kidney Wilms tumor
diseaseOn this page
Also known as adult nephroblastomaadult renal Wilms' tumourchildhood renal Wilms tumorchildhood renal Wilms tumourchildhood renal Wilms' cancerembryonal nephromanephroblastomanephroblastoma, malignantnonanaplastic renal Wilm's tumornonanaplastic renal Wilm's tumourrenal embryonic tumorrenal embryonic tumourrenal Wilms tumorrenal Wilms tumourrenal Wilms' tumorWilms tumorWilms tumor of the kidneyWilms tumourWilms tumour of the kidney
Summary
kidney Wilms tumor (MONDO:0019004) is a cancer (an umbrella term covering 8 Mondo subtypes) with 3 cohort genes (1 CIViC-evidence somatic driver) and 90 clinical trials. Top therapeutic interventions include dactinomycin, larotrectinib, and cabozantinib.
At a glance
- Classification: Cancer
- Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
- Umbrella term: 8 Mondo subtypes
- Cohort genes: 3
- Phenotypes (HPO): 27
- Clinical trials: 90
Clinical features
Epidemiology
Prevalence records
27 prevalence record(s), Orphanet, top 20 (validated / broadest geography first):
| Type | Class | Value | Geography | Validation |
|---|---|---|---|---|
| Annual incidence | 1-9 / 1 000 000 | 0.14 | Europe | Validated |
| Lifetime Prevalence | 1-9 / 100 000 | 3.65 | Europe | Validated |
| Point prevalence | 1-9 / 100 000 | Europe | Validated | |
| Prevalence at birth | 1-5 / 10 000 | 10 | Europe | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.144 | United Kingdom | Validated |
| Annual incidence | <1 / 1 000 000 | 0.072 | Bulgaria | Validated |
| Annual incidence | <1 / 1 000 000 | 0.006 | Czech Republic | Validated |
| Annual incidence | <1 / 1 000 000 | 0.089 | Germany | Validated |
| Annual incidence | <1 / 1 000 000 | 0.043 | Iceland | Validated |
| Annual incidence | <1 / 1 000 000 | 0.065 | Latvia | Validated |
| Annual incidence | <1 / 1 000 000 | 0.066 | Portugal | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.121 | Austria | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.195 | Belgium | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.155 | Croatia | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.139 | Estonia | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.158 | Finland | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.17 | Ireland | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.111 | Italy | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.12 | Lithuania | Validated |
| Annual incidence | 1-9 / 1 000 000 | 0.157 | Malta | Validated |
Signs & symptoms
Clinical features (HPO)
27 HPO clinical features (Orphanet curated; top 27 by frequency):
| HPO ID | Term | Frequency |
|---|---|---|
| HP:0002664 | Neoplasm | Very frequent (80-99%) |
| HP:0002667 | Nephroblastoma | Very frequent (80-99%) |
| HP:0031500 | Abdominal mass | Very frequent (80-99%) |
| HP:0002027 | Abdominal pain | Frequent (30-79%) |
| HP:0005580 | Duplication of renal pelvis | Frequent (30-79%) |
| HP:0012587 | Macroscopic hematuria | Frequent (30-79%) |
| HP:0000028 | Cryptorchidism | Occasional (5-29%) |
| HP:0000047 | Hypospadias | Occasional (5-29%) |
| HP:0000085 | Horseshoe kidney | Occasional (5-29%) |
| HP:0000086 | Ectopic kidney | Occasional (5-29%) |
| HP:0000526 | Aniridia | Occasional (5-29%) |
