Using intravenous trehalose to treat patients with ALS
An Expanded Access Protocol of Intravenous Trehalose Injection 90 mg/mL Treatment of Patients with Amyotrophic Lateral Sclerosis
This study is looking at how giving trehalose through an IV can help people with ALS, offering a new treatment option for those who can’t join another trial, and you can get the infusions either at the research center or at home with a nurse's help.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10649756 on NIH RePORTER |
What this research studies
This research investigates the use of intravenous trehalose, a disaccharide that activates autophagy, as a treatment for amyotrophic lateral sclerosis (ALS). Patients will receive weekly infusions of trehalose, which can be administered at the research center or at home by a trained nurse. The study aims to provide expanded access to this treatment for patients who are not eligible for the ongoing HEALEY ALS Platform Trial. By targeting multiple biological pathways involved in ALS, this approach seeks to enhance treatment efficacy beyond existing medications.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with amyotrophic lateral sclerosis who are not eligible for the current randomized controlled trial.
Not a fit: Patients with other neurological conditions or those who are not diagnosed with ALS may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that may slow the progression of ALS and improve patient outcomes.
How similar studies have performed: Previous studies have shown promising results with trehalose in animal models, but this expanded access protocol represents a novel opportunity for human treatment.
Where this research is happening
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Paganoni, Sabrina — Massachusetts General Hospital
- Study coordinator: Paganoni, Sabrina
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.