Colorectal neoplasm
diseaseOn this page
Also known as colorectal tumorcolorectal tumourcolorectum neoplasmcolorectum neoplasm (disease)colorectum tumorcolorectum tumourlarge bowel neoplasmlarge bowel tumorlarge bowel tumourlarge intestinal neoplasmlarge intestine neoplasmlarge intestine tumorlarge intestine tumourneoplasm of colorectumneoplasm of large bowelneoplasm of the large boweltumor of colorectumtumor of large boweltumor of the large bowel
Summary
Colorectal neoplasm (MONDO:0005335) is a cancer (an umbrella term covering 9 Mondo subtypes) with 1 cohort gene (1 CIViC-evidence somatic driver; 1 ClinVar predisposition record) and 722 clinical trials. Top therapeutic interventions include cetuximab, irinotecan, and pembrolizumab.
At a glance
- Classification: Cancer
- Umbrella term: 9 Mondo subtypes
- Cohort genes: 1
- ClinVar variants: 1
- Clinical trials: 722
Clinical features
No curated clinical features (Orphanet) for this disease.
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | colorectal neoplasm |
| Mondo ID | MONDO:0005335 |
| EFO | EFO:0004142 |
| MeSH | D015179 |
| NCIT | C2956 |
| UMLS | C0009404 |
| MedGen | 3171 |
| Anatomy (UBERON) | UBERON:0012652 |
| Is cancer (heuristic) | yes |
Also known as: colorectal neoplasm · colorectal tumor · colorectal tumour · colorectum neoplasm · colorectum neoplasm (disease) · colorectum tumor · colorectum tumour · large bowel neoplasm · large bowel tumor · large bowel tumour · large intestinal neoplasm · large intestine neoplasm · large intestine tumor · large intestine tumour · neoplasm of colorectum · neoplasm of large bowel · neoplasm of the large bowel · tumor of colorectum · tumor of large bowel · tumor of the large bowel (+3 more)
Data availability: 1 ClinVar variant.
Disease family
An umbrella term covering 9 Mondo subtypes.
Classification path: disease › human disease › disease by body system or component › digestive system disorder › intestinal disorder › intestinal neoplasm › colorectal neoplasm
Related subtypes (5): intestinal neuroendocrine neoplasm, intestinal benign neoplasm, small intestine neoplasm, intestinal cancer, epithelial tumor of anal canal
Subtypes (9): rectal neoplasm, colorectal leiomyoma, colonic neoplasm, colorectal adenoma, colorectal cancer, colorectal gastrointestinal stromal tumor, colorectal hamartoma, colorectal neuroendocrine tumor G1, small intestinal intraepithelial neoplasia
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
ClinVar germline variants
1 retrieved; paginated sample, class counts are floors:
1 pathogenic
| ClinVar | Variant (HGVS) | Gene | Classification | Review |
|---|---|---|---|---|
| 162793 | NM_004333.6(BRAF):c.1794_1796dup (p.Thr599dup) | BRAF | Pathogenic | criteria provided, multiple submitters, no conflicts |
Genes & proteins
Mendelian disease overlap and somatic drivers
GenCC: 0 · Orphanet: 11 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0
Somatic driver evidence (intOGen + CIViC, cohort fanout)
| Gene | intOGen role | Cancer types | CIViC |
|---|---|---|---|
| BRAF | Act | BLCA,BRCA,CHOL,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,GBM,GIST,HGGNOS,LGGNOS,LUAD,MEL,MLYM,NSCLC,OVT,PAST,PCM,PRAD,PRCC,PROSTATE,READ,SACA,SKCM,STAD,UCEC,WDTC | CIViC #5 |
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 |
Cohort genes → proteins
1 cohort genes, 1 distinct canonical proteins.
Evidence partition
| Subset | Genes |
|---|---|
| multi_evidence | 1 |
Cohort genes (full)
| Symbol | HGNC | Ensembl | UniProt | Name | Evidence |
|---|---|---|---|---|---|
| BRAF | HGNC:1097 | ENSG00000157764 | P15056 | Serine/threonine-protein kinase B-raf | clinvar |
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. |
Protein-family classification
Druggable: 1 · 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 | 1 | 27.7× | 0.036 |
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 |
Expression context
Cohort genes with no expression data: 0.
