2020 MCRI Annual Report

Above and beyond Annual Report 2020

Children need us like their future depends on it

The Murdoch Children’s Research Institute acknowledges the Traditional Custodians of the land upon which we are located. We pay our respect to their Elders past, present and emerging.

2020 Stories

1

10

Stem cell research

26

Taking on a global pandemic

12

Data science

28

Genomics highlights

14

Prizes and awards

30

Two families making a difference

16

Global mission

32

Global health

18

Benchmarking our efforts: research metrics

34

Flying the flag for Aboriginal health

20

Eyes on the COVID generation

35

Better outcomes for mental health

22

From bench to bedside … and back again

36

3 See this year’s Annual Report stories come to life in video using the Viewa app on your mobile phone.

HERE’S HOW 1 Download Viewa App using barcode below 2 Scan picture with Viewa 3 Explore in Augmented Reality

4

People and Culture

Leading by example

40

Making our mark: media engagements

42

Delivering outstanding research

Murdoch Children’s Research Institute

23

Supporting outstanding researchers 44

Scan this image to hear from our Global Ambassador and Co-Chair, Sarah Murdoch.

Research Excellence

The kids are OK

Towards a National Centre for Allergies and Anaphylaxis

2

2

Research Impact

On the cover Noah Carpino has Niemann-Pick Type C disease, a rare genetic disease.

PHOTO COURTESY JAY TOWN / NEWSPIX

45

Sustainability and Engagement

Partnering with purpose

48

Thank you

50

Mapping a solution to MD

52

A remarkable contribution

53

Our donors

54

MCRI Board of Directors

56

Year at a glance: MCRI financials

58

Our manifesto

62

Annual AnnualReport Report2020 2020

3

From the Chairman On behalf of the Board, I am proud to share with you the 2020 Annual Report, which recognises and acknowledges the world-leading research of Murdoch Children’s Research Institute and the remarkable role we are playing during this global pandemic.

Our Annual Report is a chance to reflect on our past year’s achievements and acknowledge the enormous contribution made by so many inspiring individuals who make Murdoch Children’s the pre-eminent child health research institute in the country – and one of the top three globally.

COVID-19 has demonstrated what we have always known to be true; that science really does matter. It has the power to transform and save lives.

Over this past year, we have witnessed an unprecedented and co-ordinated scientific effort on a global scale. We also saw a recognition and appreciation of our frontline healthcare workers.

In this report, we share some of our work and outline the impact it’s having, particularly around COVID-19. You will read about our world-first BCG vaccine trial led by Nigel Curtis, our COVID-19 research programs and the work we undertook to get students back to school.

In my role, it has been a privilege to be part of new and ongoing philanthropic relationships with the Macquarie Foundation, Paul Ramsay Foundation, Stafford Fox Medical Research Foundation, Minderoo Foundation and globally with the Milken Institute and the Bill and Melinda Gates Foundation. I am thrilled to be able to continue my involvement and connection with the Institute as a Global Ambassador, working with Sarah Murdoch on strategic relationships here and around the world.

This year marks Sarah’s 20th anniversary with the Institute and we are so grateful for her insight and ongoing commitment. She has always understood the importance and significance of philanthropic leadership.

Director’s message the long and wonderful journey I would end up sharing with this extraordinary organisation. More than two decades later, I write to you as outgoing Chairman – deeply humbled and grateful for this incredible opportunity.

“I have witnessed the power of adaptability, agility, creativity and genius in a way that is unlike anything I have seen before.”

SUZI CARP Last year, Sarah and Lachlan Murdoch generously endowed a $5 million Fellowship to the Institute. This gift is a stunning example of the long-term vision, impact and unconditional support of Sarah and Lachlan and their family.

As Chairman, I have never felt more pride in this remarkable Institute than in this past year.

All of us at the Institute – whether it be our Directors, Committee and Council members, Council of Ambassadors, supporters or friends – are committed to creating a world where every child can grow to reach their full potential. Children really are at the heart of everything we do. It is the invisible thread that connects us all.

Despite the physical distance that separated us in 2020 – and our inability to hold board and committee meetings, directors forums and events – we remained close and connected. To my fellow directors, thank you to each of you for your commitment and dedication to MCRI. I would also like to acknowledge our remarkable director, Professor Kathryn North. Kathryn used this pandemic as an opportunity for real impact and transformation in child health. She navigated the impossibly challenging times of COVID-19 with skill, empathy and vision.

When I joined the Development Board nearly 20 years ago, I never would have imagined

4

Murdoch Children’s Research Institute

I have witnessed first-hand the transformation of MCRI from a great child health research institute in Australia to becoming one of the best in the world. I leave at a time when the deep relationships on Campus have never been stronger.

I would like to thank and recognise our Campus Council members and partners the University of Melbourne Department of Paediatrics, The Royal Children’s Hospital and The Royal Children’s Hospital Foundation.

I have always said that MCRI and the Children’s Campus fills me with hope.

Watching how we worked together and the way our Campus responded and supported one another is another shining example of how hope permeates our corridors and now our virtual spaces.

I am delighted to welcome Pat Houlihan as the next Chairman of this Institute. He has been an invaluable director over the past seven years and reminds us always to focus on what really matters. I am also thrilled that Sarah Murdoch is joining him as Co-Chair and Global Ambassador. It is a role reflecting Sarah’s deep commitment, passion and leadership in global development, strategy and philanthropy.

I would like to finish by honouring and paying tribute to the MCRI team. Your brilliant minds, your enormous contribution to society and children’s health, and your continued curiosity and drive for discovery and impact have been inspiring beyond measure.

Thank you for trusting me, supporting me, allowing me to walk alongside you and for filling my life everyday with hope.

Sharing my life with all of you has been one of my greatest privileges.

Suzi Carp 2020 Chairman

2020 will forever be defined

by COVID-19. The agility and resourcefulness with which everyone at the Institute responded went above and beyond. It is a source of great pride for me. And so, I am excited and honoured to present this year’s Annual Report as a record of all that we achieved.

Our shared passion to improve the health and wellbeing of every child saw us make a truly gigantic contribution to the global effort to address COVID-19.

During the past year, I witnessed an unwavering resolve and determination to contribute to the COVID-19 response.

This was coupled with an unshakable commitment to ensure that our vital research continued across genomic medicine, stem cell technologies and improving the lives of children and families in Victoria, Australia and around the globe. Our collective efforts ensured our continued standing as one of the top three children’s medical research institutes in the world.

Early in the pandemic, we were bracing for huge income falls. There was uncertainty around whether government grants would continue and a precarious funding environment. Once again, the institute resolutely saw off any potential crisis and attracted $16 million in dedicated COVID-19 research funding. This was on top of significant levels of federal funding secured for projects unrelated to COVID-19.

Immediately, the Institute’s researchers initiated numerous COVID-19 research projects, spanning every one of our research strengths.

The first COVID-19 project was the BRACE trial investigating whether the BCG tuberculosis vaccine could enhance healthcare workers’ immune systems to protect them against severe COVID-19. That trial has recruited more than 6000 healthcare workers at nearly 40 sites in five countries.

After studying hospital admissions of children with COVID-19, we showed that, thankfully, children were spared the worst effects.

GenV will partner with all Victorian birthing hospitals over two years. Given the timing, GenV is also in a unique position to investigate, understand and mitigate the impact of the COVID-19 pandemic on children and families.

Despite the limitations imposed by COVID-19, we made great progress across all of our major strategic research areas.

Our people have shown great ingenuity and rapidly adapted to maintain our momentum.

Following that, we examined household transmission dynamics and families’ immune responses to infection. This work has shown how children’s immune systems protect them from the virus.

Other COVID-19 research projects assessed the safety of keeping face-to-face school learning onsite in the midst of pandemic lockdowns, families’ preferences for pregnancy-care and cystic fibrosis telehealth consultations. Our teams worked to understand broader effects on children and families during this time, such as inequities in accessing health services, how children with neurodevelopmental disorders fared during lockdown and the risk of violence and substance abuse.

Our Victorian Clinical Genetics Service ensured families and referring clinicians received the same high standard of care as always. Working COVID-safe shifts, they didn’t miss a beat in providing preconception and prenatal genetic testing. VCGS also continued genome sequencing for the sickest babies to ensure as many families as possible received a diagnosis and access to precision medicine.

MCRI has continued its membership of a number of strategic national and international partnerships such as the Centre for Population Genomics with the Garvan Institute, the Australian Strep A Vaccine Alliance with the Telethon Kids Institute and, of course, our leading roles in the Global Alliance for Genomics and Health and the Genomics in Health Implementation Forum.

At the end of 2020, our Generation Victoria (GenV) project recruited its very first family.

Baby Eliana was born on Christmas Day and will become the first of thousands of Victorian children who will, over the course of their lifetimes, help reduce the burden of allergy, asthma, obesity and mental ill health.

We were so mindful of not only the physical danger to our staff and students during 2020, but also the anxiety and stress caused by the constant barrage of bad news, homeschooling and lack of contact with family, friends and colleagues. To counter this, we introduced measures to allow staff time to look after themselves and loved ones. We held a number of staff health and wellness initiatives, which received excellent feedback in our regular staff wellbeing surveys.

2020 saw leadership changes at the Board level. All of us give our sincerest thanks, best wishes and fondest farewell to outgoing Chairman Suzi Carp. Suzi has contributed so much and brought such great strategic vision to the Institute. The five-year strategic review, established in 2019, has set us up for ongoing success.

Patrick Houlihan takes over the reins as Chairman from Suzi and is joined by Co-Chair Sarah Murdoch, who has represented MCRI on the global stage for 20 years.

2020’s incredible achievements were only made possible by our staff, students, supporters, Board, Committees and Executive working together. I remain so humbled and grateful for the continuing drive and passion shown by everyone, often under the hardest of circumstances.

Despite the myriad difficulties experienced by all of our supporters over the previous year, they have continued to place their trust and hope in our work.

We are proud and honoured to repay that trust and hope by continuing our mission to make profound improvements to children’s lives everywhere. Thank you.

Professor Kathryn North AC

Annual Report 2020

5

A family’s commitment … and tradition

Welcome to Patrick Houlihan

The year marked Sarah Murdoch’s 20th as a global ambassador for the Murdoch Children’s Research Institute.

Patrick Houlihan’s love of science and the benefits it enables in society is largely thanks to a school teacher, who instilled in him a sense of curiosity.

The Murdoch family’s longstanding involvement with MCRI began when Dame Elisabeth Murdoch established the Institute with Professor David Danks in 1986.

Patrick turned that fascination into a career, starting out as a research chemist at DuluxGroup in 1989 after finishing his Bachelor of Science (Hons) at the University of Melbourne.

He became Chief Executive Officer in 2007 and DuluxGroup Chairman in 2019, after progressing through a succession of leadership roles across research and development, sales, marketing and general management. While working at DuluxGroup he also completed a Master of Business Administration.

Dame Elisabeth was determined that treatments and cures would be found for the children she visited on the wards of The Royal Children’s Hospital (RCH), which itself celebrated its 150th anniversary in 2020. The RCH is now recognised globally as one of the world’s great paediatric hospitals.

In 2018, Sarah began supporting the Marketing Council in an effort to raise the Institute’s public profile. The following year, she formed the US Development Board to extend MCRI’s philanthropic reach internationally. A key achievement has been to foster a warm relationship with renowned US philanthropist Mike Milken.

Sarah, Kathryn North and Suzi Carp host Mike Milken at MCRI. Mike remains a firm friend of the Institute and hosted Sarah, Kathryn and Dr Hamish Graham on his 2020 COVID-19 podcast.

He is pleased and proud to take the reins as MCRI Chairman and thanked his predecessor Suzi Carp for her tireless devotion to the Institute over 20 years.

At the 2020 Annual Showcase, Sarah and Lachlan Murdoch announced a $5 million Fellowship, which will continue to bring the brightest research minds to MCRI in transformative research fields, such as stem cell technologies and genomics. As a perpetual endowment, the Fellowship will make a meaningful difference to future generations of researchers.

Having become involved with MCRI in 2014, Patrick has been unfailingly impressed by its relentless focus on its mission: ensuring children are at the heart of everything we do and the way that effort is delivered through the breadth and depth of research, and the commitment and dedication of the Institute’s researchers.

In 2021, Sarah becomes Co-Chair of the Board of Directors and will continue to play a key role in connecting US and other international philanthropists with MCRI.

Sarah was the patron of MCRI’s annual “Stepa-thon”, which ran from 2013 to 2017 and encouraged kids to get outside and get active, all while raising vital research funds.

“It truly inspires me to see how deeply Sarah cares about MCRI’s work, our staff and students but, most of all, the children, adolescents and families we work with,” says MCRI Director Professor Kathryn North.

“Sarah has such a broad and deep knowledge of the Institute, and a keen sense of where the ‘next big breakthrough’ will come from. I’m so looking forward to working even more closely with Sarah in the coming years.”

In 2020, Patrick was also appointed to the Board of Industry, Innovation and Science Australia, which advises the Federal Government on innovation, research and science and is responsible for implementing the Prime Minister’s Modern Manufacturing Strategy.

Ambassador for the Institute’s hallmark fundraising event ‘Foxtel Laps’, Sarah was on hand to encourage company-sponsored staff, corporate chiefs and celebrities alike to run or walk on a treadmill as far as they could in ten hours.

