MND research grants

The MND Research Institute of Australia has awarded $3.75 million to support research in 2017. Two new grants have been awarded this year in addition to two 3-year fellowships and 29 grants-in-aid: the Betty Laidlaw Prize for an outstanding mid-career researcher and the Charcot Grant, to be awarded annually for the highest ranking grant-in-aid.

Projects commencing in 2017

BETTY LAIDLAW PRIZE 2017  
Dr Catherine Blizzard
Menzies Research Institute University of Tasmania
Betty Laidlaw Prize 
awarded to an outstanding
mid-career researcher
TDP-43 misprocessing drives synaptic deficits and ALS
POSTDOCTORAL FELLOWSHIPS (2017-2019)  
Dr Emma Devenney
Brain and Mind Centre,
University of Sydney
Beryl Bayley MND Postdoctoral Fellowship 2017 - 2019 Behaviour, cognition, eye-movements and psychiatric disease in C9orf72 MND and FTD; a cross modal-approach to facilitate early and accurate diagnosis
Jennifer Fifita
Macquarie University, NSW
Bill Gole MND Postdoctoral Fellowship 2017 - 2019 Investigating the molecular and pathological origins of amyotrophic lateral sclerosis
     
GRANTS-IN-AID    
Dr Shyuan Ngo
School of Biomedical Sciences, University of QLD
Charcot Grant
awarded to the highest ranking grant-in-aid
Metabolic exploration in neurodegenerative disease (MEND): synergy between derangements in systemic and muscle metabolism in MND
A/Prof Julie Atkin
Macquarie University, NSW
MNDRIA grant-in-aid Telomere dysfunction in ALS/MND
A/Prof David Berlowitz
Institute for Breathing and Sleep, VIC
Mavis Gallienne MND Victoria Research Grant Lung Volume Recruitment in Neuromuscular Disease
Dr Karin Borges
School of Biomedical Sciences, University of QLD
MNDRIA grant-in-aid Triheptanoin to improve energy metabolism in MND
Dr Christopher Bye
Florey Institute of Neuroscience and Mental Health, VIC
Mick Rodger MND Research Grant  Understanding disease susceptibility in idiopathic MND
Ashley Crook
Macquarie University, NSW
Graham Lang Memorial MND Research Grant  Preventing motor neurone disease: barriers, facilitators, costs and benefits of genetic testing for MND in Australia
Dr Peter Crouch
University of Melbourne
MNDRIA grant-in-aid Mitochondrial TDP-43
Prof Victoria Flood
University of Sydney and Westmead Hospital
Jenny Simko MND Research Grant The effects of active exercise combined with an enriched diet on swallowing, speech function and weight in patients with MND; a randomised trial
Dr Fleur Garton
Institute of Molecular Bioscience, University of QLD
MNDRIA grant-in-aid Cell-free DNA and ALS; insight into disease mechanisms and progression
Dr Nimeshan Geevasinga
University of Sydney and Westmead Hospital
Dr Paul Brock MND NSW Research Grant Functional and structural connectivity in ALS
Dr Jean Giacomotto
Queensland Brain Institute University of QLD
MNDRIA grant-in-aid New and innovative polygenic approach for understanding and modelling MNDs in zebrafish
Prof Andrew Hill
La Trobe University, VIC
MNDRIA grant-in-aid Harnessing the power of exosomes to understand MND
A/Prof Mark Howard
Institute for Breathing and Sleep, VIC
Jenny Simko MND Research Grant Translation of respiratory biomarkers into MND practice
Dr Anna King
University of Tasmania
Alma Grace Holt MND Research Grant  Staying connected: determining targets to protect neuronal circuitry in ALS
Dr Albert Lee
Macquarie University, NSW

