DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for infantile myofibromatosis — 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 moduleInfantile myofibromatosis 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 infantile myofibromatosis 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
neurotrophic receptor tyrosine kinase 3 (NTRK3) — NTRK3 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 4-aminophenyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6KZD · 1.708 Å · ligand 3-[2-[6-(4-aminophenyl)imidazo[1,2-a]pyrazin-3-yl]ethynyl]-2-methyl-~{N}-[3-(4-methylpiperazin-1-yl)-5-propan-2-yl-phenyl]benzamide (DZ6). Experimental structure, not a prediction.
What the evidence adds up to
Infantile myofibromatosis occurs primarily in neonates and infants, either as a single nodular lesion or as a multicentric form. Evidence from a 1984 study of two affected neonates and one affected father supports an autosomal-dominant mode of inheritance and notes the potential for recurrence after a long period of quiescence. Most cases are limited to cutaneous lesions that resolve spontaneously.
Generalised infantile myofibromatosis with visceral involvement carries a reported mortality rate as high as 73%. A 2014 report described two paediatric patients with visceral involvement who were treated with low-dose methotrexate and vinblastine, and the authors reviewed literature describing chemotherapy for these patients. A 1993 case described massive pelvic involvement that complicated delivery and precluded meaningful therapy. A 2008 report described a three-year-old boy diagnosed with infantile myofibromatosis originating from the occipital region, contrary to initial radiological findings.
No controlled trials, no prospective data on long-term outcomes, and no validated biomarkers for predicting which cases will progress to visceral involvement are available. The evidence base lacks randomised comparisons of chemotherapy versus observation, and the optimal regimen, duration of treatment, and criteria for patient selection remain undefined.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The American Journal of Surgical Pathology · 1984 · 133 citations
Infantile myofibromatosis
AbstractInfantile myofibromatosis is a distinct clinicalpathologic entity occurring primarily in neonates and infants either as a single nodular lesion or as a multicentric form. We have recently studied two cases of this disorder in neonates, and a third involving the father of one of these infants, all documented by biopsy. Evidence in support of an autosomal-dominant mode of inheritance for infantile myofibromatosis and of its potential to recur after a long period of quiescence is presented.
Journal of Pediatric Hematology/Oncology · 2014 · 33 citations
Chemotherapy for Generalized Infantile Myofibromatosis With Visceral Involvement
AbstractInfantile myofibromatosis (IM) is most commonly limited to cutaneous lesions that resolve spontaneously. However, generalized IM with visceral involvement, which has a reported mortality rate as high as 73%, has been successfully treated with a combination of methotrexate and vinblastine. Here we report the further efficacy of low-dose methotrexate and vinblastine in 2 pediatric patients with IM and visceral involvement and review the literature describing chemotherapy for these patients.
European Journal of Pediatric Surgery · 1993 · 17 citations
Infantile Myofibromatosis: An Unusual Presentation and a Review of the Literature
AbstractAn infant with an unusual presentation of Infantile Myofibromatosis (IM) is presented. Massive involvement of the pelvic region complicated the delivery and precluded meaningful therapy. The literature is reviewed. Zusammenfassung Es wird ein Kind mit einer ungewöhnlich verlaufenden, infantilen Myofibromatosis vorgestellt. Bei dem Patienten war insbesondere die Beckenregion ganz massiv befallen, was sowohl die Entbindung wie die Therapie erschwerte. Die entsprechende Literatur wird dargestellt.
Infantile Myofibromatosis in a Three-Year-Old Boy Presented as Subdural Hematoma
AbstractInfantile myofibromatosis, mostly developing at birth or in early infancy, is a rare clinical disorder characterized by myofibroblastic lesions. We report clinical, radiological and pathological features of a three-year-old boy who was diagnosed with infantile myofibromatosis originating from the occipital region contrary to radiological findings in advanced diagnostic studies.
European transport law: = Droit européen des transports = Europäisches Transportrecht = Diritto europeo dei trasporti = Derecho europeo de transportes = Europees vervoerrecht · 1998 · 0 citations
La C.M.R. en tant que regle des transports interieurs: (h la suite de Cass. com. ler juillet 1997)
AbstractInfantile myofibromatosis (IM) is most commonly limited to cutaneous lesions that resolve spontaneously. However, generalized IM with visceral involvement, which has a reported mortality rate as high as 73%, has been successfully treated with a combination of methotrexate and vinblastine. Here we report the further efficacy of low-dose methotrexate and vinblastine in 2 pediatric patients with IM and visceral involvement and review the literature describing chemotherapy for these patients.
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.
