Target landscape
Open Targets · retrieved 2026-09-11 · weekly12 genes recurrently implicated in glioblastoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.
Of 12 recurrently implicated genes, 7 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Glioblastoma does have labelled therapy — 3 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 glioblastoma 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 |
|---|---|---|---|---|
| EGFR | 74 | 23 approvedAfatinib, Afatinib Dimaleate, Amivantamab, Aumolertinib, Brigatinib, Cetuximab, Cetuximab Sarotalocan, Dacomitinib, Erlotinib, Gefitinib, Icotinib, Lapatinib, Lapatinib Ditosylate, Lazertinib, Mobocertinib, Necitumumab, Neratinib, Nimotuzumab, Olmutinib, Osimertinib, Panitumumab, Rociletinib, VandetanibApproved in non-small cell lung carcinoma, anaplastic large cell lymphoma, malignant tumor of neck and 14 other indications. No glioblastoma indication appears on these drugs’ labels.13 active of 112 glioblastoma trials | antibody, other clinical modality, protein degrader, small molecule | 6 active of 14 trials |
| PDGFRA | 32 | 14 approvedAvapritinib, Becaplermin, Cediranib, Masitinib, Midostaurin, Nintedanib, Nintedanib Esylate, Olaratumab, Pazopanib, Quizartinib, Regorafenib, Ripretinib, Sunitinib, Sunitinib MalateApproved in gastrointestinal stromal tumor, Skin ulcer, mast cell leukemia and 12 other indications. No glioblastoma indication appears on these drugs’ labels.4 active of 64 glioblastoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| PTEN | 0 | No drug— | antibody, protein degrader, small molecule | — |
| TP53 | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, Teprasiran4 trials, none active | other clinical modality, protein degrader, small molecule | — |
| NF1 | 0 | No drug— | antibody, protein degrader, small molecule | — |
| CDK4 | 15 | 4 approvedAbemaciclib, Palbociclib, Ribociclib, TrilaciclibApproved in breast cancer, breast neoplasm, breast carcinoma and 2 other indications. No glioblastoma indication appears on these drugs’ labels.6 active of 17 glioblastoma trials | protein degrader, small molecule | — |
| MDM2 | 4 | Phase 3Alrizomadlin, Idasanutlin, Navtemadlin, Siremadlin2 trials, none active | antibody, protein degrader, small molecule | — |
| CDKN2A | 0 | No drug— | — | — |
| RB1 | 0 | No drug— | protein degrader, small molecule | — |
| PIK3CA | 31 | 3 approvedAlpelisib, Copanlisib, InavolisibApproved in breast cancer, breast neoplasm, breast carcinoma and 3 other indications. No glioblastoma indication appears on these drugs’ labels.3 active of 17 glioblastoma trials | antibody, protein degrader, small molecule | — |
| TERT | 1 | 1 approvedImetelstatApproved in anemia, myelodysplastic syndrome. No glioblastoma indication appears on these drugs’ labels.2 trials, none active | antibody, other clinical modality, protein degrader, small molecule | — |
| MGMT | 0 | No drug— | protein degrader, small molecule | — |
Dataset evidence counts studies in the 341-study ranked set whose title or abstract names the gene; the bar is scaled to EGFR. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-11. 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 · weekly3 drugs carry an FDA label naming glioblastoma: Bevacizumab, Carmustine, Temozolomide. Separately, 45 of the drugs returned for the genes in the table above are approved only for other diseases and reach glioblastoma through trials, not through their labels.
