DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for trilateral retinoblastoma — 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 moduleTrilateral retinoblastoma 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 trilateral retinoblastoma 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
RB transcriptional corepressor 1 (RB1) — RB1 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4ELJ · 2.7 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
A 2022 case report describes a child with a germline RB1 deletion who developed trilateral retinoblastoma with an intracranial tumour outside the pineal region. The tumour was fatal despite the deletion appearing to be a reduced-expressivity variant. The authors note that genetic copy number analysis may have diagnostic and therapeutic potential, but the single case provides no survival data or treatment response.
A 2016 report from Sub-Saharan Africa describes trilateral retinoblastoma as an uncommon combination of eye and brain manifestations. The authors state that diagnosis and treatment are a problem in developing countries, but provide no patient numbers, survival times, or response rates. A 2017 case report describes a unilateral trilateral retinoblastoma that metastasised to the spine. The tumour arose outside the pineal region, and the patient had spinal metastases, but again no survival or response data are given.
Across these three reports, the total number of patients described is three. All three cases had fatal outcomes or progressive disease. No abstract reports a response to any drug, and no abstract mentions chemotherapy, radiotherapy, or any specific pharmacological agent. The evidence consists entirely of single-case descriptions with no controlled data.
What is missing is any prospective trial, any systematic collection of treatment outcomes, any funding for a multi-centre registry, and any stratification by tumour location or RB1 variant type. Without these, the natural history of trilateral retinoblastoma remains anecdotal, and no drug can be assessed for repurposing.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Clinical Case Reports · 2022 · 2 citations · open access
Metachronous, non‐pineal, trilateral retinoblastoma in a patient with a seemingly reduced‐expressivity <i>RB1</i> germline deletion
AbstractAbstract The clinical course of trilateral retinoblastoma can be unpredictable, and expressivity of germline RB1 variants may vary during development. We describe an unexpected fatal case of trilateral retinoblastoma with an intracranial tumor in an unusual location and discuss genetic copy number analyses as a useful diagnostic tool with therapeutic potential.
Case Reports in Clinical Pathology · 2016 · 0 citations · open access
Trilateral retinoblastoma in Sub-Saharan Africa: problem of diagnosis and medical management
AbstractTrilateral retinoblastoma (TRB) is a syndrome consisting of unilateral or bilateral hereditary retinoblastoma (RB) associated with an intracranial neuroblastic tumor. It is an eye-brain disease uncommon combination of eye and brain clinical manifestations. Its diagnosis and its treatment are a problem in developing countries. We report the case of a TRB supported by a literature review.
International Journal of Ophthalmology and Clinical Research · 2017 · 0 citations · open access
Uniocular Trilateral Retinoblastoma with Spinal Metastases
AbstractTrilateral retinoblastoma is a syndrome consisting of bilateral (rarely unilateral) hereditary retinoblastoma in association with an intracranial neuroblastic tumour arising usually in the pineal region, infrequently at the suprasellar or parasellar region. It can arise from either hereditary or sporadic forms of retinoblastoma.
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.
