Adult glioblastoma
diseaseOn this page
Also known as adult glioblastoma multiformeglioblastomagrade IV adult astrocytic neoplasmgrade IV adult astrocytic tumorgrade IV adult astrocytic tumour
Summary
Adult glioblastoma (MONDO:0020690) is a disease with 24 cohort genes and 1,139 clinical trials. The dominant Reactome pathway is Constitutive Signaling by EGFRvIII (4 cohort genes). Molecularly, MGMT Promoter Methylation confers sensitivity to Temozolomide in Glioblastoma (CIViC Level A); 67 further subtype–drug associations are mapped below. Top therapeutic interventions include temozolomide, lomustine, and aminolevulinic acid.
At a glance
- Cohort genes: 24
- Clinical trials: 1,139
- Precision-medicine evidence (CIViC): 68 subtype–drug associations
Clinical features
No curated clinical features (Orphanet) for this disease.
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | adult glioblastoma |
| Mondo ID | MONDO:0020690 |
| NCIT | C9094 |
| UMLS | C0278878 |
| MedGen | 124527 |
| GARD | 0025213 |
| Is cancer (heuristic) | no |
Also known as: adult glioblastoma · adult glioblastoma multiforme · glioblastoma · grade IV adult astrocytic neoplasm · grade IV adult astrocytic tumor · grade IV adult astrocytic tumour
Data availability: 1,249 cell lines · 16 intOGen driver records.
Disease family
An umbrella term covering 1 Mondo subtype.
Classification path: disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › nervous system neoplasm › neuroepithelial neoplasm › glioma › astrocytic tumor › adult astrocytic tumor › adult infiltrating astrocytic neoplasm › adult glioblastoma
Subtypes (1): adult spinal cord glioblastoma
Genetics & variants
GWAS landscape
No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.
Variant details and genetic-evidence tiers
No tiered GWAS variants or ClinVar records for this disease.
Genes & proteins
Mendelian disease overlap and somatic drivers
GenCC: 0 · Orphanet: 159 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0
Orphanet rare-disease linkage (cohort genes)
| Gene | Orphanet ID | Rare disease |
|---|---|---|
| BRAF | Orphanet:1340 | Cardiofaciocutaneous syndrome |
| BRAF | Orphanet:146 | Differentiated thyroid carcinoma |
| BRAF | Orphanet:251615 | Pilomyxoid astrocytoma |
| BRAF | Orphanet:389 | Langerhans cell histiocytosis |
| BRAF | Orphanet:500 | Noonan syndrome with multiple lentigines |
| BRAF | Orphanet:54595 | Craniopharyngioma |
| BRAF | Orphanet:58017 | Classic hairy cell leukemia |
| BRAF | Orphanet:626 | Large/giant congenital melanocytic nevus |
| BRAF | Orphanet:648 | Noonan syndrome |
| BRAF | Orphanet:840 | Syringocystadenoma papilliferum |
| BRAF | Orphanet:96253 | Cushing disease |
| BRCA2 | Orphanet:1331 | Familial prostate cancer |
| BRCA2 | Orphanet:1333 | Familial pancreatic carcinoma |
| BRCA2 | Orphanet:145 | Hereditary breast and/or ovarian cancer syndrome |
| BRCA2 | Orphanet:178 | Chordoma |
| BRCA2 | Orphanet:227535 | Hereditary breast cancer |
| BRCA2 | Orphanet:319462 | Inherited cancer-predisposing syndrome due to biallelic BRCA2 mutations |
| BRCA2 | Orphanet:440437 | Familial colorectal cancer Type X |
| BRCA2 | Orphanet:654 | Nephroblastoma |
| BRCA2 | Orphanet:667662 | Breast implant-associated anaplastic large cell lymphoma |
| BRCA2 | Orphanet:694963 | Inflammatory breast cancer |
| BRCA2 | Orphanet:70567 | Cholangiocarcinoma |
| BRCA2 | Orphanet:84 | Fanconi anemia |
| SOS1 | Orphanet:2024 | Hereditary gingival fibromatosis |
| SOS1 | Orphanet:648 | Noonan syndrome |
| TERT | Orphanet:146 | Differentiated thyroid carcinoma |
| TERT | Orphanet:1501 | Adrenocortical carcinoma |
| TERT | Orphanet:1775 | Dyskeratosis congenita |
| TERT | Orphanet:2032 | Idiopathic pulmonary fibrosis |
| TERT | Orphanet:2495 | Meningioma |
| TERT | Orphanet:3322 | Hoyeraal-Hreidarsson syndrome |
| TERT | Orphanet:457246 | Clear cell sarcoma of kidney |
| TERT | Orphanet:618 | Familial melanoma |
| TERT | Orphanet:88 | Idiopathic aplastic anemia |
| TP53 | Orphanet:1333 | Familial pancreatic carcinoma |
| TP53 | Orphanet:145 | Hereditary breast and/or ovarian cancer syndrome |
| TP53 | Orphanet:1501 | Adrenocortical carcinoma |
| TP53 | Orphanet:210159 | Adult hepatocellular carcinoma |
| TP53 | Orphanet:251576 | Gliosarcoma |
| TP53 | Orphanet:251579 | Giant cell glioblastoma |
| TP53 | Orphanet:251899 | Choroid plexus carcinoma |
| TP53 | Orphanet:2807 | Papilloma of choroid plexus |
| TP53 | Orphanet:293199 | Pleomorphic rhabdomyosarcoma |
| TP53 | Orphanet:3318 | Essential thrombocythemia |
| TP53 | Orphanet:524 | Li-Fraumeni syndrome |
| TP53 | Orphanet:52688 | Myelodysplastic syndrome |
| TP53 | Orphanet:585909 | B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) |
| TP53 | Orphanet:667662 | Breast implant-associated anaplastic large cell lymphoma |
| TP53 | Orphanet:668 | Osteosarcoma |
| TP53 | Orphanet:67038 | B-cell chronic lymphocytic leukemia |
Cohort genes → proteins
24 cohort genes, 24 distinct canonical proteins.
