2017 Annual Report

Where hope begins

New beginnings and expansionOur Patrons

From January 1st 2017, we became the Perron Institute for Neurological and Translational Science, formerly the Western Australian Neuroscience Research Institute (WANRI), acknowledging the long-standing support of Jean and Stan Perron AM and their family from the very beginning.

The new Ralph and Patricia Sarich Neuroscience Research Institute building, thanks to the unrelenting efforts of Professor Bryant Stokes AM and the Sarich family, became our new home as of July 2017. After 35 years in Sir Charles Gairdner Hospital A block, the clinics moved to the more easily accessible ground floor and our laboratory and operations moved to the first floor.

In 2017 the extension of our research capability commenced with the addition of three new research groups; Neurotrauma led by Professor Melinda Fitzgerald, Spinal Cord Repair led by Associate Professor Stuart Hodgetts and Motor Neurone Disease Genetics and Therapeutics led by Professor Anthony Akkari.

In January 2018 our expansion continued with the appointment of Associate Professor Jennifer Rodger in the area of Brain Plasticity, funded by MSWA.

Advances in neuroscience worldwide have provided a significant influence on the Institute’s expansion and vision for the future.

We are working with government, research and business leaders to shape the state’s translational research agenda with the goal of putting the state on the map as a leading centre for medical innovation.

“Today’s neuroscience research takes us into exciting new worlds. Horizons once thought beyond the realms of human scientific endeavours are now in view.

As Patron of the Perron Institute for Neurological and Translational Science, I feel privileged to support their breakthrough work conducted here in Western Australia. Their translational research is delivering game-changing treatments to people worldwide who live with debilitating neurological conditions.

Together with these advances, the potential for neuroscience to take even greater strides is more hopeful than ever before.

This is particularly relevant in addressing the enormous human, economic, health and productivity costs associated with an increase in neurological conditions, and our ageing population. It is here that these scientific advances can have the most profound impact on our shared futures.

I am proud to represent the Perron Institute as Patron, and I look forward to hearing about the Institute’s further research outcomes that immeasurably enhance life for our WA communities and beyond.”

Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia

Mr Stan Perron AM

Professor Lyn Beazley AO

The Perron Institute continues on its path of research translation, where findings from the laboratory are developed into advances in neurological care for the benefit of the community.

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ScopeOur mission is to undertake quality research across the scope of the neurological disease spectrum, building on our research successes for conditions such as muscular dystrophy, stroke, Parkinson’s, multiple sclerosis, motor neurone disease, myositis and restorative neurology.

TranslationWe are committed to translating the outputs of our research into better outcomes for people living with neurological conditions, including new drugs and diagnostics, new therapies and improved ways of delivering services to the community.

QualityThrough a multidisciplinary and collaborative approach, we are committed to research that supports the innovative and integrated care of people with neurological conditions.

3 Key PillarsMedical ResearchOur medical research is centred on both laboratory and clinical research outcomes. Medical research remains the Perron Institute’s core mission, with the Institute having an enviable reputation for its contribution to medical research through its leading scientists for over 35 years.

Therapeutic ServicesWe provide therapeutic services through the Perron Institute Centre for Restorative Neurology, utilising advanced technology in robotics, balance therapy and at home devices to provide a better quality of life for people with neurological conditions.

ClinicsWe conduct 15 clinics for the treatment of various neurological conditions, in partnership and under contractual arrangements with the Western Australian Department of Health and Sir Charles Gairdner Hospital. Our medical clinics include some of Western Australia’s leading neurologists dealing with some of the state’s most complex cases.

To be recognised nationally and internationally as a leader in neurological and neuromuscular research and patient care.Vision

Mission

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A message from the Chair

Over its lifetime, the Institute has had a few names; the Australian Neuromuscular Research Institute (1983-2013), the Western Australian Neuroscience Research Institute (2013-2016) and from January 2017, the Perron Institute for Neurological and Translational Science – the Perron Institute for short.

The change of title is in recognition of the long-standing support of Stan Perron AM and his wife, Jean and their family. We are pleased to honour Stan as an outstanding Western Australian whose generous philanthropy has benefited so many across the state.

2017 saw a change in the leadership of the Institute when I succeeded Roger Hussey as Chair in May. Roger made an outstanding contribution to the Institute over the short time he was Chair and the Institute was greatly saddened by his tragic death in July. 2017 also saw the appointment of businessman Ian Green as a Director and barrister

Rohan Hardcastle will join the Board as a Director in May 2018. These two critical appointments will add significantly to the Board’s skill sets and effectiveness.

The opening of the new laboratories poses a number of challenges in terms of safety and compliance and we are fortunate that Professor Sue Fletcher has agreed to take on the role of Director of Research overseeing the Institute’s research and advancing our collaborative agenda.

On behalf of the Board and the Institute’s staff, I would like to congratulate Clinical Professor Bill Carroll, multiple sclerosis clinician and researcher, on his election as President of the World Federation of Neurology. This appointment is timely recognition of Bill’s decades of service to neurology and as a leader in the field of multiple sclerosis treatment and research.

From a strategic perspective, the theme of 2017 was growth. During 2017, the Institute moved into new state-of-the-art facilities at the QEII Medical Centre and

began the process of appointing three more senior researchers to add to the four appointed in the previous year. The quality of the scientists appointed is outstanding, which bodes well for the future of the Institute. Building on these appointments, the Institute signed a Memorandum of Understanding with Notre Dame University, which means that the Perron Institute has the unique distinction of being formally affiliated with all five of Perth’s major universities. Our links with institutions across Australia and internationally are also growing.

Finally, I would also like to take the opportunity to recognise all of the members of the Perron Institute’s research and clinic groups, our dedicated administrative team, the Institute’s Board members, our Affiliates, Ambassadors and Patrons and thank them for their contributions and commitment.

Emeritus Professor Alan Robson AO CitWAChairMay 2018

Our Patrons 1

New beginnings and expansion 2

Key Pillars, Vision & Mission 3

A message from the Chair 6

Board and Leadership Group 7

A message from the Director 8

Chief Executive Officer’s Report 9

Areas of Medical Research 10

Molecular Therapies Research 11

Precision Nucleic Acid Therapeutics Research 12

Motor Neurone Disease Genetics and Therapeutics Research 13

Stroke Research 14

Neurotrauma Research 15

Spinal Cord Repair Research 16

Neuropathology Research 17

Demyelinating Diseases Research 18

Myositis Research 19

Restorative Neurology Research 20

Clinical Psychology Research 21

Perron Institute Treatment Clinics 22

Our Consultant Neurologists 23

Highlights 24

Scientific Publications 37

Grants 43

Financial Statements 45

Thank you 46

Contents

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Jo WilkieBoard Director

Professor Steve Wilton

Director

Abigail DaviesBoard Director

Ian GreenChair of the Finance

Committee

Winthrop Professor Alan Harvey

Chair of the Research Advisory Committee

Clinical Professor David Blacker

Medical Director

Dr Liz DallimoreBoard Director

Emeritus Professor Alan Robson AO

Chair

Steve ArnottCEO

Torsten KetelsenChair of the Fundraising Committee

Professor Norman Palmer

Special Projects Director

Board and Leadership Group A message from the Director

Our focus in recent years has been on growth, with the goal of creating an Institute that has the intellectual depth and critical mass to compete on the international stage. 2017 saw the latest phase in this expansion process with the appointments of Professors Anthony Akkari and Melinda (Lindy) Fitzgerald and A/Professor Stuart Hodgetts to extend the Institute’s research into the fields of motor neurone disease and neurotrauma.

The process of expansion continued throughout 2017 with international searches for key appointments in the fields of brain plasticity, neurodegenerative disease and multiple sclerosis. This has led to the appointment of A/Professor Jennifer (Jenny) Rodger from The University of Western Australia, generously funded by MSWA, and Professor Sulev Kõks from the University of Tartu in Estonia.

Through the efforts of our CEO, Steve Arnott and others, the Perron Institute has partnered with several

of Perth’s universities in making these appointments, spreading the cost and embedding our research across the universities – Anthony Akkari and Sulev Kõks through Murdoch University, Lindy Fitzgerald with Curtin University and Jenny Rodger and Stuart Hodgetts through The University of Western Australia. Our strategy of partnering with universities and organisations such as MSWA has allowed the Perron Institute to grow at an exceptional rate, far in excess of our initial expectations. Our goal was to recruit six new research leaders over five years – the reality is that we were able to fill five of these six positions within three years, as well as securing funding for a further MS appointment to be filled in 2018, also funded by MSWA.

After growth comes consolidation, and we see our next immediate priority to be creating robust, externally funded research groups centred around each of our newly appointed research leaders. One additional consequence of the growth in recent years has been a spectacular increase in the number of PhD and Honours students, which are now approaching 50.

The highlight of 2017 for me personally was a visit last May by Billy and Terri Ellsworth. Billy suffers from Duchenne muscular dystrophy (MD) and was one of the first boys to receive Exondys 51, an antisense oligomer drug developed in the old Perron Institute laboratories in “A” Block and licensed through the University of Western Australia to Sarepta Therapeutics (Cambridge, Ma). The US Food and Drug Administration gave accelerated approval for Exondys 51 in September 2016, for the treatment of Duchenne MD. This compound continues to be trialled in the US and UK, along with two new exon skipping drugs for other common Duchenne MD-causing mutations. Natural history studies of boys with Duchenne MD suggest Billy should have lost ambulation and be restricted to a wheelchair around 11.1 years of age. However, Billy is now more than 17 years 4 months old and still ambulant thanks to Exondys 51. Seeing Billy walk and be so active brought home to me, and my colleagues, why we chose to be scientists in the first place.

Professor Steve Wilton Director May 2018 87

A special feature of the Perron Institute is its commitment to research translation; the process of taking findings from the laboratory and translating these into practical outcomes of benefit to people living with neuromuscular and neurological conditions. Research translation is part of our DNA.

Areas of Medical Research Chief Executive Officer’s Report

2017 was indeed a significant year for the Perron Institute in which a number of planned outcomes finally reached fruition.

