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DeCure for Hereditary Wilms tumor

DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for hereditary Wilms tumor — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module3 genesLead labCancer
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CancerDOID:5183$DeCureCancer

The disease map

Disease moduleHereditary Wilms tumor maps to a 3-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Current lead

No approved-drug candidate for hereditary wilms tumor is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.

Molecular view

WT1 transcription factor (WT1) — WT1 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.

Loading structure…
helix sheet apo structuredrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 6BLW · 1.835 Å · ligand none (apo structure). Experimental structure, not a prediction.

What the evidence adds up to

In a 1988 study of 96 long-term survivors of unilateral sporadic Wilms tumour, none of their 179 offspring had developed Wilms tumour. The maximum likelihood estimate of a hereditary fraction in that patient group was zero, with a 95% upper confidence limit between 0.06 and 0.11 depending on penetrance. The 95% upper bound of risk for offspring was 0.02. A 1955 case report describes bilateral Wilms tumour occurring in a family with Wilms tumour, but provides no systematic data on heritable fraction or recurrence risk.

A 2022 bioinformatics analysis of three gene expression datasets (GSE66405, GSE73209, GSE11151) identified 474 common differentially expressed genes in Wilms tumour. The top ten hub genes from a protein-protein interaction network were ALB, CDH1, EGF, AQP2, REN, SLC2A2, SPP1, UMOD, NPHS2, and FOXM1, with ALB having the highest degree. Gene set enrichment analysis linked ALB expression to 11 pathways in one dataset and 10 in another. The study reports no clinical outcomes, no survival data, and no validation in patient samples.

A 2025 commentary on Treger and colleagues’ work notes that the molecular features of Wilms tumours depend on the constitutional milieu of the patient, but provides no new data on hereditary Wilms tumour specifically. No drug, no treatment, and no clinical trial results are reported in any of these abstracts.

What is still missing: prospective studies of offspring in families with confirmed hereditary Wilms tumour, molecular stratification that distinguishes sporadic from truly heritable cases, and funding for long-term follow-up cohorts rather than retrospective bioinformatics or single-case reports.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

PEDIATRICS · 1988 · 32 citations

Heritable Fraction of Unilateral Wilms Tumor

AbstractHeritability of the unilateral-sporadic (non-familial) form of Wilms tumor was examined in the offspring of 96 long-term survivors of the neoplasm. No Wilms tumor has developed in any of the 179 offspring of these patients. The maximum likelihood estimate of a hereditary Wilms tumor in our patients is zero and the corresponding 95% upper confidence limit ranges between 0.06 and 0.11, depending on penetrance. Among their offspring, the 95% upper bound of the risk of Wilms tumor is 0.02. These figures can be applied in genetic counseling of other survivors of unilateral-sporadic Wilms tumor.

https://doi.org/10.1542/peds.81.1.147
The Journal of Urology · 1955 · 24 citations

