The Motor Neuron Disease Research Centre is hosted by the Faculty of Medicine, Health and Human Sciences. This Strength-at-Scale Centre aims to harness an integrated discovery and evidence-based clinical program to deliver advances in patient outcomes and improve the lives of Motor Neuron Disease (MND) patients and their families.
“The goal is to have a real-world impact by improving capacity to diagnose and treat MND,” says Professor Ian Blair.
The MND Centre has built a multidisciplinary research pipeline through capacity-building grants funded by the National Health and Medical Research Council (NHMRC), the Australian Research Council (ARC) and major philanthropic bodies FightMND and MND Research Australia (MNDRA). Work has focused on genetics, molecular origins, therapeutic development (drug screening, stem cells, zebrafish, mice – all at-scale and linked with industry) and clinical trials. Six trials have been completed in the past two years, including the first clinical trial to be sponsored and led by Macquarie.
The centre operates as an integrated health sciences centre, with collaborative research between the clinic (patients, family members, neurologists and genetic counsellors) and the laboratory. Patients are recruited and engaged in the centre’s research through the biobank and clinical trials. Macquarie University is the only site in the southern hemisphere to be trialling tofersen, a groundbreaking new drug designed to treat inherited MND. Read more in The Lighthouse >>
The University recently established a spin-out company, Celosia Therapeutics, to commercialise the centre’s IP, including candidate MND gene therapies based on the centre’s research outcomes.
With an aim to build research capacity – including capacity to adapt and diversify as the research field evolves – the centre aims to develop primary research themes to foster cross-disciplinary research among multiple groups, addressing big-picture problems in MND.
- diagnostics and prognostics, encompassing biomarker discovery, development and translation
- therapeutic discovery, development and translation
- in-vivo modelling.
The centre brings together some of Australia’s leading MND researchers with experts from complementary research disciplines to build an integrated and collaborative workflow to make breakthrough advances in understanding the origins of MND. This includes expertise in discovery research (to understand the basis of the disease, what causes the motor neurons to degenerate), preclinical research (to identify and develop new possibilities to diagnose and treat the disease) and clinical research (to trial the latest potential therapies).
Professor Julie Atkin, Professor in Neurobiology, Macquarie Medical School
Professor Ian Blair, NHMRC Leadership Fellow, Macquarie Medical School
Professor Dominic Rowe, Professor of Neurology, Macquarie Medical School
Project MinE – which aims to understand the genetic origins of the disease through mapping the DNA of 15,000 patients in 20 countries.
MND Collective – comprising experts from lived experience, medicine, healthcare and research, collaborating to facilitate a collective approach to research that enables better outcomes for people living with MND.
Philanthropic funding – in addition to DVCR funding and support the centre is completely run with the support of public donations and research grants. Since 2009, the MND team at Macquarie has successfully achieved more than $13 million in research funding. The philanthropic funding not only supports clinical trials, but it also supports the Neurodegenerative Diseases Biobank of patient specimens and gives MND patients access to best-practice subsidised care.
Current projects include:
The CCNF/Cyclin F research program
Genetic research among MND families identified a new MND gene. This major breakthrough launched an ongoing research program which includes:
- Cellular modelling of MND. The gene-enabled the development of new cellular models to understand MND biology by mimicking features of the disease in the laboratory.
- Animal modelling of MND. New gene-enabled development of zebrafish and mouse models to mimic the disease in vivo. Unique new tools have also been developed to understand MND biology and assess new therapeutic strategies.
- New therapeutic development opportunities. Manipulating the gene’s expression shows promise as a therapeutic. Patents have been filed and a spin-out company established to attract industry support for therapeutic development.
The genetic and genomic research programs
These programs seek to identify the origins of the disease, using the latest genome sequencing technologies. New gene defects have recently been identified by the centre, including findings published in the prestigious journal Science Advances.
How to engage with the MND Research Centre
Staff interested in contributing to the work of the MND Research Centre are encouraged to find out more on the centre’s website.
Tickets are now on sale for the 2023 MND Gala Dinner on Friday 23 June at the Art Gallery of NSW. The gala will be hosted by Professor Dominic Rowe, Professor of Neurology. All funds raised from the MND Gala Dinner will support Macquarie’s research and clinic, and the centre’s ultimate goal: to end MND. Register >>
We encourage active and constructive debate through our comments section, but please remain respectful. Your first and last name will be published alongside your comment.
Comments will not be pre-moderated but any comments deemed to be offensive, obscene, intimidating, discriminatory or defamatory will be removed and further action may be taken where such conduct breaches University policy or standards. Please keep in mind that This Week is a public site and comments should not contain information that is confidential or commercial in confidence.
I have MND. Diagnosed in 2019. I cannot speak have a peg tube…and have a problem with excessive saliva. Is there any trials at tge moment. My gastroenterologist is Anna Schultz. I was on a trial for 18 months. Cancelled by pharma world wide. I now take Teglutik. Please advise.
Hi Michael. Thank you for you for getting in touch. Please reach out to Professor Dominic Rowe at MQ Health Neurology (email@example.com) with your enquiry.