DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for liver disease — screening already-approved drugs against its 39-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleLiver disease maps to a 39-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 liver 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
glucokinase regulator (GCKR) — GCKR 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 f1pdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4BB9 · 1.47 Å · ligand 1-O-phosphono-beta-D-fructopyranose (F1P). Experimental structure, not a prediction.
What the evidence adds up to
A 2009 review of inherited metabolic liver diseases—hereditary haemochromatosis, Wilson’s disease, and alpha-1-antitrypsin deficiency—reports progress in clarifying defective molecular pathways. Hepcidin, the iron hormone defective in haemochromatosis, is now known to be controlled by iron signals and by circulatory and membrane-associated regulators, and can be measured in serum and urine. In Wilson’s disease and alpha-1-antitrypsin deficiency, an unexpected pathogenic link with early metabolic abnormality in lipid or glycogen metabolism has emerged. The review suggests that interfering with apoptotic pathways may offer new therapeutic tools, but no clinical trial results, survival data, or response rates are provided.
A 1997 review on molecular approaches to inherited liver disease, focusing on Wilson disease, haemochromatosis, and alpha-1-antitrypsin deficiency, states that identification of disease genes can have practical applications for diagnosis, provide information on prognosis, and lead to new therapies. No concrete numbers on survival or response rates are given, and no drug is mentioned.
A 1997 review of cost-effectiveness in liver disease therapy notes that many advances have been made in diagnosis and treatment, but comparatively few interventions have been rigorously subjected to full cost-effectiveness evaluation. The authors state that it is difficult to make many definitive claims, and they hope future studies will consider these aspects.
A 2019 study of bile ductular reactions in cholestatic and parenchymal liver diseases—including primary biliary cholangitis, primary sclerosing cholangitis, alcoholic liver disease, non-alcoholic steatohepatitis, and HCV and HBV infections—used high-throughput RNA sequencing to compare transcriptomic profiles of ductular reaction cells from HCV and primary sclerosing cholangitis livers. The ductular reaction transcriptomes were markedly different from neighbouring hepatocytes, and 330 genes were significantly differently expressed between the two disease aetiologies. The authors suggest that exploring these gene expression profiles could enable therapeutic targeting to inhibit fibrosis and inflammation or promote regeneration, but no drug, clinical outcome, or patient data are reported.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Gastroenterology · 2009 · 24 citations · open access
Inherited metabolic disease of the liver
AbstractPURPOSE OF REVIEW: Progress in the dissection of the molecular pathogenesis of most prevalent inherited liver diseases such as hereditary hemochromatosis, Wilson's disease, and alpha1-antitrypsin deficiency is continuing. This review highlights recent achievements that have clarified defective molecular pathways and shed new lights on the complex interplay of genetic and environmental factors in determining disease phenotype. This advancement paves the way for development of new strategies to diagnose and cure metabolic liver diseases. RECENT FINDINGS: Hepcidin, the iron hormone that is defective in hemochromatosis, is controlled not only by iron signals but also by a number of circulatory and membrane-associated regulators. Serum and urinary hepcidin can be now measured. New studies have provided important information on variable clinical expressivity of the genetic defect in hemochromatosis. The molecular and cellular events that accompany Wilson's disease and alpha-1-antitrypsin deficiency are being elucidated. In both, an unexpected pathogenic link with early metabolic abnormality in lipid or glycogen metabolism has emerged. Interference with apoptotic pathways may offer new therapeutic tools to prevent liver disease progression and acute liver failure associated with inherited metabolic diseases of the liver. SUMMARY: The field of inherited diseases of the liver is rapidly evolving. Understanding molecular pathogenesis of these disorders is improving our ability to diagnose and treat them. The most recent findings are detailed in this review.
Journal of Gastroenterology and Hepatology · 1997 · 7 citations
R<scp>eview</scp>: Molecular approaches to inherited liver disease. Focus on Wilson disease
AbstractAlthough infectious agents account for a large proportion of liver disease, inherited forms of liver disease, often treatable, must not be overlooked. New approaches to the cloning of disease genes are allowing an increased understanding of the basic defect of human diseases. Identification of disease genes can have practical applications for diagnosis, can provide information on prognosis and can lead to new therapies. This review focuses on selected inherited liver diseases and the knowledge to be gained from molecular studies. Three genetic diseases affecting the liver, Wilson disease, haemochromatosis and alpha 1-antitrypsin deficiency, are selected as examples of current approaches to the study of genetic liver disease.
Clinical economics review: cost‐effectiveness in the therapy of liver disease
AbstractThere have been many advances made in the management of patients with liver disease both in diagnosis and in the treatment of underlying liver disease and its complications, although comparatively few of these have been rigorously subjected to full cost-effectiveness evaluation. In this review, we have analysed a small number of the therapeutic interventions; while these have been well evaluated clinically, very few have been analysed from the viewpoint of cost-effectiveness and, thus, it is difficult to make many definitive claims. It is hoped that future studies will consider these aspects as well.
AbstractAim : The present study evaluated the safety and effectiveness of repeated injections of minocycline hydrochloride (MINO) for symptomatic polycystic liver disease (PLD). Methods : We retrospectively studied patients who received percutaneous MINO injections for symptomatic PLD and were followed up for at least 2 years between 2002 and 2015. Huge hepatic cysts (diameter > 10 cm) were treated by continuous drainage and multiple MINO injections. Small hepatic cysts underwent one-step drainage followed by a single injection of MINO. Only several cysts that caused complications were treated by percutaneous injection of MINO. Patients were discharged 1 day after treatment. Results : Ten patients, (4men and 6 women; mean age, 57.8 years) were studied. All patients had Type 2 PLD according to Gigot’s classification. The chief complaints were abdominal distension (n = 10), abdominal pain (n = 6), back pain (n = 4), and appetite loss (n = 8). Four patients had mild liver dysfunction. One patient complained of moderate right subscapular pain immediately after the injection, and another had pain at the site of catheter insertion. Liver dysfunction did not develop in any patient after treatment. The mean follow-up was 63.4 months. The interval from the first to last admission for MINO injection therapy was 14 to 148 months (mean, 40.8 months). The number of admissions for MINO injection therapy ranged from 2 to 12 times (mean, 4.0 times). The average interval for MINO injection therapy was 8.3 to 19 months (mean, 14.8 months).The hepatic cysts shrank in all patients after treatment. Complications improved consistently, and all patients were satisfied with the outcome of treatment. Conclusions : Symptomatic PLD was controllable by 14.8 months of repeated MINO injections on average. Repeated MINO injections are a safe, definitive treatment for symptomatic PLD.
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.
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