Copper ATSM (CuATSM) is a small molecule therapeutic compound that has demonstrated intriguing results for ALS, both in the lab and in early human trials. A phase 2/3 study, conducted by Collaborative Medicine Development (CMD) in Australia, began in September of 2019 and will run through the end of this year.

While CuATSM has been used in humans for years as an imaging agent for diagnosing things like tumors, it’s potential to deliver copper molecules to damaged cells attracted the attention of scientists who believed it could be a promising drug candidate for treating ALS. Some 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 drug works as a sort of “chaperone” for copper molecules, helping them get safely into the body without causing the toxicity that would arise from taking a dose of raw copper.

Findings by researchers at the University of Oregon and University of Melbourne demonstrated that the drug appeared to halt disease progression in mouse models of SOD1 ALS. These results were further tested – and validated – by scientists at ALS TDI in 2016. Additionally, the drug may have wider applications for those with sporadic ALS. Some research has shown it may also help to eliminate peroxynitrite, a chemical that interferes with the function of mitochondria in ALS and other neurodegenerative diseases.

The validation by ALS TDI scientists supported the advancement of CuATSM into a phase 1 trial in 2016. Results released in December 2018 appeared to show slowed progression in some patients. The phase 2/3 trial, which is still recruiting participants in Australia, will enroll 80 people at four sites and focus on testing the drug’s safety and efficacy, according to a press release from CMD. Participants will receive a daily oral dose of CuATSM, or a placebo, for six months. The expected date of completion is December 2020.

CuATSM was the first ALS treatment to have been discovered outside of the organization and have its efficacy confirmed by researchers at ALS TDI. Validating drugs, whether discovered internally or externally, is one of the key pillars of our research here at ALS TDI, and we’re eagerly awaiting the results of this study.

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