Disease Briefing

Medulloblastoma: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-30Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 222 studies · 13,524 GEO samples
Page Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in medulloblastoma — SMO, PTCH1, CTNNB1, MYC, MYCN, TP53, KMT2D, OTX2, DDX3X, KBTBD4, GFI1, PRDM6 — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

1
drugs carry an FDA label naming medulloblastoma: Carmustine. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
0
of the 12 genes above carries a drug approved in medulloblastoma — 20 drug entries reach them, 17 distinct once salt forms are merged, and 2 of those are approved for other indications. A statement about these gene targets, not about the disease
3
registered B7-H3 cell-therapy trials in medulloblastoma, 3 active. Counted from ClinicalTrials.gov across 3 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
10
targets carry an Open Targets tractability signal and no trial in medulloblastoma that the searches found (no trial naming a drug Open Targets lists for the gene, none naming it as a cell-therapy target): PTCH1, CTNNB1, MYC, MYCN, TP53, KMT2D, DDX3X, KBTBD4, GFI1, PRDM6
435
human GEO series match the disease; 222 survive on-topic filtering, and only 14 are patient cohorts of 100+ samples
158
Europe PMC full-text papers name GSE85218 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$19.9M
NIH obligations in FY2025, up 86% since 2013 — while distinct core projects went 32 to 41. Larger awards rather than more distinct core projects; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-30 · weekly

12 genes recurrently implicated in medulloblastoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 3 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Medulloblastoma does have labelled therapy — 1 drugs, listed in the next section.

Two different questions

The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what medulloblastoma is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell therapy here
SMO across cancers → 8 2 approvedGlasdegib, SonidegibApproved in juvenile myelomonocytic leukemia, acute myeloid leukemia, basal cell carcinoma. No medulloblastoma indication appears on these drugs’ labels.1 active of 14 medulloblastoma trials antibody, protein degrader, small molecule —
PTCH1 across cancers → 0 No drug— antibody, protein degrader, small molecule —
CTNNB1 across cancers → 1 Phase 2Pri-724No medulloblastoma trial of any of these drugs antibody, other clinical modality, protein degrader, small molecule —
MYC across cancers → 0 No drug— protein degrader, small molecule —
MYCN across cancers → 0 No drug— protein degrader, small molecule —
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo medulloblastoma trial of any of these drugs other clinical modality, protein degrader, small molecule —
KMT2D across cancers → 0 No drug— antibody, protein degrader, small molecule —
OTX2 0 No drug— — —
DDX3X across cancers → 0 No drug— antibody, protein degrader, small molecule —
KBTBD4 0 No drug— protein degrader, small molecule —
GFI1 0 No drug— protein degrader —
PRDM6 0 No drug— protein degrader —

Dataset evidence counts studies in the 222-study ranked set whose title or abstract names the gene; the bar is scaled to MYC. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-30. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

What is actually approved here

openFDA drug labels · weekly

1 drug carries an FDA label naming medulloblastoma: Carmustine. Separately, 2 of the drugs returned for the genes in the table above are approved only for other diseases and reach medulloblastoma through trials, not through their labels.

1Labelled for medulloblastomaFDA INDICATIONS AND USAGE names the disease
2Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
3Active cell-therapy trials naming B7-H3of 3 registered

Every label that names medulloblastoma

DrugRoleWhat the label says
CarmustineCARMUSTINE, Carmustine, carmustineLabelled hereBrain tumors glioblastoma, brainstem glioma, medulloblastoma, astrocytoma, ependymoma, and metastatic brain tumors ( 1 ) 2.

13 labels carry a positive indication naming the disease under indications_and_usage:"medulloblastoma"; they collapse to 1 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-18. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against B7-H3

ClinicalTrials.gov · retrieved 2026-09-18 · weekly

B7-H3 is the busiest cell-therapy target among those this briefing searched in medulloblastoma: 3 registered trials naming it, 3 still active. It has no gene entry of its own and is reached through CD276: the immune-checkpoint protein CD276 encodes, B7-H3, the intraventricular antibody target.

TrialPhaseStatusTitleLast update
NCT041850381RecruitingStudy of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma/Diffuse Midline Glioma and Recurrent or Refractory Pediatric Central Nervous System Tumors2026-04-13
NCT073905391RecruitingB7-H3.CD28Z.CART in CNS Neoplasms2026-09-03
NCT058356871ActiveLoc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors2026-07-06

