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.
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 Pty Ltd with support from FightMND.
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 huge breakthrough, 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 licenced the compound to Collaborative Medicinal Development, 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 Latest on: Motor neurone disease
via Google News
The Latest on: Motor neurone disease
- Leeds Rhinos legends Rob Burrow and Kevin Sinfield inspire cancer survivor's 290-mile ride for Motor Neurone Disease Associationon January 23, 2021 at 4:02 am
When cancer survivor Stephen Brown saddles up for a 290-mile bike ride later this year in aid of the Motor Neurone Disease Association, he will not just be drawing inspiration from Leeds Rhinos ...
- Damage caused by motor neurone disease could be REVERSED using existing drugs that improve energy levels in nerve cells, study revealson January 20, 2021 at 2:55 am
Researchers from Edinburgh University have pinpointed an issue in patients' nerve cells that depletes energy levels in the mitochondria - the power supply to the motor neurones.
- Motor neurone disease: Edinburgh scientists reveal breakthroughon January 19, 2021 at 4:23 am
Scientists are a step closer to being able to reverse the damage caused by motor neurone disease (MND). University of Edinburgh experts have found a problem with MND patients' nerve cells which could ...
- A breakthrough discovery could reverse Motor Neurone Disease damageon January 19, 2021 at 3:31 am
Researchers in Scotland have made a breakthrough discovery which could reverse the damage caused by motor neurone disease (MND).
- AFL 2021: Neale Daniher, new walkway, FightMND, motor neurone disease, Essendon, MCG bridge, Daniher’s Wayon January 18, 2021 at 4:39 am
Daniher, part of one of Australian football’s most iconic families, has been raising awareness of Motor Neurone Disease since being diagnosed with the illness seven years ago. Victorian premier Daniel ...
- Breakthrough discovery reveals way of reversing Motor Neurone Disease damageon January 17, 2021 at 9:24 pm
SCIENTISTS are a step closer to being able to reverse the damage caused by motor neurone disease following a breakthrough discovery by ...
- Silsden walkers boost battle against motor neurone diseaseon January 17, 2021 at 3:36 am
A WALK staged to support the battle against motor neurone disease and to remember a rugby club founder has been hailed as a huge success.
- New Imaging technique effectively measures mitochondrial dysfunction in motor neuron diseaseon January 16, 2021 at 11:30 pm
Researchers from the Sheffield Institute for Translational Neuroscience (SITraN) have used a new imaging technique to measure the function of mitochondria in patients with motor neuron ...
- Imaging technique proves effective in measuring mitochondrial dysfunction in motor neuron disease (MND)on January 15, 2021 at 11:36 am
Non-invasive imaging technique called 31-phosphorus magnetic resonance spectroscopy used to measure mitochondrial function in patients with motor neuron disease (MND) ...
- Imaging technique proves effective in measuring mitochondrial dysfunction in motor neuron diseaseon January 12, 2021 at 4:00 pm
Researchers from the Sheffield Institute for Translational Neuroscience (SITraN) have used a new imaging technique to measure the function of mitochondria in patients with motor neuron disease (MND).
via Bing News