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

Leary SES, Onar-Thomas A, Fangusaro J, Gottardo NG, Cohen K, Smith A, Huang A, Haas-Kogan D, Fouladi M. Children’s Oncology Group’s 2023 blueprint for research: Central nervous system tumors. Pediatr Blood Cancer. 2023 Sep;70 Suppl 6(Suppl 6):e30600. doi: 10.1002/pbc.30600. Epub 2023 Aug 3. PubMed PMID: 37534382; PubMed Central PMCID: PMC10569820.

Tumors 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 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.