Creating a new oral treatment for ALS
Official title: Development of a novel oral brain penetrant therapeutic for ALS disease
Funded at Aquinnah Pharmaceuticals, INC. · NIH-11186238 · Data updated 2026-11-12
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 start | June 15, 2025 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,499,212 |
| NIH project number | 1R44NS141531-01A1 |
| Project ID | NIH-11186238 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS who are experiencing early to moderate symptoms of the disease.
✕Less likely to help
Patients with advanced ALS or those who have not been diagnosed with the disease may not benefit from this research.
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.
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 gone: 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
-
Aquinnah Pharmaceuticals, INC.
Newton, United States Active
Researchers
- Principal investigator: Larsen, Glenn — Aquinnah Pharmaceuticals, INC.
- Study coordinator: Larsen, Glenn
Related research
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Oral brain-penetrant therapy for ALS that may slow or reverse progression
A promising new pill designed to reach the brain and slow or reverse ALS progression might help people living with ALS.
-
A new approach to treating Alzheimer's disease by targeting tau protein
This research is developing new medicines to help people with Alzheimer's disease by focusing on a specific protein called tau.
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.