Don’t Say. Do. Big Freeze 10 is here! 

Don’t Say. Do. Big Freeze 10 is here. 

The Big Freeze is back for its 10th year, with FightMND officially launching Big Freeze 10 in an immersive experience unlike any other, all in the aim to raise vital funds to continue the search for a cure for Motor Neurone Disease (MND).  

This year’s launch included a stunning totally immersive experience at THE LUME Melbourne, our official venue partner. The experience brought to life FightMND’s 10 year journey and 9 previous Big Freeze events that have catapulted FightMND into one of the world’s leading funders of MND research.  

This year’s message is a simple adaptation of one of Neale’s most famous mantras – “It’s not what you say, it’s what you do!” and Big Freeze 10 is an opportunity for everyone to DO their bit, and let their actions speak louder than words. 

FightMND was founded a decade ago by Neale Daniher AO, Dr Ian Davis and Pat Cunningham, with the original Big Freeze taking place on Queen’s Birthday 2015. Sadly, Dr Davis and Pat’s wife Angie Cunningham have since passed away because of the disease. But, the legacy they helped create means the fight goes on and FightMND continues to fund critical research into cure and care for those living with MND.  

Big Freeze 10 beanie launch. The Lume South Wharf. Wednesday, May 8, 2024.

FightMND has invested more than $97 million into research and care initiatives thanks to the amazing generosity of the Australian public, partners and state and federal governments. But more is needed and so ‘we go again’ with Big Freeze 10.  

Every Beanie sold and donation made brings us one step towards a cure. So, team up for Big Freeze 10 and do your bit. Buy your Beanie today at Coles, Bunnings, selected Coles Express stores and online.

Eddie McGuire Big Freeze 10 beanie launch. Lume South Wharf. Wednesday, May 8, 2024.

Keep doing, keep fighting. When you wear your Beanie with pride, you’re stepping up and fighting for Neale and everyone who has or has had MND. Share your action across social media with the hashtags #DontSayDo #BigFreeze10 and tag us @fightmnd  

Big Freeze 10 beanie launch. Lume South Wharf. Wednesday, May 8, 2024.

A huge $1.2 million investment kicks off FightMND’s inaugural ‘Care Research Grants’

A huge $1.2 million investment kicks off FightMND’s inaugural ‘Care Research Grants’

In another innovative move to ‘beat the Beast’, FightMND will invest $1.2 million into five Motor Neurone Disease (MND) research projects focused on determining and improving best-practice care for people living with MND. 

It’s the next step in the fight for the organisation, and the Care Research Grants initiative will help inform guidelines around providing the most suitable care for people living with MND, as well as MND Care innovations aiming to improve quality of life and extend survival. 

Co-founded by AFL legend Neale Daniher AO in 2014, FightMND is now one of the world’s largest independent funders of MND research.

FightMND’s vision is a world without MND, but the road to a cure also requires a continued effort to improve MND Care in Australia and addressing key challenges faced by people living with MND, and their loved ones, during and after a diagnosis. 

FightMND Director of Research and Programs, Dr Bec Sheean says this commitment to pioneering investment in MND Care research will build the capacity and capability of the Australian Care research sector and serve as a beacon of hope for Australians living with MND. 

Everyone’s journey with MND is different and we want to ensure that every Australian living with MND gets the best support available when they need it most,” says Dr Sheean. 

With this investment, we’re matching our long-term commitment to finding a cure with a targeted focus on improving care through our support of these new projects from incredible teams of researchers. 

“We know that research is the best way to defeat the Beast, but it can also help to tame it. Improving the standards of care will mean that everyone who is diagnosed with MND now, and into the future, can experience the best possible quality of life while fighting this insidious disease,” says Dr Sheean.

The investment from this initiative brings FightMND’s total investment in MND Care to over $11.6 million since 2017.