| HP:0000822 | Hypertension | Occasional (5-29%) |
| HP:0001528 | Hemihypertrophy | Occasional (5-29%) |
| HP:0001824 | Weight loss | Occasional (5-29%) |
| HP:0001901 | Polycythemia | Occasional (5-29%) |
| HP:0001903 | Anemia | Occasional (5-29%) |
| HP:0001945 | Fever | Occasional (5-29%) |
| HP:0002716 | Lymphadenopathy | Occasional (5-29%) |
| HP:0002896 | Neoplasm of the liver | Occasional (5-29%) |
| HP:0002907 | Microscopic hematuria | Occasional (5-29%) |
| HP:0003072 | Hypercalcemia | Occasional (5-29%) |
| HP:0008330 | Reduced von Willebrand factor activity | Occasional (5-29%) |
| HP:0012871 | Varicocele | Occasional (5-29%) |
| HP:0031105 | Abnormal uterus morphology | Occasional (5-29%) |
| HP:0033834 | Malaise | Occasional (5-29%) |
| HP:0100526 | Neoplasm of the lung | Occasional (5-29%) |
| HP:0002094 | Dyspnea | Very rare (<1-4%) |
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | kidney Wilms tumor |
| Mondo ID | MONDO:0019004 |
| Orphanet | 654 |
| DOID | DOID:2154, DOID:5176 |
| NCIT | C40407 |
| SNOMED CT | 302849000 |
| GARD | 0007892 |
| MedDRA | 10029145 |
| NORD | 1855 |
| Is cancer (heuristic) | yes |
Also known as: adult nephroblastoma · adult renal Wilms’ tumour · childhood renal Wilms tumor · childhood renal Wilms tumour · childhood renal Wilms’ cancer · embryonal nephroma · kidney Wilms tumor · nephroblastoma · nephroblastoma, malignant · nonanaplastic renal Wilm’s tumor · nonanaplastic renal Wilm’s tumour · renal embryonic tumor · renal embryonic tumour · renal Wilms tumor · renal Wilms tumour · renal Wilms’ tumor · Wilms tumor · Wilms tumor of the kidney · Wilms tumour · Wilms tumour of the kidney (+5 more)
Data availability: 2 GenCC gene-disease records · 59 cell lines · 8 intOGen driver records.
Disease family
An umbrella term covering 8 Mondo subtypes.
Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › cancer › malignant urinary system neoplasm › kidney cancer › kidney Wilms tumor
Related subtypes (7): kidney sarcoma, mesoblastic nephroma, renal carcinoma, Graham-Boyle-Troxell syndrome, malignant mixed epithelial stromal tumor of the kidney, childhood malignant kidney neoplasm, malignant renal pelvis neoplasm
Subtypes (8): nonanaplastic kidney Wilms tumor, metachronous kidney Wilms’ tumor, mixed cell type kidney Wilms’ tumor, blastema predominant kidney Wilms tumor, epithelial predominant Wilms’ tumor, stromal predominant kidney Wilms tumor, adult kidney Wilms tumor, childhood kidney Wilms tumor
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: 12 · Orphanet: 23 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0
Somatic driver evidence (intOGen + CIViC, cohort fanout)
| Gene | intOGen role | Cancer types | CIViC |
|---|---|---|---|
| TP53 | LoF | ACC,ALL,AML,ANGS,ANSC,BCC,BL,BLADDER,BLCA,BRCA,CCRCC,CEAD,CESC,CHOL,CHRCC,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,EGC,ES,ESCA,ESCC,GB,GBC,GBM,GIST,HCC,HGGNOS,HNSC,LGGNOS,LIPO,LMS,LNM,LUAD,LUSC,MBL,MEL,MLYM,MT,NBL,NETNOS,NHL,NPC,NSCLC,OS,OVT,PAAD,PANCREAS,PAST,PCM,PLMESO,PRAD,PRCC,PROSTATE,RCC,READ,SACA,SARCNOS,SCLC,SIC,SKCM,SKIN,SOFT_TISSUE,STAD,STOMACH,THYM,UCEC,UCS,UTUC,VULVA,WDTC,WT | CIViC #45 |
GenCC gene–disease validity (cohort genes)
the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.
