Inspiration REPORT - YEARS
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YEARS inspiration REPORT 2021-2022
Table of Contents
Our legend 4
Our story 6
Our journey so far 8
Our Board 10
Our inspiring research leaders 12
Our compassionate clinical leaders 16
Our research collaboration 20
Our clinics 21
Our links 22
Our supporters 24
Our inspiration 26
Our impact 30
Our commercialisation 38
We acknowledge the traditional
custodians of this land, the Whadjuk Our future 42
people of the Noongar nation, Our request to you 46
and pay respects to their Elders
Our gratitude 47
past and present.
4 Perron Institute Inspiration Report 5
After a lifetime of research in
neuropathology, I am proud the
Institute I started in 1982 turns
40 this year! We have made
some incredible discoveries, and
I am sure we’ll make many more.
Thank you to everyone who has
been a part of our journey so far.
I look forward to what the future
brings – including developing
new treatments for devastating
conditions which continue to
affect millions of people around
legend
the world.
Emeritus Professor Byron Kakulas AO
Founder, Perron Institute
Emeritus Professor Byron Kakulas AO
Founder, Perron Institute
6 Perron Institute Inspiration Report 7
Our story
Since its inception, the Perron Institute for Neurological and of genomics, using genetic
data to inform and personalise
Translational Science has built an international reputation as an diagnostics and therapies.
My father, the late Stan
innovative and collaborative centre of excellence for medical research. Our goal is to continue to
build on our globally relevant Perron AC, first met Professor
research capacity, nurturing and Byron Kakulas as a result of Dad’s
The Institute celebrates its 40th discoveries and treatments for and partners, from over 450 supporting our research teams,
anniversary in 2022 as Western the community’s benefit. research institutes, hospitals and
involvement with his local Rotary
widening the scope of research
Australia’s longest established universities worldwide. and developing collaborations Club and was inspired to become a
Our values are integrity, respect,
medical research organisation.
We have opened new pathways,
that generate and efficiently significant supporter on hearing of
responsibility, excellence and
translate research evidence for
We’re proud to mark this community. They guide our twice leading the world in his pioneering research on Duchenne
health benefit.
anniversary as an important activities and reflect those held research breakthroughs, muscular dystrophy. Dad’s personal
opportunity to acknowledge our by our highly respected and beginning with the remarkable Our patrons support was to become a lifelong
achievements and recognise longstanding supporters, the discovery by our founder
the contributions of the many late Stan Perron AC and his Emeritus Professor Byron commitment and was extended
talented, inspiring and dedicated family. Kakalus AO that degenerated when he established the Stan
people who are part of the muscle could regenerate in the Perron Charitable Foundation in
Perron Institute story — our Our purpose Rottnest Island quokka.
1978. The work of what is now the
leaders, our researchers and
The Institute’s core purpose
clinicians, our support teams,
Building on this quantum leap, Perron Institute was supported by
is translational medicine, the Professors Steve Wilton AO and
our benefactors and our
transfer of new understanding Sue Fletcher AO and their team
Dad because it aligned with the key
partnering organisations.
of disease mechanisms gained developed three drugs approved purpose of the Foundation, which
Integral to this, are the in the laboratory, new methods by the United States Food and is to help people in need, especially
contributions of those who of diagnosis, treatment and Drug Administration to slow
children battling chronic disease and
participate in our research prevention. The end goal is to the progression of Duchenne
projects and clinical trials. provide better quality of life and muscular dystrophy in boys and at severe disadvantage.
improve clinical outcomes for young men suffering from this
Together we have gone forward millions of people around the genetic disease. FDA approvals As Chair of the Foundation, it is my
to find answers to complex The Honourable Kim Beazley AC
world suffering from devastating are very hard to achieve. It’s Governor of Western Australia privilege to continue and extend
neurological disorders and neurological conditions. an incredible achievement in a
translate research outcomes into Dad’s philanthropy and it is my great
researcher’s lifetime, an outcome
improved diagnostics and novel Solid foundations very few experience. pleasure to congratulate Emeritus
therapies. There have been and
of discovery and Professor Kakulas AO and everyone
continue to be many challenges, Connecting our researchers with
but our successes, for example innovation med-tech focused entrepreneurs at the Perron Institute on this
world first therapies for Duchenne With our focus on unlocking to make the complex journey momentous achievement.
muscular dystrophy, have been mysteries in the neurosciences to commercialisation has been
Elizabeth Perron
significant, widening horizons and field and taking our discoveries integral to this success.
Chair, Stan Perron Charitable Foundation
improving the lives of many. from ‘bench to bedside’, the
Perron Institute is helping to
A new frontier
Our vision shape the future of health care. As part of the next phase of our
To positively change lives and Our clinicians and scientists work evolution, we are expanding the
make a difference by continuing collaboratively within a global research platforms developed
to drive our research translation network of over 900 researchers over decades and we are active
forward and develop new and health professionals in the rapidly advancing area Professor Lyn Beazley AO FTSE
8 Perron Institute Inspiration Report 9
Our journey so far
As the first medical research institute in Western Australia, we are New Research Chair
in Palliative Care
delighted to be celebrating our 40th Anniversary in 2022. Professor Samar Aoun
became Perron Institute
Research Chair in Palliative
Emeritus Professor Perron Institute The Institute takes Centre for Care at UWA.