The Care Research Grants initiative was open to researchers nationwide, with five projects identified as the top-ranked projects for support: 

  • Professor Samar Aoun: Perron Institute Research Chair in Palliative Care, University of Western Australia, WA  
  • Professor David Berlowitz: Clinician Researcher, Department of Physiotherapy at The University of Melbourne and Respiratory Physiotherapist, the Victorian Respiratory Support Service at Austin Health, VIC 
  • Dr Marnie Graco: Implementation Scientist, the Institute for Breathing and Sleep at Austin Health, VIC 
  • Dr Anne Hogden: Senior Lecturer, School of Population Health at UNSW, NSW 
  • Dr Nicole Sheers: Clinician Researcher, the Department of Physiotherapy at The University of Melbourne, and the Institute for Breathing and Sleep at Austin Health, VIC 

Professor Samar Aoun, who was awarded the 2023 WA Australian of the Year for her work in palliative care, aims to improve the quality of palliative and end-of-life care service delivery to MND patients by understanding and addressing gaps in current practice across various settings. 

“This national collaborative approach will bring together for the first time peak MND and palliative care organisations to work together… It will establish a collective of stakeholders that includes people living with MND and their families, and has the capacity to sustain and nurture future research, workforce education, training for informal carers and service development,” says Prof. Aoun. 

Professor David Berlowitz’s project aims to develop and apply artificial intelligence (AI) to the setup and optimisation of Non-Invasive Ventilation (NIV), a mechanical breathing support used by people living with MND, as people’s needs change. 

“We believe that this AI development work is unique internationally. We currently do not have NIV devices that can really keep up with the changing respiratory needs of people living with MND. This FightMND grant can help us change that. Good respiratory support does not make MND go away, but when done well, it absolutely helps people, and the families live better with MND,” says Prof. Berlowitz. 

For the last two years, Dr Marnie Graco has been leading crucial research on how to improve the uptake of Non-Invasive Ventilation (NIV) to improve quality of life and survival for people living with MND.

“My research has shown that access to NIV is inequitable across Australia, and I have identified many problems that could be addressed to improve NIV access. This new project, funded by FightMND, will allow us to work with the MND community to develop novel, targeted solutions to these problems,” says Dr Graco.  

Dr Anne Hogden’s project will co-design up-to-date, high quality Decision Support Tools (DSTs) to empower people living with MND to be able to make informed decisions for their care and quality of life. 

“The DSTs were developed with members of the MND community, and we will now improve the range to meet the current needs of people living with MND…Rather than just reading fact sheets, the DSTs assist people to work through the options at their own pace, weigh up the pros and cons, and arrive at a decision,” says Dr Hogden. 

Dr Nicole Sheers’ research looks at whether specially developed exercises can help people living with MND maintain their breathing muscle strength to breathe deeper and cough better. 

“Breathing muscle weakness, cough and swallow problems have a devastating impact on people affected by MND… This pilot study will tell us whether people living with MND can manage this novel, proactive exercise program, as well as provide some information about the benefits on breathing, cough and swallow function,” says Dr Sheers.

Funded Research Projects

Funded Research Projects

Thanks to the ongoing support of the Australian public and the FightMND Army,  FightMND has invested more than $55.9 million into MND research since 2014.

Together we have made real progress towards effective treatments and a cure since the Army answered the call seven years ago.

People living with MND now have more opportunity to participate in research or clinical trials throughout Australia. It is proof of the accelerating progress we are making to end MND.

From clinical trials to drug development projects, it is research like this that will help us find more effective treatments and ultimately a cure.

Below you will find a list of the clinical trials, drug development projects and impact grants we’ve invested in from 2016 – 2022

Clinical trials

Clinical trials test and evaluate promising new treatments to find better ways to prevent, detect or treat MND.

2022

Randomised double-blind placebo-controlled Phase 3 trial of Lithium Carbonate in MND, a sub-study of a Multi-arm, Adaptive, Groupsequential trial NETwork to evaluate drug efficacy in patients with MND (MAGNET).

