Target landscape
Open Targets · retrieved 2026-09-30 · 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 across cancers → | 72 | 15 approvedAfatinib, Aumolertinib, Brigatinib, Cetuximab Sarotalocan, Dacomitinib, Erlotinib, Icotinib, Lapatinib, Lazertinib, Mobocertinib, Neratinib, Nimotuzumab, Olmutinib, Osimertinib, RociletinibApproved in non-small cell lung carcinoma, anaplastic large cell lymphoma, head and neck cancer and 3 other indications. No glioblastoma indication appears on these drugs’ labels.9 active of 96 glioblastoma trials | antibody, other clinical modality, protein degrader, small molecule | 2 active of 8 trials |
| PDGFRA across cancers → | 30 | 7 approvedCediranib, Masitinib, Midostaurin, Nintedanib, Pazopanib, Quizartinib, RipretinibApproved in mast cell leukemia, acute myeloid leukemia by FAB classification, systemic mastocytosis and 5 other indications. No glioblastoma indication appears on these drugs’ labels.4 active of 48 glioblastoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| PTEN across cancers → | 0 | No drug— | antibody, protein degrader, small molecule | — |
| TP53 across cancers → | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, Teprasiran4 trials, none active | other clinical modality, protein degrader, small molecule | — |
| NF1 across cancers → | 0 | No drug— | antibody, protein degrader, small molecule | — |
| CDK4 across cancers → | 15 | 2 approvedRibociclib, TrilaciclibApproved in breast cancer, breast carcinoma, small cell lung carcinoma. No glioblastoma indication appears on these drugs’ labels.6 active of 17 glioblastoma trials | protein degrader, small molecule | — |
| MDM2 across cancers → | 4 | Phase 3Alrizomadlin, Idasanutlin, Navtemadlin, Siremadlin2 trials, none active | antibody, protein degrader, small molecule | — |
| CDKN2A across cancers → | 0 | No drug— | — | — |
| RB1 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| PIK3CA across cancers → | 31 | 2 approvedCopanlisib, InavolisibApproved in follicular lymphoma, non-Hodgkin lymphoma, neoplasm of mature B-cells and 1 other indications. No glioblastoma indication appears on these drugs’ labels.3 active of 17 glioblastoma trials | antibody, protein degrader, small molecule | — |
| TERT across cancers → | 1 | 1 approvedImetelstatApproved in myelodysplastic syndrome, anemia. No glioblastoma indication appears on these drugs’ labels.2 trials, none active | antibody, other clinical modality, protein degrader, small molecule | — |
| MGMT across cancers → | 0 | No drug— | protein degrader, small molecule | — |
Dataset evidence counts studies in the 1220-study ranked set whose title or abstract names the gene; the bar is scaled to EGFR. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-30. 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 · weekly3 drugs carry an FDA label naming glioblastoma: Bevacizumab, Carmustine, Temozolomide. Separately, 27 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. |
41 labels carry a positive indication naming the disease under 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 2026-09-18. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy against EGFRvIII
ClinicalTrials.gov · retrieved 2026-09-18 · weeklyEGFRvIII is the busiest cell-therapy target among those this briefing searched in glioblastoma: 8 registered trials naming it, 2 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 |
|---|---|---|---|---|
| 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 |
| 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 |
| NCT02664363 | 1 | Terminated | EGFRvIII CAR T Cells for Newly-Diagnosed WHO Grade IV Malignant Glioma | 2023-02-17 |
| NCT03726515 | 1 | Completed | CART-EGFRvIII + Pembrolizumab in GBM | 2023-06-22 |
| NCT05802693 | EARLY/1 | Unknown | A Study to Evaluate the Safety, Tolerance and Initial Efficacy of EGFRvIII CAR-T on Glioblastoma | 2023-08-25 |
8 of 8 shown, most recently active first. Other targets searched: IL13RA2 (6), B7-H3 (6), CD70 (2), HER2 (1), GD2 (1), EphA2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-18. A trial counts here when its registered conditions name glioblastoma 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 · 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% | 6.5× | 39 papers |
| Receptors, Chimeric Antigen | 0.23% | 1.33% | 5.71× | 80 papers |
| Methyltransferases | 0.08% | 0.45% | 5.4× | 27 papers |
| Adenosine | 0.1% | 0.52% | 5.17× | 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. The same figures for every disease on the site are on one table.
