DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for ganglioneuroblastoma — 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 moduleGanglioneuroblastoma 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
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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 ganglioneuroblastoma 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
CREB binding lysine acetyltransferase (CREBBP) — CREBBP 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 1vudrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9H0K · 1.75 Å · ligand propionyl Coenzyme A (1VU). Experimental structure, not a prediction.
What the evidence adds up to
A 10-month infant with ganglioneuroblastoma was observed to mature into ganglioneuroma over seven years, one of five such recorded cases by 1967. Possible mechanisms proposed include differentiation of neuroblasts by natural organisers, x-ray initiation of normal differentiation, destruction of susceptible cells by irradiation with survival of resistant ganglion cells, or inhibition of a nerve growth factor. A 1979 histopathological study described two varieties of ganglioneuroblastoma: one combining undifferentiated cells with mature typical ganglion cells, the other combining undifferentiated cells with pleomorphic atypical cells, and noted diagnostic difficulties for the second type.
In a retrospective study of 67 children with ganglioneuroma (50) and ganglioneuroblastoma-intermixed (17) at a single hospital between 1990 and 2014, median age was 6 years and median tumour diameter 6.3 cm. Upfront surgery was performed in 46 patients (69%), while 21 (31%) were observed; 4 of the observed patients later underwent resection. Post-operative complications occurred in 15 of 50 surgical patients (30%), and imaging-defined risk factors correlated with complications. Among observed patients, median growth was 0.3 cm per year, and 6 of 21 had progressive disease. At median follow-up of 2.2 years, all patients were alive; among those with evaluable imaging, there were 27 complete and 10 partial responses, 19 stable disease, and 6 progressive disease. No ganglioneuroma was reclassified to neuroblastoma at resection. The authors concluded that watch-and-wait approaches should be considered for some cases.
A 2023 report described a 5-year-old boy with intracranial leptomeningeal CNS ganglioneuroblastoma presenting with sudden loss of consciousness, a large posterior fossa lesion, and intraventricular metastases. He underwent posterior fossa resection and subtotal resection of a third ventricle lesion with adjuvant chemotherapy, but died three months after diagnosis. The authors stated that ganglioneuroblastoma is extremely likely to recur quickly and extensively, and that gross total resection followed by adjuvant radiotherapy and chemotherapy is the best documented management. What remains missing are prospective trials large enough to stratify by histological subtype and anatomical location, standardised imaging surveillance protocols for observed patients, and any evidence that adjuvant therapy improves survival in the intracranial form beyond single case reports.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Cancer · 1967 · 70 citations
Maturation of ganglioneuroblastoma to ganglioneuroma
AbstractA case of ganglioneuroblastoma in a 10-month infant maturing over a 7-year period to ganglioneuroma is reported. The four previously recorded cases are reviewed. Possible mechanisms involved may include differentiation of neuroblasts to mature ganglion cells by the influence of naturally occurring organizers, x-ray initiation of normal cell differentiation, destruction of susceptible neoplastic cells by irradiation with survival of resistent ganglion cells or inhibition of a nerve growth factor stimulating neuroblast proliferation.
Pediatric Blood & Cancer · 2018 · 43 citations · open access
Characteristics and management of ganglioneuroma and ganglioneuroblastoma‐intermixed in children and adolescents
AbstractBACKGROUND: Ganglioneuromas (GNs) usually demonstrate favorable histological and clinical features. Surgery is often performed due to clinical symptoms and/or theoretical concerns that GN may transform into neuroblastoma (NB); however, several studies have identified significant GN-surgical morbidities. OBJECTIVES: We compared the natural history, biological and clinical features of GN and ganglioneuroblastoma-intermixed (GNB-I) managed by surgery or observation to inform management and surveillance. PROCEDURES: This retrospective study includes patients (n = 67) with histological diagnosis of GN (50/67) and GNB-I (17/67) at the Hospital for Sick Children between 1990 and 2014. Clinical, pathological features, tumor dimensions, and management were recorded. RESULTS: Median age and maximal tumor diameter were 6 years (1.3-17.8) and 6.3 cm (1.4-16.9), respectively. Of the 67 patients, 46 (69%) had upfront surgery and 21 (31%) were observed. Of the 21 observed patients 4 later underwent resection. There were post-operative complications in 15 of the 50 (30%) surgical patients. The presence of imaging-defined risk factors correlated with complications (P = 0.005). Observed patients were older (median 8.4 vs. 5.3 years) and diagnosed more recently. Median growth was 0.3 cm/year and 6 of 21 had progressive disease (PD). At median follow-up of 2.2 years (0.2-14.3), all patients were alive and for those with evaluable imaging there were 27 complete and 10 partial responses, 19 stable and 6 PD. Pathology classification changed at resection for three cases, but no GN was reclassified to NB. CONCLUSIONS: GN and GNB-I have a slow growth rate and resection can be associated with significant morbidity. Watch and wait approaches should be considered for some GN and GNB-I.
British Journal of Neurosurgery · 2023 · 2 citations
Intracranial leptomeningeal CNS ganglioneuroblastoma. First report and review of the literature
AbstractBACKGROUND: CNS ganglioneuroblastoma in an extremely rare embryonal tumour, specifically in the pediatric population. Bad prognosis is documented due to aggressiveness and absence of protocolized treatment at the moment. CLINICAL DESCRIPTION: We present the case of a 5-year-old boy who presented with sudden loss of consciousness. CT scan was performed showing a large posterior fossa lesion with several intraventricular focal lesions, suggesting metastases, the largest one located inside the III ventricle. The patient underwent a posterior fossa resection of the lesion and a subtotal resection of the III ventricle lesion, with adjuvant chemotherapy. The evolution was poor and the patient finally died 3 months after diagnosis. CONCLUSION: Ganglioneuroblastoma is extremely likely to recur quickly and extensively. There is little knowledge about treatment options but is documented that gross total resection followed by adjuvant radiotherapy and chemotherapy is the best management in these patients.
Histopathological Study of an Imperfect Type Ganglioneuroblastoma
AbstractBased on their observations, the authors stress the occurrence of 2 histological varieties of ganglioneuroblastoma; the former characterized by a combination of undifferentiated with mature typical ganglion cells, the latter revealing a combination of undifferentiated with pleomorphic atypical cells. After demonstrating the ganglion origin of pleomorphic cells, attention is focused on the difficulties involved in the diagnosis of the second type of ganglioneuroblastoma, which in the author's view should be included in the present classifications of ANS tumors.
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.
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