# Acamprosate pharmacogenomics: iPSC based model of alcohol use disorder

> **NIH NIH K01** · MAYO CLINIC ROCHESTER · 2022 · $129,750

## Abstract

Project Summary/Abstract
This application for an NIAAA K01 Career Development Award proposes studies of the pharmacogenomics of
alcohol use disorder (AUD) and acamprosate treatment response. Our proposed use of AUD patient-derived
induced pluripotent stem cell (iPSC)--an innovative and comprehensive “cell line based” model system will
make it possible to advancing our understanding of both drug action and of disease pathophysiology. These
results might provide novel mechanistic insight into molecular and genomic signatures for AUD as well as
mechanisms underlying individual variation in response to acamprosate. Therefore, the proposed studies
include the following aims, Aim 1: To generate a panel of iPSCs from patient-derived LCLs as a novel cell
model system for functional genomic studies of AUD and its drug therapy. Aim 2: To identify molecular
signatures for alcohol or acamprosate exposure using human neural cells to perform genome-wide
transcriptomic profiles using patient-derived iPSC-derived neuronal and glial cell types differentiated from
iPSCs obtained from AUD patients and healthy controls. Aim 3: To determine molecular signatures for alcohol
and acamprosate exposure, and mechanisms underlying individual variation in those signatures by mapping
targeted metabolomic profiles of patient-derived iPSC. The significance of the proposed studies results from
the societal importance of AUD and of patient derived iPSC-based research, from the possibility of more highly
individualized treatment for AUD, and from the fact that these studies may suggest novel genetic mechanisms
that might influence individual variation in acamprosate response in AUD patients. As a result, the proposed
studies would have both translational and basic implications for genetic mechanisms that might
influence alcohol and acamprosate effect genome-wide and for more highly individualized
acamprosate therapy of AUD.
In summary, Dr. Ho, the applicant, proposes to take advantage of the NIAAA K01 Award mechanism to
broaden her experience with the utilization of multi-omics data (under the direction of her Primary Mentor: Dr.
Richard Weinshilboum), translational pharmacogenomics with a focus on AUD (under the guidance of co-
mentor: Dr. Victor Karpyak), and iPSC technology (under the guidance of co-mentor: Dr. Zhexing Wen). The
resources and mentoring provided by an NIAAA K01 Award would help make it possible for Dr. Ho to achieve
her goal of becoming an independent investigator studying molecular and genomic mechanisms of response to
alcohol exposure using patient-derived iPSCs and pharmacogenomic studies of acamprosate response in
patients with AUD. Receipt of an NIAAA K01 Award would represent a critical step toward Dr. Ho’s ultimate
goal of becoming an independent investigator, for obtaining the additional training required to significantly
expand the nature of the biomedical research contributions that she might ultimately make and to generate
preliminary data in support ...

## Key facts

- **NIH application ID:** 10310405
- **Project number:** 5K01AA028050-03
- **Recipient organization:** MAYO CLINIC ROCHESTER
- **Principal Investigator:** Ming-Fen Ho
- **Activity code:** K01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $129,750
- **Award type:** 5
- **Project period:** 2019-09-20 → 2024-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10310405

## Citation

> US National Institutes of Health, RePORTER application 10310405, Acamprosate pharmacogenomics: iPSC based model of alcohol use disorder (5K01AA028050-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10310405. Licensed CC0.

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