MNDRIA Grant-in-aid
Investigating the regulatory roles of Cyclin F phosphorylation in the development and prevention of ALS
Dr Jacqueline Leung
University of Tasmania
MNDRIA Grant-in-aid Identifying the role of oligodendrocytes in disease onset and progression in ALS
Dr Sean Millard
School of Biomedical Sciences, University of QLD
MNDRIA grant-in-aid Functional analysis of ALS candidate genes
A/Prof Greg Neely
Charles Perkins Centre
University of Sydney
MNDRIA grant-in-aid Functional genomic dissection of motor neuron disease
A/Prof Peter Noakes
School of Biomedical Sciences, University of QLD
MNDRIA grant-in-aid Targeting neuromuscular stability in motor neuron disease.
Prof David Pow
University of QLD Centre for Clinical Research
MND & Me Foundation Research Grant  Discovery of EAAT5 – a protein that may stop glutamate excitotoxicity in ALS
Dr Mary-Louise Rogers
Flinders University, SA
MNDRIA grant-in-aid CSF and serum p75 extracellular domain as validation of a fluid biomarker for MND
Dr Fazel Shabanpoor
Florey Institute of Neuroscience and Mental Health, VIC
Peter Stearne Familial MND Research Grant Alleviation of C9orf72-mediated ALS: A novel bi-functional peptide- oligonucleotide strategy both suppressing gene expression and activating autophagic clearance of toxic protein
A/Prof Ronald Sluyter
School of Biological Sciences
University of Wollongong
Stanford Family MND Research Grant Establishing the therapeutic potential of the P2X7 receptor ion channel in ALS
Dr Rachel Tan
Brain and Mind Centre
University of Sydney
MonSTaR Foundation MND Research Grant Is ATXN2 a potential therapeutic target in MND?
Dr Bradley Turner
Florey Institute of Neuroscience and Mental Health, VIC
Benalla Act to d'feet MND Research Grant Profiling the gene expression pathways of motor neuron vulnerability early in development of MND
Dr Mehdi Van den Bos
University of Sydney and Westmead Hospital
Stanford Family MND Research Grant Pathophysiological mechanisms underlying ALS: insights from novel cortical functional techniques
Dr Adam Walker
Macquarie University, NSW
MonSTaR Foundation MND Research Grant New proteins and pathways contributing to TDP-43-mediated neurodegeneration
A/Prof Trent Woodruff
School of Biomedical Sciences, University of QLD
Fat Rabbit MND Research Grant Therapeutic inhibition of HMGB1 to slow disease progression in MND
Prof Naomi Wray
Institute for Molecular Biosciences, University of QLD
MNDRIA grant-in-aid GWAS data for SALSA-SGC
NHMRC/MNDRIA Postgraduate Scholarship (2017 - 2019)  
Camille Paynter
University of Melbourne
NHMRC/MNDRIA Postgraduate Scholarship Communication, quality of life and advance care planning for people with MND and their caregiver


Betty Laidlaw MND Prize

Dr Catherine Blizzard
Menzies Institute for Medical Research, University of Tasmania

TDP-43 misprocessing drives synaptic defecits and ALS

Both inherited and sporadic ALS is defined by characteristic pathology of the protein TDP-43. Our research has identified a novel mechanism for how a disease associated mutation to this protein is causing degeneration of the nerve cells of the brain. We propose that the mutant protein causes a very early breakdown in communication between nerve cells. Importantly these changes occur before obvious symptoms, potentially giving significant insight into how the disease may start. We will investigate how defective function of TDP-43 alters nerve cell communication and how these changes lead to nerve cell death that charaterises ALS.
 

Postdoctoral Fellowships (2017 - 2019)

Dr Emma Devenney
Brain and Mind Centre, University of Sydney
 
Behaviour, cognition, eye-movements and psychiatric disease in C9orf72 MND and FTD; a cross modal-approach to facilitate early and accurate diagnosis

Firstly this project will measure eye movements in patients with MND, FTD, and the C9orf72 expansion, and also asymptomatic carriers of the expansion. This will identify the exact nature of eye-movement abnormalities in these conditions and determine whether they are a feature of presymptomatic disease. Secondly, this study will address the issue of psychiatric symptoms in the same disease cohort by determining the underlying causes of these symptoms, which will inform future management strategies.

