2016 Research

FightMND Funded Projects

In 2016 FightMND committed $1.75million dollars to funding the best and brightest MND researchers across Australia through the Motor Neurone Disease Research Institute of Australia’s scientific grant review process. The wide range of projects that we have funded highlights the depth of knowledge and skills that MND researchers in this country possess. Through this work we have made a significant impact in the fight against Motor Neurone Disease.  

$250,000 Grant-In-Aid Recipients

Dr Bradley Turner 

Florey Institute, VIC

A synergistic approach for treatment of MND using neurotrophic & gene therapy.

MND is a complex and multifactorial disorder that is likely to require multiple agents with synergistic effects targeting different aspects of disease for effective treatment. This novel international collaborative project will examine the efficacy of a combined gene and neurotrophic therapy approach to improve motor neuron health and connections to muscle in preclinical models of MND. Importantly, these gene and neurotrophic therapy agents can be rapidly adapted to human studies which will accelerate clinical development of this approach if supported for MND.

Prof. Gilles Guillemin 

Macquarie University, NSW

New directions for early diagnosis of MND: a large-scale longitudinal analysis of multiple biomarkers 

This project aims to assess the diagnostic potential of combining markers already identified by Australian and international research groups together with a large number of inflammatory molecules (in total 95 molecules out of 1 ml of serum) as biomarkers for ALS. This will help us develop better diagnostics as well as a more accurate understanding of the onset, triggers, environmental factors and progression of ALS. Moreover, we expect that identifying robust and sensitive sets of biomarkers may provide new hints for novel therapeutic strategies, or at least new directions for research on possible therapies.

Prof. Julian Gold 

The Albion Centre, NSW

Pilot trial of antiretroviral therapy for amyotrophic lateral sclerosis – “The Lighthouse Project”

This is a pilot clinical trial, The Lighthouse Project, to test the possibility that a virus may be the cause or trigger for motor neurone disease. The type of virus involved is actually part of our genes and therefore the therapy needs to be very specific. We will be testing an anti-retroviral drug in 30-40 patients with relatively early ALS and treating them for at least six months. There will be centres in Sydney and Melbourne and also in the U.K. This trial is an important step in trying to find the cause of MND and to contribute to our scientific understanding of this disease.

$100,000 Grant-In-Aid Recipients

Prof. Roger Chung

Macquarie University, NSW

New approaches to plasma biomarker studies in MND/ALS.

There is an urgent need to identify a series of biomarkers that can be used to improve the speed of diagnosis, and predict more accurately prognosis and other clinical parameters in ALS. This project will utilize a new proteomic technology to identify potential protein biomarkers in blood samples from ALS patients. We predict that these biomarkers may be useful in future for improving diagnostic and prognostic clinical evaluations. These protein biomarkers may also identify novel biological processes associated with disease pathogenesis, and this may lead to new insight into the causes of ALS.

Dr Nicholas Cole 

Macquarie University, NSW

Elucidating the pathogenesis of sporadic motor neurone disease using zebrafish models.

A complete understanding of the exact cause and a cure for the motor neuron disease/Amyotrophic Lateral Sclerosis (ALS) remain elusive. The majority of ALS cases (over 90%) occur sporadically with no known genetic cause or family history. Our aim is to create an animal model that develops genuine ALS-like symptoms so that we can study these animals in the laboratory to understand the biology of the human disease and ultimately find a cure. This will be done in zebrafish, a well-established laboratory research animal for human disease research that are also suitable for the rapid testing of potential new treatments.

A/Prof. Julie Atkin

Macquarie University, NSW

Studying DNA damage and neurodegeneration in ALS/MND.

Our genes are under constant attack. Hence cells have developed systems to detect/repair DNA damage, termed the ‘DNA damage response’. However, if DNA cannot be repaired correctly following damage, permanent changes to our genetic information result that lead to motor neuron death. We have identified that damage to DNA is present in cells carrying mutations that cause most genetic forms of motor neuron disease (MND). This project will examine the detailed mechanisms by which this occurs. It will examine whether drugs that inhibit the DNA damage response are protective in cellular models of MND.

A/Prof. Guillaume Lessene

Walter & Eliza Hall IMR, VIC

Novel agents to prevent neuronal apoptosis in motor neuron disease.

