Brain cancer
diseaseOn this page
Also known as adult brain tumoradult brain tumouradult malignant brain neoplasmbrain neoplasmbrain neoplasms, malignantbrain tumor, adultBT - brain tumourcancer of braincancer of the brainmalignant brain neoplasmmalignant brain tumormalignant neoplasm of brainmalignant neoplasm of the brainmalignant primary brain neoplasmmalignant primary brain tumormalignant primary brain tumourmalignant tumor of adult brainmalignant tumor of brainmalignant tumor of the brain
Summary
Brain cancer (MONDO:0001657) is a cancer (an umbrella term covering 9 Mondo subtypes) with 2 cohort genes (7 GWAS associations across 9 studies; 2 CIViC-evidence somatic drivers; 2 ClinVar predisposition records) and 396 clinical trials. Top therapeutic interventions include erlotinib, aminolevulinic acid, and flucytosine.
At a glance
- Classification: Cancer
- Umbrella term: 9 Mondo subtypes
- Cohort genes: 2
- GWAS associations: 7
- ClinVar variants: 2
- Clinical trials: 396
Clinical features
No curated clinical features (Orphanet) for this disease.
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | brain cancer |
| Mondo ID | MONDO:0001657 |
| MeSH | D001932 |
| DOID | DOID:1319 |
| ICD-10-CM | C71 |
| ICD-11 | 189650925 |
| NCIT | C3568 |
| SNOMED CT | 428061005 |
| UMLS | C0153633 |
| MedGen | 57796 |
| GARD | 0027584 |
| Anatomy (UBERON) | UBERON:0000955 |
| Is cancer (heuristic) | yes |
Also known as: adult brain tumor · adult brain tumour · adult malignant brain neoplasm · brain cancer · brain neoplasm · brain neoplasms, malignant · brain tumor, adult · BT - brain tumour · cancer of brain · cancer of the brain · malignant brain neoplasm · malignant brain tumor · malignant neoplasm of brain · malignant neoplasm of the brain · malignant primary brain neoplasm · malignant primary brain tumor · malignant primary brain tumour · malignant tumor of adult brain · malignant tumor of brain · malignant tumor of the brain (+7 more)
Data availability: 2 ClinVar variants · 7 GWAS associations (9 studies) · 1 cell line.
Disease family
An umbrella term covering 9 Mondo subtypes.
Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › cancer › nervous system cancer › central nervous system cancer › brain cancer
Related subtypes (19): central nervous system primitive neuroectodermal neoplasm, central nervous system sarcoma, primary central nervous system lymphoma, central nervous system germinoma, central nervous system melanocytic neoplasm, central nervous system endodermal sinus tumor, spinal cord cancer, malignant carotid body paraganglioma, malignant adrenal gland pheochromocytoma, malignant jugulotympanic paraganglioma, pheochromocytoma/paraganglioma syndrome 2, pheochromocytoma/paraganglioma syndrome 5, central nervous system Ewing sarcoma/peripheral primitive neuroectodermal tumor, choriocarcinoma of the central nervous system, mixed germ cell tumor of central nervous system, embryonal carcinoma of the central nervous system, malignant tumor of meninges, malignant central nervous system mesenchymal, non-meningothelial neoplasm, malignant glioma
Subtypes (9): supratentorial cancer, brain germinoma, brain sarcoma, cerebral ventricle cancer, infratentorial cancer, intracranial primitive neuroectodermal tumor, brain glioma, cancer of cerebellum, metastatic malignant neoplasm in the brain
