INSPIRE Magazine Issue 23 by Research Australia

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INVESTMENT THROUGH PHILANTHROPY

INSPIRE illustrates some of the innovative philanthropy and investment partnerships supporting Australia’s health and medical researchers.

his edition of INSPIRE focuses on the significant value of investment in health and medical r esear ch and in particular, the role of philanthropy in the value chain. We a l s o we l c o m e n ews i n th e 2022-23 Federal Budget of a longterm commitment to funding the MRFF Frontier Health and Medical Research initiative, designed by Research Australia in partnership with the Department of Health. It is such vital government investment that enables our researchers to take risks, innovate and translate discoveries that improve health ou tc om e s for Austra lia ns, a nd something we do not take for granted. However, the reality is that despite substantial government funding throu g h th e M e dic a l Re se a rc h Future Fund, National Health Medical Research Council and Australian Re se arch Council, too many worthwhile research projects with enormous potential still miss out. As highlighted by Professor Leedman AO, Director of Perth’s Harry Perkins Institute of Medical Research, in this issue, in 2021, it was announced that 248 researchers nationally will share $239 million in NHMRC Ideas Grants spanning five years, however, more than 2600 applicants across Australia were unsuccessful. In this federal budget there is a worrying continuation of the real terms decline in funding for the NHMRC. T h i s i s of g e n u i n e c o n c e r n to the health and medical research community, and it jeopardises our long-term research capability and

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increases the precariousness of research careers. The pandemic has shown us just how much we need these critical skills, and they are not developed overnight. Research is a long term; sustained investment and these funding bodies are crucial to guiding our future. This issue showcases how philanthropic investment in medical research is stepping in. This support of our world-leading scientists to undertake research to produce the health solutions is much needed to improve patient care and quality of life of the world’s populations. Developments in children’s research are showcased as we hear from the Murdoch Children’s Research Institute (MCRI) and the Telethon Kids Institute about their ongoing projects and new innovations. Our recent national web survey on Post Pandemic Opportunities for Health and Medical Research and Innovation highlighted job insecurity as a major concern for early to mid-career researchers. The Harry Perkins Institute of Medical Research tells us how they are supporting our future generation of scientists through their Safe Harbour Program, through funds raised by West Australians. Another pertinent article comes from The Black Dog Institute about their five-year suicide prevention research partnership with investment company Future Generation Global, which has invested $2.3 million to support the development of digital suicide prevention initiatives that have the potential to make a lasting impact on young people’s lives.

On a more personal note, Professor Julie Bernhardt of the Florey Institute of Neuroscience and Mental Health tells us her story of how she came to dedicate her career to stroke research after watching her uncle’s recovery journey from stroke and now with the generous support of her aunt, has developed Australia’s first comprehensive young stroke service with Professor Vincent Thijs. Thank you not just to those members who’ve shared their stories in this month’s issue but to all members of the Research Australia alliance who work with Research Australia every day. It is on your behalf that we continue to highlight the need for smarter investment, in all stages of the research pipeline, as a core plank of the National Strategy for Health and Medical Research, as announced by Minister Hunt at the Research Australia Awards in December. Best wishes, Nadia and the Research Australia Team CEO & Managing Director – Research Australia

CONTENTS

Australian Health & Medical Research & Innovation

Events overview

Congratulations to the winners of the 18th Research Australia Awards

Social investment targets suicide prevention for future generations

RESEARCH AUSTRALIA

BLACK DOG INSTITUTE

20 World-first research on urinary biomarkers for motor neurone disease FLINDERS UNIVERSITY

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Publisher Research Australia Ltd Art Direction Matthew Ware p +61 403 844 763 e matt@objktive.com For Advertising enquiries please contact the Research Australia office on p 02 9295 8546 or e admin@researchaustralia.org researchaustralia.org

INSPIRE ONLINE issuu.com/researchaustralia

16 Getting to the Heart of Sudden Death CENTENARY INSTITUTE

CHRISTOPHE KEREBEL

18 CP Commons CEREBRAL PALSY ALLIANCE RESEARCH INSTITUTE

INSPIRE is a publication of Research Australia Ltd ABN 28 095 324 379 CHRISTOPHE KEREBEL CHRISTOPHE Twitter : @chriskere KEREBEL 384 My Victoria Street Darlinghurst NSW 2010

My Twitter : @chriskere

Who can submit articles? Any current member of Research Australia who would like to share a relevant story that affects their organisation including, philanthropic donations and their outcomes, research findings, and any other related health and medical research topic that affects the Australian population. Submission guidelines & deadlines For information regarding how to submit and publishing deadlines visit the Research Australia website. Disclaimer The opinions expressed in INSPIRE do not necessarily represent the views of Research Australia. Whilst every effort has been made to ensure accuracy, no responsibility can be accepted by Research Australia for omissions, typographical or inaccuracies that may have taken place after publication. All rights reserved. The editorial material published in INSPIRE is copyright. No part of the editorial contents may be reproduced or copied in any form without the prior permission from Research Australia. © Research Australia 2020.

22 Philanthropy and family inspires world leading stroke research FLOREY INSTITUTE OF NEUROSCIENCE AND MENTAL HEALTH

24 Achieving impact takes time – the value of sharing the research journey TELETHON KIDS INSTITUTE

CONTENTS

Australian Health & Medical Research & Innovation

26 Philanthropy helps researchers bust autism myth QUEENSLAND UNIVERSITY OF TECHNOLOGY

42 Saving lives takes guts – or more specifically, stool AUSTRALIAN RED CROSS

50 Are tiny pieces of plastic inside our brains? UNIVERSITY OF QUEENSLAND

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A monumental investment into stem cell medicine

MURDOCH CHILDREN’S RESEARCH INSTITUTE

44 Donor’s vision comes to life ST VINCENT’S INSTITUTE

52 High Risk High Reward UNIVERSITY OF WOLLONGONG

32 Collective intelligence to optimise patient outcomes in Australian rheumatology OPAL RHEUMATOLOGY LTD

46 Research brings hope THE KIDS’ CANCER PROJECT

54 Smarter mental health service planning can lead to better outcomes for young people UNIVERSITY OF SYDNEY

CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

38 Partners unite to strengthen postpandemic clinical trials sector PRAXIS AUSTRALIA

36 Safe Harbour given to young scientists

Using the Eye to Measure the Brain

HARRY PERKINS INSTITUTE

UNIVERSITY OF MELBOURNE

THE LAST WORD

56 Pioneering partnership at the heart of innovative research UNIVERSITY OF WESTERN AUSTRALIA

58 The gift of time UNIVERSITY OF WESTERN AUSTRALIA

60 Philanthropy’s role in research – shaping a better future LEUKAEMIA FOUNDATION

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EVENTS ROUND UP Research Australia convenes regular specialinterest roundtables throughout the year to gain insights from key experts and refine our focus on the issues that matter most to our members. Two Roundtables were held in recent months:

UNIVERSITY ROUNDTABLE At its final meeting for 2021, the University Roundtable received an update from Research Australia’s General Manager, Lucy Clynes, on Research Australia’s proposal for a national health and medical research strategy. This was followed by a broad ranging discussion on the role of the University in Australia’s future. Research Australia is continuing to pursue development of this strategy, which received the backing of the Health Minister, the Hon. Greg Hunt MP, at Research Australia’s Annual Awards on 9 December. At it the most recent meeting in March, the Roundtable has continued examining how universities can better engage with industry, with a presentation by Emeritus Professor Roy Green, considered the Academy of Science’s proposal for an oversight body to address research misconduct, and heard from Dr Julie Glover, Executive Director, Research Foundations Branch, at the National Health and Medical research Council on the issue of gender disparity in funding from the Investigators Scheme. The next University Roundtable will be held on Wednesday 18 May.

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HEALTH ECONOMICS ROUNDTABLE Research Australia continues to use our Health Economics Roundtable as a forum to support Research Australia’s advocacy in smarter investment in health and medical research. This includes calling for better alignment of existing sources of funding, promoting the economic value of health and medical research and championing the commercialisation of Australian research. In addition to a detailed examination of the implications of the federal budget for our health system and research, the next Health Economics Roundtable will focus on preventive health, particularly obesity, and the critical role of research in delivering progress on this complex issue. The next Health Economics Roundtable will be in early June.

CHRISTOPHE KEREBEL

PRE-ELECTION SUMMIT Ahead of the 2022 Federal election Research Australia hosted a Pre-Election Summit to set the policy objectives needed to strengthen our health and medical research sector. Hosted in partnership with WEHI and CSL, the Summit saw medical researchers and innovators from around Australia join parliamentarians to advance Australia’s medical research and innovation capacity to improve health and economic outcomes for Australia.

My Twitter : @chriskere CHRISTOPHE KEREBEL

Seizing opportunities for Australian health and medical research under a future National Health and Medical Research Strategy was the focus at Research Australia’s Pre-Election Summit. The outcomes from the Summit and Research Australia’s Pre-Election Statement on behalf Australia’s health and medical research sector will be shared with Research Australia’s membership and the broader community.

Research Australia were pleased to be joined by parliamentarians from across the political spectrum including: • • • • •

Dr Katie Allen MP, Federal Member for Higgins and Co-Chair, Parliamentary Friends of Health and Medical Research Ms Ged Kearney MP, Shadow Assistant Minister for Health and Ageing and Federal Member for Cooper Senator Louise Pratt, Senator for Western Australia, Shadow Assistant Minister for Manufacturing and Shadow Assistant Minister for Employment Services Senator Dorinda Cox, Senator for Western Australia, Greens Spokesperson for Science, Research and Innovation; and Mining and Resources; and Trade. The Hon Warren Snowdon MP, Federal Member for Lingiari and Co-Chair, Parliamentary Friends of Rural and Remote Health and Deputy Chair of Standing Committee on Indigenous Affairs

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CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

CONGRATULATIONS TO THE WINNERS OF THE 18TH RESEARCH AUSTRALIA AWARDS The country’s leading scientific minds and advocates were recognised in Research Australia’s 18th Health and Medical Research Awards, celebrating the phenomenal researchers, doctors, and community champions who are working to transform the sector and change lives.

esearcher s working to revolutionise the treatment of spinal cord injuries and slash the rate of infection in our hospitals were among those honoured, as well as the dedicated advocates and philanthropists who help make their work possible.

research sector has ploughed on with remarkable dedication and world-leading discoveries. The pandemic has underscored the critical importance of health and medical research – the fact that we are all here today, vaccinated for a virus which was unknown two years ago, is a triumph for research collaboration and innovation. “Research Australia is proud to advocate for the sector, marking our twentieth year of doing so. We’re honoured to celebrate the winners of this year’s Health and Medical Research Awards – an inspirational mix of researchers, advocates and philanthropists whose work is both lifechanging and sector-defining.” Federal Health Minister Greg Hunt delivered the keynote address at the event. NSW Parliamentary Secretary for Health James Griffin MP (representing Minister Brad Hazzard) presented the Health Services Research Award, the Hon Gabrielle Upton MP co-presented the Data Innovation Award and Dr Fiona Martin MP co-presented the Griffith Discovery Award. Dr Mike Freelander MP presented the Great Australian Philanthropy Award and

Research Australia CEO Nadia Levin said the standard of the finalists was a testament to the diversity and drive of Australia’s research sector and its role as a global leader in health and medical innovation. “Australian researchers are continuously making new discoveries that transform our understanding, prevention and treatment of disease,” Ms Levin said. “These incredible scientists have demonstrated astounding commitment to our collective human progress, while making tangible improvements to our individual health, wellbeing and health services. “Throughout the pandemic, Australia’s health and medical 2022 | INSPIRE 023

for medical research and improving the experience of patients in hospital. The Data Innovation Award – sponsored by the Bupa Health Foundation – was awarded to Dr Tracy DuddingByth (Hunter New England LHD) for her work using facial recognition technology to help investigate and diagnose syndromic intellectual disability. Professor Brett Mitchell (Newcastle University) received the Health Services Award – sponsored by the NSW Government – for his leading work in infection control, including research helping to drive greater use of antiseptics to prevent infection in hospital settings.

Zali Steggall OAM MP presented the Advocacy Award. Dr Brendan Murphy received Research Australia’s flagship award, the prestigious Peter Wills Medal, in honour of his leadership during the COVID-19 pandemic. As the Government Chief Medical Officer, Professor Murphy provided expert advice to Australian governments that helped spare tens of thousands of Australian lives in the first wave of the pandemic and strengthened Australia’s international reputation in the areas of medical and health research. Professor Murphy continues to serve Australians today as Secretary of the Department of Health. Dr John Parker, founding CEO of Saluda Medical, won the Frontiers Award for his leadership towards developing an innovative treatment for chronic back pain, with potentially beneficial applications for patients with Parkinson’s disease and children with cerebral palsy. Dr Mo Chen (Griffith University) was awarded the Griffith University Discovery Award for his 3D-printed cellular nerve bridge system that creates ‘artificial’ human nerves using the patient’s own cells, potentially revolutionising treatment and recovery for more than 21,000 Australians living with a spinal cord injury. On behalf of their family, Andrew and Jean Miller graciously accepted the Great Australian Philanthropy Award for the extraordinary generosity they have shown through the Miller Foundation since it was established in 1974. More than $100 million has been bestowed through the foundation across a spectrum of medical research, including work focused on maternal and child health, cancer, infectious diseases such as malaria, heart disease and diabetes, indigenous health, and research programs addressing socially disadvantaged vulnerable young people and children. The Advocacy Award was presented to the Chimera Legacy Foundation, established by Damien Thompson and his parents Bob and Debbie, recognising their tremendous efforts towards raising awareness and funds 12

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The GSK Award for Research Excellence was jointly awarded to Professor Jamie Cooper and Professor Rinaldo Bellomo (Monash University). Professor Mark Febbraio (Monash University) was also recognised as winner of the 2020 award. Ms Levin said that each of the award categories was competitive, and every nominee displayed remarkable achievements in their respective fields. “On behalf of Research Australia, I would like to thank all our nominees, nominators and members for helping make this night a memorable celebration of the astounding ability and dedication within this sector,” she said. “I would also like to thank the organisations who have supported the awards, including the NSW Government, Bupa Health Foundation, Griffith University, Australian National University and GSK.” Supporting research is as important as the research itself.

CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

THE FULL LIST OF 2020-21 RESEARCH AUSTRALIA AWARD WINNERS PETER WILLS MEDAL:

Named in honour of Research Australia Director, Peter Wills AC, a great leader whose work led to the inception of Research Australia. The Peter Wills Medal is the flagship award and recognises someone who has made an outstanding, long-term contribution to building Australia’s international reputation in areas of health and medical research and fostering collaboration for better health. WINNER: Dr Brendan Murphy, Secretary of the Department of Health.

FRONTIERS RESEARCH AWARD:

Sponsored by Australian National University, this Award recognises the success of innovative health and medical research that extends existing knowledge, boundaries and/or understandings within health and medical research. WINNER: Dr John Parker, Founding CEO of Saluda Medical

GRIFFITH UNIVERSITY DISCOVERY AWARD:

This Award recognises an early career researcher (anytime from qualification but no more than 5 years past PhD) whose paper/patent/discovery has already demonstrated its importance or impact. WINNER: Dr Mo Chen (Griffith University)

GREAT AUSTRALIAN PHILANTHROPY AWARD:

This Award profiles personal philanthropy that is outstanding in its generosity, effectiveness, vision, high impact and transformative quality. The Award recognises and encourages personal philanthropic donations over a period of time by an individual(s) or family to Australian health and medical research. WINNER: Andrew and Jean Miller (Miller Foundation)

ADVOCACY AWARD:

This Award recognises and congratulates exceptional contributions made by research champions who help raise community awareness and understanding about the importance of health and medical research. WINNER: The Chimera Legacy Foundation, founded by Damien, Debbie and Bob Thompson

DATA INNOVATION AWARD:

This Award recognises an individual or team whose innovation is considered to represent one of the most impactful new data innovations in the HMR sector within the past five years. Sponsored by Bupa Health Foundation. WINNER: Dr Tracy Dudding-Byth (Hunter New England LHD)

HEALTH SERVICES RESEARCH AWARD:

This Award is for an individual or team who has provided leadership and made an outstanding contribution to health services research; driven research that has led to a significant improvement in healthcare; and/or has championed the development of the health services research field. Sponsored by the Victorian Government. WINNER: Professor Brett Mitchell (Newcastle University)

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SOCIAL

INVESTMENT TARGETS SUICIDE PREVENTION FOR FUTURE GENERATIONS

Karen Elliff

A shared commitment to innovation was the driving force behind a five-year suicide prevention research partnership between Black Dog Institute and Future Generation Global. 14

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uicide remains the leading cause of death in young people aged 15–24. The death of any young person represents not only the loss of a valuable life but the ongoing loss of their potential—a devastating outcome for their families, friends and the Australian community. Preventing these outcomes is a major challenge in suicide prevention research, one being tackled via an innovative f ive-year par tnership bet ween Black Dog Institute and Future Generation Global.

CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

The Future Generation companies – Future Generation Australia (ASX: FGX) and Future Generation Global (ASX: FGG) – are Australia’s first listed investment companies to provide financial and social returns. The brainchild of renowned fund manager and philanthropist Geoff Wilson AO, these companies invest 1 per cent of their net tangible assets annually into charities that support young people – the future generation. Since their inception in 2015, they’ve invested nearly $53 million into Australian organisations. Black Dog Institute, the only Australian medical research institute to address mental health across the lifespan, is proud to be one of their beneficiaries. Between 2016 and 2021, we received $2.3 million from Future Generation Global (FGG), which focuses its social investments on youth mental health. This funding was used to develop a suite of major, tech-driven suicide prevention initiatives that have the potential to make a lasting impact on young people’s lives.

LEADING THE WAY ON DIGITAL MENTAL HEALTH

Three major projects with significant translation potential are currently underway: Lifebuoy, a smartphone app designed to help young people manage suicidal distress; RAFT, an automated text-message-based intervention for young people who have been discharged from hospital after a suicide attempt; and Socialise, a passive smartphone data collection app to detect social withdrawal – a risk factor for suicide – in teenagers. Led by three postdoctoral fellows appointed using FGG funding, the projects aim to meet young people where they are: on their digital devices. “These app-based interventions delivered to prevent mental illness represent a new and innovative way to deliver evidence-based prevention programs at scale,” says Professor Jennie Hudson, Director of Research at Black Dog Institute. Over the last five years, we’ve delivered compelling results across all three research streams: through a randomised controlled trial (RCT), we showed that Lifebuoy was able to reduce the severity of suicidal ideation in young adults. The app is now being enhanced in preparation for a second RCT that will also explore the role of social media in keeping young people engaged with the LifeBuoy app. A pilot study of RAFT showed that it reduced suicidal thoughts; the next step is a large-scale RCT, with recruitment currently in progress. Finally, insights drawn from the Socialise app were integrated into the flagship Future Proofing study, a world-first, smartphone-based mental health intervention to help predict and prevent anxiety and depression in adolescents. Smartphone data collected by what’s now

called the Future Proofing app is helping to establish an evidence base for the use of digital phenotyping – a machine learning technique to identify data patterns – in predicting the warning signs of mental ill health.

TRANSFORMING MENTAL HEALTH OUTCOMES BY INVESTING IN BIG IDEAS

In establishing the funding partnership, Black Dog Institute’s commitment to truly translational research was a drawcard for FGG. As a leader in both youth and e-mental health, we recognise the importance of delivering mental health solutions to those who need it most, as quickly as possible.

Black Dog Institute has a researchto-translation remit, so it’s about generating effective approaches through rigorous research and then, crucially, getting it into the hands of young people and those who work with them to make a positive difference to their wellbeing and mental health,” says Emily Fuller, Social Impact Manager at the Future Generation companies. For Black Dog Institute, having access to FGG’s steady funding has enabled researchers to test novel ideas at an early stage, as well as to take a longer-term view of their programs. It has also provided a starting point for the acquisition of additional funding as the research matured: collectively, the three projects have attracted over $4.5 million in government, philanthropic and other funding based on the early-stage results. This willingness to invest in less established ideas is the hallmark of the partnership and remains crucial to fostering innovation and breakthroughs in suicide prevention research. It has been a privilege work with FGG towards these new horizons over the last five years. “We often work within very short timeframes because funders want to see outcomes quickly,” says Dr Michelle Tye, a Senior Research Fellow at Black Dog Institute and the Principal Investigator for Lifebuoy. “With the FGG funding, I’ve been able to see this project evolve to address new challenges. “Now that we’ve established that Lifebuoy, as a concept, works, we’ve been able to ask: how can we optimise its effects? It supports you to ask more innovative questions.”

Author: Karen Elliff, Director, Funding and Partnerships, Black Dog Institute

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GETTING TO THE HEART OF SUDDEN DEATH Philanthropy is one of the most powerful catalysts in improving health outcomes for our communities. Support from donors has played a pivotal role in the development of a world-leading cardiovascular research program located within the Centenary Institute.

he sudden and unexpected cardiac death of a loved one is a devastating and traumatic experience – even more so when that death is of a child or young adult. Often the reason is found to be genetically based which has resulted in a heart problem. Up to 1 in 200 Australians suffer from some form of genetic (inherited) heart disease. The Centenary Institute’s Agnes Ginges Centre for Molecular Cardiology was launched in 2002 to further our understanding of the genes that are responsible for these deaths. Not just to provide answers to grieving families but to ensure that appropriate diagnosis, care and treatment can be provided to remaining family members, and to others, that may be suffering from exactly the same genetic issue.

20 YEARS SAVING LIVES

Celebrating its 20th anniversary this year, the Agnes Ginges Centre has established itself as a world-leading cardiovascular program, directly saving lives, improving patient outcomes and changing the future for many individuals, families and communities. This would not have been possible without philanthropic support – and in particular, the philanthropic support of Mrs Agnes Ginges and her husband, the late Mr Berel Ginges. Their financial investment, facilitated by eminent cardiologist Professor David Richmond, who initiated and led donor discussions, was critical to the Centre’s establishment. It was their vision and belief, that they could help to make a difference, that has taken us to where we 16

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are today – celebrating a Centre that operates at the very forefront of inherited heart disease with investigators who are among the very best in the world. Medical research desperately needs philanthropists such as the Ginges family. Yes, we need excellent scientists to undertake new and exciting discoveries, but we also need generous benefactors to support our core work, to fund state-of-the-art technologies and to ensure that we can attract – and retain – the most talented people. It was the Ginges family who provided us with the means to build an integrated research program focused on tackling serious disease.

The Centre was driven by a shared belief that more needed to be done to reduce the number of Australians dying each year from sudden cardiac disease, and to help prevent the tragedy of inherited heart disease, responsible for claiming the lives of three young Australians each and every week. As the head of the Agnes Ginges Centre for Molecular Cardiology since its inception, I’m pleased to say that much has been achieved over our 20 years of dedicated and focused effort, encompassing a team of amazing individuals. This includes over 120 researchers, students, and clinicians, many who have gone on to achieve great

CHRISTOPHE KEREBEL

My Twitter : @chriskere Cardiac cells in a dish used to study CHRISTOPHE KEREBEL Image by Dr cardiomyopathies.

Seakcheng Lim and Ms Mira Holliday.

Professor Chris Semsarian AM

success across Australia, Europe, the USA and UK. A key feature of the Centre has been career development and mentoring, especially in women, and promoting gender equity and diversity. Crucially, we can already see our research directly reducing sudden cardiac death in our communities. Our new gene discoveries are being used as improved diagnostic tools, guiding prevention strategies such as implantable cardioverter defibrillator therapy, and we are actively involved in improving public health measures such as CPR Training and public access defibrillators. One of our Centre’s most celebrated breakthroughs was the identification of specific genes causing sudden death in people under the age of 35 years. We also discovered that genetic testing was able to identify cardiomyopathies in nearly a quarter of sudden cardiac arrest survivors. Knowledge such as this is critical to improving care and clinical outcomes for patients and at-risk family relatives so that we can focus on initiating prevention and treatment strategies.

THE FUTURE

As is always the case, further research is required in this incredibly complex health space. We’ve been able to unlock some of those complex genetic sequences linked to disease and death, but we need to continue to delve deeper. We know that there are around 40 cardiovascular conditions caused by underlying genetic faults. There are certainly more out there waiting to be found.

The fact that we’ve been able to come so far is testament to the belief that the Ginges family had in us right from the start. They supported us to make a difference and they saw the potential that our critical life-saving activities could make. I am extremely proud that our Centre carries the name of Agnes Ginges, our most loyal supporter. Philanthropy has been absolutely central to all that we have been able to achieve – and all that we will continue to achieve. We will continue our basic science, clinical research and public health initiatives, and continue to make a difference.

Every day we do so, I express my sincere appreciation to the philanthropists who made it possible, with our ultimate goal to improve the care of patients with inherited heart disease and prevent young sudden cardiac death in our communities. Professor Chris Semsarian AM is Deputy Director at the Centenary Institute and Director of the Agnes Ginges Centre for Molecular Cardiology. He is a cardiologist at Royal Prince Alfred Hospital and University of Sydney, with a specific research focus in the genetic basis of cardiovascular disease. A focus area of his research is in the investigation and prevention of sudden cardiac death in the young, particularly amongst children and young adults. 2022 | INSPIRE 023 17

CP COMMONS

About a third of individuals with CP studied to date have a known genetic cause, and up to another third of those tested have genetic changes that may explain their CP, but without adequate supporting evidence to be certain. 18

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CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

A world-first collaborative database driving understanding of the genome’s role in causing cerebral palsy

erebral palsy (CP) is the most common physical disability in childhood, affecting one in every 700 babies born in Australia each year. It is a lifelong condition affecting movement and posture. One in four children with CP can’t speak and one in three can’t walk.

UNDERSTANDING THE ROLE OF GENOMICS IN CP

The genetic basis of CP is highly diverse. More than 300 genes are thought to be involved, including more than 500 different genetic changes, the majority of which are unique to a single individual with CP. Identification of genomic causes of CP improves our understanding of the underlying neurobiology of CP, providing opportunities to study CP pathogenesis, drive biomarker discovery, and develop novel interventions to improve the lives of people living with CP and their families. Despite huge gains over the past two decades in CP genomics research, there has been a continued bottleneck for both discovery and translational research. Whilst there have been many individual efforts to examine the genome’s role in CP, these continue to occur in predominantly “siloed” projects, with insufficient numbers, and in non-standardised formats, prohibiting easy exchange.