MND is characterised by “apoptosis”, a cellular process leading to the death of neurons under physiological stress. Recent advances in understanding how apoptosis works have allowed the development of new drugs that stops neuronal cell death and the progression of MND. Using our expertise in apoptosis and drug development, we identified the first pharmacological inhibitors of apoptosis. Extending from these studies, we hope to develop new drugs that will provide the basis for clinical trials in MND patients. Our goal is to slow or stop the progression of MND by harnessing the therapeutic potential of apoptosis inhibition.”

Dr Marco Morsch 

Macquarie University, NSW

Does the transfer of ALS protein aggregates between motor neurons trigger neurodegeneration?

The accumulation of proteins in neurons and glia is a pathological hallmark of many neurodegenerative diseases. Clinical data from MND patients describes a focal onset and the subsequent spread of muscle paralysis to other regions. This project aims to understand how ALS proteins can transfer from one cell to the next. Our team has established a zebrafish model where we can track the release and spread of ALS-proteins. The outcome of this study will be the first visualisation of ALS-protein transfer in a living animal, and provide greater understanding of whether this represents a potential pathogenic mechanism in ALS

A/Prof. Peter Noakes

University of Queensland, QLD

Exploiting the opposing actions of complement receptors in the treatment of MND.

One major part of the immune system is the complement cascade of which C3 and C5 are the key proteins. C3 is broken down into C3a and C3b, and likewise C5 is broken down to C5a and C5b. C3a and C5a activate their receptors C3aR and C5aR1 to produce opposing actions. In the nervous system, C3aR activation can protect nerve cells from death, while C5aR1 activation can drive nerve cell death. We will exploit these two opposing actions of C3aR and C5aR1 to enhance the neuro-protective actions of C3aR within the brain-spinal cord, while at the same time block with our drug the neurodestructive actions of C5aR1, in animal models of MND.

 

A/Prof. Kenneth Rodgers

University of Technology, NSW

Identification of environmental risk factors for sporadic MND in Australia.

Toxins such as heavy metals, pesticides, fertilisers and exposure to algal blooms have been implicated as causes of sporadic MND (sMND). Long-term exposure to neurotoxins can give rise to clusters or ‘hot-spots’ of sMND. We will map sMND patients in NSW to identify any geographical clusters of sMND. We will then look for neurotoxins in plasma, hair and in the local environment to identify which toxins increase the risk of developing sMND. Limiting exposure to environmental neurotoxins could result in a gradual decline in the incidence of sMND and protect future generations of Australians.

Dr Mary-Louise Rogers 

Flinders University, SA

Development of novel immunogenes to improve growth factor support for motor neurons.

Lack of growth factor support is one reason motor neurons die in MND. Animal studies have demonstrated the potential of glial cell-derived neurotrophic factor (GDNF), and hepatocyte growth factor (HGF) for MND. We have developed a technology called immunogenes that enable delivery of therapeutic genes into the nerves affected in MND from the circulation. This project will test immunogenes in newborn mice to determine dosing for delivery of GDNF and HGF for future treatments of mice living with MND. Data from this project will answer questions about efficacy of neurotrophic therapy with immunogenes for people living with MND.

Dr Lachlan Thompson 

Florey Institute, VIC

Towards cell-based therapies for Motor Neurone Disease (MND).

Our laboratory is interested in the idea that stem cells can be used to grow new neurons that can functionally replace those lost in neurodegenerative conditions such as MND. We have all been hearing for a long time now about the promise of stem cells as a treatment option for patients with brain and spinal cord injury. So why aren’t we there yet? This project is aimed at addressing the practical challenges in translating stem cell therapies to the clinic, particularly the need for immune suppression that allows for survival of the stem cell grafts but without accelerating disease progression.

Dr Adam Walker 

Macquarie University, NSW

Pre-clinical therapeutic testing and biochemical changes in the TDP-43 model of MND.

A major hurdle in understanding what causes MND and in developing treatments has been the lack of reliable MND animal models. Recently at the University of Pennsylvania, Dr Walker characterised new genetically modified mice that for the first time develop brain and spinal cord pathology along with progressive movement deficits similar to >97% of MND patients. This project will establish these mice in Australia for pre-clinical testing of potential MND therapies, and will investigate the biochemical changes that cause neurodegeneration in the early stages of MND with the aim of identifying new therapeutic targets.


 

FightMND is the largest independent financial supporter of MND research and clinical trials in Australia

We fund the best and brightest MND researchers across Australia to help develop promising new therapies in the lab to the roll into clinical trials for Australians living with MND.

No. of Australian Research Grants Awarded

No. Of Australian Clinical Trials Funded

$million Spent on Funding Research Grants

$Million Spent on Funding Clinical Trials

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