Department of Psychiatry, School of Medicine, University of Pittsburgh, Pittsburgh, PA, 15213, USA; Department of Neurology, School of Medicine, University of Pittsburgh, Pittsburgh, PA, 15213, USA.
Brain Institute of Rio Grande do Sul, PUCRS, Porto Alegre, Brazil.
EBioMedicine. 2024 Oct;108:105322. doi: 10.1016/j.ebiom.2024.105322. Epub 2024 Oct 3.
Biomarkers have been instrumental in population selection and disease monitoring in clinical trials of recently FDA-approved drugs targeting amyloid-β to slow the progression of Alzheimer's disease (AD). As new therapeutic strategies and biomarker techniques emerge, the importance of biomarkers in drug development is growing exponentially. In this emerging landscape, biomarkers are expected to serve a wide range of contexts of use in clinical trials focusing on AD and related dementias. The joint FDA-NIH BEST (Biomarkers, EndpointS, and other Tools) framework provides standardised terminology to facilitate communication among stakeholders in this increasingly complex field. This review explores various applications of biomarkers relevant to AD clinical trials, using the BEST resource as a reference. For simplicity, we predominantly provide contextual characterizations of biomarkers use from the perspective of drugs targeting amyloid-β and tau proteins. However, general definitions and concepts can be extrapolated to other targets.
生物标志物在最近获得 FDA 批准的针对淀粉样蛋白-β以减缓阿尔茨海默病（AD）进展的药物的临床试验中的人群选择和疾病监测中发挥了重要作用。随着新的治疗策略和生物标志物技术的出现，生物标志物在药物开发中的重要性呈指数级增长。在这个新兴领域，生物标志物有望在专注于 AD 和相关痴呆症的临床试验中广泛应用于各种使用场景。FDA-NIH BEST（生物标志物、终点和其他工具）框架提供了标准化术语，以促进该领域利益相关者之间的沟通。本综述使用 BEST 资源作为参考，探讨了与 AD 临床试验相关的各种生物标志物的应用。为简单起见，我们主要从针对淀粉样蛋白-β和 tau 蛋白的药物角度提供了生物标志物使用的背景描述。然而，一般定义和概念可以外推到其他靶点。
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