Developing a new treatment for ALS and frontotemporal dementia
Development of a SYF2 antisense oligonucleotide treatment for ALS and FTD
This study is testing a new treatment that aims to help people with ALS and frontotemporal dementia by reducing a harmful protein in the brain, which could lead to better options for those affected by these conditions.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Acurastem, INC. NIH-funded |
| Lab location | 1 site (Pasadena, UNITED STATES) |
| Project ID | NIH-10896120 on NIH RePORTER |
What this research studies
This research focuses on creating a SYF2 antisense oligonucleotide treatment aimed at addressing the underlying causes of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The approach targets the depletion of a protein called TDP-43, which is known to play a critical role in the neurodegeneration associated with these diseases. By developing a treatment that can potentially rescue multiple forms of ALS and FTD, especially those with unknown genetic causes, the research aims to provide a broader therapeutic option for patients. The methodology involves advanced genetic techniques to modify RNA and restore normal cellular function.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with ALS or frontotemporal dementia, particularly those with unknown genetic causes.
Not a fit: Patients with ALS or FTD that are caused by specific known genetic mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment option that addresses a wide range of ALS and FTD cases, potentially improving patient outcomes.
How similar studies have performed: While there have been successful approaches targeting specific genetic mutations in ALS and FTD, this research is exploring a novel strategy that could address a broader range of cases.
Where this research is happening
Pasadena, UNITED STATES
- Acurastem, INC. — Pasadena, United States (Active)
Researchers
- Principal investigator: Alworth, Samuel V — Acurastem, INC.
- Study coordinator: Alworth, Samuel V
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.