Professor Matthew Kiernan
The University of Sydney, NSW

A placebo-controlled safety and efficacy of ambroxol in individuals with MND,

Associate Professor Bradley Turner
The Florey, The University of Melbourne, VIC

2021

PHASE 1B – EPHRIN RECEPTOR A4 FC (new drug)

Prof Perry Bartlett, AO

2020

PHASE 1

Dr Susan Mathers

IMPACT grants

To accelerate the development of effective therapies for MND, FightMND IMProving and ACcelerating Translation (IMPACT) grants support projects focused on overcoming one or more key barriers preventing the advancement of potential treatments through to clinical trial. Drug Development projects are focused on advancing promising new drugs or therapies through the final stages of testing in preparation for their assessment in clinical trials with MND patients.

2022

Enhanced neuronal delivery, gene targeting and neuroprotection: development of a multimodal drug against MND

Dr Loren Flynn, Murdoch University, WA

Targeted degradation of misfolded TDP-43 as a therapy for MND

Dr Luke McAlary, The University of Wollongong, NSW

Advanced modelling of upper motor neuron MND pathology using human pluripotent stem cells

Professor Clare Parish, The University of Melbourne, VIC

Evaluation of a novel inducible muscle specific TDP-43 mouse model of MND

Professor Aaron Russell, Deakin University, VIC

Therapeutic targeting of TDP-43 through selective reduction of ataxin-2 expression with peptide-conjugated antisense oligonucleotides

Dr Fazel Shabanpoor, The University of Melbourne, VIC

RNA-binding proteins involved in the pathogenesis and disease heterogeneity of sporadic MND

Dr Rachel Tan, The University of Sydney, NSW

Developing a validated C9orf72 mouse model of ALS/FTD using genome editing MND

Associate Professor Bradley Turner, The University of Melbourne, VIC

New viral-mediated TDP-43 mouse models of MND

Dr Adam Walker, The University of Queensland, QLD

Development of a human MND Neurovascular Unit model to improve therapeutic translation in drug testing

Associate Professor Anthony White, QIMR Berghofer Medical Research Institute, QLD

Profiling monocytes in MND to assess disease progression and heterogeneity

Professor Trent Woodruff, The University of Queensland, QLD

 

2021

Dr Allan McRae

Prof Julie Atkin

Prof David Wright

Dr Kelly Williams

Dr Fleur Garton

Prof P. Anthony Akkari

RESTORING AUTOREGULATION OF TDP43 IN SPORADIC MND USING SPLICE-SWITCHING ANTISENSE OLIGONUCLEOTIDES

Prof Lezanne Ooi

ENHANCING DELIVERY OF GENE THERAPY TO MOTOR NEURONS AND GLIAL CELLS USING FOCUSED ULTRASOUND

Dr Kara Vine

SUBPIAL SPINAL CORD DELIVERY AS A STEM CELL-BASED TREATMENT FOR MND

Prof Lachlan Thompson

HARNESSING PHASE SEPARATION AS A PRECLINICAL STRATEGY FOR THE TREATMENT OF MND

Dr Marco Morsch

2022 DRUG DEVELOPMENT PROJECTS

Drug Development projects are focused on advancing promising new drugs or therapies through the final stages of testing in preparation for their assessment in clinical trials with MND patients.

2022

Intramuscular allosteric agonism of purinergic P2X7 receptor as a pharmacological approach to enhance skeletal muscle regeneration in MND

Dr Giovanni Nardo
Mario Negri Institute for Pharmacological
Research, Italy

Validation of the clinical-stage drug candidate RRx-001 as a novel disease modifying therapeutic for MND.

Dr Tony Reid
EpicentRx, Inc., California, USA

2022 DISCOVERY PROJECTS

Epidemiology in a dish: using human iPSC to discover common and genotype specific molecular signatures of the multistep hypothesis of MND.

