Engineered regulatory immune cells to protect nerves in ALS
Official title: Development of CM-CS1 CAR Treg to Treat Amyotrophic Lateral Sclerosis (ALS)
Funded at Celdara Medical, LLC · NIH-11181028 · Data updated 2026-11-12
Engineered regulatory immune cells (CAR Tregs) that seek out misfolded SOD1 and calm inflammation around motor neurons in people with ALS.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Celdara Medical, LLC NIH-funded |
| Lab location | 1 site (Lebanon, United States) |
| Project start | September 19, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $921,394 |
| NIH project number | 5R44NS132666-03 |
| Project ID | NIH-11181028 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with ALS, especially those whose disease involves misfolded or aggregated SOD1, would be the most relevant candidates for this approach.
✕Less likely to help
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.
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.
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 gone: 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
-
Celdara Medical, LLC
Lebanon, United States Active
Researchers
- Principal investigator: Murad, Joana M — Celdara Medical, LLC
- Study coordinator: Murad, Joana M
Related research
Other NIH-funded projects on the same topics.
-
Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.
This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.
-
CNM-Au8 Expanded Access for ALS patients across the U.S.
A wide-access program is testing CNM-Au8, a neuroprotective medicine, for people with ALS across all 50 states.
-
Access to CNM-Au8 for people with ALS
This program offers people with ALS access to CNM-Au8, an experimental oral therapy that aims to protect motor neurons and support cellular energy.
-
Biomarker test for early ALS diagnosis using Nano-QuIC detection of misfolded TDP-43
A new blood, spinal fluid, or nasal swab test using Nano-QuIC aims to detect misfolded TDP-43 early in people at risk for ALS to speed up diagnosis and treatment.
-
Using engineered immune cells to treat ALS
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.
-
Understanding ALS Risk in Workers with Certain Occupations
This project is creating a group of people at higher risk for ALS to learn how their jobs and genes might lead to the disease.
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.