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This investigational CAR-T cell therapy consists of autologous T cells genetically modified via a retroviral vector to express a chimeric antigen receptor (CAR) targeting the epidermal growth factor receptor variant III (EGFRvIII). EGFRvIII is a tumor-specific neoantigen found in approximately 30-50% of glioblastomas but absent in normal brain tissue, making it a highly specific target for immunotherapy. Developed by researchers including Daniel Landi at Duke University, the therapy was evaluated in a Phase 1 clinical trial for patients with newly diagnosed glioblastoma and gliosarcoma. The treatment protocol involved administering the CAR-T cells during the period of lymphopenia following dose-intensified temozolomide chemotherapy to enhance T-cell expansion and anti-tumor activity. The trial was terminated early due to the conclusion of grant funding.
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