Evidence partition
| Subset | Genes |
|---|---|
| civic_only | 24 |
Cohort genes (full)
| Symbol | HGNC | Ensembl | UniProt | Name | Evidence |
|---|---|---|---|---|---|
| BRAF | HGNC:1097 | ENSG00000157764 | P15056 | Serine/threonine-protein kinase B-raf | civic_evidence |
| BRCA2 | HGNC:1101 | ENSG00000139618 | P51587 | Breast cancer type 2 susceptibility protein | civic_evidence |
| SOS1 | HGNC:11187 | ENSG00000115904 | Q07889 | Son of sevenless homolog 1 | civic_evidence |
| TERT | HGNC:11730 | ENSG00000164362 | O14746 | Telomerase reverse transcriptase | civic_evidence |
| TP53 | HGNC:11998 | ENSG00000141510 | P04637 | Cellular tumor antigen p53 | civic_evidence |
| CDKN2A | HGNC:1787 | ENSG00000147889 | P42771 | Cyclin-dependent kinase inhibitor 2A | civic_evidence |
| SLTM | HGNC:20709 | ENSG00000137776 | Q9NWH9 | SAFB-like transcription modulator | civic_evidence |
| EGFR | HGNC:3236 | ENSG00000146648 | P00533 | Epidermal growth factor receptor | civic_evidence |
| EZH2 | HGNC:3527 | ENSG00000106462 | Q15910 | Histone-lysine N-methyltransferase EZH2 | civic_evidence |
| H3-3A | HGNC:4764 | ENSG00000163041 | P84243 | Histone H3.3 | civic_evidence |
| H3C2 | HGNC:4776 | ENSG00000286522 | P68431 | Histone H3.1 | civic_evidence |
| IDH1 | HGNC:5382 | ENSG00000138413 | O75874 | Isocitrate dehydrogenase [NADP] cytoplasmic | civic_evidence |
| IDH2 | HGNC:5383 | ENSG00000182054 | P48735 | Isocitrate dehydrogenase [NADP], mitochondrial | civic_evidence |
| KIT | HGNC:6342 | ENSG00000157404 | P10721 | Mast/stem cell growth factor receptor Kit | civic_evidence |
| LRP1B | HGNC:6693 | ENSG00000168702 | Q9NZR2 | Low-density lipoprotein receptor-related protein 1B | civic_evidence |
| MGMT | HGNC:7059 | ENSG00000170430 | P16455 | Methylated-DNA–protein-cysteine methyltransferase | civic_evidence |
| ATM | HGNC:795 | ENSG00000149311 | Q13315 | Serine-protein kinase ATM | civic_evidence |
| ATRX | HGNC:886 | ENSG00000085224 | P46100 | Transcriptional regulator ATRX | civic_evidence |
| PIK3CA | HGNC:8975 | ENSG00000121879 | P42336 | Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform | civic_evidence |
| PIK3R1 | HGNC:8979 | ENSG00000145675 | P27986 | Phosphatidylinositol 3-kinase regulatory subunit alpha | civic_evidence |
| PMS2 | HGNC:9122 | ENSG00000122512 | P54278 | Mismatch repair endonuclease PMS2 | civic_evidence |
| POLE | HGNC:9177 | ENSG00000177084 | Q07864 | DNA polymerase epsilon catalytic subunit A | civic_evidence |
| PTEN | HGNC:9588 | ENSG00000171862 | P60484 | Phosphatidylinositol 3,4,5-trisphosphate 3-phosphatase and dual-specificity protein phosphatase PTEN | civic_evidence |
| RB1 | HGNC:9884 | ENSG00000139687 | P06400 | Retinoblastoma-associated protein | civic_evidence |
Cohort function summary
Lead sentence per gene, UniProt-curated.
| Symbol | Protein name | Function (lead sentence) |
|---|---|---|
| BRAF | Serine/threonine-protein kinase B-raf | Protein kinase involved in the transduction of mitogenic signals from the cell membrane to the nucleus. |
| BRCA2 | Breast cancer type 2 susceptibility protein | Involved in double-strand break repair and/or homologous recombination. |
| SOS1 | Son of sevenless homolog 1 | Promotes the exchange of Ras-bound GDP by GTP. |
| TERT | Telomerase reverse transcriptase | Telomerase is a ribonucleoprotein enzyme essential for the replication of chromosome termini in most eukaryotes. |
| TP53 | Cellular tumor antigen p53 | Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence. |
| CDKN2A | Cyclin-dependent kinase inhibitor 2A | Acts as a negative regulator of the proliferation of normal cells by interacting strongly with CDK4 and CDK6. |
| SLTM | SAFB-like transcription modulator | When overexpressed, acts as a general inhibitor of transcription that eventually leads to apoptosis. |
| EGFR | Epidermal growth factor receptor | Receptor tyrosine kinase binding ligands of the EGF family and activating several signaling cascades to convert extracellular cues into appropriate cellular responses. |
| EZH2 | Histone-lysine N-methyltransferase EZH2 | Catalytic subunit of the PRC2/EED-EZH2 complex, a Polycomb group (PcG) complex that methylates ‘Lys-9’ (H3K9me) and ‘Lys-27’ (H3K27me) of histone H3, leading to transcriptional repression of the affected target gene. |
| H3-3A | Histone H3.3 | Variant histone H3 which replaces conventional H3 in a wide range of nucleosomes in active genes. |
| H3C2 | Histone H3.1 | Core component of nucleosome. |
| IDH1 | Isocitrate dehydrogenase [NADP] cytoplasmic | Catalyzes the NADP(+)-dependent oxidative decarboxylation of isocitrate (D-threo-isocitrate) to 2-ketoglutarate (2-oxoglutarate), which is required by other enzymes such as the phytanoyl-CoA dioxygenase. |
| IDH2 | Isocitrate dehydrogenase [NADP], mitochondrial | Plays a role in intermediary metabolism and energy production. |
| KIT | Mast/stem cell growth factor receptor Kit | Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine KITLG/SCF and plays an essential role in the regulation of cell survival and proliferation, hematopoiesis, stem cell maintenance, gametogenesis, mast cell develo… |
| LRP1B | Low-density lipoprotein receptor-related protein 1B | Potential cell surface proteins that bind and internalize ligands in the process of receptor-mediated endocytosis. |
| MGMT | Methylated-DNA–protein-cysteine methyltransferase | Involved in the cellular defense against the biological effects of O6-methylguanine (O6-MeG) and O4-methylthymine (O4-MeT) in DNA. |
| ATM | Serine-protein kinase ATM | Serine/threonine protein kinase which activates checkpoint signaling upon double strand breaks (DSBs), apoptosis and genotoxic stresses such as ionizing ultraviolet A light (UVA), thereby acting as a DNA damage sensor. |
| ATRX | Transcriptional regulator ATRX | Involved in transcriptional regulation and chromatin remodeling. |
| PIK3CA | Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform | Phosphoinositide-3-kinase (PI3K) phosphorylates phosphatidylinositol (PI) and its phosphorylated derivatives at position 3 of the inositol ring to produce 3-phosphoinositides. |
| PIK3R1 | Phosphatidylinositol 3-kinase regulatory subunit alpha | Binds to activated (phosphorylated) protein-Tyr kinases, through its SH2 domain, and acts as an adapter, mediating the association of the p110 catalytic unit to the plasma membrane. |
| PMS2 | Mismatch repair endonuclease PMS2 | Component of the post-replicative DNA mismatch repair system (MMR). |
| POLE | DNA polymerase epsilon catalytic subunit A | Catalytic component of the DNA polymerase epsilon complex. |
| PTEN | Phosphatidylinositol 3,4,5-trisphosphate 3-phosphatase and dual-specificity protein phosphatase PTEN | Dual-specificity protein phosphatase, dephosphorylating tyrosine-, serine- and threonine-phosphorylated proteins. |
| RB1 | Retinoblastoma-associated protein | Tumor suppressor that is a key regulator of the G1/S transition of the cell cycle. |
Protein-family classification
Druggable: 11 · Difficult: 5 · Unknown: 8 · Druggable fraction: 0.46
Family distribution
Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.
