TIM-3 Is a Novel Therapeutic Target for Eradicating Acute Myelogenous Leukemia Stem Cells

Acute myelogenous leukemia (AML) is derived from self-renewing leukemic stem cells (LSCs). We found that T-cell immunoglobulin mucin-3 (TIM-3) is expressed on LSCs in most types of primary AML, except for acute promyelocytic leukemia (M3 by the FAB classification). TIM-3 is not expressed in normal hematopoietic stem cells (HSCs). In a xenogeneic transplantation system, we showed that targeting of TIM-3 by an anti-TIM-3 cytotoxic antibody is sufficient to eradicate human AML LSCs without affecting normal human hematopoiesis. These data strongly suggest that TIM-3 is a promising therapeutic target to cure AML patients.