Access to a new treatment for ALS using a neuroprotective drug.
Official title: Intermediate-sized Expanded Access Protocol for CNM-Au8 in Amyotrophic Lateral Sclerosis (ALS).
Funded at Columbia University Health Sciences · NIH-10934384 · Data updated 2026-11-12
This study is looking for people with ALS to try a new drug called CNM-Au8® that might help protect their nerve cells and improve their energy, with the hope of slowing down the progression of the disease.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 25, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $11,593,647 |
| NIH project number | 5U01NS136023-02 |
| Project ID | NIH-10934384 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with amyotrophic lateral sclerosis (ALS) who are seeking new treatment options.
✕Less likely to help
Patients with other neurological disorders or those not diagnosed with ALS may not benefit from this research.
What this research studies
This research aims to provide individuals living with amyotrophic lateral sclerosis (ALS) across the United States, including those in remote areas, access to CNM-Au8®, a neuroprotective drug. The study focuses on addressing the energetic impairment of motor neurons, which is a key factor in ALS progression. By administering CNM-Au8, the research seeks to enhance energy levels in neurons, potentially slowing down the disease's progression. Participants will be monitored for safety and efficacy as part of this innovative treatment approach.
Why it matters
Potential benefit: If successful, this research could offer a new therapeutic option that may slow the progression of ALS and improve quality of life for patients.
How similar studies have gone: Previous studies have shown promising results with similar neuroprotective approaches in ALS, indicating potential for success with this treatment.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Andrews, Jinsy — Columbia University Health Sciences
- Study coordinator: Andrews, Jinsy
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.
-
Engineered regulatory immune cells to protect nerves in ALS
Engineered regulatory immune cells (CAR Tregs) that seek out misfolded SOD1 and calm inflammation around motor neurons in people with ALS.
-
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.
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.