| Family | Genes | Fold | FDR |
|---|---|---|---|
| Kinase | 6 | 6.9× | 1e-03 |
| Enzyme (other) | 4 | 2.0× | 0.405 |
| Phosphatase | 1 | 3.5× | 0.500 |
| Scaffold/PPI | 2 | 1.4× | 0.612 |
| Transcription factor | 3 | 1.0× | 0.683 |
| Other/Unknown | 8 | 0.6× | 0.992 |
Per-gene assignment
| Symbol | Family | Druggable? | EC | InterPro (top 3) |
|---|---|---|---|---|
| BRAF | Kinase | yes | 2.7.10.2 | Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, PKC_DAG/PE |
| BRCA2 | Other/Unknown | no | BRCA2_repeat, NA-bd_OB-fold, BRCA2_OB_1 | |
| SOS1 | Scaffold/PPI | no | DH_dom, Ras-like_Gua-exchang_fac_N, PH_domain | |
| TERT | Other/Unknown | no | RT_dom, Telomerase_RT, Telomerase_RBD | |
| TP53 | Transcription factor | no | p53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn | |
| CDKN2A | Scaffold/PPI | no | Ankyrin_rpt-contain_sf, Ank_Repeat/CDKN_Inhibitor, Tumor_suppres_ARF | |
| SLTM | Other/Unknown | no | RRM_dom, SAP_dom, Nucleotide-bd_a/b_plait_sf | |
| EGFR | Kinase | yes | 2.7.10.1 | Rcpt_L-dom, Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom |
| EZH2 | Enzyme (other) | yes | 2.1.1.356 | SANT/Myb, SET_dom, EZH1/EZH2_N |
| H3-3A | Other/Unknown | no | Histone_H3/CENP-A, H2A/H2B/H3, Histone-fold | |
| H3C2 | Other/Unknown | no | Histone_H3/CENP-A, H2A/H2B/H3, Histone-fold | |
| IDH1 | Enzyme (other) | yes | 1.1.1.42 | Isocitrate_DH_NADP, IsoCit/isopropylmalate_DH_CS, IsoPropMal-DH-like_dom |
| IDH2 | Enzyme (other) | yes | 1.1.1.42 | Isocitrate_DH_NADP, IsoCit/isopropylmalate_DH_CS, IsoPropMal-DH-like_dom |
| KIT | Kinase | yes | 2.7.10.1 | Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Tyr_kinase_rcpt_3_CS |
| LRP1B | Other/Unknown | no | LDLR_classB_rpt, EGF-type_Asp/Asn_hydroxyl_site, EGF | |
| MGMT | Enzyme (other) | yes | 2.1.1.63 | MethylDNA_cys_MeTrfase_AS, MethylG_MeTrfase_N, MethylDNA_cys_MeTrfase_DNA-bd |
| ATM | Kinase | yes | 2.7.11.1 | PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom |
| ATRX | Transcription factor | no | SNF2_N, Helicase_C-like, Znf_FYVE_PHD | |
| PIK3CA | Kinase | yes | 2.7.1.137 | PI3K_Ras-bd_dom, PI3/4_kinase_cat_dom, PI3K_accessory_dom |
| PIK3R1 | Kinase | yes | 2.7.1.153 | RhoGAP_dom, SH2, SH3_domain |
| PMS2 | Other/Unknown | no | MutL/Mlh/PMS, DNA_mismatch_S5_2-like, Ribsml_uS5_D2-typ_fold_subgr | |
| POLE | Transcription factor | no | 2.7.7.7 | DNA-dir_DNA_pol_B_exonuc, DNA-dir_DNA_pol_B, RNaseH-like_sf |
| PTEN | Phosphatase | yes | 3.1.3.16 | Tyr_Pase_dom, Tyr_Pase_cat, Tensin_C2-dom |
| RB1 | Other/Unknown | no | RB_B, RB_A, Cyclin-like_dom |
Expression context
Cohort genes with no expression data: 0.
23 cohort genes are a single-cell marker in ≥1 SCXA experiment.
Breadth distribution (Bgee present_calls)
| Bucket | Genes |
|---|---|
| narrow (1-5 tissues) | 0 |
| moderate (6-20) | 0 |
| broad (>20) | 24 |
| unknown | 0 |
Top tissues across cohort
| Tissue | Cohort genes |
|---|---|
| calcaneal tendon | 7 |
| colonic epithelium | 5 |
| ventricular zone | 4 |
| ganglionic eminence | 3 |
| adrenal tissue | 3 |
| endothelial cell | 3 |
| male germ line stem cell (sensu Vertebrata) in testis | 2 |
| secondary oocyte | 2 |
| jejunal mucosa | 2 |
| tendon of biceps brachii | 2 |
| corpus epididymis | 2 |
| buccal mucosa cell | 1 |
| olfactory bulb | 1 |
| stromal cell of endometrium | 1 |
| type B pancreatic cell | 1 |
| cervix squamous epithelium | 1 |
| parotid gland | 1 |
| pituitary gland | 1 |
| sural nerve | 1 |
| tibia | 1 |
Per-gene tissue summary (top 30)
| Symbol | Bgee breadth | FANTOM5 breadth | SCXA | Top tissues |
|---|---|---|---|---|
| BRAF | 265 | ubiquitous | marker | buccal mucosa cell, colonic epithelium, calcaneal tendon |
| BRCA2 | 184 | ubiquitous | marker | male germ line stem cell (sensu Vertebrata) in testis, secondary oocyte, ventricular zone |
| SOS1 | 289 | ubiquitous | marker | colonic epithelium, jejunal mucosa, tendon of biceps brachii |
| TERT | 105 | broad | yes | stromal cell of endometrium, type B pancreatic cell, olfactory bulb |
| TP53 | 223 | ubiquitous | marker | ventricular zone, ganglionic eminence, tendon of biceps brachii |
| CDKN2A | 220 | ubiquitous | marker | parotid gland, cervix squamous epithelium, pituitary gland |
| SLTM | 291 | ubiquitous | marker | calcaneal tendon, sural nerve, tibia |
| EGFR | 285 | ubiquitous | marker | nipple, gingiva, gingival epithelium |
| EZH2 | 216 | ubiquitous | marker | ganglionic eminence, ventricular zone, embryo |
| H3-3A | 134 | ubiquitous | marker | ganglionic eminence, monocyte, ventricular zone |
| H3C2 | 94 | ubiquitous | marker | adrenal tissue, colonic epithelium, bone marrow cell |
| IDH1 | 294 | ubiquitous | marker | corpus epididymis, jejunal mucosa, adrenal tissue |
| IDH2 | 292 | ubiquitous | marker | apex of heart, gastrocnemius, hindlimb stylopod muscle |
| KIT | 263 | broad | marker | lateral nuclear group of thalamus, secondary oocyte, oocyte |
| LRP1B | 194 | broad | marker | endothelial cell, Brodmann (1909) area 23, cortical plate |
| MGMT | 261 | ubiquitous | marker | right lobe of liver, liver, endometrium epithelium |
| ATM | 286 | ubiquitous | marker | calcaneal tendon, colonic epithelium, corpus callosum |
| ATRX | 294 | ubiquitous | marker | endothelial cell, calcaneal tendon, colonic epithelium |
| PIK3CA | 284 | ubiquitous | marker | calcaneal tendon, adrenal tissue, tendon |
| PIK3R1 | 294 | ubiquitous | marker | calcaneal tendon, caput epididymis, corpus epididymis |
| PMS2 | 143 | ubiquitous | marker | thymus, prefrontal cortex, male germ line stem cell (sensu Vertebrata) in testis |
| POLE | 221 | ubiquitous | marker | right hemisphere of cerebellum, right testis, cerebellar hemisphere |
| PTEN | 256 | ubiquitous | marker | sperm, endothelial cell, calcaneal tendon |
| RB1 | 287 | ubiquitous | marker | epithelium of nasopharynx, choroid plexus epithelium, visceral pleura |
Protein interactions among cohort
Intra-cohort edges: 48.
