CAR-T cell therapy: potential for paediatric brain tumours—an update

Central nervous system (CNS) tumours are the deadliest cancer for children and currently present limited treatment options. Chimeric antigen receptor (CAR)-T cell therapies have emerged as an innovative approach supported by encouraging clinical results. Current clinical trials using CAR-T cells in the treatment of paediatric CNS cancers differ in a number of variables, including the CAR-T cell route of delivery, presence of lymphodepletion, identified target antigen, and CAR engineering features. Considering early learnings across these areas is an essential step to developing more effective treatment options, especially given the challenges of immunosuppressive tumour microenvironments, various toxicities, CAR-T cell exhaustion, and tumour antigen heterogeneity. In sum, while there is a need for continued innovation, CAR-T cells represent a promising treatment approach for this devastating category of diseases.