Novel Agents for Acute Myeloid Leukemia

AML is a complex hematological disease characterized by genetic and clinical heterogeneity. Recent advances in the understanding of AML pathogenesis have paved the way for the development of new agents targeting specific molecules or mechanisms that contribute to finally move beyond the current standard of care, “3+7” regimen. In particular, new therapeutic options such as targeted therapies (midostaurin and enasidenib), monoclonal antibodies (gemtuzumab ozogamicin), and a novel liposomal formulation of cytarabine and daunorubicin (CPX-351) have been recently approved and will be soon available for the treatment of adult patients with AML. In this review we will present and describe the novel agents against AML showing the most promising results or that are currently under investigation, as monotherapy or in combination with chemotherapy.