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This investigational combination therapy, developed by The Second Hospital of Shandong University, consists of allogeneic natural killer (NK) cell transfusions administered in conjunction with the hypomethylating agent azacitidine. It is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with refractory myelodysplastic syndrome with excess blasts (MDS-EB). The therapeutic approach involves a conditioning regimen of cyclophosphamide followed by azacitidine and the infusion of donor-derived NK cells. NK cells are innate immune cells that can recognize and eliminate malignant cells through various activating receptors, while azacitidine functions as a DNA methyltransferase inhibitor, potentially sensitizing the blasts to immune-mediated destruction and reducing the leukemic burden.
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