Until there's a cure ... there's care

Wall at the FloreyBy 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.

New high tech machine at the FloreyDr 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.


Read more news articles from MND Victoria:

/MND%20Week%202019%20-%20Help%20Make%20Aged%20Care%20Fair!
Join our campaign to help make aged care fair for older Australians who face long waiting lists for disability care ...
/Meet%20our%20NEW%20CEO,%20Kate%20Johnson!
Kate has enjoyed over 20 years’ experience in leadership and the strategic development of not for-profit organisations ...
/MND%20News%20|%20March-April%202019
Catch up with all the latest from MND Victoria as well as your MND stories, research updates and farewell to Rod Harris, CEO.
/Tasmanian%20Health%20Professional%20Forum
On 1 March nearly 70 people, including speakers and staff, attended the MND Health Professional Forum in Tasmania.
/Benalla%20puts%20MND%20research%20projects%20on%20the%20map
Over 500 people from Benalla, surrounding communities and families affected by MND attended the Act2D'feet MND last weekend.
/An%20incredible%20$250,000%20raised%20from%202018%20Walks
In an amazing result, the funds raised from our 2018 Walk to D'feet MND in Rosebud, Melbourne, Bendigo and Echuca is over $250,000!
/Copper-ATSM%20Update%20-%20Research%20News
Dr Peter Crouch, University of Melbourne researcher updates us on the clinical outcomes for copper-ATSM as recently reported in the media.
/Behind%20the%20Scenes%20at%20our%20Equipment%20Warehouse
Our Equipment Warehouse is a critical part of our services and makes an enormous difference to the lives of people with MND.
/Largest%20MND%20Medical%20&%20Scientific%20Conference
An exclusive report by our CEO on the Allied Professionals Forum, International Symposium on ALS/ MND and other meetings.
/Benalla%20Act%202%20D'feet%20MND%20|%2024%20February%202019
The annual Benalla Act 2 Dfeet MND Walk/Run/Cycle will be held THIS Sunday (24 February) at Benalla Lake.
/MND%20News%20|%20January-February%202019
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20November-December%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20September-October%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20July-August%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/Research%20opportunity:%20Project%20Train-ALS
Researcher opportunity to explore the role of personalised exercise training in patients with MND ...
/Research%20into%20cognitive%20change%20&%20people%20with%20MND
The focus of our neuropsychology research is to understand more about specific changes in cognition, behaviour and mood ...
/There%20are 40%20trillion%20cells%20in%20our%20body%20...
Associate Prof Justin P. Rubio, University of Melbourne, is studying the genomes of single neurons to understand MND ...
/MND%20News%20|%20May-June%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/Ask%20the%20Experts%20Research%20Seminar%202017
‘Ask the Experts’ was held on Tuesday 8 May 2018 in MND Week at the Florey Institute in Parkville ...
/Make%20Aged%20Care%20Fair%20for%20people%20with%20MND!
Aged Care is failing people with motor neurone disease - we need you to join our MND Action Campaign:
/MND%20News%20|%20March-April%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20Australia%20Research%20Conference%202017
Over 160 dedicated MND researchers and supporters attended this conference held at University of Sydney.
/MND%20News%20|%20January-February%202018
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/Research%20Update%20|%20November/December%202017
Find out about preclinical and current trials of medication being offered to patients with ALS/MND ...
/MND%20News%20|%20November/December%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20September/October%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20July/August%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/Voluntary%20assisted%20dying%20and%20MND
We are working to ensure that people with MND aren’t discriminated against if the proposed reforms take place.
/MND%20News%20|%20May/June%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20March/April%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.
/MND%20News%20|%20January/February%202017
Catch up with all the latest from MND Victoria as well as your MND stories and research updates.