Target landscape
Open Targets · retrieved 2026-09-12 · weekly12 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.
| Gene | Drugs | Approval, and whether it reached this disease | Open Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it works | Cell therapy here |
|---|---|---|---|---|
| SMO | 8 | 3 approvedGlasdegib, Sonidegib, VismodegibApproved 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 | 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 | 0 | No drug— | protein degrader, small molecule | — |
| MYCN | 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 | 0 | No drug— | antibody, protein degrader, small molecule | — |
| OTX2 | 0 | No drug— | — | — |
| DDX3X | 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 2026-09-12. 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 · weekly1 drug carries an FDA label naming medulloblastoma: Carmustine. Separately, 3 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.
Every label that names medulloblastoma
| Drug | Role | What the label says |
|---|---|---|
| CarmustineCARMUSTINE, Carmustine, carmustine | Labelled here | Brain tumors glioblastoma, brainstem glioma, medulloblastoma, astrocytoma, ependymoma, and metastatic brain tumors ( 1 ) 2. |
20 labels match 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 2026-09-12. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy against B7-H3
ClinicalTrials.gov · retrieved 2026-09-12 · weeklyB7-H3 is the busiest cell-therapy antigen in medulloblastoma: 3 registered trials, 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.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT04185038 | 1 | Recruiting | Study 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 Tumors | 2026-04-13 |
| NCT07390539 | 1 | Recruiting | B7-H3.CD28Z.CART in CNS Neoplasms | 2026-09-03 |
| NCT05835687 | 1 | Active | Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors | 2026-07-06 |
3 of 3 shown, most recently active first. Other antigens searched: GD2 (1), HER2 (1), IL13RA2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-12. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.
Research momentum
PubMed MeSH · monthlyEvery 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 term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Tumor Microenvironment | 1.22% | 5.31% | 4.34× | 50 papers |
| Postoperative Complications | 1.22% | 3.72% | 3.04× | 35 papers |
| Central Nervous System Neoplasms | 0.68% | 1.28% | 1.88× | 12 papers |
| Hydrocephalus | 0.82% | 1.49% | 1.82× | 14 papers |
| Transcriptome | 1.9% | 3.29% | 1.73× | 31 papers |
| Quality of Life | 1.77% | 2.76% | 1.56× | 26 papers |
| Biomarkers | 1.22% | 1.81% | 1.48× | 17 papers |
| Brain Neoplasms | 10.87% | 15.09% | 1.39× | 142 papers |
| Cerebellar Neoplasms | 70.65% | 94.58% | 1.34× | 890 papers |
| Hedgehog Proteins | 13.86% | 18.49% | 1.33× | 174 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Immunohistochemistry | 4.89% | 1.59% | 0.33× | 15 papers |
| Mice, Nude | 5.71% | 1.91% | 0.34× | 18 papers |
| Cell Survival | 4.21% | 1.59% | 0.38× | 15 papers |
| Signal Transduction | 16.3% | 7.44% | 0.46× | 70 papers |
| Brain | 3.26% | 1.49% | 0.46× | 14 papers |
| Cranial Irradiation | 2.72% | 1.28% | 0.47× | 12 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Cerebellar Neoplasms | 70.65% | 94.58% | 1.34× | 890 papers |
| Hedgehog Proteins | 13.86% | 18.49% | 1.33× | 174 papers |
| Brain Neoplasms | 10.87% | 15.09% | 1.39× | 142 papers |
| Prognosis | 11.96% | 14.03% | 1.17× | 132 papers |
| Cell Proliferation | 17.66% | 9.56% | 0.54× | 90 papers |
| Gene Expression Regulation, Neoplastic | 13.32% | 9.03% | 0.68× | 85 papers |
| Signal Transduction | 16.3% | 7.44% | 0.46× | 70 papers |
| Neoplasm Recurrence, Local | 6.25% | 6.91% | 1.11× | 65 papers |
| Biomarkers, Tumor | 7.88% | 5.95% | 0.76× | 56 papers |
| Magnetic Resonance Imaging | 8.15% | 5.95% | 0.73× | 56 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Randomized Controlled Trial | 0.7% | 0.6% |
| Review | 10.9% | 9.9% |
| Meta-Analysis | 0.7% | 0.6% |
| Case Reports | 0.0% | 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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | 431 cases/year | direct | 2017-2021 |
| Incidence rate | 0.47 cases per 100,000 children (0-14) per year | direct | 2017-2021 |
| Deaths each year | — | not published | CBTRUS 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 published | The 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 published | Not 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 · quarterlyNIH obligations naming medulloblastoma, after removing the 829 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.
