Recent advances in cell therapy for AML/MDS

Cellular immunotherapy has substantially changed the treatment of B-cell leukemias, lymphomas, and multiple myeloma. The success of chimeric antigen receptor (CAR)-T cell therapy in these diseases has been supported by suitable target antigens, including CD19 in B-cell malignancies and B-cell maturation antigen in multiple myeloma. In contrast, comparable progress has not yet been achieved in acute myeloid leukemia acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML) and myelodysplastic syndromes myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS), in part because an optimal target with clinically manageable on-target effects on normal cells has not been identified. Nevertheless, identification of an appropriate target could enable major therapeutic advances with CAR-T cell therapy in myeloid malignancies. To further harness the potential of cellular immunotherapy in AML/MDS, a multifaceted approach may be required. In parallel with CAR-T cell development, CAR-NK cells, T-cell receptor-engineered T cells, NK cells and other innate immune approaches are actively explored, while allogeneic hematopoietic stem cell transplantation remains an established form of cellular immunotherapy for AML/MDS. These approaches may be developed in parallel and, where appropriate, integrated with molecularly targeted and other pharmacologic therapies. This issue of Progress in Hematology reviews these emerging directions and their potential to expand cellular therapy for AML/MDS.