Introduction: the results of conventional treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) remain poor and innovative strategies are warranted. With this aim, epigenetic modulation became a promising approach over the last years. Vorinostat is an epigenetic targeted drug belonging to the histone deacetylase (HDAC) inhibitors family. It is the second-generation HDAC inhibitor that has been more extensively studied in AML and MDS.
Areas covered: in this review, we will present the rationale for its use in AML and MDS, describe the drug pharmacologic properties and review the current data available from clinical trials. The data presented here will allow the reader to overview the common mechanisms of action of this class of compounds in AML and MDS, as well as to better understand the biological specificities of vorinostat, and its current and future clinical development in the field.
Expert opinion: vorinostat has shown an acceptable toxicity profile with mainly gastrointestinal and constitutional side effects. Efficacy as a single agent is limited in that group of patients, but promising results are currently observed with combinations of vorinostat and either conventional chemotherapy or investigational agents, including demethylating agents.