Personalized anti-inflammatory T cell therapy for ALS
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-11376007 · Data updated 2026-11-12
This program offers a treatment using your own modified immune cells (RAPA-501) to reduce nerve inflammation for people with ALS who have low lung capacity.
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) | $3,993,521 |
| NIH project number | 3U01NS136020-03S1 |
| Project ID | NIH-11376007 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with ALS who have SVC (slow vital capacity) below 50% and who are not eligible for the ongoing phase 2/3 trial are the primary candidates for this expanded access program.
✕Less likely to help
People without ALS or people with ALS who have relatively preserved lung function (SVC ≥50%) or who are already enrolled in the phase 2/3 trial may not be eligible or benefit from this program.
What this research studies
You would have some of your own T cells collected and modified in the lab to boost regulatory and anti-inflammatory activity before they are returned to you. The product, called RAPA-501, is designed to act without the need for chemotherapy conditioning. This expanded access effort is for people with ALS who have a slow vital capacity (SVC) below 50% and who are not eligible for the ongoing phase 2/3 trial. The team has seen safety and early biological activity in ongoing work and aims to offer this option to higher-risk patients facing rapid respiratory decline.
Why it matters
Potential benefit: If successful, the therapy could slow respiratory decline and lower the risk of respiratory failure or death in high-risk people with ALS.
How similar studies have gone: An ongoing clinical trial of RAPA-501 (NCT04220190) has reported safety and biological anti-inflammatory activity with early trends toward stabilizing lung function, but definitive efficacy is not yet proven.
Where this research is happening
-
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.