Children's Oncology Group's 2023 blueprint for research: Central nervous system tumors

Abstract

AbstractTumors of the central nervous system (CNS) are a leading cause of morbidity and mortality in the pediatric population. Molecular characterization in the last decade has redefined CNS tumor diagnoses and risk stratification; confirmed the unique biology of pediatric tumors as distinct entities from tumors that occur in adulthood; and led to the first novel targeted therapies receiving Food and Drug Administration (FDA) approval for children with CNS tumors. There remain significant challenges to overcome: children with unresectable low‐grade glioma may require multiple prolonged courses of therapy affecting quality of life; children with high‐grade glioma have a dismal long‐term prognosis; children with medulloblastoma may suffer significant short‐ and long‐term morbidity from multimodal cytotoxic therapy, and approaches to improve survival in ependymoma remain elusive. The Children's Oncology Group (COG) is uniquely positioned to conduct the next generation of practice‐changing clinical trials through rapid prospective molecular characterization and therapy evaluation in well‐defined clinical and molecular groups.