2018 MCRI Annual Report

A N N U A L

R E P O R T

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MURDOCH CHILDREN’S RESEARCH INSTITUTE

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

ANNUAL REPORT 2018

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contents

8 Our Chairman

9 Our Director

12 Modelling a brighter future

18 Pushing the envelope for the sickest of children

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10 MCRI Board of Directors

14 A vital dose of ‘what if?’

20 A population-wide approach to health’s biggest questions

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16 Creating a system of care for child mental health

22 Helping the world’s children through the power of partnership

The Diagnosis and Acute Management of Childhood Stroke

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CLINICAL GUIDELINE 2017

Our people, our institute

28 Melbourne Children's Campus

30 years at the forefront of genetics

30 Research excellence

32 The impact of giving

33 Growing together

Australian Childhood Stroke Advisory Committee

34 Changing the world through giving

40 Year at a glance

35 Strength of partnerships

42 Financials

36 Celebrating with events

38 Thank you

46 Our manifesto

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our beliefs Every child deserves a healthy start to life.

The relentless pursuit of excellence through passion, innovation, integrity & efficiency.

A culture of curiosity and scientific rigour which leads to discovery.

Our job doesn’t end until we make a difference.

We will find a cure.

Research provides hope.

Children are at our heart.

Every child deserves a chance at childhood.

Cooperative team work makes us great.

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our values Support the individual To achieve their best and develop their skills.

Perseverance Pursue answers and embrace challenges with tenacity and resilience.

Generosity of spirit Delight in the success of others.

Creativity and innovation Curious, imaginative and open to discover new knowledge.

Scientific integrity Honest and open in our work and how we deal with others.

Relationships and collaboration With partners, community, patients, families and teams – everyone has a part to play.

Courage To be honest and seek the truth no matter what.

To be leaders and first adopters, to think outside the box.

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our chairman 2018 has seen MCRI continue to honour its visionary founders as we make strides on the world stage and open doors to future success.

It is an honour to acknowledge another remarkable year for MCRI. To me, the presentation of our annual report is a moment to pause, reflect and celebrate, not only on the transformative work that we do here but the dedication, passion and commitment of so many brilliant minds.

Since our inception over 30 years ago, the achievements of this Institute have led the world in child health medical research and, more importantly, have helped to ensure the future health and wellbeing of all children everywhere.

MCRI exists today thanks to the foresight and visionary leadership of Professor David Danks, Dame Elisabeth Murdoch and the Brockhoff and Miller families, who were committed to creating a world where all children could live life to their full potential.

Today, under the direction of Professor Kathryn North AC, and with the backing of our strong partners, our wonderful family of philanthropic supporters, and our brilliant and passionate researchers and staff, we continue to honour that vision with purpose, compassion and empathy. And it’s not just me who sees it.

One of the highlights of 2018 was the findings of an independent International Review.

The Review evaluated our research position, identified our areas of strength and focus (of which there are many), and is now laying the framework for our next five-year strategic plan – Towards Precision Child Health.

The results, I am delighted to announce, reaffirm our innovation, scientific integrity and 8

global standing. MCRI is not only the number one medical research institute in Australia, it is ranked equal second for research quality and impact amongst the most prestigious child health institutes in the world. This is a breathtaking achievement and one that can only be attributed to the enormity of the work by Kathryn and her team.

The Review recognised the importance of our relationships with our campus partners, namely The Royal Children’s Hospital, the University of Melbourne Department of Paediatrics, and The Royal Children’s Hospital Foundation.

Collaboration has long been an integral part of what we do here, and there’s no better example than the 2018 launch of Melbourne Children’s Global Health. I’m very proud of the deep respect and commitment we share with our campus partners, and the achievements these relationships foster.

The power of partnerships and philanthropy is crucial for us to continue to drive and create change in the area of children’s health. In this light, the launch of Global Friends of MCRI has even more importance. In a series of events hosted this year in New York and Los Angeles we were able to connect with a new group of supporters and I’m very much looking forward to keeping you updated on our vision for this group.

This year MCRI also hosted esteemed American philanthropist, Mike Milken, who Fortune magazine dubbed ‘the man who changed medicine’ for his work to revolutionise the way medical research is funded. It was an honour to meet and share our work with him.

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We really are entering a new stage in our history. One marked by international leadership, collaboration, bold ambition and the overwhelming desire to have a positive impact in the lives of children and families in all parts of the world.

Our global influence can be evidenced by our record success in competitive grant funding and the many awards received by the dedication of those working in our labs and out in the field. Congratulations. Your achievements truly are humbling.

At MCRI, children are at the heart of everything we do. This sentiment is true for our entire team, regardless of role, so in ending I would like to thank a group of people who may not work directly with children, but who make the great work we do possible. Thank you to our incredibly supportive Board (it is a privilege to work alongside you), the MCRI team, our magnificent donors and friends, and of course, our wonderful Council of Ambassadors. A heartfelt thanks for your generosity and unwavering commitment.

If there’s one thing my time at MCRI has shown me, it is the difference a group of individuals can have. Our home might be in Melbourne, but our reach is truly global.

Suzi Carp Chairman

our director I am proud to lead an organisation brimming with outstanding individuals who are driven to challenge the status quo and push boundaries to change the face of child health in Australia and around the world.

Through creativity, innovation, scientific integrity, courage and perseverance we strive for a world in which all children have the opportunity to live a healthy and fulfilled life. The impact of our work ripples from our world-leading Institute, across Australia and all around the globe.

The stories presented in our 2018 Annual Report provide fascinating insights into just some of the ground-breaking work being conducted across our Institute. Our work is making an impact on clinical approaches and on patients. We are changing the game for children's health.

In 2018, our published research included: • A world-first clinical trial in Indonesia giving newborns the RV3-BB vaccine to guard against severe rotavirus gastroenteritis, which results in over 500,000 deaths per year in low and middle income countries • 3D modelling of a child’s kidney cells using stem cell technologies to better understand chronic kidney disease and pave the way for patientspecific treatments •U sing neuroimaging to determine specific brain patterns linked to attention deficit disorder (ADHD) symptoms, brain development and cognition • Identifying epigenetic markers before birth that can predict if a baby will develop cerebral palsy • Determining links between preschool-aged children’s mental health and their ability to learn at school.

There were many, many more. The breadth encompassed by our research is enormous.

Our leaders and researchers – emerging and established – were recognised for their work in a record-breaking number of prestigious awards and distinctions. We take enormous delight in the success of our brilliant minds.

The following pages share the stories of our outstanding 2018 achievements, which include: • The launch of Acute Care Genomics, which is providing faster genetic diagnoses for critically ill babies with unidentified illnesses in under three days! Using genomics, our team is working alongside Australian and international genomic experts to give families rapid answers about their child’s condition, and find the appropriate treatment as quickly as possible.

• The work of our Biomedical Ethics Research Group, whose role brings a “what if” lens to examine all possible impacts of our cuttingedge work in genomics, stem cells and other novel research areas.

• Our new Drug Discovery Facility in the Stem Cell Medicine Group, which enables us to partner with industry leaders to seed the discoveries for new treatments and therapies, and further enhance our work in translation and commercialisation.

• The launch of Melbourne Children’s Global Health, working with our Campus partners in The Royal Children’s Hospital and the University of Melbourne, to reduce inequity and improve child and adolescent health in disadvantaged populations globally.

While this Annual Report is an opportunity to reflect and celebrate how far we’ve come,

MCRI continues to set its sights on the health challenges the world’s children still face.

A baby born in Australia today will have a 1 in 10 chance of developing a food allergy; 1 in 5 children in disadvantaged communities experience significant learning and developmental delays by the time they start school; 1 in 4 children and adolescents are diagnosed with mental health issues such as depression and anxiety; and 1 in 3 are overweight or obese.

New approaches to these complex conditions are needed; approaches that bring together the depth and breadth of disciplines within MCRI in a dynamic way. As we do this, we are leading the way to establish precision child health in Australia, not only to better treat disease, but to diagnose it quickly and even to prevent it from ever occurring.

Every new discovery brings us a step closer to our purpose - a world in which every child has the opportunity to live a healthy and fulfilled life.

It is only through your generous support that we can continue to succeed in changing lives and bringing hope to children, families and communities everywhere. Thank you.

We couldn’t do any of this without you.

Professor Kathryn North AC Director ANNUAL REPORT 2018

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MCRI board of directors Our Board of Directors brings a diverse range of experience and expertise across business and finance, health and research.

The Board of Directors independently and objectively guides and challenges the strategic direction of the Institute.

The MCRI Board meets six times per year.

Kathryn North AC

Suzi Carp

Chairman Fundraising Sub-Committee Co Chair

Director

Steven Casper Fundraising Sub-Committee Co Chair

John Stanway

Cheryl Jones

Thank you Cheryl Jones resigned from the Board in 2018.

We sincerely thank her for her service.

Patrick Houlihan

Audit, Finance and Risk Committee member Marketing Council Chair

Kate Mohr

Development Board Chair

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Shitij Kapur

MCRI committees and sub-committees Paul Rayner

Audit, Finance and Risk Committee Chair Investment Committee member

Audit, Finance and Risk Committee Paul Rayner - Chair

Bruce Grey

Lachlan Armstrong

Patrick Houlihan

David Gillespie

Development Board

Hon Rob Knowles AO

Kate Mohr – Chair

Marcus Freeman

Miffany Blythe – Deputy Chair

Tanya Hamersfeld

Vicky Alexiou

Clark Kirby

Anna A'Beckett

Marisa Leone

Genevieve Brammall

Peter Mastos

Elisabeth Briskin

Dahlia Sable

Vas Katos

Andrew Cox

Simon Rothery

Investment Committee Chair

Fundraising Sub-Committee Suzi Carp and Steven Casper – Co Chairs

Andrew Cox Jean Miller Kate Mohr

Anna A’Beckett Ariane Barker

Investment Committee Sarah Murdoch Marketing Council member

Simon Rothery - Chair

Paul Rayner

Ariane Barker

Adrian Redlich

Bradley Davis

Marketing Council

Bruce Grey

Thank you Bruce resigned from the MCRI Board in 2018.

We sincerely thank him for his service.

Patrick Houlihan – Chair

Ian Rowden

Sarah Murdoch

Joanne Smith

Tony Thomas

Martin Ward

Translation and Commercialisation Committee Bruce Grey - Chair

Andrew Cox

Katie Allen

Geraldine Farrell

Damien Bruno

David Link

Brandon Carp

Jenny Petering

Dominic Stevens

VCGS Board Bruce Grey – Chair

Kathryn North

Brandon Carp

Andrew Sinclair

John Dakin

John Zelcer

David Gillespie ANNUAL REPORT 2018

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modelling a brighter future Imagine a world where cells from a sick child are used to model their disease, better understand the cause of their illness and develop customised treatments or replacement organs to aid their recovery. This is the vision of MCRI’s world-leading Stem Cell Medicine research program, with 2018 bringing the potential closer to reality.

a patient’s stem cells to better understand their disease and find the most appropriate drugs for treatment,” Prof Little says.