Every label that names glioblastoma
| Drug | Role | What the label says |
|---|---|---|
| BevacizumabALYMSYS, Avastin, JOBEVNE | Labelled here | Recurrent glioblastoma in adults. |
| CarmustineCARMUSTINE, Carmustine, Gliadel | Labelled here | Brain tumors glioblastoma, brainstem glioma, medulloblastoma, astrocytoma, ependymoma, and metastatic brain tumors ( 1 ) 2. |
| TemozolomideTEMODAR, TEMOZOLOMIDE, Temozolomide | Labelled here | Newly Diagnosed Glioblastoma TEMODAR is indicated for the treatment of adults with newly diagnosed glioblastoma, concomitantly with radiotherapy and then as maintenance treatment. |
72 labels match indications_and_usage:"glioblastoma"; they collapse to 3 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-11. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy against EGFRvIII
ClinicalTrials.gov · retrieved 2026-09-11 · weeklyEGFRvIII is the busiest cell-therapy antigen in glioblastoma: 14 registered trials, 6 still active. It has no gene entry of its own and is reached through EGFR: a deletion-derived mutant form of EGFR (exons 2-7 lost) rather than a separate gene, so gene-centric resources map it through EGFR.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT07480941 | 1 | Recruiting | Dual-Targeting CAR-NK Cells for Recurrent/Progressive Glioblastoma and High-Grade Glioma | 2026-03-18 |
| NCT07523529 | 1/2 | Recruiting | Biomarker-Guided Dual-Target CAR-T Cells for Advanced Solid Tumors | 2026-04-13 |
| NCT07551336 | 1 | Recruiting | Dual-Targeting CAR-NK Cells for Recurrent/Progressive Glioblastoma and High-Grade Glioma | 2026-04-24 |
| NCT05660369 | 1 | Recruiting | CARv3-TEAM-E T Cells in Glioblastoma | 2026-07-14 |
| NCT06186401 | 1 | Recruiting | Anti-EGFRvIII synNotch Receptor Induced Anti-EphA2/IL-13Ralpha2 CAR (E-SYNC) T Cells | 2026-08-13 |
| NCT07244666 | EARLY/1 | Not yet recruiting | Safety and Preliminary Efficacy of a Metabolically Armed Chimeric Antigen Receptor T Cell Therapy Targeting EGFRvIII for Recurrent Glioblastoma | 2025-11-24 |
| NCT02844062 | 1 | Unknown | Pilot Study of Autologous Anti-EGFRvIII CAR T Cells in Recurrent Glioblastoma Multiforme | 2016-07-26 |
| NCT01454596 | 1/2 | Completed | CAR T Cell Receptor Immunotherapy Targeting EGFRvIII for Patients With Malignant Gliomas Expressing EGFRvIII | 2019-08-21 |
| NCT03283631 | 1 | Terminated | Intracerebral EGFR-vIII CAR-T Cells for Recurrent GBM | 2021-04-05 |
| NCT05063682 | 1 | Unknown | The Efficacy and Safety of Brain-targeting Immune Cells (EGFRvIII-CAR T Cells) in Treating Patients With Leptomeningeal Disease From Glioblastoma. Administering Patients EGFRvIII -CAR T Cells May Help to Recognize and Destroy Brain Tumor Cells in Patients | 2021-10-01 |
10 of 14 shown, most recently active first. Other antigens searched: IL13RA2 (11), B7-H3 (11), HER2 (3), CD70 (3), GD2 (2), EphA2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-11. 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 glioblastoma literature, not a count, because the field itself grew: 2015–2018 (n=6,000) against 2021–2025 (n=6,000). A topic whose papers doubled while the field doubled has not risen.
These are sample shares. The two windows hold 6,166 and 10,350 papers; MeSH terms were read from an evenly spaced sample of 6,000 and 6,000 of them, taken across the whole window rather than from its most recent papers. Percentages carry sampling error of roughly a percentage point and small differences between two terms are not meaningful.
Rising, with a real baseline
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Neural Networks, Computer | 0.1% | 0.65% | 6.49× | 39 papers |
| Receptors, Chimeric Antigen | 0.23% | 1.33% | 5.71× | 80 papers |
| Methyltransferases | 0.08% | 0.45% | 5.39× | 27 papers |
| Adenosine | 0.1% | 0.52% | 5.16× | 31 papers |
| Molecular Docking Simulation | 0.23% | 1.2% | 5.14× | 72 papers |
| Tumor Microenvironment | 2.98% | 15.07% | 5.05× | 904 papers |
| Biomimetics | 0.08% | 0.42% | 5.0× | 25 papers |
| CRISPR-Cas Systems | 0.08% | 0.4% | 4.8× | 24 papers |
| Nomograms | 0.1% | 0.45% | 4.5× | 27 papers |
| Iron | 0.08% | 0.37% | 4.4× | 22 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| RNA Interference | 2.47% | 0.25% | 0.1× | 15 papers |
| Immunohistochemistry | 2.95% | 0.3% | 0.1× | 18 papers |
| Dacarbazine | 9.6% | 1.4% | 0.15× | 84 papers |
| Time Factors | 2.43% | 0.37% | 0.15× | 22 papers |
| Proportional Hazards Models | 2.27% | 0.33% | 0.15× | 20 papers |
| Gene Knockdown Techniques | 2.43% | 0.4% | 0.16× | 24 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Brain Neoplasms | 68.98% | 68.5% | 0.99× | 4110 papers |
| Tumor Microenvironment | 2.98% | 15.07% | 5.05× | 904 papers |
| Cell Proliferation | 19.18% | 13.9% | 0.72× | 834 papers |
| Gene Expression Regulation, Neoplastic | 16.82% | 13.2% | 0.78× | 792 papers |
| Prognosis | 13.33% | 12.2% | 0.92× | 732 papers |
| Temozolomide | 11.63% | 11.88% | 1.02× | 713 papers |
| Glioma | 2.65% | 10.57% | 3.99× | 634 papers |
| Signal Transduction | 12.75% | 8.13% | 0.64× | 488 papers |
| Apoptosis | 12.22% | 7.6% | 0.62× | 456 papers |
| Antineoplastic Agents | 11.12% | 7.32% | 0.66× | 439 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Randomized Controlled Trial | 1.5% | 0.4% |
| Review | 10.3% | 12.6% |
| Meta-Analysis | 0.8% | 0.9% |
| Case Reports | 0.0% | 0.7% |
Query: Glioblastoma[MeSH Major Topic] NOT ("Oligodendroglioma"[MeSH] OR "Ependymoma"[MeSH] OR "Medulloblastoma"[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. Windows are read whole where they fit and sampled where they do not; the totals and the sample sizes are both in the JSON beside this page. Source: PubMed MeSH, retrieved 2026-09-11.
