In 2011, first promising results of a small phase I study with the SMO inhibitor PF-04449913 in hematologic malignancies including AML were published [39] and two studies with PF-04449913 in patients with AML and myelodysplastic syndrome (MDS) or acute leukemia, respectively, are currently recruiting participants (NCT01546038, NCT01841333). This evidence concerns the gene SMO and acute myeloid leukemia.