Collings Stevens Chronic Leukemia Research Laboratory, Terry Fox Laboratory, British Columbia Cancer Research Institute, Vancouver, BC V5Z 1L3, Canada.
Department of Medicine, University of British Columbia, Vancouver, BC V6T 1Z4, Canada.
Int J Mol Sci. 2024 Mar 14;25(6):3307. doi: 10.3390/ijms25063307.
Chronic myeloid leukemia is a multistep, multi-lineage myeloproliferative disease that originates from a translocation event between chromosome 9 and chromosome 22 within the hematopoietic stem cell compartment. The resultant fusion protein BCR::ABL1 is a constitutively active tyrosine kinase that can phosphorylate multiple downstream signaling molecules to promote cellular survival and inhibit apoptosis. Currently, tyrosine kinase inhibitors (TKIs), which impair ABL1 kinase activity by preventing ATP entry, are widely used as a successful therapeutic in CML treatment. However, disease relapses and the emergence of resistant clones have become a critical issue for CML therapeutics. Two main reasons behind the persisting obstacles to treatment are the acquired mutations in the ABL1 kinase domain and the presence of quiescent CML leukemia stem cells (LSCs) in the bone marrow, both of which can confer resistance to TKI therapy. In this article, we systemically review the structural and molecular properties of the critical domains of BCR::ABL1 and how understanding the essential role of BCR::ABL1 kinase activity has provided a solid foundation for the successful development of molecularly targeted therapy in CML. Comparison of responses and resistance to multiple BCR::ABL1 TKIs in clinical studies and current combination treatment strategies are also extensively discussed in this article.
慢性髓性白血病是一种多步骤、多谱系髓系增殖性疾病,起源于造血干细胞区染色体 9 和染色体 22 之间的易位事件。由此产生的融合蛋白 BCR::ABL1 是一种组成性激活的酪氨酸激酶,可磷酸化多个下游信号分子,促进细胞存活并抑制细胞凋亡。目前,通过阻止 ATP 进入来抑制 ABL1 激酶活性的酪氨酸激酶抑制剂(TKI)被广泛用作 CML 治疗的成功疗法。然而,疾病复发和耐药克隆的出现已成为 CML 治疗的一个关键问题。治疗中持续存在障碍的两个主要原因是 ABL1 激酶结构域中的获得性突变和骨髓中静止的 CML 白血病干细胞(LSC)的存在,这两者都可以对 TKI 治疗产生耐药性。本文系统地综述了 BCR::ABL1 的关键结构域的结构和分子特性,以及理解 BCR::ABL1 激酶活性的重要作用如何为 CML 的分子靶向治疗的成功发展提供了坚实的基础。本文还广泛讨论了临床研究中对多种 BCR::ABL1 TKI 的反应和耐药性以及当前的联合治疗策略。