Research shaping
the future of
neuroscience

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Specialist clinics
improving
quality of life

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

Unlocking the power and promise of neuroscience research

The Perron Institute, formerly the Western Australian Neuroscience Research Institute, is Western Australia’s longest established medical research institute. The Perron Institute is at the forefront of international research on muscle, nerve and brain conditions, and has been since its establishment as the Australian Neuromuscular Research Institute in 1982. Its many achievements include contributing to major breakthroughs in the discovery of genes causing disease, and providing the latest in rehabilitative technologies including robotics.

A New Era of Discovery

Neuroscience research is entering a new and exciting era of discovery. New techniques in genetics, cell and molecular biology and neuroimaging are allowing even the most complex problems in neuroscience to be addressed, often for the first time. Problems that hitherto were thought beyond the scope of medical science are now being tackled on a regular basis, often with spectacular success. The Perron Institute’s research on a new therapy for Duchenne muscular dystrophy is a case in point. This research has established for the first time that Duchenne – a disease formerly thought hopelessly incurable – is amendable to therapy.

The Perron Institute’s goal is to apply the latest research techniques to investigate the origin, diagnosis and treatment of a broad range of neurological disorders.

Our goal is to take findings from the research laboratory and translate these into new drugs and diagnostics, new approaches to rehabilitation and better ways of delivering services to the community.

Goal

The Perron Institute’s goal is to be an international leader in translating neurological research into treatment for the benefit of the community.

One of the Perron Institute’s special strengths lies in its links between the institute’s laboratory research and its specialist neurology clinics; a translational research pathway. This means being able to translate research findings from the laboratory into a better quality of life for patients and their families in the form of new diagnostics, treatments and improved ways of delivering services to the community.

Our Patrons

“Scientific research is incredibly important for our world, and particularly for those whose lives are enhanced through new discoveries. It is therefore a privilege to be able to support the world-leading research that the Western Australian Neuroscience Research Institute does as its Patron. As a passionate advocate of scientific research for life-changing benefits, it is wonderful to know that in the Perron Institute we have world-acknowledged neuroscientists unlocking genetic secrets to find answers and treatments for those who suffer debilitating neurological conditions. The work of this organisation is extremely exciting and the prospects are very hopeful. With this in mind I urge you to learn more about their work and the difference it will make to those who are affected by debilitating conditions in our great state, and to look at what you can do to help their game changing research and support the discoveries that have enormous potential to change lives today and into the future”.

Her Excellency the Honourable Kerry Sanderson AC Governor of Western Australia

Patron, the Perron Institute

Interview with Stan Perron AM

Patron Mr Stan Perron AM talks of his long time support of the Perron Institute, formerly the Western Australian Neuroscience Research Institute (WANRI), including the recent naming of the Institute in his family’s honour to become the Perron Institute for Translational and Neurological Research.

One of the Perron Institute’s special strengths lies in its links between the institute’s laboratory research and its specialist neurology clinics; a translational research pathway. This means being able to translate research findings from the laboratory into a better quality of life for patients and their families in the form of new diagnostics, treatments and improved ways of delivering services to the community.

Vision

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

Mission

Quality

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

Translation

We are committed to translating the outputs of our research into better outcomes for neurological patients, including new drugs and diagnostics, new therapies and better ways of delivering services to the community.

Scope

Our mission is to undertake quality research across the full scope of the neurological disease spectrum, building on our past research successes in muscular dystrophy, stroke, Parkinson’s, multiple sclerosis, myositis and restorative neurology.

Recent Success

The Perron Institute is enjoying the recent success of the US Food and Drug Administration (FDA) accelerated approval for a novel treatment for Duchenne muscular dystrophy, using “genetic patches” to mask the error in the gene message that causes Duchenne. The Perron Institute’s track record of research translation continues with its exciting future expansion.

Our aim 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 diseases such as stroke, Parkinson’s, motor neurone and multiple sclerosis. Building on our breakthroughs, the Perron Institute is recruiting the world’s best researchers to undertake cutting-edge research on new treatments for these devastating conditions.

