DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for retinoblastoma — screening already-approved drugs against its 43-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleRetinoblastoma maps to a 43-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
approvedVincristineApproved drug
Structures already discussed alongside retinoblastoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
bovine ABCC1 — Vincristine has a real, experimentally solved structure in complex with this target (PDB 9LGC, 2.95 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet r1qdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9LGC · 2.95 Å · ligand Vincristine (R1Q). Experimental structure, not a prediction.
What the evidence adds up to
Retinoblastoma is the most common primary intraocular malignancy in children. Modern treatment protocols and early disease detection can achieve disease-free globe and eye preservation success rates up to 100%. Treatment strategies include intravenous chemoreduction, local administration routes of chemotherapy (intra-arterial, intravitreal), focal therapy for tumour consolidation (photocoagulation, thermotherapy, cryotherapy, plaque brachytherapy), external beam radiotherapy, and surgical enucleation. Over the past decade management has shifted toward local chemotherapy and away from systemic chemotherapy. Treatment of advanced cases remains complex, requiring aggressive chemotherapy or external beam radiation. Treatment protocols are extremely diverse and dependent on local resources, so success rates are variable.
A 2016 review noted that innovations in molecular biology and targeted therapies have led to improvements in survival rates and ocular salvage, but stated that the need still exists to further assess the long-term effects of such directional changes in therapy. A 2022 narrative review reported that with modern protocols and early detection success rates can reach up to 100% of disease-free-globe and eye preservation, but that treatment of advanced cases remains complex and protocols are extremely diverse and dependent on local resources, making success rates variable.
A 2016 bioinformatics study identified seven key genes in retinoblastoma—EPARS1, FN1, HLA-DPA1, HLA-DPB1, HLA-DRA, CFI, and transforming growth factor beta receptor II—by comparing classifications of differentially expressed genes, differential pathway genes, seed genes, hub genes, and informative genes. The classification performance of these informative genes was reported as AUC = 1.00, TNR = 1.00, TPR = 1.00, and MCC = 1.00. The authors considered these potential biomarkers for detection and therapy, but this was a computational analysis, not a clinical trial.
What is still missing are prospective trials that stratify patients by disease stage and molecular markers, long-term follow-up data on the ocular and systemic consequences of current local chemotherapy approaches, and independent validation of the proposed gene signatures in patient samples. No drug repurposing candidate is mentioned in any of these abstracts.
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 Control · 2016 · 65 citations
Therapeutic Options for Retinoblastoma
AbstractBACKGROUND: Retinoblastoma is the most common primary intraocular malignancy in children. The management of retinoblastoma is complex and depends on several factors. METHODS: This review provides an update on current and emerging therapeutic options for retinoblastoma. The medical literature was searched for articles relevant to the management of retinoblastoma. The results of prospective and retrospective studies on chemotherapy and focal therapy for retinoblastoma are summarized. Animal models for novel therapeutic agents are also discussed. RESULTS: Treatment strategies for retinoblastoma involve intravenous chemoreduction, local administration routes of chemotherapy (eg, intra-arterial, intravitreal), focal therapy for tumor consolidation (eg, photocoagulation, thermotherapy, cryotherapy, plaque brachytherapy), external beam radiotherapy, and surgical enucleation. Emerging therapies include alternative chemotherapeutic agents, molecularly targeted therapies, and novel drug-delivery systems. CONCLUSION: In the past 10 years, the management strategy for retinoblastoma has significantly changed, shifting toward local chemotherapy and away from systemic chemotherapy. Innovations in the field of molecular biology and the development of targeted therapies have led to improvements in survival rates and ocular salvage for this disease. However, the need still exists to further assess the long-term effects of such directional changes in Therapy.
