# Biomarker Discovery for Nonalcoholic Fatty Liver Disease in Children

> **NIH NIH R01** · EMORY UNIVERSITY · 2020 · $354,252

## Abstract

Project Abstract
Nonalcoholic steatohepatitis (NASH) is the aggressive form of nonalcoholic fatty liver disease, the most common
liver disease in children in the United States. The disease affects almost 1/3 of all obese children, estimated to
be approximately 7 million children. Treatment is based on lifestyle changes such as a low sugar diet and
increasing exercise, but no therapeutics are approved for children or adults. Currently, diagnosis of NASH
requires a liver biopsy, which is cumbersome, expensive and has inherent risk. The lack of 1) non-invasive
biomarkers to diagnose NASH and 2) pharmacodynamic/response biomarkers of NASH to therapy are
considered by many to be an important barrier in the field. These biomarkers are needed to advance clinical
care, focusing resources on the children with the most progressive form of the disease. And they are needed to
drive therapeutic development because children with NASH are prioritized for participation in clinical trials and
response biomarkers for NASH are needed to become surrogate endpoint biomarkers for clinical trials to
accelerate drug development. This proposal represents an exceptional multidisciplinary effort to address these
important gaps in knowledge. The project will be led by Dr. Miriam Vos, expert in pediatric NAFLD and
experienced in early biomarker development research. Co-investigators including Dr. Kristal Maner-Smith,
expert in lipidomics and Dr. Ayman Akil, expert in machine learning and biomarker discovery. The team seeks
to develop a panel of biomarkers to predict NASH and to reflect response of NASH to therapy through the 2
following aims. Aim 1 will define and validate a panel of metabolites and lipids diagnostic of NASH in children.
Aim 2 will define and validate a panel of metabolites, lipids and clinical variables that are associated with
treatment induced improvement in NASH. Aims 1 and 2 will both capitalize on the exceptionally high-quality
samples available from the NASH clinical research network and deposited within the NIDDK Central Repository.
Repository serum samples are available from two pediatric clinical studies including the NAFLD Database study
and the TONIC randomized clinical trial and were collected near in time to a liver biopsy as well as with detailed
clinical phenotyping. Validation cohort and control samples will be drawn from the Emory Liver Biopsy
Biorepository study and an ongoing Healthy Control pediatric cohort at Emory. Preliminary data demonstrates
strong associations between specific metabolites and pathologic features of NASH including hepatocyte
ballooning, inflammation and steatosis supporting feasibility. These studies are designed to have a sustained,
powerful impact on the pediatric NASH biomarker field and to directly improve the health of children through
efficient diagnosis of NASH and successful therapeutic development for NASH.

## Key facts

- **NIH application ID:** 10119937
- **Project number:** 1R01DK125701-01A1
- **Recipient organization:** EMORY UNIVERSITY
- **Principal Investigator:** MIRIAM B. VOS
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $354,252
- **Award type:** 1
- **Project period:** 2020-09-16 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10119937

## Citation

> US National Institutes of Health, RePORTER application 10119937, Biomarker Discovery for Nonalcoholic Fatty Liver Disease in Children (1R01DK125701-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10119937. Licensed CC0.

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