Myelodysplastic syndromes - The epigenetic model for drug development?

Application of sequencing technology has advanced our understanding of the molecular landscape of myelodysplastic syndromes (MDS). Recurrent driver mutations in genes implicated in epigenetic regulation, and functional modelling of the disease has proven the importance of epigenetic dysregulation in MDS pathogenesis. Although available therapies such as azacitidine and decitabine are thought to exert their effect by epigenetic modulation, deep understanding of disease biology and development of specific epigenetically targeted therapies is still an area under active research. In this editorial we will focus on the molecular basis of MDS with particular focus on epigenetic dysregulation and new agents under development targeting this group of biological processes.