| 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 (proxy)
11.6 years per case, 151,082 a year, from survival and age at diagnosis rather than from a death count. derived proxy Not the WHO/GBD measure, which uses age at death and a life table; comparable across these briefings, not with published YLL figures. 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 4,072 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 | $81.1M | 243 | 210 | 251 |
| FY2014 | $93.7M | 258 | 225 | 278 |
| FY2015 | $107.6M | 280 | 240 | 267 |
| FY2016 | $109.8M | 294 | 253 | 294 |
| FY2017 | $126.8M | 334 | 278 | 296 |
| FY2018 | $132.1M | 351 | 300 | 341 |
| FY2019 | $149.4M | 397 | 337 | 326 |
| FY2020 | $163.3M | 394 | 334 | 327 |
| FY2021 | $177.7M | 417 | 334 | 341 |
| FY2022 | $171.4M | 413 | 343 | 335 |
| FY2023 | $186.6M | 431 | 361 | 350 |
| FY2024 | $193.8M | 439 | 359 | 351 |
| FY2025 | $180.5M | 380 | 334 | 315 |
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 2026-09-19.
What that buys
Against 151,082 years of life lost a year, FY2025 obligations are $1,195 per life-year — $13,809 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 9 and account for 380 of 380.
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 341 of 380.
Where it lands
Share of $180.5M in FY2025. The top three hold 18%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly2,382 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 1220-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 |
| GSE4412 | freij-affy-human-916662006 · array | 170 | 258 | 10.6 | patient cohortAPT 0.95survivalstage548 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 | 44 | 36.3 | patient cohortAPT 0.95survivalmolecularCDKN2AEGFR1,470 cites |
| GSE131928 | single cell RNA-seq analysis of adult and paediatric IDH-wildtype Glioblastomas2019 · single-cell | 2 | 301 | 78.9 | patient cohortAPT 0.952,158 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 |
| GSE7696 | Glioblastoma from a homogenous cohort of patients treated within clinical trial2008 · array | 84 | 243 | 12.8 | patient cohortAPT 0.75597 cites |
| GSE121810 | Neoadjuvant anti-PD-1 immunotherapy promotes a survival benefit with intratumoral and systemic immune responses in recurrent glioblastoma2018 · sequencing | 29 | 45 | 42.8 | patient cohortAPT 0.95survival1,142 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 |
| GSE223065 | Glioblastoma remodeling of neural circuits in the human brain decreases survival2023 · sequencing | 13 | 9 | 32.6 | patient cohortAPT 0.95survivalstage341 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 | 26 | 8.0 | mixedAPT 0.75129 cites |
| GSE278456 | Distinct Myeloid Derived Suppressor Cell Populations in Human Glioblastoma2025 · single-cell | 39 | 4 | 26.2 | patient cohortAPT 0.5survivalstage102 cites |
| GSE237183 | Integrative spatial analysis reveals a multi-layered organization of glioblastoma2024 · spatial | 19 | 12 | 33.6 | APT 0.75293 cites |
| GSE235913 | Siglec-9 acts as an immune checkpoint molecule on macrophages in glioblastoma, restricting T cell priming and immunotherapy response2023 · sequencing | 51 | 13 | 16.5 | APT 0.75206 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-19. SubSeries are collapsed to one row per study by linked PMID. Of 2,382 series retrieved, 77 were dropped by the profile’s exclusion rules and 766 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
Open Targets lists no drug against it and no trial in glioblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.
NF1
Open Targets lists no drug against it and no trial in glioblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.
CDKN2A
Open Targets lists no drug against it and no trial in glioblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.
RB1
Open Targets lists no drug against it and no trial in glioblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.
MGMT
Open Targets lists no drug against it and no trial in glioblastoma names it. A bounded negative: the sources and search terms are in the JSON beside this page.
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. 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 (2026-09-11), NIH RePORTER (2026-09-19).
Negatives stated explicitly
Where the searches find nothing for glioblastoma — 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
2 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/",
"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."
}