RARE DISEASERESEARCH ATLAS
Open navigation menu

ORPHA:360

Glioblastoma

low confidenceDisorder

Also known as: GBM · Glioblastoma multiforme

Publications

235,637

Trials

1,693

Interventional, condition-specific

Researchers

1,190

Distinct authors in sample

Gene link

—

Readiness

5/6

Stages with a signal

Clinical definition (Orphanet)

Glioblastomas are malignant astrocytic tumors (grade IV according to the WHO classification).

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (11)

GBM (glioblastoma) · WHO grade IV glioma · glioblastoma · glioblastoma (disease) · glioblastoma multiforme · glioblastoma multiforme (disease) · grade IV astrocytic neoplasm · grade IV astrocytic tumor · grade IV astrocytic tumour · grade IV astrocytoma · spongioblastoma multiforme

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

5/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    235,637 matched papers (163,614 in last 10 years) Source

  3. Phenotype characterisedPresent

    15 HPO annotations (e.g. Abnormal nervous system physiology; Seizure; Emotional lability) Source

  4. Animal modelPresent

    4 genotype models (Mus musculus) Source

  5. Orphan designationPresent

    15 FDA · 1 EMA designations (15 FDA orphan-indication approvals) — e.g. cannabidiol Source

  6. Interventional trialPresent

    1,693 matched on ClinicalTrials.gov (345 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

15

Associated phenotypes · MONDO:0018177

  • Abnormal nervous system physiology
  • Seizure
  • Emotional lability
  • Cerebral edema
  • Headache

Showing 5 of 15 — open Monarch for the full list.

Animal models (Monarch / Alliance)

4

Model associations linked to this Mondo ID

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

16

Designations · 15 with FDA orphan-indication approval

  • FDA cannabidiolGlioblastoma · 2020-04-28 · Not FDA Approved for Orphan Indication
  • FDA RegorafenibGlioblastoma multiforme · 2019-07-01 · Not FDA Approved for Orphan Indication
  • FDA IbudilastGlioblastoma · 2018-10-02 · Not FDA Approved for Orphan Indication
  • FDA (1-hydroxy-2-oxopiperidin-3-yl)phosphonic acidGlioblastoma · 2018-08-09 · Not FDA Approved for Orphan Indication
  • FDA (3R,3aS,9R,9aS,9bS)-3-((dimethylamino)methyl)-9-hydroxy-6,9-dimethyl-3,3a,4,5,7,8,9,9a-octahydroazuleno[4,5-b]furan-2(9bH)-one fumarateGlioblastoma multiforme · 2017-11-08 · Not FDA Approved for Orphan Indication
  • FDA RitanserinGlioblastoma · 2017-05-11 · Not FDA Approved for Orphan Indication
  • FDA disufenton sodiumGlioblastoma multiforme · 2016-08-22 · Not FDA Approved for Orphan Indication
  • FDA human recombinant bone morphogenetic protein 4Glioblastoma multiforme · 2016-05-04 · Not FDA Approved for Orphan Indication

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

456

Drugs / clinical candidates · MONDO_0018177

CTD chemicals (MyDisease.info)

86 associated chemicals · 627 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • 1-(2-chloroethyl)-1-nitrosourea · therapeutic
  • 2,5-dimethylcelecoxib · therapeutic
  • 4,4'-Diisothiocyanostilbene-2,2'-Disulfonic Acid · therapeutic
  • Acridines · therapeutic
  • AICA ribonucleotide · therapeutic
  • Amphotericin B · therapeutic
  • Arsenic Trioxide · therapeutic
  • Aspirin · therapeutic
  • baicalein · therapeutic
  • biochanin A · therapeutic
  • Bortezomib · therapeutic
  • butylidenephthalide · therapeutic

Pathways: Glutathione metabolism; Metabolism of xenobiotics by cytochrome P450; Drug metabolism - cytochrome P450; EGFR tyrosine kinase inhibitor resistance; Endocrine resistance; Antifolate resistance; Platinum drug resistance; Basal transcription factors

MyDisease.info · MONDO:0018177

Literature

Is anyone studying this?