Byron Kakulas AO contributed to the on the Perron name Molecular Medicine Sarich family donates
Emeritus Professor discovery of the In 2017, we proudly and Innovative significant funding
Byron Kakulas AO, SOD1 MND gene became the Perron Therapeutics $1million donated to research,
The Quokka Story then Professor of Location Institute for Neurological opening
Researchers at the including Bryant Stokes
Revolutionary discovery Neuropathology at the established Perron Institute and Translational Science This joint research Research Fund.
by Emeritus Professor University of Western at SCGH (then the Australian in recognition of Mr initiative between
Byron Kakulas AO that Australia, in partnership The WA Government Neuromuscular Research Stan Perron AC and his Murdoch University and
New Research Chair
degenerated muscle with a group of parents indicated that it was family’s long-standing
Institute) played a the Perron Institute, the in Stroke
tissue in the Rottnest whose children suffered prepared to make significant part in philanthropic support. Centre for Molecular
Island quokka could be Professor Graeme Hankey
from muscular dystrophy space available in the discovering the first Medicine and Innovative
regenerated. Moved into our became the Perron Institute
took the bold decision old Sir Charles Gairdner gene (SOD1) associated Therapeutics (CMMIT), Chair in Stroke Research
to establish the Muscular Hospital building at with motor neurone new building brings together at UWA. A cornerstone for
Dystrophy Association the Queen Elizabeth disease in collaboration Moved into the Ralph researchers and the development of a Centre
of Western Australia, II Medical Centre in with researchers in the & Patricia Sarich specialist clinicians from of Excellence in Stroke.
now Muscular Dystrophy Nedlands. United States, Canada Neuroscience Institute a range of disciplines
Western Australia. and Europe. Building to bring with a common focus Byron Kakulas Annual
neuroscience groups on developing precision Medal and Lecture
together. medicine solutions. An inaugural signature event.
1965 1971 1982 2016 2019 2021
1960 1967 1978 1993 2017 2019 2022
Inclusion Body Official Second drug Third drug for
Myositis identified establishment for Duchenne Duchenne achieves
as a clinical of ANRI achieves US US FDA accelerated
identity In 1982, the Australian FDA accelerated approval
Major International Neuromuscular Research
First Duchenne approval
Inclusion body myositis The third treatment for
[IBM] was distinguished
Congress on Institute was established drug receives Duchenne muscular
Developed by Professors
from polymyositis in a Muscle Diseases with Professor Kakulas accelerated Sue Fletcher AO and dystrophy developed by our
biopsy examined by Perth hosted a major as its Founding Director. approval from the Steve Wilton AO at the WA research team led by
Professor Byron Kakulas international congress on US FDA Perron Institute via The Professors Steve Wilton AO
AO in 1965. Neurologist muscle diseases where A ground-breaking drug University of Western and Sue Fletcher AO, received
Dr Frederick Samaha the idea of establishing developed by Professors Australia, Vyondys accelerated approval by the
went on to clearly define a neuromuscular Steve Wilton AO and 53 is the second exon United States Food and Drug
the entity which was research institute in Sue Fletcher AO to treat skipping therapy for Administration (US FDA).
later elucidated via Perth was born. Duchenne muscular Duchenne muscular
electron microscopy by dystrophy received dystrophy to receive Argenica Therapeutics
neurologist Dr Sam Chou. accelerated approval from accelerated approval by lists on ASX
the US Food and Drug the United States Food The first listing of intellectual
Administration. The first and Drug Administration. property from Perron Institute
of three Duchenne drugs and UWA to drive stroke
approved by the US FDA. discoveries forward.
10 Perron Institute Inspiration Report 11
Our Board
Our Board members
are collectively proud
of the Perron Institute’s
achievements over many
Rob McKenzie Abigail Davies Ian Green Torsten Ketelsen OAM
years. We are grateful to Board Chair Board Director Board Director Board Director
(appointed May 2022)
our Board members, who
volunteer their time, and
for the range of expertise
they provide.
Steve Arnott Professor Norman Palmer Dr Rebekah Puls Jo Wilkie
CEO Board Director Board Director, Chair of the Board Director
Research Advisory Committee
generosity Dr James Williams
Board Director
Professor Stephen Wilton AO
Board Director
12 Perron Institute Inspiration Report 13
Our
Our research team leaders are
inspiring
determined to deliver positive
outcomes for people living with
research
leaders
Dr May Aung-Htut Professor Minghao Zheng Professor Samar Aoun
neurological and neuromuscular Molecular Therapies Research Brain and Bone Axis Research Palliative Care Research
conditions. Their commitment,
dedication and expertise are The group led by Professor The group is exploring the The focus of Professor Aoun
We are a dynamic, Wilton and Dr May Aung-Htut at interaction between the brain and her group is on a public
something to behold and this is the Centre for Molecular Medicine and bones, in particular the health approach to palliative
multidisciplinary institute and Therapeutics (CMMIT), relationship between bone care, viewing the community
going to make a difference.
for neurological research Murdoch University, develops
treatments for inherited and/
fractures and the progression of
dementia. The aim is to develop
as an equal partner with health
service providers in ensuring
Research is a 24/7 undertaking. and translation. We or acquired diseases, applying treatments for age-related quality care throughout the
the successful genetic ‘patching’ disorders such as Alzheimer’s end-of-life journey.
You need passion, perseverance conduct research across a technology created for Duchenne disease, other forms of
and tenacity to succeed, along broad range of conditions muscular dystrophy. dementia and osteoporosis.
with a little luck and a lot of such as stroke, Parkinson’s,
community support. motor neurone disease,
multiple sclerosis and rare
Professor Steve Wilton AO
Perron Institute Director muscle diseases such
as Duchenne muscular
dystrophy and myositis.
We are proud of our successful record in
research translation – transforming scientific
Associate Professor Rakesh Veedu Professor Bruno Meloni Professor Merrilee Needham
discoveries into clinical treatments for people
in the community. Nucleic Acid Therapeutics Research Stroke Research Myositis Research
See our research groups The laboratory team led by The core focus of the group Led by Professor Needham,
and their leaders. Associate Professor Veedu based led by Clinical Professor Neville the group (based at CMMIT,
leadership
at CMMIT, Murdoch University Knuckey and Professor Meloni, Murdoch University) has a
focuses on developing therapeutic is to develop early intervention longstanding dedication to the
and diagnostic technologies neuroprotective treatments to investigation of clinical, genetic
designed for the treatment of minimise brain damage after and immunological aspects of the
various inherited/genetic and stroke and other neurological inflammatory myopathies, with a
acquired diseases such as macular disorders causing brain injury. focus on Inclusion Body Myositis,
degeneration, Type-2 diabetes, Argenica Therapeutics Ltd is the most common muscle disease
psoriasis and liver cancer. driving this forward. associated with ageing.
