Disease Briefing

Glioblastoma: 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 1220 studies · 45,911 GEO samples
Page Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in glioblastoma — EGFR, PDGFRA, PTEN, TP53, NF1, CDK4, MDM2, CDKN2A, RB1, PIK3CA, TERT, MGMT — 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.

3
drugs carry an FDA label naming glioblastoma: Bevacizumab, Carmustine, Temozolomide. 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 glioblastoma — 179 drug entries reach them, 161 distinct once salt forms are merged, and 27 of those are approved for other indications. A statement about these gene targets, not about the disease
8
registered EGFRvIII cell-therapy trials in glioblastoma, 2 active. Counted from ClinicalTrials.gov across 6 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
4
targets carry an Open Targets tractability signal and no trial in glioblastoma that the searches found (no trial naming a drug Open Targets lists for the gene, none naming it as a cell-therapy target): PTEN, NF1, RB1, MGMT. EGFR has cell-therapy trials, so it is undrugged rather than untouched
2,382
human GEO series match the disease; 1,220 survive on-topic filtering, and only 43 are patient cohorts of 100+ samples
301
Europe PMC full-text papers name GSE131928 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$180.5M
NIH obligations in FY2025, up 123% since 2013 — while distinct core projects went 210 to 334. Both more projects (+59%) and larger awards, the latter carrying more of the growth; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-30 · weekly

12 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.

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
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 · weekly

3 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.

3Labelled for glioblastomaFDA INDICATIONS AND USAGE names the disease
27Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
2Active cell-therapy trials naming EGFRvIIIof 8 registered

Every label that names glioblastoma

DrugRoleWhat the label says
BevacizumabALYMSYS, Avastin, JOBEVNELabelled hereRecurrent glioblastoma in adults.
CarmustineCARMUSTINE, Carmustine, GliadelLabelled hereBrain tumors glioblastoma, brainstem glioma, medulloblastoma, astrocytoma, ependymoma, and metastatic brain tumors ( 1 ) 2.
TemozolomideTEMODAR, TEMOZOLOMIDE, TemozolomideLabelled hereNewly 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 · weekly

EGFRvIII 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.

TrialPhaseStatusTitleLast update
NCT061864011RecruitingAnti-EGFRvIII synNotch Receptor Induced Anti-EphA2/IL-13Ralpha2 CAR (E-SYNC) T Cells2026-08-13
NCT07244666EARLY/1Not yet recruitingSafety and Preliminary Efficacy of a Metabolically Armed Chimeric Antigen Receptor T Cell Therapy Targeting EGFRvIII for Recurrent Glioblastoma2025-11-24
NCT028440621UnknownPilot Study of Autologous Anti-EGFRvIII CAR T Cells in Recurrent Glioblastoma Multiforme2016-07-26
NCT014545961/2CompletedCAR T Cell Receptor Immunotherapy Targeting EGFRvIII for Patients With Malignant Gliomas Expressing EGFRvIII2019-08-21
NCT050636821UnknownThe 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 Patients2021-10-01
NCT026643631TerminatedEGFRvIII CAR T Cells for Newly-Diagnosed WHO Grade IV Malignant Glioma2023-02-17
NCT037265151CompletedCART-EGFRvIII + Pembrolizumab in GBM2023-06-22
NCT05802693EARLY/1UnknownA Study to Evaluate the Safety, Tolerance and Initial Efficacy of EGFRvIII CAR-T on Glioblastoma2023-08-25

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 · monthly

Every 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 term2015–182021–25ChangeShare now
Neural Networks, Computer0.1%0.65%6.5×39 papers
Receptors, Chimeric Antigen0.23%1.33%5.71×80 papers
Methyltransferases0.08%0.45%5.4×27 papers
Adenosine0.1%0.52%5.17×31 papers
Molecular Docking Simulation0.23%1.2%5.14×72 papers
Tumor Microenvironment2.98%15.07%5.05×904 papers
Biomimetics0.08%0.42%5.0×25 papers
CRISPR-Cas Systems0.08%0.4%4.8×24 papers
Nomograms0.1%0.45%4.5×27 papers
Iron0.08%0.37%4.4×22 papers

Cooling

MeSH term2015–182021–25ChangeShare now
RNA Interference2.47%0.25%0.1×15 papers
Immunohistochemistry2.95%0.3%0.1×18 papers
Dacarbazine9.6%1.4%0.15×84 papers
Time Factors2.43%0.37%0.15×22 papers
Proportional Hazards Models2.27%0.33%0.15×20 papers
Gene Knockdown Techniques2.43%0.4%0.16×24 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Brain Neoplasms68.98%68.5%0.99×4110 papers
Tumor Microenvironment2.98%15.07%5.05×904 papers
Cell Proliferation19.18%13.9%0.72×834 papers
Gene Expression Regulation, Neoplastic16.82%13.2%0.78×792 papers
Prognosis13.33%12.2%0.92×732 papers
Temozolomide11.63%11.88%1.02×713 papers
Glioma2.65%10.57%3.99×634 papers
Signal Transduction12.75%8.13%0.64×488 papers
Apoptosis12.22%7.6%0.62×456 papers
Antineoplastic Agents11.12%7.32%0.66×439 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial1.5%0.4%
Review10.3%12.6%
Meta-Analysis0.8%0.9%
Case Reports0.0%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.

