Relapse is common in acute myeloid leukemia (AML) and thought to be due to resistance of underlying leukemic stem cells (LSCs) to current standard therapies, although a lack of tools to measure the quantity and quality of these cells in patients precludes the clinical testing of this concept. This review discusses the current knowledge of LSC properties and appraises strategies aimed to bring the therapeutic targeting of LSCs to the bedside to improve patient outcomes. We highlight pathways and targets of interest and summarize available information on drugs that might eradicate LSCs. Future research is needed to close identified gaps in knowledge and provide evidence for the clinical efficacy of LSC-directed therapies to support the development of treatments that eliminate residual disease and prevent relapse, thereby increasing the cure rates of patients with AML.
Keywords: Acute myeloid leukemia; Leukemic stem cells; Measurable residual disease.
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