Disease models created from a patient’s own cells are already helping researchers better understand conditions and develop more precise treatments. But MCRI’s stem cell researchers are working towards a day when tissue that has been gene edited to remove the disease can be returned to the patient as a potential cure.

Professor Melissa Little

MCRI’s stem cell experts broke new ground in 2018, spearheading research in which stem cells derived from a child with genetic kidney disease were grown into two sets of living mini-kidneys; one with the young patient’s kidney disease and another which corrected the gene mutation.

The discovery, described by the University of Melbourne as “mind-blowing”, showed the unparalleled value of modelling a patient’s own tissue from stem cells, compared against traditional mouse models of disease.

Led by MCRI Theme Director of Cell Biology, Professor Melissa Little, the group demonstrated that gene editing could correct the mutation and stop the disease developing in the mini-kidney, also known as an organoid.

“Following this result, we now want to understand how we can use models made from

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“Today, we can use our models to better understand a disease and develop appropriate treatments, but our long-term goal is to develop a tissue or a cell type that can ultimately go back into the patient,” Prof Little says.

But it is a slow and laborious task creating enough identical organ models, one at a time, to meet the needs of research teams.

“We want to use these models to develop new drugs for diseases, so we need to be able to create them at scale and exactly the same as each other,” Prof Little says.

A new facility at MCRI will speed up the production line, allowing for increased scale and precision to test new medications and deliver tailor-made treatments.

Heading up the new Disease Modelling and Drug Discovery Facility is Dr Alejandro Hidalgo.

He brings significant experience across research and drug discovery within a major international pharmaceutical company.

“With researcher clinicians working across MCRI and The Royal Children’s Hospital we have the invaluable opportunity to work with patients in

MURDOCH CHILDREN’S RESEARCH INSTITUTE

the clinic and then apply that real world learning within our stem cell labs,” Dr Hidalgo says.

“Now with the funding support received from the Stafford Fox Medical Research Foundation we are building and equipping those facilities to an industry-leading world standard.”

The facility will centralise and enhance MCRI’s ability to use models across the eight focus disease areas of kidney, heart, blood, immune system, brain, muscle, reproductive development, and bone and cartilage disorders.

Prof Little says this significant facility investment and new research talent have underpinned the stem cell program’s rapid growth in 2018.

“We now have experts working across many of the body’s organs and tissues, making us the largest group of researchers in Australia generating stem cells from a patient’s blood or skin cells.”

As well as precision cell therapies and drug discovery, MCRI’s Stem Cell Medicine teams are also working towards organ regeneration and replacement to address the critical shortage of donor organs.

For kidney disease alone the implications are far-reaching, with more than 4000 Australians diagnosed with chronic kidney disease annually and a six per cent increase in cases each year.

In childhood kidney disease, around half is inherited. The responsible mutation is often unknown, which significantly limits effective treatment options.

“ Investment in technology to advance our knowledge and build our research efficiency is now helping us achieve our vision of a future healthcare system that integrates evidence-based, stem cellderived treatments and possible cures..”

Professor Melissa Little

Changing the game for children's kidneys Twice a week, Charlotte Matthew’s family would take her on a four-hour round trip for dialysis.

The Wonthaggi girl, 4, who has congenital nephrotic syndrome, had one of her kidneys removed soon after birth and, as the other kidney failed late last year, she relied on dialysis to keep her alive.

Kidneys filter waste products from the blood which are then excreted in urine.

They also regulate what the body should retain. But faulty filtering resulting from Charlotte’s condition caused her to lose too many important proteins, hormones and vitamins.

Everything changed when she received a transplant at The Royal Children’s Hospital.

Dr Cathy Quinlan, from MCRI, says Charlotte is now a healthy young girl who does cartwheels and handstands in the hospital hallways when coming in for check-ups.

Dr Quinlan says what makes this even more inspiring is that a blood sample taken from Charlotte will go on to help other children with her condition.

Research led by MCRI used stem cells from Charlotte’s blood to replicate her glomerulus, the damaged part of her kidney’s waste-filtration system.

Current treatments for chronically diseased and failing kidneys include dialysis or transplantation.

But with only one in four renal failure patients receiving a transplant, there is an urgent need to develop new therapies.

colours, the researchers could see blood flow through the filtration units of the human kidney organoid, revealing the connections between the blood vessels of the mouse and the human kidney tissue.

Dr Quinlan says the development is an important step towards personalised medicine for both rare kidney conditions and the most common type of adult kidney failure, diabetic nephropathy.

In an important proof-of-concept finding published in 2018, MCRI, the University of Melbourne and Leiden University Medical Centre researchers transplanted a stem-cell derived organoid under the protective layer surrounding the kidney of a living mouse.

Four weeks after transplantation, the kidney tubules and blood vessels showed evidence of fully developed adult kidney tissue.

“The idea in studying children like Charlotte, and using models of her kidney disease, is that we start moving towards finding cures,” she says. “We can also use this model to try to find drugs that will make a huge difference for adults who have milder forms of disease.”

By using gene-edited stem cell lines of different

“There is a long way to go to make the tissue large enough for treatment, but knowing that it will begin to function is an important step along the way,” Prof Little says.

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a vital dose of ‘what if?’

Among childhood’s milestones are the ubiquitous and perplexing queries of ‘what if?’ Children can ask big questions that often have adults scratching their heads and rethinking their own points of view. It is a vital role that need not end in childhood, according to the equally rigorous minds of MCRI’s Biomedical Ethics Research Group.

If parents have the chance to spare their child from inheriting a chronic illness they would take it.

But what if it meant using another person’s DNA to make it possible.

MCRI’s Biomedical Ethics Research Group knows better than most that when it comes to ethics it’s no longer black and white.

Group Leader Professor Julian Savulescu says every new medical development or technology brings a raft of possibilities with potential for positive and adverse effects.

“Ethics is often like a brake, slowing down the impulsive or harmful use of scientific advance. But sometimes it should be the accelerator, removing obstructions to science and clinical care,” he says.

“Increasingly, ethics is no longer black and white; it is black, white and grey. The concepts and principles that were developed for the 20th century are no longer fit for purpose.”

“Science can tell us how we can do something, but it cannot tell us whether we should do it.

Our goals, values and what ultimately matters are questions of ethics, not science,” Prof Savulescu says.

“Sometimes people are overconfident that some benign or beneficial intervention is wrong. At other times, they are underconfident that some beneficial intervention is right.

“To unnecessarily delay the development of a cure for a disease which kills 1000 patients a year is to be responsible for the deaths of those 1000 patients, even if you never see them. Sometimes obstruction is as wrong as deliberately harming.”

In 2018, the Biomedical Ethics Research Group contributed to an Australian Senate inquiry into whether to allow mitochondrial replacement therapy (MRT), a new type of genetic modification.

The Biomedical Ethics Research Group continues to ask ‘what if?’; raising questions and contributing to discussion and debate on issues ranging from gene editing and brain organoid development, to moral distribution of medical resources and the ethics around human enhancement technologies.

Mitochondrial disease - a disorder that occurs when structures that produce energy for a cell malfunction – can cause muscle weakness, seizures and stroke. About 1 in 4000 people has mitochondrial disease. It’s progressive and there is no cure.

The group has evolved alongside the rise of ground-breaking new research in areas such as stem cells and genomics, applying expertise in philosophy, social science, medicine, genetics, law and public policy to concepts too complex for science to handle alone.

When a couple carries genes with a mitochondrial mutation - usually without showing any symptoms – those genes can be passed on to their child.

And though their work is steeped in evidence-based science, there are often no clear-cut answers.

Prof Savulescu says work in genomics and stem cells brings both power and responsibility.

MRT is an experimental technique that replaces the mother’s damaged mitochondria with that of a healthy donor. While the technique can prevent mitochondrial disease, it means the child will inherit genetic material from three individuals.

Beyond the ethical questions raised about involving a third parent in the conception of a child, there is also the issue of whether MRT counts as genetic manipulation. Most countries including

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Australia have laws prohibiting DNA editing in early embryos out of concerns it could create a slippery slope to a designer baby scenario.

“We cannot avoid difficult ethical questions, but practical ethics gives us tools to use our power responsibly. We’ve failed to do that in the past, but I think we have also made progress in a number of areas,” he says.

“The way we meet the ethical challenges now posed by genomic and stem cell technologies will have global and inter-generational implications.”

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“ Work in genomics and stem cells brings great power. But with power comes responsibility,” says Prof Savalescu.

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creating a system of care for child mental health MCRI researchers have identified a growing urgency to address child mental health and are working with our partners to establish how community-based health services can best tackle the issue in its earliest stage.

Growing community awareness of mental health issues is a welcome development; but when it comes to children, care and treatment developed for adults simply doesn't fit the bill.

With a wealth of research to back their case, MCRI’s child mental health experts are working hard to advocate for addressing the issue early and in an age-appropriate manner.

MCRI Group Leader of Health Services Professor Harriet Hiscock says adult treatment services had been traditionally prioritised, overlooking the individual, social and economic benefits of targeting child mental health.

“Recent investments in adolescent mental health have enabled progress for those aged over 12, but there is increasing evidence that a comprehensive and sustainable approach to prevention and early intervention needs to focus on infants and children under 12,” Prof Hiscock says.

“Evidence is mounting that a person’s mental health is influenced by potentially modifiable early life experiences and despite the availability of effective treatments, our current service system is unable to meet the need, with many children receiving substandard care or missing out on support altogether.”

The need is clearly seen in the long hospital outpatient waiting lists for children with developmental and behavioural issues, and in the national audit that found six of the top 10 diagnoses of children seeing paediatricians were mental health-related.

In addition to an insufficient service system, other barriers to effective care include stigma

" ...there is increasing evidence that a comprehensive and sustainable approach to prevention and early intervention needs to focus on infants and children under 12."

Professor Harriet Hiscock

It is this eye towards challenging the status quo and pushing for a multidisciplinary, community-based approach to child mental health that has led to MCRI’s role in the establishment of the Centre for Research Excellence in Childhood Adversity and Mental Health.

The $2.49 million CRE, funded by the NHMRC and Beyondblue, is aimed at creating an equitable system of care to tackle child mental health at a grassroots level.

Professor Harriet Hiscock

around mental health diagnoses; parents, teachers and other caregivers not knowing the signs to watch for or where to go for help; lack of clear and timely referral pathways; financial burden; and community concerns around the lack of programs designed to detect emerging mental health issues in young children.

But with half of all adult mental health conditions evident by age 14, it makes sense to reinforce childhood as an important opportunity for prevention and early intervention through a population health approach.