| Gene | Classification | Inheritance | Disease | Records |
|---|---|---|---|---|
| TRIM28 | Definitive | Autosomal dominant | childhood kidney Wilms tumor | 2 |
| TRIP13 | Supportive | Autosomal dominant | kidney Wilms tumor | 10 |
Orphanet rare-disease linkage (cohort genes)
| Gene | Orphanet ID | Rare disease |
|---|---|---|
| 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 |
| TP53 | Orphanet:70573 | Small cell lung cancer |
| TP53 | Orphanet:96253 | Cushing disease |
| TP53 | Orphanet:99756 | Alveolar rhabdomyosarcoma |
| TP53 | Orphanet:99757 | Embryonal rhabdomyosarcoma |
| TRIP13 | Orphanet:1052 | Mosaic variegated aneuploidy syndrome |
| TRIP13 | Orphanet:654 | Nephroblastoma |
| TRIM28 | Orphanet:654 | Nephroblastoma |
Cohort genes → proteins
3 cohort genes, 3 distinct canonical proteins.
Evidence partition
| Subset | Genes |
|---|---|
| civic_only | 1 |
| multi_evidence | 2 |
Cohort genes (full)
| Symbol | HGNC | Ensembl | UniProt | Name | Evidence |
|---|---|---|---|---|---|
| TP53 | HGNC:11998 | ENSG00000141510 | P04637 | Cellular tumor antigen p53 | civic_evidence |
| TRIP13 | HGNC:12307 | ENSG00000071539 | Q15645 | Pachytene checkpoint protein 2 homolog | gencc |
| TRIM28 | HGNC:16384 | ENSG00000130726 | Q13263 | Transcription intermediary factor 1-beta | gencc |
Cohort function summary
Lead sentence per gene, UniProt-curated.
| Symbol | Protein name | Function (lead sentence) |
|---|---|---|
| TP53 | Cellular tumor antigen p53 | Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence. |
| TRIP13 | Pachytene checkpoint protein 2 homolog | Plays a key role in chromosome recombination and chromosome structure development during meiosis. |
| TRIM28 | Transcription intermediary factor 1-beta | Nuclear corepressor for KRAB domain-containing zinc finger proteins (KRAB-ZFPs). |
Protein-family classification
Druggable: 0 · Difficult: 2 · Unknown: 1 · Druggable fraction: 0.0
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 |
|---|---|---|---|
| Transcription factor | 2 | 5.5× | 0.081 |
| Other/Unknown | 1 | 0.6× | 0.914 |
Per-gene assignment
| Symbol | Family | Druggable? | EC | InterPro (top 3) |
|---|---|---|---|---|
| TP53 | Transcription factor | no | p53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn | |
| TRIP13 | Other/Unknown | no | ClpA/B, AAA+_ATPase, ATPase_AAA_core | |
| TRIM28 | Transcription factor | no | Znf_B-box, Bromodomain, Znf_RING |
Expression context
Cohort genes with no expression data: 0.
3 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) | 3 |
| unknown | 0 |
Top tissues across cohort
| Tissue | Cohort genes |
|---|---|
| ventricular zone | 2 |
| ganglionic eminence | 1 |
| tendon of biceps brachii | 1 |
| bronchial epithelial cell | 1 |
| bronchus | 1 |
| epithelium of bronchus | 1 |
| left testis | 1 |
| right testis | 1 |
Per-gene tissue summary (top 30)
| Symbol | Bgee breadth | FANTOM5 breadth | SCXA | Top tissues |
|---|---|---|---|---|
| TP53 | 223 | ubiquitous | marker | ventricular zone, ganglionic eminence, tendon of biceps brachii |
| TRIP13 | 212 | ubiquitous | marker | bronchial epithelial cell, epithelium of bronchus, bronchus |
| TRIM28 | 145 | ubiquitous | marker | left testis, right testis, ventricular zone |
Protein interactions among cohort
Intra-cohort edges: 0.