14 Perron Institute Inspiration Report 15
Professor Lindy Fitzgerald Professor Allan Kermode Professor Soumya Ghosh Professor Sulev Koks Dr Athanasios Gaitatzis Dr Sarah Rea
Neurotrauma Research Demyelinating Diseases Research Restorative Neurology Research Genetic Epidemiology Research Epilepsy Research Functional Genomics Research
The group led by Professor The team, led by Clinical Led by Clinical Professor Ghosh The team led by Professor The aims of the group led Dr Rea’s group, based at
Fitzgerald (Curtin University Professor Kermode (Perron and Professor Frank Mastaglia AM, Koks (CMMIT and Perron by consultant neurologist CMMIT, is researching the
and Perron Institute) seeks to Institute and CMMIT), is the group based at Perron Institute Institute) aims to understand Dr Gaitatzis (Perron Institute underlying mechanisms that
understand whether oxidative dedicated to investigating the uses advanced methodologies, how genetic factors interact and Sir Charles Gairdner cause neurodegeneration
damage to certain cells is a causes of multiple sclerosis (MS) including balance assessment and with environmental factors Hospital) are to understand the and bone disease, particularly
feature that unifies neurotrauma, and improving the treatment therapy, pulmonary function and in Parkinson’s and motor factors that affect the clinical motor neurone disease and
neurodegenerative and and management of people with therapy and non-invasive brain neurone disease. The goal is to expression of epilepsy and its frontotemporal degeneration.
demyelinating diseases, and to MS and related diseases. This stimulation to develop treatments use genomic technologies in response to treatment, and to The group is also investigating
identify potential targets for early includes study of the influence for Parkinson’s and other longitudinal studies as predictors identify biomarkers and develop the functional impacts of disease-
preventative treatments. of immune-related genes. neurological conditions. for neurodegenerative personalised treatments. associated gene changes on
outcomes. neurons and bone cells.
Associate Professor Jenny Rodger Associate Professor Stuart Hodgetts Professor Anthony Akkari
Brain Plasticity Research Spinal Cord Repair Research Motor Neurone Disease
Genetics and Therapeutics Research
inspire
The group led by MSWA Dr Hodgetts’ objective at The group led by Professor
Senior Research Fellow and UWA and Perron Institute is Akkari (CMMIT and Perron
A/Professor Rodger (UWA and to develop clinically relevant Institute), aims to identify genetic
Perron Institute) is developing strategies for the treatment mechanisms and mutations
interventions that promote of spinal cord injury resulting in neurological conditions,
healthy brain development and from trauma (falls, sporting and particularly motor neurone
function, as well as repairing road accidents) and applying disease. The goal is to develop
damaged brains after injury and these strategies towards other personalised treatments based
non-invasively treating mental medical conditions such as on the successful antisense
illness, including depression. stroke and brain injury. oligonucleotide technology.
Pictured right: Emeritus Professor Byron Kakulas AO and Abbey Figliomeni, PhD student and Kakulas Scholarship recipient
16 Perron Institute Inspiration Report 17
Our compassionate
clinical leaders
Our on-site treatment clinics, led by Manager Clinical Services
Sue Walters, are among the Perron Institute’s key strengths. Clinical Professor David Blacker Clinical Professor William Carroll Dr Rajini (Sivarajini) Rajanayagam
Stroke Clinic and Research Multiple Sclerosis Clinic and Research Movement Disorders, MND and
MS Clinic and Research
In the late 1980s, Professor An arrangement with Sir Charles Neurone Disease, Neuromuscular
Frank (Francis) Mastaglia was Gairdner Hospital enables the Conditions and Epilepsy. Clinical Professor David Blacker, Professor William (Bill) Carroll Consultant neurologist Dr
invited by Professor Kakulas to institute to offer these specialised Perron Institute’s Medical AM was appointed President Rajini Rajanayagam provides
clinical services. Our Medical Director is Clinical Director and clinical neurologist, of the World Federation of diagnostic and treatment-
join the Institute and establish
Professor David Blacker. specialises in the diagnosis and Neurology in 2018. He is a based services in her various
a Clinical Research Program
Our clinic team includes management of stroke, provides multiple sclerosis clinician clinics. Patients have access to
which he developed with
Associate Professor Rick Stell and
consultant neurologists, See our specialist a comprehensive and ongoing and researcher at the Perron advanced disease management
nurses and other allied health
subsequently Ms Walters. professionals.
consultant service, and leads the clinic Institute, and a consultant therapies including deep brain
alongside nursing and allied neurologist at Sir Charles stimulation, Duodopa and
neurologists and health staff. He also leads Gairdner and St John Apomorphine therapies.
Our clinicians treat people Clinics include Stroke, Multiple
with some of the most complex Sclerosis, Movement Disorders
their clinics and stroke research. of God hospitals.
neurological disorders. (including Parkinson’s), Motor areas of research.
Clinical Professor Soumya Ghosh Professor Merrilee Needham Dr Dev Nathani
Movement Disorders (incl. Neuromuscular (incl. Inclusion Body Motor Neurone Disease Clinic
Parkinson’s) Clinics and Research Myositis) Clinic and Research and Research
Professor Frank Mastaglia AM A/Professor Rick Stell Sue Walters
Clinical Professor Soumya Ghosh Professor Merrilee Needham Dr Dev Nathani joined our motor
is a consultant neurologist at the leads an integrated neurone disease clinic in 2021. The
Perron Institute, and Sir Charles Inflammatory Myopathies clinic offers multidisciplinary care
Gairdner, Perth Children’s Clinic also known as the including nursing and occupational
and Fiona Stanley hospitals. Neuromuscular Clinic. Professor therapy, as well as support from
Professor Ghosh also heads the Needham is an established Sir Charles Gairdner Hospital’s
Institute’s Restorative Neurology neurologist and Head of Respiratory Sleep Disorders
Research. He completed a Myositis Research at the Perron Clinic, gastroenterology, speech
The clinics really are the human side to our success. PhD in neurophysiology at the Institute and CMMIT. pathology, dietetics, physiotherapy
Australian National University. and palliative care.
Sue Walters
Manager Clinic Services
18 Perron Institute Inspiration Report 19
Associate Professor Rick Stell Dr Jason Burton Dr Athanasios Gaitatzis Dr Srimathy Vijayan
Movement Disorders (incl. Multiple Sclerosis Clinic and Epilepsy (incl. Epilepsy and Movement Disorders (incl.