3 of 3 shown, most recently active first. Other targets searched: GD2 (1), HER2 (1), IL13RA2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-18. A trial counts here when its registered conditions name medulloblastoma or a tumour-agnostic basket, its title names an administered cell product, and the title names this target: a HER2 CAR-T trial that adds a PD-1 antibody is a HER2 trial. A trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the medulloblastoma literature, not a count, because the field itself grew: 2015–2018 (n=736) against 2021–2025 (n=941). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Tumor Microenvironment1.22%5.31%4.35×50 papers
Postoperative Complications1.22%3.72%3.04×35 papers
Central Nervous System Neoplasms0.68%1.28%1.88×12 papers
Hydrocephalus0.82%1.49%1.83×14 papers
Transcriptome1.9%3.29%1.73×31 papers
Quality of Life1.77%2.76%1.56×26 papers
Biomarkers1.22%1.81%1.48×17 papers
Brain Neoplasms10.87%15.09%1.39×142 papers
Cerebellar Neoplasms70.65%94.58%1.34×890 papers
Hedgehog Proteins13.86%18.49%1.33×174 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Immunohistochemistry4.89%1.59%0.33×15 papers
Mice, Nude5.71%1.91%0.34×18 papers
Cell Survival4.21%1.59%0.38×15 papers
Signal Transduction16.3%7.44%0.46×70 papers
Brain3.26%1.49%0.46×14 papers
Cranial Irradiation2.72%1.28%0.47×12 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Cerebellar Neoplasms70.65%94.58%1.34×890 papers
Hedgehog Proteins13.86%18.49%1.33×174 papers
Brain Neoplasms10.87%15.09%1.39×142 papers
Prognosis11.96%14.03%1.17×132 papers
Cell Proliferation17.66%9.56%0.54×90 papers
Gene Expression Regulation, Neoplastic13.32%9.03%0.68×85 papers
Signal Transduction16.3%7.44%0.46×70 papers
Neoplasm Recurrence, Local6.25%6.91%1.11×65 papers
Biomarkers, Tumor7.88%5.95%0.76×56 papers
Magnetic Resonance Imaging8.15%5.95%0.73×56 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial0.7%0.6%
Review10.9%9.9%
Meta-Analysis0.7%0.6%
Case Reports0.0%1.6%

Query: Medulloblastoma[MeSH Major Topic] NOT ("Ependymoma"[MeSH] OR "Rhabdoid Tumor"[MeSH] OR "Glioblastoma"[MeSH] OR "Neuroblastoma"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-12.

Disease burden

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

MeasureValueMatchWhat it counts
New cases each year431 cases/yeardirect2017-2021
Incidence rate0.47 cases per 100,000 children (0-14) per yeardirect2017-2021
Deaths each year—not publishedCBTRUS reports mortality for malignant brain and CNS tumours as a whole, not for medulloblastoma; SEER has no page for it.
Five-year relative survival—not publishedThe CBTRUS 2024 report gives five-year relative survival for embryonal tumours as a group (64.6% in children 0-14, 2001-2020) and not for medulloblastoma alone; a group figure that includes ATRT would understate it.
Median age at diagnosis—not publishedNot stated in the report's text for medulloblastoma as a whole; subtype medians are given (WNT 11 years, SHH TP53-wildtype 19 and 8 years).

Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming medulloblastoma, after removing the 827 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

$19.9MNIH obligations, FY2025from $10.7M in FY2013 · +86%
41distinct projects funded32 in FY2013
$19.9Mpeak year was FY2025obligations, all institutes
52%of FY2025 awards from NCI23 of 44

NIH obligations by fiscal year

$5M$10M$15M$20M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$10.7M363260
FY2014$7.9M282756
FY2015$13.2M373459
FY2016$12.6M323067
FY2017$13.8M373458
FY2018$14.7M403860
FY2019$16.3M443960
FY2020$13.1M373655
FY2021$14.5M413865
FY2022$18.0M504374
FY2023$19.1M454181
FY2024$19.3M454166
FY2025$19.9M444166

Where FY2025 money went

InstitutionObligationsAwards
St. Jude Children'S Research Hospital$3.7M8
University Of California, San Francisco$1.5M3
Stanford University$1.4M0
Columbia University Health Sciences$1.3M2
University Of Tx Md Anderson Can Ctr$0.9M2
University Of Nebraska Medical Center$0.9M3
Research Inst Of Fox Chase Can Ctr$0.8M2
Medical University Of South Carolina$0.8M2
New York University School Of Medicine$0.7M1
Sanford Burnham Prebys Medical Discovery Institute$0.6M0

Text search medulloblastoma over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-19.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

NIH institutes

NCI23 · 52%
NINDS21 · 48%

Projects by administering institute. The rows above are the top 2 and account for 44 of 44.

Award mechanisms

R0126 · 59%
R214 · 9%
P014 · 9%
F312 · 5%
U012 · 5%
K081 · 2%

R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 11 and account for 44 of 44.

