Based on these and other pre-clinical observations of a potential role of the HhP in myeloid disease as highlighted below, a clinical phase 1/1b trial of 5-Aza with the smoothened (SMO) inhibitor LDE225 (erismodegib) in AML, MDS, and MPN patients was initiated and is currently accruing patients. Here, SMO is linked to acute myeloid leukemia.