Access to CNM-Au8 for people with ALS
Official title: Intermediate-sized Expanded Access Protocol for CNM-Au8 in Amyotrophic Lateral Sclerosis (ALS).
Funded at New York University School of Medicine · NIH-11405390 · Data updated 2026-11-12
This program offers people with ALS access to CNM-Au8, an experimental oral therapy that aims to protect motor neurons and support cellular energy.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | New York University School of Medicine NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 25, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $11,639,047 |
| NIH project number | 7U01NS136023-03 |
| Project ID | NIH-11405390 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with ALS who meet the protocol's eligibility criteria and seek access to CNM-Au8, including those not enrolled in ongoing clinical trials, are the intended participants.
✕Less likely to help
People with medical contraindications to CNM-Au8, advanced organ failure, or who expect an immediate cure may not experience benefit from this treatment.
What this research studies
If you have ALS, this expanded-access program is designed to provide CNM-Au8 across the United States, including remote and rural areas. The partners (Columbia University, Clene, and Synapticure) will run an intermediate-sized protocol to ship and manage treatment outside a traditional randomized trial. CNM-Au8 is an oral suspension of gold nanocrystals that has shown in lab models the potential to raise energy metabolites like NAD and ATP and protect neurons. Earlier Phase 2 trials and open-label extensions have explored safety and signals of benefit, and this program aims to broaden patient access while following protocol eligibility and safety monitoring.
Why it matters
Potential benefit: If helpful, CNM-Au8 could preserve motor neuron function and slow functional decline in people with ALS.
How similar studies have gone: Preclinical studies showed neuroprotective effects and two Phase 2 randomized trials with open-label extensions have evaluated CNM-Au8's safety and potential signals, but definitive clinical benefit has not been proven.
Where this research is happening
-
New York University School of Medicine
New York, United States Active
Researchers
- Principal investigator: Andrews, Jinsy — New York University School of Medicine
- 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.
-
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.
-
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.