“It’s a privilege to continue MCRI’s impact-driven growth agenda, ensuring that its world-leading medical research knowledge advancement and discoveries are widely disseminated in academe and industry,’’ he says. “Above all, we are driven to ensure new knowledge is rapidly applied to generate better health outcomes for children and the wider public good.”

A guiding principle for the senior leadership, he adds, will be to continue to find new ways to accelerate MCRI’s research, to increase translational outcomes that benefit patients, and to build even stronger relationships with leading stakeholders in shaping and administering the health system.

Just like Dame Elisabeth before her, Sarah takes a keen interest in why children end up in hospital and is determined to help researchers develop treatments and eventually prevent disease.

Left: Sarah announces via video link the Sarah and Lachlan Murdoch Fellowship and co- hosts a panel discussion at the 2020 Annual Showcase.

6

Murdoch Children’s Research Institute Murdoch MurdochChildren’s Children’sResearch ResearchInstitute Institute

Annual AnnualReport Report2020 2020 Annual Report 2019

7 7

Innate immune protection Alessandro Bartesaghi and Francesca Orsini with their two children, Camilla and Beatrice, all contracted COVID-19 during Melbourne’s second wave. They were part of an MCRI study into the effects of the coronavirus on children.

See story page 10

1

Research Impact The kids are OK

10

Taking on a global pandemic

12

Genomic highlights

14

Two families making a difference 16 Global health

18

Flying the flag for Aboriginal health

20

Better outcomes for mental health

22

Towards a National Centre for Allergies and Anaphylaxis

23

PHOTO COURTESY PATRICK STONE / ABC NEWS

8

MurdochChildren’s Children’sResearch ResearchInstitute Institute Murdoch

Annual Report 2020

Annual Report 2020 RESEARCH IMPACT

9

The kids are OK: the wonder of immunity Researchers are looking at why children’s immune response to COVID-19 is more robust than that of adults.

When Leila Sawenko and her husband Tony Maguire were reunited with their children after a week away in Sydney in March 2020, there were lots of cuddles and kisses shared.

Unknowingly, the couple also shared something else with their three young children – COVID-19.

A week after arriving home, Leila and Tony began to feel unwell. Leila received an email saying some NSW relatives had tested positive to the virus, prompting the couple to get tested. The results came back positive.

The couple’s three children – Bodhi, 9, Lenny, 7, and Marley, 6 – were tested but they were negative. It was a surprise, given they lived in such close quarters with their parents.

Technically, this was a case of chronic exposure. So, what was going on?

Suddenly, the family became a perfect case study for researchers trying to understand how children’s immune response to COVID-19 differed from that of adults.

The family of five shared blood, saliva, stool and urine samples, as well as nose and throat swabs, with a team of 30 researchers led by the Murdoch Children’s Research Institute. Samples were taken every two to three days over a month.

While Leila and Tony experienced heavy flu-like symptoms, their children’s experience was more akin to a cold.

Bodhi reported a cough, sore throat and abdominal discomfort, while Lenny had a mild cough and runny nose. Marley, the couple’s youngest child, who had been sharing a bed with her parents while they were infectious, displayed no symptoms at all.

“It was jaw-droppingly amazing because they’d spent a week and a half with us while we were COVID-positive,” Leila says.

The children’s initial negative test results baffled MCRI paediatrician Dr Shidan Tosif, who tested them a further three times – even using two different laboratories.

But further studies revealed they had all contracted COVID-19.

Shidan says it was striking that all three children were found to have salivary antibodies to the virus. The one with the strongest immune response was Marley, who was asymptomatic.

“The fact these children were able to shut down the virus without even showing a positive test result suggests they have some level of their immune system which is able to respond and deal effectively with the virus,” he says.

The perfect case study Leila Sawenko and husband Tony Maguire tested positive to COVID-19.

Their children Bodhi, Jenny and Marley tested negative. MCRI researchers were thrilled when the family agreed to participate in its research.

In one of the first studies of its kind, MCRI researchers established that COVID-19 was unlikely to have severe or lasting effects on younger age groups because children’s immune systems responded differently to those of adults.

At a stage of the pandemic when so much was unknown, it was reassuring news for parents, carers and researchers alike.

The findings cemented MCRI’s reputation as one of the top children’s immune research institutes globally and armed researchers with clues as to why children can shut down the virus so effectively.

This led to the Institute launching a larger study, which has grown to become one of the world’s most in-depth examinations of children’s immune system responses to COVID-19. Almost 50 children and 70 adults from 28 Melbourne households infected with or exposed to COVID-19 are participating.

Among them are Francesca Orsini and Alessandro Bartesaghi, who contracted COVID-19 in mid2020. For more than two weeks, the couple was almost bedridden with extreme fatigue, fevers, headaches and muscle pain. They also lost their sense of taste and smell. Their daughters Beatrice, 6, and Camilla, 2, had little more than a runny nose.

“The kids, they were OK,” Alessandro says. “They were jumping and running everywhere like always.”

“The fact these children were able to shut down the virus without even showing a positive test result suggests they have some level of their immune system which is able to respond and deal effectively with the virus.”

DR SHIDAN TOSIF

As part of the Institute’s larger study, immune responses in adults and children were monitored during the acute phase of infection and up to two months afterwards.

According to Dr Melanie Neeland, the results indicated children were less likely to become infected with the virus and up to a third were asymptomatic.

“It’s strikingly different to the higher prevalence and severity observed in children for most other respiratory viruses,” she says.

The researchers found that children were likely being protected from severe COVID-19 because their innate immune cells rapidly migrate to infection sites, stopping the virus in its tracks before it has a chance to take hold.

“I think the most interesting part was that this robust, innate immune response in children was not observed in adults with COVID-19,” Melanie says. “It appears to be quite unique because in most other respiratory viruses, we know that children appear to be the most affected.”

Scan page 11 to hear more about how Leila Sawenko, Tony Maguire and their children Bodhi, Lenny and Marley helped MCRI researchers.

PHOTO COURTESY SIMON SCHLUTER / THE AGE

10

MurdochChildren’s Children’sResearch ResearchInstitute Institute Murdoch

Annual Report 2020

Annual Report 2020 RESEARCH IMPACT

11

Taking on a global pandemic “Pivot” may have been the most overused word of 2020. But it accurately captures how MCRI adapted its strengths in stem cell technology and existing large study cohorts, leveraged its government, international research and philanthropic networks and reassured families at a time of high anxiety.

Our researchers, working alongside children, families, practitioners and policymakers, played a world-leading role in making profound contributions to COVID-19 knowledge in Melbourne, Victoria, Australia and on the global stage.

Back to school ... and play A report by a team of epidemiologists and public health experts led by Professor Fiona Russell and Professor Sharon Goldfeld revealed how coronavirus cases in schools and childcare were mainly driven by community transmission.

The Victorian Government used this advice to open primary schools on October 12, ahead of the eventual easing of Stage 4 restrictions.

The report was also presented to the World Health Organisation, UNESCO, the US Centres for Disease Control and Prevention, and the Australian Health Protection Principal Committee to help inform national and international responses.

The report provided detailed plans for preventing further outbreaks of COVID-19 following the staged easing of community restrictions. These plans drew on international experiences, designed to reduce day-to-day disruptions while ensuring that teachers, students and the wider community were kept safe.

Professor Sharon Goldfeld also helped clarify the official rules around outdoor play for children, which were a cause of significant community concern and confusion.

“Children need both physically active play and imaginative play in the outdoor environment,” she explains. “There is strong evidence that children need significant time for play for their mental health and wellbeing.”

BRACEing for impact

Scan page 13 to see Nigel talking about the BRACE trial

Modelling COVID-19 in a dish MCRI is one of a handful of global institutes with the necessary equipment and staff to be able to use stem cells to study disease mechanisms, conduct high-throughput disease modelling and facilitate drug discovery.

The Stem Cell team was awarded two major grants in 2020: one from the Victorian Government’s COVID-19 Research Fund and the other from the Medical Research Future Fund’s Coronavirus Research Response.

The project uses human-derived stem cells to better understand the virus’s effects on different organs, including the lungs, heart, kidneys, brain, immune system and blood vessels. The ultimate aim is to allow the screening of existing drugs to find targeted treatments.

Thanks to years of philanthropic investment from the Stafford Fox Medical Research Foundation, the $10 million Stem Cell Based Disease Modelling Facility opened in March.

The Foundation has a long-standing interest in supporting clinically meaningful projects that have real impact.

An additional $600,000 donation from the Foundation was a key contributor to the Facility being awarded both state and federal COVID-19 research funding.

The multi-agency study includes the Doherty Institute, Monash University and WEHI. It showcases the medical research powerhouse that is Melbourne’s biomedical precinct.

Professor Melissa Little says the research team’s capacity to combine different cell types allows it to investigate how the virus spreads in organs and how infected cells respond.

“There is growing evidence that SARS-CoV-2 damages organs beyond lungs, including the heart, blood vessels, immune cells, kidney and even brain tissue,” she says. “But a limited understanding of its effects on these tissues limits our capacity to treat patients in the most appropriate way and predict long-term complications, like ‘long COVID’.

“This collaborative program will increase our understanding of disease pathology, identify underlying risk factors, change clinical care to protect the patient from severe complications, facilitate the development of targeted treatment options and better prepare us for the next pandemic.”

Victoria’s COVID-19 timeline 5th

2nd

5th

2nd

30th

Crisis Management Team meets to plan Institute’s response to COVID-19

BRACE trial receives seed funding from Sarah and Lachlan Murdoch and The Royal Children’s Hospital Foundation

BRACE trial expands internationally with $10m from the Bill & Melinda Gates Foundation

Study: COVID-19 effects in human stem cell models of the heart receives MRFF funding

COVID-19 Stem Cell Study funded by Victorian Government and Stafford Fox Foundation

11th First strategy meeting of MCRI’s COVID-19 researchers

17th Non-essential MCRI and VCGS staff advised to work from home

WHO declares pandemic

Work from home begins

NUMBER OF DAILY INFECTIONS

march 12

MurdochChildren’s Children’sResearch ResearchInstitute Institute Murdoch

‘Parenting in the age of COVID-19’ podcast launched Children’s immune response cohort study receives funding from Rio Tinto

Stage 3 restrictions

april

‘Road to a Vaccine’ podcast launched by Melbourne Vaccine Education Centre

Stage 4 restrictions Mandatory masks

14th

19th

27th BRACE trial to study BCG vaccine announced

12th MCRI study: children’s mental and physical health risk from pandemic-induced racism

18th

13th BRACE trial recruiting begins in the Netherlands

may

Extra hour of outdoor playtime allowed for Victorian families during Stage 4 lockdown, informed

by MCRI report

24th Mutations that increase risk for severe disease identified

by COVID-19 Human Genetic Effort

When the pandemic struck, and the World Health Organisation called for global assistance, a natural extension was to explore whether BCG could be used to reduce the incidence and severity of COVID-19 in frontline healthcare workers.

Initially funded by Sarah and Lachlan Murdoch, The Royal Children’s Hospital Foundation, the Minderoo Foundation, the Calvert-Jones Foundation, NAB Foundation, UHG Foundation Pty Ltd, Modara Pines Charitable Foundation and additional individual donors, Nigel’s team enrolled the first Australian healthcare workers in the BRACE trial within just a few weeks of the onset of the health emergency.

“These sorts of trials normally take around eight to 12 months to start, but with the fantastic early philanthropic support, and subsequent funding from the Bill & Melinda Gates Foundation, we were able to start testing whether BCG’s ability to enhance immunity reduces the severity of COVID-19 and other respiratory illnesses,” Nigel says.

The BRACE trial also received early support from WHO Director-General Dr Tedros Adhanom Ghebreyesus.

So far, the study team of more than 350 researchers and staff in over 30 sites in five countries have collected more than a quarter of a million samples, attracted a global media audience of more than 5 billion people and secured more than $15 million in philanthropic funding.

“The overarching goal is to pinpoint how BCG induces protective effects so that we can mimic it to make something that’s perhaps better than BCG – something you would give to everybody to induce a better immune response early or even later on in life,” Nigel explains.

If BRACE returns positive results, it means the world will have a “brand new”, 100-year-old, stop-gap immune-boosting vaccine ready to pull off the shelf in time for the next global pandemic.

25th

Stage 3 restrictions end

‘COVID-19 in Victorian ECEC and Schools’ report released

1st

22nd First Australian study showing children at low risk from COVID-19 published

by MCRI

4th

Professor Nigel Curtis had been studying the immune-boosting effects of the Bacille Calmette-Guerin (BCG) vaccine in infants for years. Although used for more than 100 years to prevent tuberculosis, research shows BCG vaccine also protects against other infections including those caused by viruses.

Stage 3 restrictions reinstated

16th

12th Recruiting in UK, Spain and Brazil for BRACE trial announced

june

Children’s immune systems and blood vessels protect them from COVID-19, report shows

Stage 4 restrictions end

july

august

september

october

MCRI warns of measles epidemic due to malnutrition and undervaccination

27th GenV project launches at Joan Kirner Women’s and Children’s Hospital

november Annual Report 2020

december Annual Report 2020 RESEARCH IMPACT

13

Genomics highlights

Committed to children and families

Not every parent gets to feel the relief and ecstasy of holding their healthy newborn just minutes after delivery; or sees them grow into happy and active babies.

