A potential new treatment for motor neurone disease (MND) developed by NuNerve, a company spun-out of The University of Queensland, has produced successful results in a human clinical trial. The lead drug candidate, NUN-004, has proven to be safe and effective in the Phase 1 study, offering a new therapeutic option for patients with MND.
Emeritus Professor Perry Bartlett from UQ’s Queensland Brain Institute (QBI) expressed encouragement by the study's results, highlighting the drug's potential to not only slow disease progression but also improve gross and fine motor movement. 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.
MND progressively attacks nerve cells in the brain and spinal cord, affecting over 2000 Australians annually. The research by Professors Bartlett and Boyd, which began in the late 1990s, focused on the EphA4 protein's role in guiding motor neurons and its impediment to their recovery after injury. Their collaboration with Dr Mike Gerometta led to the engineering and patenting of NUN-004, an effective EphA4 blocker used in this clinical trial.
Professor Bartlett emphasized the need for vital funds to advance the drug candidate through the next stage of development, pointing out the dire need for better MND treatments. Currently, only three drugs globally may slow MND progression and partially improve quality of life, with no treatment reversing the disease. This trial underscores the importance of fundamental research into the brain and nervous system, with potential broader applications for stroke, spinal cord injury, and sepsis therapies.
NuNerve, formed by The Peter Goodenough Trust and UQ’s commercialisation company UniQuest, has licensed intellectual property from UniQuest to focus on new technologies for preventing or treating MND. The research has been supported by Fight MND, the Queensland Government, and the BioPharmaceuticals Australia Development Fund, with findings published in Clinical Drug Investigations.
