High relapse rate of acute myeloid leukemia (AML) is still a crucial problem despite considerable advances in anti-cancer therapies. One crucial cause of relapse is the existence of leukemia stem cells (LSCs) with self-renewal ability, which contribute to repeated treatment resistance and recurrence. Treatments targeting LSCs, especially in combination with existing chemotherapy regimens or hematopoietic stem cell transplantation might help achieve a higher complete remission rate and improve overall survival. Many novel agents of different therapeutic strategies that aim to modulate LSCs self-renewal, proliferation, apoptosis, and differentiation are under investigation. In this review, we summarize the latest advances of different therapies in development based on the biological characteristics of LSCs, with particular attention on natural products, synthetic compounds, antibody therapies, and adoptive cell therapies that promote the LSC eradication. We also explore the causes of AML recurrence and proposed potential strategies with new dimensions for targeting LSCs in the future.

尽管抗癌治疗取得了重大进展，但急性髓细胞性白血病（AML）的高复发率仍然是一个关键问题。复发的一个重要原因是存在具有自我更新能力的白血病干细胞（LSC），这有助于反复治疗耐药性和复发。针对LSC的治疗，尤其是与现有的化疗方案或造血干细胞移植相结合，可能有助于实现更高的完全缓解率并改善总体生存率。旨在调节LSC自我更新，增殖，凋亡和分化的许多不同治疗策略的新型药物正在研究中。在这篇综述中，我们根据LSC的生物学特性总结了不同疗法在开发中的最新进展，特别关注天然产物，合成化合物，抗体疗法和可促进LSC根除的过继性细胞疗法。我们还探讨了AML复发的原因，并提出了针对未来LSCs的具有新维度的潜在策略。