Hub genes (top 10 by interactor count)
| Symbol | Interactor count |
|---|---|
| TP53 | 22,736 |
| EGFR | 18,421 |
| PTEN | 11,626 |
| EZH2 | 9,646 |
| CDKN2A | 9,311 |
| BRAF | 7,394 |
| ATM | 7,383 |
| KIT | 6,087 |
| ATRX | 5,796 |
| TERT | 5,717 |
Intra-cohort edges
| A | B | Sources |
|---|---|---|
| ATM | BRCA2 | string_interaction |
| ATM | TP53 | biogrid_interaction, string_interaction |
| ATRX | BRCA2 | string_interaction |
| ATRX | EZH2 | intact |
| ATRX | H3-3A | intact |
| ATRX | IDH1 | string_interaction |
| ATRX | MGMT | string_interaction |
| ATRX | TP53 | string_interaction |
| BRAF | BRCA2 | biogrid_interaction |
| BRAF | CDKN2A | string_interaction |
| BRAF | EGFR | biogrid_interaction |
| BRAF | PIK3CA | biogrid_interaction, string_interaction |
| BRAF | PMS2 | string_interaction |
| BRAF | POLE | intact |
| BRAF | PTEN | biogrid_interaction, string_interaction |
| BRAF | SOS1 | string_interaction |
| BRAF | TP53 | string_interaction |
| BRCA2 | PMS2 | string_interaction |
| BRCA2 | TP53 | string_interaction |
| CDKN2A | MGMT | string_interaction |
| CDKN2A | RB1 | string_interaction |
| CDKN2A | TP53 | string_interaction |
| EGFR | IDH2 | biogrid_interaction |
| EGFR | MGMT | string_interaction |
| EGFR | PIK3CA | string_interaction |
| EGFR | PIK3R1 | string_interaction |
| EGFR | PTEN | string_interaction |
| EGFR | SOS1 | intact, string_interaction |
| IDH1 | IDH2 | biogrid_interaction |
| IDH1 | MGMT | string_interaction |
| IDH1 | PTEN | string_interaction |
| IDH1 | TP53 | string_interaction |
| IDH2 | MGMT | string_interaction |
| KIT | PIK3CA | biogrid_interaction |
| KIT | PIK3R1 | biogrid_interaction, intact |
| KIT | TP53 | biogrid_interaction |
| LRP1B | PIK3CA | string_interaction |
| LRP1B | TP53 | string_interaction |
| MGMT | PMS2 | string_interaction |
| MGMT | TP53 | string_interaction |
| PIK3CA | PIK3R1 | biogrid_interaction, intact, string_interaction |
| PIK3CA | PTEN | string_interaction |
| PIK3CA | SLTM | intact |
| PIK3R1 | PTEN | string_interaction |
| PIK3R1 | TP53 | string_interaction |
| PMS2 | POLE | string_interaction |
| PTEN | TP53 | string_interaction |
| RB1 | TP53 | string_interaction |
Structural data
PDB: 22 · AlphaFold-only: 2 · No structure: 0
Cohort genes with PDB structures (top 30)
| Symbol | UniProt | PDB entries |
|---|---|---|
| H3C2 | P68431 | 548 |
| EGFR | P00533 | 388 |
| TP53 | P04637 | 313 |
| PIK3CA | P42336 | 135 |
| BRAF | P15056 | 131 |
| PIK3R1 | P27986 | 105 |
| H3-3A | P84243 | 103 |
| SOS1 | Q07889 | 91 |
| IDH1 | O75874 | 61 |
| KIT | P10721 | 52 |
| EZH2 | Q15910 | 38 |
| TERT | O14746 | 23 |
| MGMT | P16455 | 23 |
| RB1 | P06400 | 19 |
| POLE | Q07864 | 18 |
| BRCA2 | P51587 | 14 |
| ATM | Q13315 | 14 |
| ATRX | P46100 | 12 |
| PTEN | P60484 | 12 |
| IDH2 | P48735 | 11 |
| PMS2 | P54278 | 9 |
| CDKN2A | P42771 | 5 |
AlphaFold-only cohort genes (top 30 by pLDDT)
| Symbol | UniProt | pLDDT |
|---|---|---|
| SLTM | Q9NWH9 | 52.38 |
| LRP1B | Q9NZR2 |
Function
Pathway analysis
Distinct Reactome pathways touched by cohort: 474. Enrichment computed across 24 evidence-associated genes (22 with Reactome annotation).
Pathways by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 22 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| Pathway | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| Constitutive Signaling by EGFRvIII | 4 | 129.8× | 6e-06 | SOS1, EGFR, PIK3CA, PIK3R1 |
| Signaling by ERBB2 ECD mutants | 4 | 122.1× | 6e-06 | SOS1, EGFR, PIK3CA, PIK3R1 |
| Constitutive Signaling by Ligand-Responsive EGFR Cancer Variants | 4 | 103.8× | 8e-06 | SOS1, EGFR, PIK3CA, PIK3R1 |
| Signaling by phosphorylated juxtamembrane, extracellular and kinase domain KIT mutants | 4 | 94.4× | 9e-06 | SOS1, KIT, PIK3CA, PIK3R1 |
| Signaling by ERBB2 KD Mutants | 4 | 76.9× | 2e-05 | SOS1, EGFR, PIK3CA, PIK3R1 |
| IRS-mediated signalling | 3 | 141.6× | 7e-05 | SOS1, PIK3CA, PIK3R1 |
| Signaling by FGFR4 in disease | 3 | 129.8× | 7e-05 | SOS1, PIK3CA, PIK3R1 |
| Signaling by PDGFRA transmembrane, juxtamembrane and kinase domain mutants | 3 | 119.8× | 7e-05 | SOS1, PIK3CA, PIK3R1 |
| Signaling by PDGFRA extracellular domain mutants | 3 | 119.8× | 7e-05 | SOS1, PIK3CA, PIK3R1 |
| Signaling by LTK | 3 | 119.8× | 7e-05 | SOS1, PIK3CA, PIK3R1 |
| Signaling by SCF-KIT | 4 | 45.1× | 7e-05 | SOS1, KIT, PIK3CA, PIK3R1 |
| RAF/MAP kinase cascade | 6 | 16.7× | 7e-05 | BRAF, SOS1, EGFR, KIT, PIK3CA, PIK3R1 |
| Stabilization of p53 | 3 | 103.8× | 9e-05 | TP53, CDKN2A, ATM |
| Signaling by FLT3 ITD and TKD mutants | 3 | 103.8× | 9e-05 | SOS1, PIK3CA, PIK3R1 |
| Oxidative Stress Induced Senescence | 5 | 20.6× | 1e-04 | TP53, CDKN2A, EZH2, H3-3A, H3C2 |
| PI3K events in ERBB2 signaling | 3 | 91.6× | 1e-04 | EGFR, PIK3CA, PIK3R1 |
| GAB1 signalosome | 3 | 86.5× | 1e-04 | EGFR, PIK3CA, PIK3R1 |
| Tie2 Signaling | 3 | 82.0× | 1e-04 | SOS1, PIK3CA, PIK3R1 |
| Role of LAT2/NTAL/LAB on calcium mobilization | 3 | 82.0× | 1e-04 | SOS1, PIK3CA, PIK3R1 |
| Signaling by FLT3 fusion proteins | 3 | 77.9× | 2e-04 | SOS1, PIK3CA, PIK3R1 |
| Signaling by FGFR3 in disease | 3 | 67.7× | 2e-04 | SOS1, PIK3CA, PIK3R1 |
| Interleukin receptor SHC signaling | 3 | 55.6× | 4e-04 | SOS1, PIK3CA, PIK3R1 |
| Meiotic recombination | 4 | 23.6× | 4e-04 | BRCA2, H3-3A, H3C2, ATM |