One way to resolve this is to combine these data sets and compare findings. This way, rare findings become more obvious, and we can accumulate better evidence of the genes and the variants involved in CP aetiology. A GLOBAL INNOVATION TO IMPROVE RESEARCH OUTCOMES

The CP Commons was established to tackle data sharing challenges and accelerate scientific discovery in CP genomics research. The CP Commons acts as a clearinghouse for clinical and genomics data from people living with CP. By centralising these data and resources, the CP Commons means authorised researchers from around the world won’t have to query multiple databases to find the resources they require. Instead, they will be provided

the opportunity to collaborate with others; pool data to create larger cohorts; reuse data to interrogate areas of the genome not yet investigated; and identify similar cases with matching clinical and genetic features.

FUTURE-FOCUSED PHILANTHROPY

Generous support from The Ian Potter Foundation and Perpetual Trustees enabled the CP Commons to be established as a truly collaborative project, initiated by the International Cerebral Palsy Genomics Consortium (ICPGC) which comprises more than 200 researchers from 75 institutes in 20 countries and is dedicated to accelerating the understanding of the genome’s role in CP. CP Commons is a project of the ICPGC, with Cerebral Palsy Alliance (CPA) assuming the role of ongoing data custodian.

FLEXIBILITY HELPED DELIVER OUTCOMES

COVID impacted this project in multiple ways, and flexibility and understanding from our funders was key to ensuring the project could continue. Following an external cyber-security audit, the CP Commons was moved from its original home at the University of Adelaide to Amazon Web Services (AWS) and work commenced with an AWS partner. Unfortunately, COVID took its toll on the original AWS partner and after a three-month period of deteriorating outcomes and delays, the partnership was concluded. Extensive due-diligence followed, and a new partner, Kablamo, was recruited and development resumed. An extension to the project deadline based on these impacts meant the project was able to move forward and achieve outcomes.

CP COMMON LAUNCHES

In November 2021, the CP Commons officially launched, alongside a report and user guide for researchers and clinicians. The next phase of the CP Commons project is to combine and harmonise existing data of people with CP.

Author: Yana Wilson, Research Officer Cerebral Palsy Alliance Research Institute.

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WORLD-FIRST RESEARCH ON URINARY BIOMARKERS FOR MOTOR NEURONE DISEASE

Pioneering work on human fluid-based tests for motor neurone disease (MND) in progression would not have been possible without the investment from the philanthropic organisations MND Research Australia (formerly MNDRIA), FightMND and USA philanthropy (ALS Association). This philanthropic funding has also led to larger grant success from the US government National Institute of Health. 20

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CHRISTOPHE KEREBEL

motor neuron disease (MND) diagnosis means nerve cells that control muscles involved in moving, swallowing and breathing are getting sick and dying and almost always results in death within 2-3 years, from an inability to breathe. In Australia, two Australians are diagnosed with MND and two people die every day, while almost 2000 people live with the disease. (https://www2.deloitte. com/au/en/pages/economics/articles/economicanalysis-motor-neurone-disease-australia.html) For the past 11 years, Associate Professor Mary-Louise Rogers and her team (notably Dr Stephanie Shepheard and Neurologist Assoc Professor David Schultz) from Flinders University Health and Medical Research Institute (FHMRI) have focussed their MND research efforts on identifying and validating urinary biomarkers for MND progression. Currently there is no definitive biomarker for measuring progression of the disease; MND progression (especially in clinical trials) is monitored using the revised ALS Functional Rating Scale (ALSFRS-r) questionnaire to observe symptoms, and physical tests which look at things like hand muscle strength and breathing. The ALSFRS-r can be fairly insensitive; new and more sensitive biomarkers are needed.

BIOMARKERS IN URINE

A biomarker found in urine, which is easy and painless to collect, provides an objective measure to assess clinical trials. The first biomarker the team examined was a generic marker of neurodegeneration, a protein fragment called p75ECD, which is shed from nerves when they are damaged and is excreted in urine. They were the first to describe this urinary test for MND progression, publishing in several journals including Neurology, a top ranking journal in the field(https:// pubmed.ncbi.nlm.nih.gov/28228570/). The research involved a long-standing collaboration with Professor Michael Benatar, from Miami University. This breakthrough appeared in this magazine in 2017 (https:// issuu.com/researchaustralia/docs/inspire_issue_6). Publication of these promising results in such high-ranking journals led to the establishment of an MND biomarker facility at FHMRI with an innovative, high-throughput robotic test for urinary p75ECD funded by FightMND, inspired by pioneering co-founder Dr Ian Davis. The facility is enabling validation of the test and is being utilised for a number of current or recently completed MND clinical trials in the USA and Australia. An example of this was the use of the urinary p75ECD biomarker in a trial of antiretroviral treatment for MND (published here: https:// pubmed.ncbi.nlm.nih.gov/31284774/ ), that supported the use of urinary p75ECD in the next phase of testing of the anti-retroviral in a large placebo-controlled trial that has recently commenced.

My Twitter : @chriskere CHRISTOPHE KEREBEL

Associate Professor Mary-Louise Rogers was also hunting for biomarkers that could describe more specific processes that happen in MND. One of these processes is inflammation; a candidate investigated is a small molecule neopterin, released from immune cells in the periphery, and brain, in response to pro-inflammatory signals. Gaining funding from MND Research Australia and FightMND enabled her group to investigate neopterin as a biomarker for MND. Urinary neopterin had previously been suggested as a biomarker in MND, but, had not been extensively studied. Her research group found urinary neopterin levels increased each month as the disease progressed indicating it may be a useful progression marker. Again, her work was a collaborative study with Prof Benatar and joining this collaboration was Prof Andreas Malaspina from the University College London, who is also a world leader in biomarkers for MND. The research team found the concentration of neopterin was higher in patients with MND compared with controls. Neopterin levels also correlated to the ALSFRS-R. The researchers tracked the progress of 29 people with MND, measuring neopterin and p75ECD in urine samples previously taken from people with MND at 3–6-month intervals over 2–3 years. Their urine samples showed a steady increase of neopterin levels each month (in addition to p75ECD). The further MND progressed, the more neopterin was found in the urine. These finding were published in the prestigious European Journal of Neurology in February 2022r (published here: https://pubmed.ncbi.nlm.nih. gov/34967083/)..

ALTERNATIVE TO SPINAL TAPS

Urinary neopterin could be a predictive biomarker of immune dysfunction and be particularly useful in trials that target inflammation and immune dysfunction in MND. Urine, being a non-invasive source of biomarkers, also has advantages over other inflammatory biomarkers obtained by patients having to undergo invasive spinal taps for cerebrospinal fluid (CSF). The FHMRI MND biomarker program is unique in using urine from people living with MND as a non-invasive test source, aiming to build tools to help identify effective treatments for MND. This research was supported (2010-2021) with Grantin-aid/ Innovator funding from MND Research Australia, IMPACT and equipment Grants from FightMND (2018, 2020-22), and additional funds form the ALS Association and the National Institutes of Health (2015-)

Author: Associate Professor Mary-Louise Rogers at Flinders University

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PHILANTHROPY AND FAMILY INSPIRES WORLD LEADING STROKE RESEARCH Philanthropy in medical research embraces bold visions and the willingness to explore big ideas that have yet come to fruition or are thought of as impossible.

hilanthropy provides the empowerment, seedfunding and capacity building for researchers to think boldly and act with confidence to make discoveries, like that of stroke researcher Professor Julie Bernhardt of the Florey Institute of Neuroscience and Mental Health. Professor Bernhardt was just 16 when her uncle Barry experienced a stroke. The saying goes that stroke doesn’t just affect the individual but affects families, which was certainly true for Barry, his wife Ronda and their sons. It also profoundly affected Professor Bernhardt, who was inspired to become a physiotherapist and dedicate her career to stroke research after watching her uncle’s rehabilitation and recovery journey. As Julie watched her uncle Barry adapt to challenges after his stroke including movement, loss of sensation and difficulty speaking, and then his gradual return of skills and function, she became fascinated with the brain. Frustrated with the absence of evidence to help make 22

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decisions about the best treatments to improve recovery, she completed a research training degree (PhD) in motor control in the late 1990s. This equipped her to develop and study new ways to treat people with brain injury to improve their recovery. Over the years, Ronda and Barry encouraged Julie’s work as she quickly developed an acute sense of the long-term impact of stroke and the importance of striving for good quality of life despite the challenges it presented. Through her, Ronda also understood that more funding is needed to accelerate the rate of finding better treatments for people who have experienced stroke. Despite the common and devastating effects of stroke, philanthropic support for stroke research has historically been surprisingly modest.

A COMPREHENSIVE YOUNG STROKE SERVICE

Now 91 years old, Rhonda’s interest and philanthropic support for Julie’s work has been constant and impactful. Over the past seven years, Julie’s team have investigated

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Professor Julie Bernhardt and Rhonda Hall.

and identified the major unmet needs of younger people who experience stroke. Thanks to this foundation of research, Professor Bernhardt and Professor Vincent Thijs, Co-Head’s of the Stroke Theme at the Florey Institute, are now able to develop Australia’s first comprehensive young stroke service. The vision Julie and Vincent have for the project is to create a service connecting people aged 18-45 who have experienced a stroke with the care providers and peer support they need to enable their long term recovery.

This collaborative project will build, test and embed an innovative, digitally-enabled young stroke service to overcome geographic boundaries and better meet the long-term care need of young people who experience stroke,” said Prof Bernhardt. “By streamlining what are currently fragmented services, we hope to connect young people who experience

stroke to the personalised services they need and stay connected with them for five years to follow and support their stroke journey,” she added. Philtranthropy often supports the generation of research evidence needed to attract grants from government or commercial partners which help to further knowledge, translate discoveries into clinical treatments or progress to clinical trials. It is a driving force behind the ‘bench to bedside’ approach in medical science and is a key part in every researcher’s journey to complete bold, impactful investigations that improve the human condition – the core mission of a scientist’s life-long endeavour.

Authors: Eric Cheng, Executive Manager of Philanthropy and Fundraising, and Professor Julie Bernhardt AM, Co-Head of the Stroke Research Theme, from the Florey Institute of Neuroscience and Mental Health.

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ACHIEVING IMPACT TAKES TIME THE VALUE OF SHARING THE RESEARCH JOURNEY

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CHRISTOPHE KEREBEL

@chriskere In 2018, Telethon Kids built the pre-cursorMy to Twitter Grow :Impact Globally, researchers and CHRISTOPHE KEREBEL CHRISTOPHE KEREBEL (Momentum) to collate and provide a single source of truth funding bodies are increasingly for research information. required to communicate the COMMUNICATING IMPACT Dr Tobias Schoep, General Manager and Founder of impact of their activities to Grow Impact, said the software was developed to meet society and demonstrate return the need for a more effective way of telling the whole story of how research makes a difference. on investment for funders and “Grow Impact is a sof t ware that suppor ts longdonors. term partnerships between researchers, researcher

he traditional approach to this has been to wait until research is completed and then share outcomes in industry publications and via case studies. This approach, however, potentially limits the reach and opportunities to engage and attract broader stakeholders (including funding partners) and articulate and demonstrate the impact that research can have. Telethon Kids Institute (Telethon Kids) is a large medical research institute based in Western Australia. Its Vision is to bring together community, researchers, practitioners, policy makers and funders, who share its mission to improve the health, development and lives of children and young people through excellence in research. Importantly, Telethon Kids wants knowledge shared and applied so it makes a real-world difference. In 2016, Telethon Kids adopted a variant of the Canadian Academy of Health Sciences’ impact framework which proposed new indicators and metrics to define the impacts of health research and improve the return on investment. Its aim was to advance knowledge, build capacity in the health and medical research sector, inform decisions in the real world, and have health, social and economic impact. Professor Jonathan Carapetis AM, Director of The Telethon Kids Institute and leading paediatrician and infectious diseases specialist, led its adoption. “Research doesn’t just happen; it takes many years to reach the point where it has an impact on the real world,” Professor Carapetis AM said. “If we wait until we have an outcome to share, however, we are missing an important opportunity to help our stakeholders understand how their support makes a difference throughout the research, as well as what went into the discoveries that directly improve children’s lives.” Telethon Kids identified the need to expand the conversation about research impact beyond publications, guidelines and therapies and instead focus on sharing the stages of research. They soon realised, however, that much of the “journey” information lived with the researchers and was not formally or consistently captured in a central repository.

organisations and their funders, so they can work together to make a real impact,” Dr Schoep said.