MCRI Theme Director of Population Heath and Co-Group Leader of Child Health Policy and Equity Professor Sharon Goldfeld says mental health and education have traditionally remained separate.

Yet her 2018 research of 302,000 Australian children in their first year of primary school showed an incontrovertible link between mental health and learning outcomes.

“Promoting early school success is easier and more cost effective than trying to fix a child’s difficulties in the later years of their schooling,” Prof Goldfeld says.

“Ideally there needs to be an integrated approach to helping any child with mental health difficulties, while also promoting competence – such as confidence and social participation – for all children within the school.”

“If you’ve experienced some form of adversity in childhood – which means everything from poverty through to domestic violence, housing or food insecurity, or drug, alcohol or gambling problems in the family – you are four to six times more likely to end up with a mental health issue as you progress into adolescence and adulthood,” Prof Hiscock says.

Under the five-year CRE program, teams of researchers will examine the link between childhood adversity and mental health, examine existing services, analyse evidencebased best practice and develop and pilot health, education and social care service hubs in Wyndham (Victoria) and Marrickville (NSW) to address the gap.

Professor Sharon Goldfeld says the CRE aims to find solutions within existing community systems to help with the earlier identification of mental health issues and to provide further support when needed.

“The CRE will review community-based services such as antenatal care, maternal child health and early childhood education, asking questions such as ‘what might happen if we co-locate them’, or ‘how do we get them talking to each other’ and ‘how do we better deliver what we already fund and provide?”

Prof Goldfeld says.

“If we can show a service works, and it engages families, better detects adversity and delivers evidence-based care to reduce the effects of adversity, we will then think about how best to sustain it and how to spread to other locations and perhaps apply the model to other areas of the health system.”

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pushing the envelope for the sickest of children The search for answers to rare diseases often results in diagnostic delays of months and even years; time that the sickest children in neonatal or paediatric intensive care may not have. But a transformative study is changing the diagnostic landscape.

The power of genomics Vomiting uncontrollably, tiny Maelle was rushed into emergency by her parents.

Tests showed that her kidneys were failing.

To keep her alive doctors put her on dialysis while they planned for a transplant.

To determine the reason behind her sudden kidney failure, Maelle was enrolled in the Acute Care Genomics study.

The laboratory team at VCGS worked around the clock to provide a result within 72 hours.

Maelle's diagnosis prompted a complete change in treatment plan. Without this, she would have become much sicker, with risk of blindness, brain damage and broken bones.

But now, her treating doctor Dr Cathy Quinlan, says that "this little girl will go to kindergarten, she’ll paint pictures.

“Developmentally she is like any other child – no brain damage, no blindness, healthy bones.”

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“ With these young patients and their families as our core focus, this program is laying the foundations to transform the diagnostic journey for all Australians affected by rare disease.”

Professor John Christodoulou

Exponential advances in genomics research and technology are not only enabling scientists and clinicians to unravel the mystery behind rare diseases, but equipping them to do so faster than ever, resulting in life-changing answers for families.

In January 2018, MCRI joined forces with hospitals, research centres and genetic pathology services across Victoria, NSW, Queensland and South Australia to push the envelope for the nation’s sickest children through the Acute Care Genomics Flagship study.

The study – a national collaboration led by MCRI that focussed on implementing genomics into healthcare – was set up to provide ultra-rapid genomic testing for about 250 babies and children in intensive care units across Australia.

Director of MCRI’s Genomic Medicine Theme and Australian Genomics co-lead Professor John Christodoulou says the study is harnessing the collaborative power of clinical and research genetics experts, together with a network of genetic curators, bioinformaticians and genetic counsellors working around the clock to find answers.

“We knew that in instances where rapid diagnosis was required, we could deliver an answer with genomic tests in three to four weeks,” Prof Christodoulou says.

“But we really wanted to take that a notch higher and see if we could deliver results, to the sickest of children, in under a week.”

MCRI Director Professor Kathryn North AC says that while individual diseases may be rare – affecting as few as five people worldwide – collectively they are more common than people might realise.

They affect one in 17 Australians, and account for a third of deaths in children aged less than 12 months.

“Genomics happens in a space where collaboration is essential to delivery,” says Prof North, who is also on the executive of the international advocacy and standards group Global Alliance for Genomics and Health. “While MCRI is at the forefront of genomics delivery, we couldn’t find the answers without our national and international networks.”

In the study, DNA samples collected from infants and children at 18 neonatal and paediatric intensive care units in Australia are sent for genomic analysis at Genome.One in NSW or to Victorian Clinical Genetics Services (see page 26) for rapid testing.

At each stage of the process – from the DNA preparation and genomic sequencing, to bioinformatic analysis, diagnosis, return of results and counselling – teams at both centres

work to streamline processes and technological capabilities, returning results at a speed that is unprecedented in Australia.

Study co-lead Dr Sebastian Lunke of the Victorian Clinical Genetics Service (a wholly owned subsidiary of MCRI) Translational Genomics Unit says the flagship project is about genomic technology fulfilling its promise.

“We’re pushing the limits of what we thought possible and changing practice in real time,” Dr Lunke says.

“We want to be able to say to clinicians in intensive care units that we can help them piece together what’s going on, and we can do it quickly.”

The success of the team was evident by Christmas, with the Acute Care Genomics Flagship study achieving its first milestone of providing rapid genomic testing to more than 100 critically ill children, delivering their results within days.

“What has really been exciting is that we’ve been able to get an answer for more than 50 of the children tested by finding they had a genetic basis for their disorder and identifying the genetic cause on average within three days,” Prof Christodoulou says.

“These findings then led to an active change in treatment for more than 35 of the children, which in some cases was quite dramatic.”

Associate Professor Zornitza Stark, who is based at VCGS and is also a study lead, says while providing results with such rapid turnaround times carries a relatively high cost, the benefits – both short and long-term – make it cost-effective.

“When used early in the diagnostic journey it helps inform clinical decisions, avoiding unnecessary, invasive and expensive investigations and providing the opportunity for early treatment,” A/Prof Stark says.

“Having a diagnosis for their child’s illness also gives young families confidence and information when planning to have more children.”

She says the technology can also provide ongoing benefit with genetic data stored and re-examined as more gene discoveries are made over time. All of this information is invaluable as families navigate the complicated and fraught journey towards diagnosis.

“While the results so far have been promising and there have been massive achievements, the fact is that we still can’t find an answer for half of these children,” says Prof Christodoulou.

“With these young patients and their families as our core focus, this program is laying the foundations to transform the diagnostic journey for all Australians affected by rare disease.”

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a population-wide approach to health’s biggest questions

Data collected at the checkpoint between childhood and adolescence is helping researchers understand how to set kids up for the healthiest possible life.

Many of the diseases and illnesses affecting Australian adults have their roots in childhood, presenting an opportunity to stamp them out before they take hold.

Paving the way to healthy adulthood requires in-depth knowledge of the links between the physical, environmental and familial factors in childhood.

Research that looks at the relationships between physical, environmental and genetic factors and health outcomes, is known as lifecourse research.

MCRI is at the forefront of this lifecourse approach, with the richness of their Child Health CheckPoint data already helping 20

researchers and policymakers around the world to plan new studies and programs to help children and families grow well.

“The aim of the initiative was to learn more about the health of young people as they pass through the ‘checkpoint’ between childhood and teenage years, as it is expressed in their developing body and brain,” says Child Health CheckPoint study lead Professor Melissa Wake.

“CheckPoint allowed us to collect an amazing wealth of different data spanning all of the body’s physical and biological systems.”

Like piecing together a puzzle, Child Health CheckPoint collected data to create a snapshot of what child health looks like in Australia, and what adult health could look like. The project was a significant addition to Australia’s only large-scale child health and wellbeing study, Growing Up in Australia, which has tracked the development of 10,000 young people and their families since 2004.

With funding from the NHMRC, the Child Health CheckPoint team ran a series of pop-up assessment centres In 2015-2016 across 30

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Professor Melissa Wake

Australian towns and cities. They invited 1800 of the 11 and 12 year-old kids from the Growing Up in Australia cohort, together with a parent, to take part in the three-and-a-half hour sessions.

Through 17 different assessment stations, researchers collected details spanning physical activity and fitness, body composition, bone health, nutrition, sleep, vision, hearing,

“ We started to see a huge range of differences in children, in areas as simple as blood pressure and cholesterol, and not surprisingly we started to see them resemble their parents in some of these measures.”

Professor Melissa Wake

language, dental, cardiovascular, kidney and respiratory health. By collecting parent data, the researchers also created a unique crossgenerational resource at a moment in time.

The rich biological samples collected and measurements taken will also enable future study involving genetics, inflammation, metabolic and micronutrient health, ageing and many other aspects of biologic health.

Prof Wake says the great strength of longitudinal studies is the opportunity they present for measuring a range of factors in many different ways.

“By putting them all together we can reveal rich secrets and answer many different questions.”

“We started to see a huge range of differences in children, in areas as simple as blood pressure and cholesterol, and not surprisingly we started to see them resemble their parents in some of these measures,” Prof Wake says.

“One of the most surprising results was how strikingly children resemble their parents on bone strength – far more than on other

characteristics. Hearing was also linked between children and parents – and there is early evidence that even in childhood, poorer hearing could be related to factors such as obesity and inflammation.” She says this would make it another lifecourse condition, rather than an unavoidable disease of old age.

“Early adolescence is a time when there is a chance to make a real difference for a child’s health,” Dr Clifford says.

While most developed countries have some form of longitudinal study of children based on interviews and surveys, the millions of data points gathered in CheckPoint’s in-depth health assessments and biological collection is unique.

The Child Health CheckPoint has also delivered important lessons for the researchers in Prof Wake’s team who will soon embark on GenV, which received a $14 million Victorian Government commitment in late 2018 to help progress the project to its next stage.

When this large dataset was made available to researchers worldwide in 2018, they leapt at the chance to mine it for better understanding of how health develops in children and adults.

Almost 100 researchers and policymakers have already accessed the data for their own studies.

CheckPoint study project manager Dr Susan Clifford says that by making the information available to other researchers, MCRI is taking the lead in the area of Open Science; a movement that aims for faster and more transparent research, leading to faster evidence-based outcomes.

“By sharing the dataset, it means that CheckPoint could one day help to design interventions that can guide kids back on to a healthy path.”

“Through CheckPoint we found that children and parents really enjoyed the chance to be part of a comprehensive project that was creating knowledge to help future children and their families,” Prof Wake says.

“In time, Gen V will become one of the world’s largest health studies, following the lives of 160,000 babies born in Victoria in 2020 and 2021. It will fast-track solutions to the complex problems facing today’s children and tomorrow’s adults.”

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helping the world’s children through the power of partnership MCRI works to ensure the health and wellbeing of the world’s children and the adults they will become; a mission that’s advancing with the recent creation of the Melbourne Children’s Global Health initiative.

