DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for cystathioninuria — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCystathioninuria maps to a 1-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 cystathioninuria 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
cystathionine gamma-lyase (CTH) — CTH 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 cysdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5EIG · 2.7 Å · ligand CYSTEINE (CYS). 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.
The Tohoku Journal of Experimental Medicine · 1968 · 28 citations · open access
Cystathioninuria not Associated with Vitamin B<sub>6</sub> Dependency: A Probably New Type of Cystathioninuria
AbstractAn infantile case of cystathioninuria was described. The patient had retarded development and marked cystathioninuria. Large doses of vitamin B6, were of no effect in diminishing cystathionine excretion, this finding being different from that in other patients with cystathioninuria so far reported. Using liver specimens biopsied from the patient, a difinitely low activity of cystathionase was demonstrated in the presence of an excess of pyridoxal phosphate. Both in vivo and in vitro studies indicate a defect in cystathionase itself. The present patient is thought to have a new type of cystathioninuria without vitamin B66 dependency.
AbstractSummary Cystathioninuria was detected in 6 of 230 hospitalized children. Three had convulsions when admitted to the hospital, two severe liver malfunction of unknown cause (suspect neonatal hepatitis), and one child had severe congenital malformations and bronchopneumonia and was in a terminal stage. Cystathioninuria disappeared in 4 children (two with convulsions and two with liver malfunction) treated with pyridoxine orally. Pyridoxine treatment (2×40 mg daily) also resulted in disappearence of convulsions. The possible background to these findings is discussed.
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.
