DeCure's autonomous Cardio AI scientist is researching a drug-repurposing hypothesis for familial hypertrophic cardiomyopathy — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleFamilial hypertrophic cardiomyopathy maps to a 34-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 hypertrophic cardiomyopathy 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
LIM domain binding 3 (LDB3) — LDB3 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 gludrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4YDP · 1.4 Å · ligand GLUTAMIC ACID (GLU). Experimental structure, not a prediction.
What the evidence adds up to
The 2020 AHA/ACC guideline for hypertrophic cardiomyopathy updates earlier recommendations with new evidence, covering diagnostic work-up, genetic and family screening, risk stratification, lifestyle modifications, surgical and catheter interventions, and medications that constitute guideline-directed medical therapy. The guideline advises clinicians to follow dosing, contraindications and drug-drug interactions based on product insert materials for all recommended drug treatment regimens. No specific drug is named in the abstract, and no survival or response rate data are reported.
A 2005 review of paediatric hypertrophic cardiomyopathy notes that the underlying genetic basis is being characterised, but genetic testing remains for research purposes only. Medical therapy is reserved for symptomatic children. For those with important left ventricular outflow obstruction, surgical myectomy may be indicated, with little current role for alcohol septal ablation. Sudden death can occur in children, though risk factors likely differ from adults. The review emphasises that there are important differences from adults in diagnosis and management, and that care must be taken given the potential lifelong implications of therapy.
A 1990 review describes hypertrophic cardiomyopathy as a rare primary myocardial disease known for dramatic morphologic and clinical manifestations, with sudden cardiac death and functional symptoms common. It states that differing pathologic mechanisms may be responsible for similar clinical symptoms and make a unified approach to therapy impossible. No drug, survival figure, or response rate is given.
What is still missing is any randomised controlled trial testing a repurposed drug specifically for familial hypertrophic cardiomyopathy, a clear molecular stratification of patients by causal mutation, and funding for such a trial. The existing literature consists of clinical guidelines and narrative reviews that do not provide the kind of quantitative evidence needed to support a drug-repurposing hypothesis.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Circulation · 2020 · 316 citations · open access
2020 AHA/ACC Guideline for the Diagnosis and Treatment of Patients With Hypertrophic Cardiomyopathy: Executive Summary
AbstractAim This executive summary of the hypertrophic cardiomyopathy clinical practice guideline provides recommendations and algorithms for clinicians to diagnose and manage hypertrophic cardiomyopathy in adult and pediatric patients as well as supporting documentation to encourage their use. Methods A comprehensive literature search was conducted from January 1, 2010, to April 30, 2020, encompassing studies, reviews, and other evidence conducted on human subjects that were published in English from PubMed, EMBASE, the Cochrane Collaboration, Agency for Healthcare Research and Quality reports, and other relevant databases. Structure Many recommendations from the earlier hypertrophic cardiomyopathy guidelines have been updated with new evidence or a better understanding of earlier evidence. This summary operationalizes the recommendations from the full guideline and presents a combination of diagnostic work-up, genetic and family screening, risk stratification approaches, lifestyle modifications, surgical and catheter interventions, and medications that constitute components of guideline directed medical therapy. For both guideline-directed medical therapy and other recommended drug treatment regimens, the reader is advised to follow dosing, contraindications and drug-drug interactions based on product insert materials.
Current Opinion in Cardiology · 2005 · 27 citations
Management of pediatric hypertrophic cardiomyopathy
AbstractPURPOSE OF REVIEW: As the underlying genetic basis of hypertrophic cardiomyopathy is being characterized, there has been increasing recognition of the wide spectrum and variable evolution of this disease within the pediatric age range. This review outlines recent evidence relevant to the diagnosis, management, and prognosis of hypertrophic cardiomyopathy specific to children and adolescents. RECENT FINDINGS: Studies of putative causal genes are leading to the discovery of factors affecting the variability of phenotypic expression and possible avenues for new therapies. Nonetheless, the use of genetic testing currently remains for research purposes only. Echocardiography is the primary means for evaluation, with an increasing focus on diastolic performance. Useful prognostic information can be obtained from the safe performance of cardiopulmonary stress testing. Sudden death can occur in children, although the risk factors are likely different than in adults. The role and mechanisms for possible ischemia remain controversial, and likely differ between individuals. Activity restrictions are recommended, with medical therapy reserved for those who are symptomatic. For those with important left ventricular outflow obstruction, surgical myectomy may be indicated, with little current role for alcohol septal ablation. Advances in implantable defibrillators now make this therapy feasible in younger children. SUMMARY: There are important differences from adults in the approach to the diagnosis and management of hypertrophic cardiomyopathy in children and adolescents. Care regarding prognostication and therapy must be taken given the potential life-long implications.
Clinical Cardiology · 1990 · 7 citations · open access
Current perspectives in hypertrophic cardiomyopathy: Diagnosis, clinical management, and prevention of disability and sudden cardiac death
AbstractHypertrophic cardiomyopathy is a rare primary myocardial disease known for its dramatic morphologic and clinical manifestations. Sudden cardiac death and functional cardiac symptoms are common. However, differing pathologic mechanisms may be responsible for similar clinical symptoms and make a unified approach to therapy impossible. This review will discuss the genetics, criteria for diagnosis, relationship among pathophysiologic abnormalities and clinical symptoms, and management of hypertrophic cardiomyopathy.
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.