235,637

235,637 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

235,637 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

163,614 in the last 10 years · low confidence

Phrase hits: 235,637 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,190

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Li Z6 papers · 2026

    Department of Neurosurgery, Beijing Tiantan Hospital, Beijing Neurosurgical Institute, Capital Medical University, Beijing, 100070, China.

    Papers in Europe PMC
  2. 02
    Wang X6 papers · 2026

    Institute of Photomedicine, Shanghai Skin Disease Hospital, School of Medicine, Tongji University, Shanghai, 200443, China.

    Papers in Europe PMC
  3. 03
    Wang Y6 papers · 2026

    Neurosurgical Oncology, The First Bethune Hospital of Jilin University, China.

    Papers in Europe PMC
  4. 04
    Liu Y5 papers · 2026

    Department of Neurosurgery, Beijing Tiantan Hospital, Beijing Neurosurgical Institute, Capital Medical University, Beijing, 100070, China.

    Papers in Europe PMC
  5. 05
    Chen J4 papers · 2026

    Research Center for Biomedical Optics and Molecular Imaging, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen, Guangdong 518055, China.

    Papers in Europe PMC
  6. 06
    Chen Y4 papers · 2026

    Cancer Center, Medical Research Institute, State Key Laboratory of Resource Insects, Southwest University, Chongqing, 400715, China; Jinfeng Laboratory, Chongqing, 401329, China; Chongqing Engineering and Technology Research Center for Silk Biomaterials and Regenerative Medicine, Chongqing, 400716, China.

    Papers in Europe PMC
  7. 07
    Li Q4 papers · 2026

    Shanghai ImmunoBiotech Co., Ltd., Shanghai, China.

    Papers in Europe PMC
  8. 08
    Li Y4 papers · 2026

    Neurosurgical Oncology, The First Bethune Hospital of Jilin University, China.

    Papers in Europe PMC
  9. 09
    Wang P4 papers · 2026

    Department of Neurosurgery, Huashan Hospital, Fudan University, Shanghai, China.

    Papers in Europe PMC
  10. 10
    Yang Y4 papers · 2026

    Department of Neurosurgery, Qilu Hospital of Shandong University, Institute of Brain and Brain-Inspired Science, Cheeloo College of Medicine, and the School of Biomedical Engineering, Shandong University, Jinan 250012, Shandong, China.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

1,693

interventional trials for this specific condition

1,693 interventional trials matched this specific condition name; 345 currently recruiting in our sample.

Data as of 11 September 2026

1,693 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 100th percentile).

low confidence · 100th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

1,693 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

197 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 99 · after dedupe 99 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 99 · dropped 0 · fetched 2026-07-29

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (99)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Glioblastoma — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Glioblastoma" OR "Glioblastoma multiforme" OR "GBM (glioblastoma)" OR "WHO grade IV glioma" OR "glioblastoma (disease)" OR "glioblastoma multiforme (disease)" OR "grade IV astrocytic neoplasm" OR "grade IV astrocytic tumor" OR "grade IV astrocytic tumour" OR "grade IV astrocytoma" OR "spongioblastoma multiforme"

Run this search on Europe PMC

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Glioblastoma" OR "Glioblastoma multiforme" OR "GBM (glioblastoma)" OR "WHO grade IV glioma" OR "glioblastoma (disease)" OR "glioblastoma multiforme (disease)" OR "grade IV astrocytic neoplasm" OR "grade IV astrocytic tumor" OR "grade IV astrocytic tumour" OR "grade IV astrocytoma" OR "spongioblastoma multiforme"

Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 1693 interventional · 197 observational · 16 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase; with hits: phrase

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: GBM

Confidence reasoning

  • Preferred label is short or not clearly distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-26T13:33:24.301Z