Associate Professor Anthony Cook, University of Tasmania, TAS

Trouble at the ribosome in C9ORF-72-driven MND

Dr Danny Hatters, The University of Melbourne, VIC

Multiomic interrogation of patient-derived neurotoxic glia

Dr Jeffrey Liddell, The University of Melbourne, VIC

2022 COLLABORATIVE INITIATIVES PROJECTS

Pre-familial and early MND biomarker program

Associate Professor Mary-Louise Rogers, Flinders University, SA

AMII: Asia-pacific MND Imaging Initiative

Dr Sicong Tu, The University of Sydney, NSW

2022 FELLOWSHIPS

The Bill Guest Mid-Career Research Fellowship is named in recognition of the extraordinary contribution of Bill Guest AM, the inaugural Chairman at FightMND.

PROJECT: Clearing TDP-43 pathology for MND therapy

Dr Adam Walker – Bill Guest Mid-Career Research Fellow, The University of Queensland, QLD

PROJECT: Reversing TDP-43 pathology and neuronal loss in sporadic MND

Dr Rachel Tan, The University of Sydney, NSW

PROJECT: Therapeutic targeting of ferroptotic cell death in MND

Dr Taide Wang, The University of Melbourne, VIC

Dr Taide Wang was also the inaugural recipient of the Angie Cunningham Scholarship.

The gene that put Australia on the map for MND research

The gene that put Australia on the map for MND research

Australia’s contribution to 30th anniversary of the discovery of the association of the SOD1 gene with MND. Written by Professor Anthony Akkari

In 1981 then Dr, now Emeritus Professor Nigel Laing, came to Western Australia to join the proposed Australian Neuromuscular Research Institute (ANRI) (now the Perron institute) to further his research into the neurobiology of motor neurones.  In the mid 1980’s, significant advances were being made in the field of genetics. In 1987, with the support of Professor Byron Kakulas, the founding Medical Director of the institute, Professor Laing travelled to Duke University in North Carolina to join the Division of Neurology to study molecular genetics and new techniques to better understand mechanisms in neurodegenerative disease. Professor Laing worked in the laboratory of Professor Teepu Siddique and with the Chief of Neurology at Duke University, the late Professor Allen Roses. Professor Roses was among the first academics to understand the power of molecular genetics in the early 1980’s [1]. Professor Roses and his team were also the first to uncover the association of the gene APOE4 with Alzheimer’s disease risk in 1993 [2]. It was with this esteemed group and Professor Siddique, who had embarked on investigating the genetics of MND, that Professor Laing trained in molecular genetics with among then the best in the field.  

In 1988 Professor Laing returned to Perth, WA and established the neurogenetics group at the ANRI, (now the Perron Institute) continuing to apply his training in molecular genetics to the study of MND, as well as to other neuromuscular diseases. Professor Laing afforded me the opportunity to join his group as a laboratory assistant in 1990 and within a short period of time I was completely captivated by the possibilities that molecular genetics methods could bring to understanding neuromuscular diseases. A few years later I enrolled as a PhD candidate in Prof Laing’s laboratory, focussing on the molecular genetics of the congenital myopathy: nemaline myopathy.  

SOD1 link to MND discovered. 