1 cohort gene 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) | 1 |
| unknown | 0 |
Top tissues across cohort
| Tissue | Cohort genes |
|---|---|
| buccal mucosa cell | 1 |
| calcaneal tendon | 1 |
| colonic epithelium | 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 |
Protein interactions among cohort
Intra-cohort edges: 0.
Hub genes (top 10 by interactor count)
| Symbol | Interactor count |
|---|---|
| BRAF | 7,394 |
Structural data
PDB: 1 · AlphaFold-only: 0 · No structure: 0
Cohort genes with PDB structures (top 30)
| Symbol | UniProt | PDB entries |
|---|---|---|
| BRAF | P15056 | 131 |
Function
Pathway analysis
Distinct Reactome pathways touched by cohort: 39. Enrichment computed across 1 evidence-associated genes (1 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 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| Pathway | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| Signaling by MRAS-complex mutants | 1 | 2855.0× | 0.004 | BRAF |
| Signalling to p38 via RIT and RIN | 1 | 2284.0× | 0.004 | BRAF |
| Negative feedback regulation of MAPK pathway | 1 | 1903.3× | 0.004 | BRAF |
| ARMS-mediated activation | 1 | 1631.4× | 0.004 | BRAF |
| Prolonged ERK activation events | 1 | 1427.5× | 0.004 | BRAF |
| SHOC2 M1731 mutant abolishes MRAS complex function | 1 | 1427.5× | 0.004 | BRAF |
| Gain-of-function MRAS complexes activate RAF signaling | 1 | 1427.5× | 0.004 | BRAF |
| Signaling by FGFR3 | 1 | 1142.0× | 0.004 | BRAF |
| Signaling by FGFR4 | 1 | 1038.2× | 0.004 | BRAF |
| Frs2-mediated activation | 1 | 951.7× | 0.004 | BRAF |
| Signaling by FGFR1 | 1 | 815.7× | 0.004 | BRAF |
| Spry regulation of FGF signaling | 1 | 713.8× | 0.005 | BRAF |
| Signalling to ERKs | 1 | 601.0× | 0.005 | BRAF |
| Negative regulation of FGFR3 signaling | 1 | 439.2× | 0.005 | BRAF |
| Signaling by RAS mutants | 1 | 423.0× | 0.005 | BRAF |
| Negative regulation of FGFR4 signaling | 1 | 407.9× | 0.005 | BRAF |
| Signaling by FGFR2 | 1 | 407.9× | 0.005 | BRAF |
| Negative regulation of FGFR1 signaling | 1 | 368.4× | 0.005 | BRAF |
| Negative regulation of FGFR2 signaling | 1 | 368.4× | 0.005 | BRAF |
| Signaling by FGFR | 1 | 346.1× | 0.005 | BRAF |
| RAF activation | 1 | 335.9× | 0.005 | BRAF |
| Signaling by high-kinase activity BRAF mutants | 1 | 317.2× | 0.005 | BRAF |
| MAP2K and MAPK activation | 1 | 285.5× | 0.005 | BRAF |
| Signaling by RAF1 mutants | 1 | 278.5× | 0.005 | BRAF |
| Negative regulation of MAPK pathway | 1 | 265.6× | 0.005 | BRAF |
| Signaling by moderate kinase activity BRAF mutants | 1 | 253.8× | 0.005 | BRAF |
| Paradoxical activation of RAF signaling by kinase inactive BRAF | 1 | 253.8× | 0.005 | BRAF |
| Signaling downstream of RAS mutants | 1 | 253.8× | 0.005 | BRAF |
| Oncogenic MAPK signaling | 1 | 248.3× | 0.005 | BRAF |
| Signaling by NTRK1 (TRKA) | 1 | 196.9× | 0.007 | BRAF |
GO biological processes by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| GO term | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| CD4-positive or CD8-positive, alpha-beta T cell lineage commitment | 1 | 5617.3× | 0.003 | BRAF |