Parkinson’s) Clinic and Research Research Pregnancy) Clinic and Research Parkinson’s) Clinics and Research
Movement Disorders specialist Consultant neurologist Dr Athanasios Gaitatzis is Dr Srimathy Vijayan is a
Associate Professor Rick Dr Jason Burton runs a weekly a consultant neurologist consultant neurologist in the
Stell provides diagnostic and multiple sclerosis clinic at the specialising in epilepsy at Perron Institute clinic. Her key
treatment-based services in his Perron Institute and Fiona Perron Institute and Sir Charles interest is movement disorders
various clinics. Patients have Stanley Hospital. Gairdner Hospital. He qualified including the management
access to advanced disease in Greece and completed his of Parkinson’s Disease and
management therapies including specialist training in London. advanced therapies, particularly
deep brain stimulation and He runs WA’s only dedicated deep brain stimulation. She
botulinum toxin treatments. He Epilepsy and Pregnancy clinic, completed her medical training
helped establish our clinics in the the second in Australia. at John Radcliffe Hospital in
1990s and conducts research. Oxford, UK.
Dr Janavi Dunuwille Dr Wayne Yau Dr Shruthi Kamath
Epilepsy Clinic and Research Movement Disorders Clinic Multiple Sclerosis Clinic and Research
possibilities
and Research
Consultant neurologist Consultant neurologist Dr Wai Consultant Neurologist
Dr Janavi Dunuwille provides Yan (Wayne) Yau studied at Dr Shruthi Kamath joined
diagnosis, management and UWA, the University of Sydney the Perron Institute clinics in
treatments for patients with and University College London 2021. She provides specialty
epilepsy. Dr Dunuwille is also (UCL). His medical training took multiple sclerosis clinics at the
head of Neurology at Sir place in Perth, Sydney and United Perron Institute. Dr Kamath also
Charles Gairdner Hospital. Kingdom, where he completed provides general neurology and
his movement disorders stroke services at Fiona Stanley
fellowship and obtained his PhD and Bunbury Regional hospitals.
in neurogenetics at UCL.
Pictured right: Alexa, Anne and Clinical Professor Soumya Ghosh clinic consultation20 Perron Institute Inspiration Report 21
Our research collaboration Our clinics
For over 40 years, the Perron Institute has continued to build its Our clinicians deliver specialist services for some of the most
reputation as a world-class medical research institute with national complex cases. They are also working to create the future of
and worldwide links. personalised medicine.
893 454 224
researchers and health research cities
21
treatment clinics
14
clinical trials
8
neurologists leading
professionals institutes per week research projects
105
Australian universities,
44 countries
75+
PhD and master students
13
consultant neurologists
3,117
clinic visits in 2021
hospitals and medical and all involved in research
research institutes
We are a leader in delivering research
outcomes to the community.
Pictured right: Demyelinating Diseases Research Team22 Perron Institute Inspiration Report 23
Our links
collaboration
Collaboration with researchers and institutions worldwide is integral
to success in scientific research. One of our strengths is the extent
of links and collaborations globally.
and unity
Science is a global
endeavour. Ideas transcend
national borders and in an
increasingly interconnected
world, international
collaboration is absolutely
essential if Australian
science is to flourish.
Professor Steve Wilton A0
Perron Institute and CMMIT Director24 Perron Institute Inspiration Report 25
Our University partners:
Our
With our significant
supporters
cohort of partners and
supporters, we are well
With sincere appreciation,
positioned to do great
we’re proud to list our many Our supporting, corporate and community partners:
things. This includes
partners, collaborators,
supporting scientific
breakthroughs,
funding bodies and
drug development supporters. They include:
innovative models of
Our Founding and
care, new technology
longtime supporters:
and services.
Professor Alan Robson
Perron Institute Chair
appreciation
Neurotrauma
Research Program26 Perron Institute Inspiration Report 27
Our inspiration
Seeing people in our community who are suffering from Consumer and Australian Health Translation
Network (WAHTN) Consumer
devastating disorders where treatments are insufficient or, in Community and Community Involvement A few years ago,
Involvement Program, formed a Consumer
many cases, unavailable motivates us every day to pursue my greatest hope
Program Advisory Group in 2020. The
discoveries that will bring hope in areas of unmet need and Improving health outcomes for
group comprises individuals and dream was
with experience of some of the
ultimately change lives. people is the goal of medical
most debilitating conditions funding becoming
research and there is increasing
awareness of and emphasis
under investigation by available for
researchers.
Compassionate end-of-life, increasing people’s The City of Bunbury, as part given to the need for consumer drug trials. The
connectedness to their of its Strategic Community and community involvement in In 2021, the WAHTN launched
Communities community. Plan, has recently included all phases. the Consumer and Community Sirolimus trial
Network an ‘Aspiration’ to foster: ‘a Involvement handbook, a
is about to start
The Compassionate Connectors Our researchers at Perron
compassionate and inclusive practical step by step guide
Professor Samar Aoun is the Model of Care initiative, in Institute and CMMIT, with the
Perron Institute Research Chair in partnership with Palliative
community’.
assistance of the Western
for organisations, researchers, at the Perron
consumers and funders.
Palliative Care at UWA. She has Care WA, has been adopted In 2021, the first South West Institute. My
influenced rapid system change by the WA Country Health Compassionate Communities
by engaging the community Service as ‘business as usual’ Network forum was held in new hope is
through the South West function of the existing Bunbury Bunbury. Bringing together
Compassionate Communities
that clinicians
Hospital Volunteer Program. politicians, researchers and
Network which she co-founded This program was selected as people in the community to and researchers
in 2018 and chairs. a finalist for the Palliative Care discuss and share practical tips
Trial offers hope in tissue, especially in the arms
can discover a
Awards in the Outstanding on how to help each other deal and legs. At this stage, there
Compassionate Communities fight against IBM
is a global movement that Regional/Rural Team Delivering with challenging topics such as is no effective treatment and treatment or cure
encourages social networks Holistic Palliative Care in any death, dying and grief. My name is Damian Slater. people like me end up requiring
to play a much stronger role Setting award. In 2008, aged 35, I was assistive devices for daily living. for all of us living
diagnosed with Inclusion Body I am now confined to an electric
in supporting those at the
wheelchair.
with this rare
Myositis (IBM). It was just six
months after I was married, and I
The hardest thing about living
disease.
was one of the youngest people
with my condition has been the
in Australia to be diagnosed with Damian Slater
limitations it puts on what I can
this disease.
do with my son and wife. My son
The diagnosis was devastating is just amazing, though, and my
for me and my wife, but with the wife’s loving, positive outlook is
support of my wonderful team my greatest motivation.
of specialists, we remain positive
about future upcoming drug
trials and grants to find a cure.
