Targeted agents in AML: much more to do

To what degree has targeted therapy succeeded in acute myeloid leukemia (AML)? Targeted therapy has become a buzzword, with its meaning lost from overuse. In chronic myeloid leukemia (CML), gastrointestinal stromal cell tumor, and a small subset of patients with non-small cell lung cancer, a validated target has been identified and a highly specific therapeutic agent has been developed. Targeted therapy generally requires a pathophysiological Achilles heel in a tumor that can be exploited by nontoxic therapy. In most cases, the validated target has been a tyrosine kinase enzyme critical for tumor growth and survival. Are similar “drugable” targets available in AML? While our understanding of the pathophysiology of AML has advanced over the past decade, and some potential targets have been identified, no single agent will likely produce a significant proportion of remissions. On the other hand, nascent attempts with mild success have been achieved, yielding hope that this strategy will bear real fruit in the future.