The Role of Biomarkers in Alzheimer's Disease Drug Development
The Role of Biomarkers in Alzheimer's Disease Drug Development
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DOI：
10.1007/978-3-030-05542-4_2
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发表时间：
2019-01-01
期刊：
影响因子：
--
通讯作者：
Cummings, Jeffrey
中科院分区：
文献类型：
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作者：
Cummings, Jeffrey
生物标志物在阿尔茨海默病 (AD) 药物开发中发挥着关键作用。生物标志物可以协助诊断、展示目标参与、支持疾病修饰和监测安全性。淀粉样蛋白 (A)、tau (T)、神经变性 (N) 研究框架强调与疾病诊断和分期相关的脑成像和 CSF 测量，可应用于药物开发和临床试验。在将候选治疗方案推进到第 3 阶段之前，证明第 2 阶段的目标参与至关重要。生物标志物结果的试验比显示临床益处所需的试验更短、更小，并且对于了解药物的生物学影响并为是否进行决定提供信息非常重要。安全有效地使用治疗需要伴随诊断，并且可能出现在 AD 药物开发项目中。补充生物标志物为治疗的使用提供信息，但不是强制使用的。生物标志物有望降低 AD 药物开发的风险，吸引赞助商参与 AD 研究，并加速为患有 AD 或有 AD 风险的患者提供新药。
Biomarkers have a key role in Alzheimer's disease (AD) drug development. Biomarkers can assist in diagnosis, demonstrate target engagement, support disease modification, and monitor for safety. The amyloid (A), tau (T), neurodegeneration (N) Research Framework emphasizes brain imaging and CSF measures relevant to disease diagnosis and staging and can be applied to drug development and clinical trials. Demonstration of target engagement in Phase 2 is critical before advancing a treatment candidate to Phase 3. Trials with biomarker outcomes are shorter and smaller than those required to show clinical benefit and are important to understanding the biological impact of an agent and inform go/no-go decisions. Companion diagnostics are required for safe and effective use of treatments and may emerge in AD drug development programs. Complementary biomarkers inform the use of therapies but are not mandatory for use. Biomarkers promise to de-risk AD drug development, attract sponsors to AD research, and accelerate getting new drugs to those with or at risk for AD.