Hub genes (top 10 by interactor count)
| Symbol | Interactor count |
|---|---|
| TP53 | 22,736 |
| TRIM28 | 8,167 |
| TRIP13 | 4,676 |
Structural data
PDB: 3 · AlphaFold-only: 0 · No structure: 0
Cohort genes with PDB structures (top 30)
| Symbol | UniProt | PDB entries |
|---|---|---|
| TP53 | P04637 | 313 |
| TRIM28 | Q13263 | 10 |
| TRIP13 | Q15645 | 6 |
Function
Pathway analysis
Distinct Reactome pathways touched by cohort: 62. Enrichment computed across 3 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 |
|---|---|---|---|---|
| Loss of function of TP53 in cancer due to loss of tetramerization ability | 1 | 5710.0× | 0.011 | TP53 |
| Regulation of TP53 Expression | 1 | 2855.0× | 0.011 | TP53 |
| Transcriptional activation of cell cycle inhibitor p21 | 1 | 1427.5× | 0.011 | TP53 |
| Activation of NOXA and translocation to mitochondria | 1 | 951.7× | 0.011 | TP53 |
| RUNX3 regulates CDKN1A transcription | 1 | 815.7× | 0.011 | TP53 |
| PI5P Regulates TP53 Acetylation | 1 | 634.4× | 0.011 | TP53 |
| Activation of PUMA and translocation to mitochondria | 1 | 571.0× | 0.011 | TP53 |
| TP53 Regulates Transcription of Caspase Activators and Caspases | 1 | 475.8× | 0.011 | TP53 |
| TP53 Regulates Transcription of Death Receptors and Ligands | 1 | 475.8× | 0.011 | TP53 |
| Urea cycle | 1 | 439.2× | 0.011 | TP53 |
| Regulation of TP53 Activity through Association with Co-factors | 1 | 407.9× | 0.011 | TP53 |
| TP53 regulates transcription of several additional cell death genes whose specific roles in p53-dependent apoptosis remain uncertain | 1 | 380.7× | 0.011 | TP53 |
| Stabilization of p53 | 1 | 380.7× | 0.011 | TP53 |
| TP53 Regulates Transcription of Genes Involved in G1 Cell Cycle Arrest | 1 | 356.9× | 0.011 | TP53 |
| Formation of Senescence-Associated Heterochromatin Foci (SAHF) | 1 | 335.9× | 0.011 | TP53 |
| Zygotic genome activation (ZGA) | 1 | 335.9× | 0.011 | TP53 |
| Regulation of NF-kappa B signaling | 1 | 317.2× | 0.011 | TP53 |
| TP53 Regulates Transcription of Genes Involved in G2 Cell Cycle Arrest | 1 | 300.5× | 0.011 | TP53 |
| SUMOylation of transcription factors | 1 | 285.5× | 0.011 | TP53 |
| TP53 Regulates Transcription of Genes Involved in Cytochrome C Release | 1 | 271.9× | 0.011 | TP53 |
| Regulation of TP53 Activity through Methylation | 1 | 271.9× | 0.011 | TP53 |
| TP53 regulates transcription of additional cell cycle genes whose exact role in the p53 pathway remain uncertain | 1 | 259.6× | 0.011 | TP53 |
| Regulation of TP53 Activity through Acetylation | 1 | 228.4× | 0.012 | TP53 |
| Pyroptosis | 1 | 211.5× | 0.012 | TP53 |
| Regulation of endogenous retroelements | 1 | 184.2× | 0.013 | TRIM28 |
| Oncogene Induced Senescence | 1 | 167.9× | 0.014 | TP53 |
| HCMV Infection | 1 | 163.1× | 0.014 | TRIM28 |
| Association of TriC/CCT with target proteins during biosynthesis | 1 | 146.4× | 0.015 | TP53 |
| Regulation of TP53 Degradation | 1 | 146.4× | 0.015 | TP53 |
| Ovarian tumor domain proteases | 1 | 139.3× | 0.015 | TP53 |
GO biological processes by enrichment
Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.
