Personalized anti-inflammatory T cell therapy for ALS
Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
['FUNDING_U01'] · MASSACHUSETTS GENERAL HOSPITAL · NIH-11376007
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
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MASSACHUSETTS GENERAL HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11376007 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
BOSTON, UNITED STATES
- MASSACHUSETTS GENERAL HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BERRY, JAMES DALE — MASSACHUSETTS GENERAL HOSPITAL
- Study coordinator: BERRY, JAMES DALE
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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease