Department of Biology, McGill University, Montreal, Quebec H3A 1B1, Canada.
Laboratory for Therapeutic Development, Rosalind and Morris Goodman Cancer Research Centre and Department of Biochemistry, McGill University, Montreal, Quebec H3G 1Y6, Canada.
Sci Transl Med. 2016 Nov 16;8(365):365ra159. doi: 10.1126/scitranslmed.aag1093.
Microtubule-targeting agents (MTAs) are widely used anticancer agents, but toxicities such as neuropathy limit their clinical use. MTAs bind to and alter the stability of microtubules, causing cell death in mitosis. We describe DZ-2384, a preclinical compound that exhibits potent antitumor activity in models of multiple cancer types. It has an unusually high safety margin and lacks neurotoxicity in rats at effective plasma concentrations. DZ-2384 binds the vinca domain of tubulin in a distinct way, imparting structurally and functionally different effects on microtubule dynamics compared to other vinca-binding compounds. X-ray crystallography and electron microscopy studies demonstrate that DZ-2384 causes straightening of curved protofilaments, an effect proposed to favor polymerization of tubulin. Both DZ-2384 and the vinca alkaloid vinorelbine inhibit microtubule growth rate; however, DZ-2384 increases the rescue frequency and preserves the microtubule network in nonmitotic cells and in primary neurons. This differential modulation of tubulin results in a potent MTA therapeutic with enhanced safety.
微管靶向剂(MTAs)是广泛使用的抗癌药物,但神经毒性等毒性限制了它们的临床应用。MTAs 结合并改变微管的稳定性,导致有丝分裂中的细胞死亡。我们描述了 DZ-2384,这是一种临床前化合物,在多种癌症类型的模型中表现出强大的抗肿瘤活性。它具有异常高的安全边际,并且在有效的血浆浓度下在大鼠中没有神经毒性。DZ-2384 以独特的方式结合微管蛋白的长春花域,与其他结合长春花的化合物相比,对微管动力学产生结构和功能上不同的影响。X 射线晶体学和电子显微镜研究表明,DZ-2384 导致弯曲原纤维变直,这一效应被认为有利于微管蛋白的聚合。DZ-2384 和长春花生物碱长春瑞滨都抑制微管的生长速度;然而,DZ-2384 增加了非有丝分裂细胞和原代神经元中的微管网络的挽救频率并保留了微管网络。这种对微管蛋白的差异调节导致具有增强安全性的有效 MTA 治疗。
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