Department of Pharmacy, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.
Department of Pharmacy, Tongren Polytechnic College, Tongren, 554300, China.
Adv Healthc Mater. 2024 Aug;13(20):e2400109. doi: 10.1002/adhm.202400109. Epub 2024 May 3.
Proteolysis targeting chimeras (PROTACs) technology is rapidly developed as a novel and selective medicinal strategy for the degradation of cellular proteins in cancer therapy. However, the applications of PROTACs as heterobifunctional molecules are largely limited by high molecular weight, low bioavailability, poor permeability, insufficient targeting, and low efficacy in vivo. Herein, self-assembling micelles of FA-PEG-PROTAC are designed for cancer cell selective targeting and reductive-response proteolysis in tumor-bearing mice. FA-PEG-PROTAC is prepared by conjugating folic acid (FA)-PEG with EGFR-targeting PROTAC via a disulfide bond. The FA-PEG-PROTAC micelles, formed by self-assembling, are demonstrated to significantly improve tumor targeting efficacy and exhibit excellent anti-tumor efficacy in the mouse xenograft model compared to the traditional PROTACs. The strategy of applying self-assembled FA-PEG-PROTAC micelles in tumor therapy can not only improve targeted proteolysis efficiency but also broaden applications in the development of PROTAC-based drugs.
蛋白水解靶向嵌合体(PROTACs)技术作为一种新型的、选择性的癌症治疗细胞内蛋白降解的药物研发策略,发展迅速。然而,PROTAC 作为双功能杂合体分子的应用在很大程度上受到高分子量、低生物利用度、差通透性、靶向不足和体内疗效低的限制。在此,设计了 FA-PEG-PROTAC 自组装胶束用于荷瘤小鼠的癌细胞选择性靶向和还原响应性蛋白水解。FA-PEG-PROTAC 通过二硫键将叶酸(FA)-PEG 与 EGFR 靶向 PROTAC 连接,制备得到。自组装形成的 FA-PEG-PROTAC 胶束与传统的 PROTACs 相比,显著提高了肿瘤靶向效率,并在小鼠异种移植模型中表现出优异的抗肿瘤疗效。在肿瘤治疗中应用自组装的 FA-PEG-PROTAC 胶束的策略不仅可以提高靶向蛋白水解效率,还可以拓宽基于 PROTAC 药物的开发应用。