| GO term | Cohort genes | Fold | FDR | Sample cohort genes |
|---|---|---|---|---|
| negative regulation of helicase activity | 1 | 5617.3× | 0.005 | TP53 |
| cellular response to actinomycin D | 1 | 5617.3× | 0.005 | TP53 |
| regulation of intrinsic apoptotic signaling pathway by p53 class mediator | 1 | 5617.3× | 0.005 | TP53 |
| negative regulation of G1 to G0 transition | 1 | 5617.3× | 0.005 | TP53 |
| positive regulation of mitochondrial membrane permeability | 1 | 2808.7× | 0.005 | TP53 |
| oligodendrocyte apoptotic process | 1 | 2808.7× | 0.005 | TP53 |
| negative regulation of glucose catabolic process to lactate via pyruvate | 1 | 2808.7× | 0.005 | TP53 |
| negative regulation of pentose-phosphate shunt | 1 | 2808.7× | 0.005 | TP53 |
| obsolete homolactic fermentation | 1 | 1872.4× | 0.005 | TP53 |
| meiotic recombination checkpoint signaling | 1 | 1872.4× | 0.005 | TRIP13 |
| signal transduction by p53 class mediator | 1 | 1872.4× | 0.005 | TP53 |
| negative regulation of miRNA processing | 1 | 1872.4× | 0.005 | TP53 |
| intrinsic apoptotic signaling pathway in response to hypoxia | 1 | 1872.4× | 0.005 | TP53 |
| regulation of fibroblast apoptotic process | 1 | 1872.4× | 0.005 | TP53 |
| T cell proliferation involved in immune response | 1 | 1404.3× | 0.005 | TP53 |
| positive regulation of programmed necrotic cell death | 1 | 1404.3× | 0.005 | TP53 |
| oxidative stress-induced premature senescence | 1 | 1404.3× | 0.005 | TP53 |
| B cell lineage commitment | 1 | 1123.5× | 0.005 | TP53 |
| T cell lineage commitment | 1 | 1123.5× | 0.005 | TP53 |
| mRNA transcription | 1 | 1123.5× | 0.005 | TP53 |
| positive regulation of RNA polymerase II transcription preinitiation complex assembly | 1 | 1123.5× | 0.005 | TP53 |
| convergent extension involved in axis elongation | 1 | 1123.5× | 0.005 | TRIM28 |
| embryonic placenta morphogenesis | 1 | 1123.5× | 0.005 | TRIM28 |
| positive regulation of thymocyte apoptotic process | 1 | 1123.5× | 0.005 | TP53 |
| cellular response to UV-C | 1 | 1123.5× | 0.005 | TP53 |
| double-strand break repair | 2 | 135.4× | 0.005 | TP53, TRIP13 |
| regulation of mitochondrial membrane permeability involved in apoptotic process | 1 | 936.2× | 0.006 | TP53 |
| viral process | 1 | 802.5× | 0.006 | TP53 |
| mitochondrial DNA repair | 1 | 802.5× | 0.006 | TP53 |
| regulation of cell cycle G2/M phase transition | 1 | 802.5× | 0.006 | TP53 |
Therapeutics
Drug target analysis
Approved (phase 4): 1 · Phase ≥3: 1 · Phased (≥1): 1 · Undrugged: 2
Druggability breadth: 3 of 3 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 |
|---|---|
| TP53 | NITROFURANTOIN |
Top cohort targets by molecule count
| Symbol | Molecules | Max phase |
|---|---|---|
| TP53 | 196 | 4 |
| TRIP13 | 0 | 0 |
| TRIM28 | 0 | 0 |
Drugs targeting cohort genes (top 30)
| Molecule | Max phase | Targets in cohort |
|---|---|---|
| NITROFURANTOIN | 4 | TP53 |
| DIOSMIN | 4 | TP53 |
