In this episode, Dr. Fernando Vieira, CSO at the ALS Therapy Development Institute (ALS TDI), joins us to discuss Copper ATSM (CuATSM) clinical trials. CuATSM is a small molecule drug sponsored by the company Collaborative Medicinal Development (CMD). They recently completed enrollment of a small, Phase 1 clinical trial in Australia.
CuATSM is a small molecule drug sponsored by the company
Collaborative Medicinal Development (CMD) that completed enrollment of a Phase
1 trial in Australia in June 2019. Originally used as an imaging agent in Asia,
CuATSM has shown potential to treat copper zinc superoxide dismutase (SOD1)
In healthy SOD1 proteins, zinc maintains the protein’s shape
and copper is essential to its antioxidant function. SOD1 mutations, though,
can cause proteins to become unmetallated. This means that they release their
necessary zinc and copper components and can no longer sustain cellular
Scientists hypothesize that CuATSM could act as a ‘copper
chaperone’, delivering copper ions back into SOD1 proteins and enabling proper
cellular function. CuATSM could also prevent protein misfolding. It also might
prevent the buildup of peroxynitrite, a molecule that can disrupt the electron
transport chain in mitochondria.
The University of Melbourne and Oregon State University have
completed studies that demonstrate significant improvements in lifespan in SOD1
mice treated with CuATSM. Additional tests at ALS TDI in 2016 confirmed the
drug’s efficacy in SOD1 mouse models. Positive results at multiple labs have
increased confidence in CuATSM as it goes forward into clinical trial.
The Australian Phase 1 trial has so far shown promise in
people with both sporadic ALS (sALS) and familial ALS (fALS). However, the
trial size in this study was small, involving just six people with ALS.
development in Australia will be dependent on demonstrated success at this
early stage. There are currently no CuATSM trials planned in the U.S, but ALS
researchers expect results from the Phase 1 trial in Australia in April 2020.