Murdoch Childrens Research Institute Annual Report 2013 by Murdoch Children's Research Institute

murdoch childrens research institute annual report 2013

murdoch childrens research institute The Royal Childrenâ&#x20AC;&#x2122;s Hospital Flemington Road, Parkville Victoria 3052 Australia Phone +61 3 8341 6200 Fax +61 3 9348 1391 www.mcri.edu.au ABN 21 006 566 972

annual report

2013

annual report 2013

Children experience disease differently to adults and the treatments , that are needed for children are different too. That s why our research is crucial, so we can find the best way to prevent disease and treat children to give them the healthiest start to life.

urdoch Childrens Research Institute is world renowned for its research into infant, child and adolescent health. Our world-class team of 1500 researchers is dedicated to making discoveries to prevent and treat common and rare childhood conditions. We also collaborate with teams nationally and internationally to answer child health problems quickly and to make an even greater impact on the health of children around the world. Today the health of children is challenged by issues such as obesity, allergies, diabetes, cancer and mental health. As the largest child health research institute in Australia, Murdoch Childrens is well positioned to make ground-breaking discoveries and translate them into treatments and cures to help ensure current and future generations of children are healthy. www.mcri.edu.au

contents About us

Our Themes

Year at a glance

Cell Biology

Chairmanâ&#x20AC;&#x2122;s report

Clinical Sciences

Directorâ&#x20AC;&#x2122;s report

Genetics

Organisational chart

Infection & Immunity

Board of Directors

Population Health

Executive committees

Research highlights

Victorian Clinical Genetics Services

Research Support & Operations

Core groups

Financial reports

Students

Grants & awards

Special events

Events 54

Corporate partners

Donors

annual report 2013

about us The place to be to change the face of child health

urdoch Childrens Research Institute is a worldleading research institute dedicated to making discoveries to improve child health. Children experience disease differently to adults, so the treatment and care provided needs to be tailored to their needs and development. Our dedicated team of 1500 researchers is passionate about filling the gaps in what we know about healing sick kids to create a healthier and brighter future for the generations of tomorrow. Our researchers operate over three areas: laboratory work to understand how diseases arise, clinical research working side-by-side with paediatric doctors and nurses to influence diagnosis and treatment, and broad population health studies to understand how disease impacts children in the wider community. Working across five main research themes and five major cross-disciplinary research programs, the Institute conducts nationally and internationally recognised studies to make genuine changes to the delivery of health and education.

Research Themes The Institute brings together large teams of researchers working on related areas to stimulate scientific collaboration and find answers to child health problems. Each theme incorporates groups with varied skills and consists of groups from clinical, laboratory and public health fields.

Cell Biology The Cell Biology Theme seeks to understand how cells work and how disease processes alter healthy cells. The team investigates how cells work in order to help children with conditions such as developmental disorders, cancer and congenital diseases.

Clinical Sciences We are passionate about saving the lives and improving the long-term health and wellbeing of children with serious, acute and chronic conditions. Working closely with clinicians, our researchers study current diagnosis and treatment practices.

A key component of this research is the follow-up of the psychological and long-term wellbeing of those who undergo clinical care.

Genetics Murdoch Childrens was originally established with a focus on genetics research and has a long history of excellence in this area. Researchers work to discover the genetic basis of disease to understand the causes and improve the management of genetic conditions such as ataxia, thalassemia, mitochondrial and chromosome disorders. Infection and Immunity The Infection and Immunity theme addresses important health problems in children, such as allergies, common infections and immune conditions both locally and globally. One focus of the theme is vaccine development, testing, safety and quality assurance. Population Health A complex relationship of social, environmental and biological factors influences child and adolescent health. The Population Health theme studies the health of communities and translates this knowledge into prevention, intervention and treatment. We work on an international scale in many of our programs and partner with government and non-government agencies to improve the lives of children everywhere. Major Strategic Initiatives At the same time, we are working on five major strategic initiatives that we believe will significantly improve the health of children and help prevent and treat illness and disease.

Generation Victoria 'Gen V' The ambitious and innovative Gen V study offers us the chance to improve current standard models of care and may ultimately improve childhood health in Victoria, Australia and globally. This bold and ground-breaking initiative will see the recruitment of all Victorian babies born over a period of two years in order to improve child health and the health of future generations.

Genomics and Personalised Medicine The Institute is in a unique position to be a leader in developing personalised medicine to improve child health outcomes, especially when it comes to improving the diagnosis, management and treatment of genetic diseases.

Global Health As well as improving the health of kids across Australia, the Institute is committed to establishing itself as a truly global health centre. The Global Health program will enable us to support and implement interdisciplinary research, education and services to address the issues facing the worldâ&#x20AC;&#x2122;s poorest children. This includes the development and implementation of vaccines, global disease epidemiology, social and behavioural interventions.