After years of planning and then construction, the new Ralph and Patricia Sarich Neuroscience Institute building was opened in April 2017 by our Patron, Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia. The untiring efforts of Professor Bryant Stokes AM and the patience and commitment of the Sarich family to see this vision fulfilled is applauded and the Western Australian neuroscience fraternity are indebted.

The Institute moved into its new state of the-art facilities in July and despite the obvious contrast from being housed in SCGH A Block, it would be remiss of me not to thank the Health Department (through SCGH) for providing this research space to the Institute for over 20 years. This initiative by the Health Department provided accommodation for the development of world leading research in Muscular Dystrophy and the development of the “gene patching” technology.

The Perron Institute will continue the development of its remaining floor space and will partner in the establishment of a Bio Resources Centre for use by residents in the building. The move has been extremely successful. The Institute has recruited assertively to ensure both new areas of research and areas involving the extension of the platform technologies into other conditions can occur as quickly as possible. An additional 30 PhD and Honours students together with their supervisors will be housed in the new facility by the end of 2018.

The Institute’s strategy of developing collaborative links in Asia continues. Hans Michael Jebsen, a leading member of the Hong Kong community, has become the Institute’s Ambassador for Hong Kong, China, an initiative announced at an event in Government House in November 2017 and is facilitating links with universities in Hong Kong with the goal of extending our outreach into Hong Kong and China.

Research on the muscle disease myositis, has been a strength of the Perron Institute for many years led

by Professors Merrilee Needham and Frank Mastaglia. This research was given added momentum in 2017 through a generous bequest from the late Marcia Sheath. Our goal is to consolidate the Institute’s position as Australia’s leading centre for myositis research.

Operationally, the Institute has performed extremely well and its financial strength and stability enhanced considerably. One of the strengths of the Perron Institute is its network of collaborative links with researchers, institutions and organisations within Western Australia, Australia and internationally. We now have collaborative appointments and affiliation agreements with all Western Australian universities, a number of neurological condition-based organisations in Western Australia in addition to two collaborative appointments with US organisations.

2018 is shaping up to be another important year in the development of the Perron Institute as our growth is further realised.

Steve Arnott CEO May 2018

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Molecular Therapies Research

Professors Steve Wilton and Sue Fletcher continue their award winning research with their team based at the Centre for Comparative Genomics in the Health Research Precinct at Murdoch University.

Their innovative treatment for Duchenne muscular dystrophy, Exondys 51, received accelerated approval by the US Food and Drug Administration in September 2016. Two other drugs for other common Duchenne muscular dystrophy mutations are currently in advanced Phase 3 clinical trials.

Exondys 51 is the first drug to address the most common subgroup of dystrophin mutations causing Duchenne. Other research has applied the ‘genetic patch’ technology developed for Duchenne to other diseases such as spinal muscular atrophy, the most common genetic cause of death in children under the age of two years, adult onset Pompe disease and other forms of muscular dystrophy. The splice intervention technology has the potential to be applied to a wide range of target diseases, including cystic fibrosis, epilepsy, macular degeneration, multiple sclerosis, osteogenesis imperfecta, epidermolysis bullosa and motor neurone disease.

Precision Nucleic Acid Therapeutics Research

McCusker Research Fellow Dr Rakesh Veedu and his team continue their research on developing novel precision nucleic acid-based therapeutic and diagnostic molecules for tackling neurological diseases including Alzheimer’s disease, brain cancers and type 2 diabetes.

Dr Veedu’s team has developed one potential lead drug molecule towards the treatment of glioblastoma, diabetic retinopathy, age-related macular degeneration by targeting a common pathological hallmark, and another candidate to reverse insulin resistance for tackling type 2 diabetes which have gone through lead optimisation stages in vitro.

Billy Ellsworth, who participated in the USA clinical trial, Professor Steve Wilton and Professor Sue FletcherImage credit: Murdoch University

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Stroke Research Stroke Research, led by Associate Professor Bruno Meloni and Clinical Professor Neville Knuckey (a neurosurgeon at SCGH), is exploring the application of a new class of peptide with neuroprotective potential to limit brain damage after stroke and other acute brain injuries. Peptides are small chains of amino acids and the building blocks of protein.

The group has already patented a poly-arginine peptide as a treatment for stroke, and more recent studies have demonstrated that the peptide may be useful for limiting brain injury following perinatal hypoxia-ischaemia and traumatic brain injury.

Ongoing studies are examining if the peptide can reduce brain damage after cardiac arrest, and damage to the spinal cord after spinal injury. In addition, essential final stage pre-clinical studies are planned to confirm the peptide’s translational clinical effectiveness for stroke.

Motor Neurone Disease Genetics and Therapeutics Research

The major focus of Motor Neurone Disease Genetics and Therapeutics Research, led by Professor Anthony Akkari, is new strategic approaches to identifying genetic mechanisms and mutations in neurological diseases, with the aim of developing new and personalised treatments, particularly for motor neurone disease also known as amyotrophic lateral sclerosis.

The main aim of the research is to understand sporadic motor neurone disease and improve outcomes of clinical trials, a serious unmet need. One strategy is to further develop biomarkers which may enrich a patient’s response to therapeutics.

In early 2017, Professor Akkari was appointed the Head of Motor Neurone Disease Research at the Perron Institute, and as a Professor of Industrial Pharmacogenetics at Murdoch University.

PhD student Adam Edwards and Dr Diego Milani13 14

Spinal Cord Repair Research

The objective of Spinal Cord Repair Laboratory Research led by Associate Professor Stuart Hodgetts is to develop clinically relevant strategies for the treatment of spinal cord injury (SCI).

A main focus is the use of human multipotent, “mesenchymal stem cells” (MSCs) isolated from the bone marrow of adult SCI patients to promote self-repair and regeneration in preclinical models of SCI.

Other strategies include gene therapy, in vivo reprogramming, neuroprotective peptides, tissue engineering (including self-assembling peptides, scaffolds), non-invasive therapies (including magnetic stimulation and infra-red/near infra-red light) and immunomodulation.

Professor Hodgetts laboratory is soon to be co-located at the Perron Institute and The University of Western Australia.

Neurotrauma Research

The objective of Neurotrauma Research led by Professor Melinda (Lindy) Fitzgerald is focused around understanding and preventing the loss of function that occurs following neurotrauma. The group uses this information to design and assess treatment strategies including nanotechnologies and combinatorial pharmacotherapeutics.

Their drive to translate their research findings to the clinic is evidenced by their current collaborative clinical trial assessing biomarkers and MRI for prediction of post-concussion syndrome.

Professor Fitzgerald’s laboratory is soon to be located in the Curtin University precinct in the Sarich Neuroscience Research Institute.

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Demyelinating Diseases Research

Demyelinating Diseases Research, led by Clinical Professor Allan Kermode, is dedicated to investigating the causes of multiple sclerosis (MS) and improving the treatment and management of those suffering with MS and related diseases.

Last year the group completed an exciting study using ultraviolet B (UVB) phototherapy as a treatment for patients with clinically isolated syndromes (CIS). One of the most interesting results of the study showed that 30% of CIS patients treated with UVB phototherapy did not convert to MS within one year, and a number of promising biomarkers have been evaluated. The study was conducted in collaboration with the Telethon Kids Institute.

The group also evaluated patients with benign MS 20 years from first presentation. This study showed that patients labelled as benign MS are part of a heterogeneous continuum of disease progression and do not possess unique clinical characteristics. Our collaborations continue with the Telethon Kids Institute and also Sydney University with Professors David Booth and Scott Byrne with further results expected.

In addition, the group has established important international collaborations with centres in Guangzhou at Sun Yat Sen University (Professor Wei Qiu) and with Kyushu University in Japan (Professor J-I Kira), and with the IMSGC, ANZGene, MSBase and the Progressive MS Alliance.

Neuropathology Research

Emeritus Professor Byron Kakulas AO, the Perron Institute’s Founding Director, continues his neuropathological research as a consultant in the areas of restorative neurology of spinal cord injuries, muscular dystrophy, Alzheimer’s disease and motor neurone disease.

An international collaboration with Professor Milan Dimitrijevic of Baylor University, Houston, is now focused on the “incomplete” spinal cord injury syndrome in which he has shown that residual nerve fibres which have escaped the primary injury may be reactivated.

Myopathological expertise is provided for Dr Ros Quinlivan in London, who is applying the pioneer work of the late Professor John Howell as a treatment for McArdle’s disease. Using a model Professor Howell showed that Valproate can activate a dormant foetal gene in the condition. Preliminary results in the human trial are encouraging.

Professor Kakulas also provides referees reports and expert appraisals for individuals and major projects.

Emeritus Professor Byron Kakulas AO

Clinical Professor Bill Carroll, Dr Marzena Pedrini and Clinical Professor Allan Kermode

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Restorative Neurology Research

The Centre for Restorative Neurology is an initiative with a key focus on research to improve the functional recovery of patients after suffering a neurological illness or injury, such as stroke, multiple sclerosis, Parkinson’s and head injury.

Led by Clinical Professor Soumya Ghosh, the Centre is an area initially funded by Mr Stan Perron AM. The Centre is also appreciative of the ongoing support of MSWA, in particular Marcus Stafford (CEO) and Sue Shapland (General Manager, Member Services). Their continued nursing support and funding enables the Institute to continue their clinical research for people with multiple sclerosis (MS).

2017 involved ongoing research into the effectiveness of various rehabilitative treatments in patients with stroke and multiple sclerosis. In addition, increased focus was applied to neurophysiological research in healthy subjects, which will provide a greater understanding of the mechanisms involved recovery and ultimately assist in optimizing rehabilitative treatments for people with neurological illness.

Myositis Research

Myositis Research, headed by Professor Merrilee Needham, has continued a program of combined clinical and laboratory research, focusing on inclusion body myositis (IBM) which is the most disabling type of myositis which responds poorly to currently available treatments.

In 2017, Dr Jerome Coudert, who is a highly experienced immunologist joined the Myositis Research group and has commenced a program of investigation into the inflammatory T-cells responsible for the immune-attack in IBM and their antigenic targets. The long-term objective is to develop new therapies to switch off the immune attack and stop the progression of the muscle inflammation and weakness.

