Smith Blake, Ownby Raymond L
Psychiatry and Behavioral Sciences, Nova Southeastern University Dr. Kiran C. Patel College of Osteopathic Medicine, Davie, USA.
Cureus. 2024 Mar 13;16(3):e56105. doi: 10.7759/cureus.56105. eCollection 2024 Mar.
Alzheimer's disease (AD) is a progressive neurodegenerative disorder characterized by memory impairment, a loss of cholinergic neurons, and cognitive decline that insidiously progresses to dementia. The pathoetiology of AD is complex, as genetic predisposition, age, inflammation, oxidative stress, and dysregulated proteostasis all contribute to its development and progression. The histological hallmarks of AD are the formation and accumulation of amyloid-β plaques and interfibrillar tau tangles within the central nervous system. These histological hallmarks trigger neuroinflammation and disrupt the physiological structure and functioning of neurons, leading to cognitive dysfunction. Most treatments currently available for AD focus only on symptomatic relief. Disease-modifying treatments (DMTs) that target the biology of the disease in hopes of slowing or reversing disease progression are desperately needed. This narrative review investigates novel DMTs and their therapeutic targets that are either in phase three of development or have been recently approved by the U.S. Food and Drug Administration (FDA). The target areas of some of these novel DMTs consist of combatting amyloid or tau accumulation, oxidative stress, neuroinflammation, and dysregulated proteostasis, metabolism, or circadian rhythm. Neuroprotection and neuroplasticity promotion were also key target areas. DMT therapeutic target diversity may permit improved therapeutic responses in certain subpopulations of AD, particularly if the therapeutic target of the DMT being administered aligns with the subpopulation's most prominent pathological findings. Clinicians should be cognizant of how these novel drugs differ in therapeutic targets, as this knowledge may potentially enhance the level of care they can provide to AD patients in the future.
阿尔茨海默病(AD)是一种进行性神经退行性疾病,其特征为记忆障碍、胆碱能神经元丧失以及认知能力下降,并逐渐发展为痴呆症。AD的发病机制复杂,遗传易感性、年龄、炎症、氧化应激和蛋白质稳态失调均对其发生和发展有影响。AD的组织学特征是中枢神经系统内淀粉样β斑块的形成和积累以及神经原纤维缠结。这些组织学特征引发神经炎症,破坏神经元的生理结构和功能,导致认知功能障碍。目前大多数针对AD的治疗仅侧重于缓解症状。迫切需要能够针对疾病生物学机制以减缓或逆转疾病进展的疾病修饰治疗(DMT)。本叙述性综述研究了处于三期开发阶段或最近已获美国食品药品监督管理局(FDA)批准的新型DMT及其治疗靶点。其中一些新型DMT的靶点领域包括对抗淀粉样蛋白或tau蛋白积累、氧化应激、神经炎症以及蛋白质稳态失调、代谢或昼夜节律失调。神经保护和促进神经可塑性也是关键的靶点领域。DMT治疗靶点的多样性可能使AD的某些亚群获得更好的治疗反应,特别是如果所给予的DMT的治疗靶点与该亚群最突出的病理发现相匹配。临床医生应了解这些新型药物在治疗靶点方面的差异,因为这些知识可能会提高他们未来为AD患者提供护理的水平。