DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for optic nerve glioblastoma — 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 moduleOptic nerve glioblastoma 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 optic nerve glioblastoma 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
neurofibromin 1 (NF1) — NF1 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 1sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7PGU · 3.3 Å · ligand (1S)-2-{[(2-AMINOETHOXY)(HYDROXY)PHOSPHORYL]OXY}-1-[(PALMITOYLOXY)METHYL]ETHYL STEARATE (PEV). Experimental structure, not a prediction.
What the evidence adds up to
Of 36 patients with glioma of the optic nerves or chiasm followed for a median of 10.2 years, one of five patients whose tumour was initially confined to the optic nerve had a recurrence after complete resection. For 25 patients irradiated for biopsy-proven chiasmal glioma, actuarial survival was 96% at 5 years, 90% at 10 years, and 90% at 15 years; progression-free survival was 87% at 5, 10, and 15 years. Vision stabilised or improved in 86% of patients after radiotherapy. Doses above a NSD of 1385 ret gave significantly better progression-free survival, but one serious complication occurred at 1533 ret. The recommended dose was 45 to 50 Gy in 1.8 Gy fractions.
Optic nerve gliomas are generally benign in children but usually malignant and aggressive in adults, so diagnostic and management approaches differ by age. In neurofibromatosis type 1, optic pathway gliomas are low-grade tumours that can cause vision loss. Genetically engineered mice with Nf1 mutations develop optic gliomas that resemble the human disease and have been used to study tumour initiation, growth, and vision loss, as well as to test molecularly targeted therapies. A 1983 series of 40 cases (24 paediatric) argued for surgical excision of nerve tumours and at least biopsy for chiasmal lesions, but the evidence presented was limited to seven selected surgical cases.
What is still missing: prospective trials that separate paediatric from adult optic nerve glioblastoma, given their radically different behaviour; validated biomarkers to predict which tumours will progress; and funding for preclinical models that can test drugs specifically against the malignant adult form, which the available literature barely addresses.
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 · 1988 · 118 citations · open access
Management of low-grade gliomas of the optic nerve and chiasm
AbstractThirty-six patients were evaluated between 1965 and 1983 for glioma of the optic nerves and/or chiasm. Median follow-up was 10.2 years. Pathologic verification was obtained in 32 patients. Tumor initially confined to the optic nerve recurred in one of five patients after complete resection. The actuarial survival for 25 patients irradiated for biopsy-proven glioma of the optic chiasm was 96%, 90%, and 90% at 5, 10, and 15 years, respectively, and the progression-free survival was 87% at 5, 10, and 15 years. Vision stabilized or improved in 86% of patients after radiotherapy. Patients irradiated to a dose greater than a NSD of 1385 ret had a significantly improved progression-free survival (P = 0.015). One serious complication occurred after a dose of 1533 ret. The recommended radiation dose for optic glioma is 45 to 50 Gy with 1.8 Gy fractions.
Journal of Neurological Surgery Part B Skull Base · 2021 · 21 citations · open access
Optic Nerve Gliomas
AbstractAbstract Objectives To describe the diagnostic and management features of optic nerve gliomas. Design Literature review. Results Optic nerve gliomas are generally benign in the pediatric age group although they are usually malignant and aggressive in adults. As such, the mechanisms by which these lesions are diagnosed, the systemic implications, the goals of intervention, and the nature of therapeutic management all differ between these tumors. Conclusions This article addresses these lesions and discusses the diagnostic and therapeutic paradigms by which they may be approached.
Journal of Neuroscience Research · 2018 · 20 citations · open access
Insights into optic pathway glioma vision loss from mouse models of neurofibromatosis type 1
AbstractNeurofibromatosis type 1 (NF1) is a common cancer predisposition syndrome caused by mutations in the NF1 gene. The NF1-encoded protein (neurofibromin) is an inhibitor of the oncoprotein RAS and controls cell growth and survival. Individuals with NF1 are prone to developing low-grade tumors of the optic nerves, chiasm, tracts, and radiations, termed optic pathway gliomas (OPGs), which can cause vision loss. A paucity of surgical tumor specimens and of patient-derived xenografts for investigative studies has limited our understanding of human NF1-associated OPG (NF1-OPG). However, mice genetically engineered to harbor Nf1 gene mutations develop optic gliomas that share many features of their human counterparts. These genetically engineered mouse (GEM) strains have provided important insights into the cellular and molecular determinants that underlie mouse Nf1 optic glioma development, maintenance, and associated vision loss, with relevance by extension to human NF1-OPG disease. Herein, we review our current understanding of NF1-OPG pathobiology and describe the mechanisms responsible for tumor initiation, growth, and associated vision loss in Nf1 GEM models. We also discuss how Nf1 GEM and other preclinical models can be deployed to identify and evaluate molecularly targeted therapies for OPG, particularly as they pertain to future strategies aimed at preventing or improving tumor-associated vision loss in children with NF1.
Anterior optic gliomas: pros and cons of surgical and conservative treatment*
AbstractSeven surgical patients have been selected from a series of 40 cases (24 pediatric) of anterior optic glioma as examples of the advantage of surgical (excision of nerve tumors; exploration and biopsy as a minimum program for chiasmal lesions) over conservative treatment. The results obtained in these seven cases reflect those seen in the total series and those to be gleaned from most of the literature
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.
