Today, treatment of patients with acute myeloid leukemia (AML) remains predominantly a "one-fits-all" approach with intensive cytarabine-based chemotherapy as the mainstay, but we are finally beginning to reap the rewards of decades of basic, translational, and clinical research. Developing individualized, "targeted" therapy for each AML patient based on unique molecular features of disease remains a daunting goal, yet one that we can now begin to envision. Hypothesis-based study designs--from preclinical/laboratory experiments to phase 1 and subsequent efficacy trials--provide the foundation for advances in the diagnosis, risk stratification, and treatment of patients with AML. The following is an outline of several key areas of ongoing AML research.