OnCo
cancersCancer

Medulloblastoma

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.

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 usually fatal outside infants; MRI-based surveillance, cfDNA in CSF, and survivorship (neurocognition, endocrine, hearing, second cancers, cerebellar mutism) dominate follow-up.

State of the art today

  • 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.
  • Targeted therapy exists only for SHH (SMO inhibitors) and only after skeletal maturity, because of growth-plate fusion.
Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • Survival is ~70-75% overall but neurocognitive decline of 2-4 IQ points per year after CSI in young children is the price.
Who it affects

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.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.

Standard of care

4top
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.

COG ACNS0331; SIOP PNET5
CisplatinVincristineCyclophosphamideLomustine (CCNU)Proton therapyIMRT / IGRT (modern external beam)
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.

COG ACNS0332
CarboplatinCisplatinCyclophosphamideAutologous stem cell transplant (high-dose therapy)
Infants (<3 years)

Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.

MethotrexateCyclophosphamideAutologous stem cell transplant (high-dose therapy)
Relapsed

Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.

TemozolomideIrinotecan (and liposomal irinotecan)BevacizumabVismodegib

Subtypes & biomarkers

top
Subtypes
Biomarkers clinicians test

Target prevalence in this cancer

Target / alterationPrevalenceSource
Smoothened (hedgehog pathway)
30%

How common each drug target or alteration is in this cancer. Population-level and approximate; see the target page for detail. Full matrix.

History

8top
  1. 1925Bailey and Cushing name medulloblastoma
  2. 1953Craniospinal irradiation introduced (Paterson and Farr)

    First cures.

  3. 1990Chemotherapy after radiation improves survival (Packer)
    CisplatinVincristine
  4. 2006Reduced-dose CSI (23.4 Gy) safe for average risk with chemotherapy (CCG 9892/ACNS0331)
  5. 2012Four molecular subgroups agreed (Taylor et al., Acta Neuropathol)
  6. 2016WHO integrates molecular groups into classification
  7. 2017Proton CSI: equivalent control, fewer toxicities (Yock, Lancet Oncol)
    Proton therapy
  8. 2021SJMB03 subgroup outcomes; ACNS0331 shows CSI dose reduction below 23.4 Gy unsafe for most

Pipeline

3top
Not mapped hereSmall-molecule smoothened (hedgehog) inhibitor
Vismodegib · Erivedge
Proton therapyDNA methylation profiling

Open problems

  • Group 3 MYC-amplified and SHH TP53-mutant tumours: survival under 50%.
  • Relapse is almost always fatal after craniospinal irradiation.
  • Neurocognitive, endocrine and hearing sequelae in survivors.
  • Infants: curing Group 3 without radiation.
  • Access to proton therapy and methylation profiling globally.

Trials

top

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Medulloblastoma
condition: Medulloblastoma
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Expert centres

top
Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

top
Bring to your appointment

Questions to ask your oncologist about Medulloblastoma

Generated from this cancer's standard of care, biomarkers, and pipeline · 17 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. Which biomarkers have been tested on my tumour (for example Molecular subgroup, MYC / MYCN amplification, TP53 mutation, Metastatic stageby MRI and CSF cytology, Extent of resection), and what were the results?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include WNT-activated, SHH-activated, TP53-wild-type, SHH-activated, TP53-mutant.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp)

  1. For my situation (average risk (≥3 years, m0, <1.5 cm² residual, no myc amp)), which of the standard options do you recommend and why?
    Why: Guideline options include: 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.
  2. Am I a candidate for Cisplatin, Vincristine, Cyclophosphamide or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

High risk (metastatic, residual, anaplastic, MYC)

  1. For my situation (high risk (metastatic, residual, anaplastic, myc)), which of the standard options do you recommend and why?
    Why: Guideline options include: CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols.
  2. Am I a candidate for Carboplatin, Cisplatin, Cyclophosphamide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Infants (<3 years)

  1. For my situation (infants (<3 years)), which of the standard options do you recommend and why?
    Why: Guideline options include: Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.
  2. Am I a candidate for Methotrexate, Cyclophosphamide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Relapsed

  1. For my situation (relapsed), which of the standard options do you recommend and why?
    Why: Guideline options include: Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.
  2. Am I a candidate for Temozolomide, Irinotecan (and liposomal irinotecan), Bevacizumab or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Any stage

  1. Are there clinical trials I could join, for example of Proton therapy, Vismodegib, DNA methylation profiling?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. Would a second opinion at a high-volume centre change anything, and can you help arrange it?
    Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
  3. What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
    Why: Supportive care improves quality of life and helps patients complete treatment.
  4. I read that “Group 3 MYC-amplified and SHH TP53-mutant tumours: survival under 50%”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Relapse is almost always fatal after craniospinal irradiation”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.

