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Updated: Nov 27, 2025
Establishment of Orthotopic Patient-derived Xenograft Models for Brain Tumors using a Stereotaxic Device
Published on: May 2, 2025
Advances in the classification and treatment of pediatric brain tumors
Jason Fangusaro1, Pratiti Bandopadhayay2,3,4
1Children's Healthcare of Atlanta, Emory University and the Aflac Cancer Center, Atlanta, Georgia.
Purpose Of Review:
Recent genetic and molecular findings have impacted the diagnosis, prognosis, and in some instances, treatment strategies for children with pediatric central nervous system tumors. Herein, we review the most up-to-date molecular findings and how they have impacted tumor classification and clinical care.
Recent Findings:
It is now recognized that aberrations of the mitogen-activated protein kinase pathway are present in the majority of pediatric low-grade glioma. Also, there has been the identification of recurrent histone H3 K27M mutations in diffuse intrinsic pontine and other midline gliomas. Medulloblastoma is now divided into four molecular subgroups with distinct characteristics and prognoses. The classification of other unique embryonal tumors is also highlighted. Finally, we present the newest classification of ependymoma; supratentorial ependymomas comprise two subtypes based on expression of the chromosome 11 Open Reading Frame 95-reticuloendotheliosis Viral Oncogene Homolog A or yes-associated protein 1 fusion, whereas posterior fossa ependymomas are divided into two distinct molecular subgroups, posterior fossa-A and posterior fossa-B.
Summary:
These advances in the molecular classification of pediatric central nervous system tumors have not only assisted in diagnoses, but they have led to a new era of tumor classification and prognostication. They also have served as drivers for the evaluation of new targeted therapies based upon molecular aberrations with the hope for improved survival outcomes for our patients.
Insights
Recent molecular discoveries are revolutionizing pediatric central nervous system tumor diagnosis and treatment. These advances improve classification and pave the way for targeted therapies, enhancing patient outcomes.
Area of Science:
- Pediatric oncology
- Neuro-oncology
- Molecular genetics
Background:
- Pediatric central nervous system (CNS) tumors are a diverse group of cancers.
- Traditional classification relied on histology, but molecular insights are transforming understanding.
Purpose of the Study:
- To review the latest molecular findings in pediatric CNS tumors.
- To discuss the impact of these findings on tumor classification and clinical management.
Main Methods:
- Review of recent genetic and molecular studies.
- Analysis of how molecular data influences diagnostic and prognostic strategies.
Main Results:
- Mitogen-activated protein kinase pathway aberrations are common in pediatric low-grade glioma.
- Recurrent histone H3 K27M mutations identified in midline gliomas.
- Medulloblastoma now classified into four distinct molecular subgroups.
- New classifications for ependymoma subtypes based on molecular fusions and subgroups (PF-A, PF-B).
Conclusions:
- Molecular classification has significantly improved diagnosis and prognostication of pediatric CNS tumors.
- These advances are driving the development of targeted therapies.
- The goal is to improve survival outcomes for affected children.
