New therapeutics for myelodysplastic syndromes

While MDS was only recently viewed as an orphan disease without any FDA approved therapeutic options, the landscape has changed dramatically with a promise for development of exciting new therapeutics that parallels our growing understanding of the pathobiology of the disease. An array of new agents is entering clinical development, many of which were not discussed in this review. Nevertheless, our paradigm for the approach to treatment of MDS can be expected to evolve with our ever expanding insight into the disease biology, targeting not only the MDS clone, but also the surrounding microenvironment while at the same time considering the context of the dynamics of disease pathogenesis.