Microglia's role in C9orf72-linked ALS and frontotemporal dementia

Microglia contribution to disease pathogenesis in C9orf72 ALS/FTD

NIH-funded research St. Joseph's Hospital and Medical Center · NIH-11377868

This work looks at whether immune cells in the brain called microglia drive nerve-cell and synapse loss in people with C9orf72-related ALS and frontotemporal dementia.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionSt. Joseph's Hospital and Medical Center NIH-funded
Lab location1 site (Phoenix, United States)
Project IDNIH-11377868 on NIH RePORTER

What this research studies

From a patient's viewpoint, researchers will compare brain tissue from people with C9orf72 genetic changes to other patient groups to see how microglia behave. They will measure microglial activation and look for signs of synapse loss in affected brain regions tied to thinking and behavior. Laboratory models will be used to test whether altered microglia–neuron interactions cause the synapse pruning that may underlie cognitive problems. The team aims to link specific microglial changes to the cognitive and behavioral features seen in some ALS/FTD patients.

Who could benefit from this research

Good fit: Ideal candidates would be people with ALS or frontotemporal dementia who carry the C9orf72 genetic mutation or families willing to contribute clinical data or tissue for research.

Not a fit: People without C9orf72 mutations or those with unrelated medical conditions are unlikely to see direct benefits from this specific project.

Why it matters

Potential benefit: If successful, this work could point to new targets to slow or prevent synapse loss and cognitive decline in people with C9orf72-linked ALS/FTD.

How similar studies have performed: Previous studies have linked microglial activation and synapse loss to cognitive decline in Alzheimer's and some ALS/FTD cases, but focusing on the C9orf72 mechanism is relatively new.

Where this research is happening

Phoenix, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Alzheimer disease dementiaAlzheimer syndromeAlzheimer's DiseaseAlzheimer's disease and related dementiaAlzheimer's disease and related disorders
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.