Genetics & variants
GWAS landscape
7 GWAS associations across 9 studies. Top hits map to 6 distinct genes (as reported by GWAS).
Top associations by p-value
| rsID | p-value | Gene | Risk allele | Odds ratio |
|---|---|---|---|---|
| rs114611496 | 1e-13 | IDE | A | 2.33 |
| rs550254779 | 3e-13 | APOBEC3B-AS1 - APOBEC3C | G | 3.04 |
| rs192247958 | 3e-12 | FCHO2 | G | 3.77 |
| rs573448077 | 7e-12 | FOXN3, FOXN3-AS3 | C | 3.47 |
| rs111754359 | 9e-12 | CCT6B | C | 4.09 |
| rs181334777 | 1e-11 | AFAP1-AS1 | C | 3.03 |
| rs140442570 | 1e-07 | CUBNP2 - OR6D1P | ? |
Top studies (by case count)
| Study | Lead author | Year | Cases | Controls | Title |
|---|---|---|---|---|---|
| GCST90477218 | Verma A | 2024 | 1,417 | 448,701 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90477219 | Verma A | 2024 | 1,199 | 449,265 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90435624 | Zhou W | 2018 | 531 | 407,239 | Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies. |
| GCST90435625 | Zhou W | 2018 | 486 | 407,239 | Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies. |
| GCST90651848 | Liu TY | 2025 | 478 | 234,636 | Diversity and longitudinal records: Genetic architecture of disease associations and polygenic risk in the Taiwanese Han population. |
| GCST90479813 | Verma A | 2024 | 322 | 121,279 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90481519 | Verma A | 2024 | 322 | 121,279 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90479812 | Verma A | 2024 | 252 | 121,443 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
| GCST90481520 | Verma A | 2024 | 252 | 121,443 | Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program. |
Variant details and genetic-evidence tiers
Tier distribution (top 50 variants)
| Tier | Variants |
|---|---|
| Tier 1: coding | 0 |
| Tier 2: splice/UTR | 0 |
| Tier 3: regulatory | 0 |
| Tier 4: intronic/intergenic | 7 |
MAF distribution
| Bucket | Variants |
|---|---|
| common (>=0.05) | 0 |
| low_freq (0.01-0.05) | 0 |
| rare (<0.01) | 6 |
| unknown | 1 |
Functional consequences
| Consequence | Count |
|---|---|
| intron_variant | 4 |
| intergenic_variant | 3 |
Top variants
| rsID | Chr | Pos | Alleles | MAF | Consequence | Gene | p-value | Tier |
|---|---|---|---|---|---|---|---|---|
| rs114611496 | 10 | 92499682 | A>G | 0 | intron_variant | IDE | 1e-13 | Tier 4: intronic/intergenic |
| rs550254779 | 22 | 39012413 | G>A | 0.001 | intergenic_variant | APOBEC3B-AS1 - APOBEC3C | 3e-13 | Tier 4: intronic/intergenic |
| rs192247958 | 5 | 73059783 | G>C | 0 | intron_variant | FCHO2 | 3e-12 | Tier 4: intronic/intergenic |
| rs573448077 | 14 | 89360121 | C>A,T | 0.001 | intron_variant | FOXN3, FOXN3-AS3 | 7e-12 | Tier 4: intronic/intergenic |
| rs111754359 | 17 | 34974171 | C>T | 0 | intergenic_variant | CCT6B | 9e-12 | Tier 4: intronic/intergenic |
| rs181334777 | 4 | 7754890 | C>A,T | 0 | intron_variant | AFAP1-AS1 | 1e-11 | Tier 4: intronic/intergenic |
| rs140442570 | 10 | 45253087 | A>C | intergenic_variant | CUBNP2 - OR6D1P | 1e-07 | Tier 4: intronic/intergenic |
ClinVar germline variants
2 retrieved; paginated sample, class counts are floors:
1 benign/likely benign, 1 uncertain significance
| ClinVar | Variant (HGVS) | Gene | Classification | Review |
|---|---|---|---|---|
| 479094 | NM_000051.4(ATM):c.611G>T (p.Gly204Val) | ATM | Uncertain significance | criteria provided, single submitter |
| 184606 | NM_000455.5(STK11):c.1284G>C (p.Ser428=) | STK11 | Benign/Likely benign | criteria provided, multiple submitters, no conflicts |
Genes & proteins
Mendelian disease overlap and somatic drivers
GenCC: 0 · Orphanet: 9 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0
Somatic driver evidence (intOGen + CIViC, cohort fanout)
| Gene | intOGen role | Cancer types | CIViC |
|---|---|---|---|
| STK11 | LoF | ANSC,CEAD,CESC,CHOL,LUAD,NSCLC,WDTC | CIViC #5534 |
| ATM | LoF | BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,COADREAD,ESCA,HCC,LUAD,LUSC,MEL,NSCLC,PAAD,PANCREAS,PANET,PCM,PLMESO,PRAD,PROSTATE,STAD,UCEC,UTUC,WDTC | CIViC #69 |
Orphanet rare-disease linkage (cohort genes)
| Gene | Orphanet ID | Rare disease |
|---|---|---|
| STK11 | Orphanet:2869 | Peutz-Jeghers syndrome |
| ATM | Orphanet:100 | Ataxia-telangiectasia |
| ATM | Orphanet:1331 | Familial prostate cancer |
| ATM | Orphanet:145 | Hereditary breast and/or ovarian cancer syndrome |
| ATM | Orphanet:227535 | Hereditary breast cancer |
| ATM | Orphanet:370109 | Ataxia-telangiectasia variant |
| ATM | Orphanet:440437 | Familial colorectal cancer Type X |
| ATM | Orphanet:52416 | Mantle cell lymphoma |
| ATM | Orphanet:67038 | B-cell chronic lymphocytic leukemia |
Cohort genes → proteins
2 cohort genes, 2 distinct canonical proteins.