| positive regulation of axon regeneration | 1 | 3370.4× | 0.003 | BRAF |
| negative regulation of synaptic vesicle exocytosis | 1 | 3370.4× | 0.003 | BRAF |
| CD4-positive, alpha-beta T cell differentiation | 1 | 2808.7× | 0.003 | BRAF |
| myeloid progenitor cell differentiation | 1 | 2407.4× | 0.003 | BRAF |
| positive regulation of D-glucose transmembrane transport | 1 | 2106.5× | 0.003 | BRAF |
| head morphogenesis | 1 | 2106.5× | 0.003 | BRAF |
| establishment of protein localization to membrane | 1 | 1872.4× | 0.003 | BRAF |
| negative regulation of fibroblast migration | 1 | 1532.0× | 0.003 | BRAF |
| endothelial cell apoptotic process | 1 | 1296.3× | 0.003 | BRAF |
| regulation of T cell differentiation | 1 | 1203.7× | 0.003 | BRAF |
| face development | 1 | 802.5× | 0.003 | BRAF |
| synaptic vesicle exocytosis | 1 | 766.0× | 0.003 | BRAF |
| positive regulation of peptidyl-serine phosphorylation | 1 | 766.0× | 0.003 | BRAF |
| stress fiber assembly | 1 | 766.0× | 0.003 | BRAF |
| postsynaptic modulation of chemical synaptic transmission | 1 | 674.1× | 0.004 | BRAF |
| positive regulation of axonogenesis | 1 | 581.1× | 0.004 | BRAF |
| thyroid gland development | 1 | 543.6× | 0.004 | BRAF |
| negative regulation of endothelial cell apoptotic process | 1 | 495.6× | 0.004 | BRAF |
| T cell differentiation in thymus | 1 | 411.0× | 0.005 | BRAF |
| positive regulation of substrate adhesion-dependent cell spreading | 1 | 374.5× | 0.005 | BRAF |
| substrate adhesion-dependent cell spreading | 1 | 343.9× | 0.005 | BRAF |
| thymus development | 1 | 337.0× | 0.005 | BRAF |
| ERK1 and ERK2 cascade | 1 | 318.0× | 0.005 | BRAF |
| positive regulation of stress fiber assembly | 1 | 312.1× | 0.005 | BRAF |
| visual learning | 1 | 306.4× | 0.005 | BRAF |
| long-term synaptic potentiation | 1 | 280.9× | 0.005 | BRAF |
| epidermal growth factor receptor signaling pathway | 1 | 247.8× | 0.006 | BRAF |
| somatic stem cell population maintenance | 1 | 247.8× | 0.006 | BRAF |
| cellular response to xenobiotic stimulus | 1 | 240.7× | 0.006 | BRAF |
Therapeutics
Drugs indicated for this disease
16 approved, 72 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.
| Drug | Development status |
|---|---|
| Aflibercept | Approved (phase 4) |
| Arcitumomab | Approved (phase 4) |
| Bevacizumab | Approved (phase 4) |
| Capecitabine | Approved (phase 4) |
| Celecoxib | Approved (phase 4) |
| Cetuximab | Approved (phase 4) |
| Encorafenib | Approved (phase 4) |
| Fruquintinib | Approved (phase 4) |
| Ipilimumab | Approved (phase 4) |
| Levoleucovorin | Approved (phase 4) |
| Nivolumab | Approved (phase 4) |
| Panitumumab | Approved (phase 4) |
| Regorafenib | Approved (phase 4) |
| Trifluridine | Approved (phase 4) |
| Tucatinib | Approved (phase 4) |
| Votumumab | Approved (phase 4) |
| Arfolitixorin | Phase 3 (in late-stage trials) |
| Ascorbic Acid | Phase 3 (in late-stage trials) |
| Aspirin | Phase 3 (in late-stage trials) |
| Atezolizumab | Phase 3 (in late-stage trials) |
| Bcg Vaccine | Phase 3 (in late-stage trials) |
