Annual Report 2021 by Murdoch Children's Research Institute

celebrating

Annual Report 2021

Stopping Strep A Strep A disproportionately affects Aboriginal and Torres Strait Islander people. However, our researchers and partners are currently working to develop a world-first Strep A vaccine to prevent this bacterial infection in children. See our Strep A story on page 17.

2021 Stories 1

Research Impact A brief history of MCRI

Leading the way on COVID-19

Investing in stem cell medicine

The heart of the matter

Newborn vaccine set to save lives

Helping Victorian families

Cracking the genetic code

Advances in infectious disease

Reducing children’s allergies

Mental health program expands

Helping vulnerable infants thrive

Cancer under the microscope

Research Excellence GenV lifts off

Prizes and awards

Foundations of success

Measures of success

Our People, Our Values Emerging leaders

Staff awards

Taking mentoring seriously

Innovating for impact

Behind our brilliant minds

Sustainability and Engagement

The Murdoch Children’s Research Institute acknowledges the Traditional Custodians of the land upon which we are located. We pay our respects to their Elders, past, present and emerging. 2 2

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

Our donors

An enduring legacy

35 years of philanthropy

MCRI financials

2021 Boards and Committees

Annual Report 2021

Celebrating 35 years Annual Report 2019

3 3

Director’s message

Our purpose

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

Murdoch Children’s Research Institute

In 2021, Murdoch Children’s Research Institute celebrated 35 years of helping infants, children, adolescents and their families live happier, healthier lives. It gives me great pleasure to introduce this year’s Annual Report, which offers a snapshot of the amazing productivity of the Institute’s staff during a second pandemic year.

From genomics, we have expanded our scope into other scientific frontiers, integrating genomics with stem cell medicine and clinical research, the power of big data, population-wide research and global health to solve the biggest health issues affecting children and families in a changing world.

Of course, in mentioning COVID, we come to the world’s most pressing health crisis of the past century. The pandemic has amplified the importance and the benefits of health and medical research. And at MCRI we have certainly played our part over the past two years.

One of our great strengths is our co-location with The Royal Children’s Hospital and the University of Melbourne’s Department of Paediatrics. It took some dedicated people a lot of work and 10 years to bring the ‘village’ of the Melbourne Children’s Campus into being, but the final objective was bang-on. Together, we are truly greater than the sum of our parts.

In 1986, two visionaries, Professor David Danks and Dame Elisabeth Murdoch, imagined a better future for children.

Society’s major issues now are the ‘modern epidemics’ of allergy, obesity, diabetes, depression, anxiety, cardiovascular disease and cancer – all with their origins in early life. Now, one in 10 children has food allergies and one in three is overweight. One in five disadvantaged children is intellectually impaired at school entry – perpetuating the cycle of disadvantage. The dramatic rise in these problems is an ‘emergency in slow motion’. To address this challenge, our pioneering researchers have established population-based community cohorts and are making great strides in prevention and early intervention.

Our COVID team continues to make huge inroads into understanding what makes children’s immune systems so good at dealing with the coronavirus. Our stem cell team is modelling the disease in miniature organs and screening potential treatments. And our vaccine uptake group has moved mountains in convincing people that the vaccines are safe and effective for them and their children.

The catalyst was the 5 per cent of children born with a genetic disease – a major cause of disability and death. The pair’s bold vision was that genes could hold the answer to health and that genetics would one day become part of standard patient care. Along with MCRI, Professor Danks founded the Victorian Clinical Genetics Services (VCGS), a pioneering not-for-profit subsidiary of the Institute, which provides clinical care and diagnostic testing for thousands of children and families who are at risk of a genetic condition. Thirty-five years later, the Institute can point to its massive contribution to genomic medicine. We make rapid diagnoses, identify safer treatments and change health outcomes for children with previously undiagnosable and untreatable diseases. For our sickest children in intensive care, the Institute can now provide a genomic diagnosis within three days.

We now host more than 30 different population-based LifeCourse studies involving more than 50,000 families, 1 million data points and 80,000 biospecimens. But this is just the beginning of our goal to take prevention and early intervention into the community. GenV is the latest and greatest of our population cohort studies. After COVID-19 interrupted the start of GenV, recruitment ramped up in 2021 and we are now busy recruiting in every single birthing hospital in Victoria. GenV babies and their families are taking part in Australia’s biggest study – to give us a complete picture of the health and wellbeing of a whole generation.

We think of research having an impact in 20 to 30 years’ time, but increasingly our research is making an immediate difference, thanks in no small part to agile philanthropic and commercial supporters. We thank all the donors who turn our vital research into health solutions at the individual, family, community and society level. We also thank the state and federal governments for their ongoing support of the Institute’s researchers, on behalf of all the families who ultimately benefit from this support. Over the past 35 years, MCRI has grown the depth and breadth of its research to become the leading child health research institute in Australia, and ranked in the top three internationally. Our next 35 years is focused on harnessing the might of our expertise, capability and commitment to accelerate precision child healthcare – to rapidly improve the prediction, prevention, diagnosis and treatment of rare and complex diseases and common conditions for children not just in Australia, but all over the world. Professor Kathryn North AC Director Annual Report 2021

Celebrating 35 years

Chairman’s message On behalf of the Board, I am pleased to share with you the 2021 Annual Report, highlighting the remarkable achievements of the Murdoch Children’s Research Institute over this past year. It was a year in which we marked the 35th anniversary of the founding of the Institute by Dame Elisabeth Murdoch and Professor David Danks with a handful of researchers and a vision to improve childhood health through world-class genetics research and clinical services. Over the course of three decades, that vision has evolved with MCRI now the leading child health research institute in Australia and ranked among the top three such enterprises worldwide. As we build upon the amazing legacy of Dame Elisabeth and Professor Danks, we continue to stay true to why we exist in the first place. Simply, we are ‘For children’; children are at the heart of everything we do. Today, we have more than 1,800 brilliant scientists united and driven by that core purpose. Their ground-breaking research is helping to ensure that more children have the opportunity to live healthy and fulfilled lives, not only in Australia but throughout the world. My own connection with the Institute started in 2014 when I attended the Biennale and soon after joined the Board. For me, with a background and early career in science, and my ongoing involvement through my role as a member of the Federal Government’s Industry, Innovation and Science Australia advisory board, the Institute consistently shows the power and ability of science to transform lives for the better.

Murdoch Children’s Research Institute

Co-Chair’s report In 2021, MCRI continued to change the face of children’s health through its six Strategic Initiatives: Genomics; Stem Cell Medicine; GenV/Population Health; Global Health; Data Science; and Clinical Translation. Our researchers come together from across the Institute to solve the big health issues facing our children. We have multidisciplinary teams focused on areas including rare diseases, allergies, adolescent health, Aboriginal health, neurodevelopment and cancer. Every day our researchers don’t just imagine a better future for children, they turn imagination into impact, empowered by research and driven by a commitment to excellence. Among the many outstanding stories you will find in this report, is how the Victorian Government has expanded our mental health program in primary schools, how our Australia-wide infant feeding guidelines are reducing allergies, and how Professor Melissa Little leveraged sustained Australian philanthropy into the world’s biggest philanthropically backed international stem cell medicine research and translation effort – reNEW. I would like to thank our philanthropic and commercial supporters who, along with the ongoing support of federal and state governments, help make this all possible. I would also like to recognise and thank our Campus partners from The Royal Children’s Hospital, the Department of Paediatrics at the University of Melbourne, and The Royal Children’s Hospital Foundation. Our co-location and collaborative partnership ensure that our researchers and clinicians are working side-by-side to rapidly translate our research into clinical practice. Also, I wish to acknowledge the outstanding leadership of our Director, Professor Kathryn North AC, whose dedication and vision are so fundamental to the success of the Institute.

Over the past two years, she has steered the team through the unpredictability of the COVID-19 pandemic with care and compassion, while ensuring MCRI has been at the forefront of research and solutions for this immediate health crisis. At the same time, the team has not ‘missed a beat’ in progressing our ambitions to solve the biggest health issues facing children and families throughout the world. Thank you to my fellow directors and board members for your commitment and dedication to the Institute, along with your ongoing support. In particular, I acknowledge the tremendous work of Co-Chair and Global Ambassador Sarah Murdoch who has helped to place the Institute in a global context in terms of leadership in child health research. Over more than two decades, she has built the profile of Murdoch Children’s with key stakeholders, and developed strategic relationships with her insightful appreciation for the role philanthropy can play in the Institute’s success. Her personal drive and commitment to MCRI and Dame Elisabeth’s vision are inspiring and so core to all that she achieves for the Institute. In my first year as Chairman, I followed in the footsteps of the remarkable Suzi Carp AO whose amazing contribution to the Institute over more than two decades continues with her joining Sarah as a Global Ambassador. I thank Suzi for her valuable counsel and her ongoing commitment to helping Murdoch Children’s pursue its ambitions ‘For children’. Finally, thank you to the entire MCRI team. Your relentless curiosity, brilliant minds and drive to improve the lives of children and families is truly inspiring. I continue to be amazed and delighted by the potential for new discovery, all guided by putting children at the heart of everything we do.

This past year, I have often thought how incredibly proud Dame Elisabeth would be if she could see the Murdoch Children’s Research Institute now. As we celebrate 35 years of the Institute’s impact, it is breathtaking to contemplate the breadth and depth of MCRI’s work and its potential to change the lives of children for generations to come. In my role as Co-Chair and Global Ambassador, I am privileged to be able to place Murdoch Children’s in the context of the world’s other leading child health research institutes and clinical centres. In fact, because we are one of the top three paediatric research institutes globally, our shared expertise and open collaboration is more important than ever. And we truly are global. This year, our new partnership with the Novo Nordisk Foundation in Denmark sees a recordbreaking investment in stem cell medicine with the reNEW stem cell research program. Our new collaborative Centre for Population Genomics is building a massive database of genetic information that better reflects the extraordinary diversity of the Asia-Pacific region, ensuring that all of our communities can benefit from advances in genomic medicine. We have also partnered with the other top three paediatric research institutes globally to create the International Precision Child Health Partnership to tackle rare disease in children. Our first project is to find answers to childhood epilepsy.

These are a few examples of how we are working to positively impact the next generation around the world. As we reach out internationally for support, I am pleased to announce that joining me as a Global Ambassador is our former Chair, Suzi Carp AO. Suzi’s leadership in health and medical research and dedicated service to children and young people, and to the greater community, was recognised with her appointment as an Officer in the General Division of the Order of Australia. Congratulations, Suzi!

“By the time the GenV ‘COVID generation’ babies turn 35, we will have the world’s largest and deepest resource to track their health and development . . .” Suzi has continued her outstanding contribution to the Institute’s work by bringing together supporters to establish a fund to endow the Suzi Carp Postdoctoral Scholarship. Murdoch Children’s future impact will be driven by today’s students and early-career researchers, and we know you share our belief that it is crucial to support their commitment, passion and talent to address current and emerging child health challenges. With visionary philanthropic support from organisations such as the Paul Ramsay Foundation, together with vital contributions from the Victorian Government, the world’s largest longitudinal study on children, GenV has already recruited over 10,000 babies and their families.

Similarly, it was the Stafford Fox Medical Research Foundation’s decade-long support that was leveraged into the world’s largest ever philanthropic donation for the reNEW stem cell research program. Furthermore, the Bill & Melinda Gates Foundation’s support for our rotavirus vaccine work led to their joining Lachlan and me in funding the BCG vaccine for COVID-19 trial – the results of which I am eager to see. I am excited by the future possibilities of this impressive Campus. By the time the GenV ‘COVID generation’ babies turn 35, we will have the world’s largest and deepest resource to track their health and development, enabling precision, evidencebased interventions for the next generation. In the nearer term, I am also looking forward to a return to the kind of face-to-face events – here in Australia and in the United States – that keep us connected. Finally, I would like to thank Patrick Houlihan for his visionary leadership as Chairman. The Board, our executive team and I have truly benefited from his expertise. And, as the Institute goes from strength to strength it is Pat who consistently reminds us we are here for the children. Thank you to every one of the students, researchers and staff members – past and present – of this wonderful Institute for so relentlessly pursuing our founders’ dream over the past 35 years. Dame E would be immensely proud of you all, and I hope everyone enjoys reading this year’s report as much as I did. Sarah Murdoch Co-Chair Global Ambassador

Patrick Houlihan Chairman

Annual Report 2021

Celebrating 35 years

Helping premmie babies Twins Clementine (centre) and Felix (right) were born extremely prematurely at just 27 weeks but are now doing well thanks to participating in the Vibes Study, which aims to improve the long-term quality of life for children who are born preterm.