One of the first Indonesian babies to receive the new vaccine

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Back in 1973, MCRI made its first steps into the complex and diverse area of global child health when a collaboration – led by MCRI’s Professor Ruth Bishop – discovered rotavirus. Since that time, global child health has come to be a major priority for the Institute, as signalled through the launch of Melbourne Children’s Global Health (MCGH) in 2018.

The Melbourne Children’s Global Health initiative brings together partners MCRI, The Royal Children’s Hospital and the University of Melbourne to work with resource-poor countries on improving child and adolescent health through research, public health, education and advocacy.

Melbourne Children’s Global Health Co-Chair Professor Andrew Steer describes the initiative as a brilliant example of the whole being greater than the sum of the parts.

“This is an exciting time for all of our people to make a tremendous difference to the lives of children and young people around the world,” Prof Steer says.

He says the collaboration will also help secure research funding, strengthen their collective standing at international meetings and enable researchers to achieve greater impact through shared information and resources.

The Melbourne Children’s Global Health initiative will harness the research and clinical power of the group, which has already achieved lifechanging results.

“Solving the many problems of global health requires such close collaborations and partnerships in order to be truly effective.”

The rotavirus work that began decades ago, and involved The Royal Children’s Hospital and the University of Melbourne, is one example of how collaboration and partnership has always been a part of MCRI’s global health story. While rotavirus is now a low-impact disease for Australian babies, that is not the case for babies elsewhere.

In low-resource countries, rotavirus still kills more than 215,000 children aged under 5 annually and sends millions more to hospital. Responding to that urgent need, MCRI developed a new vaccine that could be given to babies safely within days of birth. A vaccine that’s safe for newborns addresses early-life risk, and ensures babies from remote areas are protected from the point where they have what may be a rare contact with the health system.

The trial of 1649 Indonesian babies showed the vaccine to be well-tolerated and protective for up to 18 months. It is expected to be available by 2022, potentially saving millions of babies from life-threatening diarrhoea.

Rotavirus was just one of three vaccines that were introduced in Fiji in 2018 following MCRI research. Pneumococcal conjugate, human

Some of the 500 midwives involved in the vaccine trial in Indonesia

papilloma virus (HPV) and rotavirus vaccines protect against of a range of common, and sometimes life-threatening, illnesses.

MCRI researchers continue to evaluate the impact of these vaccines with Fijian partners.

Early results show that pneumococcal vaccine effectively halved the number of children carrying the dangerous bacteria, including in babies too young to be vaccinated.

Pneumococcal bacteria are a common cause of meningitis, blood poisoning and pneumonia, and claim the lives of more than 650,000 children worldwide each year.

With just one round of treatment, researchers from MCRI collaborated to knock out two skin diseases in the entire population (26,000+) of Choiseul Province of the Solomon Islands.

The two skin diseases – scabies and impetigo – currently infect hundreds of millions of people, mostly in tropical countries. This work saw an almost 90 per cent reduction in infection rate a year after treatment.

These projects in Indonesia, Fiji and the Solomon Islands represent just some of the work that led to the powerful collaboration that is Melbourne Children’s Global Health. As MCGH develops, the initiative has a bold vision for the health of children worldwide.

Co-Chair of Melbourne Children’s Global Health Professor Kim Mulholland, whose projects span Fiji, Vietnam, Indonesia and Mongolia, says the initiative will bring together the expertise, knowledge and resources to keep identifying need and delivering health benefits to entire communities, including for local researchers and clinicians.

“There are not many institutions around the world that could put together this sort of breadth of resources focussed on the needs of children in the poorest circumstances,” Prof Mulholland says.

Lessons learned We worked with partners in 12 Nigerian hospitals to introduce a simple change to routine clinical practice, regularly measuring the level of oxygen in sick newborns’ and children’s blood using pulse oximetry. We explored how oximetry was adopted in different hospitals and identified ways to improve the introduction in other settings. We found that training will be more effective if it: • Communicates ‘why’ learners should change practice, not just ‘what’ they should do, and articulates this as a shared vision that learners can ‘own’ • Connects new knowledge and practices with existing knowledge and practices, in an encouraging environment that fosters nonjudgemental self-correction • Is hands-on and enhances problemsolving abilities so that learners can understand the real-world application and address particular challenges in the local context • Includes practical application for learners to apply newly acquired knowledge, including ongoing support to reinforce learning and support the gradual process of change • Involves clinical leaders who will have ongoing influence through supervision, recognising their role, expertise and relationships with other staff • Builds teamwork through joint learning and problem-solving with colleagues • Uses job aids to support learning and act as ongoing reminders in the clinical workspace.

“But that’s exactly what Melbourne Children’s Global Health has just done.”

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Our people, our Institute Growth has featured in all areas of MCRI in 2018; not only in research, but in operations. While the superstars of science may be in the public eye, the Institute works hard to attract, nurture and support all staff and students to foster the next generation of leaders.

Planting a seed MCRI has more than 60 research groups and a significant cohort of research support and operations staff, so new starters at MCRI have a lot to get their heads around.

To help address any early challenges, the onboarding process sees Human Resources personally connect with new starters at the new starter induction, supporting them to make their way in a new environment.

As staff move through their careers at MCRI, mentoring and coaching are there to make a difference. The Institute offers 12 staff annually the opportunity to take part in six 1:1, one-hour coaching sessions. Staff are drawn from all areas of the Institute after nomination from theme directors and endorsement by the Executive.

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Celebrating our diversity The program offers small groups of up to eight staff the opportunity for an Institute mentor and peer mentoring, but small group mentoring has been the powerhouse of the program. There are groups from the first year who are still meeting regularly to draw on one another’s skills and experience.

Almost 100 staff have had the opportunity to go through the mentoring program, and 14 past mentees have returned to the program as mentors.

In 2018, the MCRI Executive approved leadership principles to continue to support the development of quality leadership. Over the coming years, MCRI will roll out a series of leadership programs to address common problem points. Including:

Word about the coaching program’s professional value has spread quickly; it has grown from a small, in-the-know offering to one that staff now actively seek out.

• workshops and a Thrive program to support holistic wellbeing

One of the most popular opportunities for professional development at MCRI is the mentoring program, which has been running for four years and growing in popularity with every year.

• formal and informal professional development opportunities, such as finance for non-finance managers; having courageous conversations; and presentation skills.

• practical management workshops to develop technical leadership skills

MURDOCH CHILDREN’S RESEARCH INSTITUTE

One of the most rewarding parts of life at MCRI is embracing opportunities to celebrate the diversity of the Institute.

MCRI is proud to have an active Diversity and Inclusion Committee that meets monthly to monitor diversity and equity issues that may impede career progress; develop relevant guidelines and networks; and advocate to improve the experiences of all employees.

This dynamic group of more than 30 members from across the Institute works efficiently through three sections of Society and Culture, Gender Equity and Accessibility.

In 2018, the committee worked on a range of projects and events to strengthen the MCRI community through diversity and inclusion.

Highlights included becoming the first and only Australian medical research institute to be part of the Australian Network on Disability; and developing an internal scheme to support research staff work during times of leave and career disruption, which launched early in 2019.

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Cutting-edge science

Planning for the future

Achieving the research excellence and grant success that has characterised MCRI’s work in 2018, cannot happen without access to sophisticated and specialised technology to increase the speed, efficiency and effectiveness of lab research.

As is clear from the above chart illustrating MCRI’s financial growth in the past decade, growth and diversity have been strong. In that time, many other areas of the Institute have grown and developed too, rising to support not only game-changing science, but innovative operations.

Facilitating access and guiding the use of this equipment is the work of the Scientific Services team. But in 2018, the team believes that two of the most exciting additions to the suite of available resources were not actually machines, but teams of highly skilled specialists.

The Induced Pluripotent Stem Cells derivation core facility and the Gene Editing facility are each home to scientists with specialist expertise. That expertise is then used by labs across the Institute to enhance their own work.

The facilities are also driving upskilling across the Institute, equipping researchers across MCRI with the skills to replicate the specialised work themselves.

The work of the Business Development and Legal Office (BDLO) is an important piece of MCRI’s efforts to create business sustainability now, and into the future.

There are some 450 projects feeding into BDLO’s business development pipeline, with more than 50 currently in development across diagnostics, therapeutics, medical devices and digital health.

Some products, such as the continued development of the rotavirus vaccine, can take decades to fully bring to market; others, such as smartphone apps, can be created more quickly.

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ADJUSTED BY INCOME IN ADVANCE

The Grants team did extraordinary work in 2018 to deliver record National Health and Medical Research Council (NHMRC) grant results. Funding through other grants also continued to grow in 2018, reaching 21 per cent success.

However, to appropriate a phrase, research cannot live on grants alone.

Philanthropy and Engagement, the Victorian Clinical Genetics Service, and the BDLO all play a role in supporting the diversification of funding sources.

In every part of the Institute, the range of partnerships and collaborations in place and in progress, presents further opportunities not only to diversify funding, but to strengthen research outcomes and impact.

MCRI's clinician researchers work with families of sick children and in research.

Their unique perspectives on the gaps in healthcare can lead to opportunities to partner with industry and deliver great health outcomes for our community.

ANNUAL REPORT 2018

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30 years at the forefront of genetics In 1988, while Australia celebrated its bicentenary, Dame Elisabeth Murdoch and Professor David Danks set their sights on the emerging role of genetics in public health when they opened the doors to Victorian Clinical Genetics Services (VCGS).

Having only recently co-founded MCRI, Dame Elisabeth and Prof Danks shared extraordinary foresight. Together, they recognised the impact that genetics could have for child health and worked tirelessly to drive the evolving science in Australia.

Pivotal to this was the creation of Victorian Clinical Genetics Services (VCGS) as a specialist prenatal, childhood and adult genetics service.

It is an Australian not-for-profit subsidiary of MCRI and one of the largest contributors driving genetic healthcare, research and policy in the country.

With investment from the Victorian Department of Health, VCGS has been able to apply the latest advances in genetic technology to improve diagnostic impact; helping sick children and their families in ways that were previously unimaginable.

VCGS Laboratory Director Dr Damien Bruno says the service has grown from testing for a limited range of known genetic conditions, to incorporating technology that can sequence all genes in the search for what's causing disease.

“Over the past five years we have seen extraordinary advances in research and technologies that have enabled us to completely transform the way we analyse and make sense of genomic data,” Dr Bruno says.

“Using genomic information to inform a patient’s diagnosis and clinical care is truly

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Dame Elisabeth Murdoch and Professor David Danks founded VCGS, Australia’s first not-forprofit clinical genetic service

VCGS takes on the Newborn Bloodspot Screening service – over two million Victorian babies have been screened since then

POSSUM database wins the Australian Information Technology Award for Systems Innovation of the Year. POSSUM now hosts information on more than 4000 rare genetic disorders

VCGS begins the genetic counselling service to provide vital support to all patients and their families

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remarkable; it allows doctors to deliver healthcare tailored to the family’s needs”.