Evidence partition
| Subset | Genes |
|---|---|
| multi_evidence | 2 |
Cohort genes (full)
| Symbol | HGNC | Ensembl | UniProt | Name | Evidence |
|---|---|---|---|---|---|
| STK11 | HGNC:11389 | ENSG00000118046 | Q15831 | Serine/threonine-protein kinase STK11 | clinvar |
| ATM | HGNC:795 | ENSG00000149311 | Q13315 | Serine-protein kinase ATM | clinvar |
Cohort function summary
Lead sentence per gene, UniProt-curated.
| Symbol | Protein name | Function (lead sentence) |
|---|---|---|
| STK11 | Serine/threonine-protein kinase STK11 | Tumor suppressor serine/threonine-protein kinase that controls the activity of AMP-activated protein kinase (AMPK) family members, thereby playing a role in various processes such as cell metabolism, cell polarity, apoptosis and DNA damage… |
| 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. |
Protein-family classification
Druggable: 2 · Difficult: 0 · Unknown: 0 · Druggable fraction: 1.0
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 | 2 | 27.7× | 0.001 |
Per-gene assignment
| Symbol | Family | Druggable? | EC | InterPro (top 3) |
|---|---|---|---|---|
| STK11 | Kinase | yes | 2.7.11.1 | Prot_kinase_dom, Ser/Thr_kinase_AS, Kinase-like_dom_sf |
| ATM | Kinase | yes | 2.7.11.1 | PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom |
Expression context
Cohort genes with no expression data: 0.
2 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) | 2 |
| unknown | 0 |
Top tissues across cohort
| Tissue | Cohort genes |
|---|---|
| hindlimb stylopod muscle | 1 |
| left testis | 1 |
| right testis | 1 |
| calcaneal tendon | 1 |
| colonic epithelium | 1 |
| corpus callosum | 1 |
Per-gene tissue summary (top 30)
| Symbol | Bgee breadth | FANTOM5 breadth | SCXA | Top tissues |
|---|---|---|---|---|
| STK11 | 238 | ubiquitous | marker | left testis, right testis, hindlimb stylopod muscle |
| ATM | 286 | ubiquitous | marker | calcaneal tendon, colonic epithelium, corpus callosum |
Protein interactions among cohort
Intra-cohort edges: 1.
