Welcome to the MNDRA research update. In this report we will highlight outcomes and advances from the MND research world that have caught our attention over the last few weeks.

MND Research Australia news

• Registration is open for the 3rd Australian and New Zealand MND Research Symposium in Melbourne on Tuesday 27th and Wednesday 28th of August. Abstracts have closed, with a record number of abstracts submitted. This symposium promises to be a festival of top quality research, with a  number of international speakers also presenting. Keep an eye on our symposium webpage for updates. 
• Nominations are invited from interested individuals to be on the new NHMRC-MRFF (National Health and Medical Research Council-Medical Research Future Fund) Consumer Advisory group. A range of advisory committees are being established to provide advice on health and medical research in Australia. Nominations close on Wed 31 July 2024 at 5:00PM AEST. To find out more about the advisory group, including information on how to submit your nomination, visit: https://ow.ly/QF0X50SyrU8
• Applications for the MNDRA grant funding round for research commencing in 2025 are now open
• On the 24th of June 2024, MND Australia hosted an event at Australian Parliament House in Canberra and awarded lifetime achievement medals to 30 people who have made a significant contribution to the MND community over the last 30 years. Many researchers were included in this list as well as Janet Nash who headed up MNDRA from its inception to 2019. We are grateful for the work of these amazing people, and thank them for everything they have done to improve the lives of those affected by MND.  

Other news

Congratulations to Professor Roger Chung of Macquarie University for receiving the NHRMC 2023 Marshall and Warren Innovation Award. This award goes to the top-ranked and most innovative and potentially transformative research. Professor Chung's project will develop a new technology for gene expression to be incorporated into gene therapy for MND. Successful outcomes of this project will make an important contribution towards clinical development of future gene therapies for MND. 

Clinical trial news

• The US-based Healey ALS Platform trial is the first of its kind, allowing for multiple investigational drugs to be tested and evaluated simultaneously to accelerate the development of potential new MND therapies. PharmAust Limited will soon begin planning for inclusion of their drug Monepantel in the Healey platform trial. This will likely be a Phase 2/3 trail with the platform providing an efficient option for progressing this drug through the therapeutic testing pipeline. Unfortunately, this will mean the trial will not have any Australian sites.
• Real-world analysis suggests 3.2-month survival benefit for individuals treated with RADICAVA (edaravone) and riluzole compared to those treated with only riluzole. Mitsubishi Tanabe Pharma America have announced the findings from an ongoing real-world study using information in the ALS/MND Natural History Study Consortium (Consortium) database of people living with ALS. This analysis suggests that initiating treatment with RADICAVA (edaravone) provides an additional survival benefit of 3.2 months compared to treatment with riluzole alone. With RADICAVA having recently been approved in Australia it is good to see demonstrated benefit of this additional treatment option. You can read more about the Australian approval of RADICAVA in the May edition of Research Directions. 
• Trial Participation in Neurodegenerative Diseases: Barriers and Facilitators - A Systematic Review and Meta-Analysis. This study identified barriers to and facilitators of trial participation in four major neurodegenerative disease areas: Alzheimer disease, Parkinson disease, MND and Huntington disease. They identified a comprehensive list of reasons why patients choose to participate in clinical trials for neurodegenerative diseases. These results may help to increase participation rates, better inform patients and facilitate patient-centric approaches. The study identified similar factors across the different diseases. Key factors were health benefit and altruism, treatment and study burden, relationship with clinical staff, availability of study information and the use or absence of a placebo or sham-control arm. 

Other Research Outcomes

Serum neurofilament light chain in distinct phenotypes of amyotrophic lateral sclerosis: A longitudinal, multicentre study 

A biomarker is a measurement of a biological sample, such as blood or urine, that can indicate disease and can be used to measure disease progression. There is not yet an established biomarker for MND. 

Over the last few years, we have been hearing a lot about the potential of neurofilament light chain (NfL) as a biomarker for measuring MND progression. However, it is not clear how levels of NFL vary across different subtypes of MND and whether this can confound interpretation of disease progression. This issue is of relevance for the design of clinical trials when using NfL levels for enrolment in trials. This study showed that different MND subtypes, such as motor neuron involvement and region of onset, demonstrated a significant effect on NfL and should be taken into account when studying NfL in trials and research studies. Although NfL is very promising in some situations it is still too early to use it as general MND biomarker for all studies.