Thrombopoietin-Based CAR-T Cells Demonstrate In Vitro and In Vivo Cytotoxicity to MPL Positive Acute Myelogenous Leukemia and Hematopoietic Stem Cells

CAR T cells were designed using a ligand binding domain instead of a single chain variable fragments to target stem-like leukemia cells. Thrombopoietin, the natural ligand to the myeloproliferative leukemia protein (MPL) receptor, was used as the antigen binding domain to engage MPL expressed on HSCs and erythropoietic and megakaryocytic acute myeloid leukemias.