Precision neuro-oncology for children: Time to gear up!

Molecular profiling is central to pediatric precision oncology, yet only 15-20% of patients benefit from biology-guided treatments. This limitation underscores the urgent need for functional precision medicine, which interrogates therapeutic vulnerabilities using patient-derived models. Despite hurdles in establishing these models, such as limited biopsy material and technical screening barriers, recent advances in tumoroids, patient-derived xenografts and high-throughput platforms offer an unprecedented opportunity to bridge the translational gap. In this review, we describe the evolution of patient-derived models of pediatric brain cancers, their current strengths and weaknesses and the technological innovations redefining the field. Together, these insights provide a road map for integrating functional data into clinical decision-making, ultimately aiming to 'cure more and heal better' children with brain tumors.