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    CANCER:

    Ependymoma

    Related cancers:
    2d
    Molecular Biology and Phase 1 Study of GM-CSF and Intrathecal Trastuzumab in Children with Recurrent Posterior Fossa Ependymoma. (PubMed, Neuro Oncol)
    IT trastuzumab in combination with GM-CSF is safe in recurrent pediatric PF EPN. A larger phase 2 study that includes both recurrent PFA and RELA-ST EPN is needed to determine the long-term efficacy of this immunotherapy strategy.
    P1 data • Journal • IO biomarker
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    HER-2 (Human epidermal growth factor receptor 2) • CSF2 (Colony stimulating factor 2) • RELA (RELA Proto-Oncogene)
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    Herceptin (trastuzumab)
    6d
    Cauda equina neuroendocrine tumors: An 11-Year retrospective report on clinicopathological characteristics and postoperative recurrence. (PubMed, Neurochirurgie)
    CENETS are rare and often misdiagnosed. Serpentine dilated vessel identification at the tumor's cephalad part during surgery may be indicative, and gross total resection be curative.
    Retrospective data • Journal
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    GATA3 (GATA binding protein 3) • SYP (Synaptophysin) • GFAP (Glial Fibrillary Acidic Protein) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    9d
    Dominant clones leverage developmental epigenomic states to drive ependymoma. (PubMed, Nature)
    In vivo lineage tracing studies identified neoplastic clones that aggressively dominated tumour growth and established the entire EPN cellular hierarchy. These findings identify developmental epigenomic states that are critical for fusion-oncoprotein-driven transformation and show how these states continue to shape tumour progression.
    Journal
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    RELA (RELA Proto-Oncogene) • ZFTA (Zinc Finger Translocation Associated)
    9d
    PED-DNX2401: Intratumoral DNX-2401 for High Grade Pediatric Brain Tumors (clinicaltrials.gov)
    P2, N=39, Not yet recruiting, Clinica Universidad de Navarra, Universidad de Navarra | N=20 --> 39
    Enrollment change
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    tasadenoturev (DNX-2401)
    10d
    Discovery of genetic susceptibility variants in pediatric and adult ependymoma. (PubMed, Neurooncol Adv)
    Several significant intronic variants were harbored in genes associated with cancer and neurological disease. Future studies are needed to investigate the role of these age-specific alterations in ependymoma pathogenesis.
    Journal
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    EDIL3 (EGF Like Repeats And Discoidin Domains 3)
    18d
    PNOC027: Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma (clinicaltrials.gov)
    P=N/A, N=74, Recruiting, University of California, San Francisco | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial primary completion date
    21d
    PNOC041: Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma (clinicaltrials.gov)
    P2, N=30, Recruiting, University of California, San Francisco | Not yet recruiting --> Recruiting
    Enrollment open
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    metformin
    22d
    High frequency of mosaic NF2-related schwannomatosis diagnosed after somatic analysis of multi-nodular schwannomas in schwannomatosis patients. (PubMed, Hum Genet)
    This study illustrates the underestimated high frequency of mosaic NF2-related schwannomatosis in patients harboring multi-nodular peripheral nerve schwannomas. As multi-nodular schwannomas are associated with higher surgical morbidity, the pivotal role of the NF2 gene in their tumorigenesis opens perspectives for Schwannomatosis research.
    Journal
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    NF2 (Neurofibromin 2) • SMARCB1 (SWI/SNF Related, Matrix Associated, Actin Dependent Regulator Of Chromatin, Subfamily B, Member 1) • LZTR1 (Leucine Zipper Like Transcription Regulator 1)
    23d
    INTRATUMORAL DNX-2401 ADMINISTRATION FOR RECURRENT AND REFRACTORY HIGH GRADE BRAIN TUMORS IN PEDIATRIC AND YOUNG ADULT PATIENTS (2024-515009-24-00)
    P1/2, N=20, Not yet recruiting, Clinica Universidad De Navarra
    New P1/2 trial
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    tasadenoturev (DNX-2401)
    24d
    Adnexal Endometrioid Carcinomas With Sex Cord-Like Morphology are Frequently PAX8-Negative, SOX17-Positive, and Enriched for CTNNB1 Alterations. (PubMed, Am J Surg Pathol)
    Most of these tumors are classified in the "no specific molecular profile" subgroup and have high rates of nuclear beta-catenin/CTNNB1 alterations. Awareness of these morphologic and immunohistochemical associations may help avoid misclassification as sex cord-stromal or other neoplasms.
    Journal
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    TP53 (Tumor protein P53) • CTNNB1 (Catenin (cadherin-associated protein), beta 1) • SOX17 (SRY-Box Transcription Factor 17) • PAX8 (Paired box 8)
    24d
    PRiME II: PEP-CMV + Nivolumab for Newly Diagnosed Diffuse Midline Glioma/High-grade Glioma and Recurrent Diffuse Midline Glioma/High-grade Glioma, Medulloblastoma, and Ependymoma (clinicaltrials.gov)
    P1/2, N=68, Not yet recruiting, Washington University School of Medicine | Trial primary completion date: Mar 2031 --> Jun 2031
    Trial primary completion date
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    Opdivo (nivolumab) • temozolomide
    1m
    A Study of the Treatment of Recurrent Malignant Glioma With rQNestin34.5v.2 (clinicaltrials.gov)
    P1, N=62, Active, not recruiting, Dana-Farber Cancer Institute | Recruiting --> Active, not recruiting | Trial completion date: Jan 2027 --> Jan 2028 | Trial primary completion date: Jan 2026 --> Jan 2027
    Enrollment closed • Trial completion date • Trial primary completion date
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
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    IDH1 mutation • IDH wild-type • IDH1 R132
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    cyclophosphamide • linoserpaturev (CAN-3110)