Wilms tumor 1

Summary

Wilms tumor 1 (MONDO:0008679) is a cancer caused by WT1 (GenCC Definitive), with 10 cohort genes (4 CIViC-evidence somatic drivers; 2,877 ClinVar predisposition records) and 6 clinical trials. Top therapeutic interventions include dactinomycin, vincristine, and dociparstat sodium.

At a glance

• Classification: Cancer
• Causal gene: WT1 (GenCC Definitive)
• Cohort genes: 10
• ClinVar variants: 2,877
• Clinical trials: 6

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	Wilms tumor 1
Mondo ID	MONDO:0008679
OMIM	194070
GARD	0015124
Is cancer (heuristic)	yes

Also known as: renal Wilms tumor · renal Wilms tumour · Wilms tumor 1 · Wilms tumor type 1 · Wilms tumor, autosomal dominant, somatic mutation · Wilms tumor, somatic · Wilms tumor, type 1, autosomal dominant, somatic mutation · Wilms tumour type 1 · Wilms’ tumor · Wilms’ tumour · WT1 · WT1-related Wilms tumor predisposition

Data availability: 2,877 ClinVar variants · 3 GenCC gene-disease records · 8 intOGen driver records.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › hereditary Wilms tumor › Wilms tumor 1

Related subtypes (6): Wilms tumor 2, Wilms tumor 3, Wilms tumor 4, Wilms tumor 5, Wilms tumor 6, Wilms tumor 7

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

600 retrieved; paginated sample, class counts are floors:

304 uncertain significance, 202 likely benign, 43 conflicting classifications of pathogenicity, 32 pathogenic, 8 benign, 4 benign/likely benign, 4 likely pathogenic, 3 pathogenic/likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
126014	NM_000059.4(BRCA2):c.3458del (p.Lys1153fs)	BRCA2	Pathogenic	reviewed by expert panel
126037	NM_000059.4(BRCA2):c.4131_4132insTGAGGA (p.Thr1378Ter)	BRCA2	Pathogenic	reviewed by expert panel
1330148	NM_000059.4(BRCA2):c.6611del (p.Pro2204fs)	BRCA2	Pathogenic	criteria provided, multiple submitters, no conflicts
141199	NM_000059.4(BRCA2):c.2918C>A (p.Ser973Ter)	BRCA2	Pathogenic	reviewed by expert panel
141283	NM_000059.4(BRCA2):c.1909+1G>A	BRCA2	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
141509	NM_000059.4(BRCA2):c.6816_6820del (p.Gly2274fs)	BRCA2	Pathogenic	reviewed by expert panel
142868	NM_000059.4(BRCA2):c.2059_2063del (p.Leu686_Asp687insTer)	BRCA2	Pathogenic	reviewed by expert panel
1439264	NM_000059.4(BRCA2):c.1766dup (p.Phe590fs)	BRCA2	Pathogenic	criteria provided, multiple submitters, no conflicts
1456139	NM_000059.4(BRCA2):c.4793_4794del (p.Leu1598fs)	BRCA2	Pathogenic	criteria provided, multiple submitters, no conflicts
1067035	NM_004484.4(GPC3):c.1569del (p.Ala524fs)	GPC3	Pathogenic	criteria provided, single submitter
1069346	NC_000023.10:g.(?132670152)(133119476_?)del	GPC3	Pathogenic	criteria provided, single submitter
1069347	NC_000023.10:g.(?132670146)(132670327_?)del	GPC3	Pathogenic	criteria provided, single submitter
1069348	NC_000023.10:g.(?132795752)(132888209_?)del	GPC3	Pathogenic	criteria provided, single submitter
1074502	NM_004484.4(GPC3):c.892G>T (p.Glu298Ter)	GPC3	Pathogenic	criteria provided, multiple submitters, no conflicts
11689	NM_004484.4(GPC3):c.595C>T (p.Arg199Ter)	GPC3	Pathogenic	criteria provided, multiple submitters, no conflicts
11690	NM_004484.4(GPC3):c.361C>T (p.His121Tyr)	GPC3	Pathogenic	no assertion criteria provided
11694	NM_004484.4(GPC3):c.1159C>T (p.Arg387Ter)	GPC3	Pathogenic	criteria provided, multiple submitters, no conflicts
1333337	NM_004484.4(GPC3):c.271del (p.Gln91fs)	GPC3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1433596	NM_004484.4(GPC3):c.885C>A (p.Tyr295Ter)	GPC3	Pathogenic	criteria provided, single submitter
1453391	NM_004484.4(GPC3):c.1379_1382del (p.Ser460fs)	GPC3	Pathogenic	criteria provided, single submitter
1455474	NM_004484.4(GPC3):c.175+2T>C	GPC3	Pathogenic	criteria provided, single submitter
1455674	NM_004484.4(GPC3):c.662del (p.Lys221fs)	GPC3	Pathogenic	criteria provided, single submitter
1458423	NC_000023.10:g.(?132670152)(132888213_?)del	GPC3	Pathogenic	criteria provided, single submitter
1460460	NC_000023.10:g.(?132670132)(133119496_?)del	GPC3	Pathogenic	criteria provided, single submitter
1075590	NM_024426.6(WT1):c.455del (p.Gly152fs)	LOC107982234	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1453898	NM_024426.6(WT1):c.276del (p.Gly93fs)	LOC107982234	Pathogenic	criteria provided, single submitter
1456851	NM_024426.6(WT1):c.314_318dup (p.Trp107fs)	LOC107982234	Pathogenic	criteria provided, single submitter
1069921	NC_000011.10:g.32396408del	WT1	Pathogenic	criteria provided, single submitter
1073615	NC_000011.9:g.(?32456236)(32460464_?)del	WT1	Pathogenic	criteria provided, single submitter
1073616	NC_000011.9:g.(?32421484)(32421600_?)del	WT1	Pathogenic	criteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 7 · Orphanet: 36 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
WT1	LoF	AML,MEL,PAAD	CIViC #49
BRCA2	LoF	BLCA,BRCA,CESC,CHOL,HCC,HNSC,LUSC,MBL,OVT,PAAD,PRAD,PROSTATE,RCC,VULVA	CIViC #7
MED12	Act	ALL,CESC,CLLSLL,GBM,LGGNOS,NBL,PRAD,PROSTATE,UCEC
IGF2			CIViC #60