Research Impact

Translating research into real-life outcomes that make a tangible difference to children’s lives is a cornerstone of our work. Our people are working to discover and trial new interventions and translate our research into treatments and tools that improve the health of children.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

A brief history of MCRI

1936

1960

Melbourne Hospital for Sick Children is founded.

The hospital establishes a Medical Research Committee.

The Royal Children’s Hospital Foundation is founded under Committee of Management President, Dame Elisabeth Murdoch.

1995

1986

1962

Professor Bob Williamson AO appointed Director of the Institute after Professor David Danks retires.

The Victorian Clinical Genetics Services (VCGS) is established, an amalgamation of clinical genetics services and genetic pathology laboratories. VCGS is a wholly-owned subsidiary of Murdoch Children’s Research Institute.

The Murdoch Institute for Research in Birth Defects is co-founded by Professor David Danks, with support from Dame Elisabeth Murdoch and her family, Sir Jack Brockhoff, the Scobie and Clare Mackinnon Trust, the Miller family and others. It quickly becomes a world-class centre of genetics research and clinical genetics services.

The first Genetics Research unit is established by Dr David Danks.

2000

2003

2004

The Murdoch Institute and the RCH Research Institute merge to form the Murdoch Children’s Research Institute with Professor Bob Williamson as Director.

Sarah Murdoch becomes an ambassador for Murdoch Children’s Research Institute and later joins its Board of Directors.

Professor David Danks passes away, aged 72.

Professor Terry Dwyer AO appointed Director, a position he holds until 2012.

2011

2010

2006

Rupert Murdoch contributes a $10 million grant on behalf of his family to the Institute.

Murdoch Children’s Research Institute moves into its new world-class facility, co-located with The Royal Children’s Hospital. The facility is officially opened by Her Majesty Queen Elizabeth the following year.

The first Bill & Melinda Gates Foundation grant is awarded to the Institute for its global study into childhood pneumonia. This leads to improvements in the development of new vaccines.

The Institute plays a major role in the development of Australia’ s leading parenting resource, www.raisingchildren.net.au, establishing an ongoing partnership for translating research into practical resources for parents and carers.

2012 Murdoch Children’s patron Dame Elisabeth Murdoch passes away on December 5.

1870

Murdoch Children’s Research Institute

2013

2015

Professor Kathryn North joins the Institute as its new Director.

Professor Ruth Bishop awarded the CSL Florey Medal for her lifetime’s work on rotavirus.

The Melbourne Children’s Trials Centre is established as a national centre for paediatric clinical research ranging from trials of novel therapeutic agents to large public health prevention trials.

2016

2015

VCGS celebrates 30 successful years of newborn screening in Victoria.

The Institute, working with Fiji Ministry of Health and the Australian Government, successfully trials the introduction of three new vaccines to tackle pneumonia, diarrhoea and cervical cancer, reducing these lifethreatening diseases and hospital admissions in Fiji.

Murdoch Children’s Research Institute celebrates its 30th anniversary.

The Australian Genomics Health Alliance (AGHA), a national network of 47 partner organisations, led by Professor Kathryn North and Professor Andrew Sinclair, is established.

2020

2021

Sarah Murdoch appointed as Global Ambassador.

Murdoch Children’s Research Institute announced as major partner in a new state-of-the-art international stem cell research centre to provide new therapeutic options for patients with incurable diseases.

We look forward to what the future holds

Learn how Dame Elisabeth’s generosity has helped MCRI improve child health Annual Report 2021

Celebrating 35 years

Leading the way on COVID-19

Child-friendly COVID-19 test Children globally could soon be faced with a less-invasive COVID-19 test thanks to our partnership with The Royal Children’s Hospital (RCH). The child-friendly Rhinoswab Junior™ resembles a curly moustache and sits at the base of the nose to collect particles for testing. It is being trialled at the RCH Respiratory Infection Clinic. Rhinoswab Junior™ was developed by Melbourne-based Rhinomed Ltd. The test’s development follows research that found 74 per cent of parents were concerned the COVID-19 test might be stressful, painful or uncomfortable for children. Dr Shidan Tosif said the ability for children to insert the device themselves, its comfort and novelty all helped improve the experience. He said this would be needed as children and adults required multiple COVID-19 tests as new variants emerged.

Murdoch Children’s Research Institute

As the world grappled with the impact of COVID-19 on children and their families, Murdoch Children’s Research Institute led the way in vaccine advocacy, research and transmission data.

Vaccine FAQ

Long-term health effects

Murdoch Children’s Melbourne Vaccine Education Centre (MVEC) provided easy-todigest COVID-19 information and answers to frequently asked questions for families.

Associate Professor Margie Danchin and Dr Tosif contributed to an international study on the transmission and impact of COVID-19 in children.

The FAQ cover all aspects of vaccine administration, effectiveness and safety, including reassuring families about mild adverse reactions.

The Institute’s internationally renowned experts advised governments and those working on the frontline, playing a key role in getting Victorian students back to school through research, reports and public education.

MVEC’s website also links to research, ATAGI advice, vaccine services, safety information and reports.

Published in The Journal of Paediatric Child Health, the paper found COVID’s effects on mental health, family violence, medical attention and face-to-face school learning were likely to continue for the long term.

Among other things, Murdoch Children’s: • Published four ground-breaking COVID-19 research briefs to document emerging knowledge and inform government, media and the public. • Established the ‘COVID in schools’ weekly reports to document direct effects of the pandemic on children in school settings in Australia and around the world. • Prepared a report for the Victorian Government on COVID-19 in early childhood education and care (ECEC) and schools, analysing outbreak data and showing they were safe to open. • Saw Professor Sharon Goldfeld and Professor Andrew Steer address two Senate Select Committee inquiries into COVID-19. • Saw Associate Professor Nigel Crawford appointed Chair of the Australian Technical Advisory Group on Immunisation (ATAGI).

It provides information for at-risk groups such as children, and adults who are immune-suppressed or pregnant, as well as Aboriginal and Torres Strait Islander people. COVID-19 and families Associate Professor Crawford is leading Victorian Government-funded research on the effects of COVID-19 and vaccines on children. The team is investigating transmission, ‘long COVID’, child and adolescent vaccine safety, a swab test for children, and how special-risk groups respond to the vaccines. Further, paediatrician and researcher Dr Shidan Tosif’s household transmission and immunity study aims to better understand how infection occurs in children and their immune response over time.

Existing inequities would also be amplified, creating new disadvantage that disproportionately affected children from vulnerable families. The role of stem cells Research into how COVID-19 affects stem cell-derived organ models received additional Victorian Government funding to incorporate emerging strains, long-term symptoms and potential complications of the virus in pregnancy. The project, led by Murdoch Children’s, uses human stem cells to better understand the effects of the virus on the lungs, heart, kidneys, brain, immune system and blood vessels. An Australian first, the $2.3 million study aims to support the development of additional targeted treatments.

Associate Professor Crawford and Dr Tosif are also investigating how mRNA vaccines impact children’s immune systems. The Peter Doherty Institute for Infection and Immunity, The Royal Children’s Hospital, SAEFVIC (Surveillance of Adverse Events Following Vaccination in the Community) and medical technology company Rhinomed Pty Ltd are key partners on these projects.

Scan to watch Sharon talk about COVID and children on ABC’s 7.30 Annual Report 2021

Celebrating 35 years

A monumental investment into stem cell medicine The Murdoch Children’s Research Institute is one of three world-leading research institutes that have been backed by a major international foundation to deliver innovative stem cell-driven therapies to transform the lives of people with incurable disease. The Novo Nordisk Foundation (NNF) – one of the largest foundations in the world – has made an unprecedented donation of €300 million to establish a collaborative translational research centre for stem cell medicine. Led by newly appointed CEO Professor Melissa Little from Murdoch Children’s, the Novo Nordisk Foundation Center for Stem Cell Medicine (dubbed reNEW) was established as a tripartite consortium across three international sites: Murdoch Children’s in Australia, the University of Copenhagen and the Netherlands’ Leiden University Medical Center. Together, these three world-leading institutes will combine their strengths in biomedical research excellence to develop innovative stem cell therapies for incurable diseases. Inspiring the concept of reNEW was the pioneering vision of Professor Little, MCRI’s Chief Scientist. Professor Little is internationally renowned for her expertise in kidney development and regeneration using pluripotent stem cells and is President of the International Society for Stem Cell Research. Despite promising advances in medical technologies and healthcare, populations around the world are seeing a concerning rise in chronic illnesses, including chronic kidney disease, cancer, diabetes and neurodegenerative diseases.

Murdoch Children’s Research Institute

The rising incidence of incurable disease is exposing major gaps in global healthcare systems. An ongoing challenge for the biomedical research sector is the existing high barriers in translating discovery-based science to clinical outcomes. The pipeline to bring a therapy or intervention to the clinic is slow, risky and expensive. More than 95 per cent of products fail to reach the market. The Novo Nordisk Foundation wants to accelerate this bench-to-clinic pipeline to help more people living with chronic disease, committing €30 million every year for the next decade to establish the reNEW Stem Cell Medicine Center. The pledge represents the biggest donation made by the NNF and its first investment in this type of consortium, underpinning a dedicated effort to overcome the commercialisation barrier and achieve targeted research outcomes from stem cell medicine. “Stem cell medicine truly promises to be a game changer when it comes to addressing some of the major health challenges facing the world today … whether in the form of new medical technology or new forms of treatment for the benefit of patients,” said the CEO of the Novo Nordisk Foundation, Mads Kragsgaard Thomsen. Pioneers in this branch of medicine are in the process of using stem cells to model human diseases, regenerate tissues and create socalled “designer cells” that can improve our ability to diagnose, treat and even prevent human disease. Human stem cell-derived models offer an accurate replica of human tissues and organs for non-invasively studying disease mechanisms. They offer the opportunity for preclinical drug testing with an individual’s own cells, ushering in the era of personalised medicine.

“Such transformative stem cell medicines offer patients hope for a variety of incurable diseases and represent a genuine medical frontier as well as offering an exciting opportunity for economic growth,” said Professor Enzo Porrello, reNEW Melbourne Node Director. reNEW will build upon the capacity of the three institutes in disease modelling, gene editing and regenerative medicine to speed the innovation of novel stem-cell therapies for both adult and paediatric diseases. Each of the centres or “nodes” of the international consortium will offer research expertise, facilities and technologies to provide a mutually reinforcing, collaborative network. The researchers in the reNEW initiative include experienced leaders in cell and gene therapy, as well as a new generation of emerging scientific superstars to build critical mass for delivering targeted research outcomes. Professor Little has more than 30 years of laboratory and leadership experience. Her belief is that reNEW will not only build capacity for targeted research outcomes but also attract international investment. “reNEW will enable outcomes across the breadth of stem cell medicine – new drugs based on stem cell disease models, cell and tissue therapies, and also gene editing – and take research outcomes all the way to clinic,” she said. “This is a unique model and fantastic opportunity that will put Australia on the map for regenerative medicine.” Mia Cowley, 16, (pictured right with her mum, Lyndal) was born with congenital heart disease. She essentially lives with half a heart. Mia has undergone four open heart surgeries since she was just five weeks old. “Even a tiny step in the research could one day make a huge difference,” Mia said.

Watch our introduction to the Melbourne node of reNEW Annual Report 2021

Celebrating 35 years

The heart of the matter

Saving Eden Tania O’Meara’s daughter, Eden, was just 11 months old when she almost lost her leg to a flesh-eating bacterial infection caused by Strep A. “We put our daughter to bed with what seemed like a cold but the next morning she woke up with a fever and was very pale, limp and dehydrated,” said Tania. “We took her to the hospital but the doctor couldn’t get the IV drip in because Eden was so dehydrated and they were forced to drill it into her leg. They also noticed that the skin on her leg looked like it was turning a different colour.” Eden required surgery on her right calf to remove the dead flesh. “We were told that she could die and we braced ourselves so it was a miracle that they were able to save her leg and even her calf muscle.” Eden, now 3, needed three more surgeries and has since made a full recovery. Tania said she was relieved that a vaccine for Strep A may not be too far away. “I don’t want any other family to experience what we went through,” she said. “It is an absolute tragedy that this bacteria is cutting so many lives short.”

Besides high blood pressure, smoking and obesity, some causes of heart disease are not so well known, such as complications from infection. To address such potentially deadly conditions, experts from the Murdoch Children’s Research Institute are helping lead a global effort to prevent and treat conditions that can result in heart issues. They include Strep A bacteria, which causes throat and skin infections, often in children. Untreated, Strep A can lead to serious heart or kidney conditions, toxic shock syndrome or flesh-eating disease. Indeed, each year, Strep A causes an estimated 616 million sore throats and 500,000 deaths worldwide. In Australia, Strep A disproportionately affects Aboriginal and Torres Strait Islander people. However, the Australian Strep A Vaccine Initiative (ASAVI), a Murdoch Children’s and Telethon Kids Institute collaboration, is working steadily to develop a world-first Strep A vaccine, with the group a step closer to conducting human trials for its vaccine candidates. Funded by the Australian Government’s Medical Research Future Fund (MRFF) and Open Philanthropy, ASAVI works with vaccine developers, as well as commercial, academic and other global partners, and affected communities. Murdoch Children’s-led research published in The Lancet Microbe paved the way for testing Strep A vaccines on people. The team developed a human challenge model, which infects healthy adult volunteers with a pathogen in a controlled environment. The model has been deemed safe to trial new Strep A candidate vaccines.

