One inhibitor in particular, EPZ-5676, has shown potent activity in its ability to selectively inhibit the DOT1L histone methyltransferase, resulting in cell death of acute leukemia cell lines harboring MLL translocations as well as complete tumor regression in a rat xenograft model of MLL-rearranged leukemia following continuous iv infusion of EPZ-5676 (45). This evidence concerns the gene DOT1L and leukemia.