# Medulloblastoma

Source: https://onco.cc/cancers/medulloblastoma/  
OnCo record `medulloblastoma` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less.

## Summary

Medulloblastoma is an embryonal tumour of the posterior fossa with four consensus molecular subgroups (WNT, SHH, Group 3, Group 4) that differ in age, genetics, metastatic tendency and survival: WNT (~10%, CTNNB1 mutations, >95% survival), SHH (~30%, PTCH1/SUFU/SMO, TP53-mutant subset with poor prognosis; infants and adults), Group 3 (~25%, MYC amplification, worst prognosis) and Group 4 (~35%, most common, intermediate). WHO 2021 integrates histology and molecular group.

Standard therapy is maximal safe resection, craniospinal irradiation (CSI; 23.4 Gy for average risk, 36 Gy for high risk) with posterior fossa/tumour bed boost, and adjuvant cisplatin-based chemotherapy (cisplatin, vincristine, cyclophosphamide, lomustine). Infants under 3 receive radiation-sparing intensive chemotherapy (with high-dose chemotherapy/autologous rescue or intraventricular methotrexate) because CSI is devastating to the developing brain. Risk-adapted trials (SJMB12, ACNS1422, SIOP PNET5) are reducing CSI dose for WNT tumours and testing SMO inhibitors for SHH tumours in skeletally mature patients. Proton therapy reduces exit dose to cochlea, heart and thyroid. Relapse is rarely curable outside infants; MRI-based surveillance, cfDNA in CSF, and survivorship (neurocognition, endocrine, hearing, second cancers, cerebellar mutism) dominate follow-up.

## Fields

- Kind: Cancer
- Last checked: 2026-09-08
- Tags: gap-fill; paediatric; cns
- Group: paediatric
- Burden: The most common malignant brain tumour of childhood (~20% of paediatric CNS tumours; ~500 cases per year in the US), peak age 3-8; also occurs in adults.
- Subtypes: WNT-activated (best prognosis); SHH-activated, TP53-wild-type; SHH-activated, TP53-mutant (poor); Group 3 (MYC-amplified subset very poor); Group 4; Adult medulloblastoma (mostly SHH)
- Biomarkers: Molecular subgroup (methylation profiling; IHC surrogates β-catenin, GAB1, YAP1); MYC / MYCN amplification; TP53 mutation (SHH); Metastatic stage (Chang M0-M4) by MRI and CSF cytology; Extent of resection (residual >1.5 cm²); Histology (desmoplastic/nodular, large cell/anaplastic); Germline testing (SUFU, PTCH1, TP53, BRCA2, PALB2, APC)

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/medulloblastoma/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/medulloblastoma/#overview [3 subtypes, 4 state-of-the-art points]
- What it is (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/medulloblastoma/#what-it-is [9 subtypes]
- Finding it (on the hub): How it shows itself, how it is confirmed, what screening exists, and the biomarkers clinicians test for. https://onco.cc/cancers/medulloblastoma/#finding-it [7 biomarkers, 1 prevalence rows]
- Treating it (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/medulloblastoma/#treating-it [4 settings, 4 decisions with options]
- Evidence (on the hub): Trials recruiting now, the landmark trials, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/medulloblastoma/#evidence [7 trials, 1 key paper, 8 milestones]
- The science (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/medulloblastoma/#science [24 targets, 2 pathways, 5 preclinical models]
- Where you are (own page): Cases by country, the UK and NHS pathway and other country lenses, and the expert centres with trials on record. https://onco.cc/cancers/medulloblastoma/where-you-are/ [7 centres]
- Living with it (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/medulloblastoma/#living-with-it [17 questions, 6 red cards]
- What is coming (own page): Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/medulloblastoma/coming/ [12 medicines, 7 trials, 5 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/medulloblastoma/data/ [101 connected records]

## Standard of care

- Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp): Resection, CSI 23.4 Gy with boost to 54 Gy (proton where available), then cisplatin/vincristine/cyclophosphamide or lomustine-based chemotherapy (ACNS0331); WNT tumours receive reduced CSI in trials. ([Cisplatin](https://onco.cc/drugs/cisplatin/), [Vincristine](https://onco.cc/drugs/vincristine/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Lomustine (CCNU)](https://onco.cc/drugs/lomustine/), [Proton therapy](https://onco.cc/technologies/proton-therapy/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/))
- High risk (metastatic, residual, anaplastic, MYC): CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols. ([Carboplatin](https://onco.cc/drugs/carboplatin/), [Cisplatin](https://onco.cc/drugs/cisplatin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/))
- Infants (<3 years): Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly. ([Methotrexate](https://onco.cc/drugs/methotrexate/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/))
- Relapsed: Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare. ([Temozolomide](https://onco.cc/drugs/temozolomide/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Bevacizumab](https://onco.cc/drugs/bevacizumab/), [Vismodegib](https://onco.cc/drugs/vismodegib/))