Scan to see how doctors saved Eden from a lifethreatening Strep A infection 16

Murdoch Children’s Research Institute

The trial, which will be conducted in Melbourne, will involve about 50 participants who will receive a candidate vaccine or placebo, while applying the Strep A challenge strain.

Infection and Immunity Theme Director Professor Andrew Steer hopes it will lead to bigger field trials. “A vaccine for Strep A could save hundreds of thousands of lives every year and prevent millions of infections that send children and adults to the hospital or doctor,” he said. Meanwhile, using their human challenge model, Murdoch Children’s researchers led a study published in Nature Communications that detailed the body’s immune response to Strep A, another important step towards a vaccine.

The World Health Organization has recognised the leadership of Murdoch Children’s by nominating its Tropical Diseases Research Group as its first WHO Collaborating Centre for Scabies Control, to be led by Professor Steer. Murdoch Children’s also houses the World Scabies Program, established in 2019 with a $10 million grant from the Macquarie Group.

After infecting healthy adult volunteers with Strep A, researchers identified an immune signature associated with acute pharyngitis (sore throat) that would help in developing a Strep A vaccine. Antibiotics and heart disease Murdoch Children’s-led research also found regular, affordable antibiotic treatment significantly reduced the risk of underlying rheumatic heart disease progression in children and adolescents. The Ugandan project found early heart screening critical in preventing serious rheumatic heart disease progression and death in young children. Until this New England Journal of Medicine paper, it was unknown if antibiotics could prevent latent rheumatic heart disease progression. Controlling scabies Scabies can cause terrible itching when a parasite lays eggs under the skin. Around 455 million cases occur globally each year and infections via open sores from scratching can lead to complications including heart disease. MCRI research with the Kirby Institute and the Fiji and Solomon Islands health ministries since 2015 has confirmed that the antiparasitic drug ivermectin can reduce scabies by more than 90 per cent.

Vaccine trial scholarship Efforts to reduce heart disease caused by Strep A have been boosted by a scholarship for Murdoch Children’s PhD student Dr Yara-Natalie Abo, who is part of the team working on a vaccine for the common bacterial infection. The National Heart Foundation awarded Dr Abo a $122,100 cardiovascular postgraduate research scholarship and its $30,000 Excellence Award for topranking project. Dr Abo will test Strep A vaccines in a randomised human trial, giving the vaccine or a placebo to healthy adult participants in a controlled environment.

Annual Report 2021

Celebrating 35 years

Newborn vaccine set to save lives The award-winning team behind an affordable and effective gastro vaccine is ensuring it will save as many newborn lives as possible globally. Long before COVID-19 vaccines became the focus of many of our conversations, Melbourne scientists laid the groundwork to protect infants against a common and potentially deadly form of dehydrating diarrhoea. After more than 50 years of hard work, the team has reached another critical milestone in vaccinating babies in emerging countries – and globally – against rotavirus gastroenteritis. It all began in 1973, when Professor Ruth Bishop, Professor Ian Holmes and their Melbourne team discovered rotavirus, leading to critical Murdoch Children’s research to better understand it. The discovery has so far inspired four oral rotavirus vaccines administered to babies from six weeks of age, which have saved countless lives in more than 100 countries, including Australia. Global Vaccine Action Plan data shows that from 2011-2020, up to 900,000 deaths were prevented. Murdoch Children’s Research Institute Enteric Diseases Group and RV3 Rotavirus Vaccine Program leader, University of Melbourne’s Professor Julie Bines and her team are now collaborating internationally to ensure that even more children are protected. Their latest research in Malawi has helped pave the way for what could be a highly effective vaccine for many thousands of babies across Africa and low-middle income countries globally.

Murdoch Children’s Research Institute

Inspiring life-saving vaccines

New hope for African newborns

After discovering a strain of rotavirus in the late 1980s that didn’t make babies sick, Murdoch Children’s researchers used it to develop the oral RV3-BB vaccine, which can be administered at birth.

The latest research shows that RV3-BB is safe and produces a robust immune response in African infants.

This has many benefits. Birth is a good time for health services to access mother and baby, and research has shown that the newborn’s microbiome is immature and may present less of a barrier. “Newborn babies haven’t developed their gut microbiome,” Professor Bines explained. “There seems to be not as much of a challenge to the vaccine at that time.” Current rotavirus vaccines can only be given at six weeks, but providing protection from birth could save many more lives. Trials show promise Clinical trials for RV3-BB, which was named after Professor Bishop and colleague Professor Graeme Barnes, have been completed in Australia, New Zealand, Indonesia and now Malawi. After years of rigorous research in collaboration with Universitas Gadjah Mada and Indonesian Government manufacturer BioFarma, which has developed a halal vaccine to reflect local preferences, there are now plans to introduce the vaccine into the Indonesian National Immunisation Program in 2023. But a major challenge that existing rotavirus vaccines have faced is uneven effectiveness in some parts of the world. They appear to be less effective in South Asian and African populations than in European populations, possibly due to variations in blood types. Professor Bines’ team has previously shown that the RV3-BB vaccine produces an immune response regardless of blood type so it may have an advantage in these high-risk populations.

Just published in Lancet Infectious Diseases, the phase 2 clinical trial conducted with the University of Liverpool found a reduced dose of RV3-BB produced the same immune response as a higher dose. Professor Bines co-led the study with University of Liverpool Medical Microbiology Professor Nigel Cunliffe. Their teams assessed the safety and immune reaction to three different amounts of the RV3-BB vaccine in 711 Malawian infants at birth or in their first weeks of life (trial participants, pictured left). The mid-dose performed as well as the higher dose, providing an opportunity to reduce the cost of manufacturing the vaccine, making it more affordable. “It’s safe for newborn babies and it produces an immune response in babies in Africa,” Professor Bines said. “It should be a costeffective vaccine.” Helping those who need it most Globally, rotaviruses still kill around 215,000 children aged under five each year, down from about 800,000 pre-vaccines. Many hospital stays have also been prevented. “The vaccines have made a major difference,” Professor Bines said, adding that improved hygiene and other advances had also played a role. The World Health Organization recommends all children receive a rotavirus vaccine, but millions still miss out. Murdoch Children’s is working with manufacturers to make the RV3-BB vaccine more affordable and accessible in African and Asian countries where vaccines can be cost-prohibitive.

Eureka, she’s got it! The work of Murdoch Children’s Professor Julie Bines (back row, second from right) has been recognised with an Australian Museum Eureka Prize for her contribution in leading the development of RV3-BB, a vaccine that can prevent rotavirus gastroenteritis from birth, potentially saving thousands of young lives.

Don Cameron, Yati Soenarto, Jim Buttery and Margie Danchin, and scientists such as Ruth Bishop, Ian Holmes and Carl Kirkwood, and the many, many others who have contributed directly or indirectly to this body of work.”

Professor Bines’ work on RV3-BB has been in making it a safe, effective and affordable newborn rotavirus vaccine, particularly accessible for those in developing countries where the standard vaccines offered to Australian infants and children can be cost-prohibitive. “Rotavirus continues to be a major cause of death in young children and infants around the world, particularly in the poorest countries of Africa and Asia,” noted Professor Bines. “We’re working with emerging country vaccine manufacturers to produce a safe, effective and affordable vaccine that has the potential to save many thousands of lives and prevent suffering in many more. “This award is a wonderful acknowledgment of the efforts by many over the last four decades – including inspiring clinicians such as Graeme Barnes,

For example, PT BioFarma is conducting a phase 3 clinical trial of the halal RV3-BB vaccine in Indonesia, with results due in 2023. “Working with different manufacturers also provides an opportunity to develop the most appropriate approach for their population,” Professor Bines said.

In addition to leading the RV3 Rotavirus Vaccine Program at MCRI, Professor Bines is the Victor and Loti Smorgon Professor in the Department of Paediatrics at the University of Melbourne. She is a paediatric gastroenterologist and Head of Clinical Nutrition and Intestinal Rehabilitation at The Royal Children’s Hospital. Australian Museum Eureka Prizes are awarded annually in recognition of organisations and individuals who have contributed to science and the understanding of science.

Hear how Julie’s new rotavirus vaccine can prevent this nasty virus from birth

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

Republished with permission from pursuit.unimelb.edu.au

Annual Report 2021

Celebrating 35 years

VCGS: Helping Victorian families for over a generation Victorian Clinical Genetics Services (VCGS) is a leading provider of prenatal, childhood and adult clinical genetics services. We’ve supported thousands of Victorian families across multiple generations with life-altering health information. Who are we? We are genetics pioneers and knowledge translators. We’re also proudly a not-forprofit and wholly owned subsidiary of Murdoch Children’s. Our gifted team of researchers, clinicians, genetic counsellors, medical scientists and support staff are dedicated to making genetics and genomics accessible to everyone. Our core objective is to improve the lifelong health of children, individuals and families. People are at the heart of everything we do We believe that clinical genetics, genetic testing and genetic research are paramount to improving the health and wellbeing of children, individuals and families all over the world. Our Co-founder, Professor David Danks, inspired this approach as a pioneer of clinical genetics and genetic research in Australia. His early work positioned genetics at the forefront of life-changing healthcare, championing its contribution to the betterment of society. For the past 35 years, VCGS has provided continuous service to our newest Victorians (newborn screening), to pregnant people seeking reassurance (prenatal screening), to worried parents looking for answers (childhood microarray and exomes), and to families touched by rare disease with nowhere else to turn (acute care genomics).

Murdoch Children’s Research Institute

We strive to make genetics more accessible, understandable and useful for all our patients – from preconception through to adulthood. What do we do? We support our newest citizens – 2.2 million babies screened and counting. VCGS, in partnership with the Victorian Department of Health, delivers the hugely successful newborn bloodspot screening program. As of 2021, the program has screened more than 2.2 million newborn babies. Screening began in 1966 in Victoria and enables the early identification and treatment of babies with serious and sometimes life-threatening medical conditions. Over time, the number of conditions screened has grown from one to 27, with plans for more to be added. Detecting these conditions and intervening early to improve outcomes makes a big difference to the health and wellbeing of babies and families. We support new parents and parents to be Becoming a parent is a special privilege and we’re incredibly lucky to share in that reproductive journey. Our tests provide information and reassurance about the chances of having a child with a genetic or chromosomal condition. Pre-pregnancy screening VCGS was first in Australia to introduce genetic carrier screening in 2013. These tests identify individuals who are carriers of three common inherited genetic conditions: cystic fibrosis, fragile X syndrome and spinal muscular atrophy. Those identified as carriers can then use modern medicine to have a child who will not inherit one of these serious, life-limiting conditions. We are now leading research to broaden carrier screening options so couples have access to even more information on their journey to parenthood.

Pregnancy screening In 1996, we pioneered the use of maternal serum screening in Victoria. This was the gold standard in testing for Down syndrome during pregnancy. In recent years, we went a step further and developed an Australianbased, non-invasive prenatal test (percept NIPT), which gave Australians access to the latest reproductive technology. NIPT uses ‘cell-free DNA’ and is the most accurate screening test for Down syndrome. Significantly for pregnant people, NIPT reduces the need for risky, invasive procedures. To date, we’ve given close to 1 million pregnant people information about the health of their pregnancy. For those who receive unexpected news, our team of genetic counsellors offer one-to-one support and guidance in making decisions that are right for them and their families. We support families with answers to complex questions Over the past 35 years, knowledge about how our genetic make-up affects our health has continued to expand. Genetic testing at VCGS started with the study of whole chromosomes, the structures that carry our genetic information in the form of tightly packed DNA. These tests allowed us to identify the cause of several health and development problems. But many complex cases went unexplained.

Incredibly, genomic testing can now provide answers for patients who have exhausted all other testing avenues. For critically ill babies in intensive care, such testing can provide answers in as little as three days. This means fewer hospital days, fewer invasive procedures, a definitive diagnosis and, for some, access to life-saving treatment. Being able to make a genetic diagnosis provides invaluable comfort and clarity for families and offers a path forward. Genomic testing also has the potential to end the relentless diagnostic odyssey for thousands of families who bounce from service to service with unanswered questions. We embrace the future of genomics and use it to improve lives The future of genomics at VCGS and Murdoch Children’s is exciting. Associate Professor Sebastian Lunke believes that “as evidence of benefit grows, potentially every child from birth will have their DNA sequenced, setting them up for their best possible future. Critically ill children and their families searching for a diagnosis would have answers in hours, not days”. The future looks bright as we continue our work to make genomics universally accessible for lifelong health.