The information housed in Grow Impact allows research organisations to configure their own impact framework and talk about their successes beyond publications.” Using this information, Telethon Kids now publish an annual Impact Report where they share how research translates into real-world change for their stakeholders, highlight impactful collaborations, showcase initiatives to shift the paradigm and outline projects in the pipeline. “Our annual Impact Report encapsulates the breadth and depth of the impact of Telethon Kids’ research translation,” Professor Carapetis said. “Information from Grow Impact is included in the Impact Report to show stakeholders how our research is improving child health and development” “I have found the metrics from Grow Impact a very powerful tool to demonstrate how our researchers affect real-world change. The relationships that the Impact Report help us develop are integral to the reputation and financial sustainability of our organisation” Grow Impact currently works with research institutes (such as the Institute for Respiratory Health) and funders (such as Royal Perth Hospital Foundation) to help research organisations and funders track, understand and communicate the impact of the research that they support. “Grow Impact is a gamechanger for research, allowing transparency and context so everyone involved in the process can see progress towards clear goals. I’m excited about the opportunities it presents to health and medical research, as well as other research areas,” Dr Tobias Schoep said. For more infor mation a bout G row Impact, v isit www.growimpact.com.au For more information on the Telethon Kids Institute, visit www.telethonkids.org.au

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PHILANTHROPY

HELPS RESEARCHERS BUST AUTISM MYTH

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New Australian research has CHRISTOPHE KEREBEL helped shed light on the autismgut microbiota relationship CHRISTOPHE KEREBEL My Twitter : @chriskere CHRISTOPHE KEREBEL generous donors has sustained my group by allowing and philanthropy played an me to pursue ideas and keep my research staff employed important role in making it even when we missed out in highly competitive grant rounds.” possible by supporting the “Mater Foundation provides us with a safety net and researcher who led the study. launching platform. Its financial support allows Mater

eaningful research that advances knowledge requires significant and consistent longterm investment from both government and philanthropic sponsors if it is to lead to tangible health and community impacts. A recent study published in the prestigious journal Cell on the relationship between autism, diet and the gut microbiota, led by Mater Researchers Associate Professor Jake Gratten and MD-PhD student Chloe Yap, is a prime example. This study showed that contrary to the popular view that the gut microbiota contributes to autism, changes in the microbiota in autistic children are a downstream consequence of more selective dietary preferences (that is, “picky eating”). This suggests that autism-gut microbiome associations are more a case of “mind over microbes” than the other way around. These findings put the brakes on an expanding industry marketing unproven microbiome-based interventions for autism and highlight the importance of supporting parents at mealtimes to maintain a healthy diet for children on the spectrum. The study involved researchers from around Australia, including at Mate r Re se arch, T he Uni ve r sit y of Queensland, Queensland Children’s Hospital, Wesley Medical Research, Telethon Kids Institute, La Trobe University, the University of New South Wales and Sydney Children’s Hospital. The team analysed dietary data and genetic material from faecal samples of 247 children, including 99 children diagnosed with autism spectrum disorder (ASD), from the Australian Autism Biobank and Queensland Twin Adolescent Brain study.

Research to keep researchers employed as they bridge from one competitive grant to the next.”

Mater Research Executive Director, Professor Maher Gandhi said that philanthropic support had also allowed Mater Research to pursue important partnerships, including with the Autism CRC which funded the autismgut microbiome study.

Donations made through Mater Foundation support a significant proportion of Mater Research’s activity, allowing our world-renowned research teams to make discoveries that lead to improved patient care and quality of life.” “This assistance to get runs on the board often means our researchers are more likely to have Federal grant success in the future”. In the 2021 financial year, Mater Research received donations of more than $12 million, a majority of which came from the Mater Foundation. Philanthropic funding continuity encourages and enables Mater Researchers to pursue high risk, high return research strategies that improve competitiveness of Mater Research applications during the grant peer-review process, and ultimately accelerate health and medical research innovation.

PHILANTHROPY SUSTAINS RESEARCH

While funding for the study came from the Autism Cooperative Research Centre (Autism CRC – the world’s first national cooperative research centre effort focused on autism), A/Prof. Gratten’s leadership of the project was enabled by generous philanthropic support from the Mater Foundation to his Cognitive Health Genomics Group at Mater Research. “Research can be a difficult and uncertain sector because each new study needs money and if we don’t secure grant funding, then a potentially great idea will not be investigated, or an existing project will have to be abandoned,” A/Prof. Gratten said. “The support from Mater Foundation through their

Author: Associate Professor Jake Gratten is the leader of the Cognitive Health Genomics group at Mater Research.

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ADVERTORIAL: GSK

GLOBAL INNOVATION IN INTENSIVE CARE RRESEARCH WINS AUSTRALIAN DUO THE GSK AWARD FOR RESEARCH EXCELLENCE.

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CHRISTOPHE KEREBEL

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ach year, more than 120,000 Australians require critical care.1 The COVID-19 pandemic has seen increased admissions to intensive care units (ICUs) over the past two years, putting immense pressure on hospitals and health care systems. Despite these stresses, patients in ICUs continue to receive the highest levels of care and support. This is due in no small measure to the efforts of two local researchers who have helped transform approaches to treatment of critically ill patients, not just in Australia, but worldwide. Professor Jamie Cooper and Professor Rinaldo Bellomo, Co-Directors of the Australian and New Zealand Intensive Care Research Centre (ANZIC-RC), have been acknowledged for their global leadership and innovative research in critical care medicine with the prestigious GSK Award for Research Excellence. In a collaboration spanning 20 years, Professor Cooper and Professor Bellomo have pioneered landmark patient trials that have transformed clinical guidelines, contributed hundreds of millions of dollars in savings to healthcare systems and significantly improved outcomes for patients with conditions like sepsis, traumatic brain injury, acute kidney failure and acute respiratory failure. “We are driven by wanting to ensure the best care for critically ill patients and finding better ways to treat the conditions they are experiencing,” said Professor Bellomo, Director of Intensive Care Research and Staff Specialist in Intensive Care at Austin Health.

Collaboration is at the heart of medical research. This is a moment we can celebrate the efforts of our global and local colleagues, industry and patients who so generously participate in clinical trials.” CLINICAL TRIALS FOR INTENSIVE CARE

Profe s so r C o o p e r a nd Profe s so r B e ll o mo have championed large, multi-centric collaborative investigator initiated clinical trials, which have helped establish Australia as the epicentre of world-class research in intensive care.

“Our trials don’t just look at the clinical impact of a n inte r ve ntion we a re te sting, but a lso the pharmacoeconomic or health economic impact, so there is a global understanding of the effect of what we do.” said Professor Cooper, who is Senior Specialist in Intensive Care, at The Alfred Hospital, and Sir John Monash Distinguished Professor at Monash University.

One of the latest projects being undertaken under the Professors’ directorship at the ANZIC-RC is contributing to global understanding of the impact of highly-specialised extracorporeal membrane oxygenation (ECMO) technology in the management of cardiac arrest, severe cardiac failure and respiratory failure including COVID-19 patients. ECMO pumps and oxygenates a patient’s blood outside the body, completely substituting the function of the lungs and heart.2 In places like US and Australia, it has saved numerous lives of COVID-19 patients who were not improving on ventilators. The Professors said the $80,000 in prize money that comes with the award would be used to support their team of early to mid-level career investigators working on ECMO and other innovative projects. The GSK Award for Research Excellence was presented at Research Australia’s Health and Medical Research Awards in Sydney. The GSK Award for Research Excellence is one of the most prestigious awards available to the Australian medical research community. It has been awarded since 1980 to recognise outstanding achievements in medical research and facilitates career development with potential importance to human health and Australian research. It was first presented as the Wellcome Australia Medal and then as the Glaxo-Wellcome Medal before receiving its current name.

1 Warrillow, S., Raper, R. The evolving role of intensive care in healthcare and society. Med J August 2019; 211 (7): doi: 10.5694/mja2.50340 2 Liverpool Hospital, Intensive Care Unit ECMO Learning Package, 2016, accessed November 2021 at https://www.aci.health.nsw.gov.au/__ data/assets/pdf_file/0007/306583/ECMO_Learning_package.pdf

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A MONUMENTAL INVESTMENT INTO STEM CELL MEDICINE The Murdoch Children’s Research Institute is one of three world-leading research institutes that have been backed by the Danish Novo Nordisk Foundation to deliver innovative stemcell driven therapies to transform the lives of people with incurable disease.

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CHRISTOPHE KEREBEL

he Novo Nordisk Foundation, one of the largest foundations in the world, has made an unprecedented donation of 300 million Euros to establish a collaborative translational research centre for stem cell medicine. Led by newly-appointed CEO Professor Melissa Little from the Murdoch Children’s Research Institute (MCRI), the Novo Nordisk Center for Stem Cell Medicine (dubbed reNEW) was established as a tripartite consortium across three international sites: the University of Copenhagen in Denmark; Leiden University Medical Center in The Netherlands; and Murdoch Children’s in Australia. Together, these three world-leading institutes will combine their strengths in biomedical research excellence to develop innovative stem cell therapies for incurable diseases. We are living in world where the global population is the highest it has ever been and life-expectancy is (arguably) at its peak. But, despite promising advances in medical technologies and healthcare, these ageing populations are dealing with a concerning rise in chronic illnesses, including chronic kidney disease, cancer, diabetes, and Parkinson’s disease. The rising incidence of incurable disease worldwide is exposing major gaps in our healthcare systems. An ongoing challenge for the biomedical research sector is a barrier to pushing discovery-based science to research outcomes. Indeed, the commercialisation pipeline is increasingly slow, risky, and costly, with more than 95% of products failing to reach the market. The Novo Nordisk Foundation wants to accelerate this bench-to-clinic pipeline to help more people living with chronic disease. To that aim, they have committed 30 million Euro every year for the next 10 years to establish the reNEW Stem Cell Medicine Center. This pledge marks the biggest donation made by the NNF and their first investment into an international tripartite consortium as a dedicated step to overcome the commercialisation barrier and achieve targeted research outcomes from stem cell medicine. “Stem cell medicine truly promises to be a game changer when it comes to addressing some of the major health challenges facing the world today… whether in the form of new medical technology or new forms of treatment for the benefit of patient”- Mads Kragsgaard Thomsen, CEO of the Novo Nordisk Foundation.

STEM CELL MEDICINE

Stem cell medicine uses stem cells to model human diseases, regenerate tissues and create ‘designer cells’ that can improve our ability to diagnose, treat, and even prevent human disease. As opposed to animal models, human stem cell-derived models offer a more accurate replica of human tissues and organs for non-invasively

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studying disease mechanisms. They offer the opportunity for preclinical testing of drugs, including predictive technology of individual responses, that will usher in the era of personalised medicine. Such stem cell medicines will be transformative, representing a genuine medical frontier as well as offering an exciting opportunity for economic growth. reNEW will build upon the research expertise of the three Centers in disease modelling, gene editing, and regenerative medicine, to expedite the innovation of novel stem cell therapies for both adult and paediatric diseases. Each Center or “node” of the international consortium will offer research expertise, facilities, and technologies, to provide a mutually-reinforcing, collaborative network.

The reNEW research faculty include a carefully chosen team of experienced leaders in cell and gene therapy, as well as a new generation of emerging scientific superstars to build critical mass for delivering targeted research outcomes. The concept of reNEW was the pioneering vision of Professor Little, Chief Scientist and Theme Director of Cell Biology at the Murdoch Children’s Research Institute, located at the Royal Children’s Hospital in Melbourne. Amongst many accolades, Professor Little is internationally renowned for her expertise in kidney development and regeneration using pluripotent stem cells, and is currently President of the International Society for Stem Cell Research. (Professor Little is also a nonexecutive director of Research Australia.) “reNEW will enable outcomes across the breadth of stem cell medicine — new drugs based on stem cell disease models, cell and tissue therapies, and also gene editing — and take research outcomes all the way to the clinic.” – Professor Melissa Little, CEO and Executive Director of reNEW. Professor Little’s capacity to govern and oversee the NNF Stem Cell Medicine Center is underpinned by more than 30 years of laboratory and leadership experience. Her belief is that reNEW will not only build capacity for targeted research outcomes but also attract additional international investment. “This is a unique model and fantastic opportunity, that will put Australia on the map for regenerative medicine”. – Professor Little.

Author: Katherine Gill, Philanthropy Manager, Murdoch Children’s Research Institute, Victoria, Australia.

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COLLECTIVE

INTELLIGENCE TO OPTIMISE PATIENT OUTCOMES IN AUSTRALIAN RHEUMATOLOGY

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Rheumatic and musculoskeletal diseases account for the highest non-fatal disease burden in Australia.

ifelong care is often required, and for patients with incurable and progressive autoimmune conditions like rheumatoid arthritis or ankylosing spondylitis, this often involves treatment with highly expensive biological therapies. Many therapies are not fully understood until they are administered in the real-world setting in patient pools much larger and more diverse than clinical trials, and after enough time has passed for the trajectory of disease to become evident and long-latency adverse events to emerge. As such, clinicians, policy makers and regulatory authorities are turning to real-world data (RWD) to determine the safe and optimal use of medicines and understand the unmet needs of patients. In Australia, out-of-hospital primary and specialist care is predominantly delivered by private practitioners, including rheumatology which is 80% private. As such, conducting meaningful research requires the assembly of data scattered around Australia in independent clinics with no common thread between them. Disease registries were traditionally the gold standard for capturing RWD, but they are time consuming and expensive, rarely reaching maturity. These challenges are exacerbated if representativeness and critical mass depend on recruitment in private clinics where time and resources to obtain patient consent and enter data are limited. Before the Medical Research Future Fund was established, Australian public funding to support research was primarily invested in the academic setting, inadvertently omitting most real-world knowledge from Australian research. Nevertheless, it is simply unrealistic for a registry to be maintained for every disease, and competitive shortterm grants are not a stable foundation on which to build longitudinal research programs that inform clinical care and patient outcomes. 2022 | INSPIRE 023 33

BREAKING DOWN THE DATA SILOS

In 2009 a group of private rheumatologists realised that an enormous volume of data was being generated daily in private clinics around Australia. On an individual level this information holds little value, but when data silos are broken down and clinical knowledge is pooled, a substantial breadth of intelligence is created. The necessity for innovative methods to capture RWD at scale and the potential for real-world collective intelligence to optimise patient outcomes sparked the establishment of OPAL Rheumatology. In partnership with Australian technology company, S4S Pty Ltd, and with funding through an unrestricted educational grant from Roche, a bespoke electronic medical record was customised for rheumatologists to efficiently document each patient’s condition during the consultation, while allowing data to be extracted and aggregated for near real-time research.

