In December 2019, MND Australia, in partnership with MND Western Australia, was proud to host the 30th International Symposium on ALS/MND and affiliated meetings in the sunny city of Perth.

Over 850 delegates attended the symposium, its largest audience yet on Australian soil.

Among the delegates were experts from across the world who presented their insights into the causes and biology of ALS/MND, developments in managing the diagnosis and symptoms of the disease, and what could help in its treatment.

There is still, of course, much to learn about stopping or even slowing ALS/MND. But important progress has been made in studies of genetics, disease mechanisms, biomarkers, inflammation, assistive technology, palliative care, potential drug treatments and related areas of work.

Australian research was a major highlight, including Professor Justin Yerbury and his team’s study of the fine balance of proteostasis, and its implications for ALS/MND. There was standing room only as Justin, who is living with MND, delivered his recorded presentation and answered questions via twitter.

One of the most encouraging symposium themes was collaboration – its potential for both improving clinical trials and research outcomes and harnessing the power of a global effort in stopping MND.

Here we'll cover some of the major developments in clinical trials, the Australian research profiled at the symposium, and an overview of the ALS/MND Connect session, Allied Professionals Forum, the Global Walk to D’Feet MND and other satellite events.

More and better quality ALS/MND clinical trials

There has been major growth in clinical trials, and attempts to improve their design, showing just how essential collaboration is to finding effective treatments in the near future.

At the ALS/MND Connect interactive session for the MND community, held prior to the symposium, Professor Van den Berg, Director Netherlands ALS Center, explained that more potential therapies are now at the trial stage, and with a greater number of patients. The drug development process is accelerating.

Platform trials are one such advancement. They are new to MND, having been previously used in other areas such as cancer research. Using clever trial design, multiple drugs can be trialed in parallel with much reduced placebo group numbers. This means more patients are receiving potential treatments and a significant increase in efficient use of resources. Two platforms are being established, one in the US at the Healey Centre, and one through the European TriCALS organisation.

Other symposium presenters reported on some of the latest clinical trials underway, including: the Healey Centre ALS Platform design and outcomes of Phase 1/2 trial of ibudilast; Phase 2 NurOwn; and, Phase 1/2 SOD 1 antisense oligonucleotide (Tofersen).

The Tofersen trial attracted a lot of interest at the symposium. The trial showed some promising clinical improvements as well as 35% reductions of SOD 1 in the cerebrospinal fluid (CSF) and decrease in neurofilament biomarker.

Work on biomarkers has progressed dramatically. Better biomarkers can help to rapidly demonstrate the effectiveness of potential therapies, minimise delayed diagnosis, and variable prognosis and will avoid the complex technical requirements in measuring the current outcome measures.

One of many satellite meetings brought together those involved in TriCALS which is making important progress, too. TriCALS is a non profit organisation based in Europe developing a Platform Trial program, although with some differences to the US Healey Centre program. Australian centres are now playing key roles in the TriCALS program by enabling access to these trial treatments for Australian patients.

In the TriCALS program, each treatment has its own placebo group. But with the use of very clever statistics, the placebo numbers can be reduced, and combined with placebos from different study groups. The design allows for a wider inclusion criteria, enabling recruitment of most patients, overcoming the challenges with patients progressing at very different rates.

Australian research leading the world

The symposium only comes to Australia approximately every eight years. So 2019 was a rare opportunity for the wider Australian ALS/MND community to network with and learn from leading international researchers as well as Australian scientists, who are world leaders in their fields.

The symposium saw the highest ever number of Australian researchers presenting their work at the event. In addition to Professor Justin Yerbury and his team’s work on proteostasis, Australian researchers included:

• Dr Catherine Blizzard, University of Tasmania (Does oestrogen protect against the synaptic plasticity deficits that underlie motor cortex dysfunction in ALS);
• Professor Naomi Wray, University of Queensland (Future directions in ALS genomics);
• Professor Samar Aoun, Perron Institute Western Australia (Supporting MND family carers from diagnosis to bereavement);
• Dr Frederik Steyn, University of Queensland (Loss of appetite is associated with a loss of weight and fat mass in patients with ALS).
• A/Prof Brad Turner, Florey Institute of Neuroscience and Mental Health, University of Melbourne (Mouse models of ALS: past, present and future);
• Dr Marco Morsch, Macquarie University, (Targeting the nucleo-cytoplasmic transport machinery: why does TDP-43 mislocalise?); and,
• Professor Julian Trollor, University of New South Wales (The impact of mental health on acute health service provision in motor neurone disease: a big data study).

Of course, many other leading Australian researchers made valuable contributions to the symposium, helping to enhance Australia’s standing in the ALS/MND community. Read more on:

• Australian ALS/MND researchers at the Symposium
• Platform and poster presentations abstracts
• Australian researchers funded by our research arm the MNDRIA

MND Connect, Allied Professionals Forum and the Global Walk to D’Feet MND

Many other meetings, workshops, panel sessions and events were held as part of the symposium, and to involve the broader community working to improve care and support and stop ALS/MND.