Restoring TDP43 function to treat frontotemporal dementia and ALS
Official title: A novel approach to restore functional TDP43 for the treatment of FTD and ALS
Funded at University of Minnesota · NIH-11167100 · Data updated 2026-11-12
This study is exploring a new way to help people with frontotemporal dementia and ALS by finding small molecules that can keep a protein called TDP43 working properly, so it doesn't cause harm in the body.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Minnesota NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | May 1, 2025 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $404,058 |
| NIH project number | 1R21AG094188-01 |
| Project ID | NIH-11167100 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with frontotemporal dementia, amyotrophic lateral sclerosis, or other TDP43-related disorders.
✕Less likely to help
Patients with conditions unrelated to TDP43 proteinopathies may not benefit from this research.
What this research studies
This research investigates a new approach to restore the normal function of TDP43, a protein linked to frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), and other related conditions. The study aims to identify small molecules that can stabilize TDP43's natural form, preventing its harmful mislocalization and aggregation. Using advanced techniques, the researchers will monitor TDP43 interactions in living cells to find effective treatments. This innovative strategy shifts focus from merely reducing toxic effects to enhancing the protein's beneficial functions.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the quality of life for patients with FTD, ALS, and related dementias.
How similar studies have gone: While there has been significant research on TDP43, this specific approach to restoring its function is novel and has not been extensively tested.
Where this research is happening
-
University of Minnesota
Minneapolis, United States Active
Researchers
- Principal investigator: Sachs, Jonathan N — University of Minnesota
- Study coordinator: Sachs, Jonathan N
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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.