Engineered regulatory immune cells to protect nerves in ALS
Development of CM-CS1 CAR Treg to Treat Amyotrophic Lateral Sclerosis (ALS)
['FUNDING_SBIR_2'] · CELDARA MEDICAL, LLC · NIH-11181028
Engineered regulatory immune cells (CAR Tregs) that seek out misfolded SOD1 and calm inflammation around motor neurons in people with ALS.
Quick facts
| Phase | ['FUNDING_SBIR_2'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CELDARA MEDICAL, LLC (nih funded) |
| Locations | 1 site (LEBANON, UNITED STATES) |
| Trial ID | NIH-11181028 on ClinicalTrials.gov |
What this research studies
This project creates CAR Treg cells that specifically recognize aggregated misfolded SOD1, a protein found in many ALS patients, and activates those cells where the abnormal protein appears. The engineered Tregs are also programmed to release BDNF, a factor that supports nerve cell survival. The team is testing these cells in a newly developed mouse model that carries the ALS-linked SOD1 mutation on an immune-deficient background to permit human-like cell engraftment. Work focuses on preclinical development to show safety and effectiveness before any human testing.
Who could benefit from this research
Good fit: People diagnosed with ALS, especially those whose disease involves misfolded or aggregated SOD1, would be the most relevant candidates for this approach.
Not a fit: Patients whose ALS does not involve SOD1 aggregation or those with very advanced, irreversible motor neuron loss are less likely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this approach could reduce harmful inflammation around motor neurons and help slow ALS progression.
How similar studies have performed: Regulatory T cell therapies have shown promise in other inflammatory and autoimmune conditions, but CAR Tregs targeting aggregated proteins in ALS is a novel and largely preclinical approach.
Where this research is happening
LEBANON, UNITED STATES
- CELDARA MEDICAL, LLC — LEBANON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MURAD, JOANA M — CELDARA MEDICAL, LLC
- Study coordinator: MURAD, JOANA M
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: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease, Amyotrophic Lateral Sclerosis Motor Neuron Disease