The next State of Play will be held on Tuesday, 25 July at 7pm (AEST). The webinar features Professor Trent Woodruff and Dr Margreet Ridder, both from the University of Queensland. Margreet and Trent are 2023 MNDRA Innovator Grant recipients and for this State of Play they will look at the topic of "Bringing new approaches to developing treatments for MND". Register here. Previous episodes available here.
For more insights into how a medicine progress from scientific experiments to doctors being able to prescribe it for MND in Australia, read our latest information piece: How research and the medical system develop medicines for MND in Australia
Professor Matthew Kiernan AM has been appointed to the position of Chief Executive Officer and Institute Director of Neuroscience Research Australia (NeuRA). NeuRA is an independent, not-for-profit, medical research institute dedicated to improving the lives of people living with brain and nervous system disorders based in Sydney. Hopefully this will provide new opportunities for MND research in Australia.
Monepantel
Monepantel is a drug that works to increase the recycling and removal of excessive or misprocessed proteins. Abnormal protein aggregates in neurones in MND and other neuro-degenerative conditions are believed to disrupt cellular functions and contribute to cell toxicity and cell death.
Monepantel is used widely to treat animals and has been shown to be safe in a small phase 1 trial in humans with cancer.
PharmAust are currently running a Phase 1 trial for MND in Melbourne to test the safety and tolerability of oral monepantel tablets and find the most appropriate dose of monepantel to treat MND in people. Early results indicate the drug is targeting the correct pathway and the therapy is progressing to the next stage of the trial.
ARO-SOD1
Arrowhead Pharmaceuticals announced that it has filed an application for approval to initiate a Phase 1 clinical trial of ARO-SOD1, a genetic therapy designed to reduce expression of superoxide dismutase 1 (SOD1, a gene known to cause MND in 2% of those diagnosed with the disease), in Australia.
This is the same target as Biogen’s Tofersen that was recently approved in the US, but uses a different approach. Such competition is good as it encourages innovation and continuing improvement as well as price competition, if approved.
In 2018, a parliamentary inquiry into the Life Insurance Industry recommended that Australia urgently implement a moratorium (or ban) on the use of genetic test results in life insurance underwriting. This moratorium is industry self-regulated, with no government oversight.
An investigation into the effectiveness of this moratorium found that the moratorium is inadequate to prevent genetic discrimination in life insurance and should be replaced with a legislative model of prohibition. This is important for MND patients.
As more genetic therapies and knowledge about the role of genes in MND becomes available, more patients and their family members will undergo genetic testing. It is critical that the potential risk of discrimination by insurance companies does not inhibit willingness to undergo potentially informative genetic testing.
Neurodegenerative diseases of the motor system are now recognised to also affect non-motor pathways. Non-motor symptoms have been acknowledged as important determinants of quality of life in Parkinson’s disease, and there is increasing interest in understanding the extent and role of non-motor symptoms in MND.
This study, from Ammar Al-Chalabi’s group at King’s College, London, has reviewed what is known about non-motor symptoms in MND, using lessons from Parkinson’s disease. They showed non-motor symptoms have been understudied in MND and represent an unmet need in the care of patients.
Better definition and improving understanding of the frequency and characterization of their natural history will enable clinicians and researchers to potentially develop future therapies to address these important symptoms.
Dr Rebecca San Gil and team at the University of Queensland have undertaken large-scale analyses of the protein changes in the brain cells from a mouse model of MND. They have produced a “molecular map” of the changes that occur in the cell during disease progression and have identified specific changes occurring at different disease stages.
Targeting these changes is a promising avenue for the future development of therapies to treat MND and may provide disease stage-specific opportunities.