| Bermekimab | Phase 3 (in late-stage trials) |
| Brivanib Alaninate | Phase 3 (in late-stage trials) |
| Calcium | Phase 3 (in late-stage trials) |
| Carboplatin | Phase 3 (in late-stage trials) |
| Cefmetazole | Phase 3 (in late-stage trials) |
| Cholecalciferol | Phase 3 (in late-stage trials) |
| Cisplatin | Phase 3 (in late-stage trials) |
| Cobimetinib | Phase 3 (in late-stage trials) |
| Cyclosporine | Phase 3 (in late-stage trials) |
| Dalantercept | Phase 3 (in late-stage trials) |
| Dalteparin Sodium | Phase 3 (in late-stage trials) |
| Dexamethasone | Phase 3 (in late-stage trials) |
| Dexmedetomidine | Phase 3 (in late-stage trials) |
| Eflornithine | Phase 3 (in late-stage trials) |
| Eniluracil | Phase 3 (in late-stage trials) |
| Erlotinib | Phase 3 (in late-stage trials) |
| Etodolac | Phase 3 (in late-stage trials) |
| Etoposide | Phase 3 (in late-stage trials) |
| Favezelimab | Phase 3 (in late-stage trials) |
| Floxuridine | Phase 3 (in late-stage trials) |
| Fluorouracil | Phase 3 (in late-stage trials) |
| Folic Acid | Phase 3 (in late-stage trials) |
| Gimeracil | Phase 3 (in late-stage trials) |
| Glutamine | Phase 3 (in late-stage trials) |
| Heparin Sodium | Phase 3 (in late-stage trials) |
| Interferon Alfa | Phase 3 (in late-stage trials) |
| Irinotecan | Phase 3 (in late-stage trials) |
| Lenvatinib | Phase 3 (in late-stage trials) |
| Lidocaine | Phase 3 (in late-stage trials) |
| Maltol | Phase 3 (in late-stage trials) |
| Mebendazole | Phase 3 (in late-stage trials) |
| Melphalan | Phase 3 (in late-stage trials) |
| Metformin | Phase 3 (in late-stage trials) |
| Metronidazole | Phase 3 (in late-stage trials) |
| Mitomycin | Phase 3 (in late-stage trials) |
| Monosialotetrahexosylganglioside Sodium | Phase 3 (in late-stage trials) |
| Napabucasin | Phase 3 (in late-stage trials) |
| Nintedanib | Phase 3 (in late-stage trials) |
| Nitrogen | Phase 3 (in late-stage trials) |
| Nitrous Oxide | Phase 3 (in late-stage trials) |
| Oteracil | Phase 3 (in late-stage trials) |
| Oxaliplatin | Phase 3 (in late-stage trials) |
| POLYETHYLENE GLYCOL 3350 | Phase 3 (in late-stage trials) |
| Pembrolizumab | Phase 3 (in late-stage trials) |
| Perifosine | Phase 3 (in late-stage trials) |
| Propranolol | Phase 3 (in late-stage trials) |
| Raltitrexed | Phase 3 (in late-stage trials) |
| Ramucirumab | Phase 3 (in late-stage trials) |
| Relatlimab | Phase 3 (in late-stage trials) |
| Rivoceranib | Phase 3 (in late-stage trials) |
| Rofecoxib | Phase 3 (in late-stage trials) |
| Rosuvastatin | Phase 3 (in late-stage trials) |
| Semaxanib | Phase 3 (in late-stage trials) |
| Semustine | Phase 3 (in late-stage trials) |
| Simethicone | Phase 3 (in late-stage trials) |
| Simvastatin | Phase 3 (in late-stage trials) |
| Sintilimab | Phase 3 (in late-stage trials) |
| Sotorasib | Phase 3 (in late-stage trials) |
| Sulindac | Phase 3 (in late-stage trials) |
| Tegafur | Phase 3 (in late-stage trials) |
| Thalidomide | Phase 3 (in late-stage trials) |
| Tipiracil | Phase 3 (in late-stage trials) |
| Trastuzumab | Phase 3 (in late-stage trials) |