OUR THREE PILLARS

World Class Research

The Perron Institute undertakes pioneering research on a broad spectrum of neurological disorders, including stroke, Parkinson’s disease, multiple sclerosis, spinal cord injury, brain cancer and muscle disorders including muscular dystrophy and myositis. It brings together laboratory scientists, neurologists, neurosurgeons, clinical psychologists, physiotherapists and other health professionals to tackle the major research challenges in neurology.

In addition, our researchers supervise a growing number of PhD students from Australia and overseas and collaborate with leading universities worldwide.It has affiliations with the University of Western Australia and Murdoch University. The Perron Institute’s researchers and neurologists have links with many universities and hospitals worldwide such as Great Ormond Street, London and Nationwide Children’s Hospital, Ohio. They collaborate with the top universities recognised by The Times Higher Education world reputation rankings. These include Harvard University, Stanford University, Massachusetts Institute of Technology (MIT) and the University of Oxford.

Leading Clinics

The Clinics at the Perron Institute provide diagnosis, management and treatments for patients with complex and usually long term, neurological and neuromuscular diseases.

Such conditions include Parkinson’s disease and other movement disorders such as Essential Tremor and dystonia, Multiple Sclerosis, Inflammatory Muscle Diseases including Inclusion Body Myositis and polymyositis as well as a variety of unusual and rare muscle and nerve diseases. More recently the Clinic service has expanded to include a post-stroke/cerebral palsy Spasticity Clinic and the Refractory Epilepsy service has also been re-established.

Interview with Frank Mastaglia

Perron Institute Clinic Founder and Senior Advisor Professor Frank Mastaglia, neurologist and researcher, reflects on his career and the people who helped influence it, including Founding Director Emeritus Professor Byron Kakulas AO. Professor Mastaglia says he is forever grateful to the people who made it possible, including his parents for his great start, and his mentors and role models. Professor Kakulas started the first neuropathology centre in the country and laid the foundation for the translational research success of the recent Duchenne muscular dystrophy treatment. Professor Mastaglia says the standard of our treatment for conditions such as acute stroke is on par with the best centres nationally and internationally. Professor Mastaglia said he is proud of what he has achieved, including being the first in his family and privileged to go to university, training in Britain and the US, heading up the neurology department at Sir Charles Gairdner Hospital, holding positions at Murdoch University and The University of Western Australia, including Head of Medicine.

Breakthrough Therapies

Stroke is Australia’s second biggest killer and a leading cause of disability. Currently in WA, around 41,000 people live with the effects of a stroke. It is estimated this number will rise significantly in the future, with the likelihood many of us will know or live with someone suffering the effects of stroke.

To address this, the Perron Institute is establishing a state-of-the-art Centre for Restorative Neurology. With the huge leaps we are making in understanding stroke through research and therapy, and the treatments already offered to stroke patients, the Centre’s potential for helping to restore substantial quality of life to patients is extremely promising.

OUR HISTORY

Our Story

In 1971, Perth hosted a major international congress on muscle diseases where the idea of establishing a neuromuscular research institute in Perth was born. The culminated in the establishment in 1982 of the Australian Neuromuscular Research Institute with Professor Kakulas as its Foundation Director.

In 1967, Professor Byron Kakulas, then Professor of Neuropathology at the University of Western Australia, in partnership with a group of parents whose children suffered from muscular dystrophy took the bold decision to establish the Muscular Dystrophy Association of Western Australia, now Muscular Dystrophy Western Australia. The group’s goal was to raise funds for medical research on muscular dystrophy and were assisted in this process through the generous support of the West Perth Rotary Club and the Channel 7 Telethon Charitable Trust.

In 1978, the Western Australian Government indicated that it was prepared to make space available in the old Sir Charles Gairdner Hospital building at the Queen Elizabeth II Medical Centre in Nedlands. With the establishment of the Australian Neuromuscular Research Institute in 1982, detailed planning began on the conversion of this space into medical research facilities, which were completed over the period 1982–1990. The institute’s first research work began in part of the old building in 1983.

The institute, renamed the Western Australian Neuroscience Research Institute in 2012, and then in 2017 became the Perron Institute for Neurological and Translational Science, has continued to grow and now involves research on over fifteen neuromuscular and neurological disorders. The goal of the research continues to be translational, so that findings from the laboratory lead to better clinical outcomes for people suffering.

