The possibility of inhibiting multiple key players in this pathway (i.e. SMO, GLI1/GLI2) as well as the combination with other agents targeting important mechanisms involved in AML pathology (e.g. kinase inhibitors and epigenetic regulators such as 5-Aza, HDACi and BRD4) provide a multitude of new treatment options. The gene discussed is BRD4; the disease is acute myeloid leukemia.