Disease burden
What the public sources count, and how closely each category matches this disease.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | 13,073 cases/year | direct | 2017-2021 |
| Incidence rate | 3.27 cases per 100,000 per year | direct | 2017-2021 |
| Five-year relative survival | 6.8% | direct | 2001-2020 |
| Median age at diagnosis | 66.0 years | direct | 2017-2021 |
| New cases each year | 24,740 cases/year | proxy | counts malignant brain and other cns tumours, which is broader than this disease; 2026 SEER Cancer Stat Facts, Brain and Other Nervous System Cancer, retrieved 2026-09-11 |
| Deaths each year | 18,350 deaths/year | proxy | counts malignant brain and other cns tumours, which is broader than this disease; 2026 SEER Cancer Stat Facts, Brain and Other Nervous System Cancer, retrieved 2026-09-11 |
| Death rate | 4.4 deaths per 100,000 per year | proxy | counts malignant brain and other cns tumours, which is broader than this disease; 2020-2024 SEER Cancer Stat Facts, Brain and Other Nervous System Cancer, retrieved 2026-09-11 |
| People living with it | 196,343 people living with the disease | proxy | counts malignant brain and other cns tumours, which is broader than this disease; 2023 SEER Cancer Stat Facts, Brain and Other Nervous System Cancer, retrieved 2026-09-11 |
| New cases each year, estimated | 12,741 cases/year | derived proxy | 24,740 x 0.515, from malignant brain and other cns tumours; 2026 SEER Cancer Stat Facts, Brain and Other Nervous System Cancer, retrieved 2026-09-11 |
| Deaths each year | — | not published | Neither SEER nor CBTRUS publishes deaths for glioblastoma alone; mortality is reported for malignant brain and other CNS tumours as a whole (17,411 a year, 2017-2021). |
Years of life lost
11.6 years per case, 151,082 a year, from survival and age at diagnosis rather than from a death count. derived proxy What this costs to fund is in Funding.
3 assumptions behind this estimate
- Five-year relative survival stands in for cure; a death after five years is not counted.
- The median age at diagnosis stands in for the whole age distribution.
- Life expectancy at birth (78.4 years) is the reference, rather than remaining expectancy at the age of death from a life table.
- Each assumption moves the result by more than the difference between the two diseases currently on this site, so this figure separates a disease from one ten times its size, not from a close neighbour.
Funding
NIH RePORTER · quarterlyNIH obligations naming glioblastoma, after removing the 4,160 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 | $80.9M | 245 | 212 | 256 |
| FY2014 | $93.7M | 261 | 228 | 282 |
| FY2015 | $107.3M | 282 | 242 | 273 |
| FY2016 | $110.0M | 297 | 256 | 299 |
| FY2017 | $127.6M | 338 | 282 | 301 |
| FY2018 | $132.3M | 352 | 301 | 346 |
| FY2019 | $148.5M | 396 | 336 | 336 |
| FY2020 | $162.3M | 393 | 333 | 335 |
| FY2021 | $177.2M | 417 | 334 | 348 |
| FY2022 | $172.3M | 413 | 343 | 344 |
| FY2023 | $184.9M | 428 | 359 | 361 |
| FY2024 | $193.7M | 440 | 360 | 357 |
| FY2025 | $180.0M | 381 | 335 | 322 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| University Of California, San Francisco | $12.7M | 19 |
| Brigham And Women'S Hospital | $12.1M | 20 |
| Northwestern University | $7.3M | 14 |
| Massachusetts General Hospital | $6.7M | 15 |
| University Of California Los Angeles | $6.4M | 17 |
| Cleveland Clinic Lerner Com-Cwru | $6.3M | 14 |
| Washington University | $6.2M | 0 |
| University Of Tx Md Anderson Can Ctr | $5.7M | 17 |
| Duke University | $5.4M | 11 |
| University Of Michigan At Ann Arbor | $5.0M | 8 |
Text search glioblastoma 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 2026-09-11.