Today 113 rheumatologists from 43 clinics in 6 states and territories are using this software to manage approximately 35-40% of Australian rheumatology patients, and the dataset generated as a by-product of this routine care contains the records of 1.4 million consultations with over 225,000 patients. The OPAL dataset is one of the largest and most comprehensive rheumatology datasets in the world, and at such a scale that even rare diseases are well represented. It is a remarkable achievement for a clinician driven, notfor-profit organisation to generate a longitudinal outcomes dataset of this size from independent clinics, built on a foundation of technology and in compliance with ethics and privacy regulations. To overcome the drawbacks of registries and ensure scalability and sustainability, OPAL has made research participation a ‘value-add’ rather than a ‘trade-off’. By embedding data entry within the consultation, research data is generated passively and indefinitely for over 200 conditions as a by-product of routine care. The risk to patient confidentiality of contributing to the aggregated OPAL dataset is significantly reduced by extracting only deidentified data from the clinic. This removes recruitment barriers and allows clinicians and patients to contribute to posterity without unnecessary risk or inconvenience.

BETTER CLINCAL CARE AND RESEARCH GO HAND IN HAND

OPAL’s primary focus is providing clinical infrastructure that continually elevates the efficiency and standard of 34

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care, and the comprehensive research dataset generated through this technology is a secondary benefit. By continuously interrogating data captured within the local clinical setting, OPAL has created an integrated model for routine care and research which is constantly learning and improving through knowledge-sharing and investment in clinical tools to facilitate evidence-based chronic disease management. Ultimately, the patient is the main beneficiary as the recipient of high value, lower risk care, which is guided by comprehensive documentation of their disease, its impact on their quality of life and their response to therapies.

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FUNDING FOR THE FUTURE

Despite independently establishing and maintaining a rich, dynamic collection of data documenting the utilisation and outcomes of billions of dollars of care provided by the MBS and PBS, OPAL has not received any public funding. Instead, OPAL’s scale, sophistication, and real-world expertise has attracted global industry investment via research partnerships. To date, this has supported the ongoing activities of OPAL while remaining independent and for the benefit of patients. However, industry’s investment in rheumatology is limited to a handful of conditions, and it is imperative that

Australia has the capabilities to independently examine the safety and effectiveness of high-cost medicines across the whole range. In the future, for the Australian healthcare system to realise the full value of OPAL and secure Australia’s position as a global destination for rheumatology research, public investment in OPAL will be vital.

Author: Dr Tegan Smith, CEO, OPAL Rheumatology Ltd.

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SAFE

HARBOUR GIVEN TO YOUNG SCIENTISTS

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CHRISTOPHE KEREBEL

Donors, philanthropists and Private Ancillary Funds (PAF)s CHRISTOPHE KEREBEL My Twitter : @chriskere CHRISTOPHE KEREBEL are stepping up to fill the Federal Government’s grant funding “Many research organisations must fundraise to shortfall in medical research supplement researchers’ salaries because Australia’s funding system is cyclical despite successful that is taking a disproportionate grant research exceeding the one, two or even five-year funding toll on the careers of young and cycle. “Unless there is reform in the system, Australia will mid-career scientists.

continue to lose brilliant young scientists before their innovative discovery has reached fruition simply because their grant application, was unsuccessful.

Director of Perth’s Harry Perkins Institute of Medical Research, Professor Peter Leedman AO, said the Institute is testing the new Safe Harbour program to see if it can fill a funding gap affecting Australia’s scientific future.

DR OLIVIER CLEMENT

he innovative Safe Harbor program they have funded to retain outstanding young scientists in Western Australia has awarded its first fellowship, $850,000 over three years, to neuroscientist Dr Olivier Clement who researches the molecular mechanisms that regulate memory.

“Early to Mid-Career Researchers are the engine room of laboratories. Typically, each has had 10-15 years intensive post-secondary education and training, averaging an investment of $500,000. “Many early and mid-career scientists’ careers are dependent on their ability to secure research income from the major grant funding bodies such as the National Health and Medical Research council (NHMRC) and the Australian Research Council (ARC) and those grants are extremely difficult to get,” Professor Leedman said. In 2021, Federal Health Minister Greg Hunt announced 248 researchers nationally will share $239m in NHMRC Ideas Grants spanning five years. More than 2600 applicants across Australia were unsuccessful. “That means less than 10% received a grant.

It is utterly unsustainable for scientific discoveries with the real potential for commercialisation to be cut off at the knees by the current vagaries of Australia’s grant funding system. “It is a tragedy for Australia that many EMCRs are leaving the industry because of a lack of career stability. “If we don’t do something to nurture this cohort, I fear we will lose the next generation of scientific pioneers”, he said. The Harry Perkins Institute’s Safe Harbour program aims to support its leading EMCRs with two Fellowships a year valued at $285,715 per annum for three years. In addition, young EMCRs can apply for a small discrete amount to support further learning or research.

“Grant success is heavily influenced by a researcher’s track record, but how do you build a track record if you’re not funded, it is a terrible catch 22,” Professor Leedman said. The inaugural Safe Harbour Fellow, Dr Clement works in the genome biology and genetics laboratory at the Harry Perkins Institute of Medical Research in Perth on the molecular mechanisms that control memory. “Memories make us who we are and are essential for our survival as they help us to predict what’s coming based on past experiences. “They are formed by physical and physiological modifications to the brain. The formation or recollection of a specific memory induces activity in an area of the brain where memories are stored and can lead to genetic changes that are ultimately responsible for the permanent storage, update or forgetting of the originally acquired memory.

My project focuses on understanding the fine molecular processes regulating memory which are critical for the development of diagnoses and treatments for the myriad of neurodevelopmental and neuropsychiatric disorders that result in impaired memory function. “The Safe Harbour Fellowship will give me further time to publish,” Dr Clement said. Dr Clement obtained a Master of Science in molecular and cellular biology from the Ecole Normale Supérieure de Lyon and a PhD in Neuroscience from the University of Lyon and the Lyon Neuroscience Research Center (France). He relocated to Australia and the Harry Perkins Institute of Medical Research in 2016 and in 2018 joined the laboratory of the 2020 joint WA Scientist of the Year Professor Ryan Lister.

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PARTNERS UNITE TO STRENGTHEN POSTPANDEMIC CLINICAL TRIALS SECTOR Leading education providers PRAXIS Australia and ARCS Australia partner to close the critical skill gaps in the clinical trials sector.

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CHRISTOPHE KEREBEL

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RAXIS Australia and ARCS Australia are uniting to deliver two unique programs to close critical skill gaps identified in Australia’s clinical trials workforce. The PRA XIS Australia Clinical Trials Coordinator (CTC) Internship program and the ARCS Grow program address the skill shortages of CTCs and Clinical Research Associates (CRAs). The PRAXIS Australia CTC Internship program aims to prepare a pipeline of job-ready CTCs who have the skills needed for Australia’s post-pandemic clinical trials landscape. PRAXIS Australia will partner with ARCS and other leading industry, research, and clinical trials organisations to deliver the selective, structured, national internship program. The PRAXIS CTC internship program will offer an immersive training model to 15 interns over a 10-month period. The program will target nurses, health science graduates, and other health professionals who are pursuing careers in clinical trials. The ARCS Grow program aims to address the skill shortage of CRAs in Australia’s clinical research sector, increasing the capacity for the sector and ensuring future CRAs possess knowledge and skills that align with national and international standards The Grow program uses a three-pronged approach to increase the skills in the sector. It provides valuable and essential knowledge to working CRAs, provides appropriate skills for supervisors of program participants, and delivers a competency framework based on international and national programs that will ensure the appropriate standards for CRAs are embedded in the Australian vocational training scheme. Both programs will address critical sector skill gaps identified in MTPConnect’s REDI Initiative Skills Gap Analysis (2021). Skill gaps in the Australian clinical trials workforce pose a risk to the sector’s capability and reputation and can result in an array of negative impacts, including the absence of best-practice clinical trial conduct and the inability for clinical trial sites to participate in trials. Strengthening our post-pandemic clinical trials capability will be key to ensuring Australia remains a global leader in safe, effective and efficient clinical trials conduct, and will support the innovation and quality of clinical trials now, and into the future. Adopting a best practice co-design approach, the programs will use existing educational content from partners that is enhanced by input from industry experts and will include a mentorship component to supplement the learning experience. The PRAXIS program will be facilitated and led by subject matter experts and guest lecturers from diverse backgrounds with knowledge gained from specialised experience. This model will 2022 | INSPIRE 023 39

ensure program participants are exposed to diverse perspectives across the sector. Assessment tasks will also be included to develop and strengthen abilities in key competency areas such as planning and critical thinking. The result will be an industry benchmark for CTC training. The internship format strengthens the development of key competencies by allowing participants to implement what they have learnt directly into the workplace setting. On completion of the program, participants will be workplace ready, saving training time and costs for future employers. PR A X I S Au s tr a l i a w i l l c o l l a b o r ate w i th p a r tn e r organisations that represent a broad variety and expertise of the Australian clinical trials ecosystem. The partners are ARCS Australia, The George Institute for Public Health, Maridulu Budyari Gumal (SPHERE), South Australian Health and Medical Research Institute, Sydney Health Partners and The University of Queensland Centre for Clinical Research. By providing the ARCS Grow program, ARCS will deliver professional education that will better enable the effective and efficient translation and commercialisation of health and medical research in Australia and expand the capacity and capability of the research community to undertake translational health and medical research, contributing to the growth of the Australian MTP ecosystem and the Australian economy. ARCS’ Grow Program uses an industry-led, case study driven education approach. ARCS has pulled together a comprehensive competency-based curriculum for CRAs which draws on these skills sets for its delivery. The Grow program will become the local gold standard for professional development of CRAs as it is specifically designed to align with Australian regulations and guidelines. ARCS, through its collaborative approach, has established a network of advisory panel members with broad and extensive experience in the sector to provide guidance and assist with development of ARCS educational content. ARCS will plan and deliver their program in collaboration with companies across Australia’s research, education, and clinical trials sector. The companies in the partnership are PRAXIS Australia, BeiGene Australia, Novotech, Southern Star Research and Mobius Medical. “Time and time again we hear from the clinical research sector that they need more and better qualified CRAs, most recently at the ARCS Workforce & Capacity Summit, said Shanny Dyer, CEO ARCS Australia. Dr Dyer said that she welcomed the funding for these programs that would help to train CRAs and CTCs in Australia to increase the capacity for the sector to attract clinical trials to Australia. “I’m positively excited that, for the first time in Australia, we are able to develop a competency framework for the profession, to take another step in the professionalisation of the sector,” said Dr Dyer. “We are thrilled to be collaborating with our partners on this pivotal program that will help grow the capacity, capability and confidence of Australia’s clinical trials sector”, said Sally Armstrong - CEO, PRAXIS Australia.

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COVID has catapulted Australia’s clinical trials sector to new heights of world awareness. This country is a globally recognised leader for safe, effective and efficient clinical trials. Our reputation is generating increasing demand for a highly skilled clinical trials professionals - this program is a step toward meeting that demand”, she said. Ms Armstrong and Dr Dyer said the collaboration across leading partners is an important advance towards a stronger, more robust clinical trials workforce, with one of the greatest outcomes being the alignment of sector leaders to meet a critical need, with previous competitors becoming collaborators to help solve Australia’s clinical trials skills gap. The first cohort for the ARCS Grow program commence in October this year. The participants will be those people newly employed in a CRO or sponsor company. The second cohort will commence in October 2023. The program will, in parallel, be running a community of practise online so that participants can interact and engage with each other as they undertake their training. Companies will be supported to assist the trainees by a specifically designed course for supervisors to enhance their people management skills. The goal is to provide a boost, particularly to smaller businesses’ who would struggle with the resources to devote to the training program. It anticipated that by 2024 there will also be a competency framework that sits over the training program that will be established as a ASQA accredited program. To align with university graduation timelines, the PRAXIS Australia CTC program will commence in January 2023 and be delivered by the end of 2023. As the program is highly selective, host institutions and interested participants are encouraged to apply now. Authors: Sally Armstrong, CEO, PRAXIS Australia. Dr Shanny Dyer, CEO, ARCS. Kaylie Smith, Marketing and Communications Advisor, PRAXIS Australia. Marion Demann, Director Marketing & Communication, ARCS.

ABOUT PRAXIS AUSTRALIA

PRAXIS Australia is a leading education and training provider that helps build the capability and capacity of the clinical trials, research, and ethics sector through flexible, high quality training solutions at a national and international level.

ABOUT ARCS AUSTRALIA

A RC S i s a n Austra li a n ba se d p rofe s s i o na l organisation which focuses on career long professional development for its members in the therapeutic goods sector. ARCS provides education, competency building and information sharing within communities of practice, and targeted advocacy and collaboration with a range of stakeholders.

SAVING LIVES TAKES GUTS – OR MORE SPECIFICALLY, STOOL Australian Red Cross Lifeblood is collaborating with funders and researchers to uncover the potentially life-saving benefits of a healthy individual's stool and how it can be utilised to treat Australians suffering from debilitating and often recurrent gut infections.

