DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for von Hippel-Lindau disease — screening already-approved drugs against its 13-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleVon Hippel-Lindau disease maps to a 13-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 von hippel-lindau disease 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
endothelial PAS domain protein 1 (EPAS1) — EPAS1 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 furan-2-ylmethyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3H82 · 1.5 Å · ligand N-(furan-2-ylmethyl)-2-nitro-4-(trifluoromethyl)aniline (020). Experimental structure, not a prediction.
What the evidence adds up to
Von Hippel-Lindau disease is an autosomal dominant disorder with high penetrance. Population frequency is reported between 1 in 33,000 and 1 in 36,000, or 1 in 39,000 live births. Up to 20% of patients have no family history, meaning the mutation can arise spontaneously. The disease is characterised by multiple benign and malignant tumours: hemangioblastomas of the retina and central nervous system, pancreatic cysts and tumours, renal cell carcinomas, pheochromocytomas, and epididymal cystadenomas. The diversity of forms and the rarity of the condition complicate timely diagnosis.
Untimely monitoring of a patient with von Hippel-Lindau disease increases the probability of complications during surgical treatment and can lead to early disability. The 2020 case report emphasises the need for a multidisciplinary approach to management. The 2002 and 2011 papers add no new treatment data, only descriptions of radiological findings and the importance of understanding pathogenesis for screening decisions.
No drug treatment is mentioned in any of these abstracts. There is no evidence of any pharmacological intervention being tested or recommended. The abstracts focus entirely on diagnosis, genetic inheritance, tumour types, and the need for surveillance.
What is still missing is any clinical trial of a drug for von Hippel-Lindau disease, any evidence of repurposing, any patient stratification beyond family history, and any funding for such work. The abstracts provide no survival data, no response rates, and no sample sizes from treatment studies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of the Korean Radiological Society · 2002 · 3 citations
Von Hippel-Lindau disease: A Case Report
AbstractVon Hippel-Lindau disease is an autosomal dominant disorder characterized by the presence of multiple benign and malignant tumors including hemangioblastomas of the retina and central nervous system, pancreatic cysts and tumors, renal cell carcinomas, pheochromocytomas and epididymal cystadenomas. We report the radiologic findings of a case of von Hippel-Lindau disease, describing the family history.
Obesity and metabolism · 2011 · 2 citations · open access
Sindrom Von Hippel–Lindau
AbstractVon Hippel-Lindau syndrome - a systemic disease manifesting with multiple tumor growth, inherited by autosomal-dominant type with high penetrance. Understanding the pathogenesis of the disease is important for determining the start time of screening for the presence of the tumors and adequate treatment, including metabolic disorders.
Physical and rehabilitation medicine medical rehabilitation · 2020 · 0 citations · open access
The case of detection and differential diagnosis of Hypell-Lindau disease in a patient as a result of a comprehensive examination of a patient in a chronic critical condition
AbstractAccording to evidence in the literature occurrence of the Hippel Lindau disease changes from 1: 33000 - 1: 36000 (population frequency) to 1 in 39000 live births. Up to 20% of them have not family history. Hereditary predisposition of most patients with systemic angioreticulomatosis has patterns and attributes. Diversity of forms and infrequent cases of the Hippel Lindau disease complicate timely diagnosis. Untimely control of condition of patient with the Hippel Lindau disease increases the probability of complications in surgical treatment. It can be the reason of early disability too. The presented clinical case reflects the need for a multidisciplinary approach to the management of patients with von Hippel-Lindau syndrome.
Oxford University Press eBooks · 2017 · 0 citations
Familial CNS Tumor Syndromes: Von Hippel-Lindau Disease
AbstractVon Hippel-Lindau disease can be inherited or may be caused by a spontaneous mutation. Individuals diagnosed with this disease are prone to developing multiple benign tumors termed hemangioblastomas. This chapter addresses the epidemiology, clinical picture, and treatment of these tumors; specifically within those patients with this genetic disorder.
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.