Motor neurone disease (MND) breakthrough: Patient trial shows impressive clinical results

A new copper-delivery drug, CuATSM, developed by scientists at the Florey Institute of Neuroscience, the School of Chemistry and Bio21 Institute at the University of Melbourne has dramatically improved clinical and cognitive systems of motor neurone disease (MND), also called amyotrophic lateral sclerosis.

Motor neurone disease is a progressive, fatal neurodegenerative disease. Its key hallmark is the death of brain cells that control muscle movements. This results in muscle weakness and eventually paralysis.

In this dose-finding trial involving 32 patients, the group given the highest amount of the CuATSM compound showed improved lung function and cognitive ability, compared to the predicted declines observed in standard-of-care patients.

Further, treated patients showed a much slower overall disease progression as measured by a global disability score.

The clinical trial was led by Professor Dominic Rowe at Macquarie University and Associate Professor Susan Mathers at Calvary Health Care Bethlehem, and was sponsored by Collaborative Medicinal Development Pty Ltd with support from FightMND.

Professor Ashley Bush, Chief Scientific Officer of Collaborative Medicine Development and director of the Melbourne Dementia Research Centre, 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."

Researchers are set to begin a larger Phase 2 trial to confirm CuATSM's effectiveness in motor neurone disease.

Associate Professor Peter Crouch (Dept. Pharmacology & Therapeutics) was involved in the development and trial of the CuATSM drug over a 15-year period.

Associate Professor Crouch has been described as "the driving force behind much of the University of Melbourne's recent work in MND." (Pursuit 2017)


Read the full article on The Melbourne Newsroom here.

Read more about this research in our 2017 Pursuit article, 'Breakthrough in motor neurone disease research gives hope'.