# Advancing drug repositioning and development for Alzheimer's Disease using functional genomics and computational phenomics

> **NIH NIH R56** · VANDERBILT UNIVERSITY MEDICAL CENTER · 2021 · $755,981

## Abstract

PROJECT SUMMARY
Alzheimer’s disease (AD) is a heterogeneous neurodegenerative disorder which is highly
prevalent in the global population, affecting around 46 million individuals. Due to increased
longevity, the prevalence is rising. AD is characterized pathophysiologically by extracellular β-
amyloid deposition and intracellular hyperphosphorylated tau. Current medications for AD
provide some relief from disease symptoms but are not effective in all patients while severe side
effects are observed. The disease is highly heritable and has a complex molecular basis, with a
substantial polygenic component.
There is an urgent need for developing approaches to target discovery and drug repurposing.
The combination of methodological analytic advances, functional genomics data, and
phenotype-rich datasets can be the foundation for identifying new therapies. Our central
hypothesis is that an integrative approach spanning human phenomics and genomics will offer
new AD therapeutic possibilities, including drug repositioning, and provide a powerful approach
to drug target discovery. We will develop cutting-edge genetics-anchored computational
methodologies for AD drug repurposing, generate a community resource to advance research in
AD therapeutics development, leverage a collaboration (infrastructure and machine learning)
with Google, and conduct target validation in a zebrafish model of AD.
We have engaged an interdisciplinary team to ensure the success and broad impact of our
proposal and lay the groundwork for future innovative research.

## Key facts

- **NIH application ID:** 10459749
- **Project number:** 1R56AG068026-01A1
- **Recipient organization:** VANDERBILT UNIVERSITY MEDICAL CENTER
- **Principal Investigator:** Eric R Gamazon
- **Activity code:** R56 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $755,981
- **Award type:** 1
- **Project period:** 2021-09-05 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10459749

## Citation

> US National Institutes of Health, RePORTER application 10459749, Advancing drug repositioning and development for Alzheimer's Disease using functional genomics and computational phenomics (1R56AG068026-01A1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10459749. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*