The Perron Myositis Clinic run by Professor Needham and Dr Anna Brusch from the Department of Immunology at SCGH, provides a comprehensive program for the diagnosis and treatment of all types of myositis, with regular patient follow-up and support. As part of this, a new Myositis Registry and Biobank is being developed to provide biological samples and clinical data to support the laboratory research program. Other research projects include the development of a new treatment for swallowing difficulties in patients with IBM, recruitment of patients for a trial of testosterone for IBM, and a trial of Abatacept for myositis patients resistant to other forms of treatment.

In 2017, the late Marcia Sheath, who suffered from IBM, generously bequeathed the whole of her estate to the Perron Institute for research into myositis and finding a cure for IBM. Marcia formed a trusted connection with Professor Needham while she was living with IBM and a myositis research centre will be named in her honour.

Jennifer Eisenhauer, Research Assistant, Clinical Professor Soumya Ghosh and Jesse Dixon, Senior PhysiotherapistProfessor Merrilee Needham

2019

Clinical Psychology Research

Dr Michelle Byrnes is currently engaged in numerous grant funded research projects with other Clinical Neuroscientists, Clinical Psychologists and Neurologists in clinical research and practice in the area of functional neurology and neurorehabilitation.

The aim of these projects is to enable the development and implementation of clinically relevant therapeutic programs to assist individuals with psychosocial and neurocognitive difficulties in their everyday lives. In addition, Dr Byrnes is mentoring and supervising numerous PhD and Clinical Masters students within the Perron Institute, Schools of Psychology, Exercise Science and Engineering and Information Technology at Murdoch University and the School of Psychology at UWA, to enable them to expand their clinical and research skills.

Dr Byrnes is also a Clinical Psychologist Consultant for numerous not-for-profit and business sector clients who require her expertise in the areas of pre-employment screening and the identification and evaluation of psychosocial and neurocognitive aspects of their wellbeing and functioning.

Clinical Associate Professor Michelle Byrnes

Perron Institute Treatment Clinics

One of the Perron Institute’s special strengths lies in its depth of links between the Institute’s laboratory research and its specialist neurology clinics.

The Perron Institute is an international leader in translating research into treatment for the benefit of the community.

In 2017 the Perron Institute operated 15 clinics each week for people suffering from multiple sclerosis, motor neurone disease, neuromuscular conditions, epilepsy and movement disorders including Parkinson’s. This equates to over 2000 patient visits in a year.

Our treatment clinics are led by some of Perth’s leading consultant neurologists and clinical staff, and recognised as providing the best clinical management for people with highly complex neurological conditions.

The clinics also provide an opportunity for people to participate in the latest clinical trials.

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Our leading consultant neurologists deal with some of the most complex neurological cases in the state and conduct a significant number of consultations a year.

More than half of our specialists also undertake cutting edge research on a broad spectrum of conditions including stroke, Parkinson’s, multiple sclerosis, motor neurone disease and myositis.

Cl/Prof David BlackerMedical Director,

stroke clinic

Cl/A/Prof Rick StellMovement disorders (inc. Parkinson’s) clinic

Cl/Prof Merrilee Needham

Neuromuscular clinic

Cl/Prof Allan KermodeMultiple sclerosis clinic

Cl/Prof Soumya GhoshMovement disorders

clinic

Dr Janavi DunuwilleEpilepsy clinic

Cl/Prof Bill CarrollMultiple sclerosis clinic

Dr Rajini (Sivarajini) Rajanayagam

Movement disorders, MND and MS clinic

Dr Athanasios Gaitatzis

Epilepsy clinic

Dr Jason BurtonMultiple sclerosis clinic

Dr Rob EdisMotor Neurone Disease clinic

Our Consultant Neurologists Highlights Here is a selection of some of our many exciting developments in 2017. With so much happening, it is important to thank all of the individuals who make our Institute what it is. For latest news see our website: perroninstitute.org.

Our new name became official

In January 2017, our new name, the Perron Institute for Neurological and Translational Science, became official after a special announcement at the end of 2016 at Government House hosted by our Patron, Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia. We were thrilled to rename the Institute in honour of our longstanding supporters Jean and Stan Perron AM and their family.

The Perron family have been contributing to the Institute’s research into muscular dystrophy and other neurological conditions for more than 30 years, initially supporting the work of the Institute’s Founder, Emeritus Professor Byron Kakulas AO. This eventually led to the US Food and Drug Administration granting accelerated approval for a novel treatment for Duchenne muscular dystrophy, developed by Professors Steve Wilton and Sue Fletcher, using “genetic patches” to mask the error in the gene message that causes Duchenne.

The Perron Institute’s track record of translating research findings into positive outcomes for the community continues with its exciting future expansion. Part of our future plans is to extend this novel technology to as many other conditions and applications as possible. In addition to genetic patching, the Institute is engaged in life-changing research on conditions such as stroke, Parkinson’s, motor neurone disease and multiple sclerosis. Building on our breakthroughs, the Institute is recruiting the world’s best researchers to undertake cutting-edge research on new treatments for these devastating conditions.

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Highlights continued

Professor Alan Robson appointed as Board Chair

The Institute was delighted to announce the appointment of Professor Alan Robson AO CitWA as our new Board Chair in May 2017. Professor Robson commenced his new role at the AGM and Board meeting on 26th May.

Professor Robson, previous Vice-chancellor of UWA, brings a great deal of experience both as a leader and Chair and provides significant research and advisory experience, a strong interface with industry and a commitment to innovation.

Earlier in his career, Professor Robson was an internationally regarded scientist who contributed to the prosperity and innovation of the state.

Professor Robson has received various accolades including the Australian Medal of Agricultural Science, the Fiona Stanley Medal, an Officer of the Order of Australia in 2013, and in 2015, inducted into the WA Science Hall of Fame.

Professor Sue Fletcher appointed as Research Director

In the middle of 2017, Professor Sue Fletcher was appointed the Institute’s Director of Research. Her key role is to oversee the research activities of the Institute, ensure compliance and advance the Institute’s collaborative agenda.

Professor Fletcher first joined the Institute in 1991 and is a member of the Research Advisory Committee. She currently sits on both the Telethon Kids Institute and Murdoch University Animal Ethics Committees. Previously, Professor Fletcher was a member of the UWA Institutional Biosafety Committee for 10 years and of the Scientific Review Committee of the Telethon Institute of Child Health Research for six years.

This new role is additional to Professor Fletcher’s role as Deputy Director of the Centre for Comparative Genomics (CCG) at Murdoch University and supports the role of longtime colleague Professor Steve Wilton as Director. Professor Fletcher provides advice on the Institute’s research projects and oversight of ethical compliance and research integrity.

Bill Carroll becomes President of the World Federation of Neurology

Clinical Professor William (Bill) Carroll was elected President of the World Federation of Neurology (WFN) during the 23rd World Congress of Neurology (WCN) in Kyoto, Japan, in September 2017. The highest position in the world’s premier organisation for neurologists, Professor Carroll assumed the office of President from January 1st 2018.

Professor Carroll, a multiple sclerosis (MS) clinician and researcher at the Perron Institute and neurologist at

Sir Charles Gairdner Hospital, has been recognised for decades as a leader in the field of MS treatment and research.

Appointed as First Vice President of the WFN in 2014, Professor Carroll has been a prominent leader and member within the London-based WFN for 18 years. Professor Carroll has many accolades including 2012 Western Australian of the Year Award (Business & Professions).

Perron Institute Director, Professor Steve Wilton, Medical Director, Clinical Professor David Blacker, and Head of MS research, Clinical Professor Allan Kermode, who featured in the program, joined Professor Carroll during the momentous WCN.

New building opening and move

Our Patron, Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia, officially opened the new Ralph & Patricia Sarich Neuroscience Research Institute building on 26th April 2017, hosted by Professor Bryant Stokes AM, North Metro Health Service and the Sarich Family.

Jean and Stan Perron AM, 16 year old Billy Ellsworth - on the US trial of the drug which received accelerated approval by the US Food and Drug Administration - and his mother Terri, were special guests at the momentous celebration.

This building brings together many of Western Australia’s leading neuroscience research organisations including the Perron Institute, the Australian Alzheimer’s Research Foundation, Ear Science Institute Australia, Curtin University and Edith Cowan University Neurosciences. This consolidation of neuroscience research in the Sarich building has the potential to deliver enormous health benefits to the community.

In July 2017, we were thrilled to have moved the clinics, laboratories and operations into the ground and first floors of the new building. Our specialist neurology clinics being located on the ground floor ensures both easier access for our patients and links are maintained between laboratory research and clinical research and practice.

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New Ambassador Hans Michael Jebsen

In 2017, a special event was held at Government House in Perth, hosted by our Patron, Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia, who made two exciting announcements.

The first announcement was the appointment of Mr Hans Michael Jebsen as Perron Institute Ambassador to Hong Kong, China. Mr Jebsen (pictured left) is Chairman of the Jebsen Group in Hong Kong and known and respected internationally as a business leader and philanthropist.

The second announcement, initiated by Mr Jebsen, involves the Institute developing a new collaborative partnership with the Hong Kong University of Science and Technology.

In particular with the Key State Laboratory of Molecular Neuroscience at the Hong Kong University of Science and Technology headed by Director and neuroscientist Professor Nancy Ip.

It is hoped this partnership will lead to a broader relationship with leading scientists in Hong Kong dedicated to working toward neurological translational research outcomes. Torsten Ketelsen (pictured right), Perron Institute Board member and Chair of the Fundraising Committee, is responsible for connecting the Perron Institute with Mr Jebsen.

Highlights continued

NHMRC funding

Several members of the Institute were successful in a round of NHMRC grants announced in December 2017.

• Professors Steve Wilton, Sue Fletcher, Ming Hao Zheng and Frank Mastaglia, received $797,165 to extend the antisense technology to treat other diseases;

• Clinical Professor Neville Knuckey, head of Stroke Research, is part of a group with Professor Karol Miller, a leading mathematician from UWA, who received $370,055 to model brain shift to aid in tumour resection during surgery;

• A/Professor Jenny Rodger is part of a group with Dr Kaylene Young in Tasmania who received $664,869 for a MS project; and

• Research affiliate A/Professor Livia Hool at UWA received $1,312,606 for a hypertrophic cardiomyopathy project.