NIH obligations by fiscal year
| Year | Obligations | Awards | Distinct projects | Dropped as off-topic |
|---|---|---|---|---|
| FY2013 | $10.7M | 36 | 32 | 60 |
| FY2014 | $7.9M | 28 | 27 | 56 |
| FY2015 | $13.2M | 37 | 34 | 59 |
| FY2016 | $12.6M | 32 | 30 | 67 |
| FY2017 | $13.8M | 37 | 34 | 58 |
| FY2018 | $14.7M | 40 | 38 | 60 |
| FY2019 | $16.3M | 44 | 39 | 60 |
| FY2020 | $13.1M | 37 | 36 | 55 |
| FY2021 | $15.2M | 42 | 39 | 65 |
| FY2022 | $18.0M | 50 | 43 | 75 |
| FY2023 | $19.1M | 45 | 41 | 81 |
| FY2024 | $19.6M | 46 | 42 | 67 |
| FY2025 | $20.2M | 45 | 42 | 66 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| St. Jude Children'S Research Hospital | $3.7M | 8 |
| University Of California, San Francisco | $1.5M | 3 |
| Stanford University | $1.4M | 0 |
| Columbia University Health Sciences | $1.3M | 2 |
| University Of Tx Md Anderson Can Ctr | $0.9M | 2 |
| University Of Nebraska Medical Center | $0.9M | 3 |
| Research Inst Of Fox Chase Can Ctr | $0.8M | 2 |
| Medical University Of South Carolina | $0.8M | 2 |
| New York University School Of Medicine | $0.7M | 1 |
| Sanford Burnham Prebys Medical Discovery Institute | $0.6M | 0 |
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 2026-09-12.
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
Projects by administering institute. The rows above are the top 3 and account for 45 of 45.
Award mechanisms
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 45 of 45.
Where it lands
Share of $20.2M in FY2025. The top three hold 32%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly435 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
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE85218 | DNA methylation and gene expression profiling of primary medulloblastoma samples2017 · methylation | 1,526 | 158 | 31.0 | patient cohortAPT 0.95970 cites |
| GSE37418 | Novel mutations target distinct subgroups of medulloblastoma.2012 · array | 76 | 74 | 16.2 | cell lineAPT 0.95molecularCTNNB1DDX3X716 cites |
| GSE49243 | Gene expression data from medulloblastoma tumor samples2014 · array | 73 | 32 | 16.0 | patient cohortAPT 0.95molecularSMOSMOOTHENED616 cites |
| GSE93646 | Microarray-based DNA methylation profiles of primary medulloblastomas2017 · methylation | 428 | 7 | 14.0 | patient cohortAPT 0.95survivalstagemolecularSMO423 cites |
| GSE21140 | Genomics of medulloblastoma identifies four distinct molecular variants2010 · array | 103 | 25 | 24.9 | patient cohortAPT 0.95survival1,052 cites |
| GSE37385 | Subgroup specific somatic copy number aberrations in the medulloblastoma genome2012 · array | 1,382 | 27 | 17.3 | APT 0.95molecularMYC740 cites |
| GSE119926 | Single cell RNA-seq analysis of medulloblastoma2019 · single-cell | 36 | 31 | 9.7 | patient cohortAPT 0.95318 cites |
| GSE12992 | Beta-catenin status in pediatric medulloblastomas2009 · array | 40 | 23 | 3.3 | patient cohortAPT 0.95survivalmolecularCTNNB1156 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE202043 | Integrative Genomic Analysis of Medulloblastoma Identifies a Molecular Subgroup That Drives Poor Clinical Outcome2022 · array | 214 | 4 | 12.9 | patient cohortAPT 0.95survivalmolecularMYC568 cites |
| GSE337222 | International Phase III Medulloblastoma Trial (SJMB03)2026 · methylation | 310 | 0 | 11.5 | patient cohortAPT 0.95survivalstagemolecularERBB2199 cites |
| GSE189919 | Medulloblastoma cerebrospinal fluid reveals hypoxic indicators (metabolites and lipids) and cancer-specific RNAs2022 · sequencing | 51 | 2 | 3.8 | patient cohortAPT 0.75survival50 cites |
| GSE207266 | Unified rhombic lip origins of Group 3 and Group 4 medulloblastoma2022 · chromatin | 68 | 4 | 7.6 | APT 0.95127 cites |
| GSE294901 | Measles oncolytic virus as an immunotherapy for recurrent/refractory pediatric medulloblastoma and atypical teratoid rhabdoid tumor: results from PNOC0052025 · single-cell | 84 | 1 | 2.6 | patient cohortAPT 0.75survival10 cites |
| GSE239854 | Single nuclei RNA-seq profiles from medulloblastoma tumors with extensive nodularity2023 · methylation | 55 | 3 | 2.9 | patient cohortAPT 0.7513 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. 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 refreshEach 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
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
MYC
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
MYCN
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
KMT2D
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
OTX2
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
DDX3X
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
KBTBD4
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
GFI1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
PRDM6
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
How a machine reads this page
StaticThis 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.
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 (2026-09-12), ClinicalTrials.gov (2026-09-12), openFDA (2026-09-12), NCBI GEO (2026-09-12), PubMed (2026-09-12), NIH RePORTER (2026-09-12).
Negatives stated explicitly
Where nothing exists for medulloblastoma — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated 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/",
"note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}