Jennifer Fifita
Macquarie University

Investigating the molecular and pathological origins of amyotrophic lateral sclerosis
 
This project aims to use next-generation sequencing technologies to identify gene mutations that cause ALS, or may increase the risk of developing ALS, in patients with both familial and sporadic ALS. To understand how these new mutations cause disease, each will be studied in neuronal cell culture models, as well as in a zebrafish animal model. The identification of new ALS genes adds to DNA diagnostic testing, and provides a greater understanding of the underlying cause of ALS. New genes can be used to develop new cell and animal models, which will greatly assist in the testing and development of novel treatments for this devastating disease.
 

Grants-in-aid

Dr Shyuan Ngo
The University of Queensland

Metabolic exploration in neurodegenerative disease (MEND): synergy between derangements in systemic and muscle metabolism in MND
 
Our current study into energy needs in MND has shown that people living with MND use more energy than expected. We also have exciting new data that shows that skeletal muscle of MND patients becomes energy hungry, and that this might cause the overall increase in energy needs in people living with MND. We now aim to further investigate the relationship between the overall energy needs of MND patients and the energy needs of their skeletal muscle. Insights gained from this study will help us develop therapies that are personalised to match the precise energy needs of individual MND patients.
  
A/Prof Julie Atkin
Macquarie University

Telomere Dysfunction in ALS/MND 
 
The risk of developing MND increases with age, but the reasons why are unclear. Most genetic forms of MND are caused by mutations in a protein called C9ORF72, and we have evidence that this damages DNA and binds to telomeres. Telomeres protect the ends of our chromosomes and they shorten with age. DNA cannot be replaced so damage to chromosomes can have serious consequences for the cell. Hence, in MND, loss or shortening of telomeres could lead to motor neurone death. We aim to characterise the telomeres in C9ORF72-MND, and determine whether telomere dysfunction triggers degeneration/death of motor neurons.
 
A/Prof David Berlowitz
Institute for Breathing and Sleep

Lung Volume Recruitment in Neuromuscular Disease
 
As MND progresses, the inability to breathe deeply or cough effectively becomes more distressing. Breathing complications, chest infections and respiratory failure are debilitating and contribute to early death in people living with MND. “Breath-stacking” is a simple, inexpensive therapy that helps people take deep breaths. Doing this daily may stretch the chest wall, improve lung capacity, cough strength and slow the decline in breathing. This research trial is the first in the world to test whether performing these exercises regularly improves breathing function, symptoms, cough and quality of life for people living with MND over three months.
 
Dr Karin Borges
The University of Queensland

Triheptanoin to improve energy metabolism in MND
 
Recent research indicates that problems in energy metabolism contribute to disease progression in MND. Thus alternative fuel sources are a promising approach to treat MND. Triheptanoin, the triglyceride of heptanoate (a C7 fatty acid), has already been used safely for 15 years in other disorders for energy metabolism and neuromuscular disorders. In an MND mouse model it protects against motor neurone death and delays the onset of motor symptoms. We will generate more data to show that triheptanoin targets metabolic impairments that contribute to MND. These new data will be crucial to initiate large clinical trials of triheptanoin in MND.
 
Dr Christopher Bye
The Florey Institute of Neuroscience

Understanding Disease Susceptibility in Idiopathic MND

Sporadic MND causes 90% of disease in patients, yet we have an extremely limited understanding of why these patients become sick, in part due to our inability to model sporadic forms of the disease. The advent of iPS stem cell technology now allows us to take cells from patients, and use them to generate genetically identical motor neurons in the laboratory. In this proposal we are using iPS cells from sporadic MND patients in a newly developed long term disease model to understand why neurons from these patients are susceptible to disease, and potentially develop new treatments.

