A potential new treatment for motor neurone disease (MND) developed by a company spun-out of The University of Queensland has produced successful results in a human clinical trial.
NuNerve, formed to commercialize research from UQ's Queensland Brain Institute (QBI), has announced its lead drug candidate NUN-004 had proven to be safe and effective in thePhase 1 study.
QBI Emeritus Professor Perry Bartlettsaid the results came after more than 2 decades of MND research alongside long-term colleague Emeritus Professor Andrew Boyd.
We are enormously encouraged by the results of this study.
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."
Perry Bartlett, QBI Emeritus Professor
The trial involved 8 people with MND and 20 healthy volunteers dosed with NUN-004 over a 6 month period, under the supervision of UQ's Associate Professor Robert Henderson and Dr Jing Zhao.
"There were signs of the disease stabilising in the MND patients but also importantly, they gave positive anecdotal feedback on improved gross and fine motor movement," Professor Bartlett said.
MND progressively attacks nerve cells in the brain and spinal cord and affects more than 2000 Australians every year.
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Emeritus Professor Bartlett and Emeritus Professor Boyd began their research partnership in the late 1990s.
"We showed that the EphA4 protein was essential for guiding motor neurons from the brain to targets in the spinal cord," Professor Bartlett said.
"But further studies showed EphA4 was also responsible for impeding the recovery of motor neurons after an injury.
"Our theory was that by blocking this action we could preserve the motor neurons and potentially improve motor function and halt the disease."
The team collaborated with Dr Mike Gerometta to engineer and patent an effective EphA4 blocker, NUN-004, used in this clinical trial.
Professor Bartlett said vital funds were now needed to bring the drug candidate through the next stage of development.
"There is a dire need for better treatments for MND," he said.
"There are only 3 drugs approved globally which may slow the progression of MND and partially improve quality of life, but no treatment that reverses the disease.
"This trial demonstrates the importance of fundamental research into the brain and nervous system and it's gratifying to see more than 23 years of research now showing potential benefit to patients.
"The research could also have broader applications, as potential future therapies for stroke, spinal cord injury and sepsis."
NuNerve was formed by partners The Peter Goodenough Trust and UQ's commercialisation company UniQuest.
NuNerve has licensed intellectual property from UniQuest as part of its focus on new technologies to prevent or treat MND.
This research has been supported by Fight MND, the Queensland Government and the BioPharmaceuticals Australia Development Fund.
The research paper was published in Clinical Drug Investigations.
The University of Queensland
Journal reference:
Gerometta, M., et al. (2024) Evaluation of NUN-004, a Novel Engineered Ephrin Antagonist, in Healthy Volunteers and Patients with Amyotrophic Lateral Sclerosis: A Phase I/Ib, Open-Label, Escalating Dose and Extended Access Study. Clinical Drug Investigations. doi.org/10.1007/s40261-024-01410-x.