Using engineered immune cells to treat ALS
Development of CM-CS1 CAR Treg to Treat Amyotrophic Lateral Sclerosis (ALS)
['FUNDING_SBIR_2'] · CELDARA MEDICAL, LLC · NIH-11128419
This study is exploring a new way to help people with ALS by using specially designed immune cells to target harmful proteins in the body, with the hope of reducing inflammation and slowing down the disease.
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-11128419 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new treatment for Amyotrophic Lateral Sclerosis (ALS) by utilizing engineered immune cells known as CAR Tregs. These cells are designed to target and respond to misfolded proteins associated with ALS, specifically a protein called SOD1. By enhancing the function of these Tregs, the research aims to reduce inflammation and slow disease progression in ALS patients. The approach involves creating a mouse model to test the effectiveness of these engineered cells before moving to human trials.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS, particularly those with mutations in the SOD1 gene.
Not a fit: Patients with ALS who do not have SOD1 mutations or those with advanced stages of the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a novel therapy that slows the progression of ALS and improves the quality of life for patients.
How similar studies have performed: Other research has shown promise in using engineered immune cells for various conditions, suggesting potential success for this novel approach in ALS.
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