In its earliest days, VCGS took on the Newborn Bloodspot Screening program – known as the heel prick or “Guthrie” test – partnering with the Victorian State Government. Developed in the 1960s by Dr Robert Guthrie as a test for phenylketonuria, it now covers 25 different conditions. VCGS has screened more than two million newborns, and saved many lives through quick diagnosis and treatment.

In addition to newborns, VCGS also began applying genetic technologies to prenatal testing, including first and second trimester screening programs used to assess women at increased risk for conditions such as Down syndrome. More recently, VCGS began offering highly advanced genome-wide non-invasive prenatal testing (NIPT), which can detect a broad range of chromosome conditions in early pregnancy.

In the paediatric setting, VCGS also pioneered the implementation of single nucleotide polymorphism (SNP) microarrays. This technique, which uses a child’s blood or saliva sample, has informed diagnosis and treatment for thousands of Australian families.

But this life-saving genetic information can be complex and challenging for families. VCGS quickly recognised the importance of specially trained counsellors to act as translators

between scientists, doctors and patients.

Employing its first official genetic counsellor in 1989, VCGS soon developed an in-house training program that became the foundation for the University of Melbourne’s Master of Genetic Counselling.

Exponential growth in MCRI and VCGS’ genetic knowledge base led to the development of POSSUM; a birth defect database used by researchers and clinicians globally to inform treatment and new discoveries. VCGS Medical Director Professor Martin Delatycki describes POSSUM as “the single-most internationally recognisable output of VCGS.” VCGS has also led the way in reproductive carrier screening for conditions such as cystic fibrosis, spinal muscular atrophy and fragile X syndrome. In its anniversary year, VCGS, through MCRI, was named one of three laboratories nationally to develop a comprehensive genetic testing and patient support model for the $20 million Mackenzie’s Mission: a three-year pilot program that will screen 10,000 couples to identify their chance of having a child with a severe genetic condition.

What was once a manual, labour-intensive process, taking sometimes years to arrive at a result, can now scan an individual’s entire DNA code in just days. The implications for swift diagnosis and clinical decision-making have been game changing.

VCGS pioneered implementation of exome sequencing into clinical healthcare, resulting in hundreds of answers for patients and families with previously undiagnosed conditions.

This has set the stage for Acute Care Genomics, which has brought incredible advances in technology and enables clinicians to get rapid findings about the cause of rare and complex genetic conditions, resulting in timely, targeted treatment for children.

Together with its key partners — MCRI, The Royal Children’s Hospital, The Royal Children’s Hospital Foundation, University of Melbourne, Australian Genomic Health Alliance, Melbourne Genomic Health Alliance and the Australian and Victorian Departments of Health — VCGS continues to stand as a world-leading, not-forprofit provider of cutting-edge genetic services.

Just as it began with a vision of the future, VCGS remains dedicated to delivering the benefits of genomics to acute care and standard clinical practice, so that every child and family can reap the benefits and live their healthiest possible life.

Mackenzie’s Mission is the first project under the Federal Government’s $500 million Genomics Health Futures Mission - part of the Australian Government’s Medical Research Future Fund.

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VCGS makes microarray testing easy with saliva sampling to provide rare disease diagnosis for more than 1000 children each year

The perceptTM test starts, offering Australia’s most advanced noninvasive prenatal test (NIPT)

VCGS pioneers the use of DNA sequencing application and analysis for clinical care

VCGS is a key part of Acute Care Genomics, a study providing rapid genomic results for critically ill babies

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melbourne children's campus news The Melbourne Children's Campus brings together the research power of Murdoch Children's Research Institute, clinical expertise of The Royal Children's Hospital and educational nous of the University of Melbourne. With the support of The Royal Children's Hospital Foundation, the Campus powerhouse creates world-leading impact for children's health.

Making a difference for the world’s poorest children The launch of Melbourne Children’s Global Health has seen Melbourne Children’s Campus partners join forces for the world’s poorest children.

Supported by The Royal Children’s Hospital Foundation, Melbourne Children’s Global Health brings together researchers from MCRI; clinicians from The Royal Children’s Hospital; and educators and students from the University of Melbourne around a common goal.

Together, they will work to ensure the health and wellbeing of the world’s children and the adults they will become.

MCRI Director Professor Kathryn North AC says that working together, the Melbourne Children’s Campus partners can change the world for the better.

“MCRI researchers discovered the rotavirus at The Royal Children’s Hospital using electron microscopes at University of Melbourne, which showed that this one virus was responsible for the deaths of hundreds of thousands of young children,” Prof North says.

“Solving the many problems of global health requires such close collaborations and partnerships in order to be truly effective.”

Head of Melbourne University’s Medical School, Prof John Prins, says many of the MCGH projects focus on supporting health workers in developing countries to improve patient care.

To achieve its vision, MCGH will focus on five key areas:

“Our projects are collaborative,” he says.

2. Entering new research areas such as the health of children caught in humanitarian crises and young people living with disability.

“For example, Melbourne paediatricians work alongside paediatricians in countries in Asia Pacific and Africa designing better training programs for new doctors.”

1. Building on strengths in areas such as vaccine discovery, development and delivery.

3. Growing links with local, national and global partners.

It is also hoped the new initiative will ultimately lead to:

4. Creating career pathways into global child and adolescent health.

• faster development of vaccines and treatments for conditions including rotavirus, pneumonia, meningitis, scabies, trachoma, stomach cancer and rheumatic heart disease

5. Leading the conversation on child and adolescent health around the world.

• trials of new interventions to tackle the growing burden of adolescent mental health issues • more effective ways to detect and manage drug-resistant tuberculosis • helping save lives by improving medical training and facilities in hospitals around the world.

MCRI Board member and Chair of The Royal Children’s Hospital Board, the Hon Rob Knowles AO, says the Melbourne Children’s Campus partners are already having a major impact in the Asia Pacific region.

“This new initiative allows us to leverage the collaboration and innovation we see across the Melbourne Children’s Campus partnership and deliver improved outcomes for children across the globe.”

Melbourne Children’s Global Health (MCGH) will not only help to secure research funding, and enable researchers to achieve greater impact, but will work to support the UN’s Sustainable Development Goals (SDGs).

Equity is a core principle in global health and a central component of the UN’s SDGs. MCGH aims to contribute to the SDGs, notably SDG 3: to achieve good health and wellbeing for all children and adolescents.

Melbourne Children’s Global Health will harness the research and clinical power of Melbourne Children’s Campus partners, a group that has already achieved life-changing results.

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Ms Kathryn Snow, University of Melbourne; Prof Kathryn North AC, MCRI Director; Dr Jarir At Thobari, Gadjah Mada University (Yogyakarta); and Dr Monica Brook, The Royal Children’s Hospital and graduate of the Fiji School of Medicine.

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The Diagnosis and Acute Management of Childhood Stroke

Australia’s first clinical guidelines for children with stroke CLINICAL GUIDELINE 2017

Children will receive faster diagnosis and life-changing treatment with the first clinical guidelines in Australia to help them recover from stroke.

Doctors from MCRI and The Royal Children’s Hospital developed the childhood guidelines to improve paediatric care. Before the guidelines were introduced, children could often wait more than a day for a stroke diagnosis, missing the critical window to reduce brain injury and improve recovery.

MCRI researcher and paediatric neurologist Associate Professor Mark Mackay, who led development of the guidelines, says they aim to create better standards of care for childhood stroke across Australian paediatric hospitals.

“This will help reduce the time to diagnose stroke and ensure all children are provided with the same high-quality evidence-based care,” A/Prof Mackay says.

Stroke is among the top 10 causes of death in children; more common than brain tumours.

In Australia between 100 and 300 Australian babies and children are diagnosed with a stroke each year. More than half of those who survive will have lifelong disabilities affecting their

physical functioning, communication, learning and social behaviour.

“The lifelong burden of stroke is greater for children than adults, because more than half of strokes occur in children under 5. These children, therefore, face decades of living with disability,” A/Prof Mackay says.

“Children face difficulties ever achieving an independent life because the stroke happens while the child’s brain is still developing.”

According to MCRI’s Dr Tanya Medley, who also worked on the guidelines, the causes of stroke in children are very different to adults, requiring changes to typical treatment.

“We believe implementing a standardised approach to diagnosis and management will ensure children are not being left behind in the advances in stroke care that have transformed outcomes for adults,” Dr Medley says.

The new paediatric clinical guidelines, which began implementation in 2018 and have received endorsement from the Stroke Foundation and the Australian and New Zealand Child Neurology Society, will address the need for fast, evidence-based action.

A key recommendation of the guidelines is that rehabilitation be delivered by a team of

health professionals such as occupational therapists, physiotherapists, speech pathologists and dieticians.

Australian Childhood Stroke Advisory Committee

“The guidelines have helped us identify key research questions and develop a national collaborative network,” says A/Prof Mackay, who is also Director of The Royal Children’s Hospital Stroke Program.

“We hope that setting a national research agenda will help us attract much-needed funding to improve our knowledge of the best approaches to treating Australian children who have had a stroke.”

A world-leading partnership for adolescent health the clinical strength of campus partner The Royal Children’s Hospital and the education strength of the University of Melbourne.

Professor George Patton and Professor Susan Sawyer know adolescent health is critical to current and future generations, and work hard to keep it in the centre of policy activity locally and globally.

Since its founding 25 years ago, the Centre has contributed to shifting adolescent health from an obscure subspecialty to the forefront of national and international initiatives.

Last year the world-renowned pair published groundbreaking research outlining how children’s growth and development is affected by the health and lifestyles of their parents, well before those parents decide to have a child.

In Australia, adolescents are now central to mental health and substance abuse policies and, more recently, central to discussions of chronic physical illness policy, including non-communicable disease risks and cancer survival.

The MCRI and University of Melbourne findings, ‘Adolescence and the next generation’, were published in the journal Nature in February 2018.

Lead author of the paper, Prof Patton says while the health system for children currently kicks in from a woman’s first antenatal visit — most often eight to 14 weeks into a pregnancy — engagement needs to happen pre-conception.

“Current policies to promote the best possible start to life in Australia along with most other countries are starting too late,” he says.

“We need it to go beyond its focus on contraception to tackle broader health risks and emotional wellbeing in both young women and men.”

This means adolescence holds greater relevance for human development than ever before, according to paper co-author and passionate advocate for young people Prof Sawyer.

“An extended adolescence creates an opportunity for this generation to acquire greater assets and capabilities,” she said.

“That will make a huge difference not only for themselves but for their children.”

Profs Sawyer and Patton are co-leaders of the Centre for Adolescent Health, which brings together the research strength of MCRI, alongside

At an international level, adolescents have risen to prominence with the Global Strategy for Women’s, Children’s and Adolescents’ Health, a prominent and funded agenda within the UN’s Sustainable Development Goals. In all of these developments, the Centre for Adolescent Health has been a leading player, contributing expert recommendations that have become policy cornerstones.

