Human lab-grown livers to find medicines for metabolic fatty liver disease
Official title: Implementing A QSP Platform to Predict and Test Drugs for Metabolic Associated Fatty Liver Disease Genetic Variants in an iPSC-cell Based Human Biomimetic Liver Microphysiology System
Funded at University of Pittsburgh at Pittsburgh · NIH-11290358 · Data updated 2026-11-12
This project uses computer models and tiny human liver tissues grown from stem cells to find medicines that could help people with metabolic fatty liver disease linked to specific genes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project start | February 1, 2024 |
| Project end | November 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $650,009 |
| NIH project number | 5R01DK135606-03 |
| Project ID | NIH-11290358 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with metabolic dysfunction‑associated fatty liver disease (MAFLD), especially those known to carry genetic variants such as PNPLA3, TM6SF2, MBOAT7, HSD17B13, or MTARC1, are the most likely future candidates to benefit from follow-up trials based on this work.
✕Less likely to help
People without metabolic fatty liver disease or those with very advanced, irreversible liver failure are unlikely to benefit directly from this laboratory-focused project.
What this research studies
From a patient view, researchers will build tiny, blood‑flowing human livers in the lab using stem cells and the exact genetic changes linked to fatty liver disease. They'll combine those lab models with computer simulations to predict which existing or new drugs—or drug combinations—might fix disease features caused by different gene variants. The team will edit genes in the stem‑cell derived liver cells to mimic both risk and protective human variants and watch how the tissues respond at the molecular and functional level. The goal is to point to drug candidates and explain how certain gene changes protect or harm the liver, which could guide future clinical trials.
Why it matters
Potential benefit: If successful, this work could identify new or repurposed medicines tailored to genetic types of metabolic fatty liver disease and speed the path to patient trials.
How similar studies have gone: Related lab‑grown liver models and genetic studies have produced useful biological clues, but combining genome‑edited human iPSC livers with quantitative systems pharmacology to pick drugs is a relatively new and emerging approach.
Where this research is happening
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University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Miedel, Mark T. — University of Pittsburgh at Pittsburgh
- Study coordinator: Miedel, Mark T.
Related research
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A large NIH grant helps researchers study why fatty liver develops in Hispanic adults and how genetics interact with lifestyle to affect risk.
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Glutamine and T cells in fatty liver disease
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.