IBM is a rare, incurable disease
where the body’s immune
system attacks healthy muscle
Pictured left amd right: Samar, Drew and Maureen
at Perron Institute; Damian and Carmen Slater.
Photo credit Tony McDonough, The Australian 202028 Perron Institute Inspiration Report 29
Involving the broader community
in shaping our research is crucial
if we are to succeed in our mission
of translating findings from the
laboratory into outcomes that
directly benefit patients.
Clinical Professor David Blacker
Perron Institute Medical Director
Defying his disease Almost six years since approval
of the first drug Exondys 51
Making life easier
influence
21-year-old Billy Ellsworth, still (eteplirsen), Billy maintains Jenny Dixon is a patient of
participating in a US clinical stability and defies his disease the Perron Institute clinic and
trial for Duchenne muscular by not being confined to a is happy to talk about her
dystrophy, graduated in 2019 wheelchair. deep brain stimulation (DBS)
and is studying at college. He operation which she said has
is still able to walk due to the Billy’s mother Terri Ellsworth made her life so much easier.
pioneering and life-changing said: “I’m so grateful to the
therapies developed by Perron Institute, UWA and “Without the DBS operation I
Professors Steve Wilton, Sue Murdoch University. I think it would not be the person I am
Fletcher and their team. Three is important, motivational and today. I can feed myself without
treatments received accelerated inspirational to see Billy walking breaking teeth, cook for myself
approval by the United States well at 21 years of age.” and carry a cup of tea with one
Food and Drug Administration hand. It has changed my life for
in 2016, 2019 and 2021. the better in so many ways.”
Jenny Dixon
Pictured top and bottom right: Emeritus Professor Byron Kakulas AO and Billy Ellsworth; Anne and Alexa Jefferson in the clinic30 Perron Institute Inspiration Report 31
Our impact
Translating results into community benefit Strategic priorities Research translation is part
for research of our DNA. Patients need
The Perron Institute is of making a major contribution industry, non-governmental Foster the development and access to safe medicines as
to the health of the community. organisations, communities retention of clinician scientists
proud of its success and community organisations, to drive clinical practice quickly as possible. We have
In striving to achieve our
in delivering research
objectives in translational
patients and carers. innovation and quality built a platform to take our
Drive translational research
outcomes to the medical research, we see In working to improve the lives preclinical developments from
through engagement with
community. the rapidly advancing field and the prospects for people
of molecular genetics as a with neurological disorders,
network partners the lab, through the regulatory
tremendous opportunity. including those with rare Grow clinical trials capacity
Research excellence and participation processes and into the clinic as
The personalised therapy diseases, our researchers are
for the future pathway, to refine diagnostic tackling complex issues and Support translational research fast as possible.
processes and tailor therapies applying advanced research through engagement between
The Perron Institute, a centre clinicians, patients, academic Professor Anthony Akkari
to specific groups and technologies.
of excellence in neuroscience partners and industry Head of MND Genetics and Therapeutics
individuals, is a major focus for
research, is committed to Clinical research must be Research and Black Swan Pharmaceuticals
the Institute – an area in which Maximise success in Chief Scientific Officer
continuing on its pathway of responsive to changing
we are confident we can have a obtaining competitive grant
collaboration and affiliation, community needs. Contemporary
significant impact. and industry funding
connecting people, ideas issues include a diverse and
Improving research
and alliances with innovative
We achieve ageing population, developments
governance, support
strategies. in data acquisition and analytics
outcomes by capacity and technologies that functions and infrastructure
Growth - a engaging with enable personalised medicine. to support research to
maximise successes
continuing end-users Also, the expectation that
consumers have a voice in Increasing research profile
trajectory An essential ingredient in
the development of research through publications,
We have grown to include over successful medical research translation, leadership
priorities and how care is
250 clinicians, researchers is engaging with the various and engagement with
delivered.
and staff, with an extensive people affected by the entire the community
network of partnerships and process, so that we focus on Our strategic plan must reflect
relationships across the nation the priorities and that the these external realities.
and internationally. By working outputs of our research can
collaboratively, we have been be translated into outcomes
able to deliver many shared to benefit patients. End-
persevere
research successes with the aim users include government,32 Perron Institute Inspiration Report 33
Four major milestones in Byron Kakulas
clinician-scientists, he will lead projects and programs at
the development of a Stroke the Perron Institute. The very
Centre of Excellence. generous commitment will fund
Annual Medal and
Lecture
our 40th year
projects focusing on translating
This new chair position will
discoveries into new therapies
enable dedicated researchers to In February 2022, Professor
and clinical processes.
develop a leading international Steve Wilton advised he would
site for stroke research, Associated with the Sarich be proud to support the
education, care and advocacy. family support is the creation inaugural Byron Kakulas Annual
Medal and Lecture. The launch
Research Chair in an international leader in conditions. The core aspects of It will enable Professor Hankey of a fund named in recognition
of this award coincides with the
promoting and advocating for this program include increasing to build on the excellent stroke of distinguished neurosurgeon
Palliative Care a public health approach to support for family carers, grief research being undertaken Emeritus Professor Bryant Institute’s 40th Anniversary.
In January 2022, Professor palliative and end-of-life care. and bereavement support, at the Perron Institute and Stokes AO for his contribution
Emeritus Professor Byron
Samar Aoun was announced and improving grief and death to collaborate with several to the Perron Institute and
She has published extensively Kakulas AO, founder of the
the Perron Institute Research literacy around end-of-life and existing outstanding clinical and advancement of neurosciences
and her research outcomes Perron Institute, has been
Chair in Palliative Care at The palliative care. research stroke groups. research. The Bryant Stokes
have been influential in the an innovator throughout his
University of Western Australia. Neurological Research Fund
career. His research has tackled
development of policy and Chair in Stroke Professor Hankey will
will support seven projects
some of the bigger questions
Professor Samar Aoun joined practice guidelines at national spearhead the formation of
the institute as head of Palliative and international levels.