However, since completion of the Human Genome project in 2003, our understanding of genetics and the pace of technological development has exploded at VCGS. We’ve now moved from ‘genetics’, looking at single genes, to ‘genomics’, looking at all our genes, which gives us powerful new tools for investigating complex health problems.

Annual Report 2021

Celebrating 35 years

Cracking the genetic code Genomics delivers rapid diagnosis and treatment Children like Levi Trowbridge, who experienced his first seizure aged just three days, have a brighter future thanks to a new collaboration of world-leading genomics institutes. Murdoch Children’s Research Institute is part of the International Precision Child Health Partnership, or IPCHiP, which is improving the lives of children with complex genetic conditions. When Levi’s seizures began, he entered the care of Murdoch Children’s epilepsy team leader and the RCH paediatric neurologist Dr Katherine Howell. Levi had multiple seizures every day and at his weakest was on life support. Within days, rapid genomic testing diagnosed him with KCNQ2 epileptic encephalopathy, a rare genetic disorder. This enabled doctors to change his medication, which soon stopped the seizures. “Levi improved rapidly,” Dr Howell said. “His multiple daily seizures stopped by the time he was three weeks old; he was more alert, started to feed well and was able to go home from the hospital.”

More children will benefit from earlier diagnosis and targeted treatment thanks to the partnership, which includes Murdoch Children’s (with The Royal Children’s Hospital and the University of Melbourne), The Hospital for Sick Children in Toronto, Boston Children’s Hospital, and London’s UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital. Their experts will better analyse medical and genomic data to rapidly diagnose serious childhood illnesses, plus find and develop improved, more personalised treatments. “With individual babies like Levi we’ve had a glimpse of the possible benefits of prompt genetic diagnosis, but we now want to understand how much benefit this approach might provide to all the infants we see with these devastating conditions,” Dr Howell said. More than 7,000 rare diseases affect millions of people around the world, but only a few hundred receive approved treatments.

IPCHiP is the first major global collaboration around genomics and child health and its first project will involve severe infant epilepsy. Investigators will enrol babies diagnosed in their first year, sequence their genomes, change treatment based on the findings, and follow their development over the long term. Associate Professor Sebastian Lunke, the Head of Genetics and Genomics at Victorian Clinical Genetics Services, said another key early program of IPCHiP would be to improve acute care genomics. This would speed ultrarapid diagnosis and, hopefully, treatment for a range of neurological, metabolic, heart and other disorders. He hoped that IPCHiP would also eventually enable experts to predict future conditions. “The faster that we get an answer, the better the outcome for the child,” he said. Levi’s mum Karsha said the fast diagnosis was a godsend and the partnership would help many families globally. “By sharing resources and knowledge, researchers can test and tailor new treatments for children with rare conditions,” she said.

Advances in infectious disease The pandemic shone a light on the importance of our immune systems in dealing with infection. Murdoch Children’s continued its critical work in this space in 2021, taking on COVID-19 and other pressing global infectious diseases.

The research highlighted the potential of nondrug measures such as good hand hygiene, physical distancing, school and business closures, and travel and social restrictions in reducing the spread of infectious diseases. These simple public health measures, which helped contain COVID-19, were also likely to work against all types of infection. Infection-related hospital stays fell by 65 per cent in the seven months following the introduction of pandemic infection controls in 2020, compared with the previous five years. Murdoch Children’s research showed that children undergoing cancer treatment were still at relatively low risk of developing severe COVID-19 after becoming infected. But they were much more likely to have a severe case if they had underlying conditions such as acute co-infections and low white blood cell counts.

Murdoch Children’s Research Institute

New research conducted with the Peter Doherty Institute for Infection and Immunity found a cell-based influenza vaccine can protect against the flu in children and adolescents.

Amid the urgent challenges, came some good news. Drawing on hospital data from Victoria’s Department of Health, researchers showed that COVID-19 precautions led to fewer children and teenagers receiving hospital treatment for other common respiratory infections.

Children with cancer

Scan to watch Levi’s story

The cell-based flu vaccine production process is an alternative to the traditional manufacturing technique that requires fertilised hens’ eggs. Cell-based vaccine technology could potentially be more effective, and offer increased scalability and production speed.

The study by Murdoch Children’s, Peter MacCallum Cancer Centre and Goethe University looked at COVID-19 severity, shedding duration, symptoms and outcomes

Led by Vaccine and Immunisation Research Group (VIRGo) head Professor Terry Nolan, the study found the cell-based quadrivalent influenza vaccine (QIVc) produced a sufficiently robust immune response in children aged 2-18. Published in The New England Journal of Medicine, it was the first study looking at responses in children as young as two. in 131 children with cancer in 10 countries including Australia. The group found 95 per cent of children with cancer fully recovered from COVID-19. A third (37 per cent) required hospital treatment, 11 per cent entered ICU and four died. Those with serious cases tended to have comorbidities, infections and/or compromised immune systems. Murdoch Children’s and Peter Mac’s Associate Professor Gabrielle Haeusler said while the study identified some predictors of severe illness, prevention was still the best defence. “Ongoing surveillance is also critical in monitoring vaccine efficacy and impact of emerging COVID-19 variants in this vulnerable population,” she said. Cell-based flu vaccine In a typical year, up to 15 per cent of the world’s population contracts influenza, leading to as many as 650,000 respiratoryrelated deaths.

Antibiotics in pregnancy A Murdoch Children’s-led review found a common antibiotic, azithromycin, can reduce low birth weight and premature births if taken during pregnancy in countries with significant malaria cases. The systematic review found azithromycin reduced low birth weight and prematurity in Africa and Asia. But it didn’t lower infant deaths, infections, or hospital admissions. Azithromycin is inexpensive and widely used to treat chest and ear infections. It was unclear whether it would improve perinatal and neonatal outcomes in non-malaria endemic settings, and the potential harm on stillbirth rates needs more investigation. Similar research is underway in Fiji. Murdoch Children’s Professor Fiona Russell said all this research was likely to inform global maternal child health policy and, hopefully, benefit infants and mothers around the world.

Annual Report 2021

Celebrating 35 years

Reducing children’s allergies Australia’s unwanted title as the world’s food allergy capital is finally under threat thanks to new Murdoch Children’s research. Feeding guidelines change behaviour An MCRI-led study found that food allergy guideline changes have now led to a 16 per cent drop in infant peanut allergy. Significantly more parents were also feeding their babies peanuts. International infant feeding guidelines changed in 2016 to recommend introducing peanut and other allergenic foods before 12 months. Murdoch Children’s PhD candidate and study lead Victoria Soriano said this research was the first to test the approach in homes and measure the guidelines’ impact on peanut allergies. She said despite initial concern that parents might not follow the new advice, uptake was high. Peanut consumption by 12 months increased from 28 per cent to 89 per cent in the 10 years to 2019, with severe allergic reactions to early introduction uncommon. The study found peanut allergy prevalence among infants was 2.6 per cent in 2018-19, down from 3.1 per cent in 2007-11. Australia still has the world’s highest reported childhood food allergy rates – about one in 10 infants and one in 20 children aged up to five years has a food allergy. Most parents know that peanuts are particularly problematic, but fewer know about potential allergies to tree nuts – including cashews, almonds, walnuts and hazelnuts. Tree nuts are one of the most common foods causing acute allergic reactions and nearly all tree nuts have been associated with fatal allergic reactions.

Murdoch Children’s Research Institute

To address this, Murdoch Children’s Dr Vicki McWilliam and Associate Professor Kirsten Perrett have commenced the TreEat study, which is enrolling children with a confirmed peanut allergy, to see how a multi-nut diet compares with at-home introduction of a single tree nut. Repurposing old vaccines to reduce allergies Could the solution for allergies be hiding in plain sight? New research has found repurposing vaccines might help. The Murdoch Children’s-led study found a century-old tuberculosis (TB) vaccine could help reduce the incidence of eczema in high-risk infants due to its helpful off-target effects. The Bacille Calmette-Guérin (BCG) vaccine was used in Australia until the 1980s to prevent TB. The trial found it had a modest effect in preventing eczema in high-risk infants. However, there was insufficient evidence to recommend a neonatal BCG vaccination for all infants. The benefit was greater in children whose parents both had hay fever, asthma or eczema. A single dose of BCG soon after birth reduced their eczema incidence by 25 per cent.

Part of the long-running Melbourne Infant Study: BCG for Allergy and Infection Reduction (MIS BAIR), the project was based on the idea that BCG vaccines stimulate the immune system to better fight infections and be less allergy prone. The team is following these babies over their first five years to investigate BCG’s effect on respiratory tract infections, allergies and asthma. Whooping cough trial Another innovative trial is investigating whether an older whooping cough vaccine could help prevent allergies in early childhood. The OPTIMUM Study, conducted by Murdoch Children’s, the Telethon Kids Institute and The Children’s Hospital at Westmead, is recruiting infants aged 6-12 weeks. It is investigating whether a single dose of the discontinued ‘whole cell’ whooping cough vaccine at two months, instead of the current ‘acellular’ whooping cough vaccine, can help protect children against food allergies and eczema. Researchers want to know whether the whole cell whooping cough vaccine can train the immune system to recognise the difference between harmful germs and things it could mistakenly treat as a threat such as food.

Staying smart about dummies Sterilising dummies has been linked to increased food allergies in babies at 12 months. The Institute-led study with Deakin University compared dummy use and cleaning methods among more than 700 infants at six months and those with a confirmed food allergy at one year. Infants whose dummies were sterilised with antiseptic were more likely to have a diagnosed food allergy at 12 months. There was no increased food allergy risk at one year among dummy users when the dummies were washed in tap water, boiling water, put in the parent’s mouth, or not washed at all. Centre of Food and Allergy Research co-lead, Associate Professor Kirsten Perrett, said the link warranted further research into how good bacteria in the infant mouth and gut could help prevent food allergies. But she noted it was still important to clean dummies to prevent infectious diseases.

Hear Kirsten explain how her research is working to prevent food allergy

Annual Report 2021

Celebrating 35 years

Mental health program expands More than ever, building resilience in children and supporting young people’s mental health is vital.

A beneficial form of screen time

Healthy minds

A new digital eHub will better connect children and families with available mental health support.

Parents and carers can use a new app to distinguish between normal childhood ups and downs and emerging mental health problems.

One in four children will have a mental health disorder at some point, with puberty a key developmental stage when mental health issues emerge.

The planned online Child and Family eHub is designed to improve access and engagement with social and mental health services to enable early intervention, especially for disadvantaged families.

The Raising Children Network’s Raising Healthy Minds wellbeing app helps families tell whether the behaviour of children aged up to 12 needs further investigation.

The Mental Health in Primary Schools Pilot, developed by Murdoch Children’s with the University of Melbourne and the Victorian Department of Education and Training, has now expanded into more schools.

Murdoch Children’s Professor Sharon Goldfeld, Director of the Centre for Community Child Health, secured a National Health and Medical Research Council (NHMRC) Partnership Projects grant to design and build the eHub.

First started as a pilot program in 10 schools in 2020, the program received Victorian Government and Ian Potter Foundation funding in 2021 to expand to 100 schools by early 2022.

Her team and Melbourne Children’s Campus collaborators will co-develop and test the eHub in three communities experiencing significant vulnerability.

The program embeds a child mental health and wellbeing co-ordinator in schools to help identify and manage emerging mental health issues in students and connect education, social and health services. Early evaluation showed that it significantly increased teachers’ confidence in addressing students’ mental health issues.

The free online resource provides easily accessible local information and connections to health and social care systems. “In Australia, 14 per cent of children have a mental health disorder,” Professor Goldfeld said. “We urgently need effective prevention and early intervention strategies to mitigate the huge rise in child and family mental health issues.”

The free app is tailored to a child’s needs and circumstances, including their reaction to the pandemic. It supports parents, carers and professionals to address wellbeing, behaviour and emotions, providing the latest evidence-based information on self and family care. Murdoch Children’s Professor Frank Oberklaid and Raising Children Network Director Derek McCormack developed the app with parents and health professionals.

Ready, set, play!

Raising Children Network

Yard size does not affect children’s physical activity levels, suggesting that children’s exercise might not be affected by denser cities and shrinking backyards.

The Raising Children Network is supported by the Australian Government to provide families with free, reliable and up-to-date evidence-based information on all aspects of raising children.

