DeCure for Maturity-onset diabetes of the young type 3
DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for maturity-onset diabetes of the young type 3 — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMaturity-onset diabetes of the young type 3 maps to a 5-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 maturity-onset diabetes of the young type 3 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
insulin (INS) — INS 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 hc4drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6TC2 · 1.36 Å · ligand 4'-HYDROXYCINNAMIC ACID (HC4). Experimental structure, not a prediction.
What the evidence adds up to
A 2023 case report describes an 11-year-old girl with a de novo HNF1A mutation who was diagnosed with MODY3 only after exome-targeted capture sequencing. Initial treatment with insulin and metformin proved inefficient, but sulfonylureas were effective. The report emphasises that correct and timely diagnosis is the key to treatment, particularly for blood sugar control. No other patients or quantitative outcomes are given in this single-case report.
A 2012 study of acetyl-L-carnitine (50 mg/kg/day) plus nicotinamide (25 mg/kg/day) involved nine children at risk of type 1 diabetes, not MODY3. Treatment periods ranged from 16 to 120 months. Four patients recovered their parameters and medication was suspended; two continued treatment with favourable evolution; two evolved slowly with normal growth and development; one girl became diabetic because she was treated late. The authors concluded that the treatment delays development or remits evolution of type 1 diabetes in at-risk children. This study does not address MODY3.
A 2025 article announces the discovery of a new MODY subtype from India, labelled MODY 15, but provides no patient data, treatment outcomes, or molecular details. It only notes that historical classification of diabetes by age at onset is now understood to be inadequate.
No abstract reports a controlled trial of any drug specifically for MODY3. The 2023 case report shows that sulfonylureas can work after genetic diagnosis, but the sample is one patient. The 2012 study is about type 1 diabetes risk, not MODY3. What is missing is any randomised trial, any comparison of sulfonylureas with other agents in MODY3, any data on long-term outcomes, and any stratification by specific HNF1A mutation. Funding for such trials and systematic genetic screening to identify patients early remain absent.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Pediatric Endocrinology and Metabolism · 2012 · 6 citations
Children at risk of diabetes type 1. Treatment with acetyl-L-carnitine plus nicotinamide – Case reports
AbstractUNLABELLED: Abstract Objectives: The aim was to evaluate the treatment with acetyl-L-carnitine (50 mg/kg/day) and nicotinamide (25 mg/kg/day) in children at risk of type 1 diabetes. This treatment was effective and harmless in experimental type 1 diabetes in mice. PATIENTS: Nine out of seventy healthy participants of the type 1 diabetes risk study were treated. They were typified for diabetes with HLA-DQB1 and positive autoantibodies. Children with a first peak of insulin response ≤48 µU were randomly distributed in control and treated patients. Children evolution was followed with an intravenous glucose tolerance test. Control children were treated when was another risk parameter was added. During their evolution all children were treated. RESULTS: Treatment periods differ (range: 120-16 months) because children began treatment at different times. During the treatment 4 patients recovered their parameters and the medication was suspended; 2 patients continued the treatment with favorable evolution. Two children evolved slowly with normal growth and development. One girl became diabetic because she was treated late. CONCLUSIONS: In children at risk, this treatment delays the development or remits the evolution of type 1 diabetes.
De novo HNF1A mutation of young maturity-onset diabetes 3 of a young girl—Case report
AbstractYoung maturity-onset diabetes of the young type3(MODY3) as a special type of diabetes, the probability of diagnosis is low. This article reports on a case and reviews the relevant knowledge of the disease. We report an 11-year-and-11-month-old girl whose grandmother died from diabetic complications while the rest of the families were non-diabetes. The proband was initially treated with insulin and metformin but the threatment proved inefficient. After an exome-targeted capture sequencing test, she was diagnosed with mature-onset diabetes of young type 3 (MODY3), and sulfonylureas make sense. The key to mody treatment is a correct and timely diagnosis, which contributes to helping patients overcome the problems of MODY3, especially for blood sugar control.
Journal of the Association of Physicians of India · 2025 · 1 citations
Exciting Discovery of a New Maturity-onset Diabetes of the Young Subtype from India (MODY 15)
AbstractAt that time, the classification of diabetes was based purely on the age at onset of diabetes. Those diagnosed with diabetes below 40 years of age were labeled as "growth onset diabetes," while those with onset at or above the age of 40 years were referred to as "maturity onset diabetes." At that time, these types were believed to be equivalent to what are known as type 1 diabetes (T1D) and type 2 diabetes (T2D) today.
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.