Health Service RESEARCH It can be challenging for research evidence to reach clinical practice. To improve the delivery of healthcare and education to children, the Health Services initiative works to identify the most effective ways to organise, manage, finance and deliver high-quality care. Our research will be applied by doctors, nurses, and allied health professionals working on a daily basis with families.

, Melbourne Children s Trials Centre We are establishing a single clinical and community trials precinct in collaboration with The Royal Childrenâ&#x20AC;&#x2122;s Hospital and the University of Melbourne to deliver the latest clinical trials and care to children. The outcome will be more effective research: insights, understanding, translation and improved treatment for children everywhere.

How your fundraising dollars help Donor funds have a huge impact on medical research. Many of our research projects that receive government funding were initially established with funding from private sources. Without this support, some of our vital research would not get off the ground as governments and competitive funding bodies tend to support established research. Most importantly, we distribute philanthropic funds to our research teams based on performance. Every donation, no matter how small, has the potential to save lives. Gifts to Murdoch Childrens are an investment in the future health of children.

annual report 2013

year at a glance Research highlights: Our research continued to deliver results

INCOME

to improve the health and wellbeing of children.

Annual turnover increased to $104.55 million in 2013, up from $96.9 million in 2012.

Among the highlights:

n 2013, the Institute was awarded three clinical Centre of Research Excellence (CRE) Grants from the National Health & Medical Research Council (NHMRC), out of six available nationally. The CREs will focus on improving emergency department care, health outcomes for newborn babies and for children with cerebral palsy.

GRANTS

A study by the Institute found children who have an earlier onset of puberty have poorer mental health from as early as preschool age. The study, which followed almost 3500 children from age four through to 11, found boys with an onset of puberty by eight to nine years of age had greater behavioural difficulties and poorer emotional and social adjustment from as early as four years of age. This pattern continued through to early adolescence. Girls with early puberty had more difficulties in emotional and social adjustment from early childhood, but not the behavioural problems found in boys. Our researchers were involved in the ‘Global Burden of Disease, Injury and Risk Factor Study’ which is the largest ever study to describe the global distribution and causes of a wide array of major diseases, injuries, and health risk factors. Researchers from various disciplines worked together to measure levels and trends in all major diseases, injuries, and risk factors. The study produced new and comprehensive sets of estimates and easy-touse data visualisation tools for research and teaching. In total, the study generated nearly one billion estimates of health outcomes. A total of seven articles from the study were published in The Lancet.

Researchers discovered a lack of vitamin D increases the likelihood of food allergy in infants. They found infants who are vitamin D insufficient were three times more likely to have a food allergy. Those with vitamin D insufficiency were also more likely to have multiple than single food allergies, with the odds increasing to ten times more likely among those with two or more food allergies.

Despite the highly competitive environment, we were once again successful in securing competitive government and other grants for a wide range of research projects. This included $26.2 million from the Federal Government’s NHMRC for new initiatives commencing in 2014.

INTERNATIONAL GRANTS Murdoch Childrens received $6.1 million in grants from international funding bodies in 2013. A large amount of support was from the Bill & Melinda Gates Foundation supporting our rotavirus and pneumococcal vaccine programs for children in developing countries.

PUBLICATIONS When our researchers make discoveries, their work is submitted to peer-reviewed scientific journals around the world for communication to the medical community. This enables key discoveries to be more quickly translated to ensure better outcomes for children. In 2013, we had 713 papers published which was a significant increase, up from 610 in 2012. This number included 206 in the world’s highest ranking journals.

STAFF There were 1718 paid staff, students and honoraries at the Institute in 2013. This number includes staff from The Royal Children’s Hospital and allied health professionals and University of Melbourne academics who conduct research within Murdoch Childrens.

FUNDRAISING Our team engaged with the community to raise money to provide the initial funding for our research, to get it to the point where larger grants can be attracted. In 2013, fundraising, donation and bequest income totalled $6.9 million.

2012

2013

Total income

$96.9 million

$104.55million

NHMRC grants awarded (for projects to commence in the following year)

$28.8 million

$26.2 million

Peer reviewed publications

610

713

Peer reviewed publications in the world’s top medical journals (tier one and two)

153

206

Staff & students

1600

1718

Donations, fundraising & bequests

$6.1 million

$6.9 million

Media mentions

2075

3074

annual report 2013

, chairman s report In 2013 we embarked on an exciting new era, beginning with the appointment of Professor Kathryn North who joined the Institute as Director in February. Under Kathryn’s leadership, the Institute underwent a strategic review which looked at priorities and opportunities for the Institute in the coming years.

his review identified a number of new and important health initiatives. At varying stages of development, we are about to embark on some ambitious and innovative projects including Generation Victoria, the Melbourne Children’s Trials Centre and the Melbourne Genomics Health Alliance, which are detailed later in this annual report, and I look forward to seeing what can be achieved through these initiatives. Researchers across all of our themes had a very successful year, with a number winning prestigious awards and grants, and making significant research findings that were published in top scientific journals. Financially, Murdoch Childrens’ income grew to $104.55 million in 2013, up from $96.9 million the previous year. Philanthropic support, which is vital for providing critical equipment and facilitating new initiatives, continued to be strong despite the challenging environment, with $6.9 million donated from passionate individuals, community groups and trusts and foundations.

