DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Beckwith-Wiedemann syndrome — screening already-approved drugs against its 8-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleBeckwith-Wiedemann syndrome maps to a 8-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 beckwith-wiedemann syndrome 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
nuclear receptor binding SET domain protein 1 (NSD1) — NSD1 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 samdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3OOI · 1.75 Å · ligand S-ADENOSYLMETHIONINE (SAM). Experimental structure, not a prediction.
What the evidence adds up to
Beckwith-Wiedemann syndrome is a congenital overgrowth disorder first described in 1963 and 1964. The four most characteristic clinical abnormalities are omphalocele or umbilical hernia, macroglossia, gigantism, and renal enlargement. Additional features include macrosomia, body asymmetry, and overgrowth in the first few years of life. Early recognition is considered imperative because of a high incidence of neonatal and infant death caused by hypoglycemia, and because of a suggested increased risk of developing malignant abdominal tumours during childhood.
The syndrome carries a predisposition to tumour development, with the increased risk for neoplasia concentrated in the first eight years of life. However, at least one case report describes a late onset of adrenocortical cancer associated with Beckwith-Wiedemann syndrome, indicating that tumour risk may not be entirely confined to early childhood. No specific drug therapy for the syndrome itself is mentioned in any of the abstracts.
What remains missing is any controlled trial data on pharmacological interventions, any evidence for screening protocols that reduce mortality, and any stratification of tumour risk by the specific genetic subtype within the syndrome. No drug has been tested in a prospective study for this condition.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
American Journal of Roentgenology · 1972 · 6 citations
AbstractThe 4 most characteristic clinical abnormalities of the Beckwith-Wiedemann syndrome are omphalocele or umbilical hernia, macroglossia, gigantism, and renal enlargement. Early recognition of this entity is imperative because of the high incidence of neonatal and infant death caused by hypoglycemia, and because of the suggested increased risk of the development of malignant abdominal tumor during childhood.
Endocrine Surgery · 2014 · 1 citations · open access
A Late Onset of Adrenocortical Cancer Assosiated with Beckwith-Wiedemann Syndrome
AbstractBeckwith-Wiedemann syndrome (BWS) is a genetic overgrowth disorder involving a predisposition to tumor development. The common features of Beckwith-Wiedemann syndrome include omphalocele, macroglos- sia and macrosomia. The increased risk for neoplasia is concentrated in the first eight years of life. However, this case presents a late onset of adrenocortical cancer assosiated with Beckwith-Wiedemann syndrome.
Meditsinskiy sovet = Medical Council · 2017 · 0 citations · open access
OUTPATIENT MANAGEMENT OF A PATIENT WITH BECKWITHWIEDEMANN SYNDROME (CASE REPORT)
AbstractBeckwith - Wiedemann syndrome (ICD-10 code: Q 87.3) is a congenital disease characterized by macrosomia, macroglossia, hernia umbilical cord, overgrowth in the first few years of life, asymmetry of the body and a predisposition to embryonal tumor development. The disease was first described by American pathologist J.V. Beckwith in 1963 and, independently of him, by the German pediatrician H.R. Wiedemann in 1964. Thereafter, it was named the Beckwith-Wiedemann syndrome to honor of these scientists [1--4].
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.