Research new lines of research and
to be selected in 2022, up to
in medicine, resulting in some
$100,000 each.
Care Research in 2018 and is Another partnership between attract leading colleagues paradigm-shifting findings that
As Chair, Professor Aoun will from interstate and overseas We are indebted and very have changed the direction
the University of Western
focus on developing models of to join forces here and provide grateful to Professor Stokes, a of scientific thinking. This is
Australia and the Perron
integrated care which mobilise ongoing momentum for further former Perron Institute Board exemplified by his work on
Institute is paving the way for
community assets, improve advances. Director, who has championed muscle regeneration.
the future of medicine.
primary care engagement, and excellence in clinical practice
The award will recognise
enhance partnerships between In mid-2022, internationally Sarich family funding and medical research. His
specialist and generalist services. renowned Winthrop Professor innovators in a broad sense.
Graeme Hankey became the
and Bryant Stokes vision to bring together key
This could include researchers,
neuroscience research groups
Other areas target improved
Chair in Stroke Research. Fund to foster collaboration resulted
clinicians, health administrators
models of care for non-cancer and media figures who have
Recognised for decades as a We are extremely grateful in the development of the Ralph
conditions such as motor changed the face of medical
global leader in stroke research to receive support from the & Patricia Sarich Neuroscience
neurone disease (MND) and research and/or service delivery
and one of Australia’s leading philanthropic Sarich family to Institute Building which opened
other neurodegenerative for the better. The recipient will
fund important research at the QEll Medical Centre in 2017.
receive a medal, a cash award
of $5,000 and will present the
annual Kakulas Lecture.
Pictured: Prof Alan Robson, Prof Bryant
Stokes and Steve Arnott
excellence34 Perron Institute Inspiration Report 35
Key research Genetic variant discovered for
the progression of dementia
Recent Recognition HIGH IMPACT Duchenne resulted in three
FDA approved drugs that for
has been massive. Perron
Institute researchers proved
highlights in Parkinson’s Professor Steve Wilton AO,
Professor Sue Fletcher AO
AREAS the first time enable clinicians their adaptability by being part
Revolutionary nerve repair Parkinson’s non-contact to treat the underlying gene of this effort as shown in one
treatment developed by Prof and Professor Frank Mastaglia Translational medical research defect in Duchenne. Combined, example below. Others can be
boxing study
Minghao Zheng and team via AM recognised in Queen’s is a lengthy process. It can take the three drugs developed can found in the Commercialisation
First recruiting site for IBM Birthday Honours 2021
Orthocell many years for a discovery treat almost thirty per cent of section.
study with Abcuro in the USA
Jacinta Thorne wins the in the laboratory or clinic to patients diagnosed with this
Rapid and accurate COVID-19
test developed with Avicena
Advanced Trainee Clinic 3 Minute Thesis competition translate into an outcome such form of muscular dystrophy. A rapid paper test
program with SCGH
for large scale screening 2022 as a new diagnostic, a new
for SARS-CoV-2
$1m sepsis grant – A/Prof
New genetic marker Frances Theunissen wins the treatment or changes in health End-of-Life Care
discovered for motor neurone policy or clinical practice that In partnership with the Fiona
Rakesh Veedu and Dr Andrew Best Paper competition 2022 Professor Samar Aoun’s
disease by Prof Anthony directly benefits patients. As Stanley Hospital and Centre for
Currie, CMMIT Emily King receives Geoffrey research has led to Entrepreneurial Research and
Akkari and team an example, the first report by
2 x $250k FightMND grants Blee Neuroscience Honours development of the Public Innovation, Associate Professor
Perron Institute hosts Steve Wilton and colleagues
for motor neurone disease Prize 2021 Health Model for Bereavement Rakesh Veedu has developed a
its first cohort of Curtin on the capacity of antisense
research Alex Tang receives Young Tall Support. In turn, this has led to novel lateral flow paper test to
University Medical School oligonucleotides (AO) to
Poppy Award 2021 improved practice and policy detect the presence of SARS-
Prof Minghao Zheng’s undergraduates promote exon skipping in
at national and international CoV-2 in swabs. The test is
collaboration and PearlBone Sarah Hellewell receives Duchenne muscular dystrophy
Hole in One event raises levels. Internationally, for
discovery for bone formation Perron Institute Aspire appeared in 1997. It took until both highly accurate and yields
money for Telethon funded example, elements of the model
Award 2021 2016 – 19 years later – before results in minutes. The test is
World Stroke Organization research have been adopted by the UK
the first drug, Exondys 51 was being evaluated for sensitivity by
award for treatment and care Chidozie Anyaegbu receives National Bereavement Alliance
$1.8m grant received for IBM researchers in Singapore and is
Millennium Science Award approved by the US FDA.
Prof Sulev Koks’ cancer paper trial led by Professor Merrilee Taskforce of the European the subject of a provisional patent
2021 Despite these long timelines,
in Nature Biotechnology Needham and team Association of Palliative Care application. This research was
Perron Institute’s research
Prof Minghao Zheng’s Alex Tang nominated for and Irish Hospice Foundation.
US FDA approves third drug includes some outstanding supported by the Government
lymphedema study Premier’s Science Prize 2021
for Duchenne muscular examples of successful impact. of Western Australia’s COVID-19
dystrophy
The COVID-19 Special Initiative Scheme.
Development pandemic
of drugs for the By March 2022, reported deaths
treatment of from the pandemic had passed
six million and it is thought likely
Duchenne muscular
that the real figure is far greater.
dystrophy The worldwide research effort
Professors Steve Wilton and Sue to develop vaccines, adjunct
Fletcher and team’s research therapies and reliable and fast
on AO-based therapeutics for diagnostic screening systems
innovative, resilient
and sustainable36 Perron Institute Inspiration Report 37
Non-contact physiologist, researcher and Parkinson’s. So far, the program In 2021 to build on the program, and Precision Nucleic Acid
Perron Institute Research has improved balance, fitness Professor Fitzgerald launched Therapeutics led by Associate
Boxing study for Affiliate Dr Travis Cruickshank and overall wellbeing for and became the CEO of Professor Rakesh Veedu.
