文章：

急性早幼粒细胞白血病治疗发展史及早幼粒细胞白血病小体的作用

History of Developing Acute Promyelocytic Leukemia Treatment and Role of Promyelocytic Leukemia Bodies

原文发布日期：29 March 2024

DOI: 10.3390/cancers16071351

类型: Article

开放获取: 是

英文摘要：

The story of acute promyelocytic leukemia (APL) discovery, physiopathology, and treatment is a unique journey, transforming the most aggressive form of leukemia to the most curable. It followed an empirical route fueled by clinical breakthroughs driving major advances in biochemistry and cell biology, including the discovery of PML nuclear bodies (PML NBs) and their central role in APL physiopathology. Beyond APL, PML NBs have emerged as key players in a wide variety of biological functions, including tumor-suppression and SUMO-initiated protein degradation, underscoring their broad importance. The APL story is an example of how clinical observations led to the incremental development of the first targeted leukemia therapy. The understanding of APL pathogenesis and the basis for cure now opens new insights in the treatment of other diseases, especially other acute myeloid leukemias.

摘要翻译： 

急性早幼粒细胞白血病（APL）的发现、病理生理学及治疗历程是一段独特的医学探索之旅，它使这一最具侵袭性的白血病类型转变为治愈率最高的白血病。这一进程遵循着一条由临床突破驱动的经验性路径，这些突破推动了生物化学与细胞生物学领域的重大进展，包括PML核体（PML NBs）的发现及其在APL病理生理中的核心作用。超越APL本身，PML核体已被揭示在多种生物学功能中扮演关键角色，包括肿瘤抑制和SUMO介导的蛋白质降解，凸显了其广泛的重要性。APL的历程是一个范例，展示了临床观察如何逐步催生了首个靶向白血病疗法。对APL发病机制及其治愈基础的理解，如今为其他疾病（尤其是其他急性髓系白血病）的治疗开辟了新的视野。

原文链接：

History of Developing Acute Promyelocytic Leukemia Treatment and Role of Promyelocytic Leukemia Bodies