A NEW drug has been shown to delay the progression of motor neurone disease and improve cognitive and clinical symptoms.
Developed by scientists at the Florey Institute of Neuroscience, and the School of Chemistry and Bio21 Institute at the University of Melbourne, the drug was found to have dramatically improved clinical and cognitive symptoms of motor neurone disease, also called amyotrophic lateral sclerosis.
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 a 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.
The treated patients also showed a much slower overall disease progression as measured by a global disability score.
Huge breakthrough
Professor Ashley Bush, chief scientific officer of Collaborative Medicinal Development and director of the Melbourne Dementia Research Centre, said this was 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," Professor Bush said.
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, Collaborative Medicinal Development, to take the drug into human studies.
The researchers plan to begin enrolment for a larger, randomised, placebo-controlled double-blind Phase 2 trial in mid to late 2019.