The past 12 months saw changes on the Board, with the resignation of longstanding members Mr Martin Armstrong and Mrs Janet Calvert-Jones, who have provided invaluable counsel since 1997 and 1986 respectively. We were delighted to welcome Mr Steven Casper, who is also the Chairman of the Institute’s Development Board, and Mr Bruce Grey. The Institute’s ambassador, Mrs Sarah Murdoch, who has been tireless in her work for the organisation over the past 14 years, agreed to increase her involvement with the Institute and joined the Board in February 2014. I trust you will enjoy reading about the Institute’s achievements and future endeavours. We really are best placed to improve outcomes for children and can proudly boast about being competitive on an international stage. MR Leigh Clifford AO

annual report 2013

, director s report My first year at the Institute has been a wonderful experience - we have an outstanding team all of whom are passionate about achieving the very best for children and their families.

2013

was a very successful year for the Institute and was filled with highlights. Our researchers achieved major grant successes and several received esteemed national and international awards. We have strengthened our campus partnerships and together developed major and innovative strategic initiatives. We have made many discoveries and generated countless research findings that will translate to tangible improvements in child health. It has been a pleasure for me to immerse myself in the research undertaken at Murdoch Childrens and to appreciate its breadth and quality. We can truly boast that we have the best and brightest child health researchers across all areas of clinical, basic science and population health research.

STRATEGIC REVIEW In 2013, all of the staff of the Institute, members of our Board and our campus partners worked together to develop a new strategic plan to guide the direction of our research over the next five years. Through this process, we identified five key strategic areas in which we can make a significant difference to the health of children. With our campus partners, we are establishing the Melbourne Children’s Trials Centre, so that the latest therapies discovered anywhere in the world will be readily available in Australia. Our state-ofthe-art centre will support clinicians and researchers to initiate and perform trials of new therapies and will provide access to all of the skills needed to ensure a successful clinical research study such as biostatistics, ethics, data storage, health economic expertise and biobanking. The Institute has always been a leader in genetics research. In 2014 we will partner with six other key institutions across the Parkville precinct to form the Melbourne Genomics Health Alliance. This collaborative effort will focus on bringing the latest innovations in genomic testing into everyday healthcare so that we truly achieve a “personalised” approach to medicine.

Our researchers will continue to focus on optimising Health Service Delivery both in Australia and internationally. Our goal is to influence health policy through the development of evidence-based guidelines and to develop and promote “gold standards” in clinical practice. We will also expand our emphasis on Global Child Health with a commitment to address the inequity in child mortality in disadvantaged populations internationally. Another campus initiative is the innovative Generation Victoria (Gen V), a longitudinal study that will connect research, clinical care and service delivery to every child born in Victoria. The overarching objective of the study is to generate new knowledge about how to provide children with the best possible healthy start to life. We hope to recruit a cohort of babies before and around the time of birth and monitor their health, development and episodes of care throughout childhood and into school years. In this way, we can identify factors that contribute to common problems such as obesity, allergy, infection, developmental delay, depression and anxiety so that we can design the best approaches to prevention and intervention.

RESEARCH HIGHLIGHTS We celebrated a number of successes throughout the year, including our National Health & Medical Research Council (NHMRC) grant results; we achieved success rates well above the national average despite the highly competitive environment. We were also awarded three prestigious national clinical Centre of Research Excellence (CRE) grants by the NHMRC – out of only six awarded nationally. The CREs will focus on improving emergency

< DIRECTOR PROFESSOR KATHRYN NORTH

department care, health outcomes for newborn babies and for children with cerebral palsy. With the addition of these new Centres, the Institute will have eight national Centres of Research Excellence in total. Neuromuscular disorders, childhood food allergy, pneumonia in developing countries, biostatistics and childhood language are the focus of the five centres already based at the Institute. During 2013, many of our researchers have been recognised in Australia and internationally for their work. Professor Ruth Bishop, one of our most esteemed and respected researchers, was awarded the CSL Florey Medal, a national honor, for her

