By Alison Jones, Coordinator Information & Resourcing
‘Ask the Experts’ was held on Tuesday 8 May 2018 at the Florey Institute of Neuroscience & Mental Health in Parkville and comprised presentations by three researchers followed by tours of the laboratory facilities.
Associate Professor Brad Turner gave an update on international & Florey MND research. He explained that 10% of MND cases are inherited and 90% are sporadic (scattered or isolated). Possible risk factors include smoking, playing sport at a professional level, retroviruses, blue-green algal blooms and military deployment in the Gulf War.
He spoke about genetics, advising that 70% of MND genes have now been found and that they point to disease pathways and potential treatment clues. He believes that the remaining 30% of genes will be identified over the next 5 years.
Part of Brad’s presentation also focused on a new Drug Screening Program that is currently recruiting participants. He explained that MND is highly clinically variable with many different phenotypes (clinical presentations) and that in MND ‘one size does not fit all’ for clinical trials. One of the problems is a lack of effective treatments for MND due to factors including high disease variability, a lack of relevant sporadic MND models, a lack of human models, and until now, a lack of investment in large scale drug screening.
A new $5M program is now operating at the Florey for sporadic MND which aims to fast track drug screening. The expensive, state-of-the-art equipment involved in this project was demonstrated to participants as part of the lab tours. There were five stations that participants were rotated through including cell culture, mice, molecular, microscope and stem cells where lab staff spoke enthusiastically about their current work with MND.
Dr Fazel Shabanpoor talked about gene therapy for neurodegenerative diseases including MND. He started by advising that the US FDA (Food & Drug Administration) has recently approved the first drug for Spinal Muscular Atrophy (SMA) called Spinraza. SMA has similarities to MND hence the excitement generated by the availability of this new drug treatment.
In discussing gene therapy, Fazel explained that this involves replacing a mutated gene that causes disease with a healthy copy of the gene and removing or inactivating a mutated gene that causes disease. He also discussed anti-sense therapy which involves inactivating a mutated gene that is functioning improperly.
Dr Tristan Iseli from Neuroscience Trials Australia 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 to find out 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.
People take part in clinical trials for a variety of reasons including to advance medicine, to help improve the lives of others, to help improve their own condition, and in some cases to earn extra money and receive free medical care. It can be a long, complicated process.
To illustrate this, Tristan advised that of over 10,000 compounds synthesised in the lab across the industry, only one compound will reach the market as a new drug and that it takes on average 12-15 years to develop a drug at an average cost of up to $US800 million (from discovery through to approval). There are over 55,000 clinical trials of various types currently in operation globally with around 6,600 of these in Australia.
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