Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, 210029, China.
Key Laboratory of Hematology of Nanjing Medical University, Nanjing, 210029, China.
J Hematol Oncol. 2021 Mar 6;14(1):40. doi: 10.1186/s13045-021-01049-7.
B cell receptor (BCR) signaling is involved in the pathogenesis of B cell malignancies. Activation of BCR signaling promotes the survival and proliferation of malignant B cells. Bruton tyrosine kinase (BTK) is a key component of BCR signaling, establishing BTK as an important therapeutic target. Several covalent BTK inhibitors have shown remarkable efficacy in the treatment of B cell malignancies, especially chronic lymphocytic leukemia. However, acquired resistance to covalent BTK inhibitors is not rare in B cell malignancies. A major mechanism for the acquired resistance is the emergence of BTK cysteine 481 (C481) mutations, which disrupt the binding of covalent BTK inhibitors. Additionally, adverse events due to the off-target inhibition of kinases other than BTK by covalent inhibitors are common. Alternative therapeutic options are needed if acquired resistance or intolerable adverse events occur. Non-covalent BTK inhibitors do not bind to C481, therefore providing a potentially effective option to patients with B cell malignancies, including those who have developed resistance to covalent BTK inhibitors. Preliminary clinical studies have suggested that non-covalent BTK inhibitors are effective and well-tolerated. In this review, we discussed the rationale for the use of non-covalent BTK inhibitors and the preclinical and clinical studies of non-covalent BTK inhibitors in B cell malignancies.
B 细胞受体(BCR)信号参与 B 细胞恶性肿瘤的发病机制。BCR 信号的激活促进恶性 B 细胞的存活和增殖。布鲁顿酪氨酸激酶(BTK)是 BCR 信号的关键组成部分,确立了 BTK 作为重要治疗靶点的地位。几种共价 BTK 抑制剂在治疗 B 细胞恶性肿瘤方面显示出显著疗效,尤其是慢性淋巴细胞白血病。然而,在 B 细胞恶性肿瘤中,获得性对共价 BTK 抑制剂的耐药性并不罕见。获得性耐药的一个主要机制是 BTK 半胱氨酸 481(C481)突变的出现,该突变破坏了共价 BTK 抑制剂的结合。此外,由于共价抑制剂对 BTK 以外的激酶的非靶标抑制,常出现不良反应。如果发生获得性耐药或无法耐受的不良反应,则需要替代治疗选择。非共价 BTK 抑制剂不与 C481 结合,因此为包括已对共价 BTK 抑制剂耐药的患者在内的 B 细胞恶性肿瘤患者提供了一种潜在有效的选择。初步临床研究表明,非共价 BTK 抑制剂有效且耐受良好。在这篇综述中,我们讨论了使用非共价 BTK 抑制剂的基本原理以及非共价 BTK 抑制剂在 B 细胞恶性肿瘤中的临床前和临床研究。