Bilateral Wilms Tumor in a Wilms Tumor Family: Case Report

AbstractNo AccessJournal of Urology1 Mar 1955Bilateral Wilms Tumor in a Wilms Tumor Family: Case Report W.L. Fitzgerald and H.C. Hardin W.L. FitzgeraldW.L. Fitzgerald More articles by this author and H.C. HardinH.C. Hardin More articles by this author View All Author Informationhttps://doi.org/10.1016/S0022-5347(17)67426-3AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail © 1955 by The American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetailsCited by Lasota J (2010) MOLECULAR GENETICS OF SOFT TISSUE TUMORS Modern Soft Tissue Pathology, 10.1017/CBO9780511781049.006, (127-180) (2014) Hereditary Diseases That Implicate Defective Responses to DNA Damage DNA Repair and Mutagenesis, 10.1128/9781555816704.ch29, (1001-1047) Bonaïti-Pellié C, Chompret A, Tournade M, Hochez J, Moutou C, Zucker J, Steschenko D, Brunat-Mentigny M, Roché H, Tron P, Frappaz D, Munzer M, Bachelot C, Dusol F, Sommelet-Olive D and Lemerle J (1992) Genetics and epidemiology of Wilms' tumor: The French Wilms' tumor studyMedical and Pediatric Oncology, 10.1002/mpo.2950200404, VOL. 20, NO. 4, (284-291), . Gallo G and Chemes H (2011) The Association of Wilms' Tumor, Male Pseudohermaphroditism and Diffuse Glomerular Disease (Drash Syndrome)Pediatric Pathology, 10.1080/15513818709177840, VOL. 7, NO. 2, (175-189), Online publication date: 1-Jan-1987. François J, Spae V and Sutter E (2009) The aniridia-Wilms' tumor syndrome and other associations of aniridiaOphthalmic Paediatrics and Genetics, 10.3109/13816818209031457, VOL. 1, NO. 2, (125-138), Online publication date: 1-Jan-1982. Kantor A, Li F, Fraumeni J, Curnen M and Flannery J (1982) Childhood cancer in offspring of two wilms tumor survivorsMedical and Pediatric Oncology, 10.1002/mpo.2950100114, VOL. 10, NO. 1, (85-89), . Maurer H, Pendergrass T, Borges W and Honig G (1979) The role of genetic factors in the etiology of wilms' tumor.Two pairs of monozygous twins with congenital abnormalities (Aniridia; hemihypertrophy) and discordance for wilms' tumorCancer, 10.1002/1097-0142(197901)43:1<205::AID-CNCR2820430130>3.0.CO;2-7, VOL. 43, NO. 1, (205-208), Online publication date: 1-Jan-1979. Cotlier E, Rose M and Moel S (1978) Aniridia, Cataracts, and wilms' Tumor in Monozygous TwinsAmerican Journal of Ophthalmology, 10.1016/0002-9394(78)90029-6, VOL. 86, NO. 1, (129-132), Online publication date: 1-Jul-1978. Williams D (1974) Neoplastic Disease: The Kidney Urology in Childhood, 10.1007/978-3-642-65687-3_22, (296-308), . Fay R, Brosman S and Williams D (2018) Bilateral NephroblastomaJournal of Urology, VOL. 110, NO. 1, (119-124), Online publication date: 1-Jul-1973. Steinberg S, Brodovsky H and Goepp C (1972) Renal carcinoma in mother and daughterCancer, 10.1002/1097-0142(197201)29:1<222::AID-CNCR2820290133>3.0.CO;2-H, VOL. 29, NO. 1, (222-225), Online publication date: 1-Jan-1972. GUPTA O and DUBE M (1971) RARE PRIMARY RENAL SARCOMABritish Journal of Urology, 10.1111/j.1464-410X.1971.tb10041.x, VOL. 43, NO. 5, (546-551), Online publication date: 1-Oct-1971. Bloom H and Wallace D (1971) Tumours of the urinary tract Spezielle Strahlentherapie Maligner Tumoren / Radiation Therapy of Malignant Tumours, 10.1007/978-3-642-80566-0_3, (399-549), . Tsunoda A, Ishida M and Ohmi K (1969) Bilateral Wilm's Tumor: A Case Report and a Survey of Nineteen Cases in Japanese LiteraturePediatrics International, 10.1111/j.1442-200X.1969.tb02733.x, VOL. 11, NO. 2, (1-7), Online publication date: 1-Dec-1969. Villegas A (2018) Bilateral Primary Malignant Renal Tumors of Dissimilar Histogenesis: Report of 2 Cases and Review of the LiteratureJournal of Urology, VOL. 98, NO. 4, (450-455), Online publication date: 1-Oct-1967.Cochran W and Froggatt P (2018) Bilateral Nephroblastoma in Two SistersJournal of Urology, VOL. 97, NO. 2, (216-220), Online publication date: 1-Feb-1967. Miller R, Fraumeni J and Manning M (1964) Association of Wilms's Tumor with Aniridia, Hemihypertrophy and Other Congenital MalformationsNew England Journal of Medicine, 10.1056/NEJM196404302701802, VOL. 270, NO. 18, (922-927), Online publication date: 30-Apr-1964. STRØM T (1957) A Wilms' Tumour FamilyActa Paediatrica, 10.1111/j.1651-2227.1957.tb14489.x, VOL. 46, NO. 6, (601-604), Online publication date: 1-Nov-1957. Volume 73 Issue 3 March 1955 Page: 468-474 Advertisement Copyright & Permissions© 1955 by The American Urological Association Education and Research, Inc.Metrics Author Information W.L. Fitzgerald More articles by this author H.C. Hardin More articles by this author Expand All Advertisement PDF downloadLoading ...

https://doi.org/10.1016/s0022-5347(17)67426-3
Translational Cancer Research · 2022 · 11 citations · open access

Bioinformatics analysis and identification of genes and pathways involved in patients with Wilms tumor

AbstractBackground: Wilms tumor is the most common childhood kidney malignant tumor. However, the genes and signaling pathways associated with the disease remain incompletely understood. Methods: GSE66405, GSE73209, and GSE11151 were collected from the Gene Expression Omnibus (GEO) database, and differentially expressed genes (DEGs) were detected using R software. A protein-protein interaction (PPI) network was constructed using the STRING database, and the clustering modules and hub genes were analyzed with the Cytoscape software. Genes functional enrichment analyses were performed using the package “clusterProfiler” in R software, and the gene set enrichment analysis (GSEA) analysis was performed using GSEA v4.1.0 software. Results: Respectively, 3,092, 620, and 3,567 DEGs were screened in GSE66405, GSE73209, and GSE11151, with a total of 474 common DEGs detected in three expression profiles. For the common DEGs, the top 30 significant results of Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathways enrichment analyses were presented. Furthermore, five modules were found as the most related modules to Wilms tumor. GO term and KEGG pathway enrichment analyses of the genes in all the modules identified 10 GO terms and 5 KEGG pathways as significantly enriched. The top 10 hub DEGs of the PPI network were ALB, CDH1, EGF, AQP2, REN, SLC2A2, SPP1, UMOD, NPHS2, and FOXM1, with ALB identified as the highest degree. GSEA results showed 11 pathways were correlated with ALB expression in GSE66405 and 10 pathways were related to the expression of the ALB gene in GSE73209. Conclusions: Our study revealed robust gene signatures in Wilms tumor. Dysregulations of the signaling pathways were associated with the development and progression of the Wilms tumor, and 10 hub genes may play important roles in its diagnosis and therapy.

https://doi.org/10.21037/tcr-22-1847
Cancer Discovery · 2025 · 2 citations

Knudson’s “Two-Hit” Hypothesis and Cancer Predisposition: A Bit More Complicated but Still Going Strong

AbstractThis study by Treger and colleagues is a comprehensive evaluation of the genome and epigenome of tumors and constitutional tissue from children with Wilms tumor predisposition syndromes that demonstrates that the molecular features of Wilms tumors are dependent on the constitutional milieu of the patient in which they develop. See related article by Treger et al., p. 286.

https://doi.org/10.1158/2159-8290.cd-24-1662

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.