Jiangsu Collaborative Innovation Center of Chinese Medicinal Resources Industrialization, School of Medicine, Nanjing University of Chinese Medicine, Nanjing 210023, PR China.
Department of Hematology, the First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing 210029, PR China.
Int Immunopharmacol. 2024 Dec 25;143(Pt 2):113311. doi: 10.1016/j.intimp.2024.113311. Epub 2024 Oct 24.
The BCR:ABL T315I mutation presents a significant challenge in the current management of Chronic Myeloid Leukemia (CML), highlighting the need to identify novel targets and drugs. In our study, we observed the elevated expression of UBE2Q1 in KBM5-T315I cells compared to KBM5 cells, where it interacted with DDX3, regulating its ubiquitination. Furthermore, we found that Bortezomib (BTZ) targeted UBE2Q1, reducing its protein level expression. Consequently, BTZ dose-dependently inhibited the growth vitality of KBM5-T315I cells, inducing increased ROS production, mitochondrial membrane potential collapse, cytochrome C release, and expression of apoptosis-related proteins. These events collectively induced apoptosis in KBM5-T315I cells. Moreover, BTZ enhanced the therapeutic effects of anti-PD-1 treatment. In NOD/SCID mice bearing KBM5-T315I cell line xenografts, BTZ administration (2 mg/kg, ip, every other day for 4 weeks) significantly inhibited the growth of KBM5-T315Iderived xenografts and extended survival. In conclusion, our study sheds new light on the BTZ-induced apoptosis mechanism, suggesting the potential of BTZ as a promising chemo-immunotherapy agent against BCR:ABL T315I mutation CML.
BCR-ABL T315I 突变是慢性髓性白血病(CML)当前治疗的重大挑战,突显了需要识别新的靶点和药物。在我们的研究中,与 KBM5 细胞相比,我们观察到 KBM5-T315I 细胞中 UBE2Q1 的表达升高,它与 DDX3 相互作用,调节其泛素化。此外,我们发现硼替佐米(BTZ)靶向 UBE2Q1,降低其蛋白水平表达。因此,BTZ 呈剂量依赖性抑制 KBM5-T315I 细胞的生长活力,诱导增加 ROS 产生、线粒体膜电位崩溃、细胞色素 C 释放和凋亡相关蛋白的表达。这些事件共同诱导 KBM5-T315I 细胞凋亡。此外,BTZ 增强了抗 PD-1 治疗的疗效。在携带 KBM5-T315I 细胞系异种移植的 NOD/SCID 小鼠中,BTZ 给药(2mg/kg,ip,每隔一天给药 4 周)显著抑制 KBM5-T315I 衍生的异种移植物的生长并延长生存。总之,我们的研究为 BTZ 诱导的细胞凋亡机制提供了新的视角,表明 BTZ 作为针对 BCR-ABL T315I 突变 CML 的有前途的化疗免疫治疗药物具有潜力。