Developing targeted therapies for patients with rare forms of ALS
Official title: Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS
Funded at Columbia University Health Sciences · NIH-11034080 · Data updated 2026-11-12
This study is looking to develop special treatments for people with very rare genetic changes that cause ALS, using a new approach that targets those specific mutations, so if you have one of these unique mutations, you might get a personalized therapy just for you!
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | March 1, 2024 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $4,636,637 |
| NIH project number | 5U01NS134684-02 |
| Project ID | NIH-11034080 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are ALS patients with ultra-rare genetic mutations, particularly those affecting the TARDBP gene.
✕Less likely to help
Patients with common forms of ALS or those without identifiable genetic mutations may not benefit from this research.
What this research studies
This research focuses on creating personalized treatments for patients with ultra-rare mutations causing amyotrophic lateral sclerosis (ALS). By utilizing antisense oligonucleotides (ASOs), the project aims to directly target the genetic mutations responsible for ALS in a subset of patients. The collaboration between Columbia University and the n-Lorem Foundation seeks to expand access to these innovative therapies, which have shown promise in treating more common genetic forms of ALS. Patients with specific mutations will be identified and potentially receive tailored therapies designed to address their unique genetic profiles.
Why it matters
Potential benefit: If successful, this research could provide personalized treatment options for patients with rare genetic forms of ALS, potentially improving their quality of life and disease management.
How similar studies have gone: Previous research has shown success in using antisense oligonucleotide therapies for more common ALS mutations, indicating potential for this novel approach in ultra-rare cases.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Shneider, Neil Alan — Columbia University Health Sciences
- Study coordinator: Shneider, Neil Alan
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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.