Emerging drugs for the treatment of myelodysplastic syndrome

Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal haematopoietic stem cell disorders characterised by ineffective haematopoiesis and an increased risk of developing acute myeloid leukaemia. At present, the only curative option is allogeneic stem cell transplantation. However, the majority of patients are not eligible for this therapy, due to excessive treatment-related morbidity and mortality or lack of a suitable donor. As a result, the need for alternative therapies is great. Our improved understanding of the molecular pathogenesis of MDS has resulted in several new promising therapeutic agents. This review will consider the rational development of new agents based on the molecular biology of MDS.