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Motor neurone disease milestone: Patient trial shows impressive clinical results

A new drug delays motor neurone disease progression and improves cognitive and clinical symptoms according to recently announced trial results.

Research at a glance: 

  • The drug CuATSM slowed overall deterioration in MND patients over six months
  • Improvements were seen in lung function and cognition in some patients
  • Decline in motor disability was reduced in treated patients compared to standard-of-care patients
  • Researchers will begin a larger Phase 2 trial to confirm CuATSM’s effectiveness in motor neurone disease

A new drug developed by scientists at the Florey Institute of Neuroscience, and the School of Chemistry and Bio21 Institute at the University of Melbourne has dramatically improved clinical and cognitive symptoms of motor neurone disease, also called amyotrophic lateral sclerosis.

This is the first human evidence for a disease-modifying drug for motor neurone disease. It is a significant step forward, and we look forward to confirming the positive results in a larger study soon

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

Patients usually die of respiratory failure within three years of diagnosis, and there are no treatments or disease-modifying therapies available.

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 sponsored by Collaborative Medicinal Development LLC with support from FightMND.

The trial is currently being extended to examine the longer-term results. 

Professor Ashley Bush, Chief Scientific Officer of Collaborative Medicinal 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 significant step forward, and we look forward to confirming the positive results in a larger study soon.”

Associate Professor Kevin Barnham of the Florey, Associate Professor Anthony White at the Queensland Institute of Medical Research, and Professor Paul Donnelly and Associate Professor Peter Crouch from the University of Melbourne, developed and tested CuATSM over a 15-year period.

After showing its therapeutic potential for motor neurone disease in pre-clinical models, the researchers founded a company and licensed the compound to Collaborative Medicinal Developments to take the drug into human studies.

Professor Donnelly said, “It is gratifying to see such promising results made possible by collaborative fundamental research at the interface between chemistry and biology.”

The results were reported at the 29th International Symposium on ALS/MND in Glasgow by Dr Craig Rosenfeld, CEO of Collaborative Medicinal Development.

The researchers plan to begin enrollment for a larger, randomised, placebo-controlled double-blind Phase 2 trial in mid- to late 2019. This trial will test CuATSM’s effectiveness in motor neurone disease / amyotrophic lateral sclerosis in a larger patient sample.

More information on the phase 1 extension trial is available at the ClinicalTrials.gov site

About the Florey:

The Florey Institute of Neuroscience and Mental Health is one of the largest and highly respected brain research centres in the world. Its teams work on a range of serious diseases including stroke, epilepsy, Alzheimer’s, Parkinson’s and motor neurone diseases, depression and addiction. The Florey is a world leader in imaging technology, stroke rehabilitation and large population studies to improve patient care around the world.

About the Bio21 Institute:

The University of Melbourne’s Bio21 Molecular Science and Biotechnology Institute seeks to improve human health and disease and the environment through innovation in molecular life science and biotechnology, driven by collaborative research and dynamic interactions with industry. It is home to more than 700 research scientists, making it one of the largest biotechnology research centres in Australia. The Institute houses major analytical and imaging technology platforms providing world-leading capability to researchers in the Melbourne Biomedical Precinct.

About Collaborative Medicinal Development
CMD is a privately-held biopharmaceutical company developing innovative therapies for neurodegenerative diseases.  The Company's lead drug, CuATSM, was licensed from the University of Melbourne and entered clinical trials in ALS and Parkinson's disease in 2017 at leading clinical centers in Australia. The CMD team includes Craig Rosenfeld, MD (CEO), Kay Noel, PhD (COO), Ashley Bush, MD, PhD (CSO), and Jim Babcock (Chairman, founder of Cthulhu Ventures LLC, CMD's founding investor). 

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