Ashley Crook
Macquarie University
 
Preventing motor neurone disease: barriers, facilitators, costs and benefits of genetic testing for MND in Australia 
 
Preventing and reducing incidence of MND is only currently possible in families with a known faulty MND gene, through access to reproductive options that prevent passing on the faulty gene to future children. Little is known about how individuals from these families decide whether to have genetic counselling, have genetic testing and undergo reproductive options. We will explore what factors influence these decisions, and assess the cost-effectiveness of different genetic testing options in Australia. We will use this information to create evidencebased guidelines for MND clinics and clinicians on genetic counselling and associated genetic testing options for familial MND.
 
Dr Peter Crouch
The University of Melbourne

Mitochondrial TDP-43 
 
A protein known as TDP-43 is involved in the development of MND but the biological mechanisms through which it does so remain unclear. Over the past few years we have investigated if/how TDP-43 affects mitochondria – the energy-producing components that exist within all cells. We have established that TDP-43 interacts with mitochondria and, recently, an independent international team has shown that MND-causing changes to TDP-43 affect that way in which it interacts with mitochondria. The focus of our current work is to better understand the relationship between TDP-43 and mitochondria and how this relationship may contribute to the development of MND.
 
Prof Victoria Flood
The University of Sydney and Westmead Hospital, Western Sydney Local Health District

The effects of active exercise combined with an enriched diet on swallowing, speech function and weight in patients with MND; a randomised trial

Eating, drinking and speaking are an important part of our life and patients with MND experience a rapid decline in these functions. Research suggests that active exercise might prolong the ability to eat and drink safely and prolong the ability to speak, however no thorough research has been conducted. Other research suggests that changes in diet might slow the progression of MND, with improved maintenance of weight status and muscle function. This study will be the first to evaluate the effects of these diet changes in patients with MND combined with active exercising on swallowing function, speech and weight status.
 
Dr Fleur Garton
The University of Queensland
 
Cell-free DNA and ALS; insight into disease mechanisms and progression 
 
The last five years have seen rapid discovery in identifying the genes that cause ALS. Parallel investigations that improve diagnosis and patient monitoring would benefit from an objective blood-based marker of disease. In this proposal, we hypothesise that levels of cell death may increase with ALS and correlate with disease progression. We will measure cell death in ALS patients, healthy individuals, and an ALS mouse by investigating levels of circulating cell-free DNA (cfDNA). Using a cfDNA test as a biomarker for ALS presents a significant opportunity for early detection of motor neuron death and hence, rapid improvements in diagnosis.
 
Dr Nimeshan Geevasinga
University of Sydney

Functional and structural connectivity in ALS 
 
MND is a progressive disorder with the underlying etiology still undetermined. Recent work has suggested cortical dysfunction, with MND and frontotemporal dementia thought to represent a disease continuum. We propose on utilising novel MRI techniques to explore changes in brain connectivity in ALS patients. Whilst there is some data on the MRI structural changes, an evolving area of neuroimaging is exploring a concept of ‘Connectomics’, looking at comprehensive maps of the human brain, then identifying which areas have increased/reduced connections in ALS patients, when compared to healthy controls. Furthermore these changes will then be correlated with novel neurophysiological biomarkers and clinical demographic scales and scores.
 
Dr Jean Giacomotto
The University of Queensland

New and innovative polygenic approach for understanding and modelling MNDs in zebrafish 
 
ALS is a complex disease, lacking appropriate treatment. Although its cause appears to be multifactorial, there are strong evidences that genetics plays a role in some patients. Therefore, we believe that we can use genetics to understand this disorder and find therapeutics. Different forms of ALS exist, but they all share a common hallmark: motor neuron degeneration. We are using an innovative genetic approach in zebrafish to elucidate the individual and synergistic pathogenic role of risk-genes with the ultimate goal of generating an animal presenting motor neuron degeneration, paving the way to find therapeutics for all forms of the disease.
 