Hub genes (top 10 by interactor count)
| Symbol | Interactor count |
|---|---|
| ATM | 7,383 |
| STK11 | 5,146 |
Intra-cohort edges
| A | B | Sources |
|---|---|---|
| ATM | STK11 | string_interaction |
Structural data
PDB: 2 · AlphaFold-only: 0 · No structure: 0
Cohort genes with PDB structures (top 30)
| Symbol | UniProt | PDB entries |
|---|---|---|
| ATM | Q13315 | 14 |
| STK11 | Q15831 | 4 |
Function
Pathway analysis
Distinct Reactome pathways touched by cohort: 69. Enrichment computed across 2 evidence-associated genes (2 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 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| Pathway | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| Regulation of TP53 Activity | 2 | 132.8× | 0.002 | STK11, ATM |
| Regulation of TP53 Activity through Phosphorylation | 2 | 117.7× | 0.002 | STK11, ATM |
| Transcriptional Regulation by TP53 | 2 | 62.1× | 0.006 | STK11, ATM |
| Sensing of DNA Double Strand Breaks | 1 | 951.7× | 0.011 | ATM |
| AMPK inhibits chREBP transcriptional activation activity | 1 | 713.8× | 0.011 | STK11 |
| TP53 Regulates Transcription of Caspase Activators and Caspases | 1 | 475.8× | 0.011 | ATM |
| Pexophagy | 1 | 475.8× | 0.011 | ATM |
| Defective homologous recombination repair (HRR) due to PALB2 loss of function | 1 | 475.8× | 0.011 | ATM |
| Diseases of DNA Double-Strand Break Repair | 1 | 407.9× | 0.011 | ATM |
| Defective homologous recombination repair (HRR) due to BRCA2 loss of function | 1 | 407.9× | 0.011 | ATM |
| Stabilization of p53 | 1 | 380.7× | 0.011 | ATM |
| p53-Dependent G1 DNA Damage Response | 1 | 356.9× | 0.011 | ATM |
| p53-Dependent G1/S DNA damage checkpoint | 1 | 356.9× | 0.011 | ATM |
| FOXO-mediated transcription of cell death genes | 1 | 356.9× | 0.011 | STK11 |
| G1/S DNA Damage Checkpoints | 1 | 335.9× | 0.011 | ATM |
| Resolution of D-Loop Structures | 1 | 317.2× | 0.011 | ATM |
| Diseases of DNA repair | 1 | 285.5× | 0.011 | ATM |
| TP53 Regulates Transcription of Cell Death Genes | 1 | 271.9× | 0.011 | ATM |
| TP53 Regulates Transcription of Genes Involved in Cytochrome C Release | 1 | 271.9× | 0.011 | ATM |
| Regulation of TP53 Activity through Methylation | 1 | 271.9× | 0.011 | ATM |
| Regulation of TP53 Expression and Degradation | 1 | 259.6× | 0.011 | ATM |
| DNA Double Strand Break Response | 1 | 237.9× | 0.011 | ATM |
| Impaired BRCA2 binding to PALB2 | 1 | 228.4× | 0.011 | ATM |
| Defective homologous recombination repair (HRR) due to BRCA1 loss of function | 1 | 211.5× | 0.011 | ATM |
| Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA1 binding function | 1 | 211.5× | 0.011 | ATM |
| Defective HDR through Homologous Recombination Repair (HRR) due to PALB2 loss of BRCA2/RAD51/RAD51C binding function | 1 | 211.5× | 0.011 | ATM |
| Cellular response to heat stress | 1 | 196.9× | 0.011 | ATM |
| Energy dependent regulation of mTOR by LKB1-AMPK | 1 | 196.9× | 0.011 | STK11 |
| Resolution of D-loop Structures through Synthesis-Dependent Strand Annealing (SDSA) | 1 | 196.9× | 0.011 | ATM |
| RNA Polymerase II Transcription | 2 | 22.5× | 0.011 | STK11, ATM |
GO biological processes by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| GO term | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| response to ionizing radiation | 2 | 411.0× | 3e-04 | STK11, ATM |