| VERTEPORFIN | 4 | TP53 |
| CANDESARTAN CILEXETIL | 4 | TP53 |
| DIENESTROL | 4 | TP53 |
| CLOTRIMAZOLE | 4 | TP53 |
| COLCHICINE | 4 | TP53 |
| NABUMETONE | 4 | TP53 |
| SALMETEROL XINAFOATE | 4 | TP53 |
| AMIODARONE HYDROCHLORIDE | 4 | TP53 |
| FURAZOLIDONE | 4 | TP53 |
| AMOXAPINE | 4 | TP53 |
| RALOXIFENE HYDROCHLORIDE | 4 | TP53 |
| NICARDIPINE HYDROCHLORIDE | 4 | TP53 |
| SULCONAZOLE NITRATE | 4 | TP53 |
| PYRITHIONE ZINC | 4 | TP53 |
| LACTIC ACID | 4 | TP53 |
| OXYMETHOLONE | 4 | TP53 |
| CHLOROXINE | 4 | TP53 |
| PROPIOLACTONE | 4 | TP53 |
| CLOMIPRAMINE HYDROCHLORIDE | 4 | TP53 |
| PHENYL AMINOSALICYLATE | 4 | TP53 |
| THIORIDAZINE HYDROCHLORIDE | 4 | TP53 |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | TP53 |
| ETHOPROPAZINE HYDROCHLORIDE | 4 | TP53 |
| MECHLORETHAMINE HYDROCHLORIDE | 4 | TP53 |
| ECONAZOLE NITRATE | 4 | TP53 |
| TRIFLUPROMAZINE HYDROCHLORIDE | 4 | TP53 |
| PROCHLORPERAZINE EDISYLATE | 4 | TP53 |
| DEQUALINIUM CHLORIDE | 4 | TP53 |
Bioactivity and enzyme data
Enzyme cohort genes (≥1 EC): 0.
Cohort genes with ChEMBL bioactivity (full, sorted by assay count)
| Symbol | Assays | Type breakdown |
|---|---|---|
| TP53 | 869 | Binding:775, ADMET:83, Functional:10, Toxicity:1 |
| TRIM28 | 19 | Binding:19 |
| TRIP13 | 9 | Binding:9 |
Cohort genes with high screening signal
≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.
| Symbol | ChEMBL assays |
|---|---|
| TP53 | 869 |
Pharmacogenomics
Cohort genes with a PharmGKB record: 3; 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) |
|---|---|---|
| NITROFURANTOIN | 4 | TP53 |
| DIOSMIN | 4 | TP53 |
| VERTEPORFIN | 4 | TP53 |
| CANDESARTAN CILEXETIL | 4 | TP53 |
| DIENESTROL | 4 | TP53 |
| CLOTRIMAZOLE | 4 | TP53 |
| COLCHICINE | 4 | TP53 |
| NABUMETONE | 4 | TP53 |
| SALMETEROL XINAFOATE | 4 | TP53 |
| AMIODARONE HYDROCHLORIDE | 4 | TP53 |
| FURAZOLIDONE | 4 | TP53 |
| AMOXAPINE | 4 | TP53 |
| RALOXIFENE HYDROCHLORIDE | 4 | TP53 |
| NICARDIPINE HYDROCHLORIDE | 4 | TP53 |
| SULCONAZOLE NITRATE | 4 | TP53 |
| PYRITHIONE ZINC | 4 | TP53 |
| LACTIC ACID | 4 | TP53 |
| OXYMETHOLONE | 4 | TP53 |
| CHLOROXINE | 4 | TP53 |
| PROPIOLACTONE | 4 | TP53 |
| CLOMIPRAMINE HYDROCHLORIDE | 4 | TP53 |
| PHENYL AMINOSALICYLATE | 4 | TP53 |
| THIORIDAZINE HYDROCHLORIDE | 4 | TP53 |
| AMITRIPTYLINE HYDROCHLORIDE | 4 | TP53 |
| ETHOPROPAZINE HYDROCHLORIDE | 4 | TP53 |
| MECHLORETHAMINE HYDROCHLORIDE | 4 | TP53 |
| ECONAZOLE NITRATE | 4 | TP53 |
| TRIFLUPROMAZINE HYDROCHLORIDE | 4 | TP53 |
| PROCHLORPERAZINE EDISYLATE | 4 | TP53 |
| DEQUALINIUM CHLORIDE | 4 | TP53 |
Druggability pyramid
Cohort genes binned by druggability tier (high → low):
| Tier | Definition | Genes | Symbols |
|---|---|---|---|
| A | Approved (phase 4 drug) | 1 | TP53 |
| 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 | 2 | TRIP13, TRIM28 |
Undrugged target profiles
2 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).
