By Alison Jones, Coordinator Information & Resourcing
‘Ask the Experts’ was held on Tuesday 7 May 2019 at the Florey Institute of Neuroscience & Mental Health in Parkville and consisted of three presentations by researchers, followed by tours of the laboratory facilities.
Associate Professor Brad Turner gave an update on local and international MND research. He explained that 10% of MND cases are inherited and 90% are sporadic (scattered or isolated) cases. Possible risk factors for developing MND include smoking, playing sport at an elite level, retroviruses, blue-green algal blooms and military deployment in the Gulf War. He spoke about genetics, advising that 70% of MND related genes have now been found and that they point to various disease pathways and potential treatments. It is hoped that the remaining 30% of genes will be identified over the next 5 years.
Brad talked about how MND is a complex disease with many phenotypes (clinical presentations) and that current medical practice tends to adopt a ‘one size fits all’ approach. However precision medicine, where the focus is on identifying which approaches will be effective for which patients based on genetic, environmental and lifestyle factors, is likely to be the way of the future.
Brad also spoke about the Florey's high-throughput Drug Screening Program which aims to find potential new treatments for MND. The effects of thousands of drugs on motor neurones derived from the skin cells of people with MND will be tested. Lab tour participants were given the opportunity to see the multi-million dollar state-of-the-art drug screening equipment in action. Five groups of participants were rotated through various lab stations including cell culture, mice, molecular, microscope and stem cells where staff spoke about their current MND research and answered participants’ questions.
Associate Professor Peter Crouch from the University of Melbourne reported on the Copper- ATSM clinical trial that has been the subject of recent media attention. Peter explained that copper is found in all body tissues and plays a role in various processes such as energy production. The drug Copper- ATSM has been in existence for many years and has been recognised as having the potential to treat neurodegenerative diseases such as MND. The phase 1 trial started with a small group of MND patients in 2016 and it is hoped that the phase 2 trial, which will involve a larger number of patients, will commence in late 2019, however details are yet to be announced. Peter added that a complex disease like MND is likely to require complex treatments involving a combination of potential therapies.
Dr Tristan Iseli from Neuroscience Trials Australia was the final speaker and provided an overview of clinical trials and MND. He explained that clinical trials are performed for safety and tolerability, to find a dose that works and establish who it works for. Clinical trials must always be conducted ethically and the risks and benefits weighed up carefully. In addition, the rights, safety and wellbeing of the participants are of utmost importance.
The clinical trial process is both long and expensive, with the development of a new drug costing on average more than $US800 million from discovery to approval. It is a complicated process that typically spans 10-15 years which can be frustrating for people with MND and their families who hope for effective new treatments to be available sooner rather than later.
The knowledge that Brad, Peter and their labs collaborate with the best and brightest researchers internationally to find effective treatments for MND is a source of hope for those whose lives are touched by MND. Feedback from one MND Victoria client summed up how the event was received:
‘Thank you for the opportunity given to hear and see what is being done at the Florey Institute...it showed me that marvellous things are being done through the hard work of so many of you.’
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