Acute myelogenous and chronic myelogenous leukemia are uniformly fatal if left untreated. Treatment with conventional chemotherapeutic agents results in a cure for a small percentage of patients and substantial toxicities in all patients that receive treatment. The first of a new generation of “targeted therapies” has recently been introduced. It is hoped that these agents will demonstrate enhanced efficacy and a decreased toxicity profile. Gemtuzumab ozogamicin (Mylotarg™) is the first antibody-targeted therapy approved for use in patients with acute myelogeneous leukemia, arsenic trioxide (Trisenox®) has demonstrated efficacy in patients with acute promyelocytic leukemia, and imatinib mesylate (Gleevec™) has proven efficacy in patients with chronic myelogenous leukemia. Initially, these agents were granted FDA approval for use in those who were refractory to or intolerant of conventional treatment options. Research is ongoing to further define the full therapeutic potential of these agents and to discover additional targeted therapies with increased efficacy.
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