DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for ependymoma — 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 moduleEpendymoma 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
No approved-drug candidate for ependymoma 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
tripartite motif containing 24 (TRIM24) — TRIM24 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 benzyloxydrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4YBM · 1.46 Å · ligand N-{6-[3-(benzyloxy)phenoxy]-1,3-dimethyl-2-oxo-2,3-dihydro-1H-benzimidazol-5-yl}-3,4-dimethoxybenzenesulfonamide (4BJ). Experimental structure, not a prediction.
What the evidence adds up to
For adult ependymoma the optimum therapeutic management remains undefined because of the low incidence of the disease and because most reported series are retrospective, include small numbers of patients, and span several decades. A 2002 review noted that important therapeutic issues such as irradiation dose and volume were still being analysed, and that the main open questions concerned the translation of preclinical findings into clinical trials.
By 2012, comprehensive work had revealed molecular pathomechanisms of ependymoma, and clearly distinguishable subgroups with distinct biology had been delineated. Novel mouse models had been generated, and the first high-throughput drug screens had been conducted, leading to the identification of subgroup-specific active regimens. The next step was described as the translation of these preclinical findings into the clinical setting, with international study groups starting to implement the most recent advances into clinical trials.
A retrospective series of 33 patients treated between 1997 and 2013 included 25 WHO grade II ependymomas and eight WHO grade III anaplastic ependymomas. Six patients were paediatric (mean age 6.15 years, range 1.3–11 years) and 27 were adults (mean age 47.5 years, range 19–70 years). Among 12 adult patients with totally resected ependymomas without anaplastic pathology who received no adjuvant treatment, only one showed tumour recurrence during a mean follow-up of 93.5 months (range 27.9–162.7 months). The authors concluded that adult patients with ependymomas had better survival rates compared to paediatric patients, and suggested that totally resected adult ependymomas without anaplastic pathology could be observed without any adjuvant treatment, regardless of tumour location.
What is still missing are prospective, adequately powered trials that incorporate the molecular subgrouping now known to distinguish ependymoma biology. The existing evidence rests on small retrospective series spanning many years, and no drug has been shown in a randomised controlled trial to improve outcomes beyond surgery and radiotherapy for any subgroup. Funding for multi-centre, subgroup-stratified trials, and the development of reliable preclinical models that predict human response, remain the critical gaps.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Expert Review of Anticancer Therapy · 2002 · 23 citations
Current management and prognostic factors for adult ependymoma
AbstractThe optimum therapeutic management in adult ependymoma remains undefined because of the low incidence of this disease and because most of reported series mainly refers to childhood, are retrospective, include a small number of patients and span several decades. The purpose of this article is to analyze, discuss and summarize the current available information regarding the therapeutic approach and the prognostic factors and to provide recommendations for ordinary clinical practice. Some important therapeutic issues like the irradiation dose and volume are also analyzed. Finally, the main open questions as well as current and expected investigation trends are discussed.
Current Opinion in Neurology · 2012 · 23 citations
Molecular approaches to ependymoma
AbstractPURPOSE OF REVIEW: Effective treatment options for ependymoma apart from radical surgery and radiotherapy remain scarce, and the understanding of the molecular basis of ependymoma biology is crucial to the development of novel therapies. Comprehensive work revealing molecular pathomechanisms of ependymoma has been done; however, the elucidation of the processes underlying the origins of various clearly distinguishable ependymoma subgroups has proved to be difficult. The future challenges will be to reach consensus about molecular subgroups, to translate these into a clinical setting, and to use available models for drug screening and preclinical testing. RECENT FINDINGS: Ependymoma subgroups with clearly distinct biology have been delineated and novel mouse models generated, and the first high-throughput drug screens were successfully conducted leading to the identification of subgroup-specific active regimens. SUMMARY: Coordinated efforts to advance novel therapies into the clinic have led to breakthrough insights into the molecular biology of ependymoma. The next step will be the translation of preclinical findings into the clinical setting, and international study groups are starting to implement the most recent advances into clinical trials.
Brain Tumor Research and Treatment · 2017 · 21 citations · open access
Ependymomas: Prognostic Factors and Outcome Analysis in a Retrospective Series of 33 Patients
AbstractBACKGROUND: The purpose of this study was to evaluate the prognostic factors and outcomes in patients with ependymoma to management plans. METHODS: Between 1997 and 2013, 33 patients with 25 ependymomas (WHO grade II) and eight anaplastic ependymomas (WHO grade III) were pathologically diagnosed. Six were pediatric patients (mean age, 6.15 years; range, 1.3-11 years), while 27 were adults (mean age, 47.5 years; range, 19-70 years). Of those, there were 12 adult patients with totally resected ependymomas without anaplastic pathology and adjuvant treatment. Prognostic factors were assessed in ependymoma patients. Prognostic factors were studied using Kaplan-Meier estimates in subgroups. RESULTS: =0.06). Out of 12 patients with totally resected ependymomas without anaplastic pathology and adjuvant treatment, one patient showed tumor recurrence during follow-up (mean, 93.5 months; range, 27.9-162.7 months). CONCLUSION: Adult patients with ependymomas were found to have better survival rates compared to pediatric patients. We suggest that totally resected adult ependymomas without anaplastic pathology could be observed without any adjuvant treatment, regardless of the tumor location.
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.