Muscular Dystrophy Western Australia has continued to contribute towards to the institute’s funding for many years and even now continues a close association with the research of Professor Wilton and Fletcher on muscular dystrophy. In 2017 Muscular Dystrophy WA celebrated its 50th Anniversary.

Interview with Byron Kakulas AO

Perron Institute Founding Director Emeritus Professor Byron Kakulas AO, neuropathologist, reflects on his career and momentous discoveries that led to a treatment for sufferers of Duchenne muscular dystrophy. Professor Kakulas says he feels wonderfully fulfilled after devoting his career to muscular dystrophy research, and experiencing the rare occurrence of being able to deliver a treatment he had promised for 50 years. It began with Professor Kakulas’ muscle regenerating discovery in the Rottnest Island quokka in the 60s, which laid the foundation for DNA expert Professor Steve Wilton and longtime collaborator Professor Sue Fletcher to develop a “genetic patching” drug to treat Duchenne muscular dystrophy, approved by the US FDA last year.

The Quokka Story

Muscular Dystrophy Western Australia (MDWA) and the Perron Institute, originally the Australian Neuromuscular Research Institute (ANRI) – owe their origins to the humble Rottnest Island quokka.

The quokka, a marsupial species about the size of a domestic cat that is found in small pockets on islands off the coast of Western Australia, in particular on Rottnest Island off Perth, has played a curious role in the history of muscular dystrophy research.

In the post-World War II period, the quokka was intensively investigated by scientists from the University of Western Australia because of its unique habitat and biology. However, this important research was hampered by the animals dying of muscle paralysis when kept in small cages on the university campus.

More

In 1960, Professor Byron Kakulas, then embarking on what was to become a stellar career in neuropathology, including the study of muscle disease, was asked to investigate the problem. Despite many theories to the contrary, Professor Kakulas began to believe that the muscle breakdown in the caged quokkas was due to anti-oxidant deficiency. He showed that it was possible to successfully treat paralysed quokkas with Vitamin E, in the process making the momentous discovery that complete muscle regeneration is achievable, something that was previously believed to be impossible because the prevailing theory was that muscle as a specialised tissue was incapable of regeneration after injury. The treated quokkas completely recovered and serial biopsies of their muscle revealed a sequence of changes leading to complete regeneration.

This world-shattering discovery completely reversed the prevailing dogma towards muscle diseases and especially to the muscular dystrophies, which had been previously considered to be hopelessly incurable.

Such was the importance of this discovery that a large international meeting was held in Perth with the object of promulgating the discovery and stimulating research worldwide, which was considerably accelerated as a result. This meeting, which took place in 1971, was the first major international congress to be held in Australia and put Australia’s medical research on the international map.

Built around the discovery of complete muscle regeneration in the quokka was the academic publication Principles of Myopathology as Illustrated in the Nutritional Myopathy of the Rottnest Quokka, which was adopted as the standard teaching of the subject worldwide. Much of the tremendous progress that has taken place in muscle research since 1960 can be traced back to the early work on the quokka.

The Muscular Dystrophy Association of Western Australia (MDAWA) was established in 1967 to raise money to find a cure based on the quokka discovery. Fund raising was spearheaded by the Rotary Club of West Perth and supported by the Chanel 7 Telethon Charitable trust. Later it was possible to widen the scope of research to include other neuromuscular diseases through the establishment of the Australian Neuromuscular Research Institute (ANRI), in 1983, now the Western Australian Research Institute (the Perron Institute).

The dream that muscular dystrophy could be cured was fulfilled by Professors Steve Wilton and Sue Fletcher of the institute who have applied elegant molecular techniques to override the mutation and correct the genetic defect underlying Duchenne muscular dystrophy. Their work is now undergoing clinical trials in the United States.

It can be said that there are very few instances in the history of medical research that have progressed from blindness to cure in one lifetime, in the case of muscular dystrophy in one of the most difficult fields of human disease.

The quokka research was the subject of Professor Kakulas’ doctoral thesis in 1963 and was subsequently published by the UWA Press (1982). A concise history of muscle research may be found in the journal, Neurology India (56: 258-262, 2008).

Annual Report

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