| regulation of signal transduction by p53 class mediator | 2 | 383.0× | 3e-04 | STK11, ATM |
| negative regulation of TORC1 signaling | 2 | 324.1× | 3e-04 | STK11, ATM |
| protein autophosphorylation | 2 | 145.3× | 0.001 | STK11, ATM |
| positive regulation of vesicle transport along microtubule | 1 | 8426.0× | 0.002 | STK11 |
| establishment of RNA localization to telomere | 1 | 4213.0× | 0.002 | ATM |
| establishment of protein-containing complex localization to telomere | 1 | 4213.0× | 0.002 | ATM |
| positive regulation of telomerase catalytic core complex assembly | 1 | 4213.0× | 0.002 | ATM |
| regulation of cell cycle | 2 | 74.6× | 0.002 | STK11, ATM |
| protein phosphorylation | 2 | 68.0× | 0.002 | STK11, ATM |
| pre-B cell allelic exclusion | 1 | 2808.7× | 0.003 | ATM |
| cellular response to nitrosative stress | 1 | 2808.7× | 0.003 | ATM |
| DNA damage response | 2 | 53.5× | 0.003 | STK11, ATM |
| peptidyl-serine autophosphorylation | 1 | 1685.2× | 0.004 | ATM |
| negative regulation of telomere capping | 1 | 1685.2× | 0.004 | ATM |
| regulation of telomere maintenance via telomerase | 1 | 1404.3× | 0.004 | ATM |
| negative regulation of epithelial cell proliferation involved in prostate gland development | 1 | 1404.3× | 0.004 | STK11 |
| positive regulation of telomere maintenance via telomere lengthening | 1 | 1404.3× | 0.004 | ATM |
| lipoprotein catabolic process | 1 | 1203.7× | 0.004 | ATM |
| V(D)J recombination | 1 | 1053.2× | 0.004 | ATM |
| Golgi localization | 1 | 1053.2× | 0.004 | STK11 |
| epithelial cell proliferation involved in prostate gland development | 1 | 1053.2× | 0.004 | STK11 |
| meiotic telomere clustering | 1 | 936.2× | 0.004 | ATM |
| female meiotic nuclear division | 1 | 842.6× | 0.004 | ATM |
| histone mRNA catabolic process | 1 | 842.6× | 0.004 | ATM |
| cellular response to X-ray | 1 | 842.6× | 0.004 | ATM |
| dendrite extension | 1 | 842.6× | 0.004 | STK11 |
| DNA double-strand break processing | 1 | 766.0× | 0.005 | ATM |
| activation of protein kinase activity | 1 | 766.0× | 0.005 | STK11 |
| positive thymic T cell selection | 1 | 702.2× | 0.005 | STK11 |
Therapeutics
Drugs indicated for this disease
1 approved, 16 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.
| Drug | Development status |
|---|---|
| Lomustine | Approved (phase 4) |
| Bevacizumab | Phase 3 (in late-stage trials) |
| Carboplatin | Phase 3 (in late-stage trials) |
| Cisplatin | Phase 3 (in late-stage trials) |
| Dactinomycin | Phase 3 (in late-stage trials) |
| Dianhydrogalactitol | Phase 3 (in late-stage trials) |
| Doxorubicin | Phase 3 (in late-stage trials) |
| Etoposide | Phase 3 (in late-stage trials) |
| Filgrastim | Phase 3 (in late-stage trials) |
| Irinotecan | Phase 3 (in late-stage trials) |
| Metformin | Phase 3 (in late-stage trials) |
| Methotrexate | Phase 3 (in late-stage trials) |
| Nivolumab | Phase 3 (in late-stage trials) |
| Pembrolizumab | Phase 3 (in late-stage trials) |
| Temozolomide | Phase 3 (in late-stage trials) |
| Thioguanine | Phase 3 (in late-stage trials) |
| Vincristine | Phase 3 (in late-stage trials) |
Earlier-phase candidates (phase 2, investigational — efficacy not yet established): ANTINEOPLASTON A10, Aminolevulinic Acid, Capecitabine, Carmustine, Celecoxib, Dacomitinib Anhydrous, Dexamethasone, Fluorescein, Lapatinib, Lithium Carbonate, Oxygen, Palonosetron, Vorinostat.
