A new clinical trial gets the green light as researchers from UQ’s Australian Institute for Bioengineering and Nanotechnology (AIBN) investigate whether a common heart medication could help treat people living with the form of motor neurone disease known as Amyotrophic Lateral Sclerosis (ALS).
Led by AIBN’s Associate Professor Shyuan Ngo, the trial of 150patients with ALS will test whether the drug Trimetazidine can improve function and quality of life.
“ALS is the most common form of motor neurone disease (MND) and gradually erodes a person’s ability to control voluntary muscle and movement, and to breathe on their own,” Dr Ngo said.
“More than 2,000 people have MND in Australia and currently there is no cure or effective treatment.
“Being able to improve function, quality and life, and overall clinical outcomes for a person living with ALS would be life-changing for thousands of people and their families.”
“While ALS has traditionally been thought of as a disease of the brain or spinal cord, we now know that changes to a patient’s metabolism - or the rate they use energy - is linked to worse outcomes,” Dr Ngo said.
“If we can prove in this clinical trial that Trimetazidine improves metabolism and function in ALS patients, doors will open for more research into how other existing drugs could be repurposed to treat ALS.”
This clinical trial is part of Dr Ngo’s ongoing research exploring how metabolic factors, such as nutrition, may play a key role in managing ALS.
“Modifying metabolic flexibility, or the balance between how a cell or tissue accesses sugars or fat as an energy source, is still an emerging area of research when it comes to MND and ALS and the more we understand about it, the greater the chance we can find new treatments,” Dr Ngo said.
Set to begin early next year, the Trimetazidine clinical trial will involve participants from Australia, the Netherlands, Belgium, Spain, and Italy.
It's one of three initiatives led by Dr Ngo, who was awarded $3.1 million in total funding from FightMND’s 2024 grant round.
The additional projects will investigate the role of muscle cell particles in ALS and gather data on how the disease manifests differently in individuals, which will inform the development of a global web tool to support MND research efforts.
This exciting development builds on Dr Ngo’s success as Co-Director of UQ’s new Centre for MND Research, officially launched earlier this year.
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