A potential new treatment for motor neurone disease (MND), NUN-004, developed by NuNerve, a spin-out company from The University of Queensland (UQ), has shown promising results in a Phase 1 human clinical trial. The study indicates that NUN-004 is safe and may be effective in slowing disease progression and improving motor movement in MND patients.
The Phase 1 trial involved eight individuals with MND and twenty healthy volunteers who were administered NUN-004 over a six-month period. The trial was supervised by Associate Professor Robert Henderson and Dr. Jing Zhao from UQ. Encouragingly, the results indicated signs of disease stabilization among the MND patients. Furthermore, there were positive anecdotal reports of improvements in both gross and fine motor movement.
Targeting EphA4 for MND Treatment
The research behind NUN-004 stems from over two decades of work by Emeritus Professors Perry Bartlett and Andrew Boyd at UQ’s Queensland Brain Institute (QBI). Their research identified the EphA4 protein as a key factor in impeding the recovery of motor neurons after injury. NUN-004 is engineered to block the action of EphA4, with the goal of preserving motor neurons, improving motor function, and potentially halting the progression of MND.
"We are enormously encouraged by the results of this study," said Professor Bartlett. "It shows NUN-004 has the potential to provide a new therapeutic option for patients with motor neurone disease – not only in slowing disease progression but also improving gross and fine motor movement."
The Need for New MND Treatments
MND is a progressive disease that affects nerve cells in the brain and spinal cord. It impacts over 2,000 Australians annually. Currently, there are only three globally approved drugs that may slow the progression of MND and partially improve the quality of life, but no treatment reverses the disease. NUN-004 represents a potential breakthrough in addressing this significant unmet medical need.
Future Development
NuNerve, formed through a partnership between The Peter Goodenough Trust and UQ’s commercialization company UniQuest, has licensed intellectual property from UniQuest to focus on developing new technologies for preventing or treating MND. The development of NUN-004 has been supported by Fight MND, the Queensland Government, and the BioPharmaceuticals Australia Development Fund. Further funding is now needed to advance NUN-004 through the next stages of clinical development.
Professor Bartlett emphasized the critical need for better treatments for MND. He also noted that the research could have broader applications for potential future therapies for stroke, spinal cord injury, and sepsis.
