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.
Stone
Richard M
RM
Adult Leukemia Program, Dana Farber Cancer Institute and Harvard Medical School, 44 Binney Street, D 840 Boston, MA 02115, USA. rstone@partners.org
D000208:Acute Disease; D002986:Clinical Trials as Topic; D004791:Enzyme Inhibitors / Q000627:therapeutic use*; D051231:Farnesyltranstransferase / Q000037:antagonists & inhibitors; D015973:Gene Expression Regulation, Leukemic / Q000187:drug effects; D006801:Humans; D007951:Leukemia, Myeloid / Q000188:drug therapy*; D009857:Oncogenes / Q000187:drug effects*; D014178:Translocation, Genetic / Q000187:drug effects / Q000235:genetics; D051941:fms-Like Tyrosine Kinase 3 / Q000037:antagonists & inhibitors / Q000235:genetics
D004791:Enzyme Inhibitors; D051231:Farnesyltranstransferase; D051941:fms-Like Tyrosine Kinase 3