For one in 12 families, their little miracle will have a genetic illness that provides doctors few clues as to the underlying cause.

Clinical lead Professor Zornitza Stark agrees.

“We have provided ultra-rapid testing simultaneously across many Australian hospitals, transforming patient care and providing a blueprint for a national rapid genomic diagnosis service.’’

Victorian Clinical Genetics Services (VCGS) is Victoria’s leading specialist scientific and clinical genetics service.

Australian Genomics received an additional $5 million in 2020 to expand the program.

The national rollout is being led by MCRI and (VCGS) clinical geneticist Professor Stark.

Thus begins what researchers call the “diagnostic odyssey”. It’s a long, sometimes fruitless, journey in search of the underlying cause of a baby’s illness and an effective treatment. All this, while associated healthcare costs to the family and society mount.

“The additional funding has allowed us to scale up the national program to all the states and territories, including all children’s hospitals in Australia,” says Zornitza. “It will allow us to fully evaluate whole genome sequencing as a first-tier test for ultra-rapid diagnosis.”

VCGS is a wholly owned subsidiary and key research-translation partner of MCRI, which aspires to make genetics and genomics universally accessible for life-long health. Our commitment has never been more clearly demonstrated than during 2020. While dealing with lockdowns and school closures, teams at VCGS raced to work out how to safeguard the provision of crucial genetic testing, which benefits more than 160,000 Australian families annually.

Mackenzie’s Mission ramps up Firmly of the belief that prevention is of paramount importance in addressing genetic disease, Mackenzie’s Mission is a federally funded research program studying how best to offer reproductive carrier screening.

Couples from across Australia are being offered carrier testing for about 1300 genes that can result in an increased chance of having a child with one of around 750 severe childhood onset genetic conditions.

Recently expanded, Mackenzie’s Mission is now open to couples from across Australia, with around 8000 couples to be tested to determine how best to deliver reproductive genetic carrier screening at scale. Ultimately, the aim is to provide free screening to every couple who chooses it.

The research program is named after Mackenzie, the daughter of Rachael and Jonathan Casella (pictured), who was diagnosed with spinal muscular atrophy, a severe inherited neuromuscular condition for which there is no cure. She passed away in 2017, aged seven months.

The Casellas campaigned to raise awareness of genetic carrier screening, calling for all couples to be aware of the availability of carrier screening and for this to be free and available to all prospective parents.

14

MurdochChildren’s Children’sResearch ResearchInstitute Institute Murdoch

The Acute Care Genomics Study has already provided testing for close to 300 families and delivered a dramatic reduction in turnaround times for genomic sequencing results – as little as 47 hours in some cases.

The program was made possible by funding from the Medical Research Future Fund to Australian Genomics. Victorian Clinical Genetics Services (VCGS), as the Victorian partner of the Mission, services and supports participating Victorian couples.

Diagnosis at rapid speed MCRI Director Professor Kathryn North is the Lead Investigator of Australian Genomics, a $25 million NHMRC-funded national network of 80 partner organisations that drives the integration of genomics into mainstream healthcare.

An Australian Genomics flagship study, Acute Care Genomics, provides ultra-rapid genomics testing to critically ill babies and children with rare conditions.

The study published its first results in 2020, demonstrating that genomic test results could be returned in just three days for most paediatric patients in Australian intensive care units – a process that usually takes three to six months. Half of all families received a diagnosis, with three-quarters of the children diagnosed benefiting from a change in clinical care.

“We now have the technology and team capacity that enables us to consistently deliver a rapid diagnosis within the urgent timeframes needed when children are in intensive care,” study co-lead Dr Sebastian Lunke says.

The study was initially established with funding from Australian Genomics, The Royal Children’s Hospital Foundation, the Sydney Children’s Hospital Network and the Channel 7 Children’s Research Foundation, and has now transitioned to Genomics Health Futures Mission funding. VCGS received National Association of Testing Authorities (NATA) accreditation to perform whole genome sequencing in 2019, in addition to its accredited exome sequencing service.

Global leadership in a shrinking world Genomic medicine is rapidly transforming healthcare in Australia and around the world.

MCRI is at the forefront of this transformation, sharing data, tools and knowledge to advance genomic research and medicine, and to help reduce the burden on families and society. MCRI is helping to create a global framework to integrate genomics effectively and equitably into healthcare for children and their families.

Professor North is also Vice-Chair of the Global Alliance for Genomics and Health (GA4GH), a collaborative network of more than 600 organisations across 100 countries. The Alliance develops and implements technical and regulatory standards to share clinical and genomic data responsibly for the benefit of human health.

Below are some of the impacts VCGS made in 2020, representing the outstanding dedication and commitment of VCGS staff to ensuring continuous service provision for the benefit of children and families.

Levi Trowbridge has a rare epilepsy. Following genetic sequencing, his medication was changed and he is now seizure free.

1000+ 3 days 76,872 27%

42%

Victorian couples tested by VCGS through Mackenzie’s Mission

Expected percentage of children who will receive a diagnosis through RDNow

Mackenzie’s Mission is a research project offering genetic carrier screening to couples planning or in the early stages of pregnancy, providing information about their chances of having a child with a serious genetic condition. Couples tested through VCGS can meet with our genetic counselling team, who help couples make sense of their results and support them to make choices according to their own values.

Time it takes to complete ultra-rapid genomic sequencing for critically ill children The Acute Care Genomics study provides ultra-rapid genomic sequencing to critically ill babies and children in intensive care. Ultra-rapid testing through VCGS is regularly completed in three days, instead of three to six months.

VCGS has processed close to 300 tests, helping families in the search for a genetic diagnosis. More than half receive a diagnosis, meaning fewer hospital days, fewer invasive procedures and, in some cases, access to lifesaving treatments.

Victorian babies screened by VCGS in 2020 VCGS delivers Victoria’s newborn bloodspot screening test, also known as the heel prick test, in partnership with the Victorian Department of Health.

Newborn bloodspot screening identifies babies that have rare but serious medical conditions. Early diagnosis and treatment can improve outcomes and save lives.

Growth in clinical genomic sequencing, helping more families by uncovering the cause of rare genetic disease Clinical genomic sequencing is a powerful test that helps identify the cause of difficult health and developmental problems. In many cases, it is used to seek answers for patients where other testing has failed to find a cause.

During 2020, VCGS not only maintained its exceptional service quality, but also significantly increased the number of children and families we helped through clinical genomic sequencing.

Rare Diseases Now (RDNow) is the last tier of our genomics investigation. RDNow draws on the research and clinical expertise of MCRI and VCGS to establish a pathway for children who, despite all best efforts, remain undiagnosed.

Through cutting-edge technologies, RDNow gives these children the best chance of receiving a diagnosis and access to the latest clinical trials and treatments.

Annual Report 2020

Annual Report 2020 RESEARCH IMPACT

15

Two families making a difference David Hunter and Melinda Anderson are typical parents who only want the best for their children. Unfortunately, they have had to fight a lot harder for their kids than your average family.

Angus Hunter David’s son, Angus, and Melinda’s daughter, Sarah, both have rare, life-threatening genetic disorders for which scientists are desperately seeking a better understanding and potential treatments.

David fears that he will never hear his son call him “Dad”. Angus has an extremely rare and little understood genetic brain disorder called BPAN – beta-propeller proteinassociated neurodegeneration.

David and his wife, Edwina, first became concerned something was wrong when Angus began missing developmental milestones, like rolling over and sitting.

Initially, BPAN appears as a developmental delay; many children with BPAN, including Angus, don’t acquire speech, for example.

What frightens David most about Angus’s condition is that BPAN will one day inevitably accelerate. Should that day come ahead of a proven treatment, Angus’s health would decline rapidly. He would lose every skill he has mastered, from sitting to eating and swallowing.

Time is therefore critical: a cure or a treatment needs to be found for Angus and other children like him, before they reach their late teens or early 20s when the deterioration starts. That is why raising funds to support scientific research into BPAN is crucial.

David has become active in raising awareness and support for BPAN research, having raised over $400,000. He and Edwina believe BPAN is a mystery Australia’s top medical minds can crack and are continuing to do all they can to support BPAN research..

Sarah Anderson Melinda’s daughter, Sarah, has a different syndrome. It’s called PURA, a rare condition primarily associated with a neurodevelopmental disorder.

The first six years of Sarah’s life were dominated by epilepsy and daily seizures.

Her diagnosis came after exhaustive gene detective work that gave Sarah’s condition a name and an attachment to a unique international community that shares a common goal – to find a cure for this debilitating condition.

While Sarah’s family took comfort in finally knowing what her condition was, their response has been inspirational.

Melinda helped form a support system with families in the US, Europe and Canada.

“We weren’t alone anymore,” she reflects.

“Having the diagnosis gave us an extended family, a sense of belonging, and people who understood. It gave us a focus and a community that would celebrate our successes and provide strength to endure the hardships.”

Reaching out to other families, Melinda established the PURA Syndrome Foundation and helped create a global research network that links MCRI researchers with others around the globe. By 2018, they had established a global patient registry and biobank network, and helped invigorate epilepsy and stem cell research.

Now, funds are desperately needed to turbocharge that effort.

Angus Hunter’s cells are helping MCRI researchers uncover BPAN’s mysteries.

16

MurdochChildren’s Children’sResearch ResearchInstitute Institute Murdoch

Annual Report 2020

Annual Report 2020 RESEARCH IMPACT

17

Global health The right simply to breathe COVID-19 brought into sharp relief the importance of respiratory health and the good public health policies and practices that allow sick children in developing countries to do something most of us take for granted – breathe easily.

For decades, MCRI has been at the forefront of efforts to reduce respiratory infections and their consequences in countries in nearby Pacific regions, as well as in south and central Asia and Africa.

The pandemic has highlighted the value of medical oxygen – and exposed the inequity of oxygen access facing millions. Low blood oxygen – known as hypoxemia – is a lifethreatening complication of many illnesses, including pneumonia, sepsis, malaria and COVID-19.

Hypoxemia increases a patient’s risk of dying seven-fold, contributing to more than 1 million deaths in low and middle-income countries every year. Many of these deaths could be prevented with access to the medical oxygen we take for granted in high-income countries.

Unfortunately, many of the world’s hospitals are not properly equipped to diagnose hypoxemia or deliver oxygen to patients.

MCRI researchers have found that in places like Nigeria, over 90 per cent of facilities do not have pulse oximeters, simple handheld devices to measure blood oxygen. And fewer than half of these facilities have a reliable oxygen supply. Because of this, only 20 per cent of hypoxemic patients are diagnosed – and fewer than half of these receive the oxygen therapy they need.

But our researchers have shown that positive change is possible – even in a pandemic – getting oxygen to those who need it and saving lives.

The #InvestInOxygen campaign, launched in 2020, is a partnership between MCRI and the Clinton Health Access Initiative. It aims to raise much needed funds to reduce the burden of low blood oxygen and its effect on child mortality in five countries, which together account for one-third of the global burden of hypoxemia.

A global webinar, hosted by former US President Bill Clinton and featuring Dr Anthony Fauci, acknowledged the enormity of the inequitable access to medical oxygen and its impact on vulnerable children. The joint initiative was recognised as one of the world’s top six entries in the US-based MacArthur Foundation’s $100 million “100&Change” competition.

The team’s next steps are to turn that visibility into philanthropic funding, to make a step-change difference and save hundreds of thousands of lives.

A team in Solomon Islands conducting a scabies prevalence survey.

Protection from pneumonia In many low and middle-income countries, another respiratory illness in addition to COVID-19 wreaks havoc.

Streptococcus pneumoniae is estimated to be responsible for more than half of all pneumonia-related deaths of children under five. Respiratory infections, including severe pneumonia, cause an estimated 800,000 deaths annually, mainly in low and middleincome countries.

At the request of the Laos Ministry of Health, MCRI’s Global Health research team led by Professor Fiona Russell evaluated the benefits of the PCV13 pneumococcal vaccine to support its ongoing use.

The team showed that the vaccine was effective in protecting children in Laos against severe pneumonia, reducing hypoxic pneumonia and pneumonia requiring oxygen support by 37 per cent.

Scan page 18 to find out more about these global projects

18

Murdoch Children’s Research Institute

Defeating scabies “These results provide a compelling argument to continue childhood PCV13 vaccination in Laos and for its introduction into similar countries with high death rates from pneumonia,” Fiona says.

Globally, there are an estimated 455 million cases of scabies recorded annually, with around 1 million people affected at any one time.

The study also described a simple, lowcost, single-site method to assess vaccine effectiveness that was feasible for other low and middle-income countries.

Scabies mites thrive in hot, crowded conditions and can be widespread in some low-income communities, including in remote communities in northern Australia and the south Pacific. Infection rates are especially high among children and the elderly in resource-poor communities.

Meanwhile, Fiona’s leadership and expertise, along with that of the Global Health team, was recognised with NHMRC funding for a new $2.5 million Centre of Research Excellence in Pneumococcal Disease Control.