Parkinson’s disease and PhD student Mitchell Turner, participants. The team looks Connectivity, a not-for-profit
Perron Institute Medical Director both from ECU, along with Claire forward to publishing its results organisation working to raise WA Brain Bee
and consultant neurologist Tucak, Perron Institute senior in the coming months. awareness of concussion and Challenge The brain is
Clinical Professor David Blacker neurophysiotherapist, are also traumatic brain injury (TBI) in
is leading a non-contact boxing on the team. Concussion and the community. Connectivity Associate Professor Jenny
the last frontier.
Rodger, senior MSWA Research
exercise study for Parkinson’s Connectivity links patients, carers,
disease. The study is being
Professor Blacker has a highly
researchers, clinicians and Fellow and head of Brain If we have a good
personalised point of view as In 2019, the Federal Government Plasticity Research at UWA
health care providers to build
conducted at Edith Cowan a neurologist and having been announced $50m funding for the evidence base and improve and Perron Institute, has understanding
University’s Joondalup campus diagnosed with Parkinson’s. the Australian Government’s been involved in UWA’s Brain
in collaboration with the Perron He is a strong advocate for research funding program, the
outcomes for people following
Bee Challenge for 15 years.
of it, we will be
Institute and UWA. TBI of all severities, including
exercise, given that it has Mission for Traumatic Brain concussion. The competition encourages transformed.
Professor Blacker is significantly helped to reduce Injury. The Mission’s Expert students to consider a career
his symptoms. Working Group is chaired Among their exciting new in science and neuroscience Don’t look at your
collaborating with former
Golden Gloves Boxing Through the FIGHT-PD study,
by Professor Melinda (Lindy) projects are an online course for in particular, and to promote
interest in the
champion and fitness instructor Fitzgerald, who heads the first responders, and education the study of neuroscience as a
Rai Fazio who developed the
Dr Blacker and the team seek
Neurotrauma Research Group at and outreach with aboriginal multidisciplinary endeavour. brain in a limited
to show the benefits of boxing
the Perron Institute. communities in regional WA.
program with him. Exercise as an exercise therapy for Winners of the 2021 WA way. The sky is the
Centre for Brain Bee Challenge were
limit.
congratulated at a special
Molecular Medicine awards ceremony at the Perron
Pictured: FIGHT-PD study team
and Innovative Institute, with awards presented
Professor Amit Chakma
Therapeutics by UWA Vice-Chancellor UWA Vice-Chancellor
Professor Amit Chakma.
Established as a joint venture
between the Perron Institute
and Murdoch University, the Pictured: A/Prof Rodger, Mr Arnott,
Brain Bee 2021 winner Catherine
Centre for Molecular Medicine Yang and Prof Chakma
and Innovative Therapeutics at
Murdoch University was opened
in 2019. Professor Steve Wilton
AO and Dr May Aung-Htut are
Co-Directors.
They lead a team of over
ten research groups, seven
of which are co-located at
Murdoch University and
Perron Institute. They are:
believe educate
Demyelinating Diseases led
by Professor Allan Kermode,
Motor Neurone Disease led
by Professor Anthony Akkari,
Functional Genomics led
by Dr Sarah Rea, Molecular
Therapy led by Professor Steve
Wilton, Neurodegenerative
Diseases led by Professor
Sulev Koks, Myositis led by
Professor Merrilee Needham,38 Perron Institute Inspiration Report 39
Our commercialisation
In the latest phase of our development, building our commercialisation
capability has become a key focus. This is to ensure we maintain the
sustainability of our organisation for another 40 years.
While we hear quite often of Sarepta RAGE Biotech
innovation in industry - energy,
minerals, agriculture, for example
Therapeutics Professor Steve Wilton, in
- we sometimes lose sight collaboration with RAGE
The advanced technology
of the phenomenal medical Biotech and Monash University,
developed by Professors Steve
research history of our State. is involved in research using
Wilton AO, Sue Fletcher AO Pictured above: A/Prof Rakesh Veedu and his team. Photo credit Murdoch University
We’re proud of WA’s history of antisense oligonucleotide
and their team with their drugs
medical research innovation and technology in respiratory
for Duchenne, has provided
the contribution of the Perron disorders such as those internationally we collaborate Institute Medical Director Murdoch University and Perron
a platform for potential
Institute and its partners in associated with the coronavirus, with renowned researchers Clinical Professor David Blacker Institute) for the manufacture
treatments for numerous
that context. The next decade as well as other conditions such at Duke University and the is the Chairman of the Clinical of research grade nucleic
conditions, such as motor
for our organisation involves as sepsis and diabetes. University of Colorado. Advisory Committee and acids and to commercialise
neurone disease, Parkinson’s
new commercial partnerships and spinal muscular atrophy. Professor Meloni is the Chief intellectual property created
We also have Memoranda of
with promising molecules and
therapeutics in development
The technology also has Understanding with several A GROWING Scientific Officer. The company
(ASX:AGN) listed on the
by A/Professor Veedu in the
areas of diabetes and macular
potential for the treatment
for a range of neurological and of COVID-19.
scientific organisations locally,
nationally and internationally. In PORTFOLIO OF Australian Stock Exchange degeneration.
other conditions.
Western Australia, this includes
Preveceutical Medical, interstate
PATENTS AND mid 2021.
Avicena Systems
Here are some of the impressive
we work with GenieUs, and SPIN-OFFS Black Swan Avicena Systems was
examples.
Pharmaceuticals established by Professor
Over the past few years, several
An international collaboration Paul Watt and several
spin-off companies have been
between Australia, the other WA research leaders.
established to drive the Perron
USA and the UK, Black They have developed the
Institute’s drug development
Swan Pharmaceuticals was ‘Avicena Sentinel’ machine,
forward.
established in 2019 to expedite a highly sensitive population
screening platform for viruses
Argenica drug development, particularly
such as COVID-19, which aims
for motor neurone disease.
