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慢性髓性白血病:克服耐药性的现有治疗选择和策略。

Chronic Myeloid Leukemia: Existing Therapeutic Options and Strategies to Overcome Drug Resistance.

机构信息

Centre for Bioinformatics, School of Life Sciences, Pondicherry University, Kalapet, Puducherry 605014, India.

出版信息

Mini Rev Med Chem. 2019;19(4):333-345. doi: 10.2174/1389557518666181017124854.

Abstract

Chronic myeloid leukemia (CML) is a myeloproliferative disease caused due to translocation between chromosome 9 and 22 leading to a chimeric gene product known as Bcr-Abl. Bcr-Abl fusion protein has constitutively activated Abl tyrosine kinase activity which is responsible for the uncontrolled proliferation in CML The tyrosine kinase inhibitors (TKIs) such as Imatinib, Dasatinib, and Nilotinib are the current first-line treatments approved by the United States Food and Drug Administration (US FDA) for the treatment of the disease. Despite the spectacular progress made over the decade with the TKIs, patients develop resistance to these TKIs. In such cases, stem cell transplant therapy, which is limited by donor availability, is the only proven cure for the patients. This highlights the need for the development of new strategies for CML treatment. The Bcr-Abl point mutations, including the gatekeeper T315I mutations, are the principal cause for the development of resistance to TKIs. However, other mechanisms are also involved in the failure of TKI therapy. This review outlines the Bcr-Abl dependent and independent mechanism of TKIs resistance development and the strategies used to overcome drug resistance, such as the development of ATP site and allosteric site inhibitors. Binding mode and structural elements of Bcr-Abl inhibition are discussed with emphasis on pathways involved in this complex disease to determine alternative strategies and combination therapies.

摘要

慢性髓性白血病(CML)是一种骨髓增生性疾病,由染色体 9 和 22 之间的易位引起,导致嵌合基因产物,即 Bcr-Abl。Bcr-Abl 融合蛋白具有组成性激活 Abl 酪氨酸激酶活性,这是 CML 中不受控制增殖的原因。酪氨酸激酶抑制剂(TKI),如伊马替尼、达沙替尼和尼洛替尼,是目前美国食品和药物管理局(US FDA)批准的治疗该疾病的一线治疗药物。尽管在过去十年中 TKI 取得了显著进展,但患者仍会对这些 TKI 产生耐药性。在这种情况下,干细胞移植治疗,由于供体可用性有限,是患者唯一的有效治疗方法。这突出表明需要开发新的 CML 治疗策略。Bcr-Abl 点突变,包括守门人 T315I 突变,是导致 TKI 耐药的主要原因。然而,其他机制也参与了 TKI 治疗的失败。本综述概述了 TKI 耐药发展的 Bcr-Abl 依赖性和独立性机制,以及克服耐药性的策略,如 ATP 位点和变构位点抑制剂的开发。讨论了 Bcr-Abl 抑制的结合模式和结构要素,并重点讨论了涉及这种复杂疾病的途径,以确定替代策略和联合治疗。

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