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WT1 peptide vaccine + PR1 peptide vaccine is an investigational combination immunotherapy developed by the National Heart, Lung, and Blood Institute (NHLBI) for the treatment of myeloid malignancies, including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS). The vaccine consists of two distinct peptide components: a Wilms Tumor 1 (WT1) peptide and a PR1 peptide (derived from proteinase 3 and neutrophil elastase). These peptides are designed to be presented by HLA-A2 molecules to stimulate a cytotoxic T-lymphocyte response against leukemia cells overexpressing these antigens. The vaccine is typically co-administered with the adjuvant Montanide ISA-51 and the cytokine GM-CSF (sargramostim) to enhance the magnitude and duration of the immune response. Clinical evaluation has primarily focused on safety and the induction of antigen-specific T-cell responses in patients with minimal residual disease or high-risk myeloid cancers.
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