Developing a new treatment for ALS and frontotemporal dementia
Official title: Development of a SYF2 antisense oligonucleotide treatment for ALS and FTD
Funded at Acurastem, INC. · NIH-10896120 · Data updated 2026-11-12
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 start | August 1, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $1,402,110 |
| NIH project number | 5R44AG085411-02 |
| Project ID | NIH-10896120 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with ALS or frontotemporal dementia, particularly those with unknown genetic causes.
✕Less likely to help
Patients with ALS or FTD that are caused by specific known genetic mutations may not benefit from this research.
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.
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 gone: 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
-
Acurastem, INC.
Pasadena, United States Active
Researchers
- Principal investigator: Alworth, Samuel V — Acurastem, INC.
- Study coordinator: Alworth, Samuel V
Related research
Other NIH-funded projects on the same topics.
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Developing a treatment for ALS and frontotemporal dementia using UNC13A antisense oligonucleotides
This study is testing a new treatment for people with ALS and frontotemporal dementia that aims to help by targeting a specific gene, which could improve brain health and slow down the progression of these conditions.
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.