A team of researchers led by Australia’s Macquarie University professor Dominic Rowe and Calvary Health Care Bethlehem associate professor Susan Mathers has reported positive results from a trial that evaluated CuATSM for the treatment of motor neurone disease (MND) or amyotrophic lateral sclerosis.
MND is a progressive and fatal neurodegenerative disease that leads to muscle weakness and eventually paralysis.
Results demonstrated that CuATSM improved lung function and cognitive ability in patients compared to those treated with existing standard-of-care and was found to have slowed motor disability in patients.
CuATSM has reportedly also improved clinical symptoms of the disease and delayed the overall deterioration of MND patients within six months.
Collaborative Medicinal Development, with support from FightMND, has sponsored the trial.
The study featured a dose-finding design and enrolled 32 patients.
Collaborative Medicinal Development chief scientific officer Ashley Bush said: “This is the first human evidence for a disease-modifying drug for motor neurone disease.
“It is a huge breakthrough, and we look forward to confirming the positive results in a larger study soon.”
The researchers involved in the motor neurone disease trial intend to start enrolment for a larger, randomised, placebo-controlled double-blind Phase II trial of CuATSM this year.
This trial will be designed to investigate the effectiveness of CuATSM in motor neurone disease in a larger patient group.
CuATSM is created by scientists at Australia’s Florey Institute of Neuroscience, and University of Melbourne School of Chemistry and Bio21 Institute.
Collaborative Medicinal Development secured the licence of the drug for use in human clinical studies.