{"entity":{"id":"medulloblastoma","kind":"cancer","name":"Medulloblastoma","aka":[],"tldr":"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 shorter survival; infants and adults), Group 3 (~25%, MYC amplification, worst prognosis) and Group 4 (~35%, most common, intermediate). WHO 2021 integrates histology and molecular group.\n\nStandard 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 causes lasting harm 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.","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Medulloblastoma","links":[{"label":"NCI PDQ: childhood medulloblastoma","url":"https://www.cancer.gov/types/brain/patient/child-medulloblastoma-treatment-pdq"},{"label":"Taylor 2012 consensus subgroups","url":"https://doi.org/10.1007/s00401-011-0922-z"},{"label":"Children's Oncology Group","url":"https://childrensoncologygroup.org/"}],"tags":["gap-fill","paediatric","cns"],"related":["national-brain-tumor-society","atrt","ependymoma","cns-germ-cell-tumours","glioblastoma"],"cancers":[],"sections":[],"technologies":["proton-therapy","imrt-igrt","methylation-profiling","autologous-stem-cell-transplant","cytotoxic-chemotherapy","mri","survivorship-care-plan","antineoplastons"],"targets":["smoothened","tp53"],"drugs":["cisplatin","carboplatin","vincristine","cyclophosphamide","lomustine","methotrexate","temozolomide","irinotecan","vismodegib","carmustine"],"companies":["roche-genentech","childrens-oncology-group"],"institutions":[],"pathways":["wnt","p53-cell-cycle"],"terms":["blood-brain-barrier"],"trials":["ccss","acns0332"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"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)"],"standardOfCare":[{"setting":"Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp)","approach":"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.","refs":["cisplatin","vincristine","cyclophosphamide","lomustine","proton-therapy","imrt-igrt"],"guideline":{"version":"COG ACNS0331; SIOP PNET5","url":"https://doi.org/10.1200/JCO.20.02730"}},{"setting":"High risk (metastatic, residual, anaplastic, MYC)","approach":"CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols.","refs":["carboplatin","cisplatin","cyclophosphamide","autologous-stem-cell-transplant"],"guideline":{"version":"COG ACNS0332"}},{"setting":"Infants (<3 years)","approach":"Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.","refs":["methotrexate","cyclophosphamide","autologous-stem-cell-transplant"]},{"setting":"Relapsed","approach":"Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.","refs":["temozolomide","irinotecan","bevacizumab","vismodegib"]}],"stateOfArt":["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."],"history":[{"year":1925,"title":"Bailey and Cushing name medulloblastoma","refs":[]},{"year":1953,"title":"Craniospinal irradiation introduced (Paterson and Farr)","note":"First cures.","refs":[]},{"year":1990,"title":"Chemotherapy after radiation improves survival (Packer)","refs":["cisplatin","vincristine"]},{"year":2006,"title":"Reduced-dose CSI (23.4 Gy) safe for average risk with chemotherapy (CCG 9892/ACNS0331)","refs":[]},{"year":2012,"title":"Four molecular subgroups agreed (Taylor et al., Acta Neuropathol)","refs":[]},{"year":2016,"title":"WHO integrates molecular groups into classification","refs":[]},{"year":2017,"title":"Proton CSI: equivalent control, fewer toxicities (Yock, Lancet Oncol)","refs":["proton-therapy"]},{"year":2021,"title":"SJMB03 subgroup outcomes; ACNS0331 shows CSI dose reduction below 23.4 Gy unsafe for most","refs":[]}],"pipeline":["proton-therapy","vismodegib","methylation-profiling"],"openProblems":["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."],"parent":"brain-tumours"},"route":"/cancers/medulloblastoma/","neighbours":{"collection":[{"id":"alexs-lemonade-stand","kind":"collection","name":"Alex's Lemonade Stand Foundation (ALSF)","route":"/collections/alexs-lemonade-stand/"},{"id":"national-brain-tumor-society","kind":"collection","name":"National