Boosting UBA1 enzyme activity to treat VEXAS
Pharmacologic enhancement of UBA1 activity in models of VEXAS syndrome
This project tests whether the drug auranofin can boost the faulty UBA1 enzyme in adults with VEXAS syndrome.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Maryland Baltimore NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project ID | NIH-11126050 on NIH RePORTER |
What this research studies
This work looks at whether auranofin — a drug already used for rheumatoid arthritis — can help the defective UBA1 enzyme that causes VEXAS. In lab models and cells carrying UBA1 mutations, researchers will examine how auranofin increases ubiquitination and speeds the removal of damaged proteins. They will study effects on mutant immune cells and test drug concentrations that are lower than standard arthritis doses. These steps are intended to determine whether auranofin or similar compounds could become treatments for people with VEXAS.
Who could benefit from this research
Good fit: Adults diagnosed with VEXAS syndrome or those known to carry pathogenic UBA1 mutations would be the most relevant candidates to participate or benefit from future trials.
Not a fit: People without UBA1 mutations, children, or patients whose symptoms are caused by other diseases are unlikely to benefit from this specific research.
Why it matters
Potential benefit: If successful, this approach could restore UBA1 function and reduce the inflammation and complications that make VEXAS severe or life-threatening.
How similar studies have performed: Early laboratory data show auranofin can increase UBA1 activity in mutant cells, but clinical benefit for people with VEXAS has not yet been demonstrated.
Where this research is happening
Baltimore, United States
- University of Maryland Baltimore — Baltimore, United States (Active)
Researchers
- Principal investigator: Fang, Shengyun — University of Maryland Baltimore
- Study coordinator: Fang, Shengyun
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.