DNA and Histone Methylation in Hematopoietic Malignancy: Biology of Epigenetic Regulators in Hematological Malignancies as Basis of Therapeutic Targets

Recent advances in next-generation sequencing technologies have revealed frequent somatic mutations in hematological malignancies. As the epigenetic landscapes of hematological malignancies are unveiled, epigenetic regulatory processes have become therapeutic targets. After the success of hypomethylating agents, azacitidine and decitabine for myelodysplastic syndrome (both FDA-approved), inhibitors of other epigenetic regulators have become candidate agents for next-generation therapy against refractory hematological malignancies. In addition to tyrosine kinase inhibitors and immunotherapeutic agents, inhibitors of epigenetic regulators have been used either in a salvage monotherapy regimen or in combination therapy with conventional chemotherapy. In this chapter, alterations in DNA cytosine methylation and histone methylation as targets of therapeutics for hematological malignancies are discussed with outlines of clinical trials.