing a cure for boys with XLP that will enable them to live normal lives.
Sarah Moss, Director of Communications at Action Medical Research comments: “Action Medical Research is committed to funding research into rare childhood diseases like XLP. We are really excited to see how research in this area is developing and that research we have funded has helped lead to approval of the first clinical trial of a new cure. Thanks to supporters like Paul, we are able to help work towards a cure for boys with XLP.”
You can support Paul in the London Marathon here: www.justgiving.com/page/londonmarathonforolly.
Learn more about the work Action funds in rare disease research: www.action.org.uk/rare.