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The WT1 + PR1 peptide vaccine is an investigational therapeutic cancer vaccine composed of synthetic peptides derived from the Wilms Tumor 1 (WT1) protein and the proteinase 3 (PR3, source of the PR1 epitope). Both antigens are overexpressed in myeloid malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The combination aims to induce a cytotoxic T lymphocyte response against cells expressing these tumor-associated antigens. Clinical studies have shown that vaccination with both peptides can elicit specific CD8+ T cell responses targeting leukemic cells. The mechanism involves presentation of these peptides by HLA-A*0201 molecules on antigen-presenting cells, leading to activation and expansion of antigen-specific cytotoxic T lymphocytes that recognize and kill malignant hematopoietic cells. Early-phase clinical trials have demonstrated safety, immunogenicity, and some evidence for anti-leukemia activity in patients with AML or MDS[5][8].
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