Blocking PASK to protect nerve cells in ALS and frontotemporal dementia
PASK-regulated genes and proteins as therapeutic targets for ALS and FTD
See if blocking a protein called PASK can reduce harmful TDP‑43 protein changes in people with ALS or frontotemporal dementia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Utah State Higher Education System--University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project ID | NIH-11195087 on NIH RePORTER |
What this research studies
This work looks at a protein called PASK that helps control the cell's cleaning system (autophagy) and how it affects TDP‑43, a protein linked to nerve cell damage. Researchers will use lab-grown cells and mouse models with disease-related mutations and will lower PASK using genetic tools or a drug-like inhibitor called BioE‑115. They will measure whether reducing PASK restores normal autophagy, lowers STAU1 and TDP‑43 problems, and improves neuron health. If those results hold up, the findings could point toward new PASK‑targeting treatments for ALS and frontotemporal dementia.
Who could benefit from this research
Good fit: People diagnosed with ALS or frontotemporal dementia—especially those whose disease shows TDP‑43‑related protein changes—would be the likely candidates for eventual clinical testing.
Not a fit: Because this is primarily preclinical work, patients seeking immediate treatment or those with dementia driven by non‑TDP‑43 mechanisms should not expect direct benefit from this grant.
Why it matters
Potential benefit: If successful, this could point to new treatments that reduce TDP‑43 proteinopathy and help protect neurons in ALS and FTD.
How similar studies have performed: Early studies in related genetic ataxia models showed that lowering PASK improved autophagy and neuronal markers, but applying PASK targeting to ALS/FTD and TDP‑43 pathology is a relatively new approach.
Where this research is happening
Salt Lake City, United States
- Utah State Higher Education System--University of Utah — Salt Lake City, United States (Active)
Researchers
- Principal investigator: Scoles, Daniel R — Utah State Higher Education System--University of Utah
- Study coordinator: Scoles, Daniel R
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.