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Children's Oncology Group's 2023 blueprint for research: Central nervous system tumors
Sarah E S Leary1, Arzu Onar-Thomas2, Jason Fangusaro3
1Ben Towne Center for Childhood Cancer Research, Seattle Children's Hospital, Seattle, Washington, USA.
Abstract:
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 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.
Insights
Pediatric central nervous system (CNS) tumors require novel therapies. Advances in molecular characterization have led to targeted treatments, but challenges remain for specific CNS tumor types in children.
Area of Science:
- Pediatric oncology
- Neuro-oncology
- Molecular biology
Background:
- Central nervous system (CNS) tumors are a major cause of illness and death in children.
- Recent molecular studies have improved diagnosis and risk assessment for pediatric CNS tumors, distinguishing them from adult tumors.
- The first targeted therapies for pediatric CNS tumors have been approved by the Food and Drug Administration (FDA).
Purpose of the Study:
- To highlight the ongoing challenges in treating pediatric CNS tumors, including low-grade gliomas, high-grade gliomas, medulloblastoma, and ependymoma.
- To emphasize the need for next-generation clinical trials.
- To showcase the Children's Oncology Group's (COG) role in advancing pediatric CNS tumor research.
Main Methods:
- Molecular characterization of pediatric CNS tumors.
- Risk stratification based on molecular profiles.
- Development and evaluation of novel targeted therapies.
- Conducting practice-changing clinical trials through the Children's Oncology Group (COG).
Main Results:
- Molecular insights have redefined pediatric CNS tumor diagnoses and risk stratification.
- Unique biology of pediatric CNS tumors has been confirmed.
- Novel targeted therapies have received FDA approval for pediatric use.
Conclusions:
- Despite progress, significant challenges persist in pediatric CNS tumor treatment, affecting quality of life and long-term survival.
- The Children's Oncology Group (COG) is vital for future research, focusing on rapid molecular characterization and targeted therapy evaluation in well-defined patient groups.
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