In the mid 1980’s Dr Laing with Dr Patricia Kailis, uncovered an extended Western and South Australian, multigenerational family with autosomal dominant MND – meaning that a person needs to inherit only one copy of the defective gene from one parent with the disorder to be at risk of the disease.  By 1992 Dr Laing in collaboration with Professor Teepu Siddique, was part of the international team mapping the first MND gene.  As a student I can remember Professor Laing, laying out the extensive pedigree of this family across the ANRI Board Room table; and explaining the uncertain future for family members facing the consequences of this devastating disease, hidden in their genes. “Here Tony you can see the son who passed away in his 40’s and you can see that his mother who also has MND is still alive in her 80s.” It was that particular conversation, on that day that engaged me as a student, curious and driven to research this inherited MND. Later that same year, my own father was diagnosed with MND; these two events turned me into both a neuromuscular disease researcher and carer for someone with MND. On the 4th of March 1993, Prof Laing was a member of the international team that published the discovery of the first MND gene, SOD1. The Perth family made a major contribution to the discovery, since at that time, linkage analysis was performed in a stepwise fashion, focusing on a region of the genome that was common only to people within that family that had MND. The larger and more extended multigenerational families provide a greater chance of identifying a candidate gene region linked to the inherited disorder. Importantly, within the candidate region lay the SOD1 gene. As the international research team began examining the SOD1 gene, they found the highest probability, known as a Log of the odds score or “LOD” score, occurred with the SOD1 gene. This meant that SOD1 was the most likely candidate for familial MND in the families investigated. The international team then performed subsequent studies on SOD1, looking for disease-causing mutations and found 11 different missense mutations (changes that alter one amino acid in the protein) in 13 different families from around the world, including the West Australian family. On the 4th of March 1993 the first paper describing mutations in a gene that causes MND was reported [3]. 

The discovery of the SOD1 gene as causative in MND unequivocally opened the field of MND genetics research and since this discovery, over 30 genes have been identified and are being tested on an ongoing basis for mutations in patients with clinical presentation of MND.  

Advancing novel therapeutics for neuromuscular diseases: The Perron Institute 

Professor Laing was working at the Australian Neuromuscular Research Institute (now the Perron Institute), undertaking molecular characterisations of neuromuscular disease genes. At the same institute, another team headed by Professors Steve Wilton and Sue Fletcher began working on a novel intervention, using a type of treatment called antisense oligonucleotides for the treatment of an untreatable disease called Duchenne Muscular Dystrophy (DMD). Almost 20 years later, in 2016 the first antisense therapeutic for DMD, developed by that very team, received accelerated approval from the FDA for the treatment of some individuals with that disease, providing a mutation-specific therapy for DMD. Since then, two additional therapeutics developed by Professor Fletcher and Wilton, to treat other DMD-causing mutation subgroups, were approved by the FDA.  

The SOD1 as a therapeutic target  

Upon completing my PhD and post-doctoral research with Prof Laing, I worked on neuromuscular and neurodegenerative diseases in the USA at Duke University with Professor Allen Roses and in the pharmaceutical industry, integrating genetics into drug development. I returned to Perth in 2017 and to my home, the Perron institute, to apply my industry skills to antisense oligonucleotide drug development with an urgency to improve outcomes for patients living with MND. 

Shortly after my return, I was privileged to meet Dr Loren Flynn, who trained in the laboratory of Professors Wilton and Fletcher and with over 14 years of experience in these novel antisense therapeutics. Dr Flynn agreed to join the newly established MND genetics and therapeutics group at the Perron Institute and lead the antisense development programs targeting MND disease mechanisms. Dr Flynn’s first interest was to develop a SOD1 targeting program using an antisense therapeutic approach, in collaboration with Professors Wilton, Fletcher and myself. Dr Flynn, with exceptional support from our colleague Professor Bradley Turner (Florey Neuroscience Institute) and Black Swan Pharmaceuticals, an MND antisense drug development company, has advanced this drug for SOD1 MND patients toward the clinic. This collaboration led to the achievement of our first major milestone in taking our therapeutic toward the clinic with a $1M Drug Development grant awarded by our partners FightMND in 2020. This funding has been instrumental in advancing the SOD1 therapeutic. With funding from FightMND as well as industry support, we are moving efficiently toward clinical trials, with additional antisense therapeutics to follow.  

The first MND-linked disease gene, SOD1 was discovered through research undertaken in Australia, in collaboration with Australian patients. Now, a promising drug is under development in Australia, by Australian researchers, funded by Australians.  

Researchers worldwide continue to make significant progress in understanding MND and the role of genetics in the disease. In April 2023, the FDA approved the first antisense therapy for MND, a SOD1 suppression drug called Tofersen. This is as exciting time for genetic therapies for MND as it paves the way for future discoveries and translation of other novel antisense therapies. We are, as a global community, collaborating, sharing, and learning from each other how to progress toward successful therapies for MND. Researchers from across Australia are working together, looking beyond the horizon, and imagining better ways to understand, diagnose, model and develop therapeutics for this dreadful disease.  