With its established expertise and reputation in adolescent health, the Centre and the whole Melbourne Campus is committed to taking every opportunity to shape policy and practice for the benefit of young people around the world.

ANNUAL REPORT 2018

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research excellence In 2018, a record number of our leaders and researchers were recipients of prestigious awards and distinctions. Congratulations are due not only to those listed here, but the many MCRI team members whose work was acknowledged as outstanding in 2018.

Our culture attracts and inspires high-performing individuals to do their very best work and achieve new heights. To read about all of our 2018 winners, go to www.mcri.edu.au/researchexcellence

Dr Amanda Gwee

Dr Hamish Graham

Dr Fallon Cook

Gustav Nossal Award

Inaugural CSL Florey Next Generation Award

International Pediatric Sleep Association Young Investigator Award

This award is presented to the highest ranked applicant for an NHMRC Postgraduate Scholarship

This brand-new award went to the Dr Graham as the top PhD candidate in health and biomedical sciences

The award recognises work to increase the knowledge of pediatric sleep problems and their consequences

Australia Day Honours

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Prof Suzanne Garland

Prof Frank Oberklaid

Order of Australia

Order of Australia

Prof Garland received her AO for distinguished service to medicine in the field of clinical microbiology

Prof Oberklaid received his AO for significant service to medicine in the field of clinical paediatrics, child development, and public health policy

MURDOCH CHILDREN’S RESEARCH INSTITUTE

Dr Agnes Bankier Medal of the Order of Australia Dr Bankier received her OAM for her service to medicine as a geneticist and to medical education

Prof Melissa Little

Prof Kathryn North

Prof Kim Mulholland

NHMRC Elizabeth Blackburn Award and highest ranked NHMRC Research Fellowship

Australian Financial Review’s BOSS True Leader of 2018

Robert Austrian Award

These awards recognise Prof Little’s outstanding work as a researcher and as a woman in science

Dr Kiymet Bozaoglu Dr Dan Pellicci

Prof North was named a True Leader in 2018 for her tireless work in genomic medicine and research

Dr Enzo Porrello

The Robert Austrian award recognises Prof Mulholland’s lifesaving and paradigm-changing work in pneumonia

Flack Fellowship

CSL Centenary Fellowship

Metcalf Prize

Prof Sarath Ranganathan

The Fellowship supports Dr Bozaoglu's research into autism spectrum disorder

The $1.25 million fellowship will support Dr Pellicci’s research into unconventional T cells

Dr Porrello’s prize recognises his leadership in stem cell research

Fellow of the American Thoracic Society

Prof Cheryl Jones

Prof David Thorburn Prof John Carlin

The American Thoracic Society focuses on improving care for pulmonary diseases, critical illnesses and sleep-related breathing disorders

Fellows of the Australian Academy of Health and Medical Sciences Fellowship recognises distinguished professional achievement in health and medical research

ANNUAL REPORT 2018

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ENGAGEMENT AND PHILANTHROPY

the impact of giving The funding that comes through philanthropy acts as a seed to allow early research work to develop to a point where the researchers can apply for competitive grants.

In 2018, every dollar raised through philanthropy at MCRI was able to become seven dollars for our research.

The generosity and passion of our donors matches with the goals and ambition of our researchers. Together, the combination can deliver extraordinary outcomes.

Everyone involved in philanthropy at MCRI works to bring together the drive of our researchers with the dedication and commitment of our funders. Together, they make a significant impact to change the face of child health now and for decades — perhaps centuries — to come.

We will remember 2018 as the year that MCRI’s fundraising efforts went global, driven by the efforts of the Development Board.

The Board hosted events in LA and NYC to promote the work of MCRI to US-based philanthropists, laying the foundations for future philanthropic work.

In the following pages, you can read about some of the work that began with seed funding from our donors and went on to great achievements. You will also find the stories

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of some of our dedicated donors who have a desire for their donation to make a difference, now and into the future.

Finally, you will find a long list of some of the many donors who supported MCRI’s work in 2018. The incredible generosity of spirit is always humbling for MCRI’s Development Board, Philanthropy and Engagement team and researchers.

A sincere thank you to the MCRI Board, Development Board members, donors, supporters and volunteers for your ongoing commitment to child health. We could not do this without you.

In 2018, we sadly farewelled two Development Board members: Andrew Cox and Marisa Leone.

Thank you for your contribution.

growing together Ambitious ideas and attention to detail are characteristics shared by MCRI’s stem cell researchers and the couple whose philanthropic foundation helps support them.

It takes bold vision to move stem cell science from the laboratory to clinical use in patients.

But when you are already growing mini kidneys in a dish and sheaths of beating heart tissue that may one day mend holes in the hearts of newborn babies, anything is possible.

MCRI’s ground-breaking stem cell work began in 2013 when a trio of researchers established their laboratory at MCRI. They began these steady advances that are now attracting attention for their innovation and potential to transform medicine.

But the little known factor that is turning this into a reality has been the Stafford Fox Medical Research Foundation (SFMRF). It too was established in 2013 and came after the death of Moyna Fox. James ‘Stafford’ Fox and Moyna married in 1943; a Melbourne couple with a love of the races, fashion, gardening and sports.

In 2014, members of the Stafford Fox team met MCRI's new stem cells research team

Following a career that spanned World War II service, three decades in business, and establishing wide networks, Staf – as he was known to friends — died in 1994. The couple had no children and many wondered what would become of their considerable estate.

Nothing was known of the plans being laid by the pair to set up one of the best endowed medical research funds in Australia.

“I cannot thank the iPSC core and importantly the SFMRF enough. Without their help I would not have progressed the project as much as I have.”

Trustees Paul Brotchie and Ken Wallace, together with their wives Susan and Marian who are equally as passionate about SFMRF and the work it supports, say they are delighted by all they have seen achieved to date.

For MCRI, the SFMRF provided the springboard to initiate stem cell research and support a core workforce of researchers who have generated stem cell lines including heart, kidney, brain and cartilage for researchers at the Institute.

“We’ve seen mini kidneys growing in a dish and sheaths of beating heart tissue that may one day be able to mend holes in the hearts of newborn babies,” they say.

Critically, these core personnel have been able to train other researchers in the art of growing and differentiating cell lines for themselves, almost like an in-house academy.

With the support of the SFMRF, the Institute is progressing rapidly towards its ultimate vision – to move from fundamental stem cell science in the laboratory towards the clinical use of human stem cells in patients.

Senior Research Officer within the neurogenetics laboratory Dr Kiymet Bozaoglu says stem cells from patients are central to her project, which aims to find the genetic causes of autism and then model the condition in a dish using the patient’s own cells.

“The induced pluripotent stem cells (iPSC) core at MCRI, who are supported by Stafford Fox Medical Research funds, provide me with training in how to best care for the cells and different techniques used to culture them,” Dr Bozaoglu says.

“We hope Stafford and Moyna would be as proud and amazed by the work.”

The ability to use human stem cells to create any other cell type opens the prospect of improving patient care in many ways, including patient-specific disease modelling, personalised drug screening, novel cell therapies in patients, and one day, bioengineered organs.

MCRI is incredibly grateful to the SFMRF for helping us realise this vision.

ANNUAL REPORT 2018

33

ENGAGEMENT AND PHILANTHROPY

changing the world through giving Maureen Mcllroy’s gift will one day make a difference to children doing it tough in low‑resource countries.

Maureen has fond memories of growing up.

“I remember the fun and excitement of those days – even though we didn’t have much, we had great fun together, playing with all the children along the street.”

It was while travelling in her early 20s that Maureen realised that not all kids get to enjoy a childhood like hers. “I saw kids who, just because of where they were born, weren’t able to enjoy that sort of carefree and fun childhood.”

Maureen is still an avid traveller and, while planning a recent overseas trip, decided it was also time to get her Will up to date.

“Not long before then I had been on a visit to the Murdoch Children’s Research Institute. I’d heard from these brilliant researchers about the work they are doing here in Melbourne that’s making such a difference around the world. It made me think – if there’s anything left after I’ve remembered my family, why not put it to good use?”

Her memories of seeing children around the world growing up in tough conditions meant that she found inspiration in MCRI’s work to change outcomes in global health. Maureen discussed her idea with her nieces who agreed that it was a ‘very worthy’ decision and from there it was easy to arrange with her solicitor.

A gift from Charles Tegner’s Foundation made a huge impact, but his generosity did not stop there.

“You have to work out your purpose and vision, but one thing we all agreed was that all donations must be given joyfully.”

Over 14 years, Charles and his two co-directors supported many deserving causes who shared their vision and values.

In 2017, Charles decided that he’d like to wrap up the Foundation by the end of June 2018.

This decision meant the Foundation could make significantly larger donations to really make a difference.

“We wanted to enable recipients to immediately move ahead into future years of research, operations and other projects on their important wishlists,” he explains.

“The team at MCRI presented us with several options and from there it was easy 34

“I hope that my gift will one day make a difference for future generations. But not for a while just yet!”

with brain disorders. I thought it was a fantastic idea, so we funded that as well,” Charles says.

Brain disorders are a common diagnosis at The Royal Children’s Hospital. A single mutation in one of hundreds of different genes can be behind these disorders, which means searching for the root cause – and thus the right therapy - can take many years. Often no diagnosis can be made.

It was when Charles’s wife was very ill that she told him of her wish to ‘give back’. Inspired by her wish, Charles started the Merrin Foundation in her memory.

“In the beginning there were so many worthwhile causes that needed our help, but we soon found out that there’s a lot more to it than just giving money away,” Charles says.

Maureen McIlroy

Charles Tegner

to find an area where we felt we could make the most impact.”

The Foundation’s gift replaced a piece of vital, but outdated, research equipment, enabling processes that previously took more than 50 hours of work to be done in only two.

“In less than a year we’ve been able to analyse more than 8000 individual samples to find out if a particular neonatal vaccination could prevent allergy, eczema and infection in infants,” MCRI researcher Susie Garmano explains.

“We’ve also used it as part of a study to improve outcomes for infants after cardiac surgery.”

Spurred on by the enthusiasm of MCRI researchers and the impact of their support, Charles wanted to know what else he could do.

“I was very interested in genomics and one suggestion was to fund a new test for children

MURDOCH CHILDREN’S RESEARCH INSTITUTE

The Merrin Foundation’s gift changed that situation; enabling MCRI researchers to develop a test that examines hundreds of genes simultaneously to provide families with quicker results.

“In less than six months we were able to identify the genetic cause of brain disorders for more than half of cases tested, a remarkable improvement on previous diagnostic rates which were less than one in 10,” says MCRI researcher A/ Prof Paul Lockhart.

Charles says, “we think that our gifts to MCRI have been some of our most productive ones. We’re very pleased. It’s a joyful and uplifting feeling.”

