Rafii Michael S, Aisen Paul S
Alzheimer's Therapeutic Research Institute, Keck School of Medicine, University of Southern California, San Diego, CA, USA.
Nat Rev Neurol. 2025 Jul 21. doi: 10.1038/s41582-025-01123-5.
The treatment of Alzheimer disease (AD) has crossed a pivotal threshold, marked by the landmark approvals of the first-ever disease-modifying therapies. These immunotherapies, specifically monoclonal antibodies (mAbs) that target various amyloid-β (Aβ) species including proto-fibrillar and fibrillar forms, substantially lower levels of Aβ in the brain. The therapies have collectively demonstrated the ability to slow cognitive and clinical decline in large placebo-controlled trials, ushering a new era in the management of AD. Here, we review recent progress made in bringing amyloid-lowering mAb therapies to the clinic and explore future directions in this rapidly evolving field. We also delve into the current understanding of AD as a biological continuum, from its asymptomatic preclinical stage to its clinically overt dementia stage. We explore how this conceptualization provides a regulatory framework to evaluate amyloid-lowering mAbs across the entire spectrum of the disease. Additionally, we review key factors that affect the integration of these treatments into clinical practice.
阿尔茨海默病(AD)的治疗已跨越一个关键门槛,标志性事件是首款疾病修饰疗法获得批准。这些免疫疗法,特别是针对包括原纤维和纤维形式在内的各种淀粉样β(Aβ)亚型的单克隆抗体(mAb),可大幅降低大脑中的Aβ水平。在大型安慰剂对照试验中,这些疗法共同证明了减缓认知和临床衰退的能力,开创了AD治疗的新时代。在此,我们回顾了将降低淀粉样蛋白的单克隆抗体疗法引入临床的最新进展,并探讨了这个快速发展领域的未来方向。我们还深入研究了目前对AD作为一个生物学连续体的理解,从无症状的临床前阶段到临床明显的痴呆阶段。我们探讨了这种概念化如何提供一个监管框架,以评估在疾病的整个范围内降低淀粉样蛋白的单克隆抗体。此外,我们回顾了影响这些治疗方法融入临床实践的关键因素。