How brain immune cells (microglia) affect C9orf72-linked ALS and frontotemporal dementia
Official title: Microglia contribution to disease pathogenesis in C9orf72 ALS/FTD
Funded at St. Joseph's Hospital and Medical Center · NIH-11159585 · Data updated 2026-11-12
Researchers are looking at whether brain immune cells called microglia cause synapse loss and thinking or behavior problems in people with C9orf72-linked ALS and frontotemporal dementia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Joseph's Hospital and Medical Center NIH-funded |
| Lab location | 1 site (Phoenix, United States) |
| Project start | September 1, 2021 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $788,244 |
| NIH project number | 5R01NS120331-05 |
| Project ID | NIH-11159585 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be people with ALS or frontotemporal dementia who carry the C9orf72 mutation or family members willing to contribute clinical information or brain/tissue donations.
✕Less likely to help
People whose dementia is caused by unrelated conditions or who do not carry C9orf72 changes are less likely to see direct benefits from this specific research.
What this research studies
If you or a family member have the C9orf72 genetic change linked to ALS or frontotemporal dementia, this work studies how your brain's immune cells (microglia) might drive loss of connections between nerve cells that relate to thinking and behavior. The team will examine human brain tissue and compare findings across genetic subgroups, and they will use laboratory models to observe microglia–neuron interactions and test how changing microglia behavior affects synapses. By comparing C9orf72 cases to other genetic forms, they aim to find whether specific microglia changes explain the cognitive symptoms and point to targets for future therapies.
Why it matters
Potential benefit: If successful, this work could identify microglia-related targets to protect synapses and slow cognitive decline for people with C9orf72-linked ALS/FTD.
How similar studies have gone: Previous studies show microglia activation and increased synapse loss in dementia and ALS with cognitive symptoms, but microglia-targeted treatments remain largely unproven in humans.
Where this research is happening
-
St. Joseph's Hospital and Medical Center
Phoenix, United States Active
Researchers
- Principal investigator: Sattler, Rita — St. Joseph's Hospital and Medical Center
- Study coordinator: Sattler, Rita
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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.