DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for desmoplastic infantile ganglioglioma — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleDesmoplastic infantile ganglioglioma maps to a 1-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 desmoplastic infantile ganglioglioma 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
B-Raf proto-oncogene, serine/threonine kinase (BRAF) — BRAF 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 agsdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8VYP · 3.29 Å · ligand PHOSPHOTHIOPHOSPHORIC ACID-ADENYLATE ESTER (AGS). Experimental structure, not a prediction.
What the evidence adds up to
Desmoplastic infantile ganglioglioma is a rare intracranial tumour of childhood that involves the cerebral cortex and leptomeninges. In a 1994 report from the Pediatric Oncology Group infant brain tumor study (Protocol 8633), four children under one year of age were diagnosed with these massive cystic tumours. All four had been diagnosed by their institutions as having malignant tumours such as Grade III astrocytoma or gliosarcoma. None had metastatic disease. One child had a gross total resection; three had debulking procedures. All four received chemotherapy (cyclophosphamide, vincristine, cisplatinum, etoposide) for 12 to 24 months. Among those with postoperative measurable disease, one had a complete response, one a partial response, and one had stable disease at the end of chemotherapy. No child received radiation. All four were alive with progression-free survivals of more than 36, 42, 48, and 60 months after diagnosis.
A 2005 report describes two patients with desmoplastic infantile ganglioglioma who developed multiple cerebrospinal metastases. The authors note that only two similar cases had been reported in the literature at that time. Pathologically, these are low-grade neoplasms showing glial and ganglionic differentiation with extreme desmoplasia, but the report describes them as questionably benign.
A 2012 literature review and three new patient cases suggest the initial benign description should be amended. Nearly 23% of DIG cases occur in children older than 24 months. Approximately 40% of cases require additional medical, radiation, or further surgical intervention. Fifteen percent of infants and children develop leptomeningeal spread or die from DIG. The review concludes that DIG represents a heterogeneous disease and calls for expanded biological and molecular investigation.
What is still missing is a systematic molecular characterisation of these tumours to stratify patients by risk, a prospective trial large enough to test whether chemotherapy alone is sufficient for subtotally resected cases, and funding to support such work across the rare-disease networks that would be needed to recruit patients.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Neurosurgery · 1994 · 79 citations
Desmoplastic Infantile Gangliogliomas
AbstractDesmoplastic infantile gangliogliomas are massive cystic tumors, typically occurring in the cerebral hemispheres of infants. They are remarkable pathologically for a prominent desmoplasia and, in some cases, for a cellular mitotically active component that can be readily interpreted as a malignant neoplasm. Four children less than 1 year of age were diagnosed with desmoplastic infantile gangliogliomas in the Pediatric Oncology Group infant brain tumor study (Protocol number 8633). All had been diagnosed by their respective institutions as having malignant tumors, i.e., Grade III astrocytoma, malignant meningioma, leptomeningeal fibrosarcoma, and gliosarcoma. All had increased intracranial pressure, and two had seizures. The tumors were extremely large, with one measuring 12 x 9 x 9 cm. None had evidence of metastatic disease. One patient had a gross total resection, and the other three had debulking procedures. All four children were treated with chemotherapy (cyclophosphamide, vincristine, cisplatinum, etoposide) for periods ranging from 12 to 24 months. Of those with postoperative measurable disease, one child had a complete response, one a partial response, and one had stable disease at the conclusion of chemotherapy. No child received radiation therapy. All children are alive with progression-free survivals after diagnosis of more than 36, 42, 48, and 60 months, respectively. Although desmoplastic infantile gangliomas are rare, recognition of this tumor type is essential because, despite their massive size and pathologically malignant appearance, they may have a relatively benign clinical course. If total surgical resection can be achieved, further therapy may not be indicated. In those patients in whom residual disease is present, chemotherapy appears to be an effective form of therapy.(ABSTRACT TRUNCATED AT 250 WORDS)
Journal of Pediatric Hematology/Oncology · 2012 · 31 citations
Clinical Heterogeneity of Desmoplastic Infantile Ganglioglioma
AbstractDesmoplastic infantile gangliogliomas (DIG) are intracranial tumors described in 1987 as benign lesions of infancy. A literature review and the clinical course of 3 patients reported herein suggest that the initial description should be amended. Nearly 23% of DIG cases occur in children older than 24 months. Approximately 40% of DIG cases require additional medical, radiation, and/or further surgical intervention, and 15% of infants and children develop leptomeningeal spread or die from DIG. Such adverse outcomes, combined with the recognition that DIG represents a heterogeneous disease, underscore the need for an expanded biological and molecular investigation.
Desmoplastic infantile ganglioglioma: A questionably benign tumour
AbstractDesmoplastic infantile ganglioglioma is a rare intracranial tumour of childhood that involves the cerebral cortex and the leptomeninges. We report two patients with desmoplastic infantile gangliogliomas and multiple cerebrospinal metastases. To our knowledge, only two similar cases have been reported in the published literature. Pathologically, this rare intracranial tumour shows glial and ganglionic differentiation, accompanied by an extreme desmoplastic reaction. These are low-grade neoplasms that are questionably benign.
Central European Neurosurgery · 2009 · 3 citations
Long-Term Follow-up of a Non-Infantile Desmoplastic Ganglioglioma
AbstractDesmoplastic gangliogliomas are mixed cerebral tumors traditionally reported in infants. However, a few non-infantile cases have been documented. A case of a desmoplastic ganglioglioma in a 16-year male is presented. The patient reported severe headaches. Radiological examination revealed a large mass occupying the right frontal lobe. The lesion was totally excised. Histopathological examination confirmed the diagnosis of a desmoplastic ganglioglioma. The postoperative course was excellent. At the 10(1/2) year follow-up there was no evidence of tumor recurrence. Although desmoplastic gangliogliomas have aggressive features, complete surgical removal is the treatment of choice obviating the need for adjuvant therapy.
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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