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EGFRvIII-CAR is an investigational chimeric antigen receptor (CAR) T-cell therapy designed to target the epidermal growth factor receptor variant III (EGFRvIII), a tumor-specific mutation frequently expressed in glioblastoma multiforme (GBM) but absent in normal tissues. The therapy involves engineering a patient's own T cells to express a synthetic receptor consisting of an anti-EGFRvIII single-chain variable fragment (scFv) linked to intracellular signaling domains, typically CD3-zeta and a co-stimulatory domain such as 4-1BB or CD28. Developed primarily by Kite Pharma in collaboration with academic institutions including the University of Pennsylvania and UCSF, it aims to provide a targeted immune response against aggressive brain tumors. Clinical development has reached Phase 2 for GBM, although challenges such as antigen heterogeneity and the immunosuppressive tumor microenvironment remain significant hurdles in its therapeutic efficacy.
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