Acute Myeloid Leukemia Stem Cells: Origin, Characteristics, and Clinical Implications

The stem cells of acute myeloid leukemia (AML) are the malignancy initiating cells whose survival ultimately drives growth of these lethal diseases. Here we review leukemia stem cell (LSC) biology, particularly as it relates to the very heterogeneous nature of AML and to its high disease relapse rate. Leukemia ontogeny is presented, and the defining functional and phenotypic features of LSCs are explored. Surface and metabolic phenotypes of these cells are described, particularly those that allow distinction from features of normal hematopoietic stem cells (HSCs). Opportunities for use of this information for improving therapy for this challenging group of diseases is highlighted, and we explore the clinical needs which may be addressed by emerging LSC data. Finally, we discuss current gaps in the scientific understanding of LSCs.

Automated summary

This article reviews the biology of leukemia stem cells (LSCs) in acute myeloid leukemia (AML), focusing on their relationship with the heterogeneous nature of AML and its high relapse rate. The defining functional and phenotypic features of LSCs are discussed, including their surface and metabolic phenotypes that differentiate them from normal hematopoietic stem cells (HSCs). The potential for using this information to improve therapy and address clinical needs is highlighted, as well as the current gaps in the scientific understanding of LSCs.