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Spotlight on research: Copper-ATSM as a potential MND treatment

The idea to investigate copper-containing compounds as a possible treatment for MND began as a collaboration between Drs Kevin Barnham, Tony White and Paul Donnelly, all based at The University of Melbourne over 10 years ago. They were working on Alzheimer’s disease and Parkinson’s disease at the time. As luck would have it, they had an opportunity to test copper-ATSM in an MND mouse model under the auspices of Dr Qiao-Xin Li, also at The University of Melbourne. The researchers were thrilled to find the project was an immediate success; treating the MND model mice diminished severity of disease symptoms and extended survival. These data led to the first project to focus on investigating copper-ATSM as a potential treatment for MND. The project commenced in 2008 and was led by Drs Li, Barnham, Donnelly, White and Crouch.

Science in brief
Since the initial project, Dr Crouch and his team’s focus has been to address some fundamental research questions. What is the full therapeutic potential of copper-ATSM? How does it work? Will it work in people with MND? To answer these questions they have adopted multiple, complementary lines of investigation, including assessments performed on mice treated with the drug and biochemical analyses performed using brain and spinal cord tissue from people who had MND. The expanded knowledge gained from this research provides an ever-increasing opportunity to ensure that the successful outcomes generated from MND model mice will translate to an effective drug for people with MND.

Impact
This research over the last decade has led to the Phase I copper-ATSM clinical trial sponsored by Collaborative Medicinal Development Pty Ltd, which began in Sydney in late 2016, and more recently, in Melbourne in 2017. Dr Crouch and his team have achieved what is often referred to as “bench-to-clinic” research. This means that an idea, initially developed then tested in the research laboratory, has withstood the rigours of scientific interrogation to the point where it has become feasible to begin testing in people.  

Next steps
Dr Crouch and his team continue to undertake research that feeds new information into the clinical setting. Only 10 per cent, of drugs that begin preclinical testing ever make it to human clinical trials. Getting to this point needs many factors to align including financial support. A possible treatment resulting from this research is still many years away. Analysis of data from the clinical trial will give researchers a clearer picture on whether copper-ATSM should be tested in larger studies to further investigate its potential. 

Article sourced from MND Research Institure of Australia report '$25Million, 25 Milestones: Changing the future of MND'. 

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