Alzheimer disease (AD) is the most common contributor to dementia in the world, but strategies that slow or prevent its clinical progression have largely remained elusive, until recently. This Review highlights the latest advances in biomarker technologies and therapeutic development to improve AD diagnosis and treatment. We review recent results that enable pathological staging of AD with neuroimaging and fluid-based biomarkers, with a particular emphasis on the role of amyloid, tau and neuroinflammation in disease pathogenesis. We discuss the lessons learned from randomized controlled trials, including some supporting the proposal that certain anti-amyloid antibodies slow cognitive decline during the mildly symptomatic phase of AD. In addition, we highlight evidence for newly identified therapeutic targets that may be able to modify AD pathogenesis and progression. Collectively, these recent discoveries—and the research directions that they open—have the potential to move AD clinical care toward disease-modifying treatment strategies with maximal benefits for patients.

阿尔茨海默病（AD）是世界上最常见的痴呆症病因，但直到最近，减缓或预防其临床进展的策略在很大程度上仍然难以捉摸。本综述重点介绍了生物标志物技术和治疗开发的最新进展，以改善 AD 诊断和治疗。我们回顾了最近的结果，这些结果能够利用神经影像学和基于液体的生物标志物对 AD 进行病理分期，特别强调淀粉样蛋白、tau 蛋白和神经炎症在疾病发病机制中的作用。我们讨论了从随机对照试验中吸取的经验教训，包括一些支持某些抗淀粉样蛋白抗体在 AD 轻度症状阶段减缓认知衰退这一提议的经验教训。此外，我们强调了新发现的治疗靶点的证据，这些靶点可能能够改变 AD 的发病机制和进展。总的来说，这些最近的发现以及它们所开辟的研究方向有可能将 AD 临床护理推向疾病缓解治疗策略，为患者带来最大利益。