Cell-clearance breakdowns in brain immune cells in frontotemporal dementia
Endolysosomal defects in secretory autophagy and microglial toxicity in FTD
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-11303286
This work explores whether problems with the cell-clearance system in brain immune cells (microglia) lead to nerve cell damage in people with frontotemporal dementia.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-11303286 on ClinicalTrials.gov |
What this research studies
Researchers at UCSF study how the autophagy and endolysosomal systems in microglia and other glial cells go wrong in frontotemporal dementia (FTD). They use laboratory models, molecular analysis, and animal experiments to track how defects change secretion of extracellular vesicles and other cargo (including TDP-43) and how this promotes microglial toxicity. The team focuses on progranulin (GRN) loss and age-dependent endolysosomal defects that appear to drive abnormal secretion and neuron loss. Results aim to reveal molecular steps that could be targeted to reduce harmful microglial activity in FTD.
Who could benefit from this research
Good fit: People with frontotemporal dementia or those with genetic risk for FTD—especially individuals carrying progranulin (GRN) mutations—would be most relevant to this research.
Not a fit: Patients with unrelated types of dementia or those in very late-stage disease may not see direct benefit from these mechanistic, preclinical findings in the short term.
Why it matters
Potential benefit: If successful, this work could point to new targets to prevent microglia-driven neuron loss and guide therapies for frontotemporal dementia.
How similar studies have performed: Prior research has linked autophagy and progranulin loss to neurodegeneration, and targeting microglial pathways shows promise in preclinical models, while the specific role of secretory autophagy (LDELS) in FTD is a newer area of study.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: HUANG, ERIC J — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: HUANG, ERIC J
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 below.