A 2021 Institute-led study found no association between yard space and greenness and physical activity, highlighting a child’s capacity for outside play regardless of the size of their yard.

It also conducts research and works with other groups. Member organisations are the Parenting Research Centre, Murdoch Children’s and the RCH Centre for Community Child Health.

The study recorded the physical activity of 5,276 Melburnian six-year-olds over eight days. On average, they met physical activity guidelines of at least 60 minutes a day of moderate to vigorous physical activity. Murdoch Children’s and University of Melbourne researcher Dr Suzanne Mavoa said it is still important to identify ways to promote children’s activity levels and access to play spaces as a public health priority. “As cities become increasingly densified, urban design, shared indoor and outdoor spaces and schools will need to play a greater role in supporting young children’s physical activity and active play,” she said.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

Helping vulnerable infants thrive

Treatment and technology advances are helping more vulnerable newborns to survive and, in many cases, thrive. But we can still do better.

For the first time, Murdoch Children’s researchers have captured detailed images of newborns’ lungs as they took their crucial first breaths, which is set to improve diagnosis and treatment of babies like Milly.

When Joanna Bezette’s daughter Milly, now 2, was born at just 25 weeks, she had to be intubated and spent five months in neonatal intensive care.

In the trial, researchers used non-invasive electrical impedance tomography (EIT) technology. A small silk belt was placed around the chest of full-term babies born at the Royal Women’s Hospital to facilitate high-resolution images of the lungs.

Joanna said new technology being tested by Murdoch Children’s researchers could help others avoid the family’s trauma when it took a month to discover how underdeveloped Milly’s lungs were. “Having issues identified earlier would be very reassuring for a lot of parents with very sick babies, because it’s terrifying when you don’t have any idea what’s going on and what the future might look like,” Joanna said.

About 10 per cent of newborns, and almost all preterm infants, need resuscitation when their lungs don’t properly fill with air at birth.

The images showed how air moved through the lungs for each breath, and the breaths turned out to be far more complicated than previously thought. Murdoch Children’s neonatal and respiratory leader Associate Professor David Tingay said the new technology did not require radiation or interrupt life-saving care. He hoped the knowledge gained could also help doctors predict when a baby’s lungs might fail at birth. “This study showed that babies’ lungs are far more complicated than traditional monitoring methods had previously suggested,” he said.

Read more about Milly’s story

Murdoch Children’s Research Institute

“Improving interventions in the delivery room first requires understanding the processes that define success and failure of breathing at birth.”

Preventing bleeding in babies Melbourne Children’s Campus researchers hope to prevent bleeding in babies who need heart bypass support. The National Blood Authority-funded project investigated new treatments for babies born with heart disease who experience significant bleeding at the end of corrective surgery. Surgery usually involves a cardiopulmonary bypass, where a machine acts as the heart and lungs. Significant bleeding occurs in more than 10 per cent of child patients; 70 per cent of them aged under two. The study analysed blood samples from babies who had the surgery to understand what causes the bleeding. “Bleeding can lead to adverse outcomes such as a prolonged stay in the operating theatre, the need for life support and even death,” Project lead Professor Paul Monagle said. “The aim is to provide evidence for the use of new targeted treatments for babies undergoing cardiopulmonary bypass which will lead to less bleeding and better outcomes.”

Improving birth weights Improving birth weights and reducing complications in newborns is the goal of a new Murdoch Children’s project commissioned by North Western Melbourne Primary Health Network. It is implementing and evaluating new approaches to support pregnant women and families in Melton South. Murdoch Children’s Dr Elisha Riggs said a ‘whole of service’ approach involved a midwife and child health nurse supporting service providers

to promote equity-oriented healthcare. “The project will work with local communities to ensure services are well set up to meet the needs of culturally diverse families, including families of refugee background,” she said. The project is in partnership with Djerriwarrh Health Services, Melton City Council’s maternal child health services, VICSEG New Futures and Foundation House.

Annual Report 2021

Celebrating 35 years

From blood to brain, cancer under the microscope High-tech tools A smartphone app is helping children move on from cancer. The Murdoch Children’s-led project is developing a post-cancer app and digital tool for children, adolescents, and their families. The project was funded by the Cancer Council Victoria. Murdoch Children’s Associate Professor Maria McCarthy, of the Institute’s Take a Breath research program and the RCH Cancer Centre, is developing a smartphone app and online platform to help children and young people move on from cancer treatment. “Five-year survival rates in paediatric cancer now exceed 80 per cent,” Associate Professor McCarthy said. “Despite improved survival outcomes, children and adolescents treated for cancer are at risk of developing long-term physical and psychological difficulties related to their disease or treatment.” These ‘late effects’ may emerge over months and years after treatment ends. Until now young people and their families received little information or guidance. The Digital Survivorship Companion (DiSC) app will provide information including treatment history and health-related treatment impacts. It will also enable care and help recruit patients for appropriate trials. ‘Virtual’ radiotherapy Virtual reality helped children calmly receive radiotherapy treatment and could alleviate the need for general anaesthetic. A Murdoch Children’s and Peter MacCallum Cancer Centre trial gave 30 participants aged 6-18 virtual reality (VR) goggles to simulate cancer treatment before it began.

Murdoch Children’s Research Institute

Following the VR experience, the children and their parents reported reduced anxiety and improved understanding of the radiotherapy process. Associate Professor McCarthy said researchers tested a range of physical measures including nausea and anxiety and mood markers to get a genuine sense of whether it could impact quality of life. Young children often need a general anaesthetic to ensure they can have radiotherapy, meaning repeated anaesthetics over their radiotherapy cycle. The study showed 29 out of the 30 children did not need a general anaesthetic for their radiotherapy, indicating VR can help avoid its use. “Like most people, I was very aware of VR and its use in gaming and entertainment, but unaware of its potential utility in a clinical setting,” Associate Professor McCarthy said. “A lot of our inpatients can experience isolation, loneliness and boredom at times, so I can definitely see a place for an immersive experience like VR.” At the heart of the problem Using funding from the Australian Government and the Heart Foundation, a national network of specialist hubs are investigating how cancer treatments can cause heart damage. Associate Professor Rachel Conyers leads the project to establish three cardio-oncology hubs looking at how chemotherapy and other cardio-toxic cancer treatments damage the heart and arteries and cause strokes. “The aim is to improve the cardiovascular outcomes of Australian cancer survivors through a national network of multidisciplinary cardio-oncology hubs that combine research with clinical services,” she said.

Children and cancer Each year, almost 1,000 children are diagnosed with cancer across Australia. The new Victorian Paediatric Cancer Consortium (VPCC) will see Murdoch Children’s and the Hudson Institute of Medical Research co-lead a collaboration of Victorian hospitals and research institutes to improve childhood cancer survival rates and quality of life. The Consortium’s research will focus on finding new therapies for cancers with the greatest unmet medical need, including brain, bone and soft tissue cancers – all with low survival rates. The Federal Government’s Medical Research Future Fund (MRFF), supported by the Children’s Cancer Foundation, established the new collaboration with $9.6 million over three years. The VPCC is co-led by Hudson Institute’s Professor Ron Firestein and Murdoch Children’s Neuro-Oncology Group Leader Professor David Eisenstat, who is also Head of The Royal Children’s Hospital Cancer Centre. Researchers and clinicians across Melbourne’s Monash and Parkville medical precincts will explore nextgeneration precision oncology, new tumour immunotherapy developments and the role of epigenomics. A state-wide registry of childhood cancer survivors will be expanded to follow up, and later transition children to adult services.

Keeping hearts healthy James Bloor beat cancer when he was five, but it left him with a damaged heart. James had Burkitt’s lymphoma and developed heart damage from his anthracycline chemotherapy drug. Five years after James’ cancer was treated, he was diagnosed with cardiomyopathy – damaged heart muscle. “He went from being a little boy who ran and played all day, to a child who can’t do basic things without having to stop for a rest,” his mother Toni said. “He struggles to keep up with us, even when walking, and has to remind us to slow down.” James is part of a Murdoch Children’s study that aims to identify those at risk of heart damage following cancer treatment. “We hope that one day a prevention or cure for heart damage from cancer treatment can be found so that other children can have better outcomes,” Toni said. “We want James to beat this one too.” Murdoch Children’s is recruiting across 12 hospitals for the national Australian Cardio Oncology Registry (ACOR)/Bio-bank study, the largest of its kind in the world. Among a growing number of Murdoch Children’s cancer projects, the registry is seeking patients of all ages exposed to therapies that can damage heart tissue, like chemotherapy, radiotherapy and immunotherapy. The study is investigating biological clues using genetics and heart imaging studies to reveal who are the 15 per cent of children vulnerable to heart damage if treated with cancer drugs. Annual Report 2021

Celebrating 35 years

Screening for CMV Teddy, 3, was born profoundly deaf and with cerebral palsy. He tested positive for the common maternal infection cCMV, as part of a new study investigating whether it is feasible to add a CMV screening program to the standard hearing screening tests in Australia. See story, page 34

Research Excellence Our research is powered by state-of-the-art platforms and technology including stem cell lines, 3D printing, gene editing, LifeCourse data repositories and high-throughput drug screening. This enables our researchers and external collaborators to build capacity for children’s medical research in Victoria.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

GenV lifts off This generation faces increasing rates of complex health and development problems. To help transform the health of our newest Victorians, and generations to come, we are asking parents of newborns to choose to share their information with us to give us an inclusive and precise understanding of health and wellbeing across Victoria. This year saw the first babies recruited into GenV – a sophisticated research project, funded by the Paul Ramsay Foundation and the Victorian Government, with a simple goal: a better approach to child health, development and wellbeing.

New application of GenV Congenital cytomegalovirus (cCMV) testing as part of GenV Teddy, 3, was born profoundly deaf and with cerebral palsy. “If his cCMV diagnosis wasn’t picked up so quickly, he would be further behind in his gross motor skills,” Teddy’s mum, Victoria, said. “Having the awareness early meant we received support quickly and our specialists have been on top of everything.” Associate Professor Valerie Sung will work with GenV families to find out just how common cCMV is among Victorian newborns and the long-term outcomes for babies. The results should help determine whether universal newborn cCMV screening is warranted and support the development of a rapid same-day test that could lead to early treatment. 34

Murdoch Children’s Research Institute

Watch Teddy’s story

Scan to watch GenV in action Annual Report 2021

Celebrating 35 years

Julie Bines

Prizes and awards

Australian Museum Eureka Prize for leading the development of RV3-BB, a vaccine that can prevent rotavirus gastroenteritis from birth. Elected to the Australian Academy of Health and Medical Sciences Fellowship for her work on the RV3-BB vaccine, which has the potential to save children’s lives around the world.

Murdoch Children’s researchers were again recognised with multiple state, national and international awards in 2021. The Institute is proud to attract and foster talented medical researchers who are relentless in their pursuit of making a difference for children, young people, families and communities.

Melissa Little Homer W. Smith Award from the American Society of Nephrology for outstanding contributions that fundamentally affect the science of kidney health.

Murdoch Children’s Research Institute

Suzi Carp

Jonathan Kaufman

Igor Konstantinov

George and Joan Lefroy

Queen’s Birthday AO for distinguished service to health and medical research, to children and young people, and to the community through leadership roles.

University of Melbourne Chancellor’s Prize for Excellence in PhD Thesis for his research on diagnostic processes for young children with urinary tract infections.

Queen’s Birthday OAM for service to medicine as a cardiothoracic surgeon.

Queen’s Birthday AM for significant service to the community through philanthropic support initiatives.

Sebastian Lunke and Zornitza Stark

Margie Danchin

Jane Halliday

Amanda Fosang

Innovation Award (Research Translation and MedTech & BioTechnology) from Innovation Australia for their work in the Acute Care Genomics project.

BioMelbourne Network Women in Leadership Awards (Directors Award for leadership) for her work across the health sector during the COVID-19 pandemic last year.

Queen’s Birthday AM for her research measuring the impact of potentially harmful prenatal exposures on the health and wellbeing of infants, children and young adults.

Queen’s Birthday AM for significant service to medical research in the field of arthritis, and significant service to international societies.

George Patton

Sarah Stephenson

Harriet Hiscock

Yara-Natalie Abo

Elected to the Australian Academy of Health and Medical Sciences Fellowship for his extensive child and adolescent mental health research in Australia and global adolescent health work.

Young Tall Poppy Science Award for her work on childhood epilepsy. OUTstanding LGBT+ Role Model (Involve network) for her contributions to LGBT+ inclusion inside and outside the workplace, professional achievements in improving children’s health and the influence of her role at MCRI.

Elected to the Australian Academy of Health and Medical Sciences Fellowship for her work integrating health, social and education services to improve health and wellbeing for children.

