Breakthrough research funded by the Dear Toby Trust is now advancing towards the clinic, offering new hope for children with acute myeloid leukaemia (AML). In partnership with Dr. Sara Ghorashian and the team at Great Ormond Street Hospital (GOSH), this pioneering work is set to enter early-phase clinical trials — a major step forward in our mission to find more effective, less toxic treatments for childhood cancer.
Thanks in part to our support, Sara’s team has developed a cutting-edge CAR-T cell therapy specifically targeting AML, one of the most difficult-to-treat childhood cancers. The therapy uses three CARs (chimeric antigen receptors) to attack the disease from multiple angles — a world-first approach that has now received funding for an adult clinical trial, with the potential to extend into paediatrics soon after.
While AML treatments have traditionally relied on aggressive chemotherapy and bone marrow transplants, this new strategy aims to harness a child’s own immune system to eliminate leukaemia cells. The therapy also includes a clever “safety switch”, allowing doctors to deactivate the CAR-T cells when needed and prepare children for a rescue transplant.
The Dear Toby Trust is actively supporting efforts to shorten the timeline between adult and paediatric trials, challenge outdated regulatory norms, and make access to these life-saving innovations fairer and faster for children. Our collaboration with Sara and GOSH is at the heart of this change — providing not just funding, but patient and parent perspectives to help design trials that are as compassionate as they are scientifically rigorous.
This research builds on our wider strategy to accelerate the development of kinder, smarter, and more targeted therapies for children with cancer. Together, we’re helping to ensure that breakthroughs in the lab make it to the bedside — not in decades, but in years.
