Disease Briefing

Wilms tumour: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-16Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 67 studies · 3,443 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in Wilms tumour — WT1, CTNNB1, AMER1, IGF2, TRIM28, SIX1, SIX2, DROSHA, DGCR8, DICER1, TP53, MYCN — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

1
drugs carry an FDA label naming Wilms tumour: Dactinomycin. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
0
of the 12 genes above carries a drug approved in Wilms tumour — 14 drug entries reach them, 13 distinct once salt forms are merged, and 0 of those are approved for other indications. A statement about these gene targets, not about the disease
15
registered WT1 cell-therapy trials in Wilms tumour, 1 active. Counted from ClinicalTrials.gov across 4 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
11
targets carry an Open Targets tractability signal and have no clinical programme of any kind: CTNNB1, AMER1, IGF2, TRIM28, SIX1, SIX2, DROSHA, DGCR8, DICER1, TP53, MYCN. WT1 has cell-therapy trials, so it is undrugged rather than untouched
173
human GEO series match the disease; 67 survive on-topic filtering, and only 5 are patient cohorts of 100+ samples
28
Europe PMC full-text papers name GSE66405 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$1.1M
NIH obligations in FY2025, up -44% since 2013 — while distinct core projects went 3 to 4. More distinct projects (+33%) rather than larger ones; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-16 · weekly

12 genes recurrently implicated in Wilms tumour, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 4 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Wilms tumour does have labelled therapy — 1 drugs, listed in the next section.

Two different questions

The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what Wilms tumour is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell therapy here
WT1 2 Phase 3Galinpepimut-S, Ombipepimut-S1 active of 7 Wilms tumour trials other clinical modality, protein degrader 1 active of 15 trials
CTNNB1 across cancers → 1 Phase 2Pri-724No Wilms tumour trial of any of these drugs antibody, other clinical modality, protein degrader, small molecule
AMER1 0 No drug antibody, protein degrader
IGF2 2 Phase 2Dusigitumab, XentuzumabNo Wilms tumour trial of any of these drugs antibody
TRIM28 0 No drug protein degrader, small molecule
SIX1 0 No drug protein degrader
SIX2 0 No drug protein degrader
DROSHA 0 No drug protein degrader
DGCR8 0 No drug protein degrader
DICER1 0 No drug protein degrader
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo Wilms tumour trial of any of these drugs other clinical modality, protein degrader, small molecule
MYCN across cancers → 0 No drug protein degrader, small molecule

Dataset evidence counts studies in the 67-study ranked set whose title or abstract names the gene; the bar is scaled to WT1. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-16. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

What is actually approved here

openFDA drug labels · weekly

1 drug carries an FDA label naming Wilms tumour: Dactinomycin. Separately, 0 of the drugs returned for the genes in the table above are approved only for other diseases and reach Wilms tumour through trials, not through their labels.

1Labelled for Wilms tumourFDA INDICATIONS AND USAGE names the disease
0Approved, but for another diseasereturned for the genes in the table above
2Backbone agents listing itbroad cytotoxics whose labels name many tumours
1Active WT1 cell-therapy trialsof 15 registered

Every label that names Wilms tumour

DrugRoleWhat the label says
DactinomycinDACTINOMYCIN, Dactinomycin, dactinomycinLabelled hereWilms Tumor Dactinomycin for Injection is indicated for the treatment of adult and pediatric patients with Wilms tumor, as part of a multi-phase, combination chemotherapy regimen.
DoxorubicinDoxorubicin Hydrochloride, doxorubicin hydrochlorideBackbonefor the treatment of: acute lymphoblastic leukemia, acute myeloblastic leukemia, Hodgkin lymphoma, Non-Hodgkin lymphoma, metastatic breast cancer, metastatic Wilms' tumor, metastatic neuroblastoma, metastatic soft tissue sarcoma, metastatic bone sarcomas, metastatic ovarian carcinoma, metastatic transitional cell bladder carcinoma, metastatic thyroid carcinoma, metastatic gastric carcinoma, met...
VincristineVinCRIStine SulfateBackboneVincristine Sulfate Injection has also been shown to be useful in combination with other oncolytic agents in Hodgkin's disease, non–Hodgkin's malignant lymphomas, rhabdomyosarcoma, neuroblastoma, and Wilms' tumor.

0 labels match indications_and_usage:"Wilms tumor" OR indications_and_usage:"Wilms' tumor" OR indications_and_usage:"nephroblastoma"; they collapse to 3 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-16. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against WT1

ClinicalTrials.gov · retrieved 2026-09-16 · weekly

WT1 is the busiest cell-therapy antigen in Wilms tumour: 15 registered trials, 1 still active.

