Restoring lysosome acidity to help fatty liver (MASLD)
Lysosomal Regeneration (LysoGen) and Rescue as a Treatment for Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
['FUNDING_R01'] · BOSTON UNIVERSITY (CHARLES RIVER CAMPUS) · NIH-11195154
Researchers are developing an acid-releasing therapy to help liver cells clear fat buildup in people with metabolic-dysfunction associated steatotic liver disease (MASLD).
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BOSTON UNIVERSITY (CHARLES RIVER CAMPUS) (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11195154 on ClinicalTrials.gov |
What this research studies
This project develops LysoGen, an injectable polymer designed to release acid inside your liver cells' lysosomes so they can regain normal acidity and resume clearing damaged material and excess fat. The therapy contains a strong diacid (tetrafluorosuccinic acid, TFSA) built into a biodegradable polyester copolymer that is activated by the mildly acidic lysosomal environment. It is designed to sense lysosomes with pH around 5.7–6 and lower the pH to a healthier 4–5, restoring autophagic flux. Researchers will first test the approach in laboratory and preclinical models with the goal of advancing to human testing to see if liver fat and autophagosome buildup are reduced.
Who could benefit from this research
Good fit: Adults with metabolic-dysfunction associated steatotic liver disease (MASLD), especially middle-aged and older individuals with fatty liver related to metabolic risk factors, would be the likely candidates.
Not a fit: People without MASLD, those whose liver disease is primarily due to alcohol, or patients with advanced cirrhosis are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this approach could help liver cells clear excess fat and damaged material, potentially slowing or reversing MASLD progression.
How similar studies have performed: Targeting lysosomal acidification is a novel strategy with promising laboratory results for autophagy-related problems but has limited clinical testing to date.
Where this research is happening
BOSTON, UNITED STATES
- BOSTON UNIVERSITY (CHARLES RIVER CAMPUS) — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GRINSTAFF, MARK W. — BOSTON UNIVERSITY (CHARLES RIVER CAMPUS)
- Study coordinator: GRINSTAFF, MARK W.
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.