Enhancing UBA1 activity to treat VEXAS syndrome
Pharmacologic enhancement of UBA1 activity in models of VEXAS syndrome
['FUNDING_U01'] · UNIVERSITY OF MARYLAND BALTIMORE · NIH-10874714
This study is looking at a new treatment using a drug called auranofin to help people with VEXAS syndrome by boosting the function of a protein that’s not working properly due to a gene mutation.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF MARYLAND BALTIMORE (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-10874714 on ClinicalTrials.gov |
What this research studies
This research investigates a new treatment approach for VEXAS syndrome, a severe autoinflammatory disease caused by mutations in the UBA1 gene. The study focuses on the drug auranofin, which has shown promise in enhancing the activity of the UBA1 protein, potentially restoring its function in patients with this condition. By understanding how auranofin interacts with UBA1, the research aims to develop effective therapies that could improve patient outcomes. The approach involves both laboratory experiments and clinical assessments to evaluate the drug's efficacy in enhancing UBA1 activity.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with VEXAS syndrome who have not responded to existing treatments.
Not a fit: Patients with other autoinflammatory diseases unrelated to UBA1 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new therapeutic option for patients suffering from VEXAS syndrome, potentially improving their quality of life and survival rates.
How similar studies have performed: Preliminary studies have shown that auranofin can enhance UBA1 activity, indicating potential success for this novel treatment approach.
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.