Marcia Sheath’s significant generosity towards myositis research

Even in the depths of her cruel suffering, Marcia Sheath was positively determined that others with her debilitating disease would have a chance to be spared her fate.

While this fiercely independent and vibrant Sydney woman endured more than 15 years of the devastating muscle wasting condition known as Inclusion Body Myositis (IBM), her sensitivity and enormous generosity of spirit remained to the end.

After Marcia sadly lost her battle in May 2017, it emerged that she has left her entire estate to medical research into Myositis at the Perron Institute. Marcia’s generous gift was inspired as much by her hope that a cure for her terrible disease would be found, as it was by a trusted connection she formed over the years with Professor Merrilee Needham, one of the Institute’s leading neurologists and Head of Neurology at Fiona Stanley Hospital.

Professor Needham and the Institute’s wider research team including Professor Frank Mastaglia are at the forefront of IBM research. The Institute is extremely grateful for Marcia’s exceptional generosity and the empathy her gift expresses. The team is dedicated to ensuring her research wishes are supported, and that her legacy continues. The Myositis research centre will be named in her honour.

Further funding received from MSWA for MS research

In 2015 and 2016, Dr Marzena Fabis-Pedrini of Demyelinating Disease Research, conducted research into multiple sclerosis at the Perron Institute, supported by funding from MSWA. During 2017, Dr Fabis-Pedrini received further funding of $135,000 from MSWA to extend her work for a third year. Dr Fabis-Pedrini’s latest research focuses on benign MS and people who have low levels of disability as a result of their condition as well as aggressive/progressive MS.

Stroke protection research funded by MSWA & NRP

During 2017 our Stroke Research group was awarded $250,000 from MSWA to further investigate reducing damage to the brain after a stroke.

A/Professor Bruno Meloni, Head of Stroke Laboratory Research, and his team including Professor Neville Knuckey, Head of Stroke Research, and Clinical Professor David Blacker, Medical Director, are the Chief Investigators who will assess the effectiveness of their leading peptide in a pre-clinical stroke model to tackle this epidemic.

In August 2017, the Stroke Research group received a $99,500 grant from the Neurotrauma Research Program (NRP) of WA to further assess their lead neuroprotective peptide to reduce brain damage in neurotrauma related injuries, namely global cerebral ischaemia, traumatic brain injury and spinal cord injury.

The Chief Investigators on the project are Associate Professor Bruno Meloni, Professor Neville Knuckey, Associate Professor Stuart Hodgetts and Dr Jane Cross. The Health Minister Hon Ben Wyatt MLA announced the funding provided by the Insurance Commission of Western Australia in a media statement.

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Highlights continued

New Board Director Ian Green

The Board appointed Mr Ian Green as a new Director in 2017. Mr Green is a successful businessman through his involvement in the development and management of Gull Petroleum in both Australia and New Zealand. He is widely known and respected in Perth and the sale of Gull provided an opportunity for Mr Green to be involved in additional activities. He has an extensive financial and management background, is currently Chair of Connolly & Associates and is on the Board of Management of the Maurice Zeffert Home for the Aged. Mr Green’s strong management, business and financial skills are an asset to the Perron Institute Board.

Top Cited Paper for MS

One of the publications from Clinical Professors Allan Kermode and Bill Carroll’s collaboration with the Telethon Kids Institute became a top cited paper. The journal of Clinical and Experimental Immunology listed the paper entitled ‘Circulating Immune Cells in Multiple Sclerosis’ as one of their top five cited articles of 2017. Professors Kermode and Carroll are part of the Institute’s Demyelinating Diseases Research and Professor Kermode is the Head of the group.

Anthony Akkari MND appointment

In 2017 we welcomed Professor Anthony Akkari to the role of Foundation Chair in Industrial Pharmacogenetics and Head of Motor Neurone Disease Genetics and Therapeutics Research, based at both QEII and Murdoch University. Professor Akkari’s background in industry with Perron Institute alumni Professor Allen Roses (who sadly passed away suddenly in 2016), through Shiraz Pharmaceuticals and Duke University, creates a skillset to pursue collaborations and opportunities with a commercial focus. Professor Akkari’s research focus is genetics and motor neurone disease (MND).

In 2017 work commenced on a provisional patent for a SOD1 therapeutic, utilising the antisense oligonucleotides technology to target MND patients with SOD1 genetic mutations and in 2018 it was submitted.

Two new Neurotrauma appointments

There were two new appointments in the area of Neurotrauma Research in 2017. Professor Melinda Fitzgerald, who commenced in April 2017 in a joint role with Curtin University and the Perron Institute focusing on preventing spreading damage after neurotrauma.

Perron Institute Research Affiliate Associate Professor Stuart Hodgetts also commenced in a joint role between UWA and the Institute in 2017 in the area of spinal cord repair research. In 2017 a new collaboration was forged with Professor Gong Chen in Guangzhou, China, following a visit by key researchers from the Institute. This new collaboration involves the use of “in vivo reprogramming” technologies which is an emerging technology that promotes repair and/or regeneration following neurotrauma.

MDWA 50th Anniversary breakfast

Muscular Dystrophy WA (MDWA) celebrated their 50th Anniversary in April 2017 with a series of events including a breakfast at Frasers in Kings Park and several members of the Institute attended.

Founding Director Emeritus Professor Byron Kakulas AO was acknowledged in conjunction with a small group of parents and the Rotary Club of West Perth who started MDWA in 1967.

In the early 1990s, MDWA provided the original seed funding as well as ongoing funding, which enabled researchers Professors Steve Wilton and Sue Fletcher to develop the drug that has heralded a new frontier in the treatment of Duchenne muscular dystrophy (DMD).

At home technology ableX

In 2017, people living with MS and stroke commenced participating in an upper limb rehabilitation project with at home technology ableX across Perth, including some regional areas, through the Institute and in conjunction with MSWA.

AbleX, pioneered in New Zealand and similar to a Nintendo Wii, is a convenient and empowering device for people to take control of their own rehabilitation in their own home.

The project, which has the potential to reduce strain on the healthcare system, was set up to identify the barriers and solutions to setting up this therapy in people’s home environments in the wider community and was funded with a $92,000 grant from Lotterywest.

The project was completed in 2018 and feedback from participants was positive.

After one week a participant with MS said, “Now I can write my name and it’s readable. It’s something so simple”. Another participant post-stroke said, “I can now open a carton of milk on my own and make toast at night if I wake up hungry. I am also wearing a pair of trousers that I haven’t been able to put on for myself since the stroke [until now]”.

Health Minister Roger Cook MLA testing ableX

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Highlights continued

Alan Harvey at TEDxPerth and book published

Board Director and Chair of the Research Advisory Committee, Emeritus Professor Alan Harvey, gave an inspiring presentation about music and the brain at TEDxPerth towards the end of 2017. Research leaders at the Institute have now presented at this popular annual event two years in a row, following the Institute’s Director Professor Steve Wilton presenting in 2016.

Professor Harvey described the power of music as a social communicator and how it differs from speech to a full Perth Concert Hall. He utilised modern

science to show what happens to the brain when it responds to music and moved the whole auditorium to stand and sing at the end of one of the most enjoyed talks of the day.

Also in 2017, Oxford University Press published Professor Harvey’s book entitled “Music, Evolution, and the Harmony of Souls”. Robyn Williams on the ABC’s ‘The Science Show’ interviewed Professor Harvey about it at the time and he has been interviewed many times about his findings, including by a younger demographic on Triple J radio who were fascinated by the subject matter.

Rakesh Veedu lab expansion

Dr Rakesh Veedu, McCusker Fellow and Head of Precision Nucleic Acid Therapeutics Research at Murdoch University, and his research group significantly expanded in the past two years. In 2017 they published 11 research articles in high quality journals, and jointly filed 1 provisional patent application entitled “Thiomorpholino oligonucleotides for the treatment of muscular dystrophy” in collaboration

with Professor Marvin Caruther’s lab at the University of Colorado, USA.

One of Dr Veedu’s articles “Novel Chemically-modified DNAzyme targeting Integrin alpha-4 RNA transcript as a potential molecule to reduce inflammation in multiple sclerosis” published in the journal Nature Scientific Reports, won the Thermofisher best manuscript prize which was awarded at Combined Biological Sciences Meeting (CBSM) 2017.

Dr Veedu also presented an invited Plenary lecture at ISNAC in Tokyo, Japan in November 2017, and this conference is one of the best and largest international conferences in the field of nucleic acids, where there were 385 participants from 15 countries. His PhD student Mr Bao Le won the Perron Institute best poster prize at CBSM 2017, and both Mr Le and PhD student Mr Suxiang Chen were also selected as the top finalists of Murdoch University Annual 3 minute thesis competition in 2017.

New Affiliates

In 2017, we welcomed several new International and Research Affiliates to the Institute; Professor Jaglish Prasad Agrawal, Dr David Prentice, Professor Rohit Pokharel, Associate Professor Livia Hool, Dr Travis Cruikshank and Associate Professor Julian Heng.

• Jaglish Prasad Agrawal is Professor of Internal Medicine at the Institute of Medicine at Tribhuvan University in Kathmandu, Nepal. Professor Agrawal is one of Nepal’s most distinguished neurologists and is currently President of the Muscular Dystrophy Foundation-Nepal.

• Dr David Prentice is a Consultant Neurologist in the Department of Medicine at Royal Perth Hospital (RPH), who has worked in the RPH’s Stroke Unit for many years and has been involved in clinical trials with Clinical Professor David Blacker and Institute Research Affiliate, Professor Graeme Hankey.

• Rohit Pokharel is Professor and chief of the Spine Unit of the Department of Orthopaedics and Trauma Surgery at the Institute of Medicine, Tribhuvan University. Professor Pokharel is Founder, Board Member and current Vice President of Muscular Dystrophy Foundation-Nepal.

• A/Professor Hool, Head Cardiovascular Electrophysiology Laboratory, School of Anatomy Physiology and Human Biology, UWA, has long-standing research links with Professors Sue Fletcher and Steve Wilton focusing on the cardiac complications associated with Duchenne muscular dystrophy.

