Research Director's Report: Advance December 2023

Published: December 2023

Ups and downs in MND clinical trials and new treatments

It has been another action-packed six months in the world of MND research. 

In positive news we have seen new treatments for MND make their way to Australia, however we have also seen the failure of some clinical trials. Some of these outcomes are disappointing however they demonstrate the hard work that is underway and it is just as important to know what doesn’t work as well as what does!

Edaravone, the first MND treatment to be made available in Australia since 1997, has been approved by the Therapeutic Goods Administration. The Pharmaceutical Benefits Advisory Committee met recently to consider including Edaravone for reimbursement on the Pharmaceutical Benefits Scheme and the outcome of this will soon be announced.

Tofersen (Qalsody), the SOD1 genetic therapy, continues to be available in Australia through Biogen’s Extended Access Program (EAP). So in essence that is two new therapies in Australia in the last few months!

There have been some losses too. A second genetic therapy for C9ORF72 failed, and this may mean a return to the drawing board for this type of therapy. Pegcetacoplan, a therapeutic that targets neuro-inflammation and Reldesemtiv, a therapeutic that aimed to increase muscle function, both also failed trials.

We have also seen the news come out that several treatments have failed across the three platform trials running in the US and Europe: the Healey, MND-SMART and the TRICALS Magnet trial.

Although this news is disappointing, it should also be considered that these outcomes have been produced quickly and efficiently. This saves resources and participant burden and frees up resources to test the next drug in the pipeline. This is exactly how these trials are supposed to work.

There are now three MND clinical trials underway that have resulted from initial Australian research. These are the ALS-205, AMBALS and Metflex trials, all of which are being run in Australia. This is fantastic to see and shows the strength of Australian MND research.

What have MND Research Australian been up to?

I have taken the opportunity to get out and about in 2023 to support varied fundraising efforts around the country. Although all very different in scale and format, they are all linked by the profound impact that the resilience and bravery of someone fighting MND leaves in these communities. It is always an absolute pleasure to attend these events and meet the brilliant fundraisers.

The events included the Shag Gregory Memorial Poker Run, the Stomp for David Debutante Ball, the MND and ME Scott Sullivan Ball, the Superball and the Narrandera Rotary Golf Day.

I also had the great experience of attending my first Pan-Asian Consortium for Treatment and Research in ALS (PACTALS) meeting from the 14–16th September in Kuala Lumpur. The meeting was a fantastic opportunity to meet local MND associations from Asia. Interestingly, there was a strong representation from drug companies at the meeting, demonstrating the increasing importance of the Asian region for clinical trials.

In addition to the 2024 grant announcements, we also announced the recipients of the Daniel McLoone Major Research Initiative, which is jointly funded by MND Research Australia (MNDRA) and FightMND. This unique research initiative was made possible by an incredibly generous bequest by Daniel McLoone’s family from his estate following his death from MND. The grants aim to support an outstanding innovative and collaborative project that has the capacity to make a significant impact on Australian MND research. MNDRA worked with FightMND to double the amount available for this grant program enabling us to jointly fund two projects.

We are striving to provide a broad range of research communications to connect with the wider MND community and highlight advances and outcomes from local and international research efforts. In addition to our twice-yearly Advance publication, we now have monthly online Research Directions updates, as well as regular State of Play webinars throughout the year.

On a very sad note, the MND research community lost a giant of research and a staunch advocate with the passing of Professor Justin Yerbury in July 2023. Refusing to give in to this cruel disease that afflicted him and took many of his family members, Justin forged a brilliant research career leading an amazing team at the University of Wollongong developing new treatments for MND. As a mark of his huge contribution he received the Eureka Prize for Scientific Research and the Excellence in Medical Biological Sciences Prize in the 2022 NSW Premier’s Prizes for Science & Engineering.

MND Research Collective

The MND Research Collective continues to build momentum. The Lived Experience, Discovery Research and Clinical Care Expert Driving Teams are now well-established and meeting monthly to build collaborations, share resources and ensure the lived experience voice is central to everything we do. Most states now have local “nodes” allowing researchers right across the lab and clinical/care spectrum to meet and identify synergies and collaborations.

In May the Collective held a workshop bringing together the Discovery and Clinical Care Expert Driving Teams and establish the Lived Experience Expert Driving Team (LEED). The workshop identified a number of key issues that needed to be addressed and identified some key priorities moving forwards.

Thank you to our generous donors, who continue to allow critical MND research to continue through our research grant program. We are, as always, very grateful.

Dr Gethin Thomas
Executive Director Research