The Centre’s mission is to support Australia’s Asia-Pacific neighbours to help reduce the number of vaccine doses needed to lower pneumonia-related deaths, while improving vaccine affordability and take-up. A key part of this work is to improve understanding around new strains of the pneumococcal bacteria which can emerge following mass vaccination, and its relevance in the region’s low and middle-income countries.

Scabies are transmitted by skin-to-skin contact with another infected person.

Infection causes constant scratching, leading to bacterial skin infections that in severe cases can lead to blood poisoning, kidney failure and heart disease.

The World Scabies Program (WSP), headed

by Professor Andrew Steer, is a partnership between MCRI and the Fijian Ministry of Health and Medical Services, with funding provided by the Macquarie Group Foundation and other philanthropic groups.

The program aims to eliminate scabies.

The National Steering Committee’s inaugural meeting and official launch of the program, held in 2020 at Mataika House (Fiji Centre for Disease Control), brought together senior health and government representatives with MCRI staff from Melbourne via video conference.

The World Scabies Program will continue to build on the lessons learnt from more than a decade of research to control scabies.

Some of these lessons include that trained local healthcare workers and community volunteers can deliver effective mass drug administration, and the importance of community-based health promotion for messaging around scabies prevention and treatment.

MCRI is a signatory to the ACFID Code of Conduct, which is a voluntary, selfregulatory sector code of practice. As a signatory, we are committed and fully adhere to the ACFID Code of Conduct, conducting our work with transparency, accountability and integrity.

Annual Report 2020

Annual 2020 RESEARCH IMPACT Annual Report Report 2019

19

Flying the flag for Aboriginal health Aboriginal and Torres Strait Islander communities have occupied the continent for more than 60,000 years. Today, Aboriginal and Torres Strait Islander people continue their connection to culture, identity, knowledge and place, and their relationships to Country.

MCRI established its Aboriginal Health research program to address gaps in evidence by working with Aboriginal communities to co-design, implement and evaluate strategies to promote health, wellbeing and equity for Aboriginal children, young people and families.

The first priority is to prevent and reduce impacts of intergenerational trauma and reverse rising rates of mental health disorders, self-harm and suicide among Aboriginal children and young people.

Aboriginal and Torres Strait Islander children and families continue to experience the legacy and ongoing impact of colonisation, loss of land and decimation of culture, language and community, and experiences of racism and discrimination. While there have been significant gains in some domains of child health, there are still many areas where improvements need to be made.

Strong leadership by – and collaboration with – Aboriginal and Torres Strait Islander people is key to effective research responses to the adversity faced by these children and young people.

The MCRI Aboriginal Reference Group was formed to provide strategic guidance and cultural advice to the Aboriginal and Torres Strait Islander Health Research Leadership Team, the Institute Director and Institute Executive.

In 2020, the Group commissioned a digital artwork, Jillay Wannik – Healthy Pathway, by Dixon Patten, a Yorta Yorta and Gunnai artist, to celebrate strong culture, health and wellbeing, and equity for Aboriginal children, young people and families.

Aboriginal Reference Group members:

Jillay Wannik (Healthy Pathway), by Dixon Patten

Bambu Di Kerr

Andrew Jackomos

N’arweet Carolyn Briggs

Janine Mohamed

Justin Mohamed (Co-Chair)

Trevor Pearce

The river in the middle represents life’s flow and journey.

Helen Kennedy (Co-Chair)

Selena White

Shawana Andrews

Graham Gee

Indi Clarke

20

Murdoch Children’s Research Institute

The hands represent Aboriginal children.

The gum leaves represent the local Kulin Nation and symbolise “Welcome to Country”.

The three large circles represent the MCRI implementing programs and policy to influence greater health outcomes for Aboriginal people.

The flowers represent caring, nurturing and growth.

The U-shape symbols within depict parents, carers and support people guiding our young people.

The leaves within spiral outwards, creating a ripple and showing how positive outcomes impact on the individuals, families, mobs and communities.

Annual Report 2020

The smaller coloured circles represent the diverse nations across the Country.

The trail of circles represents our connection to each other.

Annual Report 2020 RESEARCH IMPACT

21

Better outcomes for

mental health

The Centre for Community Child Health, a joint endeavour of MCRI and The Royal Children’s Hospital, plays a key role in ensuring MCRI’s population health research supports the best start in life for every child. The Centre leads an effort that brings together a wide range of research and other projects.

Heading off problems early Half of all mental health issues in children emerge by age 14, with symptoms often starting in primary school, or even infancy.

In a classroom of 25 third-graders, about 10 students can be expected to have emotional or behavioural problems. On average, the numeracy skills of these students are a year behind their peers when they start high school.

And yet, research by Group Leader of Health Services Professor Harriet Hiscock showed the majority of children and adolescents with mental health problems in Australia do not receive the basic standard of treatment. And those who do, don’t actually get better.

MCRI is determined to shake up the system, as Harriet puts it, to improve the way mental healthcare is delivered and outcomes are measured in Australia, and to give children every chance of being as happy and healthy as possible.

In collaboration with the University of Melbourne and the Victorian Department of Education and Training, MCRI piloted a mental health program in 10 primary schools.

Early results are promising, with strong support coming from schools and indications the program can improve care pathways for children with emerging mental health issues.

22

Murdoch Children’s Research Institute

The Institute’s Group Leader of Policy and Equity, Professor Frank Oberklaid, says schools are the perfect platform for detecting mental health problems early, before they become entrenched.

The program successfully embedded a child mental health and wellbeing co-ordinator within schools to identify and manage emerging mental health issues in students as well as providing connections between education, social and health services.

This pilot success meant the program received additional state government funding for a further 16 metropolitan and regional schools, with hopes ultimately for a national rollout.

Reaching out to the regions The Institute’s innovative suite of child mental health and wellbeing programs includes the BY FIVE program, which supports rural kids to make the transition from childcare and kindergarten to primary school.

The BY FIVE project – so called because it covers five geographic clusters within regional Victoria’s Wimmera Southern Mallee and enacts significant change on five developmental outcomes by the time children reach five years of age – won the Creating Collaborative Community Partnerships category at the 2020 Victorian Government Early Years Awards.

Towards a National Centre for Allergies and Anaphylaxis Australia has one of the world’s highest rates of allergies in the world. Food allergy is a particularly pernicious problem, affecting one in 10 infants and one child in every classroom.

Mild food allergy symptoms like hives and swelling are uncomfortable, but severe symptoms such as breathing difficulties (called anaphylaxis) can be life-threatening.

Food allergies also affect a child’s quality of life as they need to be really careful they don’t accidentally eat something at school or childcare that contains foods to which they are allergic.

The BY FIVE Specialist Paediatric Support Partnership (SPSP), developed in response to the 2018 Australian Early Development Census, exposed a growing gap in the school-readiness of rural children compared with those living in cities. BY FIVE is significantly improving health and wellbeing outcomes for children by connecting the specialist expertise of paediatricians with local primary health providers.

The MCRI-led Centre for Food and Allergy Research (CFAR), is a federally funded collaboration of experts in children’s food allergy and food-related immune disorders.

In 2020, a bipartisan inquiry convened by the Federal Parliament’s health committee started investigating ways to combat Australia’s high and rising rates of allergy and severe allergic reactions, while looking into access to services for allergy sufferers.

CFAR submitted a written response to the inquiry which was then presented by MCRI’s A/Prof Kirsten Perrett along with Dr Rachel Peters, Dr Catherine Hornung and PhD student Victoria Soriano.

The inquiry announced its support for all of CFAR’s recommendations:

• Expanding CFAR to establish a National Centre for Allergies and Anaphylaxis.

• Clinical research into food allergy treatments including head-tohead trials of food-based oral immunotherapy.

• Establishing a national register of anaphylaxis episodes.

Simone Albert, a consumer advocate involved in CFAR’s inquiry submission, welcomed the announcement.

“My family has lived with severe multiple allergies for more than 14 years,’’ Simone says.

“As a mental health professional specialising in anxiety and stress management for families living with this chronic condition, I welcome the parliamentary committee’s recommendations as a big step towards understanding why food allergies and anaphylaxis are on the rise, how to prevent future cases and find effective treatments for people with existing food allergies.”

CFAR Director Dr Jennifer Koplin noted that the Centre had led the world in understanding food allergy risk factors and prevalence, with Victoria at the forefront with its anaphylaxis notification scheme.

Expanding CFAR into a comprehensive National Centre would ultimately enhance children’s safety.

Project manager Rachel Robinson says the team was thrilled to have increased the confidence and capacity of primary health services in the region to solve complex care issues in partnership with families.

“Our work in the region found many children were being referred to specialist services located in major cities for health and development issues that could be resolved locally,” she says.

“In a fantastic partnership between The Royal Children’s Hospital, MCRI and local primary care, families are being supported by services they know and professionals are learning more about assessing and managing common paediatric issues.”

PHOTO COURTESY EUGENE HYLAND / NEWSPIX

Annual Report 2020

RESEARCH IMPACT

23

Big ambitions Stem cells drawn from the blood of kidney transplant patient Charlotte Matthews, 7, are helping researchers develop treatments for rare kidney conditions.

See story, page 26

2

Research Excellence Stem cell research

26

Data science

28

Prizes and awards

30

Global mission

32

Benchmarking our efforts: research metrics 34

24

Murdoch Children’s Research Institute

Eyes on the COVID generation

35

From bench to bedside … and back again

36

Annual Report 2020

RESEARCH EXCELLENCE

25

Stem cell research: a great leap forward It’s a four-hour round trip from Wonthaggi, south-east of Melbourne, to Parkville on the CBD fringe. Charlotte Matthews and her family know the route well. They made the trip twice a week for several years so that Charlotte could have lifesaving dialysis.

The seven-year-old was born with congenital nephrotic syndrome, a rare condition where the kidneys remove too many proteins, hormones and vitamins from the blood. Left untreated, it can lead to kidney failure by early childhood.

Charlotte had one of her kidneys removed soon after birth. By 2018, her other kidney was operating at barely 10 per cent of its capacity.

The long car trips remained part of the family’s routine until Charlotte had a kidney transplant the following year.

The surgery changed everything for Charlotte. But as Murdoch Children’s Research Institute’s Dr Cathy Quinlan declared, Charlotte could improve things for other children as well.

Towards personalised medicine By extracting stem cells from Charlotte’s blood, scientists replicated the damaged part of her kidney’s waste-filtration system in order to study what went wrong.

The research raises the prospect of personalised medicine for children with rare kidney conditions, as well as the treatment of diabetic nephropathy, a common cause of kidney failure in adults.

STEM CELL LEADERSHIP Professor Melissa Little, who leads MCRI’s Stem Cell Medicine Program, is using laboratory-made mini-kidneys for her groundbreaking work investigating kidney disease.

26

Murdoch Children’s Research Institute

Scan page 26 to see Melissa discussing stem cell models of COVID-19 infection

MCRI’s Stem Cell Medicine program is led by Professor Melissa Little and has been backed with $10 million from the Stafford Fox Medical Research Foundation, including $2 million for the Stem Cell-Based Disease Modelling Facility. The generous support is allowing Melissa’s team to study tiny 3D kidneys no bigger than a fingernail, created in the lab using a 3D-bioprinter.

The mini-kidneys could be used to test the effectiveness and toxicity of new medications, making drug development less expensive by identifying the most promising candidates to take to clinical trials.

“Ultimately, we want to grow a patient’s kidney in the lab, from their own cells, to eliminate the need for kidney donation,” says Melissa. “It sounds like science fiction, but we are well on the way.”

However, to make a functioning kidney in the lab you first need to ensure its primary function works – namely, filtering waste from the blood.

Immunity in a dish

Enter another of Melissa’s research breakthroughs: eavesdropping on the conversation between the kidney’s filtration unit and the tube that carries the urine to its exit point.

The filter tissue, known as nephrons, and the tubes, or ureters, are formed by different stem cell types. But which signals are important in determining what the stem cells developed into had remained a mystery.

Melissa’s team studied these genetic signals and managed to isolate and coax the nephron cells into becoming the cells at the tip of the ureter, where the urine first enters the “exit pipe”. It’s a significant finding because if the team is able to build a working artificial kidney, the reliance on organ donation could be significantly reduced.

Understanding how the body’s earliest cells are formed is the first step in recreating the process. It’s why the work of Professors Ed Stanley and Andrew Elefanty is so fundamental.

In 2020, their team also had a breakthrough.

Using genetic engineering and a novel way of growing stem cells, they reproduced and visualised the earliest developmental steps in human immune cell production in a laboratory. It means researchers can better understand childhood diseases and autoimmune conditions.

“We can use this new knowledge to test ideas about how diseases like childhood leukaemia and type 1 diabetes develop, while understanding more about the steps these cells go through, and how we can more efficiently nudge them down a desired pathway,’’ Andrew says.

Annual Report 2020

RESEARCH EXCELLENCE

27

Data science Translating big data into precision clinical care Never content to stand still, MCRI has added another enabling platform to ensure that sick children and their families receive the right kind of care – at the right time, and in the right setting.

This concept is known as “precision clinical care”, and is the future of gold-standard healthcare.

The Institute is determined to remain a leader in using data to improve all aspects of research and clinical care by establishing the Data Science Initiative – bringing together teams of data scientists, software designers and computational biologists across an array of research fields.