• Dr Cruikshank, Postdoctoral Research Fellow, School of Medical and Health Sciences, ECU, is growing links between his research on the design and delivery of treatments for people with neurological disorders, particularly Huntington’s disease, with the Institute’s growing research in the field of restorative neurology.

• A/Professor Heng is a recent appointment at Curtin University as a Group Leader at the Curtin Neuroscience Laboratories based in the Sarich Neuroscience Institute building in close proximity to the Institute. His research on how gene mutations affect neural circuits in the brain and lead to human mental disorders, such as epilepsy and intellectual disability, has close links to the Institute’s research in the fields of genetics and neurotrauma.

Megan Bakeberg awarded Walter Gibbon Scholarship

Megan Bakeberg completed her Honours at the end of 2016 in our Stroke Research group and was awarded First Class Honours. Due to her academic merit, Ms Bakeberg was awarded a Walter Gibbon Scholarship via UWA, and in 2017 started her PhD in the area of Parkinson’s with supervisors Dr Rick Stell, Professor Frank Mastaglia, Dr Ryan Anderton and Clinical A/Professor Michelle Byrnes. Ms Bakeberg was also awarded a Perron Institute Prestige Scholarship to assist with her studies.

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Highlights continued

Science on the Swan 2017

The Perron Institute was a sponsor of the 2017 Science on the Swan Conference and Welcome Reception in May. Several ministers, along with local, interstate and international scientists and our Patrons including Professor Lyn Beazley AO, were also in attendance for the evening in Fremantle.

As Conference Co-Chair, Professor David Morrison, Deputy Vice Chancellor Research and Innovation at Murdoch University, Professor Steve Wilton and our Patron, Her Excellency the Honourable Kerry Sanderson AC, Governor of Western Australia, spoke about the work of the Institute and the role of research in the community. The Governor also asked Billy and his mother Terri Ellsworth, here for their first visit to Australia, to come to the front of the room and share their experience, at a wonderful impromptu appearance before they were due to fly back to Pittsburgh, Pennsylvania, that evening. Billy lives with Duchenne muscular dystrophy and was one of the first boys to receive Exondys 51 approved by the US FDA and he is still uncharacteristically walking at the age of 17.

At both the Welcome Reception and the Conference opening address, the breakthrough of Professors Wilton and Sue Fletcher, and being able to meet Billy in person, was highlighted by Professor Morrison as a significant example of research translation in Western Australia that extends internationally.

SWAN 2017

The WA Symposium of Neuroscience - SWAN 2017 - was held at the Harry Perkins Institute on 26th June and attracted a record 170 registrants, including undergraduate and postgraduate neuroscience students, early career and established neuroscience researchers, allied health professionals and clinicians. The Institute was a platinum sponsor of the event.

Research was presented under broad themes of neuroinflammation, cognitive neuroscience, innovative nanotherapeutics and technologies, and stroke and movement disorders.

There were a variety of speakers from various interstate and WA institutes

and universities, including Katherine Hankinson, a PhD student at UWA, investigating how the Get Rhythm music app enhances brain plasticity, and Professor Andrew Whitehouse (nominated for one of the year’s Eureka Prizes) from the Telethon Kids Institute on rewriting the rulebook for infant intervention to prevent disability in autism.

A highlight was during the ‘Stroke and Movement Disorders’ session chaired by Perron Institute researchers Professor Lindy Fitzgerald and A/Professor Bruno Meloni. Stroke survivor Peter Coghlan (pictured left) and his neurologist Dr David Blacker (pictured right), spoke with Professor Fitzgerald about his inspiring journey conquering locked in syndrome.

CBSM 2017

The 27th Annual Combined Biological Sciences Meeting (CBSM) was held in August 2017 at UWA. The Perron Institute was a sponsor, of plenary speaker Dr Nicolas Plachta’s from A*Star in Singapore, of morning tea and of the best poster presentation award. PhD and Masters candidates Li Shan Chiu

of Stroke Research and Niall Keegan from Professors Sue Fletcher and Steve Wilton’s group presented talks in the main auditorium for the Department of Health WA New Investigator Session, and PhD candidate Adam Edwards also of Stroke Research also presented during the Cell and Developmental Biology session and won the best student oral presentation. Bao Le from

Dr Rakesh Veedu’s group received the Perron Institute poster presentation award, and Madhuri Chakravarthy and Craig McIntosh from Professors Fletcher and Wilton’s group were also recipients of student prizes.

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Highlights continued

Dr Diego Milani MD graduates with PhDDr Diego Milani MD and now also PhD of the Stroke Research group, graduated at the end of 2017 and received the second unconditional pass at the Institute! His project assessed a novel treatment involving peptides to reduce brain damage after cerebral ischaemia.

Dr Milani received scholarships from Notre Dame University and the Perron Institute to complete his PhD in three years. Dr Milani was first author in four papers and second author in two papers written during his PhD candidature.

Of his experience and leaving Italy, Dr Milani says, “For my partner and I, the decision to come to Australia was a big jump, not without heartbreaks and uncertainty. I left my clinical practice to dedicate myself entirely to research. We have been very lucky to find extraordinary people in this Institute, my colleagues, students, researchers and all of the personnel. A special thank you to Bruno Meloni, for matters beyond the work.”

In the middle of 2017, Dr Milani became a Research Officer at the Institute and continues to be an integral part of Stroke Research. In 2018, he commenced a project using MRI to continue his peptide research.

Loren Price graduates with PhD

Dr Loren Price of Molecular Therapies Research based at Murdoch University, completed her PhD and graduated in December 2017. Dr Price’s thesis is titled: “Applications of antisense oligonucleotides in designing a therapy for spinal muscular atrophy [SMA]”. SMA, caused by an insufficiency of the survival motor neuron (SMN) protein that leads to loss of motor neurons, muscle denervation and wastage, is a devastating neurodegenerative disease for which there is currently no cure. Dr Price’s thesis focused on designing and evaluating splice-switching antisense oligonucleotides that increase SMN protein levels as a potential therapy for SMA. Dr Price (pictured between Professors Sue Fletcher and Steve Wilton) is utilising her unique skillset in 2018 working with Professor Anthony Akkari in the area of motor neurone disease at both the Perron Institute and Murdoch University. Dr Price wishes to “acknowledge the very generous financial support throughout her PhD from Team Spencer and the Steven family, the Parry family, Muscular Dystrophy WA (MDWA) and Telethon, for without which this work would not have been possible.”

Research Week talks at Curtin and SCGH

September 2017 was research month at Curtin University and Professor Melinda Fitzgerald, head of Neurotrauma research at the Institute and Curtin, delivered an interesting community targeted lecture focused on her and her team’s work on concussion and neurotrauma.

There were several activities for SCGH Research Week in October 2017, including a tour of the QEII precinct, which included the Sarich Neuroscience Institute building where Clinical Professor David Blacker spoke to the tour group assembled in the ground floor foyer about the purpose of and tenants housed in the building. Professor Marcus Atlas of the Ear Science Institute (ESIA) gave an overview of ESIA housed on the third floor, which was followed by a lecture and panel discussion at the Perkins Institute hosted by Norman Swan entitled “Personalised medicine-hype versus reality”. Professor Sue Fletcher participated in this panel after her keynote talk earlier in the day entitled “Invention, drug development and the uncertain path to commercialisation”.

Niall Keegan graduates with MastersNiall Keegan, also of the Molecular Therapies group, graduated in 2017 with his Masters, his thesis titled: “Splicing Behaviour and Exotic Mutations in the Duchenne muscular dystrophy (DMD) Gene”. Mr Keegan successfully devised a novel method to find and sequence breakpoint junctions of seven patient cell lines that had large deletions in their DMD genes and made discoveries to broaden knowledge of DMD mutations

and splicing. Mr Keegan is thankful of the support he received during his studies and wishes to share the following: “Having now graduated, I must express my gratitude to the Enid and Arthur Home Memorial scholarship for providing essential funding to support me and my research throughout the two years of my enrolment. I hope that I will justify their investment in me as my career in science continues.”

Welcome back Professor Frank Mastaglia

Professor Frank Mastaglia came back to the fold in a senior role within the Institute in 2017.

Professor Mastaglia started our clinics decades ago and in that time held various roles including Director of the Institute. Professor Mastaglia’s input is proving instrumental as he continues research projects and provides expertise and experience in strengthening relationships with leading Institutes. Professor Mastaglia’s advice to researchers and students is also invaluable as they prepare research papers and write their theses. Professor Mastaglia continues to be a prolific writer publishing research.

Honours students graduate

Three students from the Institute graduated with Honours at the end of 2017.

Jade Kenna graduated from The University of Western Australia with first class Honours. Her thesis is titled: “Exploring the Combined Effects of Recombinant Tissue Plasminogen Activator (rtPA) and Poly-Arginine Peptide R18D in Cultured Neural Cell Lines”. Ms Kenna has continued on at the Institute commencing her PhD in 2018 in the area of Parkinson’s research.

Maddeson Riley graduated from Notre Dame University also with first class Honours. Her thesis is titled: “Demographic and Clinical Indicators of Impulsivity in an Australian Cohort of Parkinson’s Disease Patients”. Ms Riley commenced a medical degree in Sydney in 2018, also at Notre Dame.