THANK YOU 

I would like to personally thank MND patients around the world and in Australia FightMND, The Giumelli Family, The Perron Institute and Murdoch University, MNDRIA, MSWA, Black Swan Pharmaceuticals, and our associated investor/philanthropists, racing for MNDi, as well as our collaborators in Australia; Professor Bradley Turner and the Florey Neuroscience Institute, and our international collaborators. We are very privileged to work with a committed community determined to advance disease-modifying therapeutics for MND.  

Professor Anthony Akkari is the Co-Lead of the Motor Neuron Disease Genetics and Therapeutics group at the Perron Institute for Neurological and Translational Science, and the Foundation Chair of Industrial Pharmacogenetics at Murdoch University. He is also the Chief Scientific Officer and a Co-Founder of Black Swan Pharmaceuticals, a pharmaceutical company developing antisense therapeutics for MND and Parkinson’s Disease.  

 References

  1. Snyder A: Allen RosesLancet 2016, 388(10057):2232.
  2. Strittmatter WJ, Weisgraber KH, Huang DY, DONG L-M, Salvesen GS, Pericak-Vance M, Schmechel D, Saunders AM, Goldgaber D, Roses AD: Binding of human apolipoprotein E to synthetic amyloid ,Bpeptide: Isoform-specific effects and implications for late-onset AlzheimerdiseaseProc Natl Acad 1993, 90:8098-8102. 
  3. Rosen R, Siddique T, Patterson D, Figlewicz D, Sapp P, Hentati A, Donaldson D, Goto J, O’Regan J, Deng HX et alMutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosisNature 1993, 362:59-62.

The Australian MND Registry: Providing Clinical Data to Fight the Beast

Motor Neurone Disease (MND) is a complex and variable disease, and this can cause challenges in both the lab and the clinic. To better understand the disease, in 2004 a team of clinicians established The Australian MND Registry (AMNDR), a database of clinical data from Australians with MND. One clinician involved in setting up AMNDR was Professor Paul Talman, a neurologist with a specific interest in MND, who talked to us about the registry’s beginnings and how it has changed the MND field.

“The registry began in the late 1990s when fellow neurologist, Associate Professor Susan Mathers and I established a Victorian MND registry based at Calvary Health Care Bethlehem. At that time, we recognised patients presented with different patterns of MND that had very different rates of change and outcomes, and we thought this was important to define as it may help with understanding the disease.”

It was in 2003 when pharmaceutical company Aventis approached Paul at the suggestion of Professor Sir Edward Byrne, a distinguished Australian neuroscientist and founding Director of the Melbourne Neuromuscular Research Unit. The company licenses the only approved therapy for MND in Australia, riluzole. “Aventis wanted to provide an educational grant to enhance the Victorian registry and expand it nationally,” said Paul. Knowing that the registry’s success would depend on the engagement of neurologists and clinics nationwide, Paul and Susan brought together neurologists with a specific interest in MND from around Australia.

Together, health professionals at participating clinics contributed to AMNDR by recording data from people living with MND who volunteered to take part in the registry. Data was collected on disease milestones, such as the time of symptom onset and symptom progression, to assist with the staging of a patient’s disease. “[The clinics] also collected information on health service utilisation, the diagnostic workup required, and treatments to assist patients with MND.”

What started off as paper-based data entry matured into a web-based platform. “The information was collected by clinic health professionals at each of the state-based MND clinics promoted by MND Australia and the state MND Associations. Data collection and entry was performed as a voluntary contribution by the clinics and is a credit to the health professionals’ dedication in trying to help understand this disease.”

