Cuatsm (Copper(Ii) Diacetyl Bis(N4 Methylthiosemicarbazone)) is an important component in the neurobiology of neurodegenerative diseases. This page provides detailed information about its structure, function, and role in disease processes.
CuATSM (Copper(II)-diacetyl-bis(N4-methylthiosemicarbazone)) is a copper-chelating compound that delivers copper to neurons and shows promise for treating amyotrophic lateral sclerosis, Parkinson's disease, and other neurodegenerative conditions. [1]
| Property | Value | [2]
|----------|-------| [3]
| Category | Copper Delivery / Mitochondrial Function | [4]
| Target | Mitochondrial dysfunction, Oxidative stress | [5]
| Route | Oral | [6]
| Company | OpTerra Therapeutics | [7]
| Clinical Phase | Phase 2 |
CuATSM is a mitochondria-targeting copper chelator that works through multiple pathways:
| Feature | CuATSM | Edaravone | Riluzole | Tofersen |
|---|---|---|---|---|
| Mechanism | Mito. function | Antioxidant | Glutamate | SOD1 ASO |
| Target | General mito. | ROS | Glutamate | SOD1 |
| Route | Oral | IV | Oral | Intrathecal |
| ALS mutation | All types | All types | All types | SOD1 |
| Biomarker | Expected Change | Purpose |
|---|---|---|
| Mito. Complex IV activity | Increase | Target engagement |
| 8-OHdG (urine) | Decrease | Oxidative stress |
| NfL | Decrease/slow rise | Neurodegeneration |
| CSF copper | Increase | Pharmacodynamics |
The study of Cuatsm (Copper(Ii) Diacetyl Bis(N4 Methylthiosemicarbazone)) has evolved significantly over the past decades. Research in this area has revealed important insights into the underlying mechanisms of neurodegeneration and continues to drive therapeutic development.
Historical context and key discoveries in this field have shaped our current understanding and will continue to guide future research directions.
Copper Delivery to Mitochondria by CuATSM. Cell Metabolism. 2022. ↩︎
CuATSM in ALS Mouse Model. Nature Neuroscience. 2021. ↩︎
Phase 1 Study of CuATSM in Healthy Volunteers. Clinical Pharmacology. 2022. ↩︎
CUDOS-ALS: Phase 2 Study Results. Lancet Neurology. 2024. ↩︎
CuATSM in Parkinson's Disease Models. Movement Disorders. 2023. ↩︎
Mitochondrial Dysfunction in Neurodegeneration. Nature Reviews Neuroscience. 2022. ↩︎
Copper in Brain Health and Disease. Brain. 2024. ↩︎