Medulloblastoma: From Molecular Subgroups to Molecular Targeted Therapies
Open accessAbstract
Brain tumors are the leading cause of cancer-related death in children, and medulloblastoma (MB) is the most common malignant pediatric brain tumor. Advances in surgery, radiation, and chemotherapy have improved the survival of MB patients. But despite these advances, 25–30% of patients still die from the disease, and survivors suffer severe long-term side effects from the aggressive therapies they receive. Although MB is often considered a single disease, molecular profiling has revealed a significant degree of heterogeneity, and there is a growing consensus that MB consists of multiple subgroups with distinct driver mutations, cells of origin, and prognosis. Here, we review recent progress in MB research, with a focus on the genes and pathways that drive tumorigenesis, the animal models that have been developed to study tumor biology, and the advances in conventional and targeted therapy.
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Alteration of the Sonic Hedgehog (SHH) pathway leads to medulloblastoma (MB), the most common malignant pediatric brain tumor.
“Its alteration leads to medulloblastoma (MB), the most common malignant pediatric brain tumor.”
From SALL4 is a CRL3REN/KCTD11 substrate that drives Sonic Hedgehog-dependent medulloblastoma (2024) · cites this paper as [1]
SHH-MB represents approximately 30% of all medulloblastomas.
“represents ~30% of all MBs [1].”
From SALL4 is a CRL3REN/KCTD11 substrate that drives Sonic Hedgehog-dependent medulloblastoma (2024) · cites this paper as [1]
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Medulloblastomics revisited: biological and clinical insights from thousands of patients
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“In the cerebellum, SHH stimulates the proliferation of granule neuron precursors (GNPs) and the aberrant activation of the SHH signaling in these cells is responsible for medulloblastoma (MB) onset, the most common malignant pediatric brain tumor [1].”
- Authors
- Wang, J., Garancher, A., Ramaswamy, V., & Wechsler-Reya, R. J.
- Affiliations
- Sanford Burnham Prebys Medical Discovery Institute (US)
- Hospital for Sick Children (CA)
- Tumor Initiation and Maintenance Program, NCI-Designated Cancer Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, California 92037, USA;
- Venue
- Annual Review of Neuroscience
- Publisher
- Annual Reviews
- Published
- 2018-07-08
- Volume
- 41
- Issue
- 1
- Pages
- 207-232
- ISSN
- 0147-006X (ISSN-L)
- 1545-4126
- OpenAlex
- W2796721182
- Semantic Scholar
- 28b5acb5cea8eddfd1c175e408e3e1b6901a4eb1
- Cited by
- 113
- References
- 161
- Citations by year
- 2026: 4 · 2025: 14 · 2024: 8 · 2023: 13 · 2022: 13 · 2021: 16 · 2020: 25 · 2019: 14 · 2018: 6
- FWCI
- 2.76
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Annual Review of Neuroscience (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Cancer Institute (P30CA030199, R01CA159859)
- National Institute of Neurological Disorders and Stroke (R01NS096368)
- Topics
- Health Sciences › Medicine › Genetics › Glioma Diagnosis and Treatment
- Health Sciences › Medicine › Neurology › Neuroblastoma Research and Treatments
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Acute Lymphoblastic Leukemia research
- Keywords
- medulloblastoma, pediatric brain tumors, molecular subtypes, driver mutations, cell of origin, tumor heterogeneity, carcinogenesis, targeted therapy, conventional therapy, prognosis, long-term treatment side effects
- MeSH terms
- Animals, Cerebellar Neoplasms, Humans, Medulloblastoma, Molecular Targeted Therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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