| Downstream signal transduction | 3 | 51.9× | 5e-04 | SOS1, PIK3CA, PIK3R1 |
| Constitutive Signaling by Aberrant PI3K in Cancer | 4 | 23.1× | 5e-04 | EGFR, KIT, PIK3CA, PIK3R1 |
| DAP12 signaling | 3 | 50.2× | 5e-04 | SOS1, PIK3CA, PIK3R1 |
| FLT3 Signaling | 3 | 47.2× | 6e-04 | SOS1, PIK3CA, PIK3R1 |
| Signaling by CSF1 (M-CSF) in myeloid cells | 3 | 47.2× | 6e-04 | SOS1, PIK3CA, PIK3R1 |
| Oncogene Induced Senescence | 3 | 45.8× | 6e-04 | TP53, CDKN2A, RB1 |
| Interleukin-3, Interleukin-5 and GM-CSF signaling | 3 | 43.3× | 7e-04 | SOS1, PIK3CA, PIK3R1 |
GO biological processes by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 24 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| GO term | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| replicative senescence | 4 | 165.2× | 5e-06 | TERT, TP53, CDKN2A, ATM |
| negative regulation of glial cell proliferation | 3 | 210.7× | 9e-05 | TP53, IDH2, RB1 |
| subtelomeric heterochromatin formation | 3 | 191.5× | 9e-05 | EZH2, H3-3A, ATRX |
| DNA damage response, signal transduction by p53 class mediator | 4 | 59.8× | 9e-05 | BRCA2, TP53, ATM, ATRX |
| multicellular organism growth | 5 | 28.5× | 9e-05 | SOS1, TP53, H3-3A, ATM, ATRX |
| cellular senescence | 4 | 49.3× | 1e-04 | BRCA2, TP53, CDKN2A, ATM |
| glyoxylate cycle | 2 | 702.2× | 2e-04 | IDH1, IDH2 |
| epidermal growth factor receptor signaling pathway | 4 | 41.3× | 2e-04 | BRAF, SOS1, EGFR, PIK3CA |
| phosphatidylinositol 3-kinase/protein kinase B signal transduction | 4 | 35.1× | 3e-04 | EGFR, PIK3CA, PIK3R1, PTEN |
| Ras protein signal transduction | 4 | 34.2× | 3e-04 | SOS1, TP53, CDKN2A, RB1 |
| insulin-like growth factor receptor signaling pathway | 3 | 62.0× | 9e-04 | SOS1, PIK3CA, PIK3R1 |
| isocitrate metabolic process | 2 | 280.9× | 9e-04 | IDH1, IDH2 |
| negative regulation of mammary gland epithelial cell proliferation | 2 | 280.9× | 9e-04 | BRCA2, CDKN2A |
| protein stabilization | 5 | 13.9× | 0.001 | TP53, CDKN2A, ATM, PIK3R1, PTEN |
| myeloid progenitor cell differentiation | 2 | 200.6× | 0.002 | BRAF, KIT |
| negative regulation of G1/S transition of mitotic cell cycle | 3 | 44.8× | 0.002 | EZH2, PTEN, RB1 |
| establishment of protein localization to telomere | 2 | 175.5× | 0.002 | BRCA2, TERT |
| epithelial cell proliferation | 3 | 39.0× | 0.002 | EGFR, KIT, RB1 |
| positive regulation of miRNA transcription | 3 | 36.3× | 0.002 | TERT, TP53, EGFR |
| telomere maintenance via recombination | 2 | 127.7× | 0.003 | BRCA2, TERT |
| NADP+ metabolic process | 2 | 127.7× | 0.003 | IDH1, IDH2 |
| positive regulation of transcription by RNA polymerase II | 8 | 5.0× | 0.003 | TP53, CDKN2A, EGFR, ATM, ATRX, PIK3R1, PTEN, RB1 |
| somatic stem cell population maintenance | 3 | 31.0× | 0.003 | BRAF, CDKN2A, KIT |
| cellular response to xenobiotic stimulus | 3 | 30.1× | 0.003 | BRAF, TP53, RB1 |
| insulin receptor signaling pathway | 3 | 27.7× | 0.004 | SOS1, PIK3CA, PIK3R1 |
| B cell differentiation | 3 | 27.4× | 0.004 | EZH2, KIT, PIK3R1 |
| eyelid development in camera-type eye | 2 | 87.8× | 0.005 | SOS1, EGFR |
| double-strand break repair | 3 | 25.4× | 0.005 | BRCA2, TP53, ATM |
| G1/S transition of mitotic cell cycle | 3 | 25.1× | 0.005 | EZH2, POLE, RB1 |
| rRNA transcription | 2 | 82.6× | 0.005 | TP53, CDKN2A |
Therapeutics
Drug target analysis
Approved (phase 4): 13 · Phase ≥3: 14 · Phased (≥1): 15 · Undrugged: 9
Druggability breadth: 20 of 24 evidence-associated genes (83%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).
Genes with an approved drug
The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.
| Symbol | Example approved molecule |
|---|---|
| BRAF | VEMURAFENIB |
| SOS1 | IDARUBICIN |
| TERT | BERBERINE |
| TP53 | NITROFURANTOIN |
| SLTM | CABOZANTINIB |
| EGFR | LEVODOPA |
| EZH2 | TAZEMETOSTAT |
| IDH1 | ENASIDENIB |
| IDH2 | ENASIDENIB |
| KIT | PONATINIB |
| ATM | AMIODARONE HYDROCHLORIDE |
| PIK3CA | IDELALISIB |
| PIK3R1 | IDELALISIB |
Top cohort targets by molecule count
| Symbol | Molecules | Max phase |
|---|---|---|
| TP53 | 196 | 4 |
| EGFR | 175 | 4 |
| KIT | 99 | 4 |
| PIK3CA | 67 | 4 |
| BRAF | 48 | 4 |
| ATM | 35 | 4 |
| PIK3R1 | 26 | 4 |
| TERT | 10 | 4 |
| IDH1 | 10 | 4 |
| IDH2 | 7 | 4 |
Drugs targeting cohort genes (top 30)
| Molecule | Max phase | Targets in cohort |
|---|---|---|
| VEMURAFENIB | 4 | BRAF, EGFR |
| PONATINIB | 4 | BRAF, EGFR, KIT |
| FEDRATINIB | 4 | BRAF, EGFR, KIT, PIK3CA |
| SORAFENIB | 4 | BRAF, EGFR, KIT |
| DASATINIB ANHYDROUS | 4 | BRAF, EGFR, KIT |
| RUXOLITINIB | 4 | BRAF, KIT |
| INFIGRATINIB PHOSPHATE | 4 | BRAF, KIT |
| INFIGRATINIB | 4 | BRAF, KIT |
| REGORAFENIB | 4 | BRAF, KIT |
| DABRAFENIB | 4 | BRAF |
| COBIMETINIB | 4 | BRAF |
| NILOTINIB | 4 | BRAF, KIT |
| ABEMACICLIB | 4 | BRAF, EGFR |
| ENCORAFENIB | 4 | BRAF |
| TOVORAFENIB | 4 | BRAF |
| PAZOPANIB | 4 | BRAF, KIT |
| DASATINIB | 4 | BRAF, EGFR, KIT, PIK3CA |
| ERLOTINIB | 4 | BRAF, EGFR, KIT |
| GEFITINIB | 4 | BRAF, EGFR, KIT |
| IMATINIB | 4 | BRAF, EGFR, KIT |
| IDARUBICIN | 4 | SOS1 |
| DOXORUBICIN | 4 | SOS1, TERT |
| SOTORASIB | 4 | SOS1 |
| ADAGRASIB | 4 | SOS1 |
| BERBERINE | 4 | TERT |
| NITROFURANTOIN | 4 | TP53 |
| DIOSMIN | 4 | TP53 |
| VERTEPORFIN | 4 | TP53 |
| CANDESARTAN CILEXETIL | 4 | TP53 |
| DIENESTROL | 4 | TP53 |
Bioactivity and enzyme data
Enzyme cohort genes (≥1 EC): 12.
