Analytical and Clinical Validation of Biomarkers for Alzheimer's Disease and AD-Related Dementias

NOTE: This funding opportunity expired March 6, 2026. Limited case-by-case submissions may be accepted; contact the eRA Service Desk.

Research Focus

This U01 cooperative agreement supports rigorous validation of candidate biomarkers, composite biomarkers, or biomarker signatures for Alzheimer's disease (AD) and AD-related dementias (ADRD). The National Institute on Aging seeks to close a critical gap: while many candidate biomarkers have been discovered, few progress to validation with established clinical utility. Research must achieve analytical and/or clinical validation rigor comparable to FDA Biomarker Qualification Program (BQP) standards or other FDA regulatory pathways.

Projects should validate biomarkers for use in translational research, clinical trials, medical product development, or clinical practice—with emphasis on understudied and heterogeneous populations. Eligible biomarkers include fluid biomarkers (cerebrospinal fluid, blood), imaging biomarkers, digital phenotyping, and physiologic markers, individually or in combination. Research may address mixed pathologies (AD/Lewy body dementia, AD/frontotemporal dementia, AD/LATE, AD/vascular disease) and the Amyloid-Tau-Neurodegeneration (A-T-N) framework.

Priority topics include: enrichment biomarkers for clinical trial enrollment; longitudinal trajectory studies predicting cognitive decline or treatment response; safety biomarkers; prognostic and monitoring biomarkers in diverse populations; and diagnostic biomarkers differentiating AD subtypes. Analytical validation covers accuracy, precision, sensitivity, specificity, reference intervals, and quality control. Clinical validation must demonstrate the biomarker's ability to identify, measure, or predict meaningful clinical outcomes with defined sensitivity, specificity, area under the curve (AUC), predictive values, and appropriate decision thresholds.

At a Glance

• Who can apply: Not stated (see full FOA for institutional eligibility)
• Funding & duration: Not stated in excerpt
• Award mechanism: U01 Research Project – Cooperative Agreements (milestone-driven; NIA provides significant input on validation strategy)
• Key dates: Applications due February 5, 2025 (earliest); October 5, 2025 and February 5, 2026 (subsequent rounds); expiration March 6, 2026
• Best fit for: Biomarker researchers in neurodegenerative disease with existing candidate biomarkers ready for rigorous analytical or clinical validation; emphasis on diverse populations and real-world generalizability