The new perspectives of targeted therapy in acute myeloid leukemia.

Acute myeloid leukemia (AML) is a heterogeneous disease and the results of previous treatment with cytotoxic drugs have not been satisfactory. This situation has prompted investigations into novel approaches. The breakthrough in therapy brought by all-trans retinoic acid (ATRA) in acute promyelocytic leukemia (APL) and tyrosine kinase inhibitors in neoplasms with the Philadelphia chromosome has encouraged the search for other effective targeted therapies. Among the tested substances are higher molecular mass drugs such as antibodies and various small molecules: kinase inhibitors, cell pathway inhibitors and epigenetic modulators. So far, the U.S. Food and Drug Administration (FDA) has approved the antibody-drug conjugate gemtuzumab ozogamycin (GO), the tyrosine kinase inhibitor midostaurin and the IDH2 inhibitor enasidenib. These studies have led to a better understanding of the mechanisms of leukemogenesis and may soon allow for differentiating treatments depending on baseline mutational complements. Some innovative drugs described in this article have strong therapeutic potential, but there is still a long way to go before actual success in targeted treatment.