| Uracil | Phase 3 (in late-stage trials) |
| Vitamin E | Phase 3 (in late-stage trials) |
| Xaliproden | Phase 3 (in late-stage trials) |
| Zanzalintinib | Phase 3 (in late-stage trials) |
Earlier-phase candidates (phase 2, investigational — efficacy not yet established): 6-O-BENZYLGUANINE, Afatinib, Alectinib, Alpelisib, Amifostine, Andrographolide, Artesunate, Avelumab, Avutometinib, Axitinib, Balstilimab, Becatecarin, Binimetinib, Bortezomib, Botensilimab, Brivanib, Cabazitaxel, Cabozantinib, Cadonilimab, Camrelizumab, Carmustine, Catequentinib, Cediranib, Cemiplimab, Curcumin, Defactinib, Denosumab, Disitamab Vedotin, Duloxetine, Durvalumab, Edrecolomab, Elacytarabine, Endostatin, Endostatin, N-Terminal-Mggshhhhh, Envafolimab, Enzastaurin, Erfonrilimab, Etirinotecan Pegol, Everolimus, Exisulind, Fianlimab, Figitumumab, Filgrastim, Fludeoxyglucose, G17DT IMMUNOGEN, Gefitinib, Gemcitabine, Ginger, Hydroxychloroquine, Icosapent, Incomplete Freund’S Adjuvant, Influenza Virus Vaccine, Irofulven, Ixabepilone, Izalontamab, Lamivudine, Lapatinib, Lenalidomide, Leronlimab, Levocetirizine, Mesalamine, Methotrexate, Minocycline, Monalizumab, Nibrozetone, Niclosamide, Nimotuzumab, Nogapendekin Alfa, Olaparib, Oleclumab, Omeprazole, Onartuzumab, Osimertinib, Paclitaxel, Pacritinib, Panobinostat, Pegfilgrastim, Pemetrexed, Penpulimab, Pentamidine, Pertuzumab, Prolgolimab, Pyridoxine, Pyrotinib, Quavonlimab, Romidepsin, Rubitecan, Sargramostim, Selenomethionine, Selumetinib, Serplulimab, Sorafenib, Sunitinib, Surufatinib, Temozolomide, Tepotinib, Tetanus Toxoid, Tipifarnib, Tislelizumab, Tivozanib, Tocotrienol, Toripalimab, Trametinib, Trastuzumab Deruxtecan, Tremelimumab, Tretinoin, Tucidinostat, Ursodiol, Vandetanib, Veliparib, Vemurafenib, Vibostolimab, Vicriviroc, Vorinostat, Zibotentan.
Drug target analysis
Approved (phase 4): 1 · Phase ≥3: 1 · Phased (≥1): 1 · Undrugged: 0
Druggability breadth: 1 of 1 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 |
|---|---|
| BRAF | VEMURAFENIB |
Top cohort targets by molecule count
| Symbol | Molecules | Max phase |
|---|---|---|
| BRAF | 48 | 4 |
Drugs targeting cohort genes (top 30)
| Molecule | Max phase | Targets in cohort |
|---|---|---|
| VEMURAFENIB | 4 | BRAF |
| PONATINIB | 4 | BRAF |
| FEDRATINIB | 4 | BRAF |
| SORAFENIB | 4 | BRAF |
| DASATINIB ANHYDROUS | 4 | BRAF |
| RUXOLITINIB | 4 | BRAF |
| INFIGRATINIB PHOSPHATE | 4 | BRAF |
| INFIGRATINIB | 4 | BRAF |
| REGORAFENIB | 4 | BRAF |
| DABRAFENIB | 4 | BRAF |
| COBIMETINIB | 4 | BRAF |
| NILOTINIB | 4 | BRAF |
| ABEMACICLIB | 4 | BRAF |
| ENCORAFENIB | 4 | BRAF |
| TOVORAFENIB | 4 | BRAF |
| PAZOPANIB | 4 | BRAF |
| DASATINIB | 4 | BRAF |
| ERLOTINIB | 4 | BRAF |
| GEFITINIB | 4 | BRAF |
| IMATINIB | 4 | BRAF |
| MASITINIB | 3 | BRAF |
| AVUTOMETINIB | 3 | BRAF |
| NAPORAFENIB | 3 | BRAF |
| QUERCETIN | 3 | BRAF |
| MOTESANIB | 3 | BRAF |
| DORAMAPIMOD | 2 | BRAF |
| FORETINIB | 2 | BRAF |
| REBASTINIB | 2 | BRAF |
| CEP-32496 | 2 | BRAF |
| BAFETINIB | 2 | BRAF |
Bioactivity and enzyme data
Enzyme cohort genes (≥1 EC): 1.