National Heart Foundation PhD Scholarship for her research, which aims to advance Strep A vaccine research and prevent heart disease. National Heart Foundation Excellence Award for the top-ranking PhD project submitted to the scholarship. Annual Report 2021

Celebrating 35 years

Why MCRI?

Foundations of success To achieve its purpose of wanting all children to live a healthy and fulfilled life, the Institute has over the course of its history invested in a suite of worldclass, transformative, technological and expertisebased platforms. These enable our researchers and their external collaborators to delve deeply into their questions with expert assistance, translating the outcomes to realworld applications.

Stem Cell Lines, 3D Printing, Gene Editing and Highthroughput Drug Screening

Health Economics and Health Services Research

Associate Professor Silvia Velasco, Sarah and Lachlan Murdoch Fellow

Epidemiology and Biostatistics

. . . and the platforms we hope to address in the future: Gene therapy siRNA, CRISPR libraries Advanced cellular therapy

AI/machine learning/virtual reality capability

I recently joined the Institute to lead the Neural Stem Cell group. I completed my studies in Italy at the University of Turin, then trained in Spain, New York, and most recently in Boston at the Broad Institute and Harvard University. With my research team, I am interested in understanding the causes and mechanisms behind neurodevelopmental disorders, like autism spectrum disorder, intellectual disability and developmental delay. I use human stem cells to grow “brain organoids”, which are complex neural structures mimicking human brain development, providing an invaluable tool to investigate brain diseases “in a dish” and find effective treatments.

Data – storage, analytics, visualisation, linkage and expertise

What influenced you to choose Melbourne for this stage of your career?

Enabling translation to impact via knowledge translation, health economics, and health services research pipeline

I was fortunate enough to receive the inaugural Sarah and Lachlan Murdoch Fellowship, which enabled me to join Murdoch Children’s. I was drawn to apply for the position because I was attracted by the Institute’s vision and its supportive and friendly culture. I was excited by the idea of working with outstanding and enthusiastic leaders in the stem cell and developmental biology fields and by the challenge of translating stem cell research into outcomes for patients. I was also attracted by the opportunity to work in a more clinical setting, collaborating with neurologists, geneticists and clinicians at The Royal Children’s Hospital. Finally, the idea of bringing my research closer to patients and their families was really what motivated my decision to move to the Institute.

Organs on a chip for drug development and screening Developmental Imaging

Melbourne Children’s Trial Centre

Digital Technologies

Multi-omic integration with the electronic medical records using AI/machine learning Molecular phenomics Technology to measure phenotype at scale (ePhenome) Vaccine testing platform

Cord Blood and Bioresource Centre (including Rare Cancer Bank)

Victorian Clinical Genetics Services

Health Informatics

Melbourne and Australian Genomics Health Alliances

Knowledge Translation and Impact

LifeCourse Data Repository

Imaging analytics

Flexible clinical registry platform linked with universal patient consent model 3D printing to scale

Murdoch Children’s Research Institute

Tell us a little bit about yourself?

What excites you most about the opportunities being at the Institute presents? I am excited about the opportunity to work with exceptional colleagues in an extremely multidisciplinary and collaborative environment across the Institute and the hospital. I’m thrilled to contribute to developing new experimental approaches together with the world-class stem cell medicine platforms at MCRI, like the gene editing, disease modelling, drug screening, bioinformatic and imaging platforms. I am also very excited to be part of the reNEW initiative and to see how this exceptional international effort, by forming these collaborations across world-leading scientific institutions, will facilitate the translation of basic stem cell science into targeted research. Ideally, this creates new effective and safe stem cellbased therapies to ultimately deliver on the promise of stem cell medicine. What would you say to other scientists or clinicians looking to make the next step in their research career? Look carefully at all the available options and choose the one that best enables you to realise the full potential of yourself and your research. Don’t be afraid to embrace new challenges, no matter if this means moving out of the comfort zone you have built over the years. Look outside your home institution or home country. Look for a place that will do everything it can to support you in your endeavours and that always remembers its purpose and mission. Choose a place that promotes a healthy and friendly culture, where the priority and shared goal is to find ways to increase knowledge and improve human health.

Annual Report 2021

Celebrating 35 years

Measures of success Media mentions

2020

2021

Media exposure for the Institute in Australia and around the world is a testament to the ongoing advocacy work of our experts and MCRI’s reputation as a trusted source of truth for families. Using a variety of channels ensures our child health messages are heard by a diverse range of audiences.

9,359

Research metrics

The Institute’s social media channels continued their significant growth in engagement, reflecting the myriad and emerging ways parents, carers and friends consume health information from trusted sources.

Traditionally, publication data metrics have captured the breadth of our researchers’ activities, demonstrating their productivity as well as the impact and ongoing influence of their research output. By these measures, the Institute’s researchers and collaborators place us in the top three child health research institutes globally.

5,838

5,250 4,468

Facebook

3,469

People reached:

Twitter Impressions:

Engagement:

1,014,258 154,819

695 AU online news

Social media/digital

Global coverage

YouTube

Impressions:

Views:

Engagement:

Total hours:

Instagram

Website

People reached:

Unique visitors:

52,764

Engagement:

1,737

Murdoch Children’s Research Institute

48,970

27,672

53.66% of papers had an international

Highly Cited

collaboration

Professors John Carlin and George Patton were named among the world’s most highly cited researchers in 2021.

Co-authors came from 137 countries and from 1,712 different institutions

2,314,000

Broadcast (AU)

802,154

Collaboration

52,764 1,737

382,015

Papers

1,170 items in total 2,823 total citations H-index=19 19 of our 2021 papers have been cited 19 times or more

Altmetrics

The Highly Cited Researchers 2021 list from Clarivate identifies researchers who demonstrated significant influence through the publication of multiple highly cited papers, as measured by the fact their research papers ranked in the top 1 per cent by citations in the Web of Science citation index.

Altmetrics is a system that tracks the online attention that research outputs receive.

69,536 mentions of 4,881 research outputs 600 policy mentions 942 patent mentions 404 Wikipedia mentions

Scan to see our COVID thought leaders in action The Institute’s researchers pivoted to address urgent questions around COVID-19 in global health, household transmission and stem cell-based modelling of the disease’s effects and possible treatments. Importantly, our researchers communicated their work to a concerned public, providing a trusted voice of reassurance at this worrying time. Annual Report 2021

Celebrating 35 years

Treating Levi’s seizures Levi Trowbridge was diagnosed with KCNQ2 epileptic encephalopathy, a rare genetic disorder that causes frequent seizures, at three weeks of age after taking part in a rapid genomic testing program for critically ill children at the Victorian Clinical Genetics Services, the genetic testing arm of MCRI. Mum Karsha and dad Steve said rather than waiting on tenterhooks for six months, they had a diagnosis within days, and after changing medications Levi went from having five seizures a day to not having any since February 2020. See story, page 22

Our People, Our Values Our people are at the heart of our innovative world-class research into the identification, prevention and treatment of conditions affecting the health of babies, children and adolescents. Murdoch Children’s Research Institute is the place to be for those who want to change the face of child health.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

Emerging leaders Murdoch Children’s young researchers are the leaders of tomorrow, poised to make the next 35 years of impact for child and adolescent health. During their time at MCRI, our emerging leaders demonstrate their embodiment of and commitment to the Institute’s mission, vision and values.

Meet our emerging leaders, from left:

Dr Kiymet Bozaoglu Team Leader “I want to understand how and why autism develops in children so we can find better strategies to help them.”

Dr Simran Kaur Research Officer “I want to work with families and children with rare genetic brain disorders to discover new insights and ultimately make a difference in their lives.”

Ken Knight Research Impact Manager “I want to bridge the gap between what we know and what we do for children’s health.”

Jordan Gibbs Aboriginal Leadership committee member, Peerapper man “I want to help my community build a stronger future, grounded in culture, for our Aboriginal and Torres Strait Islander children.”

Dr Melanie Neeland Senior Research Officer “I want to discover new treatments for children with lung complications.”

Associate Professor Daniel Pellicci Group Leader “I want to discover immune markers that can help find, treat and ultimately prevent childhood diseases.” 44

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

MCRI & VCGS staff awards Each year, MCRI and VCGS recognise the important contributions our staff made to child and teen health. Those contributions that might not make global headlines, but have a huge impact for individuals, families, communities and populations. The MCRI and VCGS Staff Awardees are nominated by their peers in recognition of dedication, excellence and commitment to making a difference to children’s lives.

Inspiring Others Award Presented to an MCRI or VCGS employee who has demonstrated excellent leadership qualities. Ritika Saxena Research Assistant, Blood Development, Cell Biology Andrea Frigo Head of People and Culture, Growth and Innovation

Winners of the MCRI & VCGS Staff Awards for 2021:

Organisation Innovation Award Given to an MCRI or VCGS staff member who has demonstrated innovation and creativity to improve our way of working. This category is open to all staff who facilitate interactions to generate creative workflow/organisation solutions. Miriam Fanjul Fernandez Medical Scientist, TGU Clinical Genomics, VCGS

Above and Beyond in Research Award For outstanding contribution made by a mid-level career researcher through research that enhanced the impact of their group or wider team. Ryan Toh New Vaccines, Infection and Immunity Nicole Messina Infectious Diseases, Infection and Immunity

Safety, Health and Wellbeing Award Recognises an individual who has shown outstanding initiative to promote, enhance or protect the safety, health and wellness of our staff and students. Eleanor Neal Research Assistant, AsiaPacific Health, Infection and Immunity Erin Oldaker Quality Manager, VCGS Lab Management, VCGS

Above and Beyond in Research Support Award For outstanding support for research activities in 2021. Supporting research activities could include work done by, but not limited to, administration staff, laboratory staff, research nurses and staff in Research Support and Operations, CEBU, MCTC and CR. Megan Kaegi Research Assistant, Inflammatory Origins, Infection and Immunity

Rising Star Award Presented to a student based on their exceptional participation in the Institute community, as well as research outcomes for the year. Jeremy Anderson PhD student, New Vaccines, Infection and Immunity Sean Wilson PhD student, Kidney Regeneration, Cell Biology Victoria Soriano PhD student, Population Allergy, Population Health

From the nominees, the VCGS Employee of the Year was selected by VCGS leadership and the Research Excellence Award recipients in 2021 were selected by the MCRI Director.

Above and Beyond in Service Excellence Award Given to a VCGS employee who demonstrated depth of care to our patients and/or commitment to our high quality of service. This award is open to all areas of VCGS. Sarah Jamieson Specimen Reception Co-ordinator, VCGS Laboratory Services Stefanie Eggers Research Genomics Head, TGU Research Genomics, VCGS

Murdoch Children’s Research Institute

Mentor Award Recognises an outstanding mentor, supervisor, manager or colleague who has supported and guided others in an exceptional way in 2021. Paul Licciardi Team Leader/Senior Research Fellow, New Vaccines, Infection and Immunity Marc Seal Group Leader/Senior Research Fellow, Developmental Imaging, Clinical Sciences

Pivot Award in Research The pandemic has triggered changes in research funding, priorities, topics and practices, as well as a shift to remote work. This award recognises an individual or a team who have quickly adjusted their research to explore some of these pressing issues or be able to pursue their research subject despite the logistical hurdles. Conor McCafferty PhD student, Haematology, Clinical Sciences Pivot Award in Organisation or Service Delivery In a year that has brought new ways of working, this award recognises an individual or team who have taken on additional responsibilities/changed roles to create and implement processes that enable MCRI and VCGS to continue to safely deliver services and research. Gerardo Luis Dimaguila (Ikee) Health Informatics Officer, SAEFVIC Leading or Contributing to Large Projects/Teams Award Recognises and promotes project/program coordinators and managers playing a key role in the organisation and smooth running of large projects and/or teams of people. Alisha Gulenc Senior Project Officer, GenV Research Associates Award for Professional Excellence Recognises staff members below postdoctoral level, such as project and research assistants whose roles do not involve team leadership but contribute directly to research activity. Joan Gains Community Co-ordinator, Neurodisability and Rehabilitation, Clinical Sciences VCGS Employee of the Year Award Presented to a VCGS staff member who has gone ‘above and beyond’. Sarah Jamieson Specimen Reception Co-ordinator, VCGS Laboratory Services Research Excellence Award Presented to an outstanding senior researcher based on exceptional research outcomes for the year. Melissa Little Chief Scientist, Theme Director, Cell Biology

Taking mentoring seriously Murdoch Children’s supports mentoring at all levels, informally and formally, creating the opportunity for all our people to learn, grow and develop in a way that supports their individual development needs. In 2021, the Institute established the Beyond Mentoring Program, a structured program which provides an aligned mentor, a peer-learning group and a series of skillsbased workshops and coaching sessions. The program creates space for participants to reflect on their career and development journey, while providing them with skills and coaching to apply their insights to their workplace. For mentees, the benefits include strengths-based assessment, career and development goal setting, expanded networking opportunities and confidence building. The Beyond Mentoring Program recruited and matched more than 60 mentors and mentees in 2021 who will continue to participate in group coaching sessions, networking and skills-based workshops, and also enjoy a formal program close that recognises and celebrates their achievements over the program.