| Symbol | ChEMBL assays | Drugged partners (top 3) |
|---|---|---|
| TRIP13 | 9 | — |
| TRIM28 | 19 | — |
Clinical trials & evidence
Clinical trials
Clinical trials: 90.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| PHASE2 | 28 |
| Not specified | 28 |
| PHASE1 | 25 |
| PHASE1/PHASE2 | 4 |
| PHASE3 | 3 |
| PHASE4 | 1 |
| EARLY_PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00336531 | PHASE4 | COMPLETED | Efficacy of Prophylactic Itraconazole in High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation |
| NCT00352534 | PHASE3 | ACTIVE_NOT_RECRUITING | Vincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms’ Tumor |
| NCT00379340 | PHASE3 | ACTIVE_NOT_RECRUITING | Combination Chemotherapy With or Without Radiation Therapy in Treating Young Patients With Newly Diagnosed Stage III or Stage IV Wilms’ Tumor |
| NCT00945009 | PHASE3 | ACTIVE_NOT_RECRUITING | Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor |
| NCT02867592 | PHASE2 | ACTIVE_NOT_RECRUITING | Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors |
| NCT03155620 | PHASE2 | ACTIVE_NOT_RECRUITING | Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) |
| NCT03210714 | PHASE2 | ACTIVE_NOT_RECRUITING | Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial) |
| NCT03213652 | PHASE2 | ACTIVE_NOT_RECRUITING | Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial) |
| NCT03213704 | PHASE2 | ACTIVE_NOT_RECRUITING | Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial) |
| NCT03698994 | PHASE2 | ACTIVE_NOT_RECRUITING | Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial) |
| NCT04195555 | PHASE2 | ACTIVE_NOT_RECRUITING | Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) |
| NCT04284774 | PHASE2 | ACTIVE_NOT_RECRUITING | Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial |
| NCT04320888 | PHASE2 | ACTIVE_NOT_RECRUITING | Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial |
| NCT04322318 | PHASE2 | RECRUITING | A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT |
| NCT04851119 | PHASE1/PHASE2 | RECRUITING | Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors |
| NCT04901702 | PHASE1/PHASE2 | RECRUITING | Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma |
| NCT04968990 | PHASE2 | RECRUITING | Treatment of Newly Diagnosed Patient’s With Wilm’s Tumor Requiring Abdominal Radiation Delivered With Proton Beam Irradiation |
| NCT05384821 | PHASE1/PHASE2 | RECRUITING | Metronomic Chemotherapy in Wilms Tumor (MetroWilms-1906) |
| NCT05985161 | PHASE2 | RECRUITING | A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors |
| NCT00001509 | PHASE2 | COMPLETED | A Phase II Trial of All-Trans-Retinoic Acid in Combination With Interferon-Alpha 2a in Children With Recurrent Neuroblastoma or Wilms’ Tumor |
| NCT00038207 | PHASE2 | COMPLETED | Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies |
| NCT00141765 | PHASE2 | COMPLETED | Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers |
| NCT00187031 | PHASE2 | COMPLETED | A Phase II Study of Topotecan in Children With Recurrent Wilms Tumor |