Cohort genes with ChEMBL bioactivity (full, sorted by assay count)
| Symbol | Assays | Type breakdown |
|---|---|---|
| BRAF | 1,442 | Binding:1400, Functional:37, ADMET:5 |
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 |
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 |
Pharmacogenomics
Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.
No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).
Drug repurposing candidates
29 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) |
|---|---|---|
| VEMURAFENIB | 4 | BRAF |
| PONATINIB | 4 | BRAF |
| FEDRATINIB | 4 | BRAF |
| SORAFENIB | 4 | BRAF |
| DASATINIB ANHYDROUS | 4 | BRAF |
| RUXOLITINIB | 4 | BRAF |
| INFIGRATINIB PHOSPHATE | 4 | BRAF |
| INFIGRATINIB | 4 | BRAF |
| DABRAFENIB | 4 | BRAF |
| COBIMETINIB | 4 | BRAF |
| NILOTINIB | 4 | BRAF |
| ABEMACICLIB | 4 | BRAF |
| ENCORAFENIB | 4 | BRAF |
| TOVORAFENIB | 4 | BRAF |
| PAZOPANIB | 4 | BRAF |
| DASATINIB | 4 | BRAF |
| ERLOTINIB | 4 | BRAF |
| GEFITINIB | 4 | BRAF |
| IMATINIB | 4 | BRAF |
| MASITINIB | 3 | BRAF |
| AVUTOMETINIB | 3 | BRAF |
| NAPORAFENIB | 3 | BRAF |
| QUERCETIN | 3 | BRAF |
| MOTESANIB | 3 | BRAF |
| DORAMAPIMOD | 2 | BRAF |
| FORETINIB | 2 | BRAF |
| REBASTINIB | 2 | BRAF |
| CEP-32496 | 2 | BRAF |
| BAFETINIB | 2 | BRAF |
Druggability pyramid
Cohort genes binned by druggability tier (high → low):
| Tier | Definition | Genes | Symbols |
|---|---|---|---|
| A | Approved (phase 4 drug) | 1 | BRAF |
| 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: 722.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| Not specified | 401 |
| PHASE2 | 132 |
| PHASE1 | 57 |
| PHASE3 | 53 |
| PHASE1/PHASE2 | 45 |
| PHASE4 | 20 |
| PHASE2/PHASE3 | 11 |
| EARLY_PHASE1 | 3 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04281667 | PHASE4 | ACTIVE_NOT_RECRUITING | Mechanical Bowel Preparation and Oral Antibiotics Versus Mechanical Bowel Preparation Only Prior Rectal Surgery |
| NCT05148494 | PHASE4 | RECRUITING | TES RCT Fleet Enema vs Oral Mechanical Bowel Prep |
| NCT07158164 | PHASE4 | RECRUITING | DPYD Pharmacogenomics and Fluoropyrimidine (FP) Dose-Adjustment |
| NCT00114829 | PHASE4 | UNKNOWN | Preoperative Assessment of Colon Tumor |
| NCT00134589 | PHASE4 | COMPLETED | CHOICE: Communicating Health Options Through Information and Cancer Education |
| NCT00140036 | PHASE4 | COMPLETED | Pharmacogenomics Blood Sampling Protocol For Irinotecan/Fluorouracil/Leucovorin(CPT-11/FU/LV). |
| NCT00168987 | PHASE4 | COMPLETED | Influence of an Oral Nutritional Supplement Rich in Omega-3 Fatty Acids on Functional State and Quality of Life in Malnourished Patients With Gastroenterological Tumors |
| NCT00199277 | PHASE4 | UNKNOWN | Iron Therapy in Colo-Rectal Neoplasm and Iron Deficiency Anemia: Intravenous Iron Sucrose Versus Oral Ferrous Sulphate. |
| NCT00398333 | PHASE4 | TERMINATED | Study to Assess the Effectiveness of a Omega-3 Enriched Supplement on Chemotherapy Tolerance in Colon Cancer Patients |
| NCT00764621 | PHASE4 | COMPLETED | Health Economic Evaluation of Primovist-enhanced Liver MRI |