What that buys
Against 151,082 years of life lost a year, FY2025 obligations are $1,192 per life-year — $13,772 per case. The estimate and its assumptions are in Disease burden.
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 10 and account for 381 of 381.
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 15 and account for 342 of 381.
Where it lands
Share of $180.0M in FY2025. The top three hold 18%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly2,377 human GEO series match glioblastoma. Keyword relevance cannot tell a 804-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 341-study ranked set
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE84465 | Single-Cell RNAseq analysis of diffuse neoplastic infiltrating cells at the migrating front of human glioblastoma2017 · single-cell | 3,589 | 245 | 23.9 | APT 0.75794 cites |
| GSE57872 | Single cell RNA-seq of primary human glioblastomas2014 · sequencing | 875 | 74 | 105.2 | mixedAPT 0.753,911 cites |
| GSE36278 | Methylation data from glioblastoma tumor samples2012 · methylation | 142 | 36 | 36.3 | patient cohortAPT 0.95survivalmolecularCDKN2AEGFR1,470 cites |
| GSE13041 | Gene expression analysis of glioblastomas identifies the major molecular basis for the prognostic benefit of younger age2008 · array | 267 | 135 | 3.6 | patient cohortAPT 0.75survivalmolecularEGFRMGMT174 cites |
| GSE163120 | Single-cell profiling of myeloid cells in glioblastoma across species and disease stage reveals macrophage competition and specialization2020 · single-cell | 14 | 32 | 29.0 | patient cohortAPT 0.75survivalstage569 cites |
| GSE4536 | Tumor stem cells more closely mirror the phenotype and genotype of primary human tumors than do cancer cell lines2006 · array | 101 | 33 | 39.7 | mixedAPT 0.751,939 cites |
| GSE84010 | Patients With Proneural Glioblastoma May Derive Overall Survival Benefit From the Addition of Bevacizumab to First-Line Radiotherapy and Temozolomide: Retrospective Analysis of the AVAglio Trial2016 · array | 349 | 14 | 7.0 | patient cohortAPT 0.95survival235 cites |
| GSE103659 | Radiomic subtyping improves disease stratification beyond key molecular, clinical and standard imaging characteristics in patients with glioblastoma.2018 · methylation | 181 | 8 | 7.3 | patient cohortAPT 0.95survivalmolecularMGMT170 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE174554 | A single cell atlas of human glioma under therapy2022 · single-cell | 113 | 36 | 17.7 | patient cohortAPT 0.95survival276 cites |
| GSE274546 | The Multi-Layered Transcriptional Architecture of Glioblastoma Ecosystems [10X]2025 · sequencing | 121 | 6 | 24.1 | APT 0.7596 cites |
| GSE173280 | Single-cell landscapes of primary glioblastomas and matched organoids and cell lines reveal variable retention of inter- and intra-tumor heterogeneity.2022 · single-cell | 126 | 4 | 8.0 | mixedAPT 0.75129 cites |
| GSE279073 | Longitudinal epigenome analysis of IDH-wildtype glioblastomas from initial and recurrent surgical specimens2024 · methylation | 226 | 2 | 5.8 | patient cohortAPT 0.75survival23 cites |
| GSE246293 | Astrocyte-driven suppression of tumor-specific T-cell immunity in glioblastoma2025 · single-cell | 102 | 1 | 13.4 | mixedAPT 0.5survival53 cites |
| GSE200647 | DNA methylation analysis of glioblastomas harboring FGFR3-TACC3 fusions identifies a methylation subclass with better patient survival.2022 · methylation | 244 | 4 | 1.6 | patient cohortAPT 0.75survival25 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-11. SubSeries are collapsed to one row per study by linked PMID. Of 640 series retrieved, 29 were dropped by the profile’s exclusion rules and 189 named the disease only in passing. 1 further series named in the profile as contamination are excluded here. 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.
PTEN
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
NF1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
CDKN2A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
RB1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
MGMT
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 (2026-09-11), ClinicalTrials.gov (2026-09-11), openFDA (2026-09-11), NCBI GEO (2026-09-11), PubMed (2026-09-11), NIH RePORTER (2026-09-11).
Negatives stated explicitly
Where nothing exists for glioblastoma — 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
4 exclusion patterns are applied to free text before anything is ranked, because U87, U251 and T98G is used as a model system in nanoparticle and drug-delivery testing, blood-brain-barrier transport models, multi-line compound screens. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Glioblastoma",
"mesh": "Glioblastoma",
"facts": "https://usebiotransfer.org/disease/glioblastoma.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."
}