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Volume 15
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10.3390/brainsci15080896
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Review

Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments

by
David T. Kim
1,†,
Michaela Uloho-Okundaye
1,†,
Stephen C. Frederico
2,
Santosh Guru
3,
Min J. Kim
4 and
Steven D. Chang
5,*
1
Harvard College, Harvard University, Cambridge, MA 02138, USA
2
Department of Neurological Surgery, University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA
3
School of Clinical Medicine, University of Cambridge, Cambridge CB2 0SP, UK
4
Department of Neurosurgery, Harvard Medical School and Mass General Brigham, Boston, MA 02115, USA
5
Department of Neurosurgery, Stanford School of Medicine, Palo Alto, CA 94304, USA
*
Author to whom correspondence should be addressed.
†
These authors contributed equally to this work.
Brain Sci. 2025, 15(8), 896; https://doi.org/10.3390/brainsci15080896
Submission received: 8 July 2025 / Revised: 17 August 2025 / Accepted: 18 August 2025 / Published: 21 August 2025
(This article belongs to the Section Neuro-oncology)
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Abstract

Medulloblastoma (MB), the most common malignant pediatric brain tumor, has undergone reclassification from a histologically defined disease to a genetically stratified spectrum of distinct subgroups: WNT, SHH, Group 3, and Group 4. Advances in molecular profiling, as captured in the 2021 WHO CNS5 classification, have shown meaningful heterogeneity in terms of tumor biology, prognosis, and therapeutic response. However, translating these insights into precise, less toxic treatments remains an ongoing challenge. This review synthesizes current knowledge on MB subgroup biology, treatment strategies, and emerging therapies such as subgroup-specific inhibitors, immunotherapies, and novel chemotherapeutic regimens. This review also explores risk-adapted approaches while addressing global disparities in access to diagnostics and care. As the field moves toward individualized medicine, closing the gap between molecular understanding and equitable implementation will be crucial to improving outcomes and quality of life for children with medulloblastoma worldwide.
Keywords: medulloblastoma; brain tumors; pediatric brain tumors; targeted therapy; oncology; pediatric oncology; immunotherapy; precision medicine; WHO CNS5 medulloblastoma; brain tumors; pediatric brain tumors; targeted therapy; oncology; pediatric oncology; immunotherapy; precision medicine; WHO CNS5

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MDPI and ACS Style

Kim, D.T.; Uloho-Okundaye, M.; Frederico, S.C.; Guru, S.; Kim, M.J.; Chang, S.D. Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments. Brain Sci. 2025, 15, 896. https://doi.org/10.3390/brainsci15080896

AMA Style

Kim DT, Uloho-Okundaye M, Frederico SC, Guru S, Kim MJ, Chang SD. Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments. Brain Sciences. 2025; 15(8):896. https://doi.org/10.3390/brainsci15080896

Chicago/Turabian Style

Kim, David T., Michaela Uloho-Okundaye, Stephen C. Frederico, Santosh Guru, Min J. Kim, and Steven D. Chang. 2025. "Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments" Brain Sciences 15, no. 8: 896. https://doi.org/10.3390/brainsci15080896

APA Style

Kim, D. T., Uloho-Okundaye, M., Frederico, S. C., Guru, S., Kim, M. J., & Chang, S. D. (2025). Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments. Brain Sciences, 15(8), 896. https://doi.org/10.3390/brainsci15080896

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