Our heartfelt thanks for the Merrin Foundation’s support through its founder, Charles Tegner, and co-directors, Ann Hyams, Genevieve Timmons and Mark Fairbairn.

Merrin would have been proud that such a positive impact on children’s lives has been made in her memory.

strength of partnerships Rebecca Le Souef and her son, Ryan

MCRI is proud to have the backing of a diverse range of corporate supporters who not only provide financial help for our research, but raise awareness of our story through their networks.

“ Our employees told us that they wanted our community program to improve the lives of children.”

For Latitude Financial Services, MCRI ticked all the boxes for its workplace giving program.

tree nuts after having an anaphylactic reaction to peanut butter at 2 years of age.

“Our employees told us that they wanted our community program to improve the lives of children. Given MCRI’s breadth and impact on child health, it was an obvious choice,” says Frederika Walls, Corporate Affairs Leader at Latitude Financial Services. MCRI was honoured to be selected as one of its inaugural community partners.

“It was very hard at first. I was extremely nervous about everywhere he went. I’m still conscious of the fact that every day we send him off to school, we’re relying on others to control his environment. The school is very good about this, but they can’t always control what kids bring in their lunchboxes,” says Rebecca.

A central aspect of the partnership was Latitude’s Better Giving program, which allows their 1200 Australian employees to support MCRI through workplace giving. It’s a simple and tax effective way to support MCRI, as employees are able to make contributions from pre-tax dollars. Latitude has generously matched each donation, creating an even greater impact.

Unfortunately Ryan’s story is all too common — the number of children with a food allergy or food-related immune disorder has increased dramatically in the past 10 years.

“It’s early days in our partnership, but we are delighted that some Latitude employees have had the opportunity to visit the Institute and see the impact first-hand,” says Ms Walls.

A personal reason to give Rebecca Le Souef’s reason for giving to MCRI can't get any more personal.

The Latitude’s UX Manager’s son, Ryan, was diagnosed with a severe allergy to peanuts and

Rebecca was well aware of MCRI’s worldleading research into the prevention, treatment and cure of food allergy, and was delighted when Latitude selected MCRI as a partner.

“I’m very much looking forward to seeing MCRI’s peanut oral immunotherapy treatment move through clinical trials to eventually reach the mainstream population.

This would be incredible for Ryan,” she says.

We sincerely thank Rebecca, Latitude Financial Services and all of our incredible corporate supporters who give through workplace giving or other arrangements.

ANNUAL REPORT 2018

35

ENGAGEMENT AND PHILANTHROPY

celebrating with events Each year, the team at MCRI is pleased to host events that celebrate the breadth and generosity of our donor community, encourage awareness and support of the Institute, and share our vision for the future.

We were privileged to have the opportunity to have our Dame Elisabeth Murdoch Mother’s Day Lunches, generously hosted by Mrs Jeanne Pratt AC at Raheen House in Melbourne and by Ms Lorraine Tarabay and Mr Nick Langley at their Point Piper home in Sydney.

Rotary Art Show, which raised funds in conjunction with the Australian Food Allergy Foundation in support of MCRI’s Vitality Project, and the annual Diabetes Hearts and Minds Ball which raised funds for diabetes research.

With the sprawling gardens of Raheen and sparkling views across Sydney Harbour as their respective backdrops, guests united to support our work in genomics and stem cell medicine.

We were proud to hold our third Dads of Melbourne lunch at Becco restaurant in Melbourne thanks to the invaluable efforts of Peter Mastos, Marcus Freeman, Kate Mohr, Miff Blythe and Vas Katos.

We continue to see incredible community support through events such as the Camberwell

With our research and discoveries touching the lives of millions of children and families

worldwide, a Friends of MCRI Committee was formed to further enrich our international networks and awareness. Committee members Sarah Murdoch, David Calvert-Jones, Suzi Carp, Steven Casper, Kate Mohr, Miff Blythe and Trent Blacket successfully held launch events in L.A and New York and are excited by the opportunity to take our message to the global community.

We thank you all for your special part in caring for kids.

Showcasing our research discoveries We were pleased to welcome 250 guests to the Institute for our Annual Showcase event.

Returning guests and those new to the Institute had the opportunity to hear about our success stories, current initiatives and vision for the future.

The Annual Showcase is a special chance for our Director, Kathryn North AC, our Chairman, Suzi Carp, and researchers and staff to share our achievements with those who have made it all possible.

Our researchers took guests on the journey of GenV, a project that is changing the landscape of how large-scale research happens with an aim to improve treatments, provide better services and prevent illness through predictive, tailored and targeted health care for children.

The Annual Showcase is a wonderful chance to hear from our brilliant minds; MCRI researchers who are changing the lives of our children and bringing hope for a healthy future.

36

MURDOCH CHILDREN’S RESEARCH INSTITUTE

The 2018 MCRI Annual Showcase

1

2

3

4

5

1

Jeanne Pratt AC, Prof Kathryn North AC, and Dame Quentin Bryce AD, CVO

2

Des and Margaret Dodds

3

Nicky Long, Genevieve Brammall and Carolyn Creswell

4

Sarah Murdoch, Mike Milken, Prof Kathryn North AC and Suzi Carp

5

Nick Langley and Lorraine Tarabay

6

Lucy Morton, James Calvert-Jones, Miff Blythe, Elina Wilson

6

ANNUAL REPORT 2018

37

ENGAGEMENT AND PHILANTHROPY

thank you 6A Foundation Mr & Mrs Paul & Nina Aberdeen A-esque AKA Promotions Mrs Antoinette Albert Mr Kenneth Allardice & Ms Julie Roy Mr Andrew Alston Mr Kent Anderson & Mrs Shauna Anderson Mr & Mrs John & Ros Andrews ANTG Pty Ltd ARA Consultants Ltd Mr Christopher Archibald & Ms Melissa Goode Ms Maria Ariano Ms Natalie Arrowsmith Mr Peter Austin Australian Communities Foundation Aypolsan Pty Ltd Ms Hayley Baillie Mrs & Mr Michelle & Marshall Baillieu Mr & Mrs Stuart & Jillian Bales Mr & Mrs Angus & Ariane Barker Mr & Mrs David & Penny Barlow Professor The Hon Dame Marie Bashir AD CVO Mr & Ms Andrew & Natalie Bassat Baystreet Pty Ltd Mrs Gillian Beaurepaire The Beck Family Foundation Mr Damien Beechey Mr & Mrs Andrew & Annie Bell Bellwether Foundation Pty Ltd Bendigo Truss Plant Pty Ltd Ms Danielle Besen Besen Family Foundation Big W Bill & Melinda Gates Foundation Bio Tools Pty Ltd BioAutism Mrs & Mr Stacey & Tim Bishop Black Diamondz Mr Trent Blacket & Ms Lily Romano Mr Daryl Blashki & Ms Lynn Trayer Mr Daneal Blicblau & Ms Jessica Huberman Mr & Ms Kai & Josephine Blunck Mr & Mrs James & Miffany Blythe Mr & Ms Bruce & Genevieve Brammall The Honourable Dame Quentin Bryce AD CVO & Mr Michael Bryce AM AE BUPA Australia Buzz Products Pty Ltd Calvert-Jones Foundation Cameron Foundation The Campbell Edwards Trust 38

Canaccord Genuity (Australia) Limited Carey Baptist Grammar School Carman's Fine Foods Mr Steven Casper & Ms Ilana Wald The CASS Foundation Charles Allen Charitable Trust Cher Family Foundation Children's Cancer Centre - Monash Children's Hospital Children's Cancer Foundation Children's Tumour Foundation of Australia Mr & Mrs Brandon & Davina Chizik Mrs & Mr Vanessa & Andrew Chrapot Mrs Ronda Clarke Mr Jeremy Clinton Mr & Mrs Trevor & Heather Cohen Ms Sharn Coombes Mr & Mrs Anthony & Melanie Coops Mr & Mrs Richard & Jade Coppleson Ms Sharon Cowin Mr & Mrs Andrew & Abigail Cox Mr Laurence G Cox AO & Mrs Julie Ann Cox AM Mr & Mrs Simon & Sophie Crowe The Cuming Bequest Mrs June Danks Datt Family Foundation David And Wilma Keath Family Prescribed Private Fund Mr G Davies & Mrs Jessica Mariani Dr Charles Day & Ms Elise Everest Mr & Mrs David & Veronica de la Coeur Deague Group Pty Ltd Dina and Ron Goldschlager Family Charitable Foundation Mr & Mrs Des & Margaret Dodds Doll Association of Victoria Inc. Dr Annette Domanti Mr Albert Donald DS Capital Endowment Mr Mustafa Eideh Equity-One Mortgage Fund Ltd Eureka Benevolent Foundation Mrs Meredith Evans Mrs Stephanie Fairfax Mr & Mrs Dov & Miriam Farkas Ms Heather Felix Fight Cancer Foundation Mr & Mrs Rob & Sandy Fildes Financial Markets for Children Finkel Foundation Mr & Mrs Robert & Bernadette Fitzgerald The Flew Foundation Mr Nick Fordham

MURDOCH CHILDREN’S RESEARCH INSTITUTE

Mr & Mrs Andrew & Lucy Fortey Mr Todd Foster The Fox Family Foundation Mr & Mrs Graeme & Pamela Fraser Mr & Mrs Geoff & Lesley Freeman Dr & Mrs John & Diana Frew Ms Jessica Frid Mrs Vivienne Fried Fuchs Lubricants (Australasia) Pty Ltd Mr & Mrs Tony & Helen Gandel Gandel Philanthropy Mr & Mrs Michael & Helen Gannon Mr & Mrs Ross & Judith Gardner Mr & Mrs John & Gaye Gaylard Mr & Mrs Raphael & Fiona Geminder Genesis Entertainment George & Freda Castan Families Charitable Foundation Mr Joseph Gersh AM Mr & Mrs John & Suzanne Gibson Ms Kimberly Gire & Mr Marten Touw Dr June Gleeson Mr & Ms Ergad & Roslyn Gold Goldman Sachs Gives Mr Michael Green Mr & Mrs Bruce & Anne Grey Mr & Mrs Nicholas & Olivia Haddad Mr Maurice E Hall Mr & Mrs Leonard & Tanya Hamersfeld Mr & Mrs Michael & Susan Hamson Mr & Mrs Nicholas & Adelicia Harding Heart Foundation Ms Jennifer Heath Mr & Mrs Geoffrey & Dorothy Heeley Mr William H Hodgson The Ian Potter Foundation In celebration of Jennifer Ayoub's Birthday In celebration of Zac Hamersfeld In celebration of Alex Holland In celebration of Judah Sable's Birthday In memory of Edith 'Judy' Ashton In memory of Mary Costigan In memory of Nance Elizabeth Creaton In memory L I Roach Mr & Mrs Jonathan & Nicki Isaacs Isaacson Davis Foundation The Isabel & John Gilbertson Charitable Trust Jam and Jelly Foundation Mr Bruce Jeanes Mr David Jenkins & Ms Fran H Lefroy John Burge Trust