Drug target analysis
Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 2 · Undrugged: 0
Druggability breadth: 2 of 2 evidence-associated genes (100%) 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 |
|---|---|
| STK11 | FEDRATINIB |
| ATM | AMIODARONE HYDROCHLORIDE |
Top cohort targets by molecule count
| Symbol | Molecules | Max phase |
|---|---|---|
| ATM | 35 | 4 |
| STK11 | 17 | 4 |
Drugs targeting cohort genes (top 30)
| Molecule | Max phase | Targets in cohort |
|---|---|---|
| FEDRATINIB | 4 | STK11 |
| PACRITINIB | 4 | STK11 |
| NINTEDANIB | 4 | STK11 |
| SUNITINIB | 4 | STK11 |
| MIDOSTAURIN | 4 | STK11 |
| AMIODARONE HYDROCHLORIDE | 4 | ATM |
| FURAZOLIDONE | 4 | ATM |
| ESTRADIOL ACETATE | 4 | ATM |
| NAFTIFINE HYDROCHLORIDE | 4 | ATM |
| METHYSERGIDE MALEATE | 4 | ATM |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | ATM |
| XYLOMETAZOLINE HYDROCHLORIDE | 4 | ATM |
| FLUVOXAMINE MALEATE | 4 | ATM |
| ESTRADIOL VALERATE | 4 | ATM |
| PERMETHRIN | 4 | ATM |
| MITOTANE | 4 | ATM |
| TICLOPIDINE HYDROCHLORIDE | 4 | ATM |
| ENOXIMONE | 4 | ATM |
| METHYLENE BLUE ANHYDROUS | 4 | ATM |
| DITHIAZANINE IODIDE | 4 | ATM |
| ETHACRYNIC ACID | 4 | ATM |
| SECNIDAZOLE | 4 | ATM |
| MENADIONE | 4 | ATM |
| FENOFIBRATE | 4 | ATM |
| DIPYRIDAMOLE | 4 | ATM |
| DINACICLIB | 3 | STK11 |
| DOVITINIB | 3 | STK11 |
| LESTAURTINIB | 3 | STK11 |
| RUBOXISTAURIN | 3 | STK11 |
| DACTOLISIB | 3 | ATM |
Bioactivity and enzyme data
Enzyme cohort genes (≥1 EC): 2.
Cohort genes with ChEMBL bioactivity (full, sorted by assay count)
| Symbol | Assays | Type breakdown |
|---|---|---|
| STK11 | 244 | Binding:244 |
| ATM | 240 | Binding:233, Functional:5, ADMET:2 |
Cohort enzymes (BRENDA EC)
| Symbol | EC numbers | Names |
|---|---|---|
| STK11 | 2.7.11.1 | non-specific serine/threonine protein kinase |
| ATM | 2.7.11.1 | non-specific serine/threonine protein kinase |
Cohort genes with high screening signal
≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.
| Symbol | ChEMBL assays |
|---|---|
| STK11 | 244 |
| ATM | 240 |
Pharmacogenomics
Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.
No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).
Drug repurposing candidates
30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.
| Compound | Max phase | Cohort target (bioactivity) |
|---|---|---|
| FEDRATINIB | 4 | STK11 |
| PACRITINIB | 4 | STK11 |
| NINTEDANIB | 4 | STK11 |
| SUNITINIB | 4 | STK11 |
| MIDOSTAURIN | 4 | STK11 |
| AMIODARONE HYDROCHLORIDE | 4 | ATM |
| FURAZOLIDONE | 4 | ATM |
| ESTRADIOL ACETATE | 4 | ATM |
| NAFTIFINE HYDROCHLORIDE | 4 | ATM |
| METHYSERGIDE MALEATE | 4 | ATM |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | ATM |
| XYLOMETAZOLINE HYDROCHLORIDE | 4 | ATM |
| FLUVOXAMINE MALEATE | 4 | ATM |
| ESTRADIOL VALERATE | 4 | ATM |
| PERMETHRIN | 4 | ATM |
| MITOTANE | 4 | ATM |
| TICLOPIDINE HYDROCHLORIDE | 4 | ATM |
| ENOXIMONE | 4 | ATM |
| METHYLENE BLUE ANHYDROUS | 4 | ATM |
| DITHIAZANINE IODIDE | 4 | ATM |
| ETHACRYNIC ACID | 4 | ATM |
| SECNIDAZOLE | 4 | ATM |
| MENADIONE | 4 | ATM |
| FENOFIBRATE | 4 | ATM |
| DIPYRIDAMOLE | 4 | ATM |
| DINACICLIB | 3 | STK11 |
| DOVITINIB | 3 | STK11 |
| LESTAURTINIB | 3 | STK11 |
| RUBOXISTAURIN | 3 | STK11 |
| DACTOLISIB | 3 | ATM |
Druggability pyramid
Cohort genes binned by druggability tier (high → low):
| Tier | Definition | Genes | Symbols |
|---|---|---|---|
| A | Approved (phase 4 drug) | 2 | STK11, ATM |
| B | Phased (≥1) drug, not yet approved | 0 | |
| C | Druggable family + PDB, no drug | 0 | |
| D | Druggable family + AlphaFold only, no drug | 0 | |
| E | Difficult family or no structure, no drug | 0 |
Undrugged target profiles
0 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).
