Creating a new oral treatment for ALS
Development of a novel oral brain penetrant therapeutic for ALS disease
This study is testing a new oral medication for people with ALS that aims to help slow down the disease by targeting a harmful protein, which could improve muscle control and make daily life a bit easier.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Aquinnah Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Newton, United States) |
| Project ID | NIH-11186238 on NIH RePORTER |
What this research studies
This research focuses on developing an innovative oral medication aimed at treating Amyotrophic Lateral Sclerosis (ALS), a severe neurodegenerative disease. The approach targets the accumulation of a protein called TDP-43, which is linked to the progression of ALS. By potentially slowing or reversing the disease's effects, this treatment could improve muscle control and overall quality of life for patients. The research involves laboratory testing and clinical trials to assess the safety and efficacy of the new drug candidate.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS who are experiencing early to moderate symptoms of the disease.
Not a fit: Patients with advanced ALS or those who have not been diagnosed with the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that may slow or reverse the progression of ALS, significantly improving patient outcomes.
How similar studies have performed: While there have been various approaches to ALS treatment, this specific method targeting TDP-43 is novel and has not been extensively tested in clinical settings.
Where this research is happening
Newton, United States
- Aquinnah Pharmaceuticals, INC. — Newton, United States (Active)
Researchers
- Principal investigator: Larsen, Glenn — Aquinnah Pharmaceuticals, INC.
- Study coordinator: Larsen, Glenn
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.