Where it lands

St. Jude Children'S Research Hospital$3.7M · 18.7%
University Of California, San Francisco$1.5M · 7.3%
Stanford University$1.4M · 7.0%
Columbia University Health Sciences$1.3M · 6.4%
University Of Tx Md Anderson Can Ctr$0.9M · 4.7%
University Of Nebraska Medical Center$0.9M · 4.6%

Share of $19.9M in FY2025. The top three hold 33%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

435 human GEO series match medulloblastoma. Keyword relevance cannot tell a 1,526-sample patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Composition of the 222-study ranked set

patient 79unspecified 66cell line 45mixed 29xenograft 3
79 patient66 unspecified45 cell line29 mixed3 xenograft125 carry clinical annotation71 carry survival14 patient cohorts ≥100 GEO samples13,524 GEO samples totalin 9 single-cell series the GEO sample count is the submitter’s unit (cells, libraries or pools), not patients

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE85218DNA methylation and gene expression profiling of primary medulloblastoma samples2017 · methylation1,52615831.0
patient cohortAPT 0.95970 cites
GSE37418Novel mutations target distinct subgroups of medulloblastoma.2012 · array767416.2
patient cohortAPT 0.95molecularCTNNB1DDX3X716 cites
GSE37385Subgroup specific somatic copy number aberrations in the medulloblastoma genome2012 · array1,3822717.3
patient cohortAPT 0.95molecularMYC740 cites
GSE49243Gene expression data from medulloblastoma tumor samples2014 · array733216.0
patient cohortAPT 0.95molecularSMOSMOOTHENED616 cites
GSE93646Microarray-based DNA methylation profiles of primary medulloblastomas2017 · methylation428714.0
patient cohortAPT 0.95survivalstagemolecularSMO423 cites
GSE21140Genomics of medulloblastoma identifies four distinct molecular variants2010 · array1032524.9
patient cohortAPT 0.95survival1,052 cites
GSE10327mRNA expression data of 62 human medulloblastoma tumors2008 · array625711.0
patient cohortAPT 0.95555 cites
GSE119926Single cell RNA-seq analysis of medulloblastoma2019 · single-cell36319.7
patient cohortAPT 0.95318 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE202043Integrative Genomic Analysis of Medulloblastoma Identifies a Molecular Subgroup That Drives Poor Clinical Outcome2022 · array214412.9
patient cohortAPT 0.95survivalmolecularMYC568 cites
GSE337222International Phase III Medulloblastoma Trial (SJMB03)2026 · methylation310011.5
patient cohortAPT 0.95survivalstagemolecularERBB2199 cites
GSE253557Single-nuclei RNA-Seq profiles from Group 3 and Group 4 medulloblastomas [scRNA-Seq]2025 · single-cell3945.8
patient cohortAPT 0.7525 cites
GSE189919Medulloblastoma cerebrospinal fluid reveals hypoxic indicators (metabolites and lipids) and cancer-specific RNAs2022 · sequencing5123.8
patient cohortAPT 0.75survival50 cites
GSE207266Unified rhombic lip origins of Group 3 and Group 4 medulloblastoma2022 · chromatin6847.6
APT 0.95127 cites
GSE294901Measles oncolytic virus as an immunotherapy for recurrent/refractory pediatric medulloblastoma and atypical teratoid rhabdoid tumor: results from PNOC0052025 · single-cell8412.6
patient cohortAPT 0.75survival10 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-19. SubSeries are collapsed to one row per study by linked PMID. Of 435 series retrieved, 29 were dropped by the profile’s exclusion rules and 99 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Where the gaps are

Derived from the sections above · recomputed on every refresh

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

PTCH1

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

MYC

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

MYCN

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

KMT2D

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

OTX2

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

DDX3X

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

KBTBD4

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

GFI1

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

PRDM6

Open Targets lists no drug against it and no trial in medulloblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.

How a machine reads this page

Static

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

Fully server-rendered

Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.

Every number carries a source

Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-30), ClinicalTrials.gov (2026-09-18), openFDA (2026-09-18), NCBI GEO (2026-09-19), PubMed (2026-09-12), NIH RePORTER (2026-09-19).

Negatives stated explicitly

Where the searches find nothing for medulloblastoma — no drug, no trial, no approval — the page says so in those words rather than omitting the row, and names the source searched. An absent row is unquotable; a bounded negative is the finding.

Denominators, not just percentages

Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.

The filters are published

3 exclusion patterns are applied to free text before anything is ranked, because DAOY is used as a model system in none of consequence: DAOY is used for the disease, and "Daoy" is also a surname. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Medulloblastoma",
  "mesh": "Medulloblastoma",
  "facts": "https://usebiotransfer.org/disease/medulloblastoma.json",
  "methods": "https://usebiotransfer.org/methods/",
  "all_diseases": "https://usebiotransfer.org/disease/api.json",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}