Investigating a new treatment approach for a genetic form of ALS and frontotemporal dementia.
Official title: Targeting Inflammasome Mediated Neuronal-Microglial Crosstalk as a Therapeutic Substrate in c9orf72 FTD/ALS
Funded at James J Peters VA Medical Center · NIH-10701143 · Data updated 2026-11-12
This study is looking at how a specific gene change might affect ALS and frontotemporal dementia, and it aims to see if targeting inflammation in the body can help slow down the disease and improve symptoms, especially for those with the C9orf72 mutation.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | James J Peters VA Medical Center NIH-funded |
| Lab location | 1 site (Bronx, United States) |
| Project start | April 1, 2024 |
| Project end | March 31, 2028 |
| Funding year | 2025 |
| NIH project number | 1I01BX005907-01A2 |
| Project ID | NIH-10701143 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS or FTD who carry the C9orf72 genetic mutation.
✕Less likely to help
Patients with sporadic forms of ALS or FTD without the C9orf72 mutation may not benefit from this research.
What this research studies
This research focuses on understanding how a specific genetic mutation (C9orf72) contributes to the development of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study aims to explore the role of neuroinflammation in these conditions and evaluate the effectiveness of targeting certain inflammatory pathways to improve patient outcomes. By using animal models, researchers will assess how manipulating these pathways can potentially slow disease progression and alleviate symptoms. This research is particularly relevant for patients with the C9orf72 mutation, which is linked to more severe disease manifestations.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies that significantly improve the quality of life for patients with ALS and FTD.
How similar studies have gone: Previous research has shown promise in targeting neuroinflammation for ALS and FTD, but this specific approach focusing on C9orf72 is relatively novel.
Where this research is happening
-
James J Peters VA Medical Center
Bronx, United States Active
Researchers
- Principal investigator: Pasinetti, Giulio Maria — James J Peters VA Medical Center
- Study coordinator: Pasinetti, Giulio Maria
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.