MND Australia

Research Directions | October 2024

21 October 2024

Welcome to the MNDRA research update. In this report we will highlight outcomes and advances from the MND research world that have caught our attention over the last few weeks.  

Industry and Clinical Trial news 

  • PharmAust Limited has announced it has officially changed its name to Neurizon Therapeutics Limited. The drug they are developing, Monepantel, which is about to enter into the Healey platform trial in the US, will now be known as NUZ-001.  Neurizon Therapeutics Chair, Sergio Duchini, was quoted “The Neurizon Therapeutics name and brand align with our focus on creating a promising horizon for patients facing complex neurodegenerative diseases. Starting with Amyotrophic Lateral Sclerosis (ALS), the most common form of Motor Neurone Disease, we’re committed to reshaping patient treatment and making meaningful progress in neurodegenerative research”. 
  • Following approvals in the US, Canada and Europe, China has given conditional approval for Biogen's genetic therapy, Qalsody (tofersen), to be available to MND patients carrying mutations in the SOD1 gene. Biogen are currently preparing an application to the TGA for approval in Australia. Tofersen is currently available in Australia for eligible patients through Biogen's Early Access Program. 
  • Eisai Co., Ltd. announced that it has obtained manufacturing and marketing authorization approval for ALS treatment “Rozebalamin® (mecobalamin) in Japan. Methylcobalamin is a very high dose of the biologically active form of vitamin B12 which is thought to decrease levels of homocysteine, a molecule that can contribute to neuronal degeneration. Further information is available via ACN Newswire and the International Alliance of ALS/MND Associations. 
  • Synchron have announced the results of their COMMAND brain computer interface (BCI) device trial, which was implanted in six participants over a 12-month period. They demonstrated that simple, thought-derived expressions of intent could be converted into digital actions on computers. The Synchron BCI is implanted in the blood vessel on the surface of the motor cortex of the brain via the jugular vein, through a minimally-invasive procedure. Once implanted, it is designed to detect and wirelessly transmit motor intent out of the brain. As an endovascular BCI, the fully internalized system offers long-term signal stability, providing a new path for patients to regain functional independence through digital interfaces. It is hoped the device will restore the capability for severely paralyzed people to control personal devices with hands-free point-and-click.  
  • The 14th of October was Allied Health Professionals Day and this year’s theme was ‘stronger together’. Allied health professionals include physiotherapists, speech pathologists, among others, and are critical in providing care to those living with MND.   
Other MND News
  • The 2024 Nobel Prize in Physiology or Medicine has been awarded to Victor Ambros and Gary Ruvkun for the discovery of microRNA and its role in post-transcriptional gene regulation. MicroRNAs have been implicated in regulating pretty much every system in the body, and a number of microRNAs have been shown to be associated with MND. Previous to discovering these molecules, they were thought to be junk that had no function. A recently published paper showed that a group of eight microRNAs in the blood of patients can be used as a biomarker to distinguish MND from other neurodegenerative diseases. 

Not MND specifically but might well be applicable 

  • A review has shown that there is increasing evidence that subclinical dysfunction of the cardiovascular, pulmonary, renal, gastrointestinal, hepatic and endocrine systems is associated with age-related cognitive decline and dementia. Pathways that might lead from organ dysfunction to brain dysfunction include reduced peripheral clearance of wastes (including amyloid-β and tau), systemic inflammation and alterations in the microbiome. All of these processes are thought to be disrupted in MND as well. The brain requires a supportive environment maintained by healthy organ systems to function optimally and to confer resilience to neurodegenerative diseases. This presents more evidence that there are many factors and pathways that might contribute to MND. 
  • A study in Alzheimer’s Disease has shown that microglia (immune cells that support neurones) are disrupted through changes in the STING pathway.  The STING pathway has been shown to be involved in MND (through research partially funded by MNDRA), and specific inhibitors of this pathway are currently being investigated as potential treatments. This is another example of the crossover between different neurodegenerative diseases where neuroinflammation and protein aggregation play roles.