The initiative will drive new methods and opportunities to bridge individual genomes and dynamic physiology to detect disease at an early stage and uncover lifestyle and environmental patterns associated with disease. It will also allow us to answer the big questions associated with treatment resistance and recurrence risk.

The world is entering a new era of genomic research, where Big Data is starting to exceed our existing analytical capabilities.

We now need innovative systems to appropriately manage, process and integrate huge experimental datasets to fully visualise and understand the embedded information.

With this initiative, the Institute continues to tackle the most difficult challenges in aggregating, integrating and, crucially, protecting personal data to advance our understanding of disease to enable datadriven precision care.

28

Murdoch Children’s Research Institute

The best research requires the best people to drive it forward. In 2020, MCRI recruited and retained some amazing precision health experts, delivering the right diagnosis and treatments at the right time, to the right patients.

Joining forces to accelerate gene discovery In 2020, MCRI and Sydney’s Garvan Institute of Medical Research joined forces to establish the Australian Centre for Population Genomics.

Recruited from the Broad Institute at Harvard University, Associate Professor Daniel MacArthur is leading the Centre, which aims to advance the prediction and diagnosis of inherited and complex genetic diseases, and accelerate gene discovery so as to speed the development of new therapeutics.

Australia is the ideal setting for this Centre.

While the vast majority of global genomics datasets collected and described so far relate to Europeans, Australia’s multiethnic population includes people from Africa, Asia, South America and the Asia-Pacific region, as well as, of course, Aboriginal and Torres Strait Islanders.

This means the Centre’s contribution to genomic health will be truly representative and relevant to the health of all peoples.

Grey matters

Visualising a better future

A pipeline to success

Professor David Eisenstat was recruited from the Stollery Children’s Hospital and Cross Cancer Institute at the University of Alberta, Canada, to spearhead the Institute’s mission to solve one of the hardest problems in paediatrics – childhood brain cancer.

MCRI successfully recruited expert bioinformatician Associate Professor Mirana Ramialison in 2020 to lead the Institute’s new Transcriptomics and Bioinformatics team.

To best position our genomics research for the future, MCRI in early 2020 founded the Bioinformatics Methods and Platforms group, led by Dr Simon Sadedin, a recognised expert in the field of clinical bioinformatics. Simon developed the current Genomics Informatics Platform for the Victorian Clinical Genetics Services (VCGS).

David pushes the scientific boundaries at the interface between cell fate, normal development and cancer, delving into how the massive cell proliferation in children’s developing brains can sometimes tip over into cancer, with disastrous consequences.

He is a clinician scientist and has also been appointed Director of the Children’s Cancer Centre at The Royal Children’s Hospital, where he will help develop links between basic, translational and clinical childhood cancer research within the Campus and the Parkville precinct, and across Victoria, Australia and the globe.

Mirana pioneered the 3D visualisation of genetic information (3Dcardiomics) and developed world-first computer programs to mine data. She will work closely with MCRI’s stem cell experts to examine genes involved in organ function, development and disease.

She plans to optimise a Google Earthtype tool, using virtual reality to examine gene expression in four dimensions within whole organs and tissues. This in-situ bioinformatics tool, available to only three institutes globally, will fast forward our knowledge of the genetic networks that make up a healthy embryo, and how these networks are disrupted in embryonic diseases.

This group will provide state-of-theart genomic resources, expertise and infrastructure to meet the needs of our researchers from across the Institute. The group also gives us the expertise required to leverage and support the Centre for Population Genomics.

Building the team for our next-generation genomics research requires recruiting the very best experts in the fields of software engineering, data science and genomics.

In 2020, the Bioinformatics Methods and Platforms group added six new scientists, enabling the team to build, deploy and optimise state of the art tools for making rare diseases diagnoses at the Institute.

Recruitment of the research team across MCRI and Garvan is almost complete, with several projects kicking off in 2021.

Annual Report 2020

RESEARCH EXCELLENCE

29

Prizes and awards MCRI researchers were recognised with a variety of fellowships, prizes and national honours in 2020. The Institute Amanda Gwee attracts and inspires Premier’s Awards for Health and Research finalist (Clinical high-performing Medical Researcher category). individuals to do their For research into determining the best dose antibiotic vancomycin for babies very best work and ofto the improve the treatment of bloodstream reach new heights. infections.

BUPA Emerging Health Researcher Award. Recognised for research that’s led to new antibiotic dosing guidelines for babies.

Andrew Steer Bailey K. Ashford Medal from American Society of Tropical Medicine for distinguished work in tropical medicine.

Australian Academy of Health and Medical Science Jian Zhou Medal which recognises a rising star in translational medicine.

Elected to Australian Academy of Health and Medical Science in recognition of outstanding contributions to Australian health and medical research.

Jono Kaufman

Margie Danchin

Melissa Lee

Clinical Investigator Award from Viertel Charitable Foundation.

Bob and June Prickett Churchill Fellowship to improve vaccine and risk communication to optimise COVID-19 and routine vaccine acceptance and uptake.

Premier’s Awards for Health and Medical Research (Clinical Researcher category) for improving outcomes after surgery for patients with a common congenital heart disease condition.

Premier’s Awards for Health and Medical Research finalist (Health Services Researcher category) for research into the effective investigation of urinary tract infections in young children.

Angela Morgan

Daniel Engelman

Melissa Little

Roy Robins Browne

Sharon Goldfeld

Sir William Kilpatrick Churchill Fellowship to develop a best practice approach to treating child speech disorders.

Dr Dorothea Sandars Churchill Fellowship to develop best practice approaches for population mapping of scabies and tropical skin diseases.

Julian Wells Medal, Lorne Genome Conference, for outstanding contribution to genome research in Australia.

Lifetime achievement award from the Australian Society for Microbiology.

Marles Medal, University of Melbourne, recognises excellence in research impact.

Erin Crellin

Graeme Barnes

Susan Sawyer

Melanie Neeland

Sarath Ranganathan

Individual Best Achievement in Medical Research at the Aspire Awards, celebrating leadership, achievement, contributions and abilities of people with a disability across a wide range of sectors.

Queen’s birthday AO for distinguished service to medicine.

Queen’s birthday OAM for service to tertiary education, adolescent health and international groups.

Chan Zuckerberg Award, Single-Cell Analysis of Inflammation award.

Chan Zuckerberg Award, Single-Cell Analysis of Inflammation award.

NHMRC Elizabeth Blackburn Investigator Grant Award - highest ranked female applicant (Leadership category) for research in Clinical Medicine and Science.

30

Murdoch Children’s Research Institute

Australia Day – Officer of the Order (AO) for distinguished service to education and research in the field of microbiology and immunology and to professional groups.

Annual Report 2020

RESEARCH EXCELLENCE

31

Global mission

From left:

Fiona Russell Member, WHO Pacific Joint Incident Management Team, COVID-19 Vaccine Pillars working group

32

MCRI senior staff are not only prolific researchers in their own right, but also lend their expertise and wisdom to some of the most influential national and international bodies in global medical research. Here are just some of the ways our selfless team contributes to advocacy efforts the world over.

George Patton Member, WHO Strategic and Technical Advisory Group of Experts (STAGE) on Maternal, Newborn, Child and Adolescent Health and Nutrition

Murdoch Children’s Research Institute

Jim Buttery Member, Advisory Committee on Vaccines, Therapeutic Goods Administration

Susan Sawyer President, International Association for Adolescent Health

Daniel Engelman Chair, International Alliance for the Control of Scabies

Kim Mulholland Member, WHO Strategic Advisory Group of Experts on Immunization (SAGE)

John Christodoulou AM Board, Mito Foundation

Margie Danchin Chair, Collaboration on Social Science and Immunisation (COSSI)

Kirsten Perrett Chair, National Allergy Clinical Trials Network

Andrew Steer (front row) Co-Director of the Strep A Vaccine Global Consortium

David Thorburn (back row) Board, Mito Foundation

Annual Report 2020

Melissa Little President Elect, International Society for Stem Cell Research

RESEARCH EXCELLENCE

33

Benchmarking our efforts Research metrics

Eyes on the COVID generation PHOTO COURTESY NICKI CONNOLLY / NEWSPIX

Our place in the world

Scan page 35 to hear more about GenV

MCRI has published more than 11,000 research papers with its top 20 collaborating nations over the last 35 years.

MCRI tracks the performance of its research output against Australian and international benchmarks. Over the last five years, MCRI research was cited 2.67 times more than the average for comparable research bodies. Our researchers consistently catapult the Institute into the very top echelons of global child health research and 2020 proved no exception.

34

Murdoch Children’s Research Institute

1058 21.2 Peer-reviewed publications

Citations per publication (2015-2020 )

95

32%

93

763

Publications in top 10% of their research field

7236 Publications (2015-2020)

MCRI’s 5-year ‘h-index’ (a combined measure of academic output and impact)

128

Countries collaborating with MCRI

Research collaboration agreements signed

MCRI papers were mentioned 927 times in patents in 2020

Top 3

MCRI’s international ranking in child health research according to Clarvariate Analytics

Noted and notable Professor George Patton and Dr Michael Poidinger were named among the world’s most highly cited researchers for 2020.

Dr Melanie Neeland’s research on children’s immune system responses to COVID-19 was one of Nature Publishing Group’s Top 50 most-read COVID papers.

GenV, or Generation Victoria, aims to transform the health and wellbeing of an entire generation. And because that generation has been born into a world forever altered by a global pandemic, its establishment could not have been more important.

Years in the planning, and following support from The Royal Children’s Hospital Foundation, MCRI Development Board, the Paul Ramsay Foundation and the Victorian Government, GenV recruited its first participant in December 2020 – baby Eliana, who was born on Christmas Day. Eliana will be joined eventually by thousands of other Victorian children born in 2021 and 2022 (and their parents) to provide a complete picture of the health and wellbeing of a whole generation, allowing researchers to predict, prevent and treat problems faster – and with greater precision – than ever before.

GenV also includes the development of one of the world’s biggest pregnancy and childhood biobanks to store samples for future laboratory analysis. In 2020, the new state-of-the-art facility became operational at the Melbourne Children’s Campus. This fully automated biobank is the biggest of its type in the Southern Hemisphere, transforming the way samples are stored and accessed.

Keeping it out of the family Two of the Institute’s longest running cohort studies have shed new light on how the mental health of prospective parents is linked to the premature birth of their children.

The 2000 Stories: Victorian Adolescent Health Cohort Study (VAHCS) is a landmark longitudinal study that began in 1992, involving 2000 year-9 students from across Victoria. The selected 14- and 15-year-olds completed six interviews during their school years to year 12, as well as five interviews in adulthood (when aged around 21, 24, 29, 35 and 42).

A subset of the recruits went on to have their own children, creating the unique opportunity to explore how the health of one generation can influence the next, and leading to the launch in 2006 of the Victorian Intergenerational Health Cohort Study (VIHCS).

As one of the first cohorts in the world to embed child development study within an existing longitudinal study of parent psychosocial development, the VIHCS is already producing dramatic results.

In 2020, the study team showed that both a mother’s and father’s mental health influences whether their baby will be born prematurely.

Men with persistent mental health problems through adolescence and young adulthood were more likely to have a premature baby.

Women with anxiety and depression during pregnancy were more likely to have a preterm birth.

Dr Elizabeth Spry says that prior to this study the impact of maternal and paternal mental health history on offspring preterm birth and birth weight was unknown.

The study involved 398 women and 267 men who were assessed over 15 years for anxiety and depressive symptoms from adolescence to young adulthood and during subsequent pregnancies.

“We found that men with persistent mental health symptoms in the decades leading up to pregnancy were more likely to have premature babies,” Elizabeth says. “Our study joins growing evidence of the important role that fathers play in the health and development of their children, and suggests that these links begin well before babies are conceived.”

The researchers believe that public health recommendations, almost entirely focused on what mums should and shouldn’t do when planning pregnancy or having a child, should be expanded to give guidance and support to fathers-to-be as well.

Annual Report 2020

RESEARCH EXCELLENCE

35

From bench to bedside … and back again MCRI fosters a culture of collaboration with the Melbourne Children’s Campus partners – The Royal Children’s Hospital, the University of Melbourne and The Royal Children’s Hospital Foundation – to rapidly embed research results in the fabric of everyday clinical care, and enable the careers of many talented clinician researchers.

A/Prof Ken Pang with Charlotte, a client at the Gender Service.

• 27 Fellows have been appointed since 2016 across 20 clinical departments at The Royal Children’s Hospital

Fellows have: • Leveraged more than $15 million from competitive external funding sources • Been appointed to lead two national NHMRC Centres of Research Excellence • Authored more than 400 peerreviewed publications

Critically, our Clinician Scientist Fellows are funded by The Royal Children’s Hospital Foundation to give them valuable time to perform research, whereas previously they conducted unpaid research outside their clinical hours.

Fellows who have received recognition:

The scheme enables and enhances a twoway flow of information to improve research translation. Research findings can be quickly and effectively applied in the clinical setting, with the research agenda directly set by real-life issues that clinicians and their patients face every day.

Trans20 aims to determine the biopsychosocial outcomes for transgender individuals attending the Gender Service, looking at gender identity, transition, dysphoria and gender-related healthcare, as well as mental and physical health, education, quality of life and family functioning.