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
WT1	Definitive	Autosomal dominant	Wilms tumor 1	7

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
WT1	Orphanet:220	Denys-Drash syndrome
WT1	Orphanet:242	46,XY complete gonadal dysgenesis
WT1	Orphanet:251510	46,XY partial gonadal dysgenesis
WT1	Orphanet:3097	Meacham syndrome
WT1	Orphanet:347	Frasier syndrome
WT1	Orphanet:654	Nephroblastoma
WT1	Orphanet:656	Hereditary steroid-resistant nephrotic syndrome
WT1	Orphanet:83469	Desmoplastic small round cell tumor
WT1	Orphanet:893	WAGR syndrome
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
MED12	Orphanet:1415	Hardikar syndrome
MED12	Orphanet:293707	Blepharophimosis-intellectual disability syndrome, MKB type
MED12	Orphanet:776	Lujan-Fryns syndrome
MED12	Orphanet:777	X-linked non-syndromic intellectual disability
MED12	Orphanet:93932	FG syndrome type 1
TRIM28	Orphanet:654	Nephroblastoma
GPC3	Orphanet:373	Simpson-Golabi-Behmel syndrome
GPC3	Orphanet:654	Nephroblastoma
GPC4	Orphanet:2662	Keipert syndrome
GPC4	Orphanet:373	Simpson-Golabi-Behmel syndrome
IGF2	Orphanet:2128	Isolated hemihyperplasia
IGF2	Orphanet:231117	Beckwith-Wiedemann syndrome due to imprinting defect of 11p15
IGF2	Orphanet:231140	Silver-Russell syndrome due to an imprinting defect of 11p15
IGF2	Orphanet:231144	Silver-Russell syndrome due to 11p15 microduplication
IGF2	Orphanet:397590	Silver-Russell syndrome due to a point mutation

