What if a treatment developed for spinal cord injury (SCI) could also help treat another devastating neurological disorder? This may be a possibility thanks to promising results from a recent clinical trial showing that NUN-004 may be effective in slowing the progression of motor neurone disease (MND). Apart from benefitting MND patients, this sets the stage for NUN-004 to be further tested as a treatment for other closely related conditions, including SCI.
The trial, which was published in Clinical Drug Investigations, was conducted by NuNerve, a company formed by the University of Queensland’s Queensland Brain Institute (QBI). It was led by SpinalCure Director and Scientific Chair Emeritus Professor Perry Bartlett from QBI and Professor Andrew Boyd from the Walter and Eliza Hall Institute of Medical Research (WEHI). The initial development of NUN-004 was funded by SpinalCure as a treatment for SCI, thanks to a bequest from the late Lisa Palmer – a courageous young woman who was left a quadriplegic after a car accident and later died of cancer at just 29 years old.
“Lisa Palmer’s legacy will fuel the quest for a cure; her contribution may one day prove to be priceless.” said Prof John Hay AC, then Vice-Chancellor of UQ, on accepting the funding. With the results of this trial his prediction is becoming a reality.
NUN-004 is a novel therapeutic based on blocking the action of EphA4, a protein which acts like a traffic signal for cells in the nervous system. According to Professor Bartlett, the aim of the research with MND was to explore if this blocking action could preserve motor neurons, potentially improving motor function and halting the disease. The results of the trial, which tested NUN-004 on eight people with MND and 20 healthy volunteers over a six month period, indicated just that.
“Not only did we see signs of the disease stabilising in the MND patients but, importantly, they gave positive anecdotal feedback on improved gross and fine motor movement,” he explained.
Leveraging similarities between SCI & MND
There are several parallels between SCI and MND, creating strong synergies for an integrated approach to research. With an SCI, the spinal cord consists of a complex extension of the central nervous system that acts as a telecommunications system between brain and body. When this is injured or broken the result is a loss of movement (either paraplegia or quadriplegia). It also deprives one of sensations like pleasure, touch and pressure, the control of the bladder and bowel and more. Comparatively, MND affects the nerves known as motor neurons. These are found in the brain and spinal cord and help tell the body’s muscles what to do. Consequently, MND impacts movement and can result in a wide range of symptoms including muscle weakness, cramps, spasms and pain.
Further SCI focus on the horizon
Professor Bartlett’s NUN-004 research began over 25 years ago, with original findings demonstrating its importance in regulating spinal cord motor neuron development and promoting the regeneration of neuronal connections following an SCI. The latest results further expand the possibilities for NUN-004’s application in the SCI field, paving the way for a new treatment option to be developed in the future.
“Now that it has been shown to be safe in humans, the plan is to fund and develop a clinical strategy to investigate NUN-004’s potential to promote neuronal reconnection in patients with SCI,” concluded Professor Bartlett.