The first clinical trial of copper-ATSM as a potential treatment option for motor neurone disease (MND) is expected to start recruiting in November 2016. People with MND should contact their State MND Association or neurologist if they wish to discuss the trial further. For detailed information about the copper-ATSM study > click here.
What is copper-ATSM?
Copper-ATSM therapy was developed in Australia. Its chemical name is diacetylbis(4-methylthiosemicarbazonato)copperII but it is often abbreviated to Cu-ATSM, CuII(atsm) or copper-ATSM. Research has shown copper-ATSM can protect motor neurones in the spinal cord, improve MND-like symptoms, and extend the lifespan of mice with a mutated form of SOD1. It is more effective in mutant SOD1 mice than riluzole (the only approved treatment for MND).
To read about the history of copper-ATSM > more.
What is involved?
It is anticipated the Phase 1 study will be conducted at four sites in Australia. At present, only Macquarie University has ethics approval to proceed and the study should begin in November 2016. Patients with known genetic abnormalities will be recruited into the trial at this site.
Ten participants that meet certain inclusion criteria will be recruited at each site. Copper-ATSM will be administered orally to patients with amyotrophic lateral sclerosis. The study will be conducted in three phases to determine the recommended dose of copper-ATSM and assess preliminary evidence of efficacy. To see details of what is involved > more.
Is this related to sporadic or familial MND?
The study will recruit participants who have been diagnosed with sporadic or familial amyotrophic lateral sclerosis. See inclusion and exclusion criteria > more.
Research to date has been conducted on familial MND animal models. There is no animal model for sporadic MND. However, in 2015 Peter Crouch and colleagues at The University of Melbourne and the Florey Institute found MND-affected tissues obtained from people who died because of sporadic MND had important similarities to mice that responded to copper-ATSM. This suggests that copper-ATSM may have activity in both sporadic and familial MND.
How far off is a treatment?
A possible treatment resulting from this study is many years away. This research will determine if copper-ATSM is safe to treat people with MND. Analysis of data from the trial will give researchers a clearer picture on whether copper-ATSM should be tested in larger studies to further investigate its potential.
The copper-ATSM clincial trial is sponsored by Collaborative Medicinal Development Pty Ltd (CMD).
The Motor Neurone Disease Research Institute of Australia has invested more than $1.2 million to support three projects that aim to assist developing copper-ATSM as a potential therapeutic. These projects are:
Betty Laidlaw MND Research Grant
Copper malfunction in motor neurone disease: a therapeutic target for sporadic MND
zo-ee MND Research Grant
Proteomic investigation of functional copper deficiency in MND
Jenny Barr Smith MND Collaboration Grant
Drug-specific biomarkers to faclitate clinical translation of CuII(ATSM) as a potential therapeutic for MND.
For detailed information about the research projects > more.