Cohort genes → proteins

10 cohort genes, 10 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	10

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
WT1	HGNC:12796	ENSG00000184937	P19544	Wilms tumor protein	gencc,clinvar
BRCA2	HGNC:1101	ENSG00000139618	P51587	Breast cancer type 2 susceptibility protein	clinvar
ELP4	HGNC:1171	ENSG00000109911	Q96EB1	Elongator complex protein 4	clinvar
MED12	HGNC:11957	ENSG00000184634	Q93074	Mediator of RNA polymerase II transcription subunit 12	clinvar
TRIM28	HGNC:16384	ENSG00000130726	Q13263	Transcription intermediary factor 1-beta	clinvar
WT1-AS	HGNC:18135	ENSG00000183242	Q06250	Putative Wilms tumor upstream neighbor 1 gene protein	clinvar
CCDC160	HGNC:37286	ENSG00000203952	A6NGH7	Coiled-coil domain-containing protein 160	clinvar
GPC3	HGNC:4451	ENSG00000147257	P51654	Glypican-3	clinvar
GPC4	HGNC:4452	ENSG00000076716	O75487	Glypican-4	clinvar
IGF2	HGNC:5466	ENSG00000167244	P01344	Insulin-like growth factor 2	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
WT1	Wilms tumor protein	Transcription factor that plays an important role in cellular development and cell survival.
BRCA2	Breast cancer type 2 susceptibility protein	Involved in double-strand break repair and/or homologous recombination.
ELP4	Elongator complex protein 4	Component of the elongator complex which is required for multiple tRNA modifications, including mcm5U (5-methoxycarbonylmethyl uridine), mcm5s2U (5-methoxycarbonylmethyl-2-thiouridine), and ncm5U (5-carbamoylmethyl uridine).
MED12	Mediator of RNA polymerase II transcription subunit 12	Component of the Mediator complex, a coactivator involved in the regulated transcription of nearly all RNA polymerase II-dependent genes.
TRIM28	Transcription intermediary factor 1-beta	Nuclear corepressor for KRAB domain-containing zinc finger proteins (KRAB-ZFPs).
GPC3	Glypican-3	Cell surface proteoglycan.
GPC4	Glypican-4	Cell surface proteoglycan that bears heparan sulfate.
IGF2	Insulin-like growth factor 2	The insulin-like growth factors possess growth-promoting activity.

Protein-family classification

Druggable: 0 · Difficult: 2 · Unknown: 8 · 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
Other/Unknown	8	1.4×	0.218
Transcription factor	2	1.6×	0.346

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
WT1	Transcription factor	no		Wilms_tumour_N, Znf_C2H2_type, Znf_C2H2_sf
BRCA2	Other/Unknown	no		BRCA2_repeat, NA-bd_OB-fold, BRCA2_OB_1
ELP4	Other/Unknown	no		Elongator_complex_protein_4, P-loop_NTPase
MED12	Other/Unknown	no		Mediator_Med12, Mediator_Med12_catenin-bd, Mediator_Med12_LCEWAV
TRIM28	Transcription factor	no		Znf_B-box, Bromodomain, Znf_RING
WT1-AS	Other/Unknown	no
CCDC160	Other/Unknown	no
GPC3	Other/Unknown	no		Glypican, Glypican_CS
GPC4	Other/Unknown	no		Glypican, Glypican_CS
IGF2	Other/Unknown	no		IGF2_C, Insulin-like, IGF2

Expression context

Cohort genes with no expression data: 0.

9 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)	10
unknown	0

Top tissues across cohort

Tissue	Cohort genes
ventricular zone	4
left ovary	2
germinal epithelium of ovary	1
metanephric glomerulus	1
renal glomerulus	1
male germ line stem cell (sensu Vertebrata) in testis	1
secondary oocyte	1
calcaneal tendon	1
primordial germ cell in gonad	1
right adrenal gland	1
right adrenal gland cortex	1
left testis	1
right testis	1
ovary	1
right uterine tube	1
buccal mucosa cell	1
olfactory segment of nasal mucosa	1
oviduct epithelium	1
right lung	1
tibial nerve	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
WT1	168	broad	marker	germinal epithelium of ovary, renal glomerulus, metanephric glomerulus
BRCA2	184	ubiquitous	marker	male germ line stem cell (sensu Vertebrata) in testis, secondary oocyte, ventricular zone
ELP4	250	ubiquitous	marker	ventricular zone, calcaneal tendon, primordial germ cell in gonad
MED12	281	ubiquitous	marker	right adrenal gland cortex, right adrenal gland, left ovary
TRIM28	145	ubiquitous	marker	left testis, right testis, ventricular zone
WT1-AS	106	tissue_specific	yes	right uterine tube, left ovary, ovary
CCDC160	142	broad	marker	oviduct epithelium, buccal mucosa cell, olfactory segment of nasal mucosa
GPC3	213	broad	marker	upper lobe of left lung, right lung, tibial nerve
GPC4	228	ubiquitous	marker	ventricular zone, ganglionic eminence, lower esophagus muscularis layer
IGF2	135	ubiquitous	marker	adrenal tissue, placenta, sural nerve