Clinical trials & evidence
Clinical trials
Clinical trials: 396.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| Not specified | 180 |
| PHASE1 | 83 |
| PHASE2 | 70 |
| PHASE1/PHASE2 | 35 |
| PHASE3 | 11 |
| EARLY_PHASE1 | 11 |
| PHASE4 | 6 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT07049094 | PHASE4 | RECRUITING | The Analgesic Effect of Scalp Nerve Block Using Bupivacaine Liposomes for Postoperative Pain Relief After Craniotomy |
| NCT07225101 | PHASE4 | RECRUITING | Evaluation of the Efficacy of STRATAFIX for Neurosurgical Cranial and Spine Procedures |
| NCT02193568 | PHASE4 | COMPLETED | Light Sedation or Intubated General Anesthesia in Patients With Brain Cancer Undergoing Craniotomy |
| NCT02334722 | PHASE4 | COMPLETED | 1 Week Versus 6 Weeks of Levetiracetam in Surgical Brain Tumor Patients |
| NCT02708056 | PHASE4 | COMPLETED | Sugammadex Given for the Reversal of Rocuronium Induced Neuromuscular Blockade Under Sevoflurane Anesthesia in Infants |
| NCT02964416 | PHASE4 | COMPLETED | Single Dose Tramadol Effect on Extubation Response and Quality of Emergence Post-supratentorial Intracranial Surgery |
| 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 |
| NCT07376304 | PHASE3 | NOT_YET_RECRUITING | Intraoperative Ultrasound for Brain Tumor Surgery Enhanced by AI |
| NCT00045968 | PHASE3 | UNKNOWN | Study of a Drug [DCVax®-L] to Treat Newly Diagnosed GBM Brain Cancer |
| NCT00124761 | PHASE3 | COMPLETED | A Trial Comparing Radiosurgery With Surgery for Solitary Brain Metastases |
| NCT00241670 | PHASE3 | COMPLETED | Fluorescence-guided Resection of Malignant Gliomas With 5-Aminolevulinic Acid |
| NCT00460395 | PHASE3 | COMPLETED | Surgery Versus Stereotactic Radiosurgery in the Treatment of Single Brain Metastasis: A Randomized Trial |
| NCT00548756 | PHASE3 | COMPLETED | Randomized Trial Comparing Radiosurgery With vs Without Whole Brain Radiotherapy |
| NCT01014767 | PHASE3 | TERMINATED | Intercontinental Multidisciplinary Registry and Treatment Optimization Study for Choroid Plexus Tumors |
| NCT02617589 | PHASE3 | COMPLETED | An Investigational Immuno-therapy Study of Nivolumab Compared to Temozolomide, Each Given With Radiation Therapy, for Newly-diagnosed Patients With Glioblastoma (GBM, a Malignant Brain Cancer) |
| NCT03149575 | PHASE3 | TERMINATED | VAL-083 Phase 3 Study in Temozolomide-Avastin (Bevacizumab) Recurrent GBM |
| NCT04588246 | PHASE3 | TERMINATED | Comparing Whole Brain Radiotherapy Using a Technique That Avoids the Hippocampus to Stereotactic Radiosurgery in Patients With Cancer That Has Spread to the Brain and Come Back in Other Areas of the Brain After Earlier Stereotactic Radiosurgery |
| NCT02691923 | PHASE2 | RECRUITING | Diagnostic Performance of Fluorescein as an Intraoperative Brain Tumor Biomarker |
| NCT02800486 | PHASE2 | RECRUITING | Super Selective Intra-arterial Repeated Infusion of Cetuximab (Erbitux) With Reirradiation for Treatment of Relapsed/Refractory GBM, AA, and AOA |
| NCT02861898 | PHASE1/PHASE2 | RECRUITING | Super-selective Intra-arterial Repeated Infusion of Cetuximab for the Treatment of Newly Diagnosed Glioblastoma |
