NUZ-001: a treatment option for ALS to protect nerve cells
Official title: An Intermediate-Size Expanded Access Protocol (EAP) for NUZ-001 in People with Amyotrophic Lateral Sclerosis (NUZ-001 EAP)
Funded at Massachusetts General Hospital · NIH-11564156 · Data updated 2026-11-12
A small molecule called NUZ-001 is being tested to help people with ALS by improving cell systems that keep proteins balanced and prevent harmful protein clumps.
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 1, 2026 |
| Project end | July 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $8,276,544 |
| NIH project number | 1U01NS153995-01 |
| Project ID | NIH-11564156 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with ALS who meet protocol criteria and are eligible for the expanded access program.
✕Less likely to help
Some patients may not experience benefit or may not tolerate the drug due to side effects.
What this research studies
This project tests NUZ-001 in people with ALS to see if it can support neurons by boosting protein housekeeping pathways. Researchers will study how the drug affects proteasome function, autophagy, and ER stress, and whether it slows decline or stabilizes function. The early work builds on a Phase 1 study and uses a mid-size expanded access protocol to collect safety and preliminary effectiveness data in real patients. The study aims to translate lab findings about proteostasis into a potential clinical benefit for ALS patients over several years.
Why it matters
Potential benefit: If successful, NUZ-001 could help protect motor neurons and potentially slow disease progression in ALS.
How similar studies have gone: Preliminary Phase 1 data suggested safety and signals of biologic activity, with historical data providing context but no definitive efficacy.
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.