The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USA; University of Connecticut Health Center & Connecticut Children's Medical Center, Department of Pediatrics, Farmington, CT 06032, USA.
Tufts University, Department of Biomedical Engineering, Medford, MA 02155, United States.
Acta Biomater. 2019 Aug;94:306-319. doi: 10.1016/j.actbio.2019.02.052. Epub 2019 Mar 2.
Brain access remains a major challenge in drug testing. The nearly 'impermeable' blood-brain-barrier (BBB) prevents most drugs from gaining access to brain cells via systematic intravenous (IV) injection. In this study, silk fibroin films were used as drug carrier as well as cell culture substrate to simulate the in vivo interface between drug reservoir and brain cells for testing drug delivery in the brain. In in vitro studies, film-released arabinofuranosyl cytidine (AraC), a mitotic inhibitor, selectively killed glial cells in film-supported mixed neural cell cultures; with widened dosage windows for drug efficacy and tolerance compared to drugs in solution. In the brain, the presence of silk films was well tolerated with no signs of acute neuroinflammation, cell death, or altered brain function. Topical application of silk films on the cortical surface delivered Evans blue, a BBB-impenetrable fluorescent marker, through the intact dura matter into the parenchyma of the ipsilateral hemisphere as deep as the hippocampal region, but not the contralateral hemisphere. In a mouse traumatic brain injury (TBI) model, necrosis markers by film delivery accessed more cells in the lesion core than by con-current IV delivery; whereas the total coverage including the peri-lesional area appeared to be comparable between the two routes. The complementary distribution patterns of co-delivered markers provided direct evidence of the partial confinement of either route's access to brain cells by a restrictive zone near the lesion border. Finally, film-delivered necrostatin-1 reduced overall cell necrosis by approximately 40% in the TBI model. These findings from representative small molecules of delivery route-dependent drug access are broadly applicable for evaluating drug actions both in vitro and in vivo. Combined with its demonstrated role of supporting neuron-electrode interfaces, the film system can be further developed for testing a range of neuromodulation approaches (i.e., drug delivery, electrical stimulation, cell graft) in the brain. STATEMENT OF SIGNIFICANCE: This study demonstrated that silk fibroin films can be used to evaluate drug actions both in vitro and in vivo, partially overcoming the significant delivery barriers of the brain. This system can be adapted for efficient drug access to specific brain regions and/or cell types. The film system can be further developed for testing a range of interventions with drugs, electrical signals or cell graft for analysis of treatment outcomes including cell responses and brain function.
脑内给药仍然是药物测试的一大挑战。几乎“不可渗透”的血脑屏障(BBB)阻止了大多数药物通过系统静脉(IV)注射进入脑细胞。在这项研究中,丝素蛋白薄膜既用作药物载体,也用作细胞培养基质,以模拟药物储库与脑细胞之间的体内界面,用于测试脑内的药物递送。在体外研究中,薄膜释放的胞嘧啶阿拉伯糖苷(AraC),一种有丝分裂抑制剂,在薄膜支持的混合神经细胞培养物中选择性杀死神经胶质细胞;与溶液中的药物相比,其药效和耐受性的剂量范围更宽。在大脑中,丝素薄膜的存在得到了很好的耐受,没有急性神经炎症、细胞死亡或大脑功能改变的迹象。丝素薄膜在皮质表面的局部应用,将 Evans 蓝(一种不可穿透 BBB 的荧光标记物)通过完整的硬脑膜递送到对侧半球的脑实质深处,可达海马区,但不会递送到对侧半球。在小鼠创伤性脑损伤(TBI)模型中,通过薄膜递送的坏死标志物比同时进行的 IV 递送更容易进入损伤核心的细胞;然而,两种途径的总覆盖范围包括损伤周围区域,似乎相似。共递送标志物的互补分布模式为两种途径对损伤边界附近的限制区的细胞进入提供了直接证据。最后,在 TBI 模型中,薄膜递送的 Necrostatin-1 使总体细胞坏死减少了约 40%。这些代表药物输送途径依赖性药物进入的小分子的发现结果广泛适用于评估体外和体内的药物作用。结合其支持神经元-电极界面的作用,该薄膜系统可进一步开发用于测试一系列脑内神经调节方法(即药物输送、电刺激、细胞移植)。
本研究表明,丝素蛋白薄膜可用于评估体外和体内的药物作用,部分克服了大脑的重大输送障碍。该系统可适用于特定脑区和/或细胞类型的药物高效进入。该薄膜系统可进一步开发,用于测试一系列药物、电信号或细胞移植干预措施,以分析包括细胞反应和大脑功能在内的治疗结果。