Expanded access to pridopidine for people with ALS
Official title: An Intermediate-Size Expanded Access Protocol for Amyotrophic Lateral Sclerosis with Pridopidine
Funded at Massachusetts General Hospital · NIH-11189730 · Data updated 2026-11-12
This program offers people living with ALS access to the experimental drug pridopidine while collecting information about safety and how it affects symptoms.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 25, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $7,928,222 |
| NIH project number | 5U01NS136021-03 |
| Project ID | NIH-11189730 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with ALS who meet site-specific eligibility criteria and can attend clinic visits or participate remotely are the most likely candidates to qualify for this program.
✕Less likely to help
People with medical problems or medications that make pridopidine unsafe, or those with very advanced disease where benefit is unlikely, may not receive benefit from this program.
What this research studies
Pridopidine targets the Sigma-1 receptor, a protein linked to ALS, and this expanded access protocol provides eligible patients a way to receive it outside of a randomized trial. Participants will get the medication at participating sites and attend regular clinic visits for safety checks, blood tests, and symptom measurements. The program may also collect biomarker samples and use remote tools (like a smartphone app) to track function and daily changes. It builds on animal studies and earlier human testing that showed safety and hints of benefit, and gives more people the chance to try the medicine.
Why it matters
Potential benefit: If effective for some people, pridopidine could improve motor or bulbar symptoms or slow disease progression for a subset of patients with ALS.
How similar studies have gone: Pridopidine improved outcomes in ALS mouse models and a HEALEY platform trial in 162 participants showed safety and trends toward benefit but did not meet its primary endpoint.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Paganoni, Sabrina — Massachusetts General Hospital
- Study coordinator: Paganoni, Sabrina
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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.