Developing small molecules to treat TDP-43 related brain diseases
Official title: Developing small molecule therapeutics for TDP-43 associated neurodegenerative diseases
Funded at University of Arizona · NIH-11617893 · Data updated 2026-11-12
A potential new drug candidate (XL20) for diseases like Alzheimer’s and other dementias linked to TDP-43 protein problems is being developed and refined for people who live with these conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Arizona NIH-funded |
| Lab location | 1 site (Tucson, United States) |
| Project start | September 15, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $639,943 |
| NIH project number | 1R56AG097418-01 |
| Project ID | NIH-11617893 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with Alzheimer’s disease dementia or related dementias who are affected by TDP-43 proteinopathy may benefit from future therapies developed through this program.
✕Less likely to help
People whose condition is not driven by TDP-43 toxicity or who cannot participate in drug development studies may not benefit directly.
What this research studies
This grant focuses on turning a promising compound, XL20, into a stronger, safer drug candidate by using structure-based design. Researchers will optimize XL20 derivatives to improve potency and therapeutic effects in models of TDP-43 related dementia and related neurodegenerative diseases. The work aims to advance from preclinical findings to more rigorous testing that could eventually support human trials.
Why it matters
Potential benefit: If successful, this could lead to a new treatment that reduces disease features and progression in patients with TDP-43 related dementias.
How similar studies have gone: Prior studies showed XL20’s preclinical promise, but clinical efficacy in humans remains to be demonstrated.
Where this research is happening
-
University of Arizona
Tucson, United States Active
Researchers
- Principal investigator: Wang, Xinglong — University of Arizona
- Study coordinator: Wang, Xinglong
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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.