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PR3-specific cytotoxic T lymphocytes is an investigational adoptive cellular immunotherapy developed by the Fred Hutchinson Cancer Center for the treatment of high-risk myeloid malignancies. The therapy consists of donor-derived CD8+ cytotoxic T lymphocytes (CTLs) that are ex vivo expanded to specifically target the PR1 peptide, an epitope derived from the serine protease proteinase 3 (PR3, also known as myeloblastin). PR3 is frequently overexpressed in the cells of patients with acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndromes (MDS). In the clinical setting, these HLA-A2-restricted CTL clones are administered intravenously to patients who have relapsed or progressed after allogeneic hematopoietic stem cell transplantation. The treatment protocol typically includes the co-administration of subcutaneous aldesleukin (interleukin-2) to enhance the expansion, persistence, and anti-leukemic activity of the infused T cells.
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