Prof Andrew Hill
La Trobe University
 
Harnessing the power of exosomes to understand MND

The presence of aggregates of protein in the brain is characteristic of MND; however, the process by which they spread throughout the brain has eluded the MND field despite concerted efforts. We recently developed an innovative new methodology that is capable of studying protein spread in the human brain. We aim to implement this methodology in MND to discover how proteins spread from cell to cell in the diseased human brain.
 
A/Prof Mark Howard
Institute for Breathing and Sleep

Translation of respiratory biomarkers into MND practice  
 
The inability to breathe is unfortunately the most common cause of death in people living with motor neurone disease. Our research group reported that breathing assistance using noninvasive ventilation (NIV) increases survival by 13 months. Importantly, respiratory muscle strength appeared to identify the best time to start NIV. We propose to use 25 years of Australian Motor Neurone Disease Register data to confirm our preliminary findings. These experiments will enable clinicians to advise individual people with MND about when breathing assistance should be started to maximise benefit and assist researchers to optimise the design of therapeutic trials in MND.
 
Dr Anna King
Wicking Dementia Research and Education Centre

Staying connected: determining targets to protect neuronal circuitry in ALS 
 
Nerve cells are specialized cells which communicate with each other along long axons. MND is characterized by the nerve cells losing the ability to communicate with each other in a process called axon degeneration. Axon degeneration occurs in the motor nerve cells in the brain as well as those that control the muscles. The goal of our work it to determine the mechanisms by which axons degenerate in MND in order to find therapeutic targets to stabilise and maintain the function of these axons and their connections.
 
Dr Albert Lee
Macquarie University

Investigating the regulatory roles of Cyclin F phosphorylation in the development and prevention of amyotrophic lateral sclerosis 

Our team recently identified mutations in a new ALS/FTD gene that encodes the protein Cyclin F. It is involved in maintaining cellular health by tagging unwanted proteins (ubiquitylation) for breakdown and recycling within the cell. Mutant versions of Cyclin F, found in ALS patients, are defective in that they lack the necessary features (addition of a phosphate molecule) needed to regulate proper function, which ultimately leads to increased ubiquitylation and accumulation of proteins. This project will investigate new mechanisms of regulating Cyclin F activity that will contribute to our understanding of reducing abnormal accumulation of proteins inside motor neurons.
 
Dr Jacqueline Leung
Wicking Dementia Research and Education Centre

Identifying the role of oligodendrocytes in disease onset and progression in amyotrohpic lateral sclerosis  
 
In this project we will determine the role of a cell type called the oligodendrocyte in ALS. These cells produce myelin, a fatty layer surrounding the neuron processes that facilitates rapid signal transduction and provide structural support. Although oligodendrocytes appear to be affected in ALS with the presence of insoluble TDP-ˇ43 protein, in a similar way to the nerve cells, their role in ALS is unclear. In this project we will develop a mouse model with TDP-ˇ43 pathology in oligodendrocytes to determine its effects on nerve cells. This will provide evidence supporting oligodendrocytes as a potential therapeutic target in ALS.
 
Dr Sean Millard
The University of Queensland

Functional analysis of ALS candidate genes 
 
To effectively treat ALS, the functions of the genes involved need to be determined so that biologically relevant therapies that modulate progression of the disease can be identified. This grant will assess whether genes associated with ALS in human genome-wide association studies (GWAS) function at the neuromuscular junction (NMJ) of Drosophila melanogaster. The majority of the genes for ALS identified to date are conserved in fruit flies making this an ideal platform for assessing what these genes do. These studies will enable us to assign functions to several new ALS candidate genes, laying a foundation for future therapeutic studies.
 