| NCT01564810 | PHASE4 | UNKNOWN | Cetuximab in Combination With Chemotherapy for the Treatment of Metastatic Colorectal Cancer |
| NCT01588990 | PHASE4 | COMPLETED | A Translational Study of Bevacizumab in Participants With Metastatic Colorectal Cancer |
| NCT01607255 | PHASE4 | COMPLETED | Comparative Efficacy of Water & Indigo Carmine vs. Water or Air Method on Adenoma Detection Rate (ADR) - a Randomized Controlled Trial (RCT) |
| NCT01700062 | PHASE4 | COMPLETED | Medium Calorie Parenteral Nutrition on Patients With Gastrointestinal Cancer Undergoing Surgery |
| NCT01701310 | PHASE4 | COMPLETED | IVICA: Intravenous Iron in Colorectal Cancer Associated Anaemia |
| NCT01972490 | PHASE4 | COMPLETED | Avastin in Combination With Chemotherapy for RAS Mutant Unresectable Colorectal Liver-limited Metastases |
| NCT01972503 | PHASE4 | UNKNOWN | Intraoperative Intraportal Chemotherapy Combined With Adjuvant Chemotherapy for Stage II and III Colorectal Cancer |
| NCT02057471 | PHASE4 | COMPLETED | Intravenous Iron: Measuring Response in Anemic Surgical Patients |
| NCT02800330 | PHASE4 | COMPLETED | The Effects of the Proton Pump Inhibitor Esomeprazole on the Bioavailability of Regorafenib |
| NCT02999217 | PHASE4 | UNKNOWN | Intravenous Iron for Correction of Anaemia After Colorectal Surgery |
| NCT01349881 | PHASE3 | ACTIVE_NOT_RECRUITING | S0820, Adenoma and Second Primary Prevention Trial |
| NCT02758951 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | Perioperative Systemic Therapy for Isolated Resectable Colorectal Peritoneal Metastases |
| NCT02885753 | PHASE3 | RECRUITING | Systemic Oxaliplatin or Intra-arterial Chemotherapy Combined With LV5FU2 +/- Irinotecan and an Target Therapy in First Line Treatment of Metastatic Colorectal Cancer Restricted to the Liver |
| NCT03659448 | PHASE3 | RECRUITING | Performance of SGM-101 for the Delineation of Primary and Recurrent Tumor and Metastases in Patients Undergoing Surgery for Colorectal Cancer |
| NCT05141721 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | A Study of a Patient-Specific Neoantigen Vaccine in Combination With Immune Checkpoint Blockade for Patients With Metastatic Colorectal Cancer |
| NCT05239741 | PHASE3 | ACTIVE_NOT_RECRUITING | Study of Pembrolizumab (MK-3475) Versus Chemotherapy in Chinese Participants With Stage IV Colorectal Cancer (MK-3475-C66) |
| NCT05253651 | PHASE3 | RECRUITING | A Study of Tucatinib With Trastuzumab and mFOLFOX6 Versus Standard of Care Treatment in First-line HER2+ Metastatic Colorectal Cancer |
| NCT05768178 | PHASE2/PHASE3 | RECRUITING | DETERMINE Trial Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers. |
| NCT05770102 | PHASE2/PHASE3 | RECRUITING | DETERMINE Trial Treatment Arm 02: Atezolizumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With High Tumour Mutational Burden (TMB) or Microsatellite Instability-high (MSI-high) or Proven Constitutional Mismatch Repair Deficiency (CMMRD) Disposition |