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CHRISTOPHE KEREBEL

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hree years ago, Lifeblood saw a life-saving oppor tunit y in the Austr alian healthcare landscape that would offer hope to patients suffering from recurrent Clostridioides difficile infection (rCDI). rCDI is a serious bacterial infection within the gut in some patients after they have been treated with antibiotics. It is pervasive, debilitating, and can even be fatal. Currently, the primar y treatment option is more antibiotics which may need to be used repeatedly and to limited effect. Faecal Microbiota Transplant (FMT) is a procedure where the gut microbiota (a stool donation from a screened and tested healthy individual), is transplanted into the bowel of an individual with rCDI. This attempts to rebalance the gut microbiota in the patient and encourage the growth of healthy bacteria and elimination of pathogenic or harmful bacteria. This treatment is more than 90% effective for patients with rCDI, better than the current antibiotic alternatives, and has seen a reduction in hospital bed days of around 50%. For many patients, this means being able to return to life as normal, which could include the many simple things taken for granted, such as returning to work, taking public transport or caregiving for the family.

PROVIDING LIFE-GIVING PRODUCTS

Lifeblood Microbiome has been set up within the Rotary WA Health Innovation Centre at Lifeblood’s Per th Processing Centre. Rotary’s generosity has enabled Lifeblood to create a dedicated manufacturing facility within the Processing Centre. This has become Australia’s first TGA-licenced facility for the manufacture of FMT products.

patients with rCDI and has utilised its far-reaching logistics network to supply FMT products overnight to hospitals in Queensland, New South Wales and Victoria to treat patients. The results of these treatments have been very positive with many of the patients showing significant overall improvement or even becoming asymptomatic. Without the invaluable investment from our funders – McCusker Charitable Foundation, HBF and Rotary WA – this would not have been possible. Lifeblood is proud to be able to continue to offer these treatments to our partner, Fiona Stanley Hospital, and other hospitals across Australia, and in doing so making a life-changing impact on the lives of Australians.

WHERE TO FROM HERE?

There is a growing body of evidence suggesting that treatment with faecal microbiota may be beneficial for other gastroenterological and non-gastroenterological conditions.

Lifeblood plans to expand its service to meet this need and explore the efficacy of FMT for other disease indications, in partnership with leading Australian researchers and philanthropists. Lifeblood is looking to innovate, streamline its manufacturing process, and develop next generation products (capsules and freeze-dried FMT). This will be achieved by partnering with industry experts and manufacturers.

This specialised facility enables donor screening, stool donation, processing, storage and distribution of FMT. Our incredible staff include registered nurses, scientists and processing technicians who work together to create these life changing FMT products.

Indications requiring next generation products include research programs for ulcerative colitis, Crohn’s disease, and major depressive disorders, as well as continuing to use the current generation product for research into treatment of adverse events resulting from chemotherapy.

Lifeblood has utilised the invaluable knowledge gained from over ninety years supplying blood and blood products to the Australian health care system to set up the Microbiome Program.

Saving lives takes guts, and without the support of funders who are willing to back new and emerging health innovations, this life-changing new product would not be possible. This generosity opens the door to dozens more potential life-saving treatments for Australians and is something we are excited to uncover.

Initially, via a pilot program supplying FMT products to a clinical trial at Perth’s Fiona Stanley Hospital, Lifeblood has shown that the supply of FMT products manufactured under new TGA regulation is both clinically efficacious and cost effective for the health system.

WORKING WITH FUNDERS TO CHANGE LIVES

Lifeblood has seen significant demand from hospitals across Australia for FMT products to treat seriously ill

Authors: Coco Sowada: BSc, BGS from Monash University; Stakeholder Engagement Coordinator at Australian Red Cross Lifeblood. Chris Van Diemen: BAppSc, MHA, Business Design Lead – Microbiome at Australian Red Cross Lifeblood.

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DONOR’S VISION COMES TO LIFE Associate Professor Elaine Sanij

St Vincent’s Institute was founded in the 1950s by a bequest from champion racehorse trainer Jack Holt. Today, a new generation of philanthropists continue to be central to supporting new research and breakthrough discoveries at the Institute. 44

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hen Christine Tarascio AM began her journey with St Vincent’s Institute of Medical Research (SVI) 16 years ago, she had no inkling of the enormous contribution she would make. “One of my sons, Sam, has psoriatic arthritis. When I was invited to join the SVI Foundation Board, I went on a tour of the Institute and the SVI team told me about research they were doing in the field. I would do anything for my children, so I was hooked.” Christine’s respect for SVI’s scientists and their dedication to understanding disease and improving health outcomes inspired her to establish SVI’s Discovery Fund in 2007.

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“During my time with SVI, I’ve witnessed the huge challenge researchers face in gaining sustainable support for their work. I wanted to do something about that, so I set about convincing my contacts to pledge $10,000 a year for five years, with the initial aim of building an endowment of $5 million.”

"Elaine is an incredibly inspiring mid-career researcher, with a vision for change – that’s where our purpose aligns,” says Christine. “I know she’ll do great things for ordinary people who have been dealt a terrible hand. And we intend to continue to further grow the Discovery Fund – its potential is infinite.”

In March 2020, just before Australia’s nationwide COVID-19 lockdown, Christine’s goal was achieved: the Fund had exceeded its original goal.

OVARIAN CANCER’S IMPACT

“There has been a lot of work, and a lot of loyalty from fabulous donors – many of whom are friends – so much so that we are now heading towards $10 million,” she says. “For many, the year 2020 didn’t offer much to celebrate, so it was nice to be able to have something positive to focus on.”

PHILANTHROPHIC SUCCESS LEADS TO NEW OVARIAN CANCER RESEARCH

Every day, five Australian women are diagnosed with ovarian cancer, and three will go on to die from the disease – more than 1,000 women each year. Thanks to Christine’s tireless work, SVI last year established a new laboratory focused on ovarian cancer, led by Associate Professor Elaine Sanij, supported by the Discovery Fund. Elaine uses a pioneering approach to selectively cause high stress inside cancer cells. Her goal is to delay or prevent relapse by overcoming the cancer’s ability to develop drug resistance. “Many of the treatments for women today are virtually unchanged since the 1990s; new and more personalised treatments for this cancer are desperately needed,” says Elaine. “I will push forward the development of new drugs, validate these drugs’ effectiveness in ovarian cancer models, and – when we have strong drug candidates in hand – initiate clinical trials.” Elaine recently celebrated her one-year anniversary at SVI. In addition to her collaborations in DNA repair – with SVI’s Associate Professors Andrew Deans and Wayne Crismani – Elaine has been able to develop a new cross-disciplinary partnership investigating multiple myeloma. “Multipe myeloma is the second most common blood cancer in Australia – almost seven Australians are diagnosed every day, most commonly men aged 60 or older. It can be treated, but patients commonly relapse, and – similar to ovarian cancer – it becomes more and more resistant to treatment over time. While new drugs have emerged to treat this disease, treatment resistance remains an urgent challenge,” says Elaine.

“Ugly” is how Bernadette Dennis characterises ovarian cancer, the insidious disease that claimed the lives of both her mother and sister.

In 1989, my mother was diagnosed with stage four ovarian cancer. She passed away on Christmas Eve that year. Then, in 2003, my sister Pauline was also diagnosed at stage four, with a different form of the same cancer. After several attempts at chemotherapy, doctors advised her there was nothing else that could be done,” Bernadette recalls. “What I realised at that time was that there was not a lot of support for research or new treatments for this cancer. Although there was a 15-year gap between my mother and sister being diagnosed, it was clear that little had really changed.” “My sister Trish started raising funds to support a clinical trials nurse, and some of Pauline’s friends and I joined in this ultimately successful campaign.” This first determined foray into fundraising led Bernadette to become passionate about the transformative impact of philanthropy. She is now a member of the SVI Foundation Board and of the SVI Discovery Fund. “For the many families who have experienced the loss of loved ones to ovarian cancer, it is wonderful to see this specialist research happening at SVI,” says Bernadette. “I know every member of my family will endorse the need for more awareness and discovery, with the ambition of better care, life-expectancy and quality of life for women like my mother and sister.” In addition to the SVI Discovery Fund, Associate Professor Elaine Sanij has Fellowship funding from the Victorian Government through the Victorian Cancer Agency. Her work is also supported by the National Health and Medical Research Council, The CASS Foundation and the St Vincent’s Hospital Melbourne Research Endowment Fund.

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RESEARCH BRINGS HOPE Dr Dannielle Upton is part of a ground-breaking team investigating potential treatments for one of the deadliest childhood cancers. Multiple attempts to secure government funding have been unsuccessful.

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CHRISTOPHE KEREBEL

hilanthropic suppor t from donors to organisations like The Kids’ Cancer Project is vital in helping her and other scientists establish their careers in life-changing research fields. For children with diffuse intrinsic pontine glioma (DIPG), and their loved ones, the diagnosis is devastating. This inoperable form of brain cancer typically affects children aged five to seven who are otherwise perfectly healthy. It is considered incurable, with an average survival of just nine months from diagnosis.

THE LONG ROAD TO A BREAKTHROUGH

One of the problems facing cancer researchers in the past was a lack of tumour samples. This was solved partly thanks to the work of Associate Professor David Ziegler, Group Leader of the Brain Tumours Group, and his team at Children’s Cancer Institute. Dr Dannielle Upton is carrying out a study to further enhance knowledge of this deadly disease. She is determined to improve the very limited treatment options. “DIPG is the most aggressive of all childhood cancers. Current therapeutic strategies are palliative only,” explains Dr Upton. “Innovative treatment approaches are urgently needed to change the statistics from it being incurable, to not just prolonging life but curing this cancer as well.”1 Ziegler, Upton, and their colleagues painstakingly tested thousands of clinically available drugs against DIPG samples. One drug that ticked all the boxes was Auranofin, an existing anti-inflammatory compound previously used to treat rheumatoid arthritis. Dr Upton and the team found that the agent showed potent anti-cancer activity against DIPG cells.

FUNDING ENGAGES GOOD RESEARCHERS IN GREAT RESEARCH

Now Upton is on a quest to find out exactly how Auranofin works against DIPG and identify the best combination of drugs to target tumours. But it hasn’t been easy to get to this point. Although she is five years beyond earning her PhD and has spent time as a reproductive biologist studying female reproduction and cancer, Dr Upton is still considered an early career researcher. Like many at her level, she’s struggled to find financial backing to enable her investigations to go ahead. “Big pharmaceutical companies have little interest in developing drugs for kids’ cancer, and at this point in my career, it’s really competitive to get funding from government agencies,” she says. “In fact, I have been unsuccessful every time I’ve tried for government funding so far.” 1

Dr Dannielle Upton

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Help for Dr Upton to continue her vital work has come in the form of philanthropic support. Thanks to generous donors, dedicated fundraisers and corporate partners, The Kids’ Cancer Project is funding the next steps in this powerful study.

Grants like this allow early career researchers to get our foot in the door,” Upton says. “They encourage us to push our work, grow our independence and establish ourselves.” Financial backing keeps smart, passionate people in the field, supporting scientists who can have a real-world impact on kids diagnosed with cancer, ultimately saving children’s lives. “Thanks to donors, I have foundation funding early in my career and can progress some very important work.” The Kids’ Cancer Project’s CEO Owen Finegan agrees. “Government funding only stretches so far, it’s up to the Australian public and business community to pick up the shortfall to help fund scientific work that has the greatest chance of clinical success.”

A COMMUNITY CREW

The philanthropic support for Dr Upton’s work doesn’t stop there. Throughout May, which is Brain Cancer Awareness Month, groups across Australia will be jumping aboard The Kids’ Cancer Project’s Pirate Day. Pirate Day is an annual national day of dressing up to raise awareness for childhood brain cancer. The Pirate Day community has raised almost $500,000 since 2015. This goes towards scientists who are working to turn the tide on childhood brain cancer survival statistics, further helping those who may have struggled to secure adequate funding from other sources. This year, Pirate Day funds will be directed towards Dr Upton’s work into DIPG. Dr Upton and the CCI team are thankful for the community support the initiative fosters, bringing crucial awareness and funds to such an important field. “Brain cancer is a devastating disease, even more so when it affects children,” Dr Upton says. “Anything that raises awareness will help research efforts with the hope of finding a cure.”

Authors: Original article by Chris Sheedy, edited by Emma Holifield.

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USING THE EYE TO MEASURE THE BRAIN WHY INVESTING IN TRAUMA RESPONSE SAVES LIVES

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CHRISTOPHE KEREBEL

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For Associate Professor Bang Bui and his colleagues, additional philanthropic investment in an idea has made discovery possible.

“Design and manufacture of a desktop-based prototype has provided us with initial proof of concept. This prototype device has informed the design principles for a fully handheld system,” Bang Bui said.

The approach takes advantage of the fact that the pulsing of the blood vessels just as they leave the eye is influenced by the intracranial pressure (ICP) in the sheath surrounding the optic nerve just outside of the eye.

prototype device for measuring intracranial pressure (ICP) is set to eliminate the need for brain surgery in head trauma patients, providing fast, non-invasive, critical decision-making data to clinicians to improve patient outcomes. With philanthropic suppor t from the Michael Hirshorn Medical Research Commercialisation Fund, Associate Professor Bang Bui has brought together a multidisciplinary team of experts at the Department of Optometry and Vision Sciences at the University of Melbourne to move the device into the next phase of development. The support has also provided for appointment of an Early Career Researcher to ensure the project remains sustainable over time, an important step in the commercialisation of a new device. The device uses ophthalmodynamometr y, or the measurement of blood pressure dynamics within the blood vessels in the eye, to reveal high pressure around the brain following physical trauma. Such pressure can quickly lead to brain injury but measuring this pressure to determine life-saving action is traditionally only possible through invasive brain surgery, which takes time and presents the risk of infection and complications.

