Target landscape
Open Targets · retrieved 2026-09-16 · weekly12 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.
| Gene | Drugs | Approval, and whether it reached this disease | Open Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it works | Cell 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 · weekly1 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.
Every label that names Wilms tumour
| Drug | Role | What the label says |
|---|---|---|
| DactinomycinDACTINOMYCIN, Dactinomycin, dactinomycin | Labelled here | Wilms 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 hydrochloride | Backbone | for 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 Sulfate | Backbone | Vincristine 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 · weeklyWT1 is the busiest cell-therapy antigen in Wilms tumour: 15 registered trials, 1 still active.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT07645469 | 1 | Recruiting | FH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia | 2026-08-13 |
| NCT00003887 | 2 | Completed | Lymphocyte Infusion in Treating Patients With Relapsed Cancer After Bone Marrow or Peripheral Stem Cell Transplantation | 2011-11-30 |
| NCT01513109 | 1/2 | Unknown | Safety and Immunogenicity of Recombinant WT1 Antigen-Specific Cancer Immunotherapeutic Combined With Infusion of Treg Depleted T Cells for Adult WT1 Acute Myeloid Leukemia | 2012-01-20 |
| NCT00769613 | 1 | Unknown | Emergency Use of Donor Lymphocytes in Treating Patients Who Have Undergone Donor Stem Cell Transplant and Have Cytomegalovirus Infections | 2013-12-18 |
| NCT02895412 | 1 | Unknown | Infection and Tumour Antigen Cellular Therapy | 2016-09-09 |
| NCT01621724 | 1/2 | Completed | WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study | 2018-10-02 |
| NCT02550535 | 1/2 | Completed | A Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients | 2018-10-02 |
| NCT00620633 | 1 | Completed | Dose Escalation Trial of WT1-Sensitized T Cells for Residual or Relapsed Leukemia After Allogeneic Hematopoietic Progenitor Cell Transplantation | 2021-03-02 |
| NCT02408016 | 1/2 | Terminated | Genetically Modified T Cells in Treating Patients With Stage III-IV Non-small Cell Lung Cancer or Mesothelioma | 2021-09-13 |
| NCT02770820 | 1/2 | Terminated | Laboratory-Treated (Central Memory/Naive) CD8+ T Cells in Treating Patients With Newly Diagnosed or Relapsed Acute Myeloid Leukemia | 2021-10-29 |
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 · monthlyEvery 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 term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Cohort Studies | 1.54% | 2.85% | 1.86× | 19 papers |
| Genotype | 1.1% | 1.95% | 1.78× | 13 papers |
| Tumor Burden | 1.1% | 1.8% | 1.64× | 12 papers |
| Genes, Wilms Tumor | 1.75% | 2.7% | 1.54× | 18 papers |
| Cell Movement | 2.41% | 3.3% | 1.37× | 22 papers |
| Disease Progression | 2.41% | 3.15% | 1.31× | 21 papers |
| Biomarkers | 1.54% | 1.95% | 1.27× | 13 papers |
| Prognosis | 14.25% | 18.02% | 1.26× | 120 papers |
| MicroRNAs | 5.7% | 7.21% | 1.26× | 48 papers |
| Cell Proliferation | 5.26% | 6.61% | 1.26× | 44 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Treatment Outcome | 13.6% | 4.35% | 0.32× | 29 papers |
| Tomography, X-Ray Computed | 7.68% | 2.85% | 0.37× | 19 papers |
| Combined Modality Therapy | 4.82% | 2.1% | 0.44× | 14 papers |
| Risk Factors | 4.39% | 2.1% | 0.48× | 14 papers |
| Dactinomycin | 3.95% | 2.1% | 0.53× | 14 papers |
| Follow-Up Studies | 10.09% | 5.41% | 0.54× | 36 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Kidney Neoplasms | 70.39% | 86.34% | 1.23× | 575 papers |
| Prognosis | 14.25% | 18.02% | 1.26× | 120 papers |
| Nephrectomy | 21.27% | 13.96% | 0.66× | 93 papers |
| Kidney | 10.96% | 9.16% | 0.84× | 61 papers |
| Gene Expression Regulation, Neoplastic | 7.89% | 8.86% | 1.12× | 59 papers |
| Mutation | 8.99% | 8.11% | 0.9× | 54 papers |
| Neoplasm Staging | 10.96% | 7.96% | 0.73× | 53 papers |
| Neoplasm Recurrence, Local | 7.68% | 7.51% | 0.98× | 50 papers |
| Biomarkers, Tumor | 7.02% | 7.36% | 1.05× | 49 papers |
| Antineoplastic Combined Chemotherapy Protocols | 8.11% | 7.36% | 0.91× | 49 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Randomized Controlled Trial | 1.1% | 0.2% |
| Review | 12.1% | 10.8% |
| Meta-Analysis | 1.1% | 1.1% |
| Case Reports | 0.0% | 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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | 600 cases/year | direct | American Cancer Society, Key Statistics for Wilms Tumors, retrieved 2026-09-16 |
| New cases each year | 80,450 cases/year | proxy | counts kidney and renal pelvis cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Kidney and Renal Pelvis Cancer, retrieved 2026-09-16 |
| Deaths each year | 15,160 deaths/year | proxy | counts kidney and renal pelvis cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Kidney and Renal Pelvis Cancer, retrieved 2026-09-16 |
| Incidence rate | 18 cases per 100,000 per year | proxy | counts kidney and renal pelvis cancer, which is broader than this disease; 2019-2023 SEER Cancer Stat Facts, Kidney and Renal Pelvis Cancer, retrieved 2026-09-16 |
| Death rate | 3.4 deaths per 100,000 per year | proxy | counts kidney and renal pelvis cancer, which is broader than this disease; 2020-2024 SEER Cancer Stat Facts, Kidney and Renal Pelvis Cancer, retrieved 2026-09-16 |
| People living with it | 687,999 people living with the disease | proxy | counts kidney and renal pelvis cancer, which is broader than this disease; 2023 SEER Cancer Stat Facts, Kidney and Renal Pelvis Cancer, retrieved 2026-09-16 |
| Deaths each year | — | not published | Not published on the page. |
| Five-year relative survival | — | not published | The American Cancer Society publishes survival by stage and histology (favourable, anaplastic), not one relative figure. |
| Median age at diagnosis | — | not published | Published 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 · quarterlyNIH 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.
