Collaborative Innovation Center of Advanced Drug Delivery System and Biotech Drugs, School of Pharmacy, Yantai University, Yantai, Shandong, China.
MabPlex International, Yantai, Shandong, China.
Drug Dev Res. 2024 Aug;85(5):e22241. doi: 10.1002/ddr.22241.
The role of KRAS mutation in non-small cell lung cancer (NSCLC) initiation and progression is well-established. However, "undruggable" KRAS protein poses the research of small molecule inhibitors a significant challenge. Addressing this, proteolysis-targeting chimeras (PROTACs) have become a cutting-edge treatment method, emphasizing protein degradation. A modified ethanol injection method was employed in this study to formulate liposomes encapsulating PROTAC drug LC-2 (LC-2 LPs). Precise surface modifications using cell-penetrating peptide R8 yielded R8-LC-2 liposomes (R8-LC-2 LPs). Comprehensive cellular uptake and cytotoxicity studies unveiled that R8-LC-2 LPs depended on concentration and time, showcasing the superior performance of R8-LC-2 LPs compared to normal liposomes. In vivo pharmacokinetic profiles demonstrated the capacity of DSPE-PEG2000 to prolong the circulation time of LC-2, leading to higher plasma concentrations compared to free LC-2. In vivo antitumor efficacy research underscored the remarkable ability of R8-LC-2 LPs to effectively suppress tumor growth. This study contributed to the exploration of enhanced therapeutic strategies for NSCLC, specifically focusing on the development of liposomal PROTACs targeting the "undruggable" KRAS protein. The findings provide valuable insights into the potential of this innovative approach, offering prospects for improved drug delivery and heightened antitumor efficacy.
KRAS 突变在非小细胞肺癌(NSCLC)的发生和发展中的作用已得到充分证实。然而,“不可成药”的 KRAS 蛋白给小分子抑制剂的研究带来了巨大的挑战。针对这一问题,蛋白水解靶向嵌合体(PROTAC)已成为一种前沿的治疗方法,强调蛋白质的降解。本研究采用改良的乙醇注入法制备了包裹 PROTAC 药物 LC-2(LC-2 LPs)的脂质体。通过使用穿透肽 R8 进行精确的表面修饰,得到了 R8-LC-2 脂质体(R8-LC-2 LPs)。全面的细胞摄取和细胞毒性研究表明,R8-LC-2 LPs 依赖于浓度和时间,表现出 R8-LC-2 LPs 优于普通脂质体的卓越性能。体内药代动力学研究表明,DSPE-PEG2000 能够延长 LC-2 的循环时间,使游离 LC-2 相比之下,血浆浓度更高。体内抗肿瘤功效研究强调了 R8-LC-2 LPs 有效抑制肿瘤生长的显著能力。本研究为 NSCLC 的增强治疗策略的探索做出了贡献,特别是针对针对“不可成药”KRAS 蛋白的脂质体 PROTAC 的开发。这些发现为这种创新方法的潜力提供了有价值的见解,为提高药物输送和增强抗肿瘤功效提供了前景。