Cohort genes with ChEMBL bioactivity (full, sorted by assay count)
| Symbol | Assays | Type breakdown |
|---|---|---|
| EGFR | 6,531 | Binding:6211, Functional:173, ADMET:138, Toxicity:9 |
| KIT | 2,305 | Binding:2242, ADMET:32, Functional:22, Toxicity:9 |
| PIK3CA | 2,034 | Binding:2009, ADMET:19, Toxicity:4, Functional:2 |
| BRAF | 1,442 | Binding:1400, Functional:37, ADMET:5 |
| TP53 | 869 | Binding:775, ADMET:83, Functional:10, Toxicity:1 |
| EZH2 | 839 | Binding:833, Functional:6 |
| PIK3R1 | 493 | Binding:470, ADMET:23 |
| IDH1 | 488 | Binding:475, Functional:12, ADMET:1 |
| SOS1 | 421 | Binding:409, Functional:12 |
| TERT | 391 | Binding:389, Functional:2 |
| ATM | 240 | Binding:233, Functional:5, ADMET:2 |
| MGMT | 86 | Binding:84, ADMET:2 |
| IDH2 | 84 | Binding:84 |
| RB1 | 59 | Binding:59 |
| SLTM | 14 | Binding:14 |
| PTEN | 8 | Binding:8 |
| H3-3A | 6 | Binding:6 |
| CDKN2A | 2 | Binding:2 |
| PMS2 | 1 | Binding:1 |
Cohort enzymes (BRENDA EC)
| Symbol | EC numbers | Names |
|---|---|---|
| BRAF | 2.7.10.2, 2.7.11.1 | non-specific protein-tyrosine kinase, non-specific serine/threonine protein kinase |
| EGFR | 2.7.10.1 | receptor protein-tyrosine kinase |
| EZH2 | 2.1.1.356 | [histone H3]-lysine27 N-trimethyltransferase |
| IDH1 | 1.1.1.42 | isocitrate dehydrogenase (NADP+) |
| IDH2 | 1.1.1.42 | isocitrate dehydrogenase (NADP+) |
| KIT | 2.7.10.1 | receptor protein-tyrosine kinase |
| MGMT | 2.1.1.63 | methylated-DNA-[protein]-cysteine S-methyltransferase |
| ATM | 2.7.11.1 | non-specific serine/threonine protein kinase |
| PIK3CA | 2.7.1.137, 2.7.1.153, 2.7.11.1 | phosphatidylinositol 3-kinase, phosphatidylinositol-4,5-bisphosphate 3-kinase, non-specific serine/threonine protein kinase |
| PIK3R1 | 2.7.1.153 | phosphatidylinositol-4,5-bisphosphate 3-kinase |
| POLE | 2.7.7.7 | DNA-directed DNA polymerase |
| PTEN | 3.1.3.16, 3.1.3.67 | protein-serine/threonine phosphatase, phosphatidylinositol-3,4,5-trisphosphate 3-phosphatase |
Cohort genes with high screening signal
≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.
| Symbol | ChEMBL assays |
|---|---|
| BRAF | 1,442 |
| SOS1 | 421 |
| TERT | 391 |
| TP53 | 869 |
| EGFR | 6,531 |
| EZH2 | 839 |
| IDH1 | 488 |
| KIT | 2,305 |
| ATM | 240 |
| PIK3CA | 2,034 |
| PIK3R1 | 493 |
Pharmacogenomics
Cohort genes with a PharmGKB record: 24; with CPIC/DPWG dosing guidelines: 0.
No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).
Chemical tractability of cohort targets
26 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.
| Compound | Max phase | Cohort target (bioactivity) |
|---|---|---|
| VEMURAFENIB | 4 | BRAF, EGFR |
| PONATINIB | 4 | BRAF, EGFR, KIT |
| FEDRATINIB | 4 | BRAF, EGFR, KIT, PIK3CA |
| SORAFENIB | 4 | BRAF, EGFR, KIT |
| DASATINIB ANHYDROUS | 4 | BRAF, EGFR, KIT |
| RUXOLITINIB | 4 | BRAF, KIT |
| INFIGRATINIB PHOSPHATE | 4 | BRAF, KIT |
| INFIGRATINIB | 4 | BRAF, KIT |
| REGORAFENIB | 4 | BRAF, KIT |
| COBIMETINIB | 4 | BRAF |
| NILOTINIB | 4 | BRAF, KIT |
| ENCORAFENIB | 4 | BRAF |
| TOVORAFENIB | 4 | BRAF |
| DASATINIB | 4 | BRAF, EGFR, KIT, PIK3CA |
| ERLOTINIB | 4 | BRAF, EGFR, KIT |
| IMATINIB | 4 | BRAF, EGFR, KIT |
| IDARUBICIN | 4 | SOS1 |
| DOXORUBICIN | 4 | SOS1, TERT |
| SOTORASIB | 4 | SOS1 |
| ADAGRASIB | 4 | SOS1 |
| BERBERINE | 4 | TERT |
| NITROFURANTOIN | 4 | TP53 |
| DIOSMIN | 4 | TP53 |
| VERTEPORFIN | 4 | TP53 |
| CANDESARTAN CILEXETIL | 4 | TP53 |
| DIENESTROL | 4 | TP53 |
Druggability pyramid
Cohort genes binned by druggability tier (high → low):
| Tier | Definition | Genes | Symbols |
|---|---|---|---|
| A | Approved (phase 4 drug) | 13 | BRAF, SOS1, TERT, TP53, SLTM, EGFR, EZH2, IDH1, IDH2, KIT (+3 more) |
| B | Phased (≥1) drug, not yet approved | 2 | MGMT, RB1 |
| C | Druggable family + PDB, no drug | 1 | PTEN |
| D | Druggable family + AlphaFold only, no drug | 0 | |
| E | Difficult family or no structure, no drug | 8 | BRCA2, CDKN2A, H3-3A, H3C2, LRP1B, ATRX, PMS2, POLE |
Undrugged target profiles
9 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).