Putting transgender health on the radar

Over the next 20 years, the team will be able to address important questions, such as the evolution of gender identity and dysphoria, and the role and safety of different interventions, both medical (such as puberty blockers and cross-sex hormones) and psychosocial (such as cognitive behavioural therapy).

Associate Professor Ken Pang is one of 18 Clinician Scientist Fellows funded by the Foundation. After starting out as an immunologist and genetic researcher, Ken’s clinical and research priorities have evolved into providing the best care possible for trans and gender-diverse children and adolescents.

Transgender healthcare for children and young people is a relatively new field, and more research is urgently needed to better inform the team’s clinical work and ensure safe and effective outcomes.

This is especially important given that trans and gender-diverse young people face significant health and social challenges. For example, Australian data show that 80 per cent of transgender young people self-harm and 48 per cent attempt suicide before the age of 24.

Ken runs the Institute’s Trans20 research project, a longitudinal cohort study that began in 2017 with the generous support of the Foundation.

36

Murdoch Children’s Research Institute

Fellowships at a glance

During 2020, the Trans20 team released results from studies examining rates of fertility preservation and showed that more than 60 per cent of transgender adolescents assigned male at birth attempted fertility preservation prior to hormone use at the Gender Service – a much higher rate than reported in other studies.

The research highlights the importance of offering affordable fertility preservation to these young people.

The Hugh D. T. Williamson Foundation, which has a long standing relationship with the RCH, recognised the importance of the Trans20 project, announcing in 2020 that it will join the RCH Foundation in funding Trans20 for another three and a half years, through the support of two positions, The Hugh Williamson Foundation Trans20 Research Fellow and The Hugh Williamson Foundation Trans20 Principal Research Fellow.

“Receiving a Clinician Scientist Fellowship was crucial in allowing me to switch my research interests from lab-based immunology to clinical transgender health research. The RCH Foundation plays such a pivotal role in translating the Institute’s groundbreaking research into the Hospital’s excellent clinical care and I know all the Fellows express their sincere gratitude to the Foundation.”

• Franz Babl NHMRC Practitioner Fellowship • Amanda Gwee Gustav Nossal Award (highestranked applicant for an NHMRC Postgraduate Scholarship); Research Establishment Fellowship from the Royal Australasian College of Physicians (RACP) • Amanda Gwee and Penelope Bryant MCRI/University of Melbourne Strategic Grants for Outstanding Women • Hamish Graham $20,000 CSL Florey Next Generation Award; Finalist, Premier’s Award for Health and Medical Research • Sebastian King Selected to participate in the Australian Academy of Health and Medical Sciences Mentor Program; appointed MCRI Co-Group Leader, Surgery

KEN PANG

Annual Report 2020

Annual Report 2020 RESEARCH EXCELLENCE

37

Leading the way MCRI is blessed to have many role models and inspiring leaders within its ranks, including those who have helped break down barriers to create an inclusive workplace.

See story, page 40

Pictured: Chloe, 6, is the niece of Senior Research Officer Dr Libby Hughes.

3

People and Culture

Leading by example

40

Making our mark: media engagements

42

Supporting outstanding researchers

44

Delivering outstanding research 45

38

Murdoch Children’s Research Institute

Annual Report 2020

Annual 2020 PEOPLE &Report CULTURE

39

Leading by example Acknowledged as a leader in medical research, MCRI is proud to have staff who have dedicated time and energy to breaking down barriers and creating more inclusive workplaces. Among them are Dr Sarah Stephenson and Erin Crellin, whose advocacy has made lasting contributions to the Institute and beyond.

Sarah Stephenson

Erin Crellin

Sarah (pictured, left) is a natural leader. She heads up MCRI’s Neurogenetics group and, in June 2018, co-founded QueersInScience.

She hasn’t yet completed her PhD but already Erin Crellin (right) is being described as a trailblazer.

Last year, Sarah was named an OUTstanding LGBT+ Role Model by the global consultancy group INvolve in recognition of her role in establishing QueersInScience.

It was her Master’s supervisors, including MCRI’s Dr Belinda McClaren and Professor Clara Gaff, who nominated her for an Aspire Award to recognise her exceptional leadership qualities and pioneering role in disability advocacy. The Awards are supported by Funds in Court and the Supreme Court of Victoria.

Having grown to include chapters in all states, the organisation is dedicated to improving support for LGBTQIA+ people working in the fields of science, technology, engineering, mathematics and medicine (STEMM).

“It is always lovely to be recognised for the work that we do,” she says. “For me, this award reflects the hard work and dedication of our entire team.”

The organisation’s aim is simple, adds the molecular biologist. “We aim to increase support, enable education, end harassment and discrimination, and empower professionals, students and organisations.”

QueersInScience also highlights the contributions LGBTQIA+ professionals and students make to an organisation.

With Sarah’s guidance as director, and with the support of the Walter and Eliza Hall Institute, the organisation has connected LGBT+ individuals across isolated research areas. She has secured several government grants and, with her team, organised Australia’s first LGBT+ researcher symposium.

However, Sarah, who identifies as lesbian, says the award was about much more than recognition.

“It’s an opportunity to raise the profile of QueersInScience and hopefully create new conversations and networks to advance LGBTQIA+ inclusion.”

40

Murdoch Children’s Research Institute

“[It’s] in recognition of her being an exceptional role model for young people with disabilities who hope to have careers in medical research,” her supervisors wrote.

The nomination saw Erin, who was born with spinal cord damage and has been in a wheelchair full-time since the age of 18, go on to win the Individual Best Achievement in Medical Research award.

With a Master of Biomedical Science under her belt, Erin has started her PhD research with MCRI’s Genomics in Society group, supported by an NHMRC postgraduate scholarship.

Erin will investigate how genomic testing can best be supported to ensure equitable delivery. Her research focuses on children under 10 with an intellectual disability or developmental delay as a case study.

Genome sequencing for this purpose was funded through Medicare for the first time in 2020.

Janet’s story As part of the Institute’s “day of disability” in 2020, staff were invited to share their experiences of living and working with a disability by writing stories, without the obligation of using their last name. A collection of the stories were shared on the MCRI’s intranet. Here are Janet’s thoughts and insights about working with autism and ADHD during the pandemic.

“Autism or ADHD aren’t inherently disabling. In the right environment, they are the source of my true strengths and purest joy. When given freedom to explore and communicate our knowledge in a calm environment, our divergent thinking, spontaneity, hyperfocus, pattern recognition and observational skills are a huge asset.

“COVID-19 has been undeniably terrible; the feeling of ongoing uncertainty, the discomfort of masks, loneliness, curfews, homeschooling and the awkwardness and fatigue of Zoom meetings. These are probably the closest tangible examples that I could provide to neurotypical people of what autism and ADHD can feel like on a daily basis.

“Neurodivergent people are often described as being like the ‘canary in the coalmine’ – the adjustments that work for us can benefit the whole workforce. The silver lining of COVID-19 is the switch to telecommuting and other adjustments. Many people across MCRI found it suited them wonderfully, enhancing their productivity while allowing them to fit in other commitments. Accessibility really can benefit everyone!”

“We want to make sure it doesn’t widen the existing health disparities and access to services that already exist,’’ Erin says.

A member of MCRI’s Accessibility Working Group, Erin says she is looking forward to helping improve accessibility – not just for researchers but for research participants as well.

Annual Report 2020

PEOPLE & CULTURE

41

Scan page 43 to see more about the global media splash made by the BRACE trial

In a very crowded health media space due to the pandemic, MCRI achieved a record amount of media coverage of both the Institute’s COVID-19 research and our core research strengths.

Media mentions

2019

8086

During 2020, MCRI released six episodes of our COVID-19 podcast for parents and carers, discussing ways to maintain wellbeing in toddlers and school-aged children, how to transition back to school after lockdown and outlining how COVID-19 impacts kids. These have been downloaded almost 4000 times since they were first released in April 2020.

online mentions of MCRI research papers

blog mentions

5838

5250

Our researchers fully embraced social media in 2020, leveraging and expanding their professional networks to spread the word of new publications, clinical guidelines and policies. The Office of Research uses Altmetric to track the performance of academic documents in the social media sphere.

55,249 448 39

2020

2709 695

1442

AU online news

Global coverage

Broadcast (AU)

Social media/Digital

2020

policy document mentions

Youtube Twitter Facebook

62,789 likes/shares

42

Murdoch Children’s Research Institute

27,954 likes and RTs

LinkedIn

19,321 likes and shares

23,798 views Totalling

893 hours

Annual Report 2020

Website

440,209 unique browsers

PEOPLE & CULTURE

43

Supporting outstanding researchers The Institute’s groundbreaking research can only be carried out by world-leading researchers, supported by first-class operational, infrastructure and administrative teams.

In a year that was immensely challenging – both personally and professionally – MCRI’s dedicated Operations teams:

•

CO-ORDINATED the safe and speedy repatriation of researchers from across the world ahead of Australia’s border closure

•

ESTABLISHED the ‘Flexi-75’ program, in which staff could reduce their hours to 75 per cent to support their families

•

INITIATED a number of fun events and activities to keep the organisation’s 2000-plus team engaged, happy and involved

•

RECRUITED 167 staff while working from home, many of whom only met their co-workers for the first time at their Christmas picnics

•

KEPT staff engaged, with high open rates of all-staff communiques, and more than 650 employees attending the monthly virtual ‘town hall’ events

Staff ‘pulse’ surveys conducted during 2020 showed:

95 % 93 % 85 96% 89% %

of staff reported the organisation was agile and competent in its response to changing COVID-19 circumstances thought that leaders made effective decisions given the complexity of COVID-19 reported their manager kept them informed, providing guidance and support reported being informed about safety and staying healthy when working on and off site were agile in adapting to new systems and processes to support getting work done efficiently

Some staff statistics:

2808 73% 27%

Total staff (paid employees, unpaid staff and students)

Female employees

Male employees

44

Murdoch Children’s Research Institute

Delivering outstanding research MCRI is home to more than 350 Doctoral, Master’s and Honours students, whose youth, energy and optimism contribute so much to the fabric of the Institute.

Added to their ranks are the postdoctoral researchers – the engine room of the Institute – responsible for hands-on training of students, drafting grant applications, and transforming complex laboratory and clinical research results into manuscripts.

With Suzi Carp stepping down from the Board, the Institute’s donors stepped up, creating the Suzi Carp Fund as a mark of thanks and in recognition of Suzi’s contribution to MCRI. The fund will provide an annual stipend for a doctoral student in the early stages of their research career.

We caught up with two of our students to ask how they continued to produce groundbreaking research, provide excellent patient care and look after themselves during 2020.

CONOR McCAFFERTY

MELISSA LEE

Second-year PhD student in Haematology, supervised by Professors Vera Ignjatovic, Paul Monagle and Dr Chantal Attard

Early-career clinicianresearcher in the Heart Research group.

How did you come to be interested in a research career?

I started my research career as an MCRI summer student. From there, I caught the research bug – becoming a volunteer, Honours student and now a PhD student.

What’s your current research topic, and can you share one particularly interesting finding from 2020?

At the start of 2020 I pivoted with my project and am now looking at the relationship between COVID-19 and blood clotting. We have seen some fascinating proof of the way COVID-19 works differently in children and adults, especially in regards to clotting risk.

How did you find studying and working in 2020?

Being involved in COVID-19 research, I was on site for most of the year, but it was still difficult to see an empty MCRI. I missed the buzz of MCRI and I really missed seeing my full team in person. Needing to do almost everything over Zoom added so many layers of difficulty to planning and co-ordinating a new project.

On the flip side, did anything great come out of 2020?

I got to work with some incredibly talented teams and researchers inside MCRI and around Parkville that I likely wouldn’t have met otherwise.

What has 2020 highlighted for you about MCRI, and what would you say to students/junior doctors thinking about joining?

I saw how quickly different people in MCRI could come together when we were all addressing a common problem. I would just want future students to know how respected and validated I was made to feel when I was suddenly thrown into big meetings with senior researchers.

How did you come to be interested in a research career?

I completed a year of research in 2010 as part of my medical school training at the University of Melbourne. I kept going during the rest of my medical school years, which turned into a PhD (2019) alongside my junior doctor years.

What’s your current research topic, and can you share one particularly interesting finding from 2020?

My entire research path has been investigating long-term outcomes after coarctation repair, a congenital heart defect that babies can be born with where there is a narrowing in the aorta. Many patients go on to develop hypertension later in life though even as early as adolescence and young adulthood. One particularly interesting finding is that this hypertension often develops despite successful surgery and without any further narrowing in the aorta.

How did you find studying and working in 2020?

I think one of the key things has been our ability to move almost entirely to virtual platforms – not only for clinical and research meetings but even for telehealth interactions with patients.

On the flip side, did anything great come out of 2020?

I was awarded my first major post-PhD grant, a Medical Research Future Fund (MRFF) Investigator Grant, to support my coarctation research. I was also extremely humbled to receive the 2020 Premier’s Award for Health and Medical Research (Clinical Researcher).

What has 2020 highlighted for you about MCRI, and what would you say to students/junior doctors thinking about joining?

It is a wonderful place to work not only due to the endless opportunities and world-class knowledge and facilities on offer, but also because of the friendliness and support of all who work there.

