A Phase I clinical trial (CARPALL) has opened at Great Ormond Street Hospital (GOSH) to investigate the use of immunotherapy with genetically modified T cells for acute lymphoblastic leukemia (ALL). The trial is led by Professor Persis Amrolia, who is supported by the NIHR Great Ormond Street BRC.
Relapse is the primary cause of treatment failure for paediatric ALL with 25 children in the UK dying per year from untreatable leukaemia relapse. Emerging data shows unprecedented responses to immunotherapy, with genetically redirected T cells expressing second-generation chimeric antigen receptors (CARs) enabling them to recognise and kill ALL cells. However, it is unclear how sustained these remissions are, as most responding patients have been consolidated with stem cell transplant (SCT).
The trial will take place at GOSH, University College London Hospital (UCLH) and Manchester Children’s hospital. It aims to treat 15 children and young adults with high-risk relapsed ALL to investigate the durability of responses to CD19CAR T cells, and whether this strategy could remove the need for SCT. If successful, it could mean that the short term mortality and late toxicities of SCT could be avoided.