TIME IS OF THE ESSENCE

“When it comes to any trauma, but especially head trauma, we know time is of the essence. Many of the existing tools we rely on for diagnostics are not portable. A clinically viable device to be used for trauma patients must be handheld and portable and able to be used both in upright patients and those patients lying down.” Having shown preliminary feasibility of this method, Bang Bui and his team now aim to develop a handheld device that can be used to calculate the correlation between vascular instability using ophthalmodynamometry and ICP in patients admitted to the Royal Melbourne Hospital due to head trauma.

The automated handheld ophthalmodynamometry unit will comprise a feedback force sensor to compensate for movement – allowing for use in a wide range of postures while processing images of retinal blood vessels.

In addition to faster, non-invasive diagnosis, this new technique will significantly reduce the cost of monitoring ICP by eliminating the need for invasive surgery,” he said. Associate Professor Bui and the team including Associate Professor Lauren Ayton, Dr Sam John, Professor David Grayden and Assocaite Professor Andrew Morokoff are deeply appreciative for the support from the Michael Hirshorn Medical Research Commercialisation Fund. The support was essential to maintaining continuity in their research during the shutdown throughout 2020. Having the seed funding in place at the very beginning of 2020 meant that they were able to collect substantial proof of concept data through the first quarter of 2020. This allowed them to develop image analysis approaches and to interpret the data through the second half of 2020.

FURTHER WORK

“Having this pilot data reassured us of the commercial potential of this project. Our team has already submitted two grants applications to support conducting clinical trials in the hospital setting and to use the portable device in critical care field settings.” “We will continue to work to refine the device on laboratory eye models and healthy eyes, to ensure that the system can be robustly used by clinicians.” “Philanthropic support for biomedical research is a key aspect for advancing discoveries to commercial application and out to market to save lives. “

Author: Associate Professor Bang Bui

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ARE TINY PIECES OF PLASTIC INSIDE OUR BRAINS? Humans are exposed to plastics daily, through the air we breathe, the food we eat, and the water we drink.

e know that we ingest and inhale microplastics which are visible to the naked eye and that they pass through our gut. However, we don’t know whether smaller pieces of plastic – some as tiny as a virus – cross the gut wall and accumulate in our bodies. A partnership between The University of Queensland and Minderoo Foundation, The Minderoo Centre – Plastics and Human Health is dedicated to developing methods that are sensitive enough to detect plastic in our blood, urine or brain. UQ researchers are currently working on the initial phase of the project inside one of the world’s first plastics contamination-controlled laboratories based in UQ’s Queensland Alliance for Environmental Health Sciences, located in Brisbane, Queensland. But there are challenges associated with carrying out this important work – it takes a unique facility and team to do the job. “The plastic particles are much, much harder to detect when you get down to the size of viruses,” says Professor 50

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Sarah Dunlop, Director of Plastics and Human Health at Minderoo Foundation. “It is like looking for something the size of a tennis ball from a hundred kilometres away.”

BUILDING A STAINLESS-STEEL LAB

Due to the all-pervasive nature of plastics, the ambitious initial objective was to design and build a lab with the lowest plastic contamination in the world. “We conducted extensive testing of construction materials including paint, floor coverings and adhesives,” Minderoo Foundation Chair Dr Andrew Forrest says. “As most of these materials contained plastics or plastic chemicals that can leach into the lab, it was built almost entirely out of welded stainless steel.”

A COLLABORATION BEYOND PHILANTHROPY

UQ Vice-Chancellor and President Professor Deborah Terry says the partnership extends beyond philanthropy. “Minderoo has its own expert researchers working on plastics and human health so we can effectively

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supercharge our impact by working together,” Professor Terry says. “Minderoo also brings broader skills and knowledge, including a deep understanding of chemical regulation in Australia, that will help to ensure the research findings are able to effect changes to improve health outcomes.” Professor Dunlop says Minderoo’s role in this ambitious research project was absolutely essential because many other funding agencies have a more conservative approach. “To crack urgent and seemingly intractable problems such as plastic pollution of the human body, we need audacious out-of-the-box thinking and the capital to support it right now.” she says.

UQ PROVIDING WORLD-LEADING EXPERTISE

UQ was selected as the Centre’s base because of its world-leading research team with expertise in measuring very small concentrations of molecules, enabling them to work on detecting and measuring plastics in samples.

Testing will look for plastics such as polyethylene, polypropylene and PVC, along with commonly used plastic additives such as phthalates and bisphenols. The lab team wear brightly coloured, 100 per cent cotton scrubs to minimise fibre shedding and to ensure any loose fibres are easily identified. The Sydney Brain Bank supplies samples to the lab to allow the research team to test for plastic in the brain. “Our next goal is to measure chemicals and plastics within the body accurately so we can determine whether plastic particles are in humans or not, and the level of chemicals in our bodily fluids and tissue,” Professor Thomas says. “The collaboration has given UQ’s researchers an opportunity to conduct the world-leading research required to detect and measure nanoplastics in our bodies.” “Once we release our first findings, expected by the end of the year, we will be looking to join with other leading institutions working on similar missions around the world.”

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HIGH RISK HIGH REWARD Philanthropy is funding the highrisk high-reward research needed to advance our fight against motor neurone disease (MND).

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CHRISTOPHE KEREBEL

pproximately 2,100 Australians are currently HOW DO YOU DEVELOP NEW MOTOR CHRISTOPHE KEREBEL My Twitter : @chriskere living with motor neurone disease (MND), a NEURONES? CHRISTOPHE KEREBEL devastating neurodegenerative disease. MND We are using three different models to carry out this involves the progressive loss of the neurones in the research. For one of these models, Dr Lambert-Smith brain and spinal cord that connect to and control our utilises a technology that enables her to reprogram skin muscles – the motor neurones. cells donated by ALS patients into a stem cell-like state, The loss of these neurones leads to paralysis and, and then direct them to turn into sensory neurones. eventually, death. Diagnosis of amyotrophic lateral sclerosis (ALS), the most common motor neurone disease, is a prolonged process and occurs after the symptoms appear, at which stage motor neurone degeneration is advanced.

“I’m also using sensory neurones isolated from adult mice that I similarly culture in a dish. With these sensory neurones I’m studying the neurone conversion process to figure out the most effective way to turn sensory neurones into motor neurones,” Dr Lambert-Smith says.

The team of researchers in the Proteostasis and Disease Research Centre at the University of Wollongong (UOW) focus on increasing understanding of the molecular processes that cause MND and use that knowledge to develop therapeutic strategies to treat MND. The team’s research so far provides strong evidence that a protein imbalance in the motor neurones, and that proteome dysfunction, is a fundamental part of the process underlying cellular dysfunction and death in MND. However, why this happens is still unclear.

Using sensory neurones from adult mice is important as it provides a steppingstone to pre-clinical testing, which is essential before it can be tested in humans.

There is an unquestionably urgent need for effective treatments, and it is vital that we investigate methods of replacing motor neurones, in addition to saving remaining neurones.

PHILANTHROPIC FUNDING

The third model uses the microscopic worm Caenorhabditis elegans. These tiny critters have a very well-studied nervous system, and every single neurone within them can be clearly visible when fluorescent reporters are used that light up each neurone.

I’m using this model to examine if converted sensory-motor neurones can remodel their connections in the body, and ‘synapse’ (connect) with muscles in the same way as native motor neurones,” Dr Lambert-Smith says.

The support that we have received locally and nationally has been phenomenal. Working with UOW’s philanthropy team, we have been able to raise $463,810 since 2018. These funds have been provided to us from average families, and generous individuals and groups. In 2018, children in the Illawarra held a lemonade stand raising $1,000; a group of cyclists “Ladies back on your bikes” raised $16,500, and UOW alumni have given generously.

Thanks to this funding, Dr Lambert-Smith has been able to demonstrate that the method of converting sensory neurones into motor neurones alters the activity of specific genes, causing the neurones to start expressing several motor neurone-specific genes (genes that are not expressed in sensory neurones). This is very promising, critical preliminary data showing that sensory neurones have the potential to be reprogrammed into motor neurones.

DEVELOPING NEW MOTOR NEURONES

This regenerative strategy has the potential to revolutionise ALS research and shape the lens through which we understand and treat this devastating disease. Converting sensory neurones into motor neurones has not previously been investigated; a missed opportunity to combat motor neurone loss and prevent or reverse subsequent paralysis.

With traditional funding it is almost impossible to get unconventional projects (projects that ‘think outside the box’) approved. We believe that conventional drug therapies will not work for MND. With this in mind, we have come up with a way to develop new motor neurones inside the body. Using philanthropic donations, we have been able to fund new research, which is being undertaken by Dr Isabella Lambert-Smith, a post-doctoral researcher in the Proteostasis and Disease Research Centre at UOW. “My research is aiming to test if we can regenerate motor neurone circuitry in the body and re-establish connections with the muscles, by converting sensory neurones that are not affected by ALS into motor neurones.” She hopes that this will prove to be an effective therapeutic strategy that will enable people living with ALS “to regain muscle function and have an improved quality of life,” Dr LambertSmith explains.

Authors: Dr Isabella Lambert-Smith, PostDoctoral Research Fellow at the Illawarra Health & Medical Research Institute, and member of the Proteostasis and Disease Research Centre at the University of Wollongong. Professorial Fellow Justin Yerbury, Professor of Neurodegenerative Diseases, Illawarra Health & Medical Research Institute, and Director of the Proteostasis and Disease Research Centre at the University of Wollongong. Justin Yerbury was diagnosed with Motor Neurone Disease (MND) in May 2016.

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SMARTER MENTAL HEALTH SERVICE PLANNING CAN LEAD TO BETTER OUTCOMES FOR YOUNG PEOPLE

Anyone who has experience with the mental health system will tell you that it is difficult to access the care you need.

hey will tell you stories about being denied a service for not being ill enough or being sent away for being too ill.

They will tell you about the distress they and their families have felt trying to organise appointments with different

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providers to address their social, emotional, or physical health needs. Equally, health professionals will tell you about the burdens they face trying to provide or find the right type of care for a person. They will tell you about the time pressures, lack of resources or redundant practices which reduce the time they spend providing care. These accounts are indicators of an underperforming mental health system in which pressure continues to mount due to the social and economic impacts of the pandemic. This pressure amplifies the vicious cycle between system inefficiencies (e.g., delays between helpseeking and treatment) and poor outcomes for people.

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To address this problem the BUPA Foundation funded the KEREBEL CHRISTOPHE Twitter : @chriskere BOX 1. FIVE INNOVATIONS TO My THE WAY CHRISTOPHE KEREBEL ‘Best care, First time’ project led by USYD’s Brain and YOUTH MENTAL HEALTH SERVICES USE Mind Centre. The projects aim is to determine how digital DIGITAL TECHNOLOGIES technology can support the coordination of personalised care for young people accessing public and private 1. S tandardised care entry pathway that uses online services. The BUPA Foundation has filled a critical gap by multidimensional assessment and triage – to help enabling this innovative practical health-services research determine which part of the mental health system for an area that has historically been poorly funded and a person is best suited for. Stratified care is used under-researched. to direct those with very low needs to online or low intensity services, and to fast track entry into services This work has brought together young people, their for those with very severe needs. families and health professionals to reimagine how people get access to the care they need. There has been real 2. A personalised model of care to allocate care dialogue between health providers and consumers about based on clinical stage and need – using the how current services tend to be siloed and fail to meet the multidimensional assessment results to recommend needs of young people, who are often left to navigate the care plans complexities of the health system by themselves. 3. Automated progress reviews and real-time monitoring The valuable knowledge gained from this participatory – tracking of an individual’s progress and needs to research was used to guide smarter health systems dynamically update their current care plan planning and the implementation of a digital technology 4. Highly skilled clinical facilitators are utilised early in into youth mental health services. the intake process and for ongoing care planning – SMARTER HEALTH SERVICE PLANNING Skilled clinical facilitators are proposed as a new role within the service to review progress and facilitate any Smarter health service planning has involved leveraging changes to care plans. dynamic simulation modelling to guide decisions about service pathways, resourcing, and the optimal use of digital 5. Reduced duplication and increased interoperability technologies before they are implemented in the real-world. of information systems – to improve referral time and This work demonstrates the benefits of creating a smart health service infrastructure with embedded dynamic simulation modelling to improve operational efficiency and clinical outcomes through participatory and data driven health service planning.

This research approach has facilitated major ongoing collaboration between industry and multiple public and private health care providers to continually improve the digital technologies utility and integration with existing services. This has led to five innovations that transform the way services operate (box 1). Preliminary six-month evaluation data has already shown the benefits of this type of practical health-services research with notable improvements in various clinical practices. Clinicians report feeling more confidence in the assessment and proactive response to suicidality, better care coordination between providers, improved shared decision-making practices and increased use of other online apps and e-tools for a young person’s care.

information flows about care plans (i.e. continuity of care)

This work shows the power of a whole of system approach to research funding and focus. This has challenged traditional funding and health system structures to determine how we can leverage the accessibility, scalability, and standardisation of technologies in health care. Digital health is not going to be a silver bullet, but it has the potential to advance youth mental health services in ways that just cannot be achieved via conventional approaches. With smarter health services planning we can create a youth mental health system that meets the needs of young people.

