Developing a treatment for ALS and frontotemporal dementia using UNC13A antisense oligonucleotides
Official title: Development of an UNC13A antisense oligonucleotide treatment for ALS and FTD
Funded at Acurastem, INC. · NIH-10914945 · Data updated 2026-11-12
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.
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) | $1,279,990 |
| NIH project number | 5R44NS132698-02 |
| Project ID | NIH-10914945 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with ALS or FTD, particularly those with genetic risk factors related to the UNC13A gene.
✕Less likely to help
Patients with other forms of neurodegenerative diseases that do not involve TDP-43 pathology may not benefit from this research.
What this research studies
This research focuses on creating a new treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) by using antisense oligonucleotides to target the UNC13A gene. The approach aims to suppress the inclusion of cryptic exons in UNC13A mRNA, which is linked to the progression of these diseases. By addressing the underlying genetic factors associated with ALS and FTD, the treatment could potentially restore normal protein function and improve neuronal health. Patients will be monitored for changes in symptoms and overall health as the treatment is developed and tested.
Why it matters
Potential benefit: If successful, this research could lead to a novel therapy that slows the progression of ALS and FTD, improving quality of life for patients.
How similar studies have gone: Previous research has shown promise in targeting genetic factors in neurodegenerative diseases, suggesting that this approach could be effective.
Where this research is happening
-
Acurastem, INC.
Pasadena, United States Active
Researchers
- Principal investigator: Chang, Wen-Hsuan — Acurastem, INC.
- Study coordinator: Chang, Wen-Hsuan
Related research
Other NIH-funded projects on the same topics.
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Developing a new treatment for ALS and frontotemporal dementia
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.
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.