Fluid Biomarkers in Clinical Trials for Alzheimer's Disease: Current and Future Application

Alzheimer's disease (AD) research is entering a unique moment in which enormous information about the molecular basis of this disease is being translated into therapeutics. However, almost all drug candidates have failed in clinical trials over the past 30 years. These many trial failures have highlighted a need for the incorporation of biomarkers in clinical trials to help improve the trial design. Fluid biomarkers measured in cerebrospinal fluid and circulating blood, which can reflect the pathophysiological process in the brain, are becoming increasingly important in AD clinical trials. In this review, we first succinctly outline a panel of fluid biomarkers for neuropathological changes in AD. Then, we provide a comprehensive overviewof current and future application of fluid biomarkers in clinical trials for AD. We also summarize the many challenges that have been encountered in efforts to integrate fluid biomarkers in clinical trials, and the barriers that have begun to be overcome. Ongoing research efforts in the field of fluid biomarkers will be critical to make significant progress in ultimately unveiling disease-modifying therapies in AD.

Automated summary

AD research is currently at a crucial point where the molecular understanding of the disease is being translated into potential therapies, despite previous failures in drug development. Biomarkers measured in cerebrospinal fluid and blood are playing an increasingly important role in clinical trials for AD, reflecting the pathophysiological processes in the brain. Challenges in incorporating these biomarkers into trials have been acknowledged, but progress is being made to overcome these obstacles. Ongoing efforts in this area will be crucial for the development of disease-modifying treatments for AD.