Rare & Orphan Lab · DeCure for X

DeCure for Juvenile Xanthogranuloma

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Juvenile Xanthogranuloma — screening already-approved drugs against its 10-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module10 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:4424$DeCureRare

The disease map

Disease moduleJuvenile Xanthogranuloma maps to a 10-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 juvenile xanthogranuloma 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

isocitrate dehydrogenase (NADP(+)) 2 (IDH2)IDH2 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 ndpdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 5I96 · 1.55 Å · ligand NADPH DIHYDRO-NICOTINAMIDE-ADENINE-DINUCLEOTIDE PHOSPHATE (NDP). Experimental structure, not a prediction.

What the evidence adds up to

Juvenile xanthogranuloma is an uncommon diagnosis, with the head, neck and trunk being the most common sites. One case report describes a five-month-old girl with a circumscribed papule below the right nasal ala and above the right vermilion border; the lesion was histologically diagnosed after surgical resection. No drug treatment was involved in that case.

A 2023 report describes a congenital, rapidly growing giant tumour in a newborn that mimicked a malignant tumour but was histologically a variant of juvenile xanthogranuloma. The authors discuss novel treatment options based on recent literature and refer to the results of therapy in their patient, but the abstract does not name any specific drug, give response rates, survival figures, or sample sizes. The case is presented as an extraordinary variant of a benign, mostly spontaneously regressing condition.

Across these three abstracts, no drug is mentioned by name, no systemic therapy is evaluated, and no quantitative outcomes from any treatment are reported. The condition is described as benign and often self-limiting, with surgery used for diagnosis or removal of isolated lesions.

What is missing is any controlled trial, any prospective data on drug efficacy, any patient stratification, and any funding for a repurposing study. Without a named drug, a measurable endpoint, or a treated cohort, no evidence for drug repurposing exists 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.

Plastic Surgery · 2008 · 53 citations · open access

Juvenile Xanthogranuloma: Case Report and Review of the Literature

AbstractThe present case report describes a juvenile xanthogranuloma in a five-month-old girl. A circumscribed papule was located below the right nasal ala and above the right vermilion border. The lesion was histologically diagnosed as a juvenile xanthogranuloma after surgical resection of the mass. Juvenile xanthogranuloma is an uncommon diagnosis, with the head, neck and trunk being the most common sites.

https://doi.org/10.1177/229255030801600309
Cornea · 2009 · 16 citations

New Treatment Option for Adult-onset Limbal Xanthogranuloma

AbstractPURPOSE: The purposes of this study are to report a case of adult-onset juvenile xanthogranuloma and suggest a new treatment option. METHODS: A 35-year-old man presented with an isolated mass of the limbus. Biopsy revealed a histiocytic tumor with Touton's giant cells staining positive for CD68 and S-100 but negative for CD1a, consistent with JXG. Two intratumoral injections of triamcinolone acetonide 6 weeks apart were performed. The main outcome measure was 6-months follow up clinically and by ultrasound biomicroscopy. RESULTS: The lesion regressed, and corneal thickness returned to normal 3 months after the first injection. An increase in intraocular pressure required topical treatment. No recurrence was seen throughout follow up. CONCLUSION: Limbal juvenile xanthogranuloma can be successfully treated with intralesional triamcinolone acetonide, thus avoiding a lamellar keratectomy approach.

https://doi.org/10.1097/ico.0b013e3181a06efa
Plastic Surgery · 2008 · 15 citations · open access

Juvenile xanthogranuloma: Case report and review of the literature

AbstractThe present case report describes a juvenile xanthogranuloma in a five-month-old girl. A circumscribed papule was located below the right nasal ala and above the right vermilion border. The lesion was histologically diagnosed as a juvenile xanthogranuloma after surgical resection of the mass. Juvenile xanthogranuloma is an uncommon diagnosis, with the head, neck and trunk being the most common sites.

https://doi.org/10.4172/plastic-surgery.1000572
Bőrgyógyászati és Venerológiai Szemle · 2023 · 0 citations · open access

Congenital rapidly growing giant tumors in a newborn

AbstractThe authors present a juvenile xanthogranuloma case with unusual clinical features. The results of the checkupand the differential diagnosis’ respects. They discuss the novel treatment options based on the recent literature and refer to the results of the therapy in their patient. The case presents an extraordinary, rapidly growing, malignant tumor-mimicking variant of the benign, mostly spontaneously regressing juvenile xanthogranuloma.

https://doi.org/10.7188/bvsz.2023.99.5.6

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.