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EGFRvIII CAR-PBL is an investigational autologous chimeric antigen receptor (CAR) T-cell therapy developed by the National Cancer Institute (NCI) Surgery Branch. It consists of a patient's own peripheral blood lymphocytes (PBLs) that are genetically engineered ex vivo using a retroviral vector to express a CAR specific for the EGFRvIII mutation. EGFRvIII is a tumor-specific variant of the epidermal growth factor receptor frequently expressed in malignant gliomas but absent in normal tissues, making it an ideal target for immunotherapy. The CAR construct includes a signaling domain (typically CD28 and CD3-zeta) to trigger T-cell activation upon binding to the target antigen. This therapy has been evaluated in Phase I/II clinical trials for patients with EGFRvIII-positive glioblastoma and gliosarcoma, administered intravenously following lymphodepleting chemotherapy and alongside aldesleukin (IL-2).
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