Therapeutics Professor Anthony Akkari to make repeated testing
quicker and easier without
pioneer
In 2020, Argenica Therapeutics is the Chief Scientific Officer,
Ltd was set up for the Dr Loren Flynn is the Director of compromising testing quality.
commercialisation of very Therapeutic Development and Professor Sulev Koks (Perron
promising neuroprotective Professor Steve Wilton AO is on Institute and CMMIT) is one of
peptides for the early the Scientific Advisory Board. the researchers collaborating
intervention of stroke with the multidisciplinary team.
discovered by Clinical Professor SynGenis
Neville Knuckey and Professor
SynGenis was established in
Bruno Meloni at the Perron
2020 by Associate Professor
Institute and UWA. Perron
Rakesh Veedu (CMMIT,
Pictured left: Dr May Aung-Htut and Professor Steve Wilton40 Perron Institute Inspiration Report 41
SCIENTIFIC
COLLABORATION
Brain and Bone Since joining the institute,
his research has expanded
and orthopaedics to include the role of bone
One of the
Professor Minghao Zheng, cell-derived factors in brain defining features
head of the Perron Institute’s function and disease.
Brain and Bone Axis Research of the Perron
Professor Zheng and team
group, is best known for his
have developed a patented Institute is
work on bone and joint disease
technology for the manufacture
and regenerative medicine. its success in
of a collagen device, CelGro®,
He has published over 250
papers and holds 11 patent
to enhance the capability for translational
nerve repair in patients with
families in orthopaedics and
peripheral nerve injury. This has research and
cellular therapy worldwide.
been licensed to Orthocell Ltd, commercialisation,
an ASX-listed company founded
to commercialise technologies which can bring
developed by Professor Zheng,
including the cell therapies
significant and
Ortho-ATI™ and Ortho-ACI™ timely benefits
for the regeneration of
damaged tendon and cartilage, to the Western
respectively, and now extended
Australian
to include CelGro®.
community and
beyond.
Professor Minghao Zheng
Head of Brain and Bone
Axis Research (Perron Institute
and UWA) and Orthocell
Co-Founder and Consultant
Chief Scientific Officer
imagine42 Perron Institute Inspiration Report 43
Our future Genomics Research
Other exciting new areas to find answers to complex
include developing synthetic diseases and their future
molecules for mRNA drugs management. With our partners, we have an
and investigating the role evolving focus and investment
of genetics in depression to We are supporting the exciting in genomics. This work is led
tailor treatments, particularly work of our research groups as by Professor Sulev Koks at
they broaden their horizons and Perron Institute and CMMIT,
Cultivating an inclusive culture of creativity for people whose condition is
networks to take their discoveries Murdoch University, looking to
resistant to medication.
and leadership from bench to bedside. expand research by studying
Achieving greater understanding the structure of the genome to
of the brain and bone axis and We encourage and support
stratify treatments to achieve
its implications for potential new cross-institutional collaboration,
improved targeting.
therapies for Alzheimer’s and creativity, and innovation
As we celebrate our 40th All the above are helping to to push the limits of what
dementia is also promising. The findings have potential
anniversary and look to the ensure that the Perron Institute is possible. Creating multi- for many facets of science
Our goal is to be future, we will continue to is well placed to achieve a high
Strategic Research disciplinary collaborations and health, including disease
build on our foundations as a level of research impact focused between scientific and non-
an international resilient and innovative centre on improving health outcomes. Review detection, healthy ageing,
scientific experts with the mental health and optimising
leader in delivering of medical research excellence We are working to continue to With the help of an external resources, capability and healthy brain function.
with a translational focus. build on our research scope panel, the Institute undertook networks to take translation With a growing number of
high-quality and and capacity by investing in a Strategic Research Review processes forward is an ageing patients presenting
Our Board, chaired by Professor collaborations that generate
comprehensive Alan Robson AO CitWA, brings and translate research evidence.
in early 2022 to gauge how essential part of this. with complex conditions in
a depth of knowledge and we’re tracking and to provide the contemporary health
patient-focused experience to its governance Ensuring that we remain insights to help shape our future
Alignment of research focus
environment, these are vital.
areas with key research
direction. The key learnings will
neuroscience care and strategic leadership roles, financially sustainable through partners will drive discovery and
working closely with senior diverse funding sources, be considered in developing the Developing
improvements in translational
through research, management. Our highly including philanthropy, success Institute’s priorities, goals and antisense therapies
medicine to benefit people
strategies for our development
collaboration regarded neuroscientists and in attracting research grants
over the next few years.
with neurological disorders. for Psoriasis
clinicians, our very talented and achieving commercialisation In creating new knowledge and
and service. We early career researchers, our is vitally important. Professor Sulev Koks is also
achieve this by
clinic managers and allied
One of several focus areas we
SHAPING THE therapeutic products, there
is also significant commercial
expanding his research on
the chronic and complex
continuing on the
health professionals and
support staff are making an
are excited about expanding is FUTURE OF potential which could benefit
the Institute, our partners and
autoimmune skin disease
path of innovation,
outstanding contribution.
stroke, where we are well on the
way to fulfilling a ten year vision HEALTH CARE the broader Western Australian
psoriasis, working with
Postdoctoral researcher Dr Vidya
economy.
Other strengths are our close to be recognised nationally and The Perron Institute is playing Krishnan who joined the Perron
developing new links with clinical and health internationally as a centre of its part in the future of health There are many exciting areas Institute in 2020.
knowledge and services, our collaborative excellence. care by bringing together of investigation and research
approach in pursuing complex, some of the brightest minds being developed and expanded.
delivering services multi-disciplinary research, our
Other important fields are
in neuroscience and medicine
genetics, phenomics and
affiliations with academic and
to help reduce research institutions Australia-
pathology and the role these can
play in fast-tracking research and
the burden of
opportunities
wide and internationally, and
translation, including in tackling
our close relationships with
neurological government and community-
rare diseases - a huge area of
unmet need. We are committed
based health support groups.
disorders in the to harnessing information
As well as our pride in our encoded in the genome to better
community
research achievements and the diagnose, predict, treat, and
quality of care delivered by our prevent diseases that profoundly
Steve Arnott clinics, we strive to be a great impact society.
Perron Institute CEO place to work.You can also read