## State of the art

- Four molecular subgroups (2012) now define trials; the goal is to de-escalate for WNT and SHH-favourable tumours and intensify for MYC-amplified Group 3.
- Proton CSI reduces long-term hearing, endocrine and cardiac toxicity without loss of control.
- Survival is ~70-75% overall but neurocognitive decline of 2-4 IQ points per year after CSI in young children is the price.
- Targeted therapy exists only for SHH (SMO inhibitors) and only after skeletal maturity, because of growth-plate fusion.

## Open problems

- Group 3 MYC-amplified and SHH TP53-mutant tumours: survival under 50%.
- Relapse after craniospinal irradiation is rarely curable; it is where new approaches are most needed.
- Neurocognitive, endocrine and hearing sequelae in survivors.
- Infants: curing Group 3 without radiation.
- Access to proton therapy and methylation profiling globally.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Medulloblastoma
- NCI PDQ: childhood medulloblastoma: https://www.cancer.gov/types/brain/patient/child-medulloblastoma-treatment-pdq
- Taylor 2012 consensus subgroups: https://doi.org/10.1007/s00401-011-0922-z
- Children's Oncology Group: https://childrensoncologygroup.org/

## Connected records

- collections: [Alex's Lemonade Stand Foundation (ALSF)](https://onco.cc/collections/alexs-lemonade-stand/), [National Brain Tumor Society (NBTS)](https://onco.cc/collections/national-brain-tumor-society/), [St. Baldrick's Foundation](https://onco.cc/collections/st-baldricks/)
- cancers: [Atypical teratoid/rhabdoid tumour (ATRT)](https://onco.cc/cancers/atrt/), [Brain and spinal cord tumours (all types)](https://onco.cc/cancers/brain-tumours/), [Central nervous system germ cell tumours (germinoma and non-germinomatous)](https://onco.cc/cancers/cns-germ-cell-tumours/), [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Ependymoma](https://onco.cc/cancers/ependymoma/), [Glioma & glioblastoma](https://onco.cc/cancers/glioblastoma/), [Group 3 and group 4 medulloblastoma (non-WNT/non-SHH)](https://onco.cc/cancers/medulloblastoma-group-3-4/), [Pineoblastoma](https://onco.cc/cancers/pineoblastoma/), [SHH-activated medulloblastoma](https://onco.cc/cancers/medulloblastoma-shh/), [WNT-activated medulloblastoma](https://onco.cc/cancers/medulloblastoma-wnt/)
- technologies: [Antineoplastons (Burzynski clinic)](https://onco.cc/technologies/antineoplastons/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [DNA methylation profiling](https://onco.cc/technologies/methylation-profiling/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/), [MRI](https://onco.cc/technologies/mri/), [Proton therapy](https://onco.cc/technologies/proton-therapy/), [Proton therapy machines: cyclotrons, synchrotrons and single-room systems](https://onco.cc/technologies/proton-therapy-systems/), [Survivorship care and late-effects surveillance](https://onco.cc/technologies/survivorship-care-plan/), [TomoTherapy and Radixact (helical radiotherapy)](https://onco.cc/technologies/tomotherapy/)
- targets: [CCAR1](https://onco.cc/targets/ccar1/), [CHD7](https://onco.cc/targets/chd7/), [DDX3X](https://onco.cc/targets/ddx3x/), [DDX6](https://onco.cc/targets/ddx6/), [ELP1](https://onco.cc/targets/elp1/), [FAM174B](https://onco.cc/targets/fam174b/), [FOXO3](https://onco.cc/targets/foxo3/), [GPR161](https://onco.cc/targets/gpr161/), [HIP1](https://onco.cc/targets/hip1/), [KAT6B](https://onco.cc/targets/kat6b/), [KDM3B](https://onco.cc/targets/kdm3b/), [LDB1](https://onco.cc/targets/ldb1/), [PRKAR1A](https://onco.cc/targets/prkar1a/), [SMARCB1](https://onco.cc/targets/smarcb1/), [Smoothened (hedgehog pathway)](https://onco.cc/targets/smoothened/), [SUFU](https://onco.cc/targets/sufu/), [TCF4](https://onco.cc/targets/tcf4/), [TP53](https://onco.cc/targets/tp53/), [TPR](https://onco.cc/targets/tpr/), [TRIM33](https://onco.cc/targets/trim33/), [WAS](https://onco.cc/targets/was/), [ZMYM3](https://onco.cc/targets/zmym3/), [ZNF274](https://onco.cc/targets/znf274/)