lifetime of work on rotavirus. Professor Frank Oberklaid, who leads the Centre for Community Child Health, was awarded the Nils Rosen von Rosenstein medal by Sweden’s Uppsala University for outstanding contribution to paediatrics. Two of our researchers were recognised in the NHMRC Excellence Awards; Dr Katherine Howell was awarded the Gustav Nossal Scholarship as the highest-ranked applicant for a medical postgraduate scholarship. Professor Melissa Wake was recognised as the top-ranked research fellow, and was awarded the Elizabeth Blackburn Fellowship as the highestranked female researcher overall. These grants and awards highlight our groundbreaking research and the outstanding contributions that we are making towards better therapy and prevention of childhood disorders. >

annual report 2013

, MELBOURNE CHILDREN S

In 2013, the name of the campus was changed to Melbourne Children’s; this enhancement will better place us to promote our position as a global leader in paediatric healthcare, research and education. As testament to the strength of the campus partnership, we received the 2013 Melbourne Award for our “contribution to the profile of the city of Melbourne” for our work locally, nationally and internationally.

The Royal Children s Hospital Professor Christine Kilpatrick

Murdoch Childrens Research Institute board of directors chairman

Mr Leigh Clifford AO Executive director Professor Kathryn North AM

chief operating officer Mr John Dakin

LOOKING TO

2014

& BEYOND

Next year will be big year for our researchers and staff as we launch and expand a number of our major initiatives. We will also embed our new research strategy and consolidate our research groups and structure. We will continue to remain focused on our overall goal: To change the face of child health by improving health outcomes and treatments for children in Australia and around the world.

associate directors laboratory sciences

Professor Andrew Sinclair

University of Melbourne, Department of Paediatrics Professor Paul Monagle

board committees Audit, Finance & Risk Development Board Fundraising Investment Remuneration Translation & Commercialisation Victorian Clinical Genetics Services Board

office of the director Dr Leanne Mills

clinical and public health

research support & operations

Professor Sheena Reilly

research strategy Dr MaryAnne Aitken

finance

Mr Viren Abeyasinghe

The Institute is also an active player in the Parkville Precinct, which is acknowledged as one of Australia’s leading centres for medical, education, clinical practice, clinical trials and development of biotechnology products. Together with our precinct partners we are focused on building on our basic and translational research programs, enhancing research infrastructure and positioning the precinct as a major international player.

murdoch childrens research institute

The campus partnership between The Royal Children’s Hospital, the University of Melbourne’s Department of Paediatrics and the Institute is unique in Australia and acclaimed internationally. As a fully integrated paediatric teaching hospital and research institute, our research discoveries can be quickly translated into practical treatments for children. Many of our researchers are also clinicians, which means they can use research to solve problems they confront in their day-to-day clinical work.

organisational chart

theme directors

disease model unit

population health

Ms Sophie Agius

Professor Katie Allen

& development Ms Lyn Amy

engagement

clinical services

Professor Vicki Anderson

grants

cell biology

Ms Marianne Ciavarella

Professor John Bateman

& legal Dr James Dromey

commercialisation

quantitative sciences Professor John Carlin , melbourne children s trials centre Professor Andrew Davidson

infection & immunity Professor Andy Giraud

human resources

genetics

Ms Andrea Frigo

Professor David Thorburn

operations

Mr Andrew Grimes Mr Neil Harker

victorian clinical genetics services

information technology

Associate Professor David Amor

internal auditor

director

Mr Wayne Mather communications

Ms Simone Myers scientific services

Dr Benjamin Ong

I am very grateful to our researchers, staff, Board and committees, and Ambassador Sarah Murdoch for their ongoing support and commitment. I warmly thank all of our funding bodies, our supporters and donors, The Royal Children’s Hospital Foundation and the allocation of $3.3 million from the Victorian State Government’s Operational Infrastructure Support Scheme. Your generous and ongoing support is vital to us continuing our cutting-edge research.

annual report 2013

board of directors Mr Leigh Clifford AO

Mr Martin J Armstrong

Mrs Janet Calvert-Jones AO

Non-Executive Director, Chairman Chairman of Qantas Airways Ltd, Director of Bechtel Group Inc (USA), Chairman of Bechtel Australia Pty Ltd, Senior Advisor to Kholberg Kravis and Roberts, and Board Member of the National Gallery of Victoria Foundation. Director from 2007, Chairman from December 2009.

Non-Executive Director Principal of Plante & Henty, Chairman of Carnbrea Holdings Ltd, Director of the Jack Brockhoff Foundation and other public, private and non-profit organisations. Member of the Murdoch Childrens Research Institute’s Audit, Finance and Risk Committee and Translation and Commercialisation Committee. Director from 1997-October 2013.

Non-Executive Director Former Chairman of Herald and Weekly Times Limited, Chairman of Tapestry Foundation of Australia. Director from 1986-February 2014.

Non-Executive Director Director of River Capital, Director of Young