Annual Report 2020

PEOPLE & CULTURE

45

What we achieve together “During such a challenging year, we greatly appreciate the ongoing support and commitment of our donors.

When we put our heads and hearts together, we can make a remarkable impact on children’s lives.”

Matthew Hannan, Head of Engagement and Philanthropy E: philanthropy@mcri.edu.au T: 03 8341 6200 Pictured: Maria and Eddie Solaka with Baby Eliana and Jaala Pulford, MP.

4

Sustainability and Engagement

46 46

Murdoch Murdoch Children’s Children’s Research Research Institute Institute

Partnering with purpose

48

Thank you

50

Mapping a solution to MD

52

A remarkable contribution

53

Our donors

54

MCRI Board of Directors

56

The year at a glance: MCRI financials

58

Annual Report 2020

Report 2020 SUSTAINABILITY &Annual ENGAGEMENT

47

Partnering with purpose Government funding helps drive our ambition Both the Victorian and Commonwealth governments are crucial partners in MCRI’s relentless drive for medical breakthroughs.

From research into single genes, molecules and cells, to cohort studies on large populations, government grants are critical in underpinning the Institute’s success.

This valuable financial support spans doctoral students just embarking on their careers to whole multidisciplinary teams.

In 2020, MCRI received more than $20 million in funding from the National Health and Medical Research Council and the Medical Research Future Fund, allowing child health researchers to investigate matters as diverse as: • The medicinal properties of cannabis oil, and whether they can reduce behavioural outbursts in children with intellectual disability • Improved detection of child spine and neck injury, and the management of persistent post-concussion symptoms • Reduced antibody doses and associated anti-microbial resistance needed for clearing urinary tract infections • Preterm birth and how it affects brain development in newborns, and assessing the risk of poor cognitive and motor outcomes for these children • Improved detection techniques for the trickiest of genetic disorders, those caused by repeating 'stutters' of DNA that have so far confounded even the most advanced sequencing machines.

Recognising MCRI’s leading role in advocating for effective change to the status quo, two new $2.5 million NHMRC Centres of Research Excellence are also springing into existence, joining another seven already hosted at MCRI. In 2020, the NHMRC funded the $2.5 million Stronger Futures Centre of Research Excellence (CRE), to be led by Professor Stephanie Brown.

The Centre’s goal is to build family and community resilience to break cycles of intergenerational trauma and social inequity.

Focusing on Aboriginal and Torres Strait Islanders, refugee and socially disadvantaged families and communities, the voices of children, young people, families, communities and other key stakeholders will inform bestpractice approaches.

“Genomics is revolutionising medicine. But peering into the genome of a child, even to save their life, raises especially difficult ethical questions. The Centre for Ethics of Paediatric Genomics will unravel these puzzles and help develop evidencebased advice and guidelines to improve policy and practice.”

DR CHRIS GYNGELL

The second CRE to be funded in 2020 is the CRE for Pneumococcal Disease Control in the Asia-Pacific region, led by Professor Fiona Russell (read more on page 18).

Adding another string to the Institute’s genomics bow, $460,000 from the MRFF’s Genomics Health Futures Mission is being used to establish the Centre for Ethics of Paediatric Genomics. The Centre, the first of its kind to focus specifically on paediatric genomics, will be overseen by Dr Christopher Gyngell and Professor Julian Savulescu.

“This research will improve our understanding of the impact of rapid genomic sequencing on critically ill children and their families, clinicians and health systems,” says Chris.

In 2020, more than $20 million of federal funding went towards

6 MRFF grants 5 Postgraduate scholarships 5 Investigator grants 2 Centres of Research Excellence 2 Ideas grants 48

Murdoch Children’s Research Institute

Annual Report 2020

SUSTAINABILITY & ENGAGEMENT

49

Thank you Despite 2020 being such a difficult year for so many people, we saw a renewed passion, commitment and determination among our donors and supporters to ensure that we had a significant impact on people’s lives during the pandemic.

In a year where adverse circumstances were predicted to have a calamitous effect on charitable giving, our incredible network of supporters instead helped us raise a record $26.8 million.

We are so privileged to nurture these relationships every day, as your stories about your reasons for giving inspire us to ever greater heights. We remain so humbled by the generosity and selflessness of all our donors and corporate partners. Here are just some examples of that generosity.

In memory of Zaidee Zaidee Rose Alexander Turner, aged 7 years and 22 days, died suddenly in 2004 from a cerebral aneurism. Determined to make the most of her short life, her family started Zaidee’s Rainbow Foundation, raising awareness about the need for more people to sign up as organ and tissue donors like Zaidee, and funds for the Institute’s Heart Regeneration team.

In 2020, the Foundation encouraged people to run, jog, walk, skip or hop and log their kilometres on their favourite fitness app, raising money for MCRI’s stem cellbased heart regeneration research at the same time.

Associate Professor Enzo Porello says the team is making steady progress in using heart stem cells to grow new tissue to repair damaged hearts – hopefully, one day, removing the need for heart transplants.

An enduring legacy As a passionate supporter of MCRI, Elizabeth chose to help children beyond her lifetime with a bequest. “I experienced scarlet fever as a child in the 1940s when antibiotics were new,” she says. “I’m so pleased and grateful that children can now avoid illnesses such as these thanks to incredible advances in our medical knowledge.

"When writing my Will, it was important to me to continue supporting issues that I care deeply about now into the future. I’m very pleased that my gift will have a lasting impact on the health of younger generations.”

For more information, visit mcri.edu.au/bequest

Great corporate citizens Morgan Stanley Foundation provided significant support for the COVID Wellbeing project led by Professor Sharon Goldfeld.

Leveraging our existing LifeCourse cohorts, COVID Wellbeing provides the unique opportunity to understand how the prepandemic experiences of children and families shaped risk and resilience for children and young people during the COVID-19 crisis, and in its aftermath.

Insights gained from the work will help inform targeted prevention and interventions for this pandemic and similar future crises.

News Corp Australia and Foxtel have generously supported MCRI in various ways for more than a decade as the Institute’s media and events partner. Previously, their Foxtel Laps and Step-a-thon initiatives helped raise $6 million for vital research.

In 2020, News Corp Australia and Foxtel provided significant media and advertising in-kind support worth more than $1.8 million, demonstrating their shared commitment to building strong and healthy communities.

We are deeply grateful for their ongoing partnership and commitment to improving children’s health.

“We are thrilled that Foxtel and News Corp Australia can play our part in the health and future of Australian children. We are delighted to support MCRI and the incredible research you do.”

PENNY FOWLER News Corp Australia Community Ambassador PATRICK DELANY Foxtel CEO

50

Murdoch Children’s Research Institute

Annual Report 2020

SUSTAINABILITY & ENGAGEMENT

51

Mapping a solution to MD Tribute to a tireless supporter

A remarkable contribution The impact of Suzi Carp’s remarkable contribution to MCRI will be felt for many years to come. The Suzi Carp Recognition Fund was established at the end of 2020 to honour her more than two decades of service to the Institute, including five years as Chairman.

With scholarships to be awarded in perpetuity, the fund will support the most promising students and early-career researchers at MCRI. This vital support will enable them to focus on their research and achieve their full potential, ensuring that Suzi’s influence on the next generation of child health researchers continues into the future.

Thanks to the incredible generosity of the below philanthropists, more than $1 million was received to launch the fund and foster our nation’s brightest young scientists.

Brandon and Nicky Carp

Bernard and Helen Carp

George & Freda Castan Family

The Calvert-Jones Family

Steven Casper and Ilana Wald

The Orloff Family Charitable Trust

George and Rose Kailis The Fox Family Foundation Michael and Helen Gannon Leigh and Sue Clifford Chestnut Foundation Brian Hamersfeld and Natalie Bloom Zac and Rebecca Fried Nathan and Dahlia Sable James and Miffany Blythe

The Muscular Dystrophy Australia (MDA) building is just seven minutes’ walk from MCRI. Maps of Victoria and New South Wales line the office, speckled with small coloured flags representing an individual and their community affected by muscular dystrophy.

Boris M Struk first founded MDA in 1984 after learning that his three-year-old son, Ryan, had the most common and severe form of muscular dystrophic disease, Duchenne Muscular Dystrophy (DMD). Muscular dystrophies are a group of genetically inherited diseases causing weakness and degeneration of the skeletal muscles.

52

Murdoch Children’s Research Institute

Most children with DMD lose their ability to walk by their mid-teens. Worsening heart and lung problems cause life-threatening complications and eventually death, usually before 30 years of age.

Boris describes MDA as a refuge for families left devastated after a muscular dystrophy diagnosis. He is a strong believer that “people with muscular dystrophies need access to two things that MDA is able to offer: information and support ‘in the now’, and hope through research for a timely solution”. To that end, MDA has been supporting MCRI for 15 years, including through the generous donation of more than $1.5 million to support research into muscular diseases, including DMD. MDA is also helping support the next generation of leading researchers, including Dr Chantal Coles, who works closely with the Muscle Research Group, co-led by MCRI’s Director, Professor Kathryn North, and Associate Professor Peter Houweling.

Dr Coles is conducting several exciting projects, including using patient stem cells to grow muscle tissues that can be “exercised” in a dish and screened with new and existing drug compounds using the Stafford Fox Drug Screening Platform. This research will enable us to better understand why muscular dystrophies occur and test drugs that may be able to stop muscular dystrophy in its tracks.

Boris recently retired after 33 years of leading MDA. Boris’s most endearing features have always been a combination of pride, humility and passion for his cause.

Sadly, Ryan passed away on October 9, 2020. His legacy will live on through MDA. MCRI pays tribute to both Boris and Ryan for their contribution towards our research, and for their ongoing support of MD communities in Australia and overseas. Our partnership brings hope for finding better therapies for patients diagnosed with muscular dystrophies and, eventually, a cure.

Andrew and Amanda Rettig Jeffrey Mahemoff AO and Helen Mahemoff OAM

Paul and Julia Rayner

Jim & Claudia CraigBellwether Foundation Nicholas Tyshing and Narelle Curtis Kate and Dean Mohr The Jack & Robert Smorgon Families Foundation Vicki S Vidor Rosie Lew AM Castan Family David and Kathie Smorgon Andrew and Natalie Bassat

George Lefroy AM and Joan Lefroy AM

Wolf Foundation and SALT Catalyst

Dean and Shareen Joel

Laini Liberman and Ben Rozenes

Tony and Helen Gandel Ariane and Angus Barker

Rodney and Ann Smorgon

Paul and Sharon Bassat

Ian Darling AO and Min Darling

Cassy Liberman and Ben Krasnostein

Loti & Victor Smorgon Family Foundation

For further information about supporting the Suzi Carp Recognition Fund, please visit: https://www.mcri.edu.au/remarkablecontribution

PHOTO COURTESY JOE ARMAO / THE AGE

Annual Report 2020

SUSTAINABILITY & ENGAGEMENT

53

Our donors With life-changing support from our donors, together we have made a positive impact around the world this year.

Sue a’Beckett

Estate of Mary Ellen Macaulay

Wendy James-Ross

Antoinette Albert

Estate of Nance Elizabeth Creaton

David Jenkins and Fran Lefroy

The Ray and Margaret Wilson Foundation

Kenneth Allardice and Julie Roy

Estate of Olive Meuleman

Paul and Julia Rayner

Andrew Alston

Estate of Rosemary Agnes Warner

Jim & Claudia Craig-Bellwether Foundation

Steve Redman

Roseanne Amarant

Eureka Benevolent Foundation

Dean and Shareen Joel

Jennifer Renton

The Andrews Family Trust Charitable Fund

Jane Fenton AM and David Miles AM

The John Barnes Foundation

Rio Tinto

John Burge Trust

Judith Robinson

Angior Family Foundation

Fight Cancer Foundation

Johnstone Family Foundation

Christopher Archibald and Melissa Goode

Flicker of Hope Foundation

Joan Kaaden

Royal Children’s Hospital Foundation

Peter Austin

The Fox Family Foundation

George and Rose Kailis

Scott Farquar and Kim Jackson

Ian Kennedy AM and Sandra Hacker AO

Jo Baevski Balcon Group

Latitude Financial Services

Ariane and Angus Barker

Future Generation Investment Company

Ken and Liz Launder

David and Anne Barton

Amy Gallasch

Andrew and Natalie Bassat

Coral and Tony Gallasch

George Lefroy AM and Joan Lefroy AM

Norma Beaconsfield

Tony and Helen Gandel

Gillian Beaurepaire

Michael and Helen Gannon

Besen Family Foundation

Ross and Judith Gardner

Big W

John and Gaye Gaylard

BioTools Pty Ltd

Andrew and Bev Gelbart

James and Miffany Blythe

George & Freda Castan and the Suzi & Barry Carp families

Garry Bromham The Honourable Sally Brown AM BUPA Australia Brett Burns The Calvert-Jones Family The Calvert-Jones Foundation Cameron Foundation Krystyna Campbell-Pretty AM and Family Cann Group Limited Carla Zampatti Foundation Bernard and Helen Carp Rhea Carr-Davies Hannah Casper Steven Casper and Ilana Wald The Centre for Paediatric Allergies at Epworth Cher Family Foundation Chestnut Foundation Children’s Cancer Foundation Children’s Tumour Foundation of Australia Sandra Clark Leigh Clifford AO and Sue Clifford Corella Fund Rodney Costigan

