Acute myeloid leukemia (AML) is a heterogeneous, complex, and deadly disease, whose treatment has hardly evolved for decades and grounds on the use of intensive chemotherapy regimens. Chemotherapy helps reduce AML bulk, but promotes relapse in the long-run by selection of chemoresistant leukemia stem cells (LSC). These may diversify and result in progression to more aggressive forms of AML. In vivo models suggest that the bone marrow stem cell niche helps LSC stay dormant and protected from chemotherapy. Here, we summarize relevant changes in stem cell niche homing and adhesion of AML LSC vs. healthy hematopoietic stem cells, and provide an overview of clinical trials aiming at targeting these processes for AML treatment and future directions within this field. Promising results with various non-mutation-targeted novel therapies directed to LSC eradication via interference with their anchoring to the stem cell niche have encouraged on-going or future advanced phase III clinical trials. In the coming years, we may see a shift in the focus of AML treatment to LSC-directed therapies if the prospect of improved cure rates holds true. In the future, AML treatment should lean toward personalized therapies using combinations of these compounds plus mutation-targeted agents and/or targeted delivery of chemotherapy, aiming at LSC eradication with reduced side effects.

急性髓细胞性白血病（AML）是一种异质，复杂且致命的疾病，几十年来其治疗方法几乎没有发展，其原因是采用了强力化疗方案。化学疗法有助于减少AML体积，但通过选择化学抗性白血病干细胞（LSC）长期促进复发。这些可能会多样化并导致发展为更具攻击性的AML形式。体内模型表明，骨髓干细胞的生态位有助于LSC保持休眠状态并免受化学疗法的侵害。在这里，我们总结了AML LSC与健康造血干细胞在干细胞生态位归巢和粘附方面的相关变化，并概述了旨在针对这些过程进行AML治疗的临床试验以及该领域的未来方向。各种针对非LSC的非突变靶向新疗法的成功前景令人鼓舞，这些疗法通过干扰其在干细胞生态位上的锚定而得以根除。这鼓励了正在进行的或未来的高级III期临床试验。在未来几年中，如果提高治愈率的前景成立，我们可能会看到AML治疗的重点转移到LSC导向的治疗上。在将来，