Background
An Australian company called Collaborative Medicinal Development; LLC are clinically testing a compound called CuATSM. In 2018, results were released from a phase 1 clinical trial that was designed only to determine the safety and dosing of CuATSM. Later trials (phase 2 or 3) would be designed to further examine safety and to eventually determine if it has the ability to alter ALS/MND disease progression.
On January 7th, 2019, television spots and a media release stated that CuATSM slowed disease progression in the phase 1 clinical trial participants. This resulted in an international outcry for people living with ALS/MND to access CuATSM. Further studies 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 although the outcome of the trial has yet to be announced. It is very disappointing that Collaborative Medicinal Development, LLC have not released the results.
Summary
The Scientific Advisory Council (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. At the doses tested, using clinical grade CuATSM, it was considered safe, but a press release also states that the company has seen a substantial slowing of disease progression. This 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.
Please refer to this MND Association blog for a more detailed explanation.
International Alliance of ALS/MND Associations
May 2023
The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.