DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for anaplastic ependymoma — screening already-approved drugs against its 32-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAnaplastic ependymoma maps to a 32-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 anaplastic 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
kelch like ECH associated protein 1 (KEAP1) — KEAP1 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 2r,3sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8IXS · 1.48 Å · ligand (2R,3S)-3-[[(2S)-2-fluoranyl-2-(5,6,7,8-tetrahydronaphthalen-2-yl)ethanoyl]amino]-2-methyl-3-(4-methylphenyl)propanoic acid (T6I). Experimental structure, not a prediction.
What the evidence adds up to
In 53 paediatric infratentorial ependymomas, higher than median expression of miR-17-5p was associated with reduced overall survival (p = 0.036) and event-free survival (p = 0.002). Multivariate analysis adjusted for age, sex, tumour grade and location confirmed a statistically significant association with event-free survival (p = 0.004) and a borderline association with overall survival (p = 0.057). Lower LAMA2 mRNA expression was linked to increased risk of death. Three miRNAs (miR-17-5p, miR-19a-3p, miR-106b-5p) differentiated WHO grade II from grade III tumours. These are correlative findings, not a treatment.
Nine children under three years old with anaplastic ependymoma received intensity-modulated radiotherapy (IMRT) at a median dose of 52.0 Gy. Five-year overall survival was 40.6%, freedom from local recurrence was 53.3%, and progression-free survival was 26.7%. Five of nine patients recurred, and five died from disease progression. Motor dysfunction occurred in three patients, all of whom had hydrocephalus and neurological deficits before radiotherapy. The authors state that neurologic morbidity was not caused by radiotherapy alone.
Eight patients with refractory anaplastic ependymoma (median age 36 years) were treated with metronomic cyclophosphamide, cisplatin, and bevacizumab. Median overall survival was 19.9 months, median progression-free survival was 12.3 months. Three patients achieved a partial response, four had stable disease, and one progressed during induction. The authors claim the clinical benefit appears superior to that described in the literature, but the sample is small and uncontrolled.
A single twelve-year-old girl with WHO grade III anaplastic ependymoma and CDK4 amplification was treated with the CDK4/6 inhibitor dalpiciclib plus bevacizumab after relapse following surgery, radiotherapy, and chemotherapy. She survived for over 48 months after surgery. This is one case report; no controlled data exist. No other abstracts describe a drug treatment for anaplastic ependymoma.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
PLoS ONE · 2016 · 29 citations · open access
Altered MicroRNA Expression Is Associated with Tumor Grade, Molecular Background and Outcome in Childhood Infratentorial Ependymoma
AbstractBACKGROUND: Ependymal tumors are the third most common group of brain tumors in children, accounting for about 10% of all primary brain neoplasms. According to the current WHO classification, they comprise four entities with the most frequent ependymoma and anaplastic ependymoma. The most of pediatric tumors are located within the posterior fossa, with a tendency to infiltrate the vital brain structures. This limits surgical resection and poses a considerable clinical problem. Moreover, there are no appropriate outcome prognostic factors besides the extent of surgical resection. Despite definition of molecular subgroups, the majority of childhood ependymomas present a balanced genome, which makes it difficult to establish molecular prognostic factors. METHODS: The purpose of our study was to explore whether miRNA expression could be used as prognostic markers in pediatric infratentorial ependymomas. We also performed a mRNA expression pattern analysis of NELL2 and LAMA2 genes, with immunohistochemical illustrations of representative cases. The miRNA and mRNA expression was measured in 53 pediatric infratentorial ependymomas using a real-time quantitative PCR. RESULTS: Three miRNAs were shown to efficiently differentiate between grade II and III ependymomas: miR-17-5p, miR-19a-3p, and miR-106b-5p. Survival analysis showed that the probabilities of overall (p = 0.036) and event-free survival (p = 0.002) were reduced with higher than median miRNA expression levels of miR-17-5p. Using multivariate analysis adjusted for patient's age, sex, tumor grade and localization, we showed statistically significant associations with event-free survival (p = 0004) and borderline statistical significance with overall survival (p = 0.057) for miR-17-5p. Correlation analysis of miR-19a, miR-17-5p, miR-106b revealed that their expression levels were significantly correlated with EZH2 expression, suggested marker of PFA ependymomas. Furthermore, lower expression level of LAMA2 mRNA was shown to be associated with an increased risk of death in covariate-adjusted analyses. CONCLUSIONS: Our data provide a better understanding of pediatric ependymoma and suggests the presence of plausible molecular biomarkers connected with the outcome.