Ms Kerry-Anne Johnston Mr Luke Jones Dr Joan Kaaden Mr Ian Kennedy AM & Dr Sandra Hacker AO The Honourable Jeffrey G Kennett AC & Mrs Felicity Kennett The Kimberley Foundation Ms & Mr Adriana & Joel Kligman Mr Peter Konidaris L Kornhauser family Mrs Dinah Krongold Mr Nick Kutcher La Prairie Group Australia Pty Ltd Mr & Mrs Jeremy & Miranda Lansell Latitude Financial Services Mr & Mrs Ken & Liz Launder Le Louvre Australia Dr & Mrs George & Joan Lefroy Ms Marj Lefroy Mr Sam Riggall & Ms Nichola Lefroy Mr Jeremy Leibler Mrs & Mr Glenda & Greg Lewin Mr Adam Lindell Listamere Pty Ltd Mr Brian Little Ms Denise Livingstone Logan Family Foundation Mrs Christine MacGill Macquarie Group Foundation The Marian & E.H. Flack Trust Ms Linda Martin Mr & Mrs Peter & Elaine Mastos Mrs Judy A Matear Mr Andrew McCann Denis and Chris McConnell Mr Mike McGrath Ms Tina McMeckan McNamara Foundation The McPhee Charitable Fund Mrs & Mr Rosemary & Douglas Meagher Merrin Foundation Mr & Ms Quentin & Amanda Miller The Miller Foundation Ltd Minimax Mr & Ms Dean & Kate Mohr Mrs Victoria Morish & Mr Iggi Damiani Mount Scopus Memorial College Gandel Besen House Mr & Mrs Jason & Danni Murray Muscular Dystrophy Australia My Room Mr Baillieu Myer AC & Mrs Sarah Myer The Myer Foundation

Naranda Meadows Pty Ltd Nelson Alexander Real Estate Dr Philip Neri News Corp Australia Nick Baldi Constructions Pty Ltd Nissan Casting Australia Pty Ltd Mr & Mrs Rodney & Judith O'Neil The Orloff Family Charitable Trust Mr Tony Osmond & Ms Fiona Griffiths The Paul Ramsay Foundation Professor David Penington AC & Dr Sonay Hussein Ms Karan Perkins PKU Association of NSW Inc Lady Primrose Potter AC Predikkta Pty Ltd Prior Foundation The Rebecca L Cooper Medical Research Foundation Ltd Mr & Mrs Adrian & Christina Redlich Rellim Foundation Ms Jenny Renton Mr Troy Rike Mrs Sue Ritchie Mrs Judith Robinson & Mr Michael Robinson AO Mrs Belinda Roper Mr & Mrs Jonathan & Nadine Rosham Mrs & Mr Susan & Garry Rothwell Royal Children's Hospital Foundation Adam & Yoko Ryan and Family Mr & Mrs Gerry & Valerie Ryan Mr & Mrs Radek & Helen Sali The Samuel Nissen Charitable Foundation Mrs Pamela Sargood Save our Sons Duchenne Foundation Mr Matt Schmidt The Scobie & Claire Mackinnon Trust Mrs Holly Sherman Mr Bernie Shinners Mr Ian Simmonds Mr Leon Skaliotis Mr & Mrs Colin & Jan Smith Mr & Mrs Dean & Ellie Smorgon Mr & Mrs Rodney & Ann Smorgon Ms & Mr Sandra & Barry Smorgon Sorrento Golf Club Lady Southey AC Mr Peter Spencer Mr Neil Spitzer Splitrock Spring Water Spotlight Charitable Foundation Stafford Fox Medical Research Foundation Mr & Mrs Grant & Terri Stephenson

Mr Robert Stone The Sunraysia Foundation Mr Ben Suttie Mrs Emma Suttie Ms Sophie Swann Swann Family Foundation Mr & Mrs David & Susan Syme Ms Lorraine Tarabay & Mr Nick Langley Mr Sean Taylor Mrs Amy Tennent Mr Kevin Teoh Tour de Cure Mrs Caroline Travers OAM HUG Foundation Mr & Mrs Patrick & Margaret Upfold Mr & Mrs Raja & Harshita Uppal Mr & Mrs Tony & Judy Van Bergen Vanguard Investments Australia Ltd Vincent Chiodo Charitable Foundation W V James and Son Mr & Mrs Stephen & Brenda Wellard Mr & Mrs Geoffrey & Shirley Williams Dr Nicholas Wilsmore Mr & Mrs Ron & Joan Wilson The Wolf Foundation Dr Ian Woolf & Mrs Julie Woolf Dr Elizabeth Xipell Yabby Lake Wines Mr & Mrs Edward & Mandy Yencken Mr Derek Young AM & Mrs Caroline Young Mr Bobby Zagame Ms Carla Zampatti

Gifts received over $1000 ANNUAL REPORT 2018

39

F I N A N C I A L S TAT E M E N T S

year at a glance

149.61m

$

Total revenue ▲ 8.9%

1133 Peer reviewed publications 41 N HMRC Projects and Fellowships funded 40 n on-NHMRC grants funded (30% success rate)

(21% success rate)

261 Clinical trials 59 Drug or device trials 1287 Staff 2167 Media mentions

2018 40

MURDOCH CHILDREN’S RESEARCH INSTITUTE

75.06m

$

Research and government grants

27.89m

$

VCGS

30.60m

$

Contract research, trials & other

16.05m

$

Donations, fundraising & bequests ANNUAL REPORT 2018

41

F I N A N C I A L S TAT E M E N T S

statement of financial position As at 31 December 2018 Consolidated

Company

2018

2017

2018

2017

$’000

$’000

$’000

$’000

Cash and cash equivalents

21,482

21,322

19,464

15,376

Trade receivables and other assets

14,486

11,519

11,155

8,691

Other investments

36,905

30,546

30,312

24,966

Total Current Assets

72,873

63,387

60,931

49,033

Trade receivables and other assets

56,341

57,683

56,341

57,683

Other investments

55,314

50,590

50,240

50,590

Property, plant & equipment

11,420

14,173

10,485

12,810

411

823

-

-

Total Non-Current Assets

123,486

123,269

117,066

121,082

Total Assets

196,359

186,655

177,997

170,115

Trade and other payables

37,542

31,671

33,336

28,596

Employee benefits

15,866

13,921

10,692

9,353

Total Current Liabilities

53,411

45,592

44,028

37,949

Employee benefits

2,579

2,211

2,216

1,833

Total Non-Current Liabilities

2,579

2,211

2,216

1,833

55,990

47,802

46,244

39,782

140,369

138,853

131,755

130,333

136,233

134,717

127,659

126,237

4,096

4,096

4,096

4,096

40

40

-

-

140,369

138,853

131,755

130,333

In Australian dollars

Current Assets

Non-Current Assets

Intangibles

Current Liabilities

Non-Current Liabilities

Total Liabilities

Net Assets

Members’ Funds Accumulated funds Fair value reserve Other reserve Total Members’ Funds

42

MURDOCH CHILDREN’S RESEARCH INSTITUTE

statement of cash flows As at 31 December 2018 Consolidated

Company

2018

2017

2018

2017

$’000

$’000

$’000

$’000

Patient fees received

23,799

22,361

-

-

Government and other grants received

80,496

75,167

73,070

68,622

Donations received

15,682

17,214

15,682

17,214

992

465

764

465

26,703

23,257

30,577

23,257

-

-

78

(90)

(136,900)

(133,158)

(111,640)

(104,729)

10,772

5,306

8,531

4,738

545

2,295

436

2,114

Acquisition of property, plant and equipment

(2,586)

(5,734)

(2,394)

(5,545)

Acquisition of investments

(9,872)

(2,555)

(3,786)

(2,553)

(11,913)

(5,995)

(5,744)

(5,985)

Net increase / (decrease) in cash and cash equivalents

(1,141)

(689)

2,787

(1,247)

Cash equivalents at 1 January

21,322

22,670

15,376

17,281

1,301

(659)

1,301

(659)

21,482

21,322

19,464

15,376

In Australian dollars

Cash flows from operating activities

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

Cash flows from operating activities

Cash flows from investing activities Investment income received

Net cash provided from (used in) investing activities

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

ANNUAL REPORT 2018

43

F I N A N C I A L S TAT E M E N T S

statement of profit or loss and other comprehensive income For the year ended 31 December 2018 Consolidated

Company

2018

2017

2018

2017

$’000

$’000

$’000

$’000

132,020

117,407

104,467

90,888

(7,093)

(7,160)

(6,059)

(5,962)

(140,970)

(127,948)

(114,208)

(101,965)

(16,043)

(17,702)

(15,802)

(17,039)

Donation, estates, bequest and fundraising income

16,054

17,214

16,054

17,214

Fundraising expense

(1,242)

(1,584)

(1,242)

(1,584)

Net surplus obtained from fundraising activities

14,812

15,629

14,812

15,629

1,536

2,759

1,201

2,579

305

687

210

-

1,602

-

1,602

305

2,289

210

2,770

In Australian dollars

Revenue from research and clinical activities Depreciation and amortisation Other expenses for research and clinical activities Surplus/(deficit) from research and clinical activities

Net finance income/(expense) Operating surplus for the period

1,169

Other comprehensive income Items that may subsequently be reclassified to profit or loss Net change in available for sale financial assets Total comprehensive surplus for the period

To read our Independent Auditor's Report from 2018, please go to www.mcri.edu.au/about/annual-reports/auditors-report

44

MURDOCH CHILDREN’S RESEARCH INSTITUTE

your support can help to create possible E philanthropy@mcri.edu.au T

03 8341 6200

W mcri.edu.au/get-involved “ It is a privilege to connect the passions and commitment of our donors with the brilliant research minds at MCRI. "This connection creates extraordinary outcomes for children and their families in Australia and around the world.�

Matthew Hannan, Head of Engagement and Philanthropy ANNUAL REPORT 2018

45

46

MURDOCH CHILDREN’S RESEARCH INSTITUTE

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

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

For every obstacle there must be a way around.

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

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

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

We believe every child deserves a healthy start to life.

And a happy and prosperous community needs healthy children.

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

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

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

So every child can have a childhood.

So every child can grow to reach their full potential.

Children are at the heart of everything we do.

ANNUAL REPORT 2018

47

Murdoch Children’s Research Institute Royal Children’s Hospital Flemington Road, Parkville Victoria 3052 Australia P +61 (3) 8341 6200 F +61 (3) 8341 6212 FREE CALL 1300 766 439 E mcri@mcri.edu.au W mcri.edu.au Donate to Murdoch Children’s Research Institute mcri.edu.au/donate