Blocking acid ceramidase to slow liver scarring
Targeting Acid Ceramidase for Hepatic Fibrogenesis
A new oral medicine that blocks the enzyme acid ceramidase is being developed to slow liver scarring in people with chronic liver disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11241079 on NIH RePORTER |
What this research studies
Researchers are designing and improving a pill that stops an enzyme called acid ceramidase, which helps drive scarring in the liver. They use structure-guided medicinal chemistry to create covalent inhibitors with good potency, stability, and once-daily oral properties. The optimized compounds will be tested in mouse models and in human liver tissue slices to see whether they reduce activation of scar-forming cells and lower fibrosis. The project brings together liver disease experts and drug developers to move a promising preclinical lead toward a potential medicine.
Who could benefit from this research
Good fit: People with chronic liver disease and ongoing liver fibrosis who might be eligible for future early-phase clinical trials at centers like UCSF would be the most likely candidates.
Not a fit: Patients without significant fibrosis, with acute liver failure, or whose disease is driven by processes unrelated to acid ceramidase are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this approach could slow or reverse liver fibrosis and lower the risk of progression to liver failure by providing a targeted oral antifibrotic therapy.
How similar studies have performed: Preclinical studies in mice and in precision-cut human liver slices have shown that inhibiting acid ceramidase reduces fibrosis, but this specific oral drug series has not yet been tested in people.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Jo, Hyunil — University of California, San Francisco
- Study coordinator: Jo, Hyunil
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.