Everything relevant

46top

Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

14
Allogeneic stem cell transplantationAnti-angiogenic therapyAutologous stem cell transplant (high-dose therapy)CAR-T cell therapyCytotoxic chemotherapyDNA methylation profilingIMRT / IGRT (modern external beam)Monoclonal antibodiesMRIPlatinum agentsProton therapySmall-molecule kinase inhibitorsSurvivorship care and late-effects surveillanceTopoisomerase-I inhibitors (and ADC payloads)

targets

3
Smoothened (hedgehog pathway)TP53VEGF / VEGFR

drugs

11
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab · Avastin (and biosimilars)
ApprovedCytotoxic chemotherapy (platinum)
Carboplatin
ApprovedCytotoxic chemotherapy (platinum)
Cisplatin · Platinol (generic)
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
Not mapped hereOral nitrosourea chemotherapy
Lomustine (CCNU) · Gleostine
Not mapped hereAntifolate (DHFR inhibitor)
Methotrexate · Trexall / Otrexup / Xatmep
Not mapped hereSmall-molecule chemoprotectant
Sodium thiosulfate (otoprotectant) · Pedmark
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
Not mapped hereSmall-molecule smoothened (hedgehog) inhibitor
Vismodegib · Erivedge

companies

3
IpsenPfizer (incl. Seagen)Roche / Genentech

institutions

7
Children's Oncology Group (COG)Juravinski Cancer Centre / Escarpment Cancer Research InstituteNationwide Children's HospitalSt. Jude Children's Research HospitalTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)University of Florida Health Cancer Center

pathways

2
p53 / RB / cell-cycle checkpointWnt / β-catenin

terms

2
Blood-brain barrier (BBB)Li-Fraumeni syndrome (germline TP53)

collections

3
Alex's Lemonade Stand Foundation (ALSF)National Brain Tumor Society (NBTS)St. Baldrick's Foundation

people

1
Ashya King

Latest papers

top
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Medulloblastoma" OR ABSTRACT:"Medulloblastoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Medulloblastoma, not a curated reading list.

Connected

36top

Pages like this

not linked directly; found by shared links

technologies

7
Autologous stem cell transplant (high-dose therapy)Cytotoxic chemotherapyDNA methylation profilingIMRT / IGRT (modern external beam)MRIProton therapySurvivorship care and late-effects surveillance

targets

2
Smoothened (hedgehog pathway)TP53

drugs

11
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab · Avastin (and biosimilars)
ApprovedCytotoxic chemotherapy (platinum)
Carboplatin
ApprovedCytotoxic chemotherapy (platinum)
Cisplatin · Platinol (generic)
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
Not mapped hereOral nitrosourea chemotherapy
Lomustine (CCNU) · Gleostine
Not mapped hereAntifolate (DHFR inhibitor)
Methotrexate · Trexall / Otrexup / Xatmep
Not mapped hereSmall-molecule chemoprotectant
Sodium thiosulfate (otoprotectant) · Pedmark
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
Not mapped hereSmall-molecule smoothened (hedgehog) inhibitor
Vismodegib · Erivedge

companies

1
Roche / Genentech

institutions

7
Children's Oncology Group (COG)Juravinski Cancer Centre / Escarpment Cancer Research InstituteNationwide Children's HospitalSt. Jude Children's Research HospitalTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)University of Florida Health Cancer Center

pathways

2
p53 / RB / cell-cycle checkpointWnt / β-catenin

terms

2
Blood-brain barrier (BBB)Li-Fraumeni syndrome (germline TP53)

collections

3
Alex's Lemonade Stand Foundation (ALSF)National Brain Tumor Society (NBTS)St. Baldrick's Foundation

people

1
Ashya King