Their main goal was to record how MND evolves in patients within Australia. With the large scale of data collected via the registry, it was hoped that clinicians and researchers would have information available to inform research and clinical care. There were also hopes to assist clinical trial design to increase the potential of successful trial results: “We believed from this beginning [with AMNDR], we would be able to further develop and enhance clinical trials by considering the different disease trajectories that could be defined by data analysis.”

Initially, the data were used to categorise patients based on their symptoms, disease onset, progression, and survival. This helped clinicians when diagnosing new patients with MND, and in identifying the best potential treatment options for each patient.

“The data has also been used by the individual clinics to look at the milestones of their own patient cohort and delivery of care,” Paul said. By looking at disease milestones—for example, when a patient receives non-invasive ventilation (NIV) or a gastrostomy (feeding tube)—clinics have been able to evaluate the care they provide against the best-known standards and continuously work to improve patient outcomes.

“AMNDR has confirmed that forming teams focused on data will enhance care while providing data to external researchers wishing to understand the impact different treatments may have,” Paul said. He notes the work of Professor David Berlowitz (University of Melbourne & Victorian Respiratory Support Service, Austin Hospital) on the use of NIV as one example of how the data and external researchers have helped to transform care for people living with MND. Using data from AMNDR, Professor Berlowitz and his team compared the survival of people with MND who received NIV with those who didn’t, finding that NIV prolongs survival by 13 months. When they compared the data between different forms of MND, patients with ALS-bulbar onset benefited from NIV the most. As a result of these findings, NIV is now offered to most MND patients during their disease.

Many researchers and research initiatives have collaborated with AMNDR, using the clinical data in AMNDR for projects such as the Sporadic ALS Australia-Sporadic Genomics Consortium (SALSA-SGC, with Prof Naomi Wray), induced pluripotent stem cell (iPSC) program at the Florey Neuroscience Institute (with Prof Brad Turner), and the Victorian Brain Bank (with Prof Catriona McClean).

In 2021, a new registry platform called MiNDAUS (www.mindaus.org) was developed, following a successful NHMRC Partnership grant application, and it now replaces AMNDR. MiNDAUS collects the same registration and type of data, along with the ability for patients to self-register and maintain their own health information which they can consent to share with other health providers and researchers. The MiNDAUS Registry is the new platform linking people living with MND, clinicians and researchers – with the ability to collect the data to answer questions on care, treatments and policy – informed by the MND Community itself.

While data entry into AMNDR has now closed, the registry remains a useful resource for MND researchers and clinicians. “The AMNDR legacy dataset provides information on the natural history of MND and can be used into the future to assess the potential benefits of emerging therapies,” said Paul. “It also provides a dataset to validate new disease staging metrics used to measure disease progression, which will help determine whether emerging therapies are having a beneficial effect.”

Similar to research, the registry depended on funding, which included $150,000 from FightMND over three years. “Throughout its lifetime, AMNDR was funded entirely philanthropically by Sanofi-Aventis, MND Australia, the Pratt Foundation, Wesfarmers, and the Fox Family,” Paul said. “The registry survived on around $650,000 for over 15 years, and while this provided enough money to run the registry, it limited our ability with respect to outputs and the technological development of the registry.”

Despite funding limitations, AMNDR has made a significant impact for clinicians, researchers and, most importantly, people living with MND. Now nearly 20 years on since its national expansion, the registry has helped to change the face of MND research and care in Australia—and it is all thanks to the patients who participated in the registry, and the dedication of the MND clinics, the clinicians involved, external researchers and collaborators.

For Paul, AMNDR’s greatest impacts rest amongst the team it helped to bring together in 2003, and the patients and carers that contributed to the registry: “The greatest outcome has been the network of clinicians that grew from AMNDR, and the sustained development and enhancement of clinics focused on patients with MND and their care within Australia. The generosity of patients and carers in donating precious time has been the cornerstone for the longevity of AMNDR.”

If you or someone you know has MND, and you would like more information on the MiNDAUS registry or would like to register, visit https://www.mindaus.org/