Treatment outcome of anaplastic ependymoma under the age of 3 treated by intensity-modulated radiotherapy
AbstractPURPOSE: Intensity-modulated radiotherapy (IMRT) allows for more precise treatment, reducing unwanted radiation to nearby structures. We investigated the safety and feasibility of IMRT for anaplastic ependymoma patients below 3 years of age. MATERIALS AND METHODS: A total of 9 anaplastic ependymoma patients below 3 years of age, who received IMRT between October 2011 and December 2017 were retrospectively reviewed. The median equivalent dose in 2 Gy fractions was 52.0 Gy (range, 48.0 to 60.0 Gy). Treatment outcomes and neurologic morbidities were reviewed in detail. RESULTS: The median patient age was 20.9 months (range, 12.1 to 31.2 months). All patients underwent surgery. The rates of 5-year overall survival, freedom from local recurrence, and progression-free survival were 40.6%, 53.3%, and 26.7%, respectively. Of the 9 patients, 5 experienced recurrences (3 had local recurrence, 1 had both local recurrence and cerebrospinal fluid [CSF] seeding, and 1 had CSF seeding alone). Five patients died because of disease progression. Assessment of neurologic morbidity revealed motor dysfunction in 3 patients, all of whom presented with hydrocephalus at initial diagnosis because of the location of the tumor and already had neurologic deficits before radiotherapy (RT). CONCLUSION: Neurologic morbidity is not caused by RT alone but may result from mass effects of the tumor and surgical sequelae. Administration of IMRT to anaplastic ependymoma patients below 3 years of age yielded encouraging local control and tolerable morbidities. High-precision modern RT such as IMRT can be considered for very young patients with anaplastic ependymoma.
Metronomic Cyclophosphamide With Cisplatin and Bevacizumab for Refractory Anaplastic Ependymoma: A Retrospective Study
AbstractBACKGROUND/AIM: Anaplastic ependymoma is a rare cancer of the central nervous system. The treatment includes optimal resection with focal radiotherapy. Some case reports or retrospective studies have suggested efficacy of regimens containing platinum or bevacizumab. We describe the feasibility and clinical benefit of the cisplatin-bevacizumab-cyclophosphamide treatment of anaplastic ependymoma. PATIENTS AND METHODS: Patients were identified through the Adolescent and Young Adults (AYAS) brain tumor national Web conference. We estimated the median progression-free (PFS) and overall survival (OS). RESULTS: There were eight patients with anaplastic ependymoma, with a median age of 36 years. The median OS was 19.9 months and median PFS was 12.3 months. Three patients obtained partial response, four stable disease, and one patient had disease progression during induction. Six patients received maintenance with a median duration of 224 days. CONCLUSION: This study confirms the tolerance of bevacizumab-cyclophosphamide-cisplatin treatment of anaplastic ependymoma. The clinical benefit seems even superior to that described in the literature.
Recent Patents on Anti-Cancer Drug Discovery · 2025 · 0 citations
Targeted Therapy with Dalpiciclib in a Pediatric Patient with Anaplastic Ependymoma: A Case Study and Literature Review
AbstractINTRODUCTION: Ependymoma is the third most common brain tumor in children, with a high recurrence rate and poor prognosis. The traditional treatment of ependymoma is surgery and radiation therapy. However, the effectiveness of conventional chemotherapy has been modest. Recent breakthroughs in molecular biology and genetics have opened doors to more targeted and effective therapeutic approaches. Complex mutations, such as CDK4 amplification in anaplastic ependymoma, are infrequently documented, warranting further investigation. CASE PRESENTATIONS: This case study presents a twelve-year-old girl with a WHO Grade III anaplastic ependymoma. She underwent two surgical procedures, followed by radiotherapy and chemotherapy. Despite this comprehensive treatment, she experienced a relapse after one year. Genetic testing identified CDK4 amplification in the tumor tissue. Subsequently, based on the genetic finding, she was treated with a CDK4/6 inhibitor, Dalpiciclib, in combination with bevacizumab. So far, she has survived for over 48 months following her surgery and continues to be monitored, underscoring the remarkable efficacy of the treatment. CONCLUSIONS: This report represents the first use of the CDK4/6 inhibitor Dalpiciclib for the treatment of anaplastic ependymoma. The treatment was guided by insights from next-generation sequencing (NGS) analysis and has shown promising results in terms of patient survival.
Additional file 8: of Significance of molecular classification of ependymomas: C11orf95-RELA fusion-negative supratentorial ependymomas are a heterogeneous group of tumors
AbstractFigure S7. Histological features in RELA-negative/YAP1-negative supratentorial ependymoma cases. EP3 (EP300-BCORL1 fusion-positive) exhibits typical findings of anaplastic ependymoma, including hypercellularity, perivascular pseudorosettes (a), calcification (arrows, b) and high MIB-1 labeling index (c). In EP57 (FOXO1-STK24 fusion-positive), perivascular pseudorosettes (d), calcification (arrows, e), microcyst formation (e), vacuolated cells (f), GFAP-positive cells (g), EMA positive reaction (h) and low MIB-1 labeling index (i) were observed. (TIF 58932 kb)
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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