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PXFK peptide is a tumor-specific neoantigen vaccine component developed by the National Cancer Institute (NCI) for the treatment of alveolar rhabdomyosarcoma (ARMS). It is a synthetic peptide that mimics the unique amino acid sequence at the translocation breakpoint of the PAX3-FKHR (also known as PAX3-FOXO1) fusion protein, which is present in approximately 70-80% of ARMS cases. The peptide is designed to be presented by antigen-presenting cells (APCs) to induce a cytotoxic T-cell response against tumor cells. In clinical research, it is typically administered as part of a multi-peptide vaccination protocol (often alongside EWS-FLI1 peptides for Ewing sarcoma) using autologous APCs and supplemented with cytokines such as IL-2 and GM-CSF to enhance immunogenicity.
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