i3S - Instituto de Investigação e Inovação em Saúde, Universidade do Porto, Rua Alfredo Allen 208, Porto, 4200-393, Portugal.
INEB - Instituto Nacional de Engenharia Biomédica, Universidade do Porto, Rua Alfredo Allen 208, Porto, 4200-393, Portugal.
Small. 2023 Jun;19(22):e2300029. doi: 10.1002/smll.202300029. Epub 2023 Feb 28.
Minimal therapeutic advances have been achieved over the past two decades for glioblastoma (GBM), which remains an unmet clinical need. Here, hypothesis-driven stimuli-responsive nanoparticles (NPs) for docetaxel (DTX) delivery to GBM are reported, with multifunctional features that circumvent insufficient blood-brain barrier (BBB) trafficking and lack of GBM targeting-two major hurdles for anti-GBM therapies. NPs are dual-surface tailored with a i) brain-targeted acid-responsive Angiopep-2 moiety that triggers NP structural rearrangement within BBB endosomal vesicles, and ii) L-Histidine moiety that provides NP preferential accumulation into GBM cells post-BBB crossing. In tumor invasive margin patient cells, the stimuli-responsive multifunctional NPs target GBM cells, enhance cell uptake by 12-fold, and induce three times higher cytotoxicity in 2D and 3D cell models. Moreover, the in vitro BBB permeability is increased by threefold. A biodistribution in vivo trial confirms a threefold enhancement of NP accumulation into the brain. Last, the in vivo antitumor efficacy is validated in GBM orthotopic models following intratumoral and intravenous administration. Median survival and number of long-term survivors are increased by 50%. Altogether, a preclinical proof of concept supports these stimuli-responsive multifunctional NPs as an effective anti-GBM multistage chemotherapeutic strategy, with ability to respond to multiple fronts of the GBM microenvironment.
过去二十年,胶质母细胞瘤(GBM)的治疗进展甚微,仍然存在未满足的临床需求。本研究报告了一种基于假设的、具有刺激响应功能的载多西紫杉醇(DTX)纳米颗粒(NPs)用于治疗 GBM,其具有多功能特性,可克服血脑屏障(BBB)通透性不足和缺乏 GBM 靶向性这两大抗 GBM 治疗的主要障碍。NPs 具有双重表面修饰:i)脑靶向的酸响应性 Angiopep-2 部分,可触发 NP 在 BBB 内体囊泡中的结构重排;ii)L-组氨酸部分,可使 NP 在 BBB 穿越后优先积累到 GBM 细胞中。在肿瘤侵袭边缘的患者细胞中,刺激响应多功能 NPs 可靶向 GBM 细胞,使细胞摄取增加 12 倍,并在 2D 和 3D 细胞模型中诱导 3 倍更高的细胞毒性。此外,体外 BBB 通透性增加了 3 倍。体内生物分布试验证实,NP 在大脑中的积累增加了 3 倍。最后,在 GBM 原位模型中通过瘤内和静脉注射验证了体内抗肿瘤疗效。中位生存期和长期存活者的数量增加了 50%。总之,临床前概念验证支持这些刺激响应多功能 NPs 作为一种有效的抗 GBM 多阶段化疗策略,具有针对 GBM 微环境多个方面的响应能力。
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