RAPA-501 immune-cell therapy for ALS (expanded access for advanced disease)
Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
['FUNDING_U01'] · MASSACHUSETTS GENERAL HOSPITAL · NIH-11184331
This program offers an experimental immune-cell treatment called RAPA-501 to people with ALS who have advanced breathing weakness (SVC <50%).
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-11184331 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
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