| Symbol | ChEMBL assays | Drugged partners (top 3) |
|---|---|---|
| PTEN | 8 | TP53 |
| BRCA2 | 0 | — |
| CDKN2A | 2 | — |
| H3-3A | 6 | — |
| H3C2 | 0 | — |
| LRP1B | 0 | — |
| ATRX | 0 | — |
| PMS2 | 1 | — |
| POLE | 0 | — |
Clinical trials & evidence
Clinical trials
Clinical trials: 1,139.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| PHASE2 | 307 |
| PHASE1 | 279 |
| Not specified | 254 |
| PHASE1/PHASE2 | 123 |
| EARLY_PHASE1 | 66 |
| PHASE3 | 52 |
| PHASE2/PHASE3 | 13 |
| PHASE4 | 6 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03975829 | PHASE4 | RECRUITING | Pediatric Long-Term Follow-up and Rollover Study |
| NCT05342883 | PHASE4 | ACTIVE_NOT_RECRUITING | GammaTile and Stupp in Newly Diagnosed GBM |
| NCT07546669 | PHASE4 | NOT_YET_RECRUITING | Efficacy of Zoster Vaccination in Glioblastoma Patients |
| NCT00686725 | PHASE4 | COMPLETED | Standard Temodal (Temozolomide) Regimen Versus Standard Regimen Plus Early Postsurgery Temodal for Newly Diagnosed Glioblastoma Multiforme (Study P05572) |
| NCT05900908 | PHASE4 | WITHDRAWN | Post-operative Adjuvant Therapy w/wo GammaTile + Systemic Therapy |
| NCT06625047 | PHASE4 | COMPLETED | Comparing Telehealth and In-person Assessments in Glioma Patients Receiving Oral Chemotherapy |
| NCT02152982 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | Temozolomide With or Without Veliparib in Treating Patients With Newly Diagnosed Glioblastoma Multiforme |
| NCT02685605 | PHASE3 | ACTIVE_NOT_RECRUITING | Intraoperative Radiotherapy in Newly Diagnosed Glioblastoma Multiforme |
| NCT03663725 | PHASE3 | ACTIVE_NOT_RECRUITING | Treatment Intensification With Temozolomide in Adults With a Glioblastoma |
| NCT03970447 | PHASE2/PHASE3 | RECRUITING | A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma |
| NCT04250922 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | LAM561 With RT and TMZ for Adults With Glioblastoma |
| NCT05095376 | PHASE3 | ACTIVE_NOT_RECRUITING | Testing the Addition of the Chemotherapy Drug Lomustine (Gleostine) to the Usual Treatment (Temozolomide and Radiation Therapy) for Newly Diagnosed MGMT Methylated Glioblastoma |
| NCT05100641 | PHASE3 | NOT_YET_RECRUITING | AV-GBM-1 vs Control as Adjunctive Therapy Following Surgery and RT/TMZ in Newly Diagnosed GBM |
| NCT05118776 | PHASE3 | ACTIVE_NOT_RECRUITING | Study to Evaluate the Safety and Efficacy of ASC40 Tablets in Combination With Bevacizumab in Subjects With rGBM |
| NCT05271240 | PHASE3 | RECRUITING | Repeated Superselective Intraarterial Cerebral Infusion (SIACI) of Bevacizumab With Temozolomide and Radiation Compared to Temozolomide and Radiation Alone in Newly Diagnosed GBM |
| NCT05318612 | PHASE3 | ACTIVE_NOT_RECRUITING | Effectiveness of MR-guided LITT Therapy in Irresectable Glioblastoma (EMITT) |
| NCT05326464 | PHASE3 | ACTIVE_NOT_RECRUITING | Tofacitinib in Recurrent GBM Patients |
| NCT05439278 | PHASE3 | RECRUITING | Conventional Versus Hypofractionated Radiotherapy With Temozolomide in Elderly Glioblastoma |
| NCT05669820 | PHASE2/PHASE3 | RECRUITING | Antisecretory Factor Glioblastoma Phase 2 |
| NCT05902169 | PHASE3 | RECRUITING | Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM |
| NCT06105619 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | A Study of PLB1001 Enteric Capsules in the Treatment of sGBM/IDH Mutant Glioblastoma Patients With the ZM Fusion Gene (FUGEN). |
| NCT06388733 | PHASE3 | RECRUITING | A Study Comparing Niraparib With Temozolomide in Adult Participants With Newly-diagnosed, MGMT Unmethylated Glioblastoma |
| NCT06448286 | PHASE3 | NOT_YET_RECRUITING | PH Weighted Chemical Exchange Saturation Transfer MRI-Based Surgical Resection to Improve Survival in Patients With Glioblastoma |
| NCT06477939 | PHASE3 | NOT_YET_RECRUITING | Study of Adding or Not Liposomal Transcrocetin (L-TC) With Concomitant HypoFractionated Radiation ThErapy and TEmozolomide in Newly Diagnosed GLioblastoma (GBM) Patients |
| NCT06496971 | PHASE3 | RECRUITING | A Prospective Pivotal Study to Evaluate the Efficacy and Safety of Avastin® Bevacizumab (BEV) With or Without Microbubble-mediated Focused Ultrasound (FUS-MB) Using NaviFUS System in Recurrent Glioblastoma Multiforme Patients |
| NCT06556563 | PHASE3 | RECRUITING | EF-41/KEYNOTE D58: Phase 3 Study of Optune Concomitant With Temozolomide Plus Pembrolizumab in Newly Diagnosed Glioblastoma |
| NCT06749925 | PHASE3 | NOT_YET_RECRUITING | Clinical Trial Assessing the Efficacy and Safety of Dendritic Cell-Based Immunotherapy for Glioblastoma |
| NCT07100730 | PHASE3 | RECRUITING | Study of TLX101-Tx Plus Standard of Care (SoC) Versus SoC Alone for the Treatment of Patients With Recurrent Glioblastoma |
| NCT07195591 | PHASE3 | RECRUITING | Beginning Radiation Immediately With GammaTile at GBM Excision Versus Standard of Care |
| NCT07461948 | PHASE3 | RECRUITING | Advanced Imaging Techniques for Evaluating the Tumor Immune Microenvironment in Glioblastoma Patients |
| NCT07605364 | PHASE2/PHASE3 | NOT_YET_RECRUITING | Testing the Addition of an Anti-Cancer Drug, Mycophenolate Mofetil, to the Usual Treatment (Radiation Therapy and Temozolomide) for Advanced Brain Cancer |
| NCT00045968 | PHASE3 | UNKNOWN | Study of a Drug [DCVax®-L] to Treat Newly Diagnosed GBM Brain Cancer |
| NCT00068952 | PHASE3 | COMPLETED | Study of IV Edotecarin Vs Temozolomide or Carmustine (BCNU) or Lomustine (CCNU) in Patients With Glioblastoma Multiforme |
