Paediatric brain tumours are the most common solid tumours and the leading cause of cancer-related mortality in children. Current treatment, which includes surgery, chemotherapy, and radiotherapy, has improved survival for some types of them but is associated with significant long-term side effects that reduce the quality of life. These unique limitations highlight the urgent need for novel therapeutic strategies tailored to the specific biology and vulnerabilities of the developing brain. This review focuses on clinical trials conducted over the past decade in paediatric brain tumours to elucidate emerging therapeutic directions. By analysing trial phases, type of interventions, and molecular targets, we aimed to identify trends that could inform future treatment development. Our findings show that targeted therapies, particularly kinase inhibitors, epigenetic modulators, and immunotherapies, represent the most frequently explored interventions, although most trials remain in early clinical phases. Diagnostic innovations, including hybrid PET/MRI and molecular profiling, highlight a shift to precision medicine. Collectively, these trends reveal a diversifying therapeutic field that may, with international collaboration and long-term safety evaluation, transform outcomes for children with paediatric brain tumours.
Shah et al. (2025) studied this question.