A/Prof Greg Neely
University of Sydney

Functional genomic dissection of motor neuron disease
 
There are currently there are no effective therapies for treating motor neurone disease and potential drug targets are desperately needed. Recent human genetics efforts to identify MND disease genes have been difficult, suggesting complimentary approaches may be useful. Since MND involves defects in synaptic function in motor neurons, here we will define the core synaptic machinery required for proper motor neuron function and identify synaptic targets that can suppress development of MND in vivo. These efforts will illuminate new conserved regulators of motor neuron function, information that can then be used to develop novel therapies for this devastating illness.
 
A/Prof Peter Noakes
The University of Queensland

Targeting neuromuscular stability in motor neuron disease

Motor neurones attach to muscle cells in the body and make muscles work. Muscle weakness occurs in MND when the end of the motor neurone moves away from the muscle cell. We believe that there is a breakdown in the communication between the motor neuron and the muscle causing the neuron to withdraw. We will take muscle cells from patients with MND, place them in culture, and test them to see if they respond to chemicals normally released from motor neurons which make muscles work, and look at ways to stabilize the connection between them to improve muscle strength.
 
Prof David Pow
The University of Queensland

Discovery of EAAT5 – a protein that may stop glutamate excitotoxicity in ALS

The cause of ALS is unlcear; however, much evidence suggests that toxicity from a chemical called glutamate plays a key role. Indeed, the only therapy that improves survival in ALS acts through modulating glutamate. Therefore, finding additional mechanisms to prevent glutamate toxicity is an attractive target for the treatment of ALS. This project has a new approach to this. We have identified a new protein (EAAT5) that turns off glutamate release. It also acts as a transporter to mop up glutamate, terminating the excitatory effects. Preliminary studies showed that EAAT5 is reduced in the spinal cord of an animal ALS model. This loss of EAAT5 would cause a rise in glutamate levels and cause death of motor neurons. This study will define the anatomical and cellular distribution of EAAT5 in normal and diseased human nervous tissues, and may help identify potentially new therapeutic strategies to treat ALS.
 
Dr Mary-Louise Rogers
Flinders University

CSF and serum p75 extracellular domain as validation of a fluid biomarker for MND 
 
A lack of biomarkers that can measure the effect of treatment has been identified as one of the reasons that clinical trials of treatments for MND have failed. Our group has shown a protein in urine called p75ECD is a biomarker of MND, and is the only identified so far that is a progression and prognostic marker. We now plan to validate our findings in urine by examining if serum and cerebrospinal p75ECD correlates with urinary p75ECD. This also involves collaboration with Pam Shaw’s group in Sheffield, UK. This grant will enable our marker to move forward to clinical trials.
 
Dr Fazel Shabanpoor
Florey Institute of Neuroscience and Mental Health

Alleviation of C9orf72-mediated ALS: A novel bi-functional peptide- oligonucleotide strategy both suppressing gene expression and activating autophagic clearance of toxic protein

Abnormal expansion of the C9ORF72 gene is the most common genetic cause of MND. The product of expanded region within this gene are proteins which aggregate inside a particular group of nerve cells known as motor neurons. These toxic proteins cause the degeneration of motor neurons and progressive muscle weakness. The central objective of this study is to develop dual purpose biotherapeutic molecules known as peptide-oligonuclotides as a potential therapy for C9ORF72-linked ALS. This novel therapeutic approach prevent the formation and simultaneously clear the toxic protein aggregates.
 
A/Prof Ronald Sluyter
University of Wollongong

Establishing the therapeutic potential of the P2X7 receptor ion channel in amyotrophic lateral sclerosis 
 
There are currently no effective treatments for MND. Findings from our group and others indicate a role for a communication pathway (termed the ATP-P2X7 pathway) between motor neurones and other cells of the central nervous system in the progression of MND. Through the use of a new drug in a classic mouse model of MND, this project will investigate whether blockade of the ATP-P2X7 pathway can prevent MND progression. This research will provide further insight into the mechanisms in MND and assist in planning possible drug trials in people with MND.
 
Dr Rachel Tan
Brain and Mind Centre, University of Sydney

Is ATXN2 a potential therapeutic target in MND? 
 