Brain Tumor Society (NBTS)","route":"/collections/national-brain-tumor-society/"},{"id":"st-baldricks","kind":"collection","name":"St. Baldrick's Foundation","route":"/collections/st-baldricks/"}],"cancer":[{"id":"atrt","kind":"cancer","name":"Atypical teratoid/rhabdoid tumour (ATRT)","route":"/cancers/atrt/"},{"id":"brain-tumours","kind":"cancer","name":"Brain and spinal cord tumours (all types)","route":"/cancers/brain-tumours/"},{"id":"cns-germ-cell-tumours","kind":"cancer","name":"Central nervous system germ cell tumours (germinoma and non-germinomatous)","route":"/cancers/cns-germ-cell-tumours/"},{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"ependymoma","kind":"cancer","name":"Ependymoma","route":"/cancers/ependymoma/"},{"id":"glioblastoma","kind":"cancer","name":"Glioma & glioblastoma","route":"/cancers/glioblastoma/"},{"id":"medulloblastoma-group-3-4","kind":"cancer","name":"Group 3 and group 4 medulloblastoma (non-WNT/non-SHH)","route":"/cancers/medulloblastoma-group-3-4/"},{"id":"pineoblastoma","kind":"cancer","name":"Pineoblastoma","route":"/cancers/pineoblastoma/"},{"id":"medulloblastoma-shh","kind":"cancer","name":"SHH-activated medulloblastoma","route":"/cancers/medulloblastoma-shh/"},{"id":"medulloblastoma-wnt","kind":"cancer","name":"WNT-activated medulloblastoma","route":"/cancers/medulloblastoma-wnt/"}],"technology":[{"id":"antineoplastons","kind":"technology","name":"Antineoplastons (Burzynski clinic)","route":"/technologies/antineoplastons/"},{"id":"autologous-stem-cell-transplant","kind":"technology","name":"Autologous stem cell transplant (high-dose therapy)","route":"/technologies/autologous-stem-cell-transplant/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"methylation-profiling","kind":"technology","name":"DNA methylation profiling","route":"/technologies/methylation-profiling/"},{"id":"rejuv-ayac-education-and-work","kind":"technology","name":"Education, work and the years that were interrupted","route":"/technologies/rejuv-ayac-education-and-work/"},{"id":"rejuv-paed-growth-and-height","kind":"technology","name":"Growth and final height after treatment in childhood, and growth hormone","route":"/technologies/rejuv-paed-growth-and-height/"},{"id":"rejuv-paed-hearing","kind":"technology","name":"Hearing after platinum and cranial radiotherapy in a developing child","route":"/technologies/rejuv-paed-hearing/"},{"id":"rejuv-paed-chronic-disease-burden","kind":"technology","name":"How much illness childhood cancer survivors carry, and at what age","route":"/technologies/rejuv-paed-chronic-disease-burden/"},{"id":"imrt-igrt","kind":"technology","name":"IMRT / IGRT (modern external beam)","route":"/technologies/imrt-igrt/"},{"id":"rejuv-paed-late-mortality","kind":"technology","name":"Late deaths after childhood cancer, what causes them, and the proof that gentler treatment worked","route":"/technologies/rejuv-paed-late-mortality/"},{"id":"rejuv-paed-uk-long-term-follow-up","kind":"technology","name":"Long-term follow-up in the United Kingdom: what a survivor is actually offered","route":"/technologies/rejuv-paed-uk-long-term-follow-up/"},{"id":"rejuv-paed-neurocognitive","kind":"technology","name":"Memory, attention and learning after treatment of a childhood cancer","route":"/technologies/rejuv-paed-neurocognitive/"},{"id":"mri","kind":"technology","name":"MRI","route":"/technologies/mri/"},{"id":"proton-therapy","kind":"technology","name":"Proton therapy","route":"/technologies/proton-therapy/"},{"id":"proton-therapy-systems","kind":"technology","name":"Proton therapy machines: cyclotrons, synchrotrons and single-room systems","route":"/technologies/proton-therapy-systems/"},{"id":"rejuv-paed-second-cancers","kind":"technology","name":"Second cancers after childhood cancer: the risk by treatment, and why it is falling","route":"/technologies/rejuv-paed-second-cancers/"},{"id":"survivorship-care-plan","kind":"technology","name":"Survivorship