DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for mucopolysaccharidosis type 1 — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMucopolysaccharidosis type 1 maps to a 3-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 mucopolysaccharidosis type 1 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
alpha-L-iduronidase (IDUA) — IDUA 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 1~{r},2~{r},3~{r},4~{s},6~{s}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I6R · 2.02 Å · ligand (1~{R},2~{R},3~{R},4~{S},6~{S})-6-azanyl-2,3,4-tris(oxidanyl)cyclohexane-1-carboxylic acid (H62). Experimental structure, not a prediction.
What the evidence adds up to
The MPS I Registry analysed 987 untreated patients, with 60.9% classified as Hurler (most severe), 23.0% as Hurler-Scheie, and 12.9% as Scheie (least severe). Median age at symptom onset was 6 months for Hurler, 1.5 years for Hurler-Scheie, and 5.3 years for Scheie. Median age at treatment initiation was 1.5 years, 8.0 years, and 16.9 years respectively, showing a clear delay between symptom onset and treatment, especially in attenuated forms. Coarse facial features and corneal clouding were the most common symptoms across all phenotypes. Most patients were from Europe (45.5%) and North America (34.8%), with smaller numbers from Latin America and Asia Pacific.
A case report describes a patient with typical clinical signs from very early age who was diagnosed only at 22 months old and then began enzyme replacement therapy. The report states that earlier detection leads to better and more effective treatment, with options being enzyme replacement therapy or, as a last resort, haematopoietic stem cell transplantation.
Current challenges in MPS I include the need for adequate classification linked to therapeutic indications, early diagnosis through neonatal screening, management of spinal and eye disease, allergic reactions caused by enzyme replacement therapy, and transition to adult care.
What is still missing is widespread neonatal screening implementation, better classification systems that predict treatment response, and dedicated studies on long-term outcomes for spinal and eye disease. No trial data on repurposed drugs for MPS I were provided in these abstracts.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Genetics in Medicine · 2014 · 173 citations · open access
The natural history of MPS I: global perspectives from the MPS I Registry
AbstractPURPOSE: In this study, we aimed to describe the natural history of mucopolysaccharidosis I. METHODS: Data from 1,046 patients who enrolled in the MPS I Registry as of August 2013 were available for descriptive analysis. Only data from untreated patients and data prior to treatment for patients who received treatment were considered. Age at symptom onset, diagnosis, and treatment initiation were examined by geographic region and phenotype (from most to least severe: Hurler, Hurler-Scheie, and Scheie). For each symptom, frequency and age at onset were examined. RESULTS: Natural history data were available for 987 patients. Most patients were from Europe (45.5%), followed by North America (34.8%), Latin America (17.3%), and Asia Pacific (2.4%). Phenotype distribution was 60.9% for Hurler, 23.0% for Hurler-Scheie, and 12.9% for Scheie (3.2% undetermined) syndromes. Median age at symptom onset for Hurler, Hurler-Scheie, and Scheie syndromes was 6 months, 1.5 years, and 5.3 years, respectively; median age at treatment initiation was 1.5 years, 8.0 years, and 16.9 years, respectively. Coarse facial features and corneal clouding were among the most common symptoms in all three phenotypes. CONCLUSION: A delay between symptom onset and treatment exists, especially in patients with attenuated mucopolysaccharidosis I. A better understanding of disease manifestations may help facilitate prompt diagnosis and treatment and improve patient outcomes.
Archivos Argentinos de Pediatria · 2021 · 0 citations · open access
Algunos desafíos en mucopolisacaridosis tipo I
AbstractHere we describe the current challenges of mucopolysaccharidosis type I: the need for an adequate classification, establishing its relationship to therapeutic indications; an early diagnosis, from neonatal screening, its advantages and barriers, to clinical suspicion of severe and attenuated forms; spinal and eye disease care, from diagnosis to follow-up and treatment; allergic reactions caused by enzyme replacement therapy, their diagnosis and treatment. And lastly, transition to adult care.
Archivos Argentinos de Pediatria · 2021 · 0 citations · open access
A few challenges in mucopolysaccharidosis type I
AbstractHere we describe the current challenges of mucopolysaccharidosis type I: the need for an adequate classification, establishing its relationship to therapeutic indications; an early diagnosis, from neonatal screening, its advantages and barriers, to clinical suspicion of severe and attenuated forms; spinal and eye disease care, from diagnosis to follow-up and treatment; allergic reactions caused by enzyme replacement therapy, their diagnosis and treatment. And lastly, transition to adult care.
Residência Pediátrica · 2021 · 0 citations · open access
Hurler syndrome: diagnostic journey in an orphan disease - case report
AbstractOBJECTIVES: Promote the early detection of the disease through pertinent clinical findings in patients with mucopolysaccharidosis. CASE REPORT: Patient with MPS I with typical clinical signs of the disease from a very early age were diagnosed only at 22 months old and will begin enzymatic replacement therapy. DISCUSSION: The disease, the earlier it is detected, the better and more effective the treatment, which may be enzyme replacement therapy, or, in the last case, transplantation of hematopoietic stem cells.
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.
