DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for glycogen storage disease II — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleGlycogen storage disease II maps to a 4-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 glycogen storage disease ii 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
phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA) — PIK3CA 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 2sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4JPS · 2.2 Å · ligand (2S)-N~1~-{4-methyl-5-[2-(1,1,1-trifluoro-2-methylpropan-2-yl)pyridin-4-yl]-1,3-thiazol-2-yl}pyrrolidine-1,2-dicarboxamide (1LT). Experimental structure, not a prediction.
What the evidence adds up to
A 2025 review of all glycogen storage diseases states that each type is caused by deficiency of a specific enzyme and that clinical symptoms vary by affected enzyme and organ system. The review lists dietary management, enzyme replacement therapy, and emerging genetic therapies as current treatment strategies, and notes that early diagnosis and personalised treatment plans are essential for improving prognosis. No concrete survival or response rates are given for any drug in this review.
A 2017 review focused on the subset of glycogen storage disorders that involve polyglucosan body accumulation, which includes Adult Polyglucosan Body Disease, Andersen Disease, Tarui Disease, and Lafora Disease. It argues that a pharmacological approach should be the leading therapeutic strategy for these disorders, and describes structure-based drug development, multi-targeting, and image-based high-throughput screening as methodological platforms. No tested drug, clinical trial result, or patient outcome data are reported in this review.
A 1963 announcement from the National Institutes of Health requested physician referrals of patients with any form of glycogen storage disease for a study of metabolic errors and methods for prevention and treatment. It specified that diagnosis should be confirmed by enzymatic assays of liver and muscle tissue obtained by surgical biopsy. No drug, treatment result, or patient outcome is described.
Across these three sources, no drug is tested in patients, no response or survival rates are reported, and no compound is named as effective. What is missing are clinical trial data for any pharmacological agent in any glycogen storage disease, funding for such trials, and patient stratification by enzyme deficiency type.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Expert Opinion on Orphan Drugs · 2017 · 1 citations
Pharmacological approaches for treating glycogen storage disorders involving polyglucosan body accumulation
AbstractIntroduction: Glycogen storage disorders (GSDs) are mainly caused by over-accumulation of normal or malconstructed glycogen. A few GSDs, Adult Polyglucosan Body Disease (APBD), Andersen Disease, Tarui Disease and Lafora Disease are also associated with pathogenic inclusion bodies called polyglucosan bodies (PB) consisting of malconstructed glycogen (polyglucosan) in complex with several enzymes of glycogen metabolism. A treatment for GSDs is urgently required. This review examines the pharmacological avenue for curing PB-involving GSDs.Areas covered: I describe here the pros and cons of the structure-based drug development approach. This functional module-based approach can generate efficacious drugs, but with a large pleiotropic potential. Solutions based on modulations of affinity and specificity of putative drugs and on multi-targeting are then described. Next I discuss the targets of GSD pharmacological therapy: glycogen synthase (GYS), glycogen metabolizing enzymes and inclusion bodies. Finally, image-based high-throughput screening (HTS) is described as a methodological platform for PB-involving GSD drug discovery.Expert opinion: The conclusion of this review is that the pharmacological approach should be the leading therapeutic strategy for curing PB-involving GSDs. This approach enables broad characterization of underlying causes of PB-involving GSDs, an exhaustive examination of multiple mechanistic strategies for therapy and fast translational potential.
AbstractThe continued co-operation of physicians is requested in the referral of patients with glycogen storage disease for a study currently in progress at the Clinical Center, National Institutes of Health, Bethesda, Maryland. Patients known or suspected of having one of the forms of glycogen storage disease are needed for further study of metabolic errors leading to this group of disorders and for the elaboration of methods for the prevention and treatment of these conditions. It is desirable that the diagnosis be established by means of enzymatic assays of liver and muscle tissue obtained by surgical biopsy.
Public Health Weekly Report · 2025 · 0 citations · open access
당원병에 대한 종합적 리뷰: 분자 메커니즘, 진단 및 치료 전략
AbstractGlycogen storage disease (GSD) is a hereditary metabolic disorder caused by defective enzymes involved in carbohydrate metabolism. GSD leads to abnormal glycogen accumulation in tissues, such as the liver, muscles, and heart, resulting in various clinical symptoms, including hypoglycemia, hepatomegaly, muscle weakness, and heart dysfunction. There are several types of GSD. Each type of GSD is caused by a deficiency of a specific enzyme, and its clinical symptoms can vary significantly depending on the affected enzyme and organ systems. Advances in molecular genetics have elucidated the genetic basis of GSD, improving its diagnostic accuracy, and enhancing our understanding of its pathophysiology. This review focused on the molecular mechanisms, clinical symptoms, diagnostic approaches, and current treatment strategies for all types of GSD, including dietary management, enzyme replacement therapy, and emerging genetic therapies. It also addressed challenges relating to treatment adherence, long-term outcomes, and future research directions. Early diagnosis and personalized treatment plans are essential for improving the prognosis of patients with GSD. Continued research to discover more effective treatment options, and ultimately, cure the disease is crucial.
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.
