MND Australia

Copper - ATSM

Phase 2/3

Phase 2/3 of the Copper-ATSM clinical trial will soon commence at Macquarie University, Sydney and then at other sites across Australia.  

The first clinical trial of copper-ATSM as a potential treatment option for motor neurone disease (MND) started recruiting for Phase 1 in November 2016. An announcement regarding the outcomes of this trial was made in December, 2018.

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).

What is involved?

Phase 1 of the study was conducted at two sites in Australia. This phase identified a safe dosage of copper-ATSM.

The phase 2/3 trial will commence initially at Macquarie University in Sydney and then at other sites across Australia. Anyone interested in taking part in the trial will need to contact their neurologist to discuss suitability and eligibility.

Is this related to sporadic or familial MND?

Phase 1 of the study recruited participants who have been diagnosed with sporadic or familial amyotrophic lateral sclerosis. See inclusion and exclusion criteria for Phase 2/3 of the trial here.

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 these studies is many years away. Phase 1 determined a safe dose of copper-ATSM. Data from this trial has guided researchers in setting up the next phase of the trial. The Phase 2 Cu-ATSM clinical trial will involve 80 participants and will serve to evaluate efficacy (whether the compound works as intended) and to further evaluate safety. Patients involved in this phase of the trial will be randomly assigned Cu-ATSM or a placebo for 6x28 day cycles.

Motor Neurone Disease Research 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 facilitate clinical translation of CuII(ATSM) as a potential therapeutic for MND,