What is the drug thought to do in the body?
CuATSM (Copper ATSM) is designed to deliver copper to damaged mitochondria, which is the powerhouse of the cell. Previous research has shown that mitochondria can be become damaged and not perform as well it should in people with MND. Other research suggests that a lack of copper ions may play a role in the SOD1 protein becoming faulty in people with changes in the SOD1 gene.
Phase 2/3
Current status: Waiting on results
Primary objective: To see if CuATSM changes the rate of disease progression, measured by the ALS Functional Rating Score (ALSFRS-R)
The Phase 2/3 began recruiting participants in September 2019 to explore whether the drug is beneficial to people with MND. The trial is investigating the efficacy and safety of CuATSM. The multicenter, randomized, double-blinded, placebo-controlled study planned to recruit up to 80 people with MND. You can find out more about the trial here.
The trial recruited in Australia only.
Previous Clinical Trials
Phase 1
The Phase 1 results were announced in December 2018, and showed that the treatment was safe and well-tolerated. There were some hints thet CuATSM may slow disease progression, but additional research is needed to gather more evidence to confirm this. You can more about the trial here.
Latest News
2023
In 2023 an article was published which investigated brain tissue from 6 people who took part in this trial, but sadly died. No changes to motor neurones or the damage known to be caused by the disease were found, suggesting that CuATSM was not beneficial for those who received the drug. However, the final results from the trial have still not been published.
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Last upadated: 21/08/2024