Center for Nanomedicine and Department of Anesthesiology, Brigham and Women's Hospital , Harvard Medical School , Boston , Massachusetts 02115 , United States.
Center for Research Development and Evaluation of Pharmaceutical Excipients and Generic Drugs, Department of Pharmaceutics, State Key Laboratory of Natural Medicines , China Pharmaceutical University , Nanjing , Jiangsu 210009 , China.
ACS Nano. 2019 Jan 22;13(1):357-370. doi: 10.1021/acsnano.8b06400. Epub 2018 Dec 4.
Spurred by recent progress in medicinal chemistry, numerous lead compounds have sprung up in the past few years, although the majority are hindered by hydrophobicity, which greatly challenges druggability. In an effort to assess the potential of platinum (Pt) candidates, the nanosizing approach to alter the pharmacology of hydrophobic Pt(IV) prodrugs in discovery and development settings is described. The construction of a self-assembled nanoparticle (NP) platform, composed of amphiphilic lipid-polyethylene glycol (PEG) for effective delivery of Pt(IV) prodrugs capable of resisting thiol-mediated detoxification through a glutathione (GSH)-exhausting effect, offers a promising route to synergistically improving safety and efficacy. After a systematic screening, the optimized NPs (referred to as P6 NPs) exhibited small particle size (99.3 nm), high Pt loading (11.24%), reliable dynamic stability (∼7 days), and rapid redox-triggered release (∼80% in 3 days). Subsequent experiments on cells support the emergence of P6 NPs as a highly effective means of transporting a lethal dose of cargo across cytomembranes through macropinocytosis. Upon reduction by cytoplasmic reductants, particularly GSH, P6 NPs under disintegration released sufficient active Pt(II) metabolites, which covalently bound to target DNA and induced significant apoptosis. The PEGylation endowed P6 NPs with in vivo longevity and tumor specificity, which were essential to successfully inhibiting the growth of cisplatin-sensitive and -resistant xenograft tumors, while effectively alleviating toxic side-effects associated with cisplatin. P6 NPs are, therefore, promising for overcoming the bottleneck in the development of Pt drugs for oncotherapy.
受近年来药物化学进展的推动,过去几年涌现出了许多先导化合物,尽管大多数先导化合物受到疏水性的阻碍,这极大地挑战了其成药性。为了评估铂(Pt)候选物的潜力,本文描述了纳米化方法在发现和开发环境中改变疏水性 Pt(IV)前药药理学的情况。构建了一个自组装纳米颗粒(NP)平台,由两亲性脂质-聚乙二醇(PEG)组成,能够有效地递送到 Pt(IV)前药,通过谷胱甘肽(GSH)耗尽效应抵抗硫醇介导的解毒,为协同提高安全性和疗效提供了有前途的途径。经过系统筛选,优化的 NPs(称为 P6 NPs)表现出小的粒径(99.3nm)、高的 Pt 载量(11.24%)、可靠的动态稳定性(约 7 天)和快速的氧化还原触发释放(3 天内约 80%)。随后在细胞上的实验支持 P6 NPs 作为一种通过巨胞饮作用高效地将致死剂量的货物运输穿过细胞质膜的有效手段的出现。在细胞质还原剂,特别是 GSH 的还原作用下,P6 NPs 解体释放出足够的活性 Pt(II)代谢物,与靶 DNA 共价结合,诱导明显的细胞凋亡。PEG 化赋予了 P6 NPs 体内的长寿性和肿瘤特异性,这对于成功抑制顺铂敏感和耐药异种移植肿瘤的生长是至关重要的,同时有效缓解了与顺铂相关的毒性副作用。因此,P6 NPs 有望克服铂类药物在肿瘤治疗中的发展瓶颈。
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