What does the drug do?
CuATSM (Copper ATSM) is a candidate MND drug that is being investigated by the Collaborative Medicinal Development company for its effects of slowing down progression and improving respiratory and cognitive function. The drug is thought to work by delivering copper to cells damaged mitochondria, allowing for selective delivery of the drug (i.e. to only those cells that have been damaged). Other research suggests a lack of copper ions may be behind the misfolding of the SOD1 proteins caused by certain cases of familial ALS, which in turn leads to cell death.
The Phase 1 results were first announced in December 2018, showing that the treatment was safe and well-tolerated, with some beneficial effect observed. You can more about the trial here.
The Phase 2/3 has begun recruiting participants in September 2019 to explore whether the drug is beneficial to people with MND, in a larger cohort of people. The trial is aimed at evaluating the efficacy and safety of CuATSM. The multicenter, randomized, double-blinded, placebo-controlled study is planning to recruit up to 80 ALS patients at four clinical sites in Australia. You can find out more about the trial here.
The trial was recruiting in Australia only.
Results are expected in 2022.