Protein interactions among cohort

Intra-cohort edges: 2.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
TRIM28	8,167
BRCA2	4,839
IGF2	4,294
WT1	3,938
MED12	3,322
GPC3	2,419
ELP4	1,740
GPC4	1,361
CCDC160	989
WT1-AS	35

Intra-cohort edges

A	B	Sources
GPC3	GPC4	string_interaction
GPC3	IGF2	string_interaction

Structural data

PDB: 6 · AlphaFold-only: 4 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
WT1	P19544	28
IGF2	P01344	16
BRCA2	P51587	14
TRIM28	Q13263	10
GPC3	P51654	4
MED12	Q93074	3

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
GPC4	O75487	83.68
ELP4	Q96EB1	74.49
CCDC160	A6NGH7	72.79
WT1-AS	Q06250	61.60

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 81. Enrichment computed across 10 evidence-associated genes (8 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 8 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Defective EXT2 causes exostoses 2	2	203.9×	9e-04	GPC3, GPC4
Defective EXT1 causes exostoses 1, TRPS2 and CHDS	2	203.9×	9e-04	GPC3, GPC4
Attachment and Entry	2	150.3×	9e-04	GPC3, GPC4
Defective B4GALT7 causes EDS, progeroid type	2	142.8×	9e-04	GPC3, GPC4
Defective B3GAT3 causes JDSSDHD	2	142.8×	9e-04	GPC3, GPC4
Defective B3GALT6 causes EDSP2 and SEMDJL1	2	142.8×	9e-04	GPC3, GPC4
RSV-host interactions	3	58.7×	9e-04	MED12, GPC3, GPC4
HS-GAG degradation	2	124.1×	1e-03	GPC3, GPC4
Respiratory syncytial virus (RSV) attachment and entry	2	124.1×	1e-03	GPC3, GPC4
Initiation of coagulation cascade	2	119.0×	1e-03	GPC3, GPC4
Glycosaminoglycan-protein linkage region biosynthesis	2	98.5×	0.001	GPC3, GPC4
HS-GAG biosynthesis	2	86.5×	0.002	GPC3, GPC4
Dengue Virus Attachment and Entry	2	64.9×	0.002	GPC3, GPC4
Retinoid metabolism and transport	2	62.1×	0.003	GPC3, GPC4
Regulation of clotting cascade	2	58.3×	0.003	GPC3, GPC4
Impaired BRCA2 translocation to the nucleus	1	475.8×	0.010	BRCA2
Impaired BRCA2 binding to SEM1 (DSS1)	1	475.8×	0.010	BRCA2
Regulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)	2	21.6×	0.016	GPC3, IGF2
Epigenetic regulation of gene expression	2	17.8×	0.022	MED12, TRIM28
SHC-related events triggered by IGF1R	1	142.8×	0.028	IGF2
IRS-related events triggered by IGF1R	1	129.8×	0.029	IGF2
Signaling by Type 1 Insulin-like Growth Factor 1 Receptor (IGF1R)	1	119.0×	0.029	IGF2
Defective homologous recombination repair (HRR) due to PALB2 loss of function	1	119.0×	0.029	BRCA2
HDR through MMEJ (alt-NHEJ)	1	109.8×	0.029	BRCA2
Nephron development	1	109.8×	0.029	WT1
Diseases of DNA Double-Strand Break Repair	1	102.0×	0.029	BRCA2
Defective homologous recombination repair (HRR) due to BRCA2 loss of function	1	102.0×	0.029	BRCA2
Transcriptional regulation of testis differentiation	1	89.2×	0.032	WT1
Resolution of D-Loop Structures	1	79.3×	0.034	BRCA2
Dengue Virus-Host Interactions	2	11.4×	0.034	GPC3, GPC4