The death of neurones in MND is caused by a normal protein called TDP-43 becoming toxic. Cell and animal models have shown that another protein, called ataxin-2 (ATXN2), may be involved, a concept that appears more certain as genetic variability in the ATXN2 gene increases the risk of getting MND and also shortens survival. This study will assess whether variability in the ATXN2 gene impacts on protein levels (different forms of ATXN2 and toxic TDP-43) and neuronal integrity in patients with MND in order to provide critical information on whether therapeutic strategies for MND should target ATXN2.
 
Dr Bradley Turner
Florey Institute of Neuroscience and Mental Health

Profiling the gene expression pathways of motor neuron vulnerability early in development of MND 
 
While MND typically presents in mid to late-life, the seeds for development of MND may be sown years or even decades before symptom onset. This implies the pathological processes of MND may start early in life. Using gene profiling technology, this project will identify the earliest genetic changes occurring in motor neurons in a mouse model of MND for the first time. Identification of the earliest genetic changes in motor neurons may provide new insights into potential critical players responsible for triggering motor neurone vulnerability and therefore highlight relevant gene targets and pathways for effective intervention in MND.
 
Dr Mehdi Van den Bos
Westmead Hospital and University of Sydney

Pathophysiological mechanisms underlying ALS: insights from novel cortical functional techniques
 

MND is a progressive and invariably fatal disease affecting central and peripheral parts of the nervous system, the cause and evolution of which remains incompletely understood. A growing body of evidence has provided support for a “dying forward” model of the disease and is centered on excessive excitatory activity (“glutamate excito-toxicity”) at the cortical level initiating a degenerative cascade. Whilst recent critical work at a cellular level has provided dramatic insight into this process a translational demonstration in humans is awaited. Our project will provide direct neurophysiological evidence of increased cortical excitation in humans, demonstrate how the disease spreads from this cortical excitatory origin, and is likely to provide a critical earlier biomarker by which treatment efficacy may also be judged.
 
Dr Adam Walker
Macquarie University

New proteins and pathways contributing to TDP-43-mediated neurodegeneration
 
In almost all patients with MND, a protein known as TDP-43 misbehaves in the brain and spinal cord. I have created genetically modified mice that develop TDP-43 pathology and disease very similar to human patients, to allow the analysis of brain and spinal cord tissues at early stages of disease development. In collaboration with other researchers, my team is using advanced biochemical analyses to narrow down the biochemical changes involved in disease in these mice. This project will characterize the role that newly identified proteins play in disease, with the goal of identifying new ways to treat MND.
 
A/Prof Trent Woodruff
The University of Queenslsand

Therapeutic inhibition of HMGB1 to slow disease progression in MND 
 
In MND there is death of nerve cells. As yet there is no way to stop these cells from dying and new approaches are thus needed. We are studying the role of the immune system in MND. We have evidence that activation of the immune system contributes to the progression of disease. In particular we have been studying a group of proteins called the toll-like receptor (TLR) system. We suggest that the therapeutic targeting of this system could slow the progression of MND. In this study we will investigate this further, using a novel therapeutic drug to treat MND mice. If this study is successful, we will then be able to perform a trial of our novel drug, which acts on this TLR pathway.
 
Prof Naomi Wray
The University of Queensland
 
GWAS data for SALSA-SGC 
 
The 2015 MNDRIA Ice Bucket Challenge Grant has provided funding to establish the Sporadic ALS Australia Systems Genomics Consortium (SALSA-SGC). The consortium has established common clinical data and biological sample collection protocols and online sample tracking systems, which are being rolled out to all major ALS clinics in Australia. This harmonised resource of patient data will underpin many future studies (including non-genomics studies). We will now take the SALSA-SGC samples forward through to scientific discovery by generation of basic genome-wide genetic data, contributing to international collaborative efforts in a paradigm proven to work in providing new leads for research


 
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