Abioye Amos, Akintade Damilare, Mitchell James, Olorode Simisade, Adejare Adeboye
College of Pharmacy and Health Sciences, Belmont University, Nashville, TN 37212, USA.
Department of Biomedical Sciences, School of Health, Leeds Beckett University, Leeds LS1 3HE, UK.
Pharmaceutics. 2024 Apr 30;16(5):609. doi: 10.3390/pharmaceutics16050609.
Despite the extensive research successes and continuous developments in modern medicine in terms of diagnosis, prevention, and treatment, the lack of clinically useful disease-modifying drugs or immunotherapeutic agents that can successfully treat or prevent neurodegenerative diseases is an ongoing challenge. To date, only one of the 244 drugs in clinical trials for the treatment of neurodegenerative diseases has been approved in the past decade, indicating a failure rate of 99.6%. In corollary, the approved monoclonal antibody did not demonstrate significant cognitive benefits. Thus, the prevalence of neurodegenerative diseases is increasing rapidly. Therefore, there is an urgent need for creative approaches to identifying and testing biomarkers for better diagnosis, prevention, and disease-modifying strategies for the treatment of neurodegenerative diseases. Overexpression of the endogenous α-synuclein has been identified as the driving force for the formation of the pathogenic α-synuclein (α-Syn) conformers, resulting in neuroinflammation, hypersensitivity, endogenous homeostatic responses, oxidative dysfunction, and degeneration of dopaminergic neurons in Parkinson's disease (PD). However, the conformational plasticity of α-Syn proffers that a certain level of α-Syn is essential for the survival of neurons. Thus, it exerts both neuroprotective and neurotoxic (regulatory) functions on neighboring neuronal cells. Furthermore, the aberrant metastable α-Syn conformers may be subtle and difficult to detect but may trigger cellular and molecular events including immune responses. It is well documented in literature that the misfolded α-Syn and its conformers that are released into the extracellular space from damaged or dead neurons trigger the innate and adaptive immune responses in PD. Thus, in this review, we discuss the nonintuitive plasticity and immunogenicity of the α-Syn conformers in the brain immune cells and their physiological and pathological consequences on the neuroimmune responses including neuroinflammation, homeostatic remodeling, and cell-specific interactions that promote neuroprotection in PD. We also critically reviewed the novel strategies for immunotherapeutic delivery interventions in PD pathogenesis including immunotherapeutic targets and potential nanoparticle-based smart drug delivery systems. It is envisioned that a greater understanding of the nonintuitive immunogenicity of aberrant α-Syn conformers in the brain's microenvironment would provide a platform for identifying valid therapeutic targets and developing smart brain delivery systems for clinically effective disease-modifying immunotherapeutics that can aid in the prevention and treatment of PD in the future.
尽管现代医学在诊断、预防和治疗方面取得了广泛的研究成果并不断发展,但缺乏能够成功治疗或预防神经退行性疾病的具有临床实用价值的疾病修饰药物或免疫治疗药物仍是一个持续存在的挑战。迄今为止,在过去十年中,用于治疗神经退行性疾病的244种临床试验药物中只有一种获得批准,失败率达99.6%。相应地,已批准的单克隆抗体并未显示出显著的认知益处。因此,神经退行性疾病的患病率正在迅速上升。因此,迫切需要创新方法来识别和测试生物标志物,以实现更好的诊断、预防以及用于治疗神经退行性疾病的疾病修饰策略。内源性α-突触核蛋白的过表达已被确定为致病性α-突触核蛋白(α-Syn)构象异构体形成的驱动力,导致神经炎症、超敏反应、内源性稳态反应、氧化功能障碍以及帕金森病(PD)中多巴胺能神经元的退化。然而,α-Syn的构象可塑性表明一定水平的α-Syn对神经元的存活至关重要。因此,它对邻近的神经元细胞发挥神经保护和神经毒性(调节)功能。此外,异常的亚稳态α-Syn构象异构体可能很微妙且难以检测,但可能引发包括免疫反应在内的细胞和分子事件。文献充分证明,从受损或死亡神经元释放到细胞外空间的错误折叠的α-Syn及其构象异构体会引发PD中的先天性和适应性免疫反应。因此,在本综述中,我们讨论了α-Syn构象异构体在脑免疫细胞中的非直观可塑性和免疫原性,以及它们对神经免疫反应(包括神经炎症、稳态重塑和促进PD神经保护的细胞特异性相互作用)的生理和病理影响。我们还批判性地回顾了PD发病机制中免疫治疗递送干预的新策略,包括免疫治疗靶点和基于纳米颗粒的潜在智能药物递送系统。可以预见,更深入地了解大脑微环境中异常α-Syn构象异构体的非直观免疫原性将为识别有效的治疗靶点和开发智能脑递送系统提供一个平台,以用于未来临床上有效的疾病修饰免疫治疗,从而有助于预防和治疗PD。