Topotecan and vincristine combination is effective against advanced bilateral intraocular retinoblastoma and has manageable toxicity
AbstractBACKGROUND: New, effective chemotherapeutic agents are needed for intraocular retinoblastoma. METHODS: This institutional clinical trial sought to estimate the rate of response to 2 courses of vincristine and topotecan (VT) window therapy in patients with bilateral retinoblastoma and advanced disease (Reese-Ellsworth group IV or V) in at least 1 eye. The topotecan dose started at 3 mg/m(2) /day for 5 days and was adjusted to target a systemic exposure of 140 ± 20 ng/mL · hour. The vincristine dose was 0.05 mg/kg for patients <12 months of age and 1.5 mg/m(2) for those >12 months of age at diagnosis. RESULTS: From February 2005 to June 2010, 27 patients received VT window therapy. Median age at enrollment was 8.1 months (range, 0.7-22.1 months). Twenty-four patients (88.9%) responded to window therapy (95% confidence interval = 71.3%-96.9%). Hematologic toxicity comprised grade 4 neutropenia (n = 27), grade 3 anemia (n = 19), and grade 3/4 thrombocytopenia (n = 16). Thirteen patients had grade 3 nonhematologic toxicity. Granulocyte colony-stimulating factor support was added after 10 patients had been treated, and it significantly reduced the duration of grade 4 neutropenia (median, 7 vs 24 days; P < .001). Pharmacokinetic studies showed rapid changes in topotecan clearance rates during the first year of life. CONCLUSIONS: The combination of topotecan and vincristine is effective for the treatment of advanced intraocular retinoblastoma. Granulocyte colony-stimulating factor treatment alleviates the duration of grade 4 neutropenia. Appropriate topotecan starting doses for patients 0-3, 3-6, 6-9, 9-12, and >12 months of age are specified.
A Narrative Review - Therapy Options and Therapy Failure in Retinoblastoma
AbstractRetinoblastoma (RB) management has evolved over the last three decades. Goals of modern RB treatment are first to protect life and prevent metastatic disease, then preservation of the globe and useful vision. With modern treatment protocols and early disease detection success rates can reach up to 100% of disease-free-globe and eye preservation. Treatment of advanced cases remains complex, requiring aggressive chemotherapy or/and external beam radiation. Treatment protocols are extremely diverse and dependent on local resources thus success rates are variable. Here we review narratively current treatment protocols and failure rates based on a PubMed search using keywords of retinoblastoma, retinoblastoma seed, retinoblastoma treatment, enucleation.
Retinoblastoma: An Update on Clinical, Genetic Counseling, Epidemiology and Molecular Tumor Biology
AbstractRetinoblastoma is the first tumor suppressor gene discovered ever. The discovery opened a new avenue in the field of oncology leading to the identification of 35 tumor suppressor genes, till date in our genome. This book is an excellent compilation of both clinical and basic science information that meets the needs of a young clinician and a researcher at the same time. It also has abundant information on recent advances and cutting-edge knowledge in intracellular molecular cross-talking of retinoblastoma protein with various cellular viral-like proteins.
Journal of Cancer Research and Therapeutics · 2016 · 3 citations · open access
Identifying key genes in retinoblastoma by comparing classifications of several kinds of significant genes
AbstractOBJECTIVE: The objective of this paper was to investigate key genes in retinoblastoma using a novel method which is mainly based on five kinds of genes, differentially expressed genes (DEGs), differential pathway genes (DPGs), seed genes (common genes between DEGs and DPGs), hub genes and informative genes (common genes of hub genes and DEGs), and support vector machines (SVM) model. MATERIALS AND METHODS: In the proposed method, the first step was to identify five types of significant genes. DEGs were identified using linear models for microarray data (Limma) package (The Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia). DPGs were originated from differential pathways based on attract method. Hub genes of mutual information network which is constructed by the context likelihood of relatedness algorithm were obtained according to topological degree centrality analysis. For the second step, SVM model was implemented to assess the classification performance of DEGs, DPGs, seed genes, hub genes, and informative genes, depending on its induces the area under the receiver operating characteristics curve (AUC), true negative rate (TNR), true positive rate (TPR) and the Matthews coefficient correlation classification (MCC). RESULTS: We detected 479 DEGs, 747 DPGs, 29 seed genes, 34 hub genes, and 7 informative genes in total for retinoblastoma. The classification performance of informative genes was the best of all with AUC = 1.00, TNR = 1.00, TPR = 1.00, and MCC = 1.00, hence they were considered to key genes which included EPARS1, FN1, HLA-DPA1, HLA-DPB1, HLA-DRA, CFI, and transforming growth factor, beta receptor II. CONCLUSIONS: We have successfully identified seven key genes, which might be potential biomarkers for detection and therapy of retinoblastoma for current and future study.
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.
