Munir Iqra, Nazir Faiqa, Yesiloz Gurkan
National Nanotechnology Research Center (UNAM) Bilkent University, Cankaya, Ankara, 06800, Türkiye.
Institute of Material Science and Nanotechnology, Bilkent University, Cankaya, Ankara, 06800, Türkiye.
ACS Appl Mater Interfaces. 2024 Dec 25;16(51):70187-70204. doi: 10.1021/acsami.4c12524. Epub 2024 Dec 11.
In the existing development of extensive drug screening models, 3D cell cultures outshine conventional 2D monolayer cells by closely imitating the in vivo tumor microenvironment. This makes 3D culture a more physiologically relevant and convenient system in the regime of preclinical drug testing. In the nanomedicinal world, nanoconjugates as nanocarriers are largely hunted due to their capability of precisely binding to target cells and distributing essential dosages of therapeutic drugs with enhanced safety profiles. Thus, for boosted drug availability, the evolution from conventional drug treatment to combination therapies and last switching to drug carriers has gained significant progression in cancer cure. In contrast to conventional engineered nanoparticles, herein, we successfully designed biomolecule (ferritin)-based drug nanoconjugates effective both as a single drug (valproic acid-VPA) and twin-drug (valproic acid/doxorubicin-Dox) carriers, which dramatically enhance the proficiency of the tumor therapeutic modality. To question the reported adjuvant drug property of VPA, we progressed utilizing at first VPA alone as an effective yet exclusive tumor therapy when delivered via some carrier molecule, in particular protein. Subsequently, we paralleled this comprehensive investigation output to compare and test the coloading strategy of drugs and observe the synergistic and/or additive behavior of VPA in conjugation with other anticancer agents (Dox) while given via a carrier molecule. To approach this, VPA and/or Dox molecules were encapsulated into the ferritin (F) cavity using a thermosensitive synthesis method by maintaining the temperature at 60 °C. The successful encapsulation of drugs in the protein nanocage was confirmed through various characterization techniques. The F-VPA/F-VPA-Dox nanoconjugates exhibited similar morphology and structural characteristics to the hollow ferritin cage and showed significant cytotoxicity than the naked drugs when tested on physiologically relevant 3D spheroid models. Precisely, our first designed carrier nanoconjugate, i.e., F-VPA, offered more than a 3-fold increased intratumoral drug concentration than free VPA and significantly suppressed tumor growth after a single-dose treatment. However, our second modeled carrier nanoconjugate, viz. F-VPA-Dox, revealed an extended median survival period and lesser toxicity when administered at a much more effective dose (∼3-5 μM), in 3D tumor spheroid models of various cancer cell lines. All in all, importantly, ferritin nanoconjugates exhibited an enhanced tumor inhibition rate with a single-dose treatment, which further confirms the benefits of the active targeting property of these nanocarriers. Moreover, these nanocarriers also offer to deliver a significant dose of the therapeutic drug into tumor cells, alongside tremendous biocompatibility and safety profiles in numerous tumor 3D spheroid models.
在现有的广泛药物筛选模型开发中,3D细胞培养通过紧密模拟体内肿瘤微环境,优于传统的2D单层细胞。这使得3D培养在临床前药物测试中成为一个更具生理相关性且便捷的系统。在纳米医学领域,作为纳米载体的纳米缀合物因其能够精确结合靶细胞并以更高的安全性分布必需剂量的治疗药物而备受青睐。因此,为提高药物可用性,从传统药物治疗到联合疗法,再到最终转向药物载体的发展在癌症治疗中取得了显著进展。与传统的工程纳米颗粒不同,在此,我们成功设计了基于生物分子(铁蛋白)的药物纳米缀合物,其作为单一药物(丙戊酸-VPA)和双药(丙戊酸/阿霉素-Dox)载体均有效,显著提高了肿瘤治疗方式的效率。为质疑已报道的VPA的辅助药物特性,我们首先通过某些载体分子,特别是蛋白质,单独使用VPA作为一种有效但唯一的肿瘤治疗方法展开研究。随后,我们将这一全面的研究结果进行对比,以测试药物的共负载策略,并观察VPA与其他抗癌剂(Dox)通过载体分子给药时的协同和/或加成行为。为此,通过在60℃保持温度,采用热敏合成方法将VPA和/或Dox分子封装到铁蛋白(F)腔内。通过各种表征技术证实了药物成功封装在蛋白质纳米笼中。F-VPA/F-VPA-Dox纳米缀合物与中空铁蛋白笼表现出相似的形态和结构特征,并且在生理相关的3D球体模型上测试时,显示出比游离药物更高的细胞毒性。确切地说,我们首次设计的载体纳米缀合物,即F-VPA,其瘤内药物浓度比游离VPA增加了3倍以上,单剂量治疗后显著抑制肿瘤生长。然而,我们第二个构建的载体纳米缀合物,即F-VPA-Dox,在各种癌细胞系的3D肿瘤球体模型中,以更有效的剂量(约3-5μM)给药时,显示出延长的中位生存期和更低的毒性。总而言之,重要的是,铁蛋白纳米缀合物单剂量治疗显示出增强的肿瘤抑制率,这进一步证实了这些纳米载体主动靶向特性的益处。此外,这些纳米载体还能够在众多肿瘤3D球体模型中,将大量治疗药物输送到肿瘤细胞中,同时具有出色的生物相容性和安全性。