Emeritus Professor Perry Bartlett of UQ's Queensland Brain Institute.
(Photo credit: The University of Queensland. )
An Australian-developed therapy aimed at halting motor neurone disease progression and restoring function will advance to a national Phase 2 clinical trial.
The 75-participant trial follows encouraging earlier trial results developed from research at The University of Queensland's Queensland Brain Institute (QBI) and advanced by clinical-stage biotechnology company NuNerve.
NuNerve was established by UQ's commercialisation company, UniQuest, and the Estate Peter Goodenough Unit Trust to advance more than 25 years of research originating at QBI by Emeritus Professors Perry Bartlett and Andrew Boyd.
Emeritus Professor Bartlett AO and NuNerve chair said a protein-based therapy, called NUN-004, was designed to inhibit a receptor, EphA4, whose excessive signalling has been linked to motor neuron degeneration and impaired recovery of nerve connections.
Targeting nerve repair
Professor Bartlett said the therapy was developed to pursue a more ambitious goal than slowing decline alone.
"No approved treatment has been shown to halt or reverse progression across the broad MND population," he said.
"Current treatments largely focus on slowing the loss of function.
"NUN-004 was designed to go further to protect motor neurons and create the conditions for damaged neural connections to repair.
"We have spent more than 25 years following the biology of EphA4, from understanding its role in motor neuron connections to developing a therapy that can now be tested rigorously in people living with MND."
Motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), is a progressive neurological condition that affects the nerve cells in the brain and spinal cord responsible for movement.
As these motor neurons deteriorate and die, the brain can no longer send signals to the muscles, leading to muscle weakness, wasting, and loss of function over time.
Discovery to clinical translation
The trial will recruit 75 participants across 6 Australian clinical sites at the Sunshine Coast, Sydney, Newcastle, Brisbane, and Perth.
Researchers will assess change in function, safety and tolerability, as well as respiratory, neurophysiological, quality-of-life and biomarker measures.
NuNerve Clinical Lead Associate Professor Antony Winkel said the study had been carefully designed to determine whether NUN-004 could meaningfully change the course of MND.
"This trial combines measures of everyday function with respiratory, neurophysiological and biomarker assessments to determine whether the early Phase I signal is a real treatment effect," Professor Winkel said.
"If those findings are confirmed, they would represent an important result for people living with MND and place Australian MND research firmly on the international stage.''
UniQuest CEO Dr Dean Moss said progressing NUN-004 into a Phase 2 clinical trial was an important milestone in the long journey from university research to potential new treatments.
"This is a powerful example of what research translation looks like over the long term, with more than 25 years of UQ discovery now progressing through a company created to take that science forward," he said.
"We're proud of UniQuest's role in helping establish NuNerve and supporting a pathway for this research to be rigorously tested in people living with MND."