The Suzi Carp Scholarship Dr Elena Tucker from the Murdoch Children’s Genetics Theme was awarded the 2021 Suzi Carp Postdoctoral Scholarship for her research into reproductive development. Dr Tucker uses genomic approaches to investigate the genetic basis of human ovarian disease, and has shown research excellence and strong leadership potential during her PhD and postdoc work at the Institute. The scholarship is an Early Career Researcher Award that is intended to support a Murdoch Children’s ECR who is displaying research excellence and leadership potential through the time late in their PhD studies to early in their first postdoctoral research position. The scholarship aims to support and help set the stage for a successful research career by contributing to the recipient’s research track record and increasing their competitiveness in attracting external funding.

Annual Report 2021

Celebrating 35 years

Innovating for impact With a focus on impact powered by research with children at the heart, the Institute established a new unit called Growth & Innovation in 2021. The focus is driving greater impact from MCRI’s work and identifying new pathways to growth that will help build long-term sustainability.

Chief Innovation Officer and Head of Growth Strategy, Dr James Dromey, said: “We work in the nexus between innovation, stakeholder engagement, funding and people and culture, aligning and enabling internal capabilities in these areas to create meaningful external impact.” The establishment of Growth & Innovation also recognises that to realise the potential impact and value of the Institute’s work to benefit children and families around the world requires a strategic focus and approach, working closely with partners, funders and other supporters.

AllergyPal — a friend in your phone AllergyPal, our food allergy management companion app, is now ready to go global with the support of the AllergyPal company, spun out from the Institute in 2020. With over 50,000 Australian users, the app is now being adapted for launch in the US market. This means thousands more children and families will benefit from AllergyPal to better manage their food allergies in their everyday lives.

Behind our brilliant minds Sleep with Kip — teaching Australian children to sleep well

Gut health for life We have all heard about the importance of maintaining a healthy gut. Prevatex is a spin-out company arising from research conducted with the Barwon Infants Study (BIS) – a joint cohort research study with Murdoch Children’s, Barwon Health and Deakin University. Researchers had found that high levels of Prevotella, an ancient type of bacteria, in pregnant mums’ digestive tracts was associated with reduced food allergy incidence in their babies. 48

Murdoch Children’s Research Institute

Prevatex has built on the studies of the BIS team by licensing intellectual property related to this discovery to develop and bring to market a Prevotella-based probiotic. The product aims to establish and maintain a healthy maternal microbiome during pregnancy, and hopefully minimise the risk of food allergy in their babies.

Backed by 20 years of our sleep research, the Sleep with Kip storybooks are a collection of six children’s books incorporating research-proven behavioural techniques to support 3-9 year olds (and their families) to sleep well. The books are due to hit libraries in 2022 and are already supporting children through an online reading platform.

In another challenging year for welcoming new staff and students remotely, our People and Culture team again excelled in helping our new starters to feel at home, while working between their own homes and the organisation. Four hundred new people were welcomed across the Murdoch Children’s and Victorian Clinical Genetics Services, who were supported with an expanded Leadership Development program, now spanning: • Leadership fundamentals • Leadership in practice • Leadership excellence Our Institute in numbers:

Visit the Sleep with Kip website

• • • •

MCRI paid staff – 1,332 VCGS paid staff – 271 All Students (MCRI & VCGS) – 232 Honorary Staff and Contractors (MCRI & VCGS) – 1,022

Champions for gender equity The Murdoch Children’s Research Institute has enrolled in a Science in Australia Gender Equity (SAGE) program to better support and champion gender equity in the workplace. SAGE aims to improve gender equity in the Australian higher education and research sector, which particularly experiences a high loss rate of women wanting to pursue careers in science, technology, engineering, maths and medicine. The program applies the Athena Swan Charter, which is based on 10 key principles such as addressing unequal gender representation, committing to tackling the gender pay gap and removing the obstacles faced by women.

MCRI strives to not just be leaders in groundbreaking, world-class research, but also by setting the standard for an inclusive and dynamic workplace culture. The Institute will undergo an evaluation to receive SAGE accreditation, which will involve: • Forming an MCRI self-assessment team of volunteers from a range of backgrounds and roles • Collecting quantitative and qualitative data to understand any gaps and barriers to gender equity, diversity and inclusion • Developing and implementing a plan in accordance with SAGE guidelines • Collating the outcomes of the selfassessment and submitting for SAGE accreditation. MCRI will be given a Bronze Award if successful.

Annual Report 2021

Celebrating 35 years

Sustainability & Engagement

Through collaboration with others, we strive to support children across Australia and the world. We work closely with government to ensure our research has an impact on government policy and with philanthropic partners to ensure our impact spreads to the people and communities who need it most.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

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

6A Foundation

Eisen Family Private Fund

The John Barnes Foundation

Judith Robinson

Anna and James a’Beckett

Equity-One Mortgage Fund Ltd

John Hart Pty Ltd

The Ross Trust

Sue a’Beckett

Estate of B O Williams

Aaron Johnson

Simon and Alison Rothery

Antoinette Albert

Estate of Janice Carter

Johnstone Family Foundation

Royal Children’s Hospital Foundation

Kenneth Allardice and Julie Roy

Estate of Joe Axiak

Joan Kaaden

Alicia and Michael Ruhl

Suzanne Allen

Estate of Dr Mary Remnant

Ian Kennedy AM and Sandra Hacker AO

Gerry Ryan OAM and Valerie Ryan

Roseanne Amarant

Estate of Melanie Jan Paramor

Kim Kerr

SALT Catalyst

The Andrews Family Trust Charitable Fund

Estate of Nance Elizabeth Creaton

Lidia Kerr

Pam Sargood

Peter Austin

Estate of Rosemary Agnes Warner

Foisal Khan

Schiavello Group Charitable Foundation

Estate of Sylvia Gelman

Anders Kruse

The Scobie & Claire Mackinnon Trust

Jane Fenton AM and David Miles AM

Jamie Kuhn

Michael and Rita Shearer

Fight Cancer Foundation

Latitude Financial Services

Anne and Bernie Shinners

Flicker of Hope Foundation

George Lefroy AM and Joan Lefroy AM

Silicon Valley Community Fund

Diana and John Frew

Marj Lefroy

Leon Skaliotis

Amy Gallasch

Nichola Lefroy and Sam Riggall

Andrew and Heather Slutzkin

Coral and Tony Gallasch

Lefroy Family

Cathy Smith

Michael and Maureen Barden

Ross and Judy Gardner

Diana Lempriere

Colin and Jan Smith

Anne and David Barton

GAVI Alliance

The Lewin Family Foundation

Jacob and Fiona Smith-Hughes

Beverley Batson

John and Gaye Gaylard

Life Healthcare

Lady Marigold Southey AC

Norma Beaconsfield

Brian Little

Patricia Spencer

Gillian Beaurepaire

George & Freda Castan and the Suzi & Barry Carp families

Neil Spitzer

Beige Technologies Pty Ltd

Sonia Gilderdale

Logan Family Foundation

Janet Bell

Goldman Sachs Gives

Loti & Victor Smorgon Family Foundation

Stafford Fox Medical Research Foundation

Danielle Besen

Goldschlager Family Charitable Foundation

Luca Miraglia Foundation

Roy Stanley

Melissa Goode and Christopher Archibald

Christine MacGill

Grant and Terri Stephenson

The Marian & E.H. Flack Trust

Claire Stubbs

Judy Matear

The Sunraysia Foundation

Christine and Denis McConnell

Peter and Mary Tallis

Elizabeth McLaurin

Angie and Vidoja Tasic

McKenzie Family

Carmel Taylor

McMeckan Family Foundation

Polly Taylor

McNally Family Foundation

Charles Tegner

The McPhee Charitable Fund

Thelma and Paul Constantinou Foundation

Australian Mitochondrial Disease Foundation Diana Avila Stuart and Jillian Bales Ballandry (Peter Griffin Family) Fund, a subfund of Australian Communities Foundation

Kara Best John Bickerstaff Big W Bill & Melinda Gates Foundation BioAutism BioTools Pty Ltd Blue Bus Narelle Bulley Bupa Australia John Calvert-Jones AM and Janet Calvert-Jones AO The Calvert-Jones Foundation Cameron Foundation Krystyna Campbell-Pretty AM Carla Zampatti Foundation Paul Carr Lorraine Cash Steven Casper and Ilana Wald Centre for Paediatric Allergies at Epworth

Dean Grant Gras Foundation Trust Elaine and Len Greenhall GrewIan Investments Pty Ltd Rosemary Griffith Grove Group Maurice Hall Jillian Harris Heart Foundation Geoffrey Heeley Raymond Hemphill Yung Shing Ho The Holckner Family Wilson Homes Jess and Grant Hooper

Chan Zuckerberg Initiative DAF Tom Chikulin

Elizabeth Huang

Children’s Cancer Foundation

In celebration of Bruce Lefroy Foundation

John and Catherine Christodoulou Sandra Clark Ronda and Ernest Clarke Christine and John Collingwood John Collins AM and Mandy Collins Corella Fund Caroline and Philip Cornish Rodney Costigan Laurence Cox AO and Julie Ann Cox AM N. Crux & L. Healey CSL via a Centenary Fellowship The Cuming Bequest

In celebration of Raph Casper’s Bar Mitzvah In lieu of birthday gifts Raie Goodwach In memory of Jackson Lyons In memory of Julie Weston In support of Achondraplasia Research In support of Dr Hamish Graham In support of Saskia Dahl In support of Sebastian Wearn-Jarvis In support of The Suzi Carp Recognition Fund Inner Wheel Australia Foundation The Isabel & John Gilbertson Charitable Trust

Datt Family Foundation

Kim Jackson and Scott Farquhar

Gerry Davies and Jessica Mariani

Jam and Jelly Foundation

Charles Day and Elise Everest

Peter and Margaret James

Richard and Sarah de Crespigny

Jamie and Rebecca Gray Endowment

Jasmin Diab

Samari Jayarajah

Des and Margaret Dodds

David Jenkins and Fran Lefroy

Patricia Dykes

Jim & Claudia Craig-Bellwether Foundation

The Eirene Lucas Foundation

Murdoch Children’s Research Institute

Gourlay Charitable Trust

Tim Hovey

Children’s Tumour Foundation of Australia

Louise Gourlay AM

Merricks Capital Services Pty Ltd John Miller Edwina Molony and David Hunter Margaret Molony Susan Molony Mark Montag Muscular Dystrophy Australia Rupert and Jerry Murdoch Sarah Myer and the late Baillieu Myer AC The Myer Foundation

Anita Thomas Peter and Debbie Thomas Thyne Reid Foundation Juan Torres Trevor & Heather Cohen Charitable Legacy UHG Foundation Pty Ltd Uncle Bobs Club Lyn SL Unsworth Patrick and Margaret Upfold

Ida Nathan

Tom Valenta OAM

National Stem Cell Foundation of Australia

Tony and Judy Van Bergen Alasdair Vance

Philip Myles Neri

Vanguard Group Foundation

Victoria Newton

VCF Bird Charitable Trust

Dorothea Nossbaum

Viertel Charitable Foundation

ONE in 5000 Foundation

Vincent Chiodo Charitable Foundation

Tony Osmond and Fiona Griffiths

Robert Watmuff

Maria Paikos

Kelvin Wearn

William Palmer

Wellcome Trust

Susan Parker

Phillip Weston, in memory of Julie Weston

Rowly and Judy Paterson The Paul Ramsay Foundation

Tom and Sarah Whinney

David Penington AC and Sonay Hussein

Ariane Whitehouse John and Irene Williams

William Phillips

Paul Williams

PKU Association of NSW Inc

Shirley and Geoff Williams

Proceeds from “Never Work with Animals or Children”

Jillian Woinarski

Jenny Renton

Lesley Woodall

Andrew Rettig and Amanda Briskin-Rettig RM Ansett Trust Judy Roach, Richard Roach and Sally Gough

The Wolf Foundation Mandy and Edward Yencken The Yulgilbar Foundation Elane Zelcer

Annual Report 2021

Celebrating 35 years

An enduring legacy MCRI is built on a foundation of philanthropy. It was the generosity of donors, many personal friends of our Founders Dame Elisabeth Murdoch and Professor David Danks, that helped establish what was then called the Murdoch Institute for Research in Birth Defects. Since its inception the Institute has grown from a handful of researchers to become Australia’s most influential and highest rated child health research institute and one of the top three worldwide. The success of MCRI and the impact it has had on the lives of children would not have been possible without the generosity of visionary philanthropists including Sir Jack Brockhoff, the Scobie and Clare Mackinnon Trust and the Miller family. Through the leadership of our Global Ambassadors, Sarah Murdoch and Suzi Carp AO, the commitment of our Global Advisory Board, the Development Board led by Miffany Blythe and the Council of Ambassadors, the spirit of generosity continues to power the growth of MCRI. We are grateful for the passionate commitment of our donors to invest in our brilliant researchers, cutting-edge projects and strategic initiatives which will continue to transform child health for generations. Together, they have driven and accelerated the medical breakthroughs that have and will continue to give children the opportunity to live healthy and fulfilled lives. As we look ahead to the next 35 years, we continue to draw inspiration from Dame Elisabeth’s hands-on approach to philanthropy, as we welcome a new wave of modern philanthropists inspired to make transformational social change through innovative funding approaches focused on delivering impact on a global scale. With children at the heart of everything we do, there is no greater privilege than to work alongside our donors and witness the lasting impact of their support. With sincere appreciation, Matthew Hannan, Head of Philanthropy The late Dame Elisabeth Murdoch on her 99th birthday, at Cruden Farm.