Gerry Ryan OAM and Valerie Ryan Nathan and Dahlia Sable SALT Catalyst The Samuel Nissen Charitable Foundation

Marj Lefroy

The Scobie and Claire Mackinnon Trust

Nichola Lefroy and Sam Riggall

Colin and Jan Smith

The Lefroy Family

David and Kathie Smorgon

Leukodystrophy Australia

Gita Smorgon

Rosie Lew AM

Rodney and Ann Smorgon

The Lewin Family Foundation

Nathaniel and Christie Snary

Sonia Gilderdale

Cassy Liberman and Ben Krasnostein

Sohn Hearts & Minds Investment Leaders Conference

Goldschlager Family Charitable Foundation

Laini Liberman and Ben Rozenes

Lady Southey AC

Life Healthcare

Patricia Spencer

Gourlay Charitable Trust

Christine MacGill

Neil Spitzer

J R Gregory

Macquarie Group

Maurice Hall

Jeffrey Mahemoff AO and Helen Mahemoff OAM

The Stafford Fox Medical Research Foundation

Brian Hamersfeld and Natalie Bloom

Grant and Terri Stephenson

The Marian & E.H. Flack Trust

Robert and Laura Stone

Sophia Hatzipanagiotidis

Jessica Mariani and Gerry Davies

The Sunraysia Foundation

Heart Foundation

Judy and Bruce Matear

Patricia Tarquinio

Geoffrey Heeley

Chris and Denis McConnell

Polly Taylor

Helen Macpherson Smith Trust

Neil McGregor

Charles Tegner

Raymond Hemphill

The McPhee Charitable Fund

John Henwood

Miranda Milne

Thelma and Paul Constantinou Foundation

The Hersch Family

Kate and Dean Mohr

The Holckner Family

Margaret Molony

Tim Hovey

Mark Montag

David Hunter and Edwina Molony

Morgan Stanley Foundation

The Ian Potter Foundation

Sarah and Lachlan Murdoch

In celebration of Ian & Eileen Whitbourn

Muscular Dystrophy Australia

In memory of Edith ‘Judy’ Ashton

Michael and Vannessa Myer

VCF Bird Family Charitable Trust

The Myer Foundation

Vicki S Vidor

NAB Foundation

Vincent Chiodo Charitable Foundation

In memory of Julie Weston In memory of Kevin Roy Robson In memory of Lina Brotto

Nicholas Tyshing and Narelle Curtis UHG Foundation Uncle Bobs Club Tony and Judy Van Bergen Vanguard Investments Australia Ltd

Baillieu Myer AC and Sarah Myer

Laurence Cox AO and Julie Ann Cox AM

In memory of Matthew R Paikos

National Stem Cell Foundation of Australia

In memory of Sean Martin

Nicholes Family Lawyers

Stephen and Brenda Wellard

Creswick Foundation

In support of Andronicus Parharidis and the KBG community

The Orloff Family Charitable Trust

Tom and Sarah Whinney

Tony Osmond and Fiona Griffiths

Danial and Jackie Whitbourn

P&S Bassat Foundation

Kriss Will and Noel Blake

Rowly and Judy Paterson

Catherine Williams

Paul Ramsay Foundation

Geoff and Shirley Williams

The Phyl Waterhouse Memorial Fund

John and Irene Williams

The Cuming Bequest Mandy and Jonathan Daddia Ian Darling AO and Min Darling Datt Family Foundation

In support of Barney In support of Bruce Lefroy Foundation

Charles Day and Elise Everest

In support of Lachlan Elmer

Doll Association of Victoria

In support of Prof Frank Oberklaid

Annette Domanti

In support of Sebastian Wearn Jarvis

William Downey Equity-One Mortgage Fund Ltd Estate of Daryl Giles Howard Estate of L I Roach Murdoch Children’s Research Institute

Adam and Yoko Ryan and Family

Kim Kerr Jason and Diana Kowaltzke

Maureen and Michael Barden

54

John and Diana Frew Zac and Rebecca Fried

Stuart and Jillian Bales

Newborn Charlie de la Bere and his mum, Rebecca, are two of the newest participants in GenV.

Graeme and Pamela Fraser

Jeremy Ruskin QC and Roz Zalewski

PKU Association of NSW Inc Power Tynan Charitable Trust

The Isabel and John Gilbertson Charitable Trust

QMS Media Limited

The Jack & Robert Smorgon Families Foundation

Rare Friends

Adrian and John Quilter

Annual Report 2020

Kelvin Wearn

The Wolf Foundation Edward and Mandy Yencken The Yulgilbar Foundation Zaidee’s Rainbow Foundation Anonymous Donors

SUSTAINABILITY & ENGAGEMENT

55

MCRI 2020 Board of Directors The Institute’s Board gathers six times a year, and while 2020 saw these meetings take place virtually, the Board seamlessly continued its independent and objective governance and strategic direction setting. We thank all new Board members and those who stepped down for their service.

Committees Development Board

Suzi Carp *

Simon Rothery †

Kate Mohr

Chairman US Development Board member

Deputy Chairman Investment Committee Chair

Development Board Chair US Development Board member

Kate Mohr – Chair Miffany Blythe – Deputy Chair Trent Blacket Anna a’Beckett Genevieve Brammall Elizabeth Briskin Clark Kirby Tanya Hamersfeld Peter Mastos Vas Katos Vicky Alexiou Dahlia Sable Tamara Lasky (commenced Aug 2020) US Development Board

Prof Kathryn North AC

Sarah Murdoch #

Patrick Houlihan ^

Paul Rayner

Director VCGS Board member

US Development Board Chair Global Ambassador

Audit, Finance and Risk Committee Deputy Chair Marketing Council Chair

Audit, Finance and Risk Committee Chair Investment Committee member

Sarah Murdoch – Chair Suzi Carp Kate Mohr Miffany Blythe Katie O’Reilly Nick Stone Sarah Harden Trent Blacket Tristen Langley Steve Hasker David Calvert-Jones Lauren Thurin VCGS Board

Prof John Prins

Dominic Stevens

Steven Casper

Dr Brandon Carp

Translation and Commercialisation Committee

VCGS Board Chair Translation and Commercialisation Committee

Dr Brandon Carp – Chair David Gillespie Helen Kurincic Prof Kathryn North AC Prof Andrew Sinclair Dr John Zelcer

Hon Rob Knowles AO

Andrew Wilson

Prof Shitij Kapur

56

Murdoch Children’s Research Institute

† Stepped down as Deputy Chairman Nov 2020

^ Chairman from Nov 2020

Translation and Commercialisation Committee Geraldine Farrell – Chair Dr Damien Bruno Dr Brandon Carp Steven Casper (commenced Sept 2020) Andrew Cox Ms Melinda Griffith (commenced Sept 2020) David Link Steve Pask Jenny Petering Investment Committee Simon Rothery – Chair Adrian Redlich – Deputy Chair Ariane Barker Paul Rayner Council of Ambassadors Dame Quentin Bryce AD CVO Jeanne Pratt AC Jean Miller Paula Fox AO Carla Zampatti AC* Janet Calvert-Jones AO Frances Underwood Lady Primrose Potter AC

* MCRI notes with sadness the passing of Carla Zampatti in April 2021

Enjoy that video at mcri.edu.au/annualreport2019 * Stepped down from the Board Nov 2020

Paul Rayner – Chair Patrick Houlihan – Deputy Stuart Boxer Elise Elliot David Gillespie

Marketing Council Patrick Houlihan – Chair Ian Rowden Joanne Smith Martin Ward

Find out more At the 2019 Dame Elisabeth Luncheon, we were delighted to share a video honouring our inspirational co-founder Dame Elisabeth Murdoch AC DBE. John Stanway

Audit, Finance and Risk Committee

# Co-Chair from Nov 2020 Annual Report 2020

Report 2020 SUSTAINABILITY &Annual ENGAGEMENT

57

The year at a glance MCRI financials

$83.20m

Research and government grants revenue

$26.83m

Donations, fundraising and bequests

$51.52m

Contract research, clinical trials and others

$31.95m VCGS

$193.50m Total revenue

Statement of profit or loss and other comprehensive income for the year ended December 31, 2020 Consolidated 2020

2019

2020

2019

$’000

$’000

$’000

$’000

162,424

147,333

130,665

116,130

(7,350)

(7,060)

(6,630)

(6,040)

Other expenses for research and clinical activities

(180,389)

(161,461)

(149,174)

(130,985)

Surplus/(deficit) from research and clinical activities

(25,315)

(21,188)

(25,139)

(20,895)

Donation, estates, bequest and fundraising income

26,834

17,736

26,830

17,736

Fundraising expense

(1,710)

(1,548)

(1,710)

(1,548)

25,124

16,188

25,120

16,188

4,244

7,285

4,052

6,912

Operating surplus for the period

4,053

2,285

4,033

2,205

Total comprehensive surplus for the period

4,053

2,285

4,033

2,205

Revenue from research and clinical activities Depreciation and amortisation

Net surplus obtained from fundraising activities

Finance income

58

Murdoch Children’s Research Institute

Company

Annual Report 2020 Annual Report 2020 FINANCIALS

59

Statement of cash flows For the year ended December 31, 2020 Consolidated

Company

2020

2019

2020

2019

$’000

$’000

$’000

$’000

30,000

25,629

-

-

130,867

106,726

123,376

98,382

Donations received

26,834

17,736

26,831

17,736

JobKeeper subsidy

17,709

-

14,617

-

666

1,087

571

903

2020

2019

2020

2019

25,921

31,722

34,295

35,069

$’000

$’000

$’000

$’000

-

-

778

(94)

(175,430)

(161,828)

(145,961)

(132,618)

56,567

21,072

54,507

19,378

Cash and cash equivalents

63,279

29,318

61,471

28,046

Trade receivables and other assets

16,019

14,475

12,552

10,364

Other investments

52,444

55,350

44,536

47,249

131,742

99,143

118,559

85,659

Trade receivables and other assets

53,659

55,000

53,659

55,000

Other investments

69,995

49,046

64,667

43,800

Property, plant and equipment

14,036

14,985

11,720

13,714

179

7

179

7

Total Non-Current Assets

137,869

119,038

130,225

112,521

Total Assets

269,611

218,181

248,784

198,180

Trade and other payables

98,190

54,722

93,411

49,708

Employee benefits

21,565

18,082

14,983

12,227

119,755

72,804

108,394

61,935

3

3

-

-

Employee benefits

3,418

3,015

2,669

2,580

Total Non-Current Liabilities

3,421

3,018

2,669

2,580

Total Liabilities

123,176

75,822

111,063

64,515

Net Assets

146,435

142,359

137,721

133,665

146,395

142,319

137,721

133,665

40

40

-

-

146,435

142,359

137,721

133,665

Cash flows from operating activities Patient fees received Government and other grants received

Interest received Other receipts Advances to (from) related parties Cash paid to suppliers and employees Net cash provided from (used in) operating activities

Cash flows from investing activities Investment income received

3,578

6,198

3,481

6,009

(5,235)

(8,881)

(3,471)

(7,937)

(18,020)

(12,470)

(18,132)

(10,790)

(19,677)

(15,153)

(18,122)

(12,718)

Net increase/(decrease) in cash and cash equivalents

36,890

5,919

36,385

6,660

Cash equivalents at January 1

29,318

21,482

28,046

19,464

(2,928)

1,917

(2,960)

1,922

63,280

29,318

61,471

28,046

Acquisition of property, plant and equipment Acquisition of investments Net cash provided from (used in) investing activities

Effect of foreign exchange on opening cash balances Cash equivalents at December 31

Statement of financial position As at December 31, 2020 Consolidated

Company

Current Assets

Total Current Assets Non-Current Assets

Intangibles

Current Liabilities

Total Current Liabilities Non-Current Liabilities Trade and other payables

Members’ Funds Accumulated funds Other reserve Total Members’ Funds

60

Murdoch Children’s Research Institute

Annual Report 2020 Annual Report 2020 FINANCIALS

61

Our manifesto We believe that for every question there’s an answer.

For every child’s illness there must be a cure.

For every obstacle there must be a way around.

What inspires us is asking the big questions – Why is it so? Why does it happen? How can we fix it?

What excites us is tackling the big issues affecting children’s health.

Children are at our heart, in our blood, and in our bones.

We believe every child deserves a healthy start to life.

And a happy and prosperous community needs healthy children.

We believe in the power of curiosity, cleverness and cutting-edge research.

We are excited by discovery and new knowledge to make a difference.

The future is purchased by the present, we can shape the future, we can change the world.

So every child can have a childhood.

So every child can grow to reach their full potential.

Children are at the heart of everything we do.

Scan page 62 to see our manifesto brought to life

62

Murdoch Children’s Research Institute

Annual AnnualReport Report2020 2020

63

OUR PURPOSE

We want all children to have the opportunity to live a healthy and fulfilled life.

Murdoch Children’s Research Institute Royal Children’s Hospital 50 Flemington Road, Parkville Victoria 3052 Australia +61 (3) 8341 6200 Free Call 1300 766 439

mcri@mcri.edu.au mcri.edu.au donate.mcri.edu.au ABN 21 006 566 972