| NCT00088400 | PHASE3 | COMPLETED | Comparison of TransMID vs Standard Treatment of Cancerous Brain Tumors |
| NCT00295815 | PHASE3 | COMPLETED | Enzastaurin Versus Lomustine in Glioblastoma |
| NCT00335075 | PHASE3 | COMPLETED | Efficacy and Safety of Temodal vs Semustine in Subjects With Recurrent Glioblastoma or Anaplastic Astrocytoma (Study P03644) |
| NCT00689221 | PHASE3 | COMPLETED | Cilengitide, Temozolomide, and Radiation Therapy in Treating Patients With Newly Diagnosed Glioblastoma and Methylated Gene Promoter Status |
| NCT00761280 | PHASE3 | TERMINATED | Efficacy and Safety of AP 12009 in Patients With Recurrent or Refractory Anaplastic Astrocytoma or Secondary Glioblastoma |
| NCT00777153 | PHASE3 | COMPLETED | Cediranib in Combination With Lomustine Chemotherapy in Recurrent Glioblastoma |
| NCT00807027 | PHASE3 | COMPLETED | Clinical Trial to Assess the Efficacy and Safety of ‘Immuncell-LC’ With Temozolomide in Newly Diagnosed Glioblastoma of Korea |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| TEMOZOLOMIDE | 4 | 35 |
| LOMUSTINE | 4 | 30 |
| AMINOLEVULINIC ACID | 4 | 6 |
| DISULFIRAM | 4 | 6 |
| PLERIXAFOR | 4 | 6 |
| ERLOTINIB HYDROCHLORIDE | 4 | 5 |
| PAZOPANIB | 4 | 5 |
| TEMSIROLIMUS | 4 | 5 |
| FERUMOXYTOL | 4 | 4 |
| NIRAPARIB | 4 | 4 |
| SORAFENIB TOSYLATE | 4 | 4 |
| VORINOSTAT | 4 | 4 |
| ABEMACICLIB | 4 | 3 |
| CARMUSTINE | 4 | 3 |
| DURVALUMAB | 4 | 3 |
| FLUDEOXYGLUCOSE F 18 | 4 | 3 |
| TRAMETINIB | 4 | 3 |
| CERITINIB | 4 | 2 |
| CHLOROQUINE | 4 | 2 |
| COPPER | 4 | 2 |
| DABRAFENIB | 4 | 2 |
| DACOMITINIB ANHYDROUS | 4 | 2 |
| ETOPOSIDE PHOSPHATE | 4 | 2 |
| GEFITINIB | 4 | 2 |
| HYDROXYUREA | 4 | 2 |
| MANNITOL | 4 | 2 |
| NINTEDANIB | 4 | 2 |
| SORBITOL | 4 | 2 |
| SUNITINIB MALATE | 4 | 2 |
| AURANOFIN | 4 | 1 |
Precision-medicine subtype map (CIViC)
Drug × molecular subtype: 68 predictive associations from 71 curated evidence items; also 18 prognostic, 17 diagnostic, 11 oncogenic.
| Molecular subtype | Therapy | Effect | Level | CIViC |
|---|---|---|---|---|
| MGMT Promoter Methylation | Temozolomide | Sensitivity/Response | CIViC A | EID307 |
| MGMT Promoter Methylation | Lomustine + Temozolomide | Sensitivity/Response | CIViC A | EID7303 |
| IDH1 R132 | Cetuximab | Sensitivity/Response | CIViC B | EID3718 +1 |
| MGMT Underexpression | Temozolomide | Sensitivity/Response | CIViC B | EID2899 +1 |
| DRD5 low expression | Dordaviprone | Sensitivity/Response | CIViC B | EID7600 |
| EGFR Expression | Temozolomide + Nimotuzumab + 3-Dimensional Conformal Radiation Therapy | Sensitivity/Response | CIViC B | EID8299 |
| EGFR Overexpression | Lomustine + Bevacizumab | Sensitivity/Response | CIViC B | EID10894 |
| EGFR VIII | Erlotinib + Gefitinib | Sensitivity/Response | CIViC B | EID1128 |
| EGFR VIII | Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitor | Sensitivity/Response | CIViC B | EID772 |
| EGFR VIII | Rindopepimut | Sensitivity/Response | CIViC B | EID971 |
| EGFR::VSTM2A Fusion | Bevacizumab/Lomustine Regimen | Sensitivity/Response | CIViC B | EID11101 |
| IDH1 R132S | Cetuximab | Sensitivity/Response | CIViC B | EID4033 |
| KIT EXPRESSION | Sunitinib | Sensitivity/Response | CIViC B | EID10180 |
| MGMT Promoter Methylation | Carmustine | Sensitivity/Response | CIViC B | EID308 |
| MGMT RS16906252 | Temozolomide | Sensitivity/Response | CIViC B | EID822 |
| PIK3CA Mutation | Buparlisib | Sensitivity/Response | CIViC B | EID7072 |
| PIK3R1 Mutation | Buparlisib | Sensitivity/Response | CIViC B | EID7073 |
| PTEN Expression | Erlotinib + Gefitinib | Sensitivity/Response | CIViC B | EID1129 |
| PTEN Expression | Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitor | Sensitivity/Response | CIViC B | EID774 |
| PTEN Mutation | Buparlisib | Sensitivity/Response | CIViC B | EID7074 |
| SLC12A2 OVEREXPRESSION | Antisecretory Factor-enriched Egg Yolk Powder Supplement + Temozolomide | Sensitivity/Response | CIViC B | EID12689 |
| VEGFA Overexpression | Bevacizumab | Sensitivity/Response | CIViC B | EID7013 |
| PTEN Mutation | Nivolumab + Pembrolizumab | Resistance | CIViC B | EID8176 |
| TP53 Mutation | Temozolomide | Resistance | CIViC B | EID7978 |
| VEGFA Overexpression of VEGF121 | Bevacizumab | Resistance | CIViC B | EID9490 |
| ALK Expression | Crizotinib | Sensitivity/Response | CIViC C | EID7866 |
| ARHGEF2::NTRK1 Fusion | Entrectinib | Sensitivity/Response | CIViC C | EID12606 |
| BRAF V600E | Vemurafenib | Sensitivity/Response | CIViC C | EID3771 |
| BRCA2 K3326* | Olaparib + Temozolomide | Sensitivity/Response | CIViC C | EID7724 |
| EGFR Amplification AND EGFR EGFRVIII | Afatinib | Sensitivity/Response | CIViC C | EID773 |
+38 more predictive associations (showing top 30 by evidence level).
Related Atlas pages
- Cohort genes: BRAF, BRCA2, SOS1, TERT, TP53, CDKN2A, SLTM, EGFR, EZH2, H3-3A, H3C2, IDH1, IDH2, KIT, LRP1B, MGMT, ATM, ATRX, PIK3CA, PIK3R1, PMS2, POLE, PTEN, RB1
- Drugs: Temozolomide, Lomustine, Aminolevulinic Acid, Disulfiram, Plerixafor, Erlotinib, Pazopanib, Temsirolimus, Ferumoxytol, Niraparib, Sorafenib Tosylate, Vorinostat, Abemaciclib, Carmustine, Durvalumab, FLUDEOXYGLUCOSE F 18, Trametinib, Ceritinib, Chloroquine, Copper, Dabrafenib, Dacomitinib, Etoposide Phosphate, Gefitinib, Hydroxyurea, Mannitol, Nintedanib, Sorbitol, Sunitinib Malate, Auranofin, Cetuximab, Dordaviprone, Rindopepimut, Buparlisib, Bevacizumab, Crizotinib, Entrectinib, Vemurafenib, Afatinib