care and late-effects surveillance","route":"/technologies/survivorship-care-plan/"},{"id":"rejuv-paed-teeth-and-face","kind":"technology","name":"Teeth, jaws and facial growth after treatment in childhood","route":"/technologies/rejuv-paed-teeth-and-face/"},{"id":"rejuv-paed-cog-ltfu-guidelines","kind":"technology","name":"The Children's Oncology Group Long-Term Follow-Up Guidelines","route":"/technologies/rejuv-paed-cog-ltfu-guidelines/"},{"id":"rejuv-paed-transition-to-adult-care","kind":"technology","name":"The handover from children's to adult services, where follow-up falls away","route":"/technologies/rejuv-paed-transition-to-adult-care/"},{"id":"rejuv-paed-pituitary-and-puberty","kind":"technology","name":"The pituitary, puberty and the hypothalamic axis after cranial radiotherapy in childhood","route":"/technologies/rejuv-paed-pituitary-and-puberty/"},{"id":"tomotherapy","kind":"technology","name":"TomoTherapy and Radixact (helical radiotherapy)","route":"/technologies/tomotherapy/"}],"target":[{"id":"ccar1","kind":"target","name":"CCAR1","route":"/targets/ccar1/"},{"id":"chd7","kind":"target","name":"CHD7","route":"/targets/chd7/"},{"id":"ddx3x","kind":"target","name":"DDX3X","route":"/targets/ddx3x/"},{"id":"ddx6","kind":"target","name":"DDX6","route":"/targets/ddx6/"},{"id":"elp1","kind":"target","name":"ELP1","route":"/targets/elp1/"},{"id":"fam174b","kind":"target","name":"FAM174B","route":"/targets/fam174b/"},{"id":"foxo3","kind":"target","name":"FOXO3","route":"/targets/foxo3/"},{"id":"gpr161","kind":"target","name":"GPR161","route":"/targets/gpr161/"},{"id":"hip1","kind":"target","name":"HIP1","route":"/targets/hip1/"},{"id":"kat6b","kind":"target","name":"KAT6B","route":"/targets/kat6b/"},{"id":"kdm3b","kind":"target","name":"KDM3B","route":"/targets/kdm3b/"},{"id":"ldb1","kind":"target","name":"LDB1","route":"/targets/ldb1/"},{"id":"prkar1a","kind":"target","name":"PRKAR1A","route":"/targets/prkar1a/"},{"id":"smarcb1","kind":"target","name":"SMARCB1","route":"/targets/smarcb1/"},{"id":"smoothened","kind":"target","name":"Smoothened (hedgehog pathway)","route":"/targets/smoothened/"},{"id":"sufu","kind":"target","name":"SUFU","route":"/targets/sufu/"},{"id":"tcf4","kind":"target","name":"TCF4","route":"/targets/tcf4/"},{"id":"tp53","kind":"target","name":"TP53","route":"/targets/tp53/"},{"id":"tpr","kind":"target","name":"TPR","route":"/targets/tpr/"},{"id":"trim33","kind":"target","name":"TRIM33","route":"/targets/trim33/"},{"id":"was","kind":"target","name":"WAS","route":"/targets/was/"},{"id":"zmym3","kind":"target","name":"ZMYM3","route":"/targets/zmym3/"},{"id":"znf274","kind":"target","name":"ZNF274","route":"/targets/znf274/"}],"drug":[{"id":"bevacizumab","kind":"drug","name":"Bevacizumab","route":"/drugs/bevacizumab/"},{"id":"carboplatin","kind":"drug","name":"Carboplatin","route":"/drugs/carboplatin/"},{"id":"carmustine","kind":"drug","name":"Carmustine","route":"/drugs/carmustine/"},{"id":"cisplatin","kind":"drug","name":"Cisplatin","route":"/drugs/cisplatin/"},{"id":"cyclophosphamide","kind":"drug","name":"Cyclophosphamide","route":"/drugs/cyclophosphamide/"},{"id":"irinotecan","kind":"drug","name":"Irinotecan (and liposomal irinotecan)","route":"/drugs/irinotecan/"},{"id":"lomustine","kind":"drug","name":"Lomustine (CCNU)","route":"/drugs/lomustine/"},{"id":"methotrexate","kind":"drug","name":"Methotrexate","route":"/drugs/methotrexate/"},{"id":"sodium-thiosulfate","kind":"drug","name":"Sodium thiosulfate (otoprotectant)","route":"/drugs/sodium-thiosulfate/"},{"id":"temozolomide","kind":"drug","name":"Temozolomide","route":"/drugs/temozolomide/"},{"id":"vincristine","kind":"drug","name":"Vincristine","route":"/drugs/vincristine/"},{"id":"vismodegib","kind":"drug","name":"Vismodegib","route":"/drugs/vismodegib/"}],"company":[{"id":"childrens-oncology-group","kind":"company","name":"Children's Oncology Group (COG)","route":"/companies/childrens-oncology-group/"},{"id":"roche-genentech","kind":"company","name":"Roche / Genentech","route":"/companies/roche-genentech/"}],"pathway":[{"id":"p53-cell-cycle","kind":"pathway","name":"p53 / RB / cell-cycle checkpoint","route":"/pathways/p53-cell-cycle/"},{"id":"wnt","kind":"pathway","name":"Wnt / β-catenin","route":"/pathways/wnt/"}],"term":[{"id":"blood-brain-barrier","kind":"term","name":"Blood-brain barrier (BBB)","route":"/terms/blood-brain-barrier/"},{"id":"li-fraumeni","kind":"term","name":"Li-Fraumeni syndrome (germline TP53)","route":"/terms/li-fraumeni/"},{"id":"medulloblastoma-molecular-groups","kind":"term","name":"Medulloblastoma molecular groups (WNT, SHH, group 3, group 4)","route":"/terms/medulloblastoma-molecular-groups/"}],"trial":[{"id":"nct05382338","kind":"trial","name":"A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss","route":"/trials/nct05382338/"},{"id":"acns1422","kind":"trial","name":"ACNS1422","route":"/trials/acns1422/"},{"id":"ccss","kind":"trial","name":"Childhood Cancer Survivor Study (CCSS)","route":"/trials/ccss/"},{"id":"acns0331","kind":"trial","name":"COG ACNS0331","route":"/trials/acns0331/"},{"id":"acns0332","kind":"trial","name":"COG ACNS0332","route":"/trials/acns0332/"},{"id":"sjmb12","kind":"trial","name":"SJMB12","route":"/trials/sjmb12/"},{"id":"nct03709680","kind":"trial","name":"Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors","route":"/trials/nct03709680/"}],"pairing":[{"id":"proton-vs-imrt","kind":"pairing","name":"Caution: proton therapy vs IMRT","route":"/pairings/proton-vs-imrt/"}],"person":[{"id":"amar-gajjar","kind":"person","name":"Amar Gajjar","route":"/people/amar-gajjar/"},{"id":"ashya-king","kind":"person","name":"Ashya King","route":"/people/ashya-king/"},{"id":"brandon-wainwright","kind":"person","name":"Brandon Wainwright","route":"/people/brandon-wainwright/"},{"id":"giles-robinson","kind":"person","name":"Giles W. Robinson","route":"/people/giles-robinson/"},{"id":"louis-chesler","kind":"person","name":"Louis Chesler","route":"/people/louis-chesler/"},{"id":"michael-taylor","kind":"person","name":"Michael D. Taylor","route":"/people/michael-taylor/"},{"id":"sarah-leary","kind":"person","name":"Sarah E. S. Leary","route":"/people/sarah-leary/"},{"id":"steven-clifford","kind":"person","name":"Steven Clifford","route":"/people/steven-clifford/"}],"paper":[{"id":"paper-singh-brain-tumour-initiating-cells-nature-2004","kind":"paper","name":"Singh 2004: identification of human brain tumour initiating cells","route":"/key-papers/paper-singh-brain-tumour-initiating-cells-nature-2004/"}],"institution":[{"id":"juravinski","kind":"institution","name":"Juravinski Cancer Centre / Escarpment Cancer Research Institute","route":"/institutions/juravinski/"},{"id":"nationwide-childrens","kind":"institution","name":"Nationwide Children's Hospital","route":"/institutions/nationwide-childrens/"},{"id":"seattle-childrens","kind":"institution","name":"Seattle Children's Hospital","route":"/institutions/seattle-childrens/"},{"id":"st-jude","kind":"institution","name":"St. Jude Children's Research Hospital","route":"/institutions/st-jude/"},{"id":"texas-childrens","kind":"institution","name":"Texas Children's Cancer and Hematology Center","route":"/institutions/texas-childrens/"},{"id":"sickkids","kind":"institution","name":"The Hospital for Sick Children (SickKids)","route":"/institutions/sickkids/"},{"id":"uf-health-cancer-center","kind":"institution","name":"University of Florida Health Cancer Center","route":"/institutions/uf-health-cancer-center/"}],"journal":[{"id":"brain-tumor-pathology","kind":"journal","name":"Brain tumor pathology","route":"/journals/brain-tumor-pathology/"},{"id":"cns-oncology","kind":"journal","name":"CNS oncology","route":"/journals/cns-oncology/"},{"id":"journal-of-neuro-oncology","kind":"journal","name":"Journal of neuro-oncology","route":"/journals/journal-of-neuro-oncology/"}]}}