| NCT00335556 | PHASE2 | COMPLETED | Combination Chemotherapy, Radiation Therapy, and/or Surgery in Treating Patients With High-Risk Kidney Tumors |
| NCT01095926 | PHASE2 | COMPLETED | Pharmacokinetic Study of Doxorubicin in Children With Cancer |
| NCT02452554 | PHASE2 | COMPLETED | Lorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma |
| NCT02581384 | PHASE1/PHASE2 | TERMINATED | Stereotactic Body Radiotherapy (SBRT) for Pulmonary Metastases in Ewing Sarcoma, Rhabdomyosarcoma, and Wilms Tumors |
| NCT02624388 | PHASE2 | TERMINATED | Study of Genistein in Pediatric Oncology Patients (UVA-Gen001) |
| NCT02689336 | PHASE2 | WITHDRAWN | Erlotinib in Combination With Temozolomide in Treating Relapsed/Recurrent/Refractory Pediatric Solid Tumors |
| NCT03213665 | PHASE2 | COMPLETED | Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial) |
| NCT03213678 | PHASE2 | COMPLETED | Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial) |
| NCT03220035 | PHASE2 | COMPLETED | Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial) |
| NCT03233204 | PHASE2 | COMPLETED | Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial) |
| NCT03526250 | PHASE2 | COMPLETED | Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial) |
| NCT04791228 | PHASE2 | WITHDRAWN | A Pilot Study of Thermodox and MR-HIFU for Treatment of Relapsed Solid Tumors |
| NCT05302921 | PHASE2 | COMPLETED | Neoadjuvant Dual Checkpoint Inhibition and Cryoablation in Relapsed/Refractory Pediatric Solid Tumors |
| NCT03618381 | PHASE1 | ACTIVE_NOT_RECRUITING | EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults |
| NCT04308330 | PHASE1 | RECRUITING | Vorinostat in Combination With Chemotherapy in Relapsed/Refractory Solid Tumors and CNS Malignancies |
| NCT04377932 | PHASE1 | ACTIVE_NOT_RECRUITING | Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors |
| NCT04483778 | PHASE1 | ACTIVE_NOT_RECRUITING | B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| DACTINOMYCIN | 4 | 4 |
| LAROTRECTINIB | 4 | 4 |
| CABOZANTINIB | 4 | 3 |
| ENSARTINIB | 4 | 2 |
| ERDAFITINIB | 4 | 2 |
| IVOSIDENIB | 4 | 2 |
| SELPERCATINIB | 4 | 2 |
| SELUMETINIB | 4 | 2 |
| TAZEMETOSTAT | 4 | 2 |
| VEMURAFENIB | 4 | 2 |
| ITRACONAZOLE | 4 | 1 |
| PEGFILGRASTIM | 4 | 1 |
| SELINEXOR | 4 | 1 |
| TALAZOPARIB | 4 | 1 |
| TOPOTECAN | 4 | 1 |
| TIPIFARNIB | 3 | 2 |
| BECOTATUG VEDOTIN | 3 | 1 |
| DOCIPARSTAT SODIUM | 3 | 1 |
| GALINPEPIMUT-S | 3 | 1 |
| TARIQUIDAR | 3 | 1 |
| SAMOTOLISIB | 2 | 2 |
| ULIXERTINIB | 2 | 2 |
| ENOBLITUZUMAB | 2 | 1 |
| GENISTEIN | 2 | 1 |
| LORVOTUZUMAB MERTANSINE | 2 | 1 |
| TEGAVIVINT | 2 | 1 |
| CHEMBL4748391 | 0 | 4 |
| CHEMBL3415553 | 0 | 2 |
| CHEMBL4209555 | 0 | 2 |
| CHEMBL5398431 | 0 | 2 |
Related Atlas pages
- Cohort genes: TP53, TRIP13, TRIM28
- Drugs: Dactinomycin, Larotrectinib, Cabozantinib, Ensartinib, Erdafitinib, Ivosidenib, Selpercatinib, Selumetinib, Tazemetostat, Vemurafenib, Itraconazole, Pegfilgrastim, Selinexor, Talazoparib, Topotecan, Tipifarnib, Becotatug Vedotin, Dociparstat, Galinpepimut-S, Tariquidar