Rare & Orphan Lab · DeCure for X

DeCure for Gangliocytoma

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for gangliocytoma — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module2 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:2426$DeCureRare

The disease map

Disease moduleGangliocytoma maps to a 2-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 gangliocytoma 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(+)) 1 (IDH1)IDH1 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 ictdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 6BKX · 1.65 Å · ligand ISOCITRIC ACID (ICT). Experimental structure, not a prediction.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Revista de Neurología · 1998 · 8 citations

Tumores de células ganglionares

AbstractINTRODUCTION: Tumours of ganglion cells are very rare. They include: gangliocytoma, ganglioneuroma, Lhermitte-Duclos disease and dysembryoplastic neuroepithelial tumour. All require microscope identification of well differentiated neurons for diagnosis. Their pathogenesis is not fully understood. Some workers consider them to be dysplasias rather than true neoplasias; others refer to them as malformations. OBJECTIVE: We aim to analyze the most characteristic findings of these tumours with regard to the elements of which they are composed, their epidemiology, behavior on imaging investigations (CT and MR) and treatment. CLINICAL CASES: We present two cases: one patient with a gangliocytoma and one with a ganglioglioma. Both were treated surgically, confirmed on histological study and had good clinical results.

https://doi.org/10.33588/rn.27160.97337
AME Case Reports · 2023 · 1 citations · open access

A case report of giant gangliocytoma of mediastinum

AbstractBackground: Gangliocytoma is an uncommon disease. In this report, we report a patient who was admitted to the hospital with a mediastinal tumor which is giant rarely. Case Description: A 30-year-old male patient was found to have a mediastinal mass 2 weeks ago during a routine examination. The patient occasionally had chest pain before 1 month. Computed tomography of the chest showed a mass occupying the posterior mediastinum and located anterior to the spine, above the diaphragm. The mass is removed by thoracoscopic surgery. In the surgical field of view, the tumor was loosely adhered to the surrounding organs and was freed by blunt-sharp combination Histologically confirmed mediastinal gangliocytoma with a size of 9.5 cm × 6.0 cm × 3.0 cm. The surgery was successful, and the patient's symptoms were completely relieved after the surgery. The patient underwent chest X-ray review at the 6th month after discharge, and no recurrence was found. Conclusions: Gangliocytomas are rare tumors of the peripheral nervous system. However, most of these tumors are retroperitoneal and are more common in children and young adults. Ganglioneuromas arise from neural crest cells. Most of these tumors are asymptomatic, but some may develop high blood pressure and flushing. Surgery is the best way to treat such tumors, and attention should be paid to protecting the normal tissue around the tumor during surgery to prevent postoperative complications.

https://doi.org/10.21037/acr-23-55

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.