NIH obligations by fiscal year
| Year | Obligations | Awards | Distinct projects | Dropped as off-topic |
|---|---|---|---|---|
| FY2013 | $2.0M | 3 | 3 | 34 |
| FY2014 | $1.9M | 3 | 3 | 28 |
| FY2015 | $1.5M | 3 | 3 | 29 |
| FY2016 | $2.0M | 4 | 4 | 33 |
| FY2017 | $2.2M | 6 | 6 | 24 |
| FY2018 | $2.0M | 4 | 4 | 38 |
| FY2019 | $1.2M | 5 | 5 | 26 |
| FY2020 | $1.1M | 4 | 4 | 18 |
| FY2021 | $1.1M | 5 | 5 | 10 |
| FY2022 | $0.4M | 3 | 3 | 15 |
| FY2023 | $0.3M | 2 | 2 | 16 |
| FY2024 | $0.4M | 2 | 2 | 20 |
| FY2025 | $1.1M | 4 | 4 | 19 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| University Of Pennsylvania | $0.5M | 1 |
| University Of Wisconsin-Madison | $0.4M | 2 |
| St. Jude Children'S Research Hospital | $0.2M | 1 |
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
Projects by administering institute. The rows above are the top 1 and account for 4 of 4.
Award mechanisms
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
Share of $1.1M in FY2025. The top three hold 100%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly173 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
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE10320 | Predicting Relapse in Favorable Histology Wilms Tumor Using Gene Expression2009 · array | 144 | 11 | 0.9 | patient cohortAPT 0.75stage44 cites |
| GSE66405 | mRNA expression profiles in Wilms tumor subtypes2016 · array | 32 | 28 | 1.7 | patient cohortAPT 0.549 cites |
| GSE57370 | miRNA expression profiles in Wilms tumor subtypes2015 · array | 66 | 13 | 7.1 | APT 0.95249 cites |
| GSE31403 | Clinically Relevant Subsets Identified by Gene Expression Patterns Support a Revised Ontogenic Model of Wilms Tumor: A Children’s Oncology Group Study2012 · array | 224 | 13 | 2.1 | APT 0.7585 cites |
| GSE29505 | Custom Illumina array DNA methylation analysis of 384 CpGs across five cancer types2011 · methylation | 290 | 1 | 17.5 | APT 0.95824 cites |
| GSE56955 | Somatic mutations in DROSHA and DICER1 impair microRNA biogenesis in Wilms tumors2015 · sequencing | 12 | 1 | 5.3 | patient cohortAPT 0.75molecularDICER1DROSHA199 cites |
| GSE59157 | Methylome analysis of normal kidney, nephrogenic rest and Wilms tumor2014 · methylation | 95 | 11 | 0.9 | patient cohortAPT 0.532 cites |
| GSE50505 | microRNA Profiling in Wilms Tumors and Kidney Tissues2013 · array | 28 | 4 | 3.6 | APT 0.5molecularIGF2134 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE292896 | Somatic development of Wilms tumour via normal kidneys in predisposed children2025 · methylation | 455 | 0 | 3.5 | patient cohortAPT 0.7512 cites |
| GSE226480 | The Genetic and Epigenetic Features of Bilateral Wilms Tumor Predisposition2023 · methylation | 210 | 1 | 2.5 | APT 0.7529 cites |
| GSE175698 | Single cell transcriptomics of fetal kidney and Wilms tumor nephrogenic progenitors2023 · sequencing | 5 | 1 | 1.6 | xenograftAPT 0.5stagemolecularSIX221 cites |
| GSE269241 | DNA methylation biomarkers predict Wilms tumor disease progression2024 · methylation | 24 | 1 | 0.2 | patient cohortAPT 0.05survivalstagemolecularIGF21 cites |
| GSE200256 | Single nuclear RNASeq Analysis of Anaplastic and Favourable Histology Hu_Wilms Tumor2022 · sequencing | 40 | 5 | — | patient cohortsurvival |
| GSE246479 | Modeling high-risk blastemal and metastatic Wilms tumors by reprogramming WiT49 cells2024 · sequencing | 6 | 1 | 0.8 | 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 refreshEach 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
StaticThis 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."
}