Immunotherapy Approaches for Pediatric CNS Tumors and Associated Neurotoxicity

Treatment for brain tumors has recently shifted to using the power of the immune system to destroy cancer cells with promising results. Many immunotherapeutic approaches that have been used in adults, including checkpoint inhibitors, vaccine therapy, adoptive immunotherapy, such as chimeric antigen receptor T cell therapy, and viral therapy, are now being evaluated in children. Although these treatments work through different mechanisms, they all activate the immune system and can result in inflammation at the site of disease. This can be especially problematic in the confined area of the brain causing potentially severe neurological side effects, which are of special concern in children with central nervous system malignancies. Steroids can be helpful in the management of neurological complications but carry the risk of making immunotherapeutic approaches less effective. Alternative therapeutic interventions to mitigate side effects are being evaluated. This review describes the most common immunotherapeutic modalities that are now under study for the treatment of pediatric brain tumors, their rationale, associated neurotoxicities, and current management.