Gabrielle Shepherd graduated from Notre Dame University with her thesis titled: “Determining the neuroprotective efficacy of R18D in an in vitro TBI mechanical injury model”.35 36

12. Restoration of Cftr Function by Antisense Oligonucleotide Splicing Modulation Oren Y, Tur-Sinai MI, Ozeri-Galai E, Avizur O, Mutyam V, Wilton SD, Rowe SM, Kerem B 2017 Pediatric Pulmonology 52: S313-S313

13. Response to “Railroading at the FDA” Muntoni F, Fletcher S and Wilton SD 2017 Nat Biotechnol 35: 207-209

14. Corrigendum: Response to “Railroading at the FDA” Muntoni F, Fletcher S, Wilton SD 2017 Nat Biotechnol 35: 481

15. Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction Mishra MK, Loro E, Sengupta K, Wilton SD, Khurana TS 2017 PLoS One 12: e0182676

16. Translational development of splice-modifying antisense oligomers Fletcher S, Bellgard MI, Price L, Akkari AP, Wilton SD 2017 Expert Opin Biol Ther 17: 15-30

17. A Dominant-Negative COL6A1 Pseudoexon Insertion Is Skippable Using Splice-Modulating Oligonucleotides Bolduc V, Foley AR, Donkervoort S, Hu Y, Cummings BB, Lek M, Sarathy A, Sizov K, Degefa HS, Wagener R, Hennig GW, Hanssen E, Lamande SR, Muntoni F, Wilton SD, Macarthur DG, Bonnemann CG 2017 Molecular Therapy 25: 119-120

18. A common dominant-negative COL6A1 pseudo-exon insertion is skippable using splice-modulating oligonucleotides Bolduc V, Foley A, Donkervoort S, Hu Y, Cummings B, Lek M, Sarathy A, Sizov K, Degefa H, Wagener R, Hennig G, Hanssen E, Lamande S, Muntoni F, Wilton SD, Macarthur DG, Bonnemann C 2017 Neuromuscular Disorders 27: S177-S177

19. Comprehending the Health Informatics Spectrum: Grappling with System Entropy and Advancing Quality Clinical Research Bellgard MI, Chartres N, Watts GF, Wilton SD, Fletcher S, Hunter A, Snelling T 2017 Front Public Health 5: 224

20. Serum ferritin and sleep disturbance associate with high impulsivity in male Parkinson’s disease patients Anderton RS, Byrnes M, Jefferson A, Walters S, Ghosh S, Stell, R, Mastaglia FL 2017 American J Psych Neurosci 5(4), 45-52

21. Lack of evidence for decreased protein stability in the 2397 (Met) haplotype of the leucine rich repeat kinase 2 protein implicated in Parkinson’s disease Anderton RS, Hill L, Morris R, Mastaglia FL, Greene WK, Boulos S 2017 Advances in Parkinson’s Disease 6(4), 113-123

22. Extended “Timed Up and Go” assessment as a clinical indicator of cognitive state in Parkinson’s disease Evans T, Jefferson A, Byrnes M, Walters S, Ghosh S, Mastaglia FL, Power B, Anderton RS 2017 J Neurological Sciences 375, 86-91

23. Repeated Mild Traumatic Brain Injury in Female Rats Increases Lipid Peroxidation in Neurons Yates NJ, Lydiard S, Fehily B, Bartlett CA, Fitzgerald M 2017 Experimental Brain Research 235(7), 2133-2149

24. Limiting oxidative stress following neurotrauma with a combination of ion channel inhibitors Milbourn HR, Toomey LM, Gavriel N, Gray CGG, Gough AH, Fehily B, Giacci MK, Fitzgerald M 2017 Discovery Medicine 23 (129): 361-369

25. Repeated mild traumatic brain injury: potential mechanisms of damage Fehily B, Fitzgerald M 2017 Cell Transplantation Vol 26(7) 1131-1155

Scientific Publications

1. Neuroprotective efficacy of R18 poly-arginine and NA-1 (TAT-NR2B9c) peptides following transient middle cerebral artery occlusion in the rat Milani, D, Cross, JL, Anderton, RS, Blacker, DJ, Knuckey, NW, Meloni, BP 2017 Neuroscience Research 114:9-15

2. Characterisation of neuroprotective efficacy of modified poly-arginine-9 (R9) peptides in neuronal glutamic acid excitotoxicity model Edwards AB, Cross JL, Anderton RS, Knuckey NW, Meloni BP 2017 Mol Cell Biochem 426:75-85

3. The neuroprotective peptide poly-arginine-12 (R12) reduces cell surface levels of NMDA NR2B receptor subunit in cortical neurons; investigation into the involvement of endocytic mechanisms MacDougall G, Anderton RS, Edwards AB, Knuckey NW, Meloni BP 2017 Journal of Molecular Neuroscience 61:235-246

4. Peptide pharmacological approaches to treating traumatic brain injury: a case for arginine-rich peptides Chiu LS, Anderton RS, Knuckey NW, Meloni BP 2017 Molecular Neurobiology. DOI 10.1007/s12035-016-0287-3

5. Assessment of the neuroprotective effects of arginine-rich protamine peptides, poly-arginine peptides (R12-cylic, R22) and arginine-tryptophan containing peptides following in vitro excitotoxicity and/or permanent middle cerebral artery occlusion in rats Meloni BP, Milani D, Cross JL, Clark VW, Edwards AB, Anderton RS, Blacker DJ, Knuckey NW 2017 Neuromolecular Medicine 19: 271-285

6. Modification to the Rice-Vannucci perinatal hypoxic-ischaemic encephalopathy model in the P7 rat improves the reliability of cerebral infarct development after 48 hours Edwards AB, Feindel KW, Cross JL, Anderton RS, Clark VW, Knuckey NW, Meloni BP 2017 Journal of Neuroscience Methods 288:62-71

7. Delayed 2-hour post-stroke administration of R18 and NA-1 (TAT-NR2B9c) peptides after permanent and/or transient middle cerebral artery occlusion in the rat Milani D, Cross JL, Anderton RS, Blacker DJ, Knuckey NW, Meloni BP 2017 Brain Research Bulletin 135:62-68

8. Assessment of R18, COG1410, and APP96-110 in excitotoxicity and traumatic brain injury Chiu LS, Anderton RS, Cross JL, Clark VW, Edwards AB, Knuckey NW, Meloni BP 2017 Translational Neuroscience 15; 8:147-157

9. Pathophysiology and Neuroprotective Strategies in Hypoxic-Ischemic. Brain Injury and Stroke Meloni BP 2017 Editorial, Brain Sciences 7, 110; doi:10.3390/brainsci7080110

10. Inherited retinal disease therapy targeting precursor messenger ribonucleic acid Huang D, Fletcher S, Wilton SD, McLenachan S, Mackey DA, Chen FK Vision (2017) in press

11. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM 2017 Journal Neuromuscul Dis 4: 199-207

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35. The autoimmune risk gene ZMIZ1 is a vitamin D responsive marker of a molecular phenotype of multiple sclerosis Fewings NL, Gatt PN, McKay FC, Parnell GP, Schibeci SD, Edwards J, Basuki MA, Goldinger A, Fabis-Pedrini MJ, Kermode AG, Manrique CP, McCauley JL, Nickles D, Baranzini SE, Burke T, Vucic S, Stewart GJ, Booth DR J Autoimmun. 2017 Mar;78:57-69. doi:10.1016/j.jaut.2016.12.006

36. Narrowband UVB Phototherapy for Clinically Isolated Syndrome: A Trial to Deliver the Benefits of Vitamin D and Other UVB-Induced Molecules Hart PH, Lucas RM, Booth DR, Carroll WM, Nolan D, Cole JM, Jones AP, Kermode AG Front Immunol. 2017 Jan 24;8:3. doi: 10.3389/fimmu.2017.00003. eCollection 2017

37. Azathioprine therapy in a case of pediatric multiple sclerosis that was seropositive for MOG-IgG Zhou Y, Huang Q, Lu T, Sun X, Fang L, Lu Z, Hu X, Kermode A, Qiu W J Clin Neurosci. 2017 Apr;38:71-73. doi:10.1016/j.jocn.2016.12.022. Epub 2017 Jan 19

38. Altered regulatory T-cell fractions and Helios expression in clinically isolated syndrome: clues to the development of multiple sclerosis Jones AP, Trend S, Byrne SN, Fabis-Pedrini MJ, Geldenhuys S, Nolan D, Booth DR, Carroll WM, Lucas RM, Kermode AG, Hart PH Clin Transl Immunology. 2017 May 26;6(5):e143. doi: 10.1038/cti.2017.18. eCollection 2017 May

39. Evolving Identification of Blood Cells Associated with Clinically Isolated Syndrome: Importance of Time since Clinical Presentation and Diagnostic MRI Trend S, Jones AP, Geldenhuys S, Byrne SN, Fabis-Pedrini MJ, Nolan D, Booth DR, Carroll WM, Lucas RM, Kermode AG, Hart PH Int J Mol Sci. 2017 Jun 15;18(6). pii: E1277. doi: 10.3390/ijms18061277

40. Cladribine versus fingolimod, natalizumab and interferon for multiple sclerosis Kalincik T, Jokubaitis V, Spelman T, Horakova D, Havrdova E, Trojano M, Lechner-Scott J, Lugaresi A, Prat A, Girard M, Duquette P, Grammond P, Solaro C, Grand’Maison F, Hupperts R, Prevost J, Sola P, Ferraro D, Terzi M, Butler E, Slee M, Kermode A, Fabis-Pedrini M, McCombe P, Barnett M, Shaw C, Hodgkinson S, Butzkueven H; MSBase Study Group Mult Scler. 2017 Aug 1:1352458517728812.doi: 10.1177/1352458517728812. (WMC is a member of MSBase)

41. Data characterizing the ZMIZ1 molecular phenotype of multiple sclerosis Fewings N, Gatt PN, McKay FC, Parnell GP, Schibeci SD, Edwards J, Basuki MA, Goldinger A, Fabis-Pedrini MJ, Kermode AG, Manrique CP, McCauley JL, Nickles D, Baranzini SE, Burke T, Vucic S, Stewart GJ, Booth DR. Data Brief. 2017 Feb 21;11:364-370. doi: 10.1016/j.dib.2017.02.040

42. Fabry heterozygote mimicking multiple sclerosis Yau WY, Fabis-Pedrini MJ, Kermode AG BMJ Case Rep. 2017 Jun 2;2017. pii: bcr-2017-220274. doi: 10.1136/bcr-2017-220274

43. Infections in neuromyelitis optica spectrum disorder Zhong X, Zhou Y, Lu T, Wang Z, Fang L, Peng L, Kermode AG, Qiu W J Clin Neurosci. 2018 Jan;47:14-19. doi: 10.1016/j.jocn.2017.10.005

