Current Status and Future Prospects of Drug Target Discovery for Alzheimer Disease

Abstract

The approval of Lecanemab, a monoclonal antibody that targets amyloid-β (Aβ) aggregates, marks a major milestone in drug discovery research for Alzheimer disease (AD). It confirms that Aβ is a key molecular driver of AD pathogenesis and a valid therapeutic target. However, it has also been shown that clearing Aβ aggregates from the brain does not fully stop the progression of AD. Therefore, new approaches are needed in addition to anti-Aβ antibodies. One promising avenue is targeting aggregated tau, another pathological hallmark of AD that is directly linked to neurodegeneration. Moreover, large-scale observational studies and clinical trials with anti-Aβ antibodies, as well as advances in biomarker development, have revealed the potential role of glial cells as a new target for drug discovery. Furthermore, drug discovery strategies for patients with advanced neurodegeneration are still lacking. In this presentation, I will summarize the current status and future challenges of AD drug discovery.