MeasureValueMatchWhat it counts
New cases each year13,073 cases/yeardirect2017-2021
Incidence rate3.27 cases per 100,000 per yeardirect2017-2021
Five-year relative survival6.8%direct2001-2020
Median age at diagnosis66.0 yearsdirect2017-2021
New cases each year24,740 cases/yearproxycounts malignant brain and other cns tumours, which is broader than this disease; 2026
Deaths each year18,350 deaths/yearproxycounts malignant brain and other cns tumours, which is broader than this disease; 2026
Death rate4.4 deaths per 100,000 per yearproxycounts malignant brain and other cns tumours, which is broader than this disease; 2020-2024
People living with it196,343 people living with the diseaseproxycounts malignant brain and other cns tumours, which is broader than this disease; 2023
New cases each year, estimated12,741 cases/yearderived proxy24,740 x 0.515, from malignant brain and other cns tumours; 2026
Deaths each year—not publishedNeither 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 · quarterly

NIH 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.

$180.5MNIH obligations, FY2025from $81.1M in FY2013 · +123%
334distinct projects funded210 in FY2013
$193.8Mpeak year was FY2024obligations, all institutes
62%of FY2025 awards from NCI235 of 380

NIH obligations by fiscal year

$48M$97M$145M$194M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$81.1M243210251
FY2014$93.7M258225278
FY2015$107.6M280240267
FY2016$109.8M294253294
FY2017$126.8M334278296
FY2018$132.1M351300341
FY2019$149.4M397337326
FY2020$163.3M394334327
FY2021$177.7M417334341
FY2022$171.4M413343335
FY2023$186.6M431361350
FY2024$193.8M439359351
FY2025$180.5M380334315

Where FY2025 money went

InstitutionObligationsAwards
University Of California, San Francisco$12.7M19
Brigham And Women'S Hospital$12.1M20
Northwestern University$7.3M14
Massachusetts General Hospital$6.7M15
University Of California Los Angeles$6.4M17
Cleveland Clinic Lerner Com-Cwru$6.3M14
Washington University$6.2M0
University Of Tx Md Anderson Can Ctr$5.7M17
Duke University$5.4M11
University Of Michigan At Ann Arbor$5.0M8

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

NCI235 · 62%
NINDS124 · 33%
NIGMS8 · 2%
NIBIB6 · 2%
NIAID2 · 1%
NCATS2 · 1%

Projects by administering institute. The rows above are the top 9 and account for 380 of 380.

Award mechanisms

R01200 · 53%
U1920 · 5%
R2117 · 4%
P5014 · 4%
P0113 · 3%
R3712 · 3%

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

University Of California, San Francisco$12.7M · 7.0%
Brigham And Women'S Hospital$12.1M · 6.7%
Northwestern University$7.3M · 4.0%
Massachusetts General Hospital$6.7M · 3.7%
University Of California Los Angeles$6.4M · 3.5%
Cleveland Clinic Lerner Com-Cwru$6.3M · 3.5%

Share of $180.5M in FY2025. The top three hold 18%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

2,382 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

unspecified 401patient 398cell line 229mixed 154xenograft 38
401 unspecified398 patient229 cell line154 mixed38 xenograft531 carry clinical annotation423 carry survival43 patient cohorts ≥100 GEO samples45,911 GEO samples totalin 95 single-cell series the GEO sample count is the submitter’s unit (cells, libraries or pools), not patients

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE84465Single-Cell RNAseq analysis of diffuse neoplastic infiltrating cells at the migrating front of human glioblastoma2017 · single-cell3,58924523.9
APT 0.75794 cites
GSE4412freij-affy-human-916662006 · array17025810.6
patient cohortAPT 0.95survivalstage548 cites
GSE57872Single cell RNA-seq of primary human glioblastomas2014 · sequencing87574105.2
mixedAPT 0.753,911 cites
GSE36278Methylation data from glioblastoma tumor samples2012 · methylation1424436.3
patient cohortAPT 0.95survivalmolecularCDKN2AEGFR1,470 cites
GSE131928single cell RNA-seq analysis of adult and paediatric IDH-wildtype Glioblastomas2019 · single-cell230178.9
patient cohortAPT 0.952,158 cites
GSE13041Gene expression analysis of glioblastomas identifies the major molecular basis for the prognostic benefit of younger age2008 · array2671353.6
patient cohortAPT 0.75survivalmolecularEGFRMGMT174 cites
GSE7696Glioblastoma from a homogenous cohort of patients treated within clinical trial2008 · array8424312.8
patient cohortAPT 0.75597 cites
GSE121810Neoadjuvant anti-PD-1 immunotherapy promotes a survival benefit with intratumoral and systemic immune responses in recurrent glioblastoma2018 · sequencing294542.8
patient cohortAPT 0.95survival1,142 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE174554A single cell atlas of human glioma under therapy2022 · single-cell1133617.7
patient cohortAPT 0.95survival276 cites
GSE223065Glioblastoma remodeling of neural circuits in the human brain decreases survival2023 · sequencing13932.6
patient cohortAPT 0.95survivalstage341 cites
GSE173280Single-cell landscapes of primary glioblastomas and matched organoids and cell lines reveal variable retention of inter- and intra-tumor heterogeneity.2022 · single-cell126268.0
mixedAPT 0.75129 cites
GSE278456Distinct Myeloid Derived Suppressor Cell Populations in Human Glioblastoma2025 · single-cell39426.2
patient cohortAPT 0.5survivalstage102 cites
GSE237183Integrative spatial analysis reveals a multi-layered organization of glioblastoma2024 · spatial191233.6
APT 0.75293 cites
GSE235913Siglec-9 acts as an immune checkpoint molecule on macrophages in glioblastoma, restricting T cell priming and immunotherapy response2023 · sequencing511316.5
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 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.

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

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 (2026-09-11), 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."
}