44. Association of serum gamma-glutamyltransferase and C-reactive proteins with neuromyelitis optica and multiple sclerosis Shu Y, Li R, Qiu W, Chang Y, Sun X, Fang L, Chen C, Yang Y, Lu Z, Hu X, Kermode AG Mult Scler Relat Disord. 2017 Nov;18:65-70. doi: 10.1016/j.msard.2017.09.021

45. Serum CCL20 and its association with SIRT1 activity in multiple sclerosis patients Li R, Sun X, Shu Y, Wang Y, Xiao L, Wang Z, Hu X, Kermode AG, Qiu W J Neuroimmunol. 2017 Dec 15;313:56-60. doi: 10.1016/j.jneuroim.2017.10.013

46. Ectrodactyly in a Chinese patient born to a mother with neuromyelitis optica spectrum disorder Chang Y, Shu Y, Sun X, Xu C, He D, Fang L, Chen C, Hu X, Kermode A, Qiu W Mult Scler Relat Disord. 2017 Nov 8;19:70-72. doi:10.1016/j.msard.2017.11.009

26. Preferential enrichment of a range of proteins distinguishes the corona of PEGylated poly (glycidyl methacrylate) nanoparticles Naidu PSR, Norret M, Smith NM, Dunlop SA, Taylor NL, Fitzgerald M, Iyer KS 2017 Langmuir 33(45) DOI 10.1021/acs.langmuir.7b02568

27. Resistance wheel exercise from mid-life has minimal effect on sciatic nerves from old mice in which sarcopenia was prevented Krishnan VS, Zoe White Z, Terrill JR, Hodgetts SI, Fitzgerald M, Shavlakadze T, Harvey AR, et al 2017 Biogerontology 18:769-790

28. Specific ion channels contribute to key elements of pathology during secondary degeneration following neurotrauma O’Hare Doig RL, Chiha W, Giacci MK, Yates NJ, Bartlett CA, Smith NM, Hodgetts SI, Harvey AR, Fitzgerald M 2017 BMC Neurosci 18:62

29. Contribution of different relapse phenotypes to disability in multiple sclerosis Stewart T, Spelman T, Havrdova E, Horakova D, Trojano M, Izquierdo G, Duquette P, Girard M, Prat A, Lugaresi A, Grand’Maison F, Grammond P, Sola P, Shaygannejad V, Hupperts R, Alroughani R, Oreja-Guevara C, Pucci E, Boz C, Lechner-Scott J, Bergamaschi R, Van Pesch V, Iuliano G, Ramo C, Taylor B, Slee M, Spitaleri

D, Granella F, Verheul F, McCombe P, Hodgkinson S, Amato MP, Vucic S, Gray O, Cristiano E, Barnett M, Sanchez Menoyo JL, van Munster E, Saladino ML, Olascoaga J, Prevost J, Deri N, Shaw C, Singhal B, Moore F, Rozsa C, Shuey N, Skibina O, Kister I, Petkovska-Boskova T, Ampapa R, Kermode A, Butzkueven H, Jokubaitis V, Kalincik T; MSBase Study Group Mult Scler 2017 Feb;23(2):266-276. doi:10.1177/1352458516643392. Epub 2016 Jul 11

30. Lymphocyte reconstitution following autologous stem cell transplantation for progressive MS Cull G, Hall D, Fabis-Pedrini MJ, Carroll WM, Forster L, Robins F, Ghassemifar R, Crosbie C, Walters S, James I, Augustson B, Kermode AK Mult Scler J Exp Transl Clin. 2017 Mar 23;3(1):2055217317700167. doi: 10.1177/2055217317700167. eCollection 2017 Jan-Mar

31. Incidence and prevalence of NMOSD in Australia and New Zealand Bukhari W, Prain KM, Waters P, Woodhall M, O’Gorman CM, Clarke L, Silvestrini RA, Bundell CS, Abernethy D, Bhuta S, Blum S, Boggild M, Boundy K, Brew BJ, Brown M, Brownlee WJ, Butzkueven H, Carroll WM, Chen C, Coulthard A, Dale RC, Das C, Dear K, Fabis-Pedrini MJ, Fulcher D, Gillis D, Hawke S, Heard R, Henderson APD, Heshmat S, Hodgkinson S, Jimenez-Sanchez S, Killpatrick T, King J, Kneebone C,

Kornberg AJ, Lechner-Scott J, Lin MW, Lynch C, Macdonell R, Mason DF, McCombe PA, Pender MP, Pereira JA, Pollard JD, Reddel SW, Shaw C, Spies J, Stankovich J, Sutton I, Vucic S, Walsh M, Wong RC, Yiu EM, Barnett MH, Kermode AG, Marriott MP, Parratt JDE, Slee M, Taylor BV, Willoughby E, Wilson RJ, Vincent A, Broadley SA J Neurol Neurosurg Psychiatry 2017 Aug;88(8):632-638. doi: 10.1136/jnnp-2016-314839

32. CADASIL mimicking multiple sclerosis: The importance of clinical and MRI red flags Joshi S, Yau W, Kermode A J Clin Neurosci. 2017 Jan;35:75-77. doi: 10.1016/j.jocn.2016.09.025. Epub 2016 Oct 20

33. Circulating immune cells in multiple sclerosis Jones AP, Kermode AG, Lucas RM, Carroll WM, Nolan D, Hart PH Clin Exp Immunol. 2017 Feb;187(2):193-203. doi: 10.1111/cei.12878

34. Pregnancy in neuromyelitis optica spectrum disorder: A multicenter study from South China Huang Y, Wang Y, Zhou Y, Huang Q, Sun X, Chen C, Fang L, Long Y, Yang H, Wang H, Li C, Lu Z, Hu X, Kermode AG, Qiu W J Neurol Sci. 2017 Jan 15;372:152-156. doi: 10.1016/j.jns.2016.11.054

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60. Antisense oligonucleotide modified with serinol nucleic acid (SNA) induces exon skipping in mdx myotubes Le BT, Murayama K, Shabanpoor F, Asanuma H and Veedu RN RSC Advances, 10, 34049-34052

61. Development of an efficient G-quadruplex stabilized thrombin binding aptamer containing 3-carbon spacer molecule Aaldering LJ, Poongavanam P, Langkjær N, Murugan NA, Jørgensen PT, Wengel J, Veedu RN ChemBioChem, 18(8):755-763

62. Practice Patterns for Neurosurgical Utilization and Outcome in Acute Intracerebral Hemorrhage: Intensive Blood Pressure Reduction in Acute Cerebral Hemorrhage Trials 1 and 2 Studies Guo R, Blacker DJ, Wang X, Arima H, Lavados PM, Lindley RI, Chalmers J, Anderson CS, Robinson T; INTERACT Investigators Neurosurgery 2017 Dec 1; 81(6):980-985 doi: 10.1093/neuros/nyx129

63. In Reply: ORACLE Stroke Study: Opinion Regarding Acceptable Outcome Following Decompressive Hemicraniectomy for Ischemic Stroke Honeybul S, Ho KM, Blacker DJ Neurosurgery 2017 Apr 1; 80(4):237-238 doi: 10.1093/neuros/nyw155. No abstract available

64. Penumbral freeze: travel distance and delays provide an opportunity to study prerecanalization therapy neuroprotection Blacker DJ Future Neurology, 2017 Nov 10:185-188 Published Online 10 Nov 2017 https://doi.org/10.2217/fnl-2017-0025

65. Outcomes of Endovascular Thrombectomy with and without Thrombolysis for Acute Large Artery Ischaemic Stroke at a Tertiary Stroke Centre Wee CK, McAuliffe W, Phatouros CC, Phillips TJ, Blacker D, Singh TP, Baker E, Hankey GJ Cerebrovasc Dis Extra 2017;7(2):95-102 doi: 10.1159/000470855 Epub 2017 May

66. Mechanical thrombectomy for anterior circulation stroke: 5-year experience in a statewide service with differences in pretreatment time metrics across two hospitals sites Kabra R, Phillips TJ, Saw JL, Phatouros CC, Singh TP, Hankey GJ, Blacker D, Ghia D, Prentice D, McAuliffe W J Neurointerv Surg 2017 Jun;9(6):535-540 doi: 10.1136/neurintsurg-2016-012304 Epub 2016 May 9

67. Recognition of giant cell arteritis in patients with polymyalgia rheumatica who have a stroke: a cautionary tale Gutteridge DH, Mastaglia FL Internal Medicine, Vol 47 Issue 10 October 2017, pg 1199-1201

68. Advances in inclusion body myositis: genetics, pathogenesis and clinical aspects Needham M, Mastaglia F Expert Opinion on Orphan Drugs, Volume 5, Issue 5, 4 May 2017, Pages 431-443

69. Serum high-density lipoprotein is associated with better cognitive function in a cross-sectional study of aging women Bates KA, Sohrabi HR, Rainey-Smith SR, Weinborn M, Bucks RS, Rodrigues M, Beilby J, Howard M, Taddei K, Martins G, Paton A, Shah T, Dhaliwal SS, Foster JK, Martins IJ, Lautenschlager NT, Mastaglia FL, Gandy, SE, Martins RN International Journal of Neuroscience, Volume 127, Issue 3, 4 March 2017, Pages 243-252

70. Amplitude Integrated Electroencephalography Compared with Conventional Video EEG for Neonatal Seizure Detection: A Diagnostic Accuracy Study Rakshasbhuvankar A, Rao S, Palumbo L, Ghosh S, Nagarajan L Journal of Child Neurology, Volume 32, Issue 9, 1 August 2017, Pages 815-822

71. Dystonic tics induced by deep brain stimulation of the posterior subthalamic area for essential tremor Chandran AD, Joshi S, Thorburn M, Stell R, Lind CRP Journal of Neurosurgery, Volume 126, Issue 2, February 2017, Pages 386-390

47. Anti-inflammatory disease-modifying treatment and short-term disability progression in SPMS Lorscheider J, Jokubaitis VG, Spelman T, Izquierdo G, Lugaresi A, Havrdova E, Horakova D, Trojano M, Duquette P, Girard M, Prat A, Grand’Maison F, Grammond P, Pucci E, Boz C, Sola P, Ferraro D, Spitaleri D, Lechner-Scott J, Terzi M, Van Pesch V, Iuliano G, Bergamaschi R, Ramo-Tello C, Granella F, Oreja-Guevara C, Butzkueven H, Kalincik T; MSBase Study Group (Kermode AG and Carroll WM are members of MSBase) Neurology. 2017 Sep 5;89(10):1050-1059. doi: 10.1212/WNL.0000000000004330