Authors: Dr Frank Iorfino is an early career researcher for the Brain and Mind Centre at the University of Sydney. His research involves multidisciplinary partnerships with health service organisations, data scientists and industry to drive real-world impact that improves the quality of youth mental health care and leads to better outcomes for young people. Professor Ian Hickie is Co-Director, Health and Policy at The University of Sydney’s Brain and Mind Centre. He is an internationally renowned researcher in clinical psychiatry, with particular reference to medical aspects of common mood disorders, depression and bipolar disorder.

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PIONEERING PARTNERSHIP AT THE HEART OF INNOVATIVE RESEARCH

As one of the leading causes of death in healthy individuals under the age of 40, hypertrophic cardiomyopathy can lead to sudden death in otherwise fit, healthy children and to cardiac arrest in healthy adults. 56

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ypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. Medications and treatments focus on reducing the over-contraction of the heart caused by its thickened muscle, using beta-blockers, calcium channel blockers, heart rhythm drugs and blood thinners. In some cases, implanted cardiac defibrillators are used to treat the irregular heartbeat sometimes caused by HCM. Professor Livia Hool, Wesfarmers UWA-VCCRI Chair in Cardiovascular Research at The University of Western

CHRISTOPHE KEREBEL

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Australia, says almost one in 500 people are affected by the disease, but this number is growing and could be as common as one in every 200 people. “Right now, we have no way of preventing or reversing damage to the heart. All we can do is alleviate the symptoms,” explains Professor Hool, who is also head of the Western Australia Victor Chang Cardiac Research Institute’s (VCCRI) cardiovascular electrophysiology laboratory. Established in late 2021, the new laborator y aims to become the centre of world-class heart research in Western Australia. The laboratory is the result of philanthropic support and collaboration and will build upon Professor Hool’s innovative research into familial hypertrophic cardiomyopathy and her research into understanding the mechanisms of sudden death and heart failure.

FUNDING A DEDICATED TEAM

“Heart disease has been underfunded for far too long in WA, but thanks to this pioneering partnership between The University of Western Australia, the Victor Chang Cardiac Research Institute and Wesfarmers, that is now set to change,” explained Professor Hool. “This will not only help us to save lives in Western Australia but deliver scientific breakthroughs that will benefit people the world over.” Generous funding from Wesfarmers last year has allowed Professor Hool, UWA and VCCRI to create a pre-clinical chair of cardiovascular disease discovery, based at UWA, which will further expand the capacity of her team and laboratory to undertake the necessary research into the prevention and reversal of hypertrophic cardiomyopathy. Wesfarmers, which also funds the Centre for Vaccines and Infectious Disease at the Telethon Kids Institute, is committed to supporting research into critical health issues as part of its community partnerships program.

We are very pleased that our commitment to endow a new Chair in cardiovascular research and establish the new hub of cardiovascular excellence will help improve people’s lives and their health outcomes,” said Wesfarmers Managing Director Rob Scott. This generous funding has built on the previous philanthropy of global organisations such as CSL, which supported VCCRI as part of its Research Acceleration Initiative in order to further the study of hypertrophic cardiomyopathy. Woodside Energy has also invested in this important collaboration between UWA and VCCRI to

bring about Western Australia’s first dedicated centre of cardiovascular research. “The contribution from Woodside is extremely welcome, and will enable us to spend more time in the laboratory making discoveries,” Professor Hool explained. “It will be essential to enhancing the ground-breaking projects already under way, as well as helping us deliver future scientific breakthroughs.”

GROUNDBREAKING RESEARCH

“About one in 500 people carry a genetic mutation for hypertrophic cardiomyopathy – or an enlarged heart. Excitingly, my team has discovered a way to prevent the heart from becoming larger. If it is effective in human trials, it will be the first-ever treatment to prevent the development of the disease,” explained Professor Hool. The continuing research into this possible treatment builds on years of work from Professor Hool, beginning in 2010, in which her team discovered a novel mechanism of the calcium channel of the heart. Usually regulating mitochondrial function and energetics of the heart, in those with HCM the communication between the calcium channel and mitochondria is altered, leading to a hypermetabolic heart. New research is currently investigating the possibility of using this relationship and channel to reverse the resulting cardiomyopathy. The most recent breakthrough has built on Professor Hool’s research over the last decade, with medication targeting the calcium channel proving ef fective in preventing the disease from occurring, and excitingly, partially reducing the overall size of the heart of those who had or were affected by the disease. Continuing to work in partnership with VCCRI, UWA and the many philanthropic supporters of the research, Professor Hool is expanding the areas of research interest to include developing 3D-structure guided drug treatments, optimising gene therapy for dilated cardiomyopathy, and exploring therapy which can reverse hypertrophy associated with HCM.

Author: Professor Livia Hool is the Wesfarmers UWA-VCCRI Chair in Cardiovascular Research at The University of Western Australia. Professor Hool decided to pursue a career in heart research after observing how quickly heart attack patients deteriorated due to heart failure in the Coronary Care Unit. Professor Hool joined the Victor Chang Cardiac Research Institute in 2013 and is the current Institute faculty head.

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A multi-generational family of Raine Study participants

THE GIFT OF TIME THE IMPACT OF PHILANTHROPIC FUNDING ON LONGITUDINAL HEALTH RESEARCH

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Results that stand the test of time require long-term vision, partnership, and support

three decades, providing support for the Raine Study’s operating costs as well as scholarships and prize awards for young researchers using the Raine Study data.

In that same year, 1989, the Women and Infants Research Foundation (WIRF) commenced its support for the Raine Study. Located at Perth’s King Edward Memorial Hospital, WIRF provided a home for the Raine Study during the pregnancy and newborn research phase.

Established in Perth in 1989, it was the first medical study to recruit and track the health of its participants in utero. It has continued to survey their health at regular intervals since birth, to determine the role that factors from the womb onwards have on health and wellbeing throughout life’s course.

While the Raine Study’s core participant group has aged and the Study has moved location, first to Telethon Kids Institute and then to its current host, the University of Western Australia, WIRF remains one of the Raine Study’s core funders and research partners.

CHRISTOPHE KEREBEL My Twitter Since providing that very first grant in 1989, the: @chriskere Raine CHRISTOPHE KEREBEL the Raine Study over Foundation has continued to support

he Raine Study is a long-running Australian cohort study, and one of the most successful ongoing studies of pregnancy, child, adolescent and adult health anywhere in the world.

Because of the Raine Study, the world knows that ultrasounds in pregnancy are safe, that there is no difference in the long-term health outcomes of children conceived naturally and those conceived via assisted reproductive technology, and that many common health ailments have their roots not just in childhood but in the factors that shaped the foetus.

What began as a short-term study of pregnant women has become a longterm multi-generational study, recognised internationally for its unique and ongoing contributions to understanding human health. Until 2017, it was funded year to year, grant to grant with no allocation for core infrastructure. To this day it receives no government funding and would not survive without the far-sighted support of three philanthropic organisations.

IN THE BEGINNING

The Raine Study’s very first and longest-standing funder, the Raine Medical Research Foundation, was created from one woman’s drive to prevent others from suffering the loss of a loved one to preventable disease. Mary Raine was a visionary and saw the Foundation she established on her husband’s death in 1957 as a unique opportunity to support health and medical research that improves outcomes for all, in perpetuity. Three decades later, John Newnham, a young obstetrician with a passion for research, had a chance meeting with the Raine Foundation’s accountant where he learned that it was looking to award a sizeable grant to fund one largescale ambitious project. John and a group of Perth-based investigators Lou Landau, Con Michael and Fiona Stanley wanted to develop a long-term cohort to study the role that early life events had on later life. Their application was successful, and the Raine Study commenced with a study of the effect of frequent ultrasounds during pregnancy.

A recent world-first study draws on a decade of Raine Study data to examine how early life exposure to chemicals influences male and female reproductive development.

THE FUTURE

Throughout its existence, the Raine Study has survived fluctuating levels of funding, and increasingly scarce access to national grant schemes. Now in 2022, the Raine Study is working to secure its future with the award of substantial new funding from the Stan Perron Charitable Foundation. The Raine Study will use the Perron Foundation grant to safeguard its future through investments in staff capacity to plan strategically for the Raine Study’s future direction, and to enable investment in the specialist staff required to protect and improve access to the Raine Study’s 30-plus years of biosamples and health data. As it enters its fourth decade, the mission of the Raine Study remains to improve lifelong health and quality of life through ground-breaking, impactful research that examines influences and outcomes from before birth and throughout life’s course. It is only through the Raine Study’s philanthropic supporters that the Study has been able to survive and continue to collect important longitudinal data. Philanthropic suppor t has been instrumental in enabling researchers to take a long-term approach to understanding the causes of health and wellbeing from before birth into adulthood, making many world-first discoveries along the way. If longitudinal research is a marathon endeavour, it is philanthropic vision, partnership and support which pave the road on which this race is being run.

Author: Kate Rowlands, Communications Manager, The Raine Study

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THE LAST WORD

PHILANTHROPY’S ROLE IN RESEARCH – SHAPING A BETTER FUTURE The Leukaemia Foundation has proudly supported blood cancer research for over two decades, investing into medical research that drives rapid advancements in diagnosis and treatments for blood cancer, encourages the careers of promising scientists and clinicians, and gives Australians access to the latest therapies through clinical trials. The Leukaemia Foundation’s CEO Chris Tanti reflects on what philanthropy can bring to research. 60

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he Leukaemia Foundation has been funding research for over 20 years, investing $54 million into research, including supporting over 500 researchers and clinicians to undertake over 300 PhDs, clinical trials, and multi-year strategic research projects, across 60 research institutes here in Australia and overseas. Through our National Research Program we fund all phases of research from early and pre-clinical through to translational and clinical trials. We know that good research can take time and investments in research today are needed to ensure that we develop the new treatments of tomorrow. It’s not a short-term win, but rather a longer-term aspiration. It’s ultimately about knowing innovation in science takes time to go from the lab to the bedside. That’s what drew me to the Leukaemia Foundation. It’s an ambitious, tenacious organisation with an inspiring vision to dramatically improve the lives of Australians living with blood cancer. As an organisation we’re complex – we invest in research and support clinical trials; and for people living with blood cancer, we provide information and education as well as practical

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My Twitter : @chriskere CHRISTOPHE KEREBEL

psychosocial support and accommodation. We also lead the public discussion about the range of issues faced by the more than 110,000 Australians who are currently living with a blood cancer. I love this complexity. I also love the audacious targets that we set for ourselves, which I think is such an important thing for community driven organisations to do. I’m proud that the Leukaemia Foundation continues to stretch itself every day with its vision and support of Australians impacted by blood cancer. I am grateful to co-chair, with Professor John Seymour, the Blood Cancer Taskforce. The Taskforce developed the National Strategic Action Plan for Blood Cancer – a once-in-a-generation roadmap to reduce the impact of blood cancer. With support from the Australian Government, we have been able to start implementing some of the actions outlined in the National Action Plan, but there is still so much more to do. That is why the Leukaemia Foundation recently entered into a public-private partnership with the Department of Health to implement more of the National Action Plan.

had been personally impacted by blood cancer. Like philanthropy, its beginnings were about shaping a better future. It’s grown into a nationally relevant passionate, ambitious and brave organisation with a workforce, senior leadership team and Board who are willing to take risks, make brave decisions and play the long game to ensure we can make a difference to the people we serve, today and tomorrow and beyond 2035. This will continue thanks to investment, commitment and vision from inspiring philanthropists, many who are directly impacted by blood cancer.

We have set a vision of zero lives lost to blood cancer by 2035. That is just a short 13 short years away. To achieve this ambitious goal more effort needs to be focused on developing the next generation therapies and treatments. To guide this effort, the Leukaemia Foundation is funding a key National Action Plan recommendation – the Research Roadmap. This Roadmap will identify key Australian areas of expertise and will guide future investment to address unmet medical need across all blood cancers. When it comes to research, what role can philanthropy play? Philanthropy provides an opportunity to shape a better future. The Leukaemia Foundation has a proud track record of finding and supporting leading research and clinical trials. By bringing together the resources of philanthropists and the expertise within the Foundation, we can together focus scarce resources on solving the biggest scientific and medical challenges of blood cancer. Our portfolio of projects ranges from foundational science to understand the underlying causes of blood cancer though to fast tracking access to new treatments through clinical trials. We have created opportunities for philanthropists to have immediate and/or longer-term impact. Investment in CAR T-cell therapy is a great example of this. The first investment in this treatment was made 20 years ago, with philanthropic investment an important part of the funding mix. What began as a small initial investment resulted in a significant transformation in terms of treatment and outcomes for blood cancer patients. That’s the power of philanthropy. The Leukaemia Foundation was started by people who

Author: Chris Tanti, CEO, Leukaemia Foundation. With an extensive background in the health sector, Chris joined the Leukaemia Foundation in February 2021 and brings a wealth of knowledge to the organisation. Chris began his career in social work and in clinical settings as a psychotherapist working across acute and public mental health before transitioning into management some 25 years ago. He has since held national and international senior executive positions including CEO of Australia’s National Disability Services and establishing and leading the mental health organisation, Headspace.

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