Targeted progranulin therapy for TDP-43 proteinopathy
Funded at University of Alabama at Birmingham · NIH-11614876 · Data updated 2026-11-12
A potential new therapy aims to raise progranulin levels to help people affected by TDP-43 proteinopathy, a driver of ALS, some forms of FTD, and Alzheimer’s-related changes.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Alabama at Birmingham NIH-funded |
| Lab location | 1 site (Birmingham, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $486,779 |
| NIH project number | 1R56AG097867-01 |
| Project ID | NIH-11614876 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include individuals with ALS, FTD-TDP, or Alzheimer’s disease pathology where TDP-43 proteinopathy is involved, and who could participate in future translational studies.
✕Less likely to help
Patients without TDP-43 proteinopathy or those unable to participate in future trials may not benefit.
What this research studies
This grant focuses on a way to boost progranulin to protect nerve cells that are harmed by TDP-43 protein buildup, which occurs in ALS, many cases of frontotemporal dementia, and some Alzheimer’s disease pathology. Researchers will explore targeted delivery approaches to increase progranulin and understand how this may reduce neuronal damage and clinical decline. The work aims to identify mechanisms, balance benefits with potential side effects, and guide safer, more effective therapies for patients with these conditions. Results could inform future clinical testing in people with relevant neurodegenerative diseases.
Why it matters
Potential benefit: If successful, the therapy could slow or alter disease progression for patients with TDP-43 proteinopathy.
How similar studies have gone: Prior work with progranulin mutations shows potential for neuroprotection, and this project builds on those findings.
Where this research is happening
-
University of Alabama at Birmingham
Birmingham, United States Active
Researchers
- Principal investigator: Arrant, Andrew Emmett — University of Alabama at Birmingham
- Study coordinator: Arrant, Andrew Emmett
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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.