| NCT05786716 | PHASE2/PHASE3 | RECRUITING | DETERMINE Trial Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating Mutations |
| NCT06226857 | PHASE3 | RECRUITING | Other Oncogene Mutations for Anti-EGFR Efficacy in Patients With Left-sided RAS-wild Type Metastatic Colorectal Cancer |
| NCT06662786 | PHASE3 | RECRUITING | A Study of Amivantamab and mFOLFOX6 or FOLFIRI Versus Cetuximab and mFOLFOX6 or FOLFIRI as First-line Treatment in Participants With KRAS/NRAS and BRAF Wild-type Unresectable or Metastatic Left-sided Colorectal Cancer |
| NCT06750094 | PHASE3 | RECRUITING | A Study of Amivantamab and FOLFIRI Versus Cetuximab/Bevacizumab and FOLFIRI in Participants With KRAS/NRAS and BRAF Wild-type Colorectal Cancer Who Have Previously Received Chemotherapy |
| NCT06857773 | PHASE3 | RECRUITING | Induction Treatment for Initially Unresectable Colorectal Liver Metastases: Combined Hepatic Arterial Infusion Pump Therapy With Systemic Therapy |
| NCT07222800 | PHASE3 | RECRUITING | Symbiotic-GI-03: A Study to Learn About the Study Medicine Called PF-08634404 in Combination With Chemotherapy in Adult Participants With Metastatic Colorectal Cancer |
| NCT07440290 | PHASE2/PHASE3 | NOT_YET_RECRUITING | DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers. |
| NCT00056446 | PHASE3 | COMPLETED | Study of Oxaliplatin/5-FU/Leucovorin Plus Vatalanib Versus Oxaliplatin/5-FU/Leucovorin in Patients With Previously Treated Metastatic Colorectal Cancer |
| NCT00056459 | PHASE3 | COMPLETED | Study of Oxaliplatin/5-FU/Leucovorin Plus Vatalanib Versus Oxaliplatin/5-FU/Leucovorin in Patients With Metastatic Colorectal Cancer. |
| NCT00101686 | PHASE3 | COMPLETED | Trial Of Irinotecan In Combination With Three Methods Of Administration Of Fluoropyrimidine. |
| NCT00123760 | PHASE2/PHASE3 | COMPLETED | Study of 18F-Fluorodeoxyglucose (FluGlucoScan) in Patients With Cancer or Suspected Cancer |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| CETUXIMAB | 4 | 25 |
| IRINOTECAN | 4 | 16 |
| PEMBROLIZUMAB | 4 | 10 |
| REGORAFENIB | 4 | 9 |
| TALAPORFIN | 4 | 5 |
| FLOXURIDINE | 4 | 4 |
| LEVOLEUCOVORIN | 4 | 4 |
| NINTEDANIB | 4 | 4 |
| OXALIPLATIN | 4 | 4 |
| PANITUMUMAB | 4 | 4 |
| AFATINIB | 4 | 3 |
| BEVACIZUMAB | 4 | 3 |
| CAPECITABINE | 4 | 3 |
| LEUCOVORIN | 4 | 3 |
| PROPRANOLOL | 4 | 3 |
| AMIVANTAMAB | 4 | 2 |
| AXITINIB | 4 | 2 |
| CALCIUM | 4 | 2 |
| ETODOLAC | 4 | 2 |
| FERRIC CARBOXYMALTOSE | 4 | 2 |
| FLUOROURACIL | 4 | 2 |
| FOLIC ACID | 4 | 2 |
| GLUTAMINE | 4 | 2 |
| INDIGOTINDISULFONATE | 4 | 2 |
| SUNITINIB | 4 | 2 |
| TIPIRACIL | 4 | 2 |
| TRAMETINIB | 4 | 2 |
| TREMELIMUMAB | 4 | 2 |
| TRIFLURIDINE | 4 | 2 |
| AFLIBERCEPT | 4 | 1 |
Related Atlas pages
- Cohort genes: BRAF
- Drugs: Cetuximab, Irinotecan, Pembrolizumab, Regorafenib, Talaporfin, Floxuridine, Levoleucovorin, Nintedanib, Oxaliplatin, Panitumumab, Afatinib, Bevacizumab, Capecitabine, Propranolol, Amivantamab, Axitinib, Calcium, Etodolac, Ferric Carboxymaltose, Fluorouracil, Folic Acid, Glutamine, Sunitinib, Tipiracil, Trametinib, Tremelimumab, Trifluridine, Aflibercept