RAPA-501 immune-cell therapy for ALS (expanded access for advanced disease)
Official title: Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
Funded at Massachusetts General Hospital · NIH-11184331 · Data updated 2026-11-12
This program offers an experimental immune-cell treatment called RAPA-501 to people with ALS who have advanced breathing weakness (SVC <50%).
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, 2026 |
| Funding year | 2025 |
| Award (this year) | $6,151,947 |
| NIH project number | 5U01NS136020-03 |
| Project ID | NIH-11184331 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with ALS who have SVC under 50% of predicted, who are considered high-risk for respiratory failure and are not eligible for the phase 2/3 trial are the intended candidates.
✕Less likely to help
Patients with milder ALS and SVC ≥50%, or those who cannot undergo blood collection or cell infusion for medical reasons, are unlikely to be eligible or to receive benefit from this expanded-access program.
What this research studies
You would receive an autologous (your own) T cell therapy called RAPA-501 that is manufactured outside the body to increase anti-inflammatory TREG/Th2 activity and a T-stem phenotype. The cells are made from your blood and infused back without requiring chemotherapy conditioning. This expanded-access program is specifically for people with ALS who have reduced slow vital capacity (SVC <50%) and who are not eligible for the ongoing phase 2/3 trial. Earlier participants showed the treatment was safe, produced anti-inflammatory biological effects, and had early signs of slowing pulmonary decline.
Why it matters
Potential benefit: If successful, RAPA-501 could reduce nervous-system inflammation and slow respiratory decline, potentially lowering short-term risk of respiratory failure in high-risk ALS patients.
How similar studies have gone: A prior clinical trial of RAPA-501 (NCT04220190) reported no product-related safety issues, measurable anti-inflammatory effects, and early trends toward stabilizing pulmonary function, though larger controlled trials are still needed.
Where this research is happening
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Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Berry, James Dale — Massachusetts General Hospital
- Study coordinator: Berry, James Dale
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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.