Developing new therapies for ALS and related neurodegenerative diseases
Official title: Multi-Targeting Oligonucleotide Therapeutics for Familial and Sporadic Amyotrophic Lateral Sclerosis
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11092284 · Data updated 2026-11-12
This study is looking for new ways to treat neurodegenerative diseases like ALS and Alzheimer's by using special medicines that target specific genes, and it invites patients to join trials to explore these exciting new options.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | May 15, 2019 |
| Project end | May 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $665,737 |
| NIH project number | 5R01NS111990-07 |
| Project ID | NIH-11092284 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with familial or sporadic ALS and other related neurodegenerative disorders.
✕Less likely to help
Patients with neurodegenerative diseases not related to ALS or those who do not have a genetic component may not benefit from this research.
What this research studies
This research focuses on creating innovative treatments for neurodegenerative diseases like ALS and Alzheimer's by targeting specific genes responsible for these conditions. The approach involves using RNA-targeted medicines, such as antisense oligonucleotides, to suppress harmful gene expressions. The study aims to address the limitations of current therapies, particularly their toxicity and the need for multi-target strategies to effectively manage complex diseases. Patients may have the opportunity to participate in trials that explore these new therapeutic options.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for ALS and other neurodegenerative diseases, potentially improving patient outcomes and quality of life.
How similar studies have gone: Previous research has shown promise with RNA-targeted therapies in treating other neurodegenerative conditions, indicating a potential for success in this novel approach.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Watts, Jonathan K — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Watts, Jonathan K
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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.