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 8 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
embryonic placenta morphogenesis	2	842.6×	3e-04	TRIM28, IGF2
genomic imprinting	2	247.8×	0.002	TRIM28, IGF2
Wnt signaling pathway, planar cell polarity pathway	2	113.9×	0.005	MED12, GPC3
positive regulation of DNA-templated transcription	4	14.0×	0.005	WT1, BRCA2, MED12, TRIM28
branching involved in ureteric bud morphogenesis	2	91.6×	0.007	WT1, GPC3
body morphogenesis	1	2106.5×	0.009	GPC3
negative regulation of metanephric glomerular mesangial cell proliferation	1	2106.5×	0.009	WT1
regulation of animal organ formation	1	1053.2×	0.009	WT1
adrenal cortex formation	1	1053.2×	0.009	WT1
visceral serous pericardium development	1	1053.2×	0.009	WT1
mesenchymal cell proliferation involved in ureteric bud development	1	1053.2×	0.009	GPC3
posterior mesonephric tubule development	1	1053.2×	0.009	WT1
mesonephric duct morphogenesis	1	1053.2×	0.009	GPC3
cell proliferation involved in metanephros development	1	1053.2×	0.009	GPC3
mitotic recombination-dependent replication fork processing	1	1053.2×	0.009	BRCA2
regulation of non-canonical Wnt signaling pathway	1	1053.2×	0.009	GPC3
positive regulation of metanephric ureteric bud development	1	1053.2×	0.009	WT1
cell proliferation involved in kidney development	1	702.2×	0.012	GPC3
axis elongation involved in somitogenesis	1	702.2×	0.012	MED12
regulation of DNA-templated transcription	3	11.8×	0.013	WT1, BRCA2, IGF2
positive regulation of heart growth	1	526.6×	0.013	WT1
metanephric S-shaped body morphogenesis	1	526.6×	0.013	WT1
regulation of protein localization to membrane	1	526.6×	0.013	GPC3
negative regulation of female gonad development	1	526.6×	0.013	WT1
thorax and anterior abdomen determination	1	421.3×	0.013	WT1
negative regulation of mammary gland epithelial cell proliferation	1	421.3×	0.013	BRCA2
positive regulation of skeletal muscle tissue growth	1	421.3×	0.013	IGF2
convergent extension involved in axis elongation	1	421.3×	0.013	TRIM28
cardiac muscle cell fate commitment	1	421.3×	0.013	WT1
metanephric epithelium development	1	421.3×	0.013	WT1

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 1 · Undrugged: 9

Druggability breadth: 5 of 10 evidence-associated genes (50%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
MED12	1	2
WT1	0	0
BRCA2	0	0
ELP4	0	0
TRIM28	0	0
WT1-AS	0	0
CCDC160	0	0
GPC3	0	0
GPC4	0	0
IGF2	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
MOLIBRESIB	2	MED12

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
TRIM28	19	Binding:19
MED12	6	Binding:6
GPC3	5	Binding:5

Pharmacogenomics

Cohort genes with a PharmGKB record: 10; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

1 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)
MOLIBRESIB	2	MED12

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	1	MED12
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	9	WT1, BRCA2, ELP4, TRIM28, WT1-AS, CCDC160, GPC3, GPC4, IGF2

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)
WT1	0	—
BRCA2	0	—
ELP4	0	—
TRIM28	19	—
WT1-AS	0	—
CCDC160	0	—
GPC3	5	—
GPC4	0	—
IGF2	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 6.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	2
PHASE1	2
Not specified	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00038207	PHASE2	COMPLETED	Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies
NCT00335556	PHASE2	COMPLETED	Combination Chemotherapy, Radiation Therapy, and/or Surgery in Treating Patients With High-Risk Kidney Tumors
NCT00011414	PHASE1	COMPLETED	Phase I Trial of Tariquidar (XR9576) in Combination With Doxorubicin, Vinorelbine, or Docetaxel in Pediatric Patients With Solid Tumors
NCT02164097	PHASE1	TERMINATED	ODSH + ICE Chemotherapy in Pediatric Solid Tumors
NCT00503893	Not specified	UNKNOWN	Genetics of Wilms’ Tumor and/or the Associated Conditions of Aniridia, Hemihypertrophy, and Genitourinary Anomalies
NCT05179850	Not specified	UNKNOWN	Computer Aided Diagnostic Tool on Computed Tomography Images for Diagnosis of Retroperitoneal Tumor in Children

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
DACTINOMYCIN	4	1
VINCRISTINE	4	1
DOCIPARSTAT SODIUM	3	1
TARIQUIDAR	3	1
CHEMBL4748391	0	1