Background
In 2018, an Australian company called Collaborative Medicinal Development; LLC released results from a Phase 1 clinical trial for a compound called CuATSM. Later trials (Phase 2 or 3) would be designed to further examine safety and to eventually determine if CuATSM had the ability to alter ALS/MND disease progression.
On January 7th, 2019, television spots and a media stated that CuATSM slowed disease progression in the Phase 1 clinical trial participants (Newswire press release). However, this Phase 1 trial was designed to only determine safety and dosing of CuATSM. These statements resulted in an international outcry for people living with ALS/MND to access CuATSM. Further studies, however, were required to provide a clear indication of whether these preliminary results were reliable.
Trial Design & Results
In late 2019, a Phase 2/3 clinical trial of 80 people started recruiting in Australia (NCT04082832). It was setup to provide stronger evidence for a treatment effect than the Phase 1 trial. The size of this Phase 2/3 trial meant however, that a very large slowing of disease progression would be required for a statistically significant effect to be seen.
The Phase 2/3 trial was completed in late 2021 however, the outcome of the trial has yet to be announced. It is very disappointing that Collaborative Medicinal Development, LLC decided not to release the results to this date.
In 2023, a peer reviewed article showed no alleviation of TDP-43 pathology or astrogliosis in 6 post-mortem brain samples from individuals affected by ALS and treated with CuATSM in clinical trials. This is in contrast with what had been observed in preclinical studies (Yang et al., 2023).
Summary
The SAC recommends that Alliance members refer to CuATSM as a drug that was recently studied for human safety in a small Phase 1 clinical trial by the company Collaborative Medicinal Development, LLC in Australia. It is the opinion of the SAC that, at the doses tested, clinical grade CuATSM is considered safe. However, a press release also states that the company has seen a substantial slowing of disease progression. This Phase 1 clinical trial was not designed to make such a public statement and a number of aspects in its design require this result to be taken with as much caution as possible. Essentially, there is no substantiated scientific evidence that CuATSM has any advantageous effect on ALS/MND in humans.
To date, the follow-on trial results have not been published despite the trial completing in 2021. The SAC urges Collaborative Medicinal Development, LLC to report the outcomes of the trial as soon as possible to the community that has supported this trial. However, given that Collaborative Medicinal Development has been unresponsive to this request, the SAC has decided to move this note to the “drugs no longer in development” section of the Alliance website.
Please refer to the MND Association blog for a more detailed explanation.
International Alliance of ALS/MND Associations
April 2026
Disclaimer: The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.