- drugs: [Bevacizumab](https://onco.cc/drugs/bevacizumab/), [Carboplatin](https://onco.cc/drugs/carboplatin/), [Carmustine](https://onco.cc/drugs/carmustine/), [Cisplatin](https://onco.cc/drugs/cisplatin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Lomustine (CCNU)](https://onco.cc/drugs/lomustine/), [Methotrexate](https://onco.cc/drugs/methotrexate/), [Sodium thiosulfate (otoprotectant)](https://onco.cc/drugs/sodium-thiosulfate/), [Temozolomide](https://onco.cc/drugs/temozolomide/), [Vincristine](https://onco.cc/drugs/vincristine/), [Vismodegib](https://onco.cc/drugs/vismodegib/)
- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/), [Roche / Genentech](https://onco.cc/companies/roche-genentech/)
- pathways: [p53 / RB / cell-cycle checkpoint](https://onco.cc/pathways/p53-cell-cycle/), [Wnt / β-catenin](https://onco.cc/pathways/wnt/)
- terms: [Blood-brain barrier (BBB)](https://onco.cc/terms/blood-brain-barrier/), [Li-Fraumeni syndrome (germline TP53)](https://onco.cc/terms/li-fraumeni/), [Medulloblastoma molecular groups (WNT, SHH, group 3, group 4)](https://onco.cc/terms/medulloblastoma-molecular-groups/)
- trials: [A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss](https://onco.cc/trials/nct05382338/), [ACNS1422](https://onco.cc/trials/acns1422/), [Childhood Cancer Survivor Study (CCSS)](https://onco.cc/trials/ccss/), [COG ACNS0331](https://onco.cc/trials/acns0331/), [COG ACNS0332](https://onco.cc/trials/acns0332/), [SJMB12](https://onco.cc/trials/sjmb12/), [Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors](https://onco.cc/trials/nct03709680/)
- pairings: [Caution: proton therapy vs IMRT](https://onco.cc/pairings/proton-vs-imrt/)
- people: [Amar Gajjar](https://onco.cc/people/amar-gajjar/), [Ashya King](https://onco.cc/people/ashya-king/), [Brandon Wainwright](https://onco.cc/people/brandon-wainwright/), [Giles W. Robinson](https://onco.cc/people/giles-robinson/), [Louis Chesler](https://onco.cc/people/louis-chesler/), [Michael D. Taylor](https://onco.cc/people/michael-taylor/), [Sarah E. S. Leary](https://onco.cc/people/sarah-leary/), [Steven Clifford](https://onco.cc/people/steven-clifford/)
- key papers: [Singh 2004: identification of human brain tumour initiating cells](https://onco.cc/key-papers/paper-singh-brain-tumour-initiating-cells-nature-2004/)
- institutions: [Juravinski Cancer Centre / Escarpment Cancer Research Institute](https://onco.cc/institutions/juravinski/), [Nationwide Children's Hospital](https://onco.cc/institutions/nationwide-childrens/), [Seattle Children's Hospital](https://onco.cc/institutions/seattle-childrens/), [St. Jude Children's Research Hospital](https://onco.cc/institutions/st-jude/), [Texas Children's Cancer and Hematology Center](https://onco.cc/institutions/texas-childrens/), [The Hospital for Sick Children (SickKids)](https://onco.cc/institutions/sickkids/), [University of Florida Health Cancer Center](https://onco.cc/institutions/uf-health-cancer-center/)
- journals: [Brain tumor pathology](https://onco.cc/journals/brain-tumor-pathology/), [CNS oncology](https://onco.cc/journals/cns-oncology/), [Journal of neuro-oncology](https://onco.cc/journals/journal-of-neuro-oncology/)

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JSON: https://onco.cc/api/v1/entities/medulloblastoma.json