TrialPhaseStatusTitleLast update
NCT076454691RecruitingFH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia2026-08-13
NCT000038872CompletedLymphocyte Infusion in Treating Patients With Relapsed Cancer After Bone Marrow or Peripheral Stem Cell Transplantation2011-11-30
NCT015131091/2UnknownSafety and Immunogenicity of Recombinant WT1 Antigen-Specific Cancer Immunotherapeutic Combined With Infusion of Treg Depleted T Cells for Adult WT1 Acute Myeloid Leukemia2012-01-20
NCT007696131UnknownEmergency Use of Donor Lymphocytes in Treating Patients Who Have Undergone Donor Stem Cell Transplant and Have Cytomegalovirus Infections2013-12-18
NCT028954121UnknownInfection and Tumour Antigen Cellular Therapy2016-09-09
NCT016217241/2CompletedWT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study2018-10-02
NCT025505351/2CompletedA Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients2018-10-02
NCT006206331CompletedDose Escalation Trial of WT1-Sensitized T Cells for Residual or Relapsed Leukemia After Allogeneic Hematopoietic Progenitor Cell Transplantation2021-03-02
NCT024080161/2TerminatedGenetically Modified T Cells in Treating Patients With Stage III-IV Non-small Cell Lung Cancer or Mesothelioma2021-09-13
NCT027708201/2TerminatedLaboratory-Treated (Central Memory/Naive) CD8+ T Cells in Treating Patients With Newly Diagnosed or Relapsed Acute Myeloid Leukemia2021-10-29

10 of 15 shown, most recently active first. Other antigens searched: B7-H3 (2), PD-1 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-16. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the Wilms tumour literature, not a count, because the field itself grew: 2015–2018 (n=456) against 2021–2025 (n=666). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Cohort Studies1.54%2.85%1.86×19 papers
Genotype1.1%1.95%1.78×13 papers
Tumor Burden1.1%1.8%1.64×12 papers
Genes, Wilms Tumor1.75%2.7%1.54×18 papers
Cell Movement2.41%3.3%1.37×22 papers
Disease Progression2.41%3.15%1.31×21 papers
Biomarkers1.54%1.95%1.27×13 papers
Prognosis14.25%18.02%1.26×120 papers
MicroRNAs5.7%7.21%1.26×48 papers
Cell Proliferation5.26%6.61%1.26×44 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Treatment Outcome13.6%4.35%0.32×29 papers
Tomography, X-Ray Computed7.68%2.85%0.37×19 papers
Combined Modality Therapy4.82%2.1%0.44×14 papers
Risk Factors4.39%2.1%0.48×14 papers
Dactinomycin3.95%2.1%0.53×14 papers
Follow-Up Studies10.09%5.41%0.54×36 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Kidney Neoplasms70.39%86.34%1.23×575 papers
Prognosis14.25%18.02%1.26×120 papers
Nephrectomy21.27%13.96%0.66×93 papers
Kidney10.96%9.16%0.84×61 papers
Gene Expression Regulation, Neoplastic7.89%8.86%1.12×59 papers
Mutation8.99%8.11%0.9×54 papers
Neoplasm Staging10.96%7.96%0.73×53 papers
Neoplasm Recurrence, Local7.68%7.51%0.98×50 papers
Biomarkers, Tumor7.02%7.36%1.05×49 papers
Antineoplastic Combined Chemotherapy Protocols8.11%7.36%0.91×49 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial1.1%0.2%
Review12.1%10.8%
Meta-Analysis1.1%1.1%
Case Reports0.0%3.8%

Query: Wilms Tumor[MeSH Major Topic] NOT ("Carcinoma, Renal Cell"[MeSH] OR "Neuroblastoma"[MeSH] OR "Leukemia, Myeloid, Acute"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-16.

Disease burden

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

MeasureValueMatchWhat it counts
New cases each year600 cases/yeardirect
New cases each year80,450 cases/yearproxycounts kidney and renal pelvis cancer, which is broader than this disease; 2026
Deaths each year15,160 deaths/yearproxycounts kidney and renal pelvis cancer, which is broader than this disease; 2026
Incidence rate18 cases per 100,000 per yearproxycounts kidney and renal pelvis cancer, which is broader than this disease; 2019-2023
Death rate3.4 deaths per 100,000 per yearproxycounts kidney and renal pelvis cancer, which is broader than this disease; 2020-2024
People living with it687,999 people living with the diseaseproxycounts kidney and renal pelvis cancer, which is broader than this disease; 2023
Deaths each yearnot publishedNot published on the page.
Five-year relative survivalnot publishedThe American Cancer Society publishes survival by stage and histology (favourable, anaplastic), not one relative figure.
Median age at diagnosisnot publishedPublished as an average, not a median: "The average age of children when they are diagnosed is about 3 to 4 years."

Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming Wilms tumour, after removing the 310 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

$1.1MNIH obligations, FY2025from $2.0M in FY2013 · -44%
4distinct projects funded3 in FY2013
$2.2Mpeak year was FY2017obligations, all institutes
100%of FY2025 awards from NCI4 of 4

NIH obligations by fiscal year

$1M$1M$2M$2M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$2.0M3334
FY2014$1.9M3328
FY2015$1.5M3329
FY2016$2.0M4433
FY2017$2.2M6624
FY2018$2.0M4438
FY2019$1.2M5526
FY2020$1.1M4418
FY2021$1.1M5510
FY2022$0.4M3315
FY2023$0.3M2216
FY2024$0.4M2220
FY2025$1.1M4419

Where FY2025 money went

InstitutionObligationsAwards
University Of Pennsylvania$0.5M1
University Of Wisconsin-Madison$0.4M2
St. Jude Children'S Research Hospital$0.2M1

Text search Wilms tumor OR nephroblastoma over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-16.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

NIH institutes

NCI4 · 100%

Projects by administering institute. The rows above are the top 1 and account for 4 of 4.

Award mechanisms

R212 · 50%
K081 · 25%
R011 · 25%

R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 3 and account for 4 of 4.

Where it lands

University Of Pennsylvania$0.5M · 47.7%
University Of Wisconsin-Madison$0.4M · 35.1%
St. Jude Children'S Research Hospital$0.2M · 17.2%

Share of $1.1M in FY2025. The top three hold 100%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

173 human GEO series match Wilms tumour. Keyword relevance cannot tell a 455-sample patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Composition of the 67-study ranked set

unspecified 27patient 22cell line 8mixed 6xenograft 4
27 unspecified22 patient8 cell line6 mixed4 xenograft28 carry clinical annotation10 carry survival5 patient cohorts ≥100 GEO samples3,443 GEO samples totalin 3 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE10320Predicting Relapse in Favorable Histology Wilms Tumor Using Gene Expression2009 · array144110.9
patient cohortAPT 0.75stage44 cites
GSE66405mRNA expression profiles in Wilms tumor subtypes2016 · array32281.7
patient cohortAPT 0.549 cites
GSE57370miRNA expression profiles in Wilms tumor subtypes2015 · array66137.1
APT 0.95249 cites
GSE31403Clinically Relevant Subsets Identified by Gene Expression Patterns Support a Revised Ontogenic Model of Wilms Tumor: A Children’s Oncology Group Study2012 · array224132.1
APT 0.7585 cites
GSE29505Custom Illumina array DNA methylation analysis of 384 CpGs across five cancer types2011 · methylation290117.5
APT 0.95824 cites
GSE56955Somatic mutations in DROSHA and DICER1 impair microRNA biogenesis in Wilms tumors2015 · sequencing1215.3
patient cohortAPT 0.75molecularDICER1DROSHA199 cites
GSE59157Methylome analysis of normal kidney, nephrogenic rest and Wilms tumor2014 · methylation95110.9
patient cohortAPT 0.532 cites
GSE50505microRNA Profiling in Wilms Tumors and Kidney Tissues2013 · array2843.6
APT 0.5molecularIGF2134 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE292896Somatic development of Wilms tumour via normal kidneys in predisposed children2025 · methylation45503.5
patient cohortAPT 0.7512 cites
GSE226480The Genetic and Epigenetic Features of Bilateral Wilms Tumor Predisposition2023 · methylation21012.5
APT 0.7529 cites
GSE175698Single cell transcriptomics of fetal kidney and Wilms tumor nephrogenic progenitors2023 · sequencing511.6
xenograftAPT 0.5stagemolecularSIX221 cites
GSE269241DNA methylation biomarkers predict Wilms tumor disease progression2024 · methylation2410.2
patient cohortAPT 0.05survivalstagemolecularIGF21 cites
GSE200256Single nuclear RNASeq Analysis of Anaplastic and Favourable Histology Hu_Wilms Tumor2022 · sequencing405
patient cohortsurvival
GSE246479Modeling high-risk blastemal and metastatic Wilms tumors by reprogramming WiT49 cells2024 · sequencing610.8
mixedAPT 0.05survivalstage6 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-16. SubSeries are collapsed to one row per study by linked PMID. Of 173 series retrieved, 38 were dropped by the profile’s exclusion rules and 45 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Where the gaps are

Derived from the sections above · recomputed on every refresh

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

WT1

15 cell-therapy trials against WT1, 1 active, and no approved product. The clinical activity is real and none of it has reached a label.

AMER1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

TRIM28

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

SIX1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

SIX2

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

DROSHA

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

DGCR8

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

DICER1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

MYCN

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

How a machine reads this page

Static

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

Fully server-rendered

Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.

Every number carries a source

Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-16), ClinicalTrials.gov (2026-09-16), openFDA (2026-09-16), NCBI GEO (2026-09-16), PubMed (2026-09-16), NIH RePORTER (2026-09-16).

Negatives stated explicitly

Where nothing exists for Wilms tumour — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated negative is the finding.

Denominators, not just percentages

Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.

The filters are published

5 exclusion patterns are applied to free text before anything is ranked, because none: WiT49 is the only line in regular use is used as a model system in Wilms tumour. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Wilms tumour",
  "mesh": "Wilms Tumor",
  "facts": "https://usebiotransfer.org/disease/wilms-tumor.json",
  "methods": "https://usebiotransfer.org/methods/",
  "all_diseases": "https://usebiotransfer.org/disease/api.json",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}