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

35 years of philanthropy Wanting to help shine a light on NF1 sufferers, Zoe Petropoulos and her mother Anne established Flicker of Hope. Zoe, now 21, was diagnosed with NF1 at the age of four months. She has multiple tumours, including in her brain and spine. Zoe also has dyslexia and co-ordination problems, but considers herself lucky as many others with NF struggle with far more severe health conditions and complications. Foundation’s tireless efforts shine a light on NF Established in 2018, the Flicker of Hope Foundation aims to raise awareness of neurofibromatosis (NF). This little-known genetic disorder affects about one in 2,500 people worldwide, with about 10,000 Australians currently living with the condition. Neurofibromatosis type 1 (NF1) is a genetic condition characterised by tumours in parts of the nervous system, such as the brain, spinal cord or nerves that send signals throughout the body. It is caused by a mutation in the NF1 gene, which reduces the neurofibromin protein, a tumour-suppressor. With less tumour-suppressor, certain types of tumours can form, such as in the optic nerves or peripheral nerves, which cause pain, disfigurement and disability. Tumours growing near or around important neural structures like the spinal cord can be difficult to remove safely.

Through their determination, commitment and fundraising events such as Kicking Butt for NF Research, the foundation has raised and donated more than $1.3 million to support research on neurofibromatosis. Thanks to Flicker of Hope’s generosity, two research projects are currently underway here at MCRI. One is looking into trametinib, an anti-cancer drug that has shown promising early results in treating some NF1 tumours and a second research project that aims to understand how NF1 affects brain development and function in relation to autism which occurs in 25-30 per cent of children with NF1. State-of-the-art stem cell modelling methods and high throughput drug screening facilities at MCRI are being used to support this project.

Murdoch Children’s Research Institute

Joe and his brother Andy spent their lives working on the family property and were well known throughout the district for their farming expertise. They kept to themselves and led a frugal life, only needing each other, trusted friends, and their beloved animals.

Eden O’Meara almost her leg to Strep A infection. Read about her story on page 16 and our efforts to develop a vaccine against this insidious infection.

After Andy died suddenly in 2017, life was never the same for Joe. His health began to deteriorate, and it was during this time he decided to write his Will and include a bequest for MCRI. While Joe did not have a family of his own, he always had empathy for children and wanted to give them the opportunity of a better, healthier and happier life.

A powerful legacy for children

Joe will never be forgotten at MCRI. His story will continue through our lifechanging research projects and the children who have their whole lives ahead of them thanks to his kindness. We receive all types of bequests from generous, everyday Australians and are grateful for every single one. Please visit mcri.edu.au/bequest to find out how to leave your legacy for the benefit of children.

Joe Axiak will forever be remembered as a hard-working cattle farmer from Dunedoo, NSW, and for making an enormous difference to children’s lives across the country.

Images from left: Anne (left) and Zoe Petropoulos. Joe Axiak (left) as a young boy; Joe (centre) with his family.

As well as tumours, 80 per cent of children with NF1 will have moderate to severe cognitive difficulties. About 40 per cent of affected kids have ADHD, one quarter have autism spectrum disorder and up to half have learning difficulties such as dyslexia. Sadly, there is no cure, and treatment includes surgical tumour removal or chemotherapy. Moreover, treatments for the underlying cognitive problems are limited.

After a lifetime on the land, when Joe died at the age of 78, he left his entire estate to help sick children. His significant gift is now helping our researchers find new preventions, treatments and cures for many childhood conditions.

The Danielle Besen Scholar in Mental Health Leadership As a result of the ongoing pandemic and the related public health measures through 2021, children experienced significant impacts on their mental health, which will continue to be felt over many years to come. By establishing the Danielle Besen Scholar in Mental Health Leadership within the Centre for Community Child Health (CCCH), the Institute is empowered to take action and make a difference. The Scholar position was generously established by Danielle Besen, a mother

of three who has been involved in youth philanthropy for more than 23 years. Danielle believes this role will enable the Institute to have a voice in how governments engage school systems and the medical community to improve youth mental health, as well as remove the stigma of mental health in our society. Rachel Whiffen, the inaugural Danielle Besen Scholar in Mental Health Leadership – Mental Health Advocacy Lead, is excited about the opportunity to contribute. “I feel it has never been more important to ensure that all children have a strong foundation for lifelong wellbeing,” she said.

“The opportunity to be able to work extensively with leaders in child mental health and development is incredible.” According to Professor Sharon Goldfeld, the pandemic has exacerbated the pressures on young people’s mental wellbeing, with children facing “generation-defining disruption”. “Danielle’s vision and generosity ensures that Rachel and the CCCH will deliver an advocacy agenda focused on equity, prevention and early intervention. We will have the power to inform the changes needed to redress adversity,” she said.

Annual Report 2021

Celebrating 35 years

The year at a glance Murdoch Children’s Research Institute financials

$111.3m

Research and government grants revenue

$33.2m

Donations, fundraising and bequests

$44.5m

Contract research, clinical trials and others

$33.8m VCGS

$222.8m Total revenue

Murdoch Children’s Research Institute

Statement of profit or loss and other comprehensive income for the year ended December 31, 2021 Consolidated

Company

2021

2020

2021

2020

$’000

180,399

162,424

146,770

130,665

(8,665)

(7,350)

(7,612)

(6,630)

Other expenses for research and clinical activities

(198,692)

(180,389)

(165,988)

(149,174)

Surplus/(deficit) from research and clinical activities

(26,958)

(25,315)

(26,829)

(25,139)

Donation, estates, bequest and fundraising income

33,240

26,834

33,235

26,830

Fundraising expense

(2,066)

(1,710)

(2,066)

(1,710)

31,174

25,124

31,169

25,120

9,157

4,244

8,996

4,052

Operating surplus for the period

13,373

4,053

13,335

4,033

Total comprehensive surplus for the period

13,373

4,053

13,335

4,033

Revenue from research and clinical activities Depreciation and amortisation

Net surplus obtained from fundraising activities

Finance income

Annual Report 2021

Celebrating 35 years

Statement of cash flows For the year ended December 31, 2021 Consolidated

Company

2021

2020

2021

2020

$’000

31,694

30,000

134,207

130,867

124,354

123,376

33,240

26,834

33,235

26,831

17,709

14,617

356

666

320

571

2021

2020

2021

2020

27,694

25,921

35,859

34,295

$’000

(269)

778

(201,218)

(175,430)

(167,984)

(145,961)

Cash and cash equivalents

68,417

63,279

67,817

61,471

25,973

56,567

25,515

54,507

Trade receivables and other assets

17,814

16,019

14,030

12,552

Other investments

67,866

52,444

60,458

44,536

154,097

131,742

142,305

118,559

Trade receivables and other assets

52,317

53,659

52,317

53,659

Other investments

79,054

69,995

73,623

64,667

Property, plant and equipment

13,051

14,036

10,270

11,720

1,513

179

847

179

Total Non-Current Assets

145,935

137,869

137,057

130,225

Total Assets

300,032

269,611

279,362

248,784

111,810

98,190

107,945

93,411

24,846

21,565

17,597

14,983

136,656

119,755

125,542

108,394

Employee benefits

3,565

3,418

2,762

2,669

Total Non-Current Liabilities

3,568

3,421

2,762

2,669

Total Liabilities

140,224

123,176

128,304

111,063

Net Assets

159,808

146,435

151,056

137,721

159,768

146,395

151,056

137,721

159,808

146,435

151,056

137,721

Cash flows from operating activities Patient fees received Government and other grants received Donations received JobKeeper subsidy Interest received Other receipts Advances to (from) related parties Cash paid to suppliers and employees Net cash provided from (used in) operating activities

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

As at December 31, 2021 Consolidated

8,801

3,578

8,676

3,481

(7,673)

(5,235)

(5,487)

(3,471)

(24,482)

(18,020)

(24,877)

(18,132)

(23,354)

(19,677)

(21,689)

(18,122)

Non-Current Assets

Intangibles Net increase/(decrease) in cash and cash equivalents Cash equivalents at January 1 Effect of foreign exchange on opening cash balances Cash equivalents at December 31

Company

Current Assets

Total Current Assets

Cash flows from investing activities Investment income received

Statement of financial position

2,619

36,890

3,826

36,385

63,279

29,318

61,471

28,046

2,519

(2,928)

2,520

(2,960)

Current Liabilities

68,417

63,280

67,817

61,471

Trade and other payables Employee benefits Total Current Liabilities Non-Current Liabilities Trade and other payables

Members’ Funds Accumulated funds Other reserve Total Members’ Funds

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

2021 Board of Directors

Committees Global Advisory Board

Patrick Houlihan

Sarah Murdoch

Miffany Blythe

Dr Brandon Carp

Board Chairman; Audit, Finance and Risk Committee Member (retired February 2021)

Board Co-Chair; Global Advisory Board Chair

Joined May 2021; Australian Development Board Chair; Global Advisory Board Member

VCGS Board Chairman; Translation and Commercialisation Committee Member

Chair: Sarah Murdoch Suzi Carp AO Trent Blacket Miffany Blythe David Calvert-Jones Sarah Harden Steve Hasker Tristen Langley Chelsey Martin Kate Mohr Katie O’Reilly Nicholas Stone Lauren Thurin Australian Development Board Chair: Miffany Blythe (commenced Chair position in April 2021)

Steven Casper

Prof Jane Gunn

The Hon Robert Knowles AO

Translation and Commercialisation Committee Member

Bernadette McDonald Joined October 2021

Vicky Alexiou Anna a’Beckett Trent Blacket Genevieve Brammall Elizabeth Briskin Marcus Freeman Tanya Hamersfeld Vas Katos Clark Kirby Peter Mastos Tamara Lasky Georgia Danos Kate Mohr (previous Chair, retired April 2021)

Kate Mohr

Prof Kathryn North AC

Retired April 2021

Director; VCGS Board Member

Prof John Prins

Paul Rayner Audit, Finance and Risk Committee Chair; Investment Committee Member

Simon Rothery

John Stanway

Investment Committee Chair

Retired July 2021

Enjoy that video at mcri.edu.au/annualreport2019

(retired March 2021)

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

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

Dahlia Sable

(retired February 2021)

Andrew Wilson

Audit, Finance and Risk Committee Chair: Paul Rayner Stuart Boxer Elise Elliot David Gillespie Patrick Houlihan (retired February 2021)

Andrew Wilson Translation and Commercialisation Committee Chair: Geraldine Farrell Dr Brandon Carp Steven Casper Andrew Cox Melinda Griffith David Link Steve Pask Jenny Petering Investment Committee Chair: Simon Rothery Ariane Barker Paul Rayner Adrian Redlich (Deputy Chair)

Craig Dandurand (Joined October 2021)

Council of Ambassadors Prof Kathryn North AC The Honourable Quentin Bryce AD CVO Jeanne Pratt AC Paula Fox AO Janet Calvert-Jones AO Frances Underwood Jean Miller Marketing Council Chair: Patrick Houlihan (retired February 2021)

Chair: Ian Rowden Joanne Smith Martin Ward

Audit, Finance and Risk Committee Member

Prof Shitij Kapur retired February 2021 62

Murdoch Children’s Research Institute

Annual Report 2021

Celebrating 35 years

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 cuttingedge 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.

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

Annual Report 2021

Articles inside

Mental health program expands

Helping vulnerable infants thrive

A brief history of MCRI

Advances in infectious disease

Reducing children’s allergies

Newborn vaccine set to save lives

Cracking the genetic code

The heart of the matter

Investing in stem cell medicine

Leading the way on COVID-19