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EGFRvIII CAR-T is a chimeric antigen receptor T cell therapy engineered to target the EGFR variant III (EGFRvIII), a tumor-specific mutation of the epidermal growth factor receptor found in various cancers—most notably glioblastoma. The therapy involves collecting T cells from patients (autologous) or donors (allogeneic), genetically modifying them ex vivo to express a CAR that recognizes EGFRvIII, and then infusing them back into the patient. Upon administration, these modified T cells bind specifically to EGFRvIII-expressing tumor cells and mediate their destruction through cytotoxic immune mechanisms. This approach aims for high specificity since EGFRvIII is absent in normal tissues but overexpressed in certain tumors. Preclinical and early clinical studies have shown promising antitumor activity with limited off-target toxicity[2][4][5]. Variants of this therapy include additional genetic modifications such as co-expression of cytokines like IL-15 and chemokines like CCL19 to enhance persistence and efficacy[8]. The primary indication under investigation is glioblastoma.
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