Rare & Orphan Lab · DeCure for X

DeCure for Familial polycythemia

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for familial polycythemia — 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 labRare & Orphan
All cures
Rare & OrphanDOID:10780$DeCureRare

The disease map

Disease moduleFamilial polycythemia 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 familial polycythemia 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.

Evidence

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

Cleveland Clinic Journal of Medicine · 1989 · 18 citations

Familial polycythemia vera

AbstractThe occurrence of polycythemia vera in a father, mother, and two sons is reported. Thirteen kindreds with familial polycythemia vera in 31 members are reviewed. Comprehensive records were available for all four patients as well as other family members, since all were diagnosed and treated at the author's institution over a period of nearly 50 years. The mean age at diagnosis, sex predominance, symptoms, and incidence of chromosomal abnormalities, leukocytosis, thrombocytosis, and elevated leukocyte alkaline phosphatase levels were similar to those of nonfamilial cases. The mean RBC volume at diagnosis and the incidence of splenomegaly appear to be higher in familial than nonfamilial cases. The mode of inheritance is unclear, but genetic factors may be involved in the pathogenesis of this myeloproliferative disorder.

https://doi.org/10.3949/ccjm.56.8.813
PEDIATRICS · 1958 · 9 citations

BENIGN FAMILIAL POLYCYTHEMIA IN CHILDHOOD

AbstractThis is a review of benign familial polycythemia—a disease marked by high values for erythrocyte count and hemoglobin, not associated with significant symptoms, leukocytosis or thrombocytosis. Two families are described, some of whose members have this condition. It is hoped that greater knowledge of prognosis may be obtained by further observations of these patients.

https://doi.org/10.1542/peds.21.1.54

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.