Targeting a pathway to help with fatty liver disease and related cancers
Selective mTORC1 inhibition to prevent and treat NAFLD and NASH
This research explores a new way to control a specific cell pathway to help people with non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH), which can lead to liver damage and cancer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11097287 on NIH RePORTER |
What this research studies
Many people in the US have non-alcoholic fatty liver disease (NAFLD), which can worsen into non-alcoholic steatohepatitis (NASH), causing liver damage and potentially leading to cirrhosis or liver cancer. Currently, there are no approved treatments for NAFLD or NASH. This project focuses on a cell pathway called mTORC1, which helps control how the liver handles fats. Researchers have found a specific part of this pathway, regulated by the FLCN protein, that could protect the liver by reducing fat production and increasing fat breakdown. We aim to understand how this specific pathway works and confirm if targeting FLCN could be a new treatment for these liver conditions and related cancers.
Who could benefit from this research
Good fit: This research is relevant for individuals with non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), cirrhosis, or hepatocellular carcinoma (HCC).
Not a fit: Patients without non-alcoholic fatty liver disease or related liver conditions would not directly benefit from this specific therapeutic approach.
Why it matters
Potential benefit: If successful, this research could lead to the first FDA-approved treatment for non-alcoholic fatty liver disease and non-alcoholic steatohepatitis, potentially preventing severe liver damage and liver cancer.
How similar studies have performed: While the mTOR pathway has been studied, this research explores a newly uncovered, highly specific branch of mTORC1 signaling, making this a novel approach within a known area.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Arany, Zoltan P — University of Pennsylvania
- Study coordinator: Arany, Zoltan P
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 below.