| NCT03213002 | PHASE1/PHASE2 | RECRUITING | Oral Capecitabine and Temozolomide (CAPTEM) for Newly Diagnosed GBM |
| NCT03510208 | PHASE1/PHASE2 | RECRUITING | Panitumumab-IRDye800 in Diagnosing Participants With Malignant Glioma Undergoing Surgery |
| NCT03649880 | PHASE2 | RECRUITING | Feasibility of FMISO in Brain Tumors |
| NCT04478279 | PHASE1/PHASE2 | ACTIVE_NOT_RECRUITING | A Phase 1-2 Study of ST101 in Patients With Advanced Solid Tumors |
| NCT04755920 | PHASE2 | RECRUITING | SGM-101 in Colorectal Brain Metastases. |
| NCT04899908 | PHASE2 | ACTIVE_NOT_RECRUITING | Stereotactic Brain-directed Radiation With or Without Aguix Gadolinium-Based Nanoparticles in Brain Metastases |
| NCT05139043 | PHASE2 | ACTIVE_NOT_RECRUITING | Low Dose Versus Standard Dose Dexamethasone for Reduction of Swelling in Patients with Primary or Metastatic Brain Tumors |
| NCT05554302 | PHASE2 | RECRUITING | Characterization of 18F-Fluciclovine PET Amino Acid Radiotracer in Resected Brain Metastasis |
| NCT06012695 | PHASE1/PHASE2 | RECRUITING | NBM-BMX Administered Orally to Patients With Solid Tumors or Newly Diagnosed Glioblastoma |
| NCT06058988 | PHASE2 | RECRUITING | Trastuzumab Deruxtecan (T-DXd) for People With Brain Cancer |
| NCT06132685 | PHASE2 | RECRUITING | Post-Operative Dosing of Dexamethasone in Patients With Brain Tumors After a Craniotomy, PODS Trial |
| NCT06377696 | PHASE2 | RECRUITING | Remote Cognitive Assessment and Wearable Device While Assessing the Impact of Metformin in Patients With History of Cranial Radiation Therapy |
| NCT06640582 | PHASE1/PHASE2 | RECRUITING | TIL Therapy Combined With Pembrolizumab for Advanced Brain Cancer Including Gliomas and Meningiomas |
| NCT06667726 | PHASE2 | RECRUITING | An Investigational Scan (18F-DOPA PET/CT) for Improving the Clinical Management of Brain Tumors |
| NCT06991101 | PHASE2 | RECRUITING | Ruxolitinib With Radiation and Temozolomide Compared to Radiation and Temozolomide for Newly Diagnosed Glioblastoma |
| NCT07003139 | PHASE1/PHASE2 | RECRUITING | A Study of the Boron Neutron Capture Therapy (BNCT) Using B10 L-BPA in Malignant Brain Tumors |
| NCT07326566 | PHASE2 | RECRUITING | Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII |
| NCT07383649 | PHASE2 | RECRUITING | Trial to Study Anti- HCMV Therapy in Breast Cancer Patients With Progressive Intracranial Metastases and CMV Infection |
| NCT07416188 | PHASE1/PHASE2 | RECRUITING | Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma |
| NCT07569042 | PHASE1/PHASE2 | RECRUITING | A Rollover Study of NBM-BMX in Combination With Temozolomide in Patients With Newly Diagnosed Glioblastoma |
Drugs tested across these trials (top 30)
Related Atlas pages
- Cohort genes: STK11, ATM
- Drugs: Erlotinib, Aminolevulinic Acid, Flucytosine, Irinotecan, Sugammadex, Temozolomide, Temsirolimus, Vorinostat, Doxorubicin, Ferumoxytol, FLUDEOXYGLUCOSE F 18, Fluorescein, FLUORODOPA F 18, Gadobutrol, Lomustine, Mannitol, Sorafenib Tosylate, Sorbitol, Afatinib, Arsenic Trioxide, Bortezomib D-Mannitol, Cabazitaxel, Cabozantinib, Dacomitinib, Dactinomycin, EDOTREOTIDE GALLIUM GA-68, FLUCICLOVINE F18, Gadobenate Dimeglumine, Hydroxyurea