Using plasma neurofilament light chain to monitor disease in Wolfram syndrome
Official title: Plasma neurofilament light chain as a potential disease monitoring biomarker in Wolfram syndrome
Funded at Washington University · NIH-10727328 · Data updated 2026-11-12
This study is looking at whether a blood test for a protein called neurofilament light chain (NfL) can help track how Wolfram syndrome progresses, making it easier and cheaper for people with the condition to monitor their health over time.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | September 5, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $427,625 |
| NIH project number | 1R21HD112910-01 |
| Project ID | NIH-10727328 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Wolfram syndrome, particularly those who have experienced childhood-onset type 1 diabetes.
✕Less likely to help
Patients without Wolfram syndrome or those with other unrelated conditions may not benefit from this research.
What this research studies
This research investigates the potential of plasma neurofilament light chain (NfL) as a biomarker for monitoring disease progression in Wolfram syndrome, a rare disorder that often begins with childhood-onset type 1 diabetes. The study aims to determine if NfL levels in the blood can provide a reliable and cost-effective way to track neurodegeneration associated with the syndrome, compared to traditional clinical measures that are more complex and resource-intensive. By analyzing blood samples from participants, researchers hope to establish a clearer understanding of how NfL levels correlate with disease severity and progression over time.
Why it matters
Potential benefit: If successful, this research could provide a simpler and more accessible method for monitoring disease progression in patients with Wolfram syndrome.
How similar studies have gone: Previous research has shown that biofluid markers like NfL can be effective in other neurodegenerative diseases, suggesting potential for success in this novel application.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Hershey, Tamara G — Washington University
- Study coordinator: Hershey, Tamara G
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.