The Alzheimer's disease (AD) research field has entered a new era, where our fundamental understanding of the pathophysiology of AD and advances in biomarkers have not only allowed for earlier, timely, and accurate detection and diagnosis of the disease, but that amyloid removal has been shown to be associated with signals of slowing cognitive and functional decline. Although recent FDA-approved amyloid plaque-lowering monoclonal antibody therapies have shifted the trajectory of AD, additional treatment options will be key to further slowing clinical decline or stopping disease progression. Thus, new and emerging therapies for AD have created an evolving therapeutic landscape. The Alzheimer's Association Research Roundtable (AARR) Spring meeting held on May 23–34, 2023, brought together a broad array of scientific leaders from the AARR membership, academia, industry, and government and regulatory agencies to discuss the future of clinical trials in an era of regulator-approved amyloid-targeting therapies. Participants discussed lessons learned from other neurological diseases where disease-modifying treatments were first approved and key considerations for future clinical trials, for example, trial population real-world representativeness, duration, biomarker screening, efficacy endpoints, combination therapy, as well as overall trial design and the ethical and equity concerns that clinicians, patients, and their families face when considering clinical trial participation.
Measurements of health-related quality of life (HRQoL) are important for capturing disease impact beyond physical health and relative to other diseases but have rarely been assessed in primary progressive aphasia (PPA).
HRQoL was characterized overall, by sex and subtype in PPA (n = 118) using the Health Utilities Index-2/3 (HUI2/3). Multiple linear regression assessed associations between HRQoL and language severity.
Multi-attribute HUI2/3 summary scores indicated moderate to severe impairment. Scores did not differ by sex and were more severe for semantic than non-semantic PPA. Language severity scores showed significant associations with HUI multi-attribute scores and select single-attribute measures (hearing, sensation, cognition, and speech) with less language impairment associated with better functional capacity related to HRQoL.
This study identified poor HRQoL in a relatively large PPA cohort. HRQoL measures aid in determining patient perspective, policy decision making, and resource allocation. Results may be used to advocate for PPA support.
The Alzheimer's disease (AD) research community continues to make great strides in expanding approaches for early detection and treatment of the disease, including recent advances in our understanding of fundamental AD pathophysiology beyond the classical targets: beta-amyloid and tau. Recent clinical trial readouts implicate a variety of non-amyloid/non-tau (NANT) approaches that show promise in slowing cognitive decline for people with AD. The Alzheimer's Association Research Roundtable (AARR) meeting held on December 13–14, 2022, reviewed the current state of NANT targets on underlying AD pathophysiology and their contribution to cognitive decline, the current data on a diverse range of NANT biomarkers and therapeutic targets, and the integration of NANT concepts in clinical trial designs. Participants also discussed the current definition of therapies that target underlying AD pathophysiology, what endpoints best define what is considered a meaningful change beyond the current approved definition for clinical efficacy, and how the recent NANT findings should inform the development of future guidelines for AD classification and personalized treatment strategies.
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