48. Diagnosis of multiple sclerosis: 2017 revisions of the McDonald criteria Thompson AJ, Banwell BL, Barkhof F, Carroll WM, Coetzee T, Comi G, Correale J, Fazekas F, Filippi M, Freedman MS, Fujihara K, Galetta SL, Hartung HP, Kappos L, Lublin FD, Marrie RA, Miller AE, Miller DH, Montalban X, Mowry EM, Sorensen PS, Tintoré M, Traboulsee AL, Trojano M, Uitdehaag BMJ, Vukusic S, Waubant E, Weinshenker BG, Reingold SC, Cohen JA Lancet Neurol. 2017 Dec 21. pii: S1474-4422(17)30470-2. doi: 10.1016/S1474-4422(17)30470-2

49. The need for a Global Neurology Alliance Carroll WM J Neurol Sci. 2017 Aug 15;379:321-323. doi: 10.1016/j.jns.2017.05.067

50. Rational Design of Short Locked Nucleic Acid-Modified 2’-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro Le BT, Adams AM, Fletcher S, Wilton SD and Veedu RN 2017 Mol Ther Nucleic Acids 9: 155-161

51. Development of cell-specific aptamers: Recent advances and insight into the selection procedures Rahimizadeh K, AlShamaileh H, Fratini M, Chakravarthy M, Stephen M, Shigdar S, Veedu RN Molecules 2017, 22, 2070

52. Nucleic acid-based theranostics for tackling alzheimer’s disease Chakravarthy M, Chen S, Dodd PR, Veedu RN Theranostics 2017, 7, 3933-3947

53. Novel chemically-modified DNAzyme targeting integrin alpha-4 rna transcript as a potential molecule to reduce inflammation in multiple sclerosis Chakravarthy M, Aung-Htut MT, Le BT, Veedu RN Sci Rep 2017, 7, 1613

54. Polyglutamine ataxias: From Clinical and Molecular Features to Current Therapeutic Strategies McIntosh CS, Aung-Htut MT, Fletcher S, Wilton SD Journal of Genetic Syndromes and Gene Therapies 8: 319. doi: 10.4172/2157-7412.1000319

55. Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2’-deoxyuridines nucleotides induce exon-skipping in vitro Le BT, Hornum M, Sharma P, Nielsen P and Veedu RN 2017 RSC Advances 7, 54542

56. Construction of a bivalent thrombin binding aptamer and its antidote with improved properties Hughes Q, Le BT, Gilmore G, Baker R, Veedu RN Molecules, 22(10), 1770

57. Integrative Approaches in HIV‐1 non-nucleoside reverse transcriptase inhibitor design Poongavanam V, Namasivayam V, Vanangamudi M, Shamaileh H, Veedu RN, Kihlberg J, Murugan AN WIREs: Computational Molecular Science, e1328

58. Evaluation of Novel Anti-VEGF Molecules in the Animal Model of Human Lung Cancer Chakrabarti S, Barrow CJ, Kanwar RK, Ramana V, Veedu RN, Kanwar JR Aptamers and Synthetic Antibodies, 3, 74-93

59. Next generation nucleic acid aptamers with two base modified nucleotides improve the binding affinity and potency AlShamaileh H and Veedu RN ChemBioChem, 18:1565-1567

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Engaging the MS Community to Promote Exercise. Yvonne Learmonth; Co-Investigators: Helen Correia, Cath Smith, Allan Kermode, David Hathorn2017 $20,000 MSRA Incubator Grant Murdoch University

Pre-clinical assessment of R18D following strokeMeloni/Blacker/Knuckey2017/18: $250,000. MSWA

Pre-clinical assessment of poly-arginine peptide R18D as a therapeutic to reduce central nervous system injury following neurotraumaMeloni/Knuckey/Hodgetts/Cross2017: $99,650. Neurotrauma Research Program of WA

Establishment of an in vitro TBI model: 2017Anderton /Meloni/Edwards2017: $5,000. Notre Dame University

Precision Medicine for Children with Rare Disorders: Cell & Bank & DatabaseValidating the Efficacy and Safety of a Dual-Gene Therapy Approach for Obesity & Diabetes using Smart-siRNA and Tissue Targeted Bio-responsive Carrier Systems Veedu$378,352. Preveceutical Medical Inc

Aptamer Molecule development to an Isolated Purified Protein and to a Synthesized PeptideVeedu $405,028. George Bautovich (Royal Northshore Hospital, Sydney)

Grants

Splice correction as a treatment for rare diseasesWilton SD, Fletcher S, Bellgard MI, Goldblatt J2015 – 2018 $792,656 NHMRC Grant ID: 1086311

An integrated platform connecting registries, biobanks and clinical bioinformatics for rare disease research. Bellgard M, Nolan D, Mallal S, Wilton S, Fletcher S, Kermode A2013-2017 $989,884 NHMRC RD-CONNECT Murdoch University

RAGE and ACE2 shedding as therapeutic targets in diabetes and cardiovascular diseaseThomas M, Jandeleit-Dahm K, Wilton SD, Tikellis C2017-2019 $468,447 NHMRC Grant ID: APP1127526

Establishing a centre of research excellence in juvenile macular disease.Chen F, De Roach J, Hunt D, Wilton S, McLaren T, Thompson J, McLenachan S, Carvalho L, Mellough C, Frost S, Xiao D, Alonso-Caneiro D, Sampson D, Clark A, Carroll R2017-2019 $249,880 Telethon – Perth Children’s Hospital Research Fund

Characterisation of Dux 4 protein - protein interactions in FSHD cell lines and tissue biopsies by cross-linking and mass-spectrometryKalisman N, Wilton S, Coppee F2017-2019 $300,000 FSHD Global Research Foundation

Ground breaking research into Muscular DystrophyWilton SD2016-2018 $154,000 Channel 7 Telethon Trust

Mapping redundant exons through the generation of animal models of human diseaseWilton S, Fletcher S2017-2018 $25,875 Perron Institute for Neurological and Translational Science

In vivo validation of a novel treatment for Multiple SclerosisWilton S, Aung-Htut, Fletcher S2017-2018 $30,000 Perron Institute for Neurological and Translational Science

Sponsored Research Projects – Research Project PlansWilton SD, Fletcher S2015 – 2019 Sarepta Therapeutics Sponsored Collaborative Research Agreement

Targeting early multiple sclerosis with UVB phototherapy. Prue Hart, David Booth, Allan Kermode, David Nolan, William (Bill) Carroll, Judy Cole2014-2017 $660,558 NHMRC Project Grant University of Western Australia

Exploring epigenetic and environmental contributors to and modifiers of multiple sclerosis. Marzena Fabis-Pedrini2016-2018 $396,401 MSWA Post-Doctoral Research Fellowship Perron Institute for Neurological and Translational Research

Do MS drugs make a difference to the long-term outlook for people with MS? Investigator: Bruce Taylor; Co-Investigators: Ingrid van der Mei, Andrew Palmer, Christopher Blizzard, Helmut Butzkueven, Steve Simpson Jr, Tomas Kalincik, William (Bill) Carroll, Deborah Mason2016-2017 $180,000 MSRA Project Grant Menzies Institute for Medical Research

Enhancing the cognitive functioning of adults with multiple sclerosis: Integrated cognitive and psychosocial therapy. Michelle Byrnes, Allan Kermode, Jason Burton2016-2017 $100,237 MSWA Project Grant Perron Institute for Neurological and Translational Research43 44

2017 $

2016 $

TOTAL INCOME 6,742,327 7,154,665

TOTAL EXPENSES 4,338,011 5,862,568

NET PROFIT 2,404,316 1,292,096

Profit and Loss

Financial Report Perron Institute for Neurological and Translational Science A.B.N. 99 070 870 398

Statement of financial position As at 31 December 2017Extract from Audited Financial Statements 2017

2017 $

2016 $Current Assets

Cash assets 5,936,533 2,302,308Receivables 458,224 1,335,675Total Current Assets 6,394,757 3,637,983

Non-Current Assets

Investments 2,073,571 951,572Property, plant & equipment 817,232 101,087Total Non-Current Assets 2,890,803 1,052,659TOTAL ASSETS 9,285,560 4,690,642

Current Liabilities

Employee benefits 62,570 21,718Trade and other payables 3,028,532 915,073Total Current Liabilities 3,091,102 936,791

Non-Current Liabilities

Leasing Liability 35,116 30,188Total Non-Current Liabilities 35,116 30,188TOTAL LIABILITIES 3,126,218 966,979

Net Assets 6,159,342 3,723,663EquityRetained earnings 6,159,342 3,723,663

TOTAL EQUITY 6,159,342 3,723,663

Thank you

The Perron Institute wishes to thank you, our supporters and donors for your generosity in assisting the Institute by continuing to provide hope and a better quality of life for people with neuromuscular and neurological conditions in this exciting time of expansion.

Financial Statements

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For more information see: perroninstitute.org

Perron Institute for Neurological and Translational Science

Ralph & Patricia Sarich Neuroscience Research Institute 8 Verdun St | Queen Elizabeth II Medical CentreNedlands | Western Australia 6009

Phone + 61 (08) 6457 0312 Fax + 61 (08) 6457 0281Email info@perron.uwa.edu.au

ACN: 070 870 398

For support opportunities please contact Jim Murphy, Fundraising and Business DevelopmentMobile +61 (0) 416 273 277 Email jim.murphy@perron.uwa.edu.au

Media contactTennille Kroemer Communications and Marketing ManagerMobile + 61 (0) 426 044 223 Email tennille.kroemer@perron.uwa.edu.au

Published: May 2018.

Our overall logo design is based on collaboration. The two interlocking arms create a feeling of community, care and compassion.

The circle in the middle of our logo characterises our core focus of neuroscience.

The two parts also represent what makes us unique in WA, our laboratory research and our treatment clinics that symbiotically work together to create a whole. The whole is greater than the sum of its parts.

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