Cell and tissue changes that lead to sporadic ALS and frontotemporal dementia
Official title: The Physical Biology of Neurodegeneration in Sporadic Amyotrophic Lateral Sclerosis/Frontotemporal Dementia
Funded at Columbia University Health Sciences · NIH-11163326 · Data updated 2026-11-12
This project looks at how physical changes inside cells and in their tissue can make TDP‑43 protein clump in people with sporadic ALS and frontotemporal dementia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 1, 2021 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $1,715,367 |
| NIH project number | 5R01NS127186-05 |
| Project ID | NIH-11163326 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with sporadic ALS or ALS‑FTD (and people willing to donate relevant tissue samples) would be most relevant to this work and any future related trials.
✕Less likely to help
People whose disease is caused by known familial genetic mutations or who do not have TDP‑43 pathology may not directly benefit from findings focused on sporadic TDP‑43 mechanisms.
What this research studies
Researchers will examine why normally functioning proteins like TDP‑43 begin to aggregate in sporadic ALS/FTD by focusing on physical changes at the cell and tissue level. They will study altered molecular crowding inside cells and how mechanical changes in the tissue microenvironment, such as stiffening, may trigger protein buildup. Work will compare different cell types to explain selective neuronal vulnerability and use laboratory models alongside human tissue samples to track how protein homeostasis affects cell-to-cell communication. The goal is to create a new framework linking biophysical changes to the clinical ALS‑FTD spectrum.
Why it matters
Potential benefit: If successful, this work could reveal new targets to prevent or reverse harmful protein aggregation and lead to therapies that slow or stop ALS/FTD progression.
How similar studies have gone: Related basic‑science studies on protein aggregation and cell mechanics have provided useful insights, but translating these biophysical findings into effective treatments remains largely unproven.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Phatnani, Hemali — Columbia University Health Sciences
- Study coordinator: Phatnani, Hemali
Related research
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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.