How stathmin-2 helps nerve fibers stay healthy and regrow
Mechanism of stathmin-2-dependent axon maintenance, regeneration, and function
The team is looking at whether restoring a protein called stathmin‑2 can protect and help regrow nerve fibers in conditions linked to TDP‑43 such as ALS, frontotemporal dementia, and some Alzheimer's cases.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11133842 on NIH RePORTER |
What this research studies
This project studies how the protein stathmin‑2 keeps axons (nerve fibers) healthy, how its loss contributes to TDP‑43 related neurodegeneration, and whether fixing stathmin‑2 can restore nerve function. Scientists will use laboratory experiments, cell and animal models, and analyses of human brain or spinal cord tissue to map the molecular steps that lead from TDP‑43 dysfunction to stathmin‑2 loss and axon damage. They are also testing antisense oligonucleotides (ASOs) that can bring stathmin‑2 levels back up in nervous tissue to see if this improves nerve maintenance and regeneration in models. The goal is to build the knowledge needed to guide future clinical therapies and potential biomarkers for people with ALS, FTD, and TDP‑43 positive Alzheimer's disease.
Who could benefit from this research
Good fit: People with TDP‑43 proteinopathies—such as many ALS patients, some frontotemporal dementia patients, and a subset of Alzheimer's or LATE patients—would be the primary group who could benefit or be eligible for future trials.
Not a fit: People whose condition does not involve TDP‑43 dysfunction or whose nerve damage has progressed beyond repair are less likely to benefit from stathmin‑2–targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to therapies that protect or restore nerve fibers and slow or reverse symptoms in people with TDP‑43 related diseases like ALS and some forms of dementia.
How similar studies have performed: Restoring proteins with antisense oligonucleotides has worked in other neurological diseases (for example, nusinersen in spinal muscular atrophy), and preclinical work already shows ASOs can restore stathmin‑2 in models, but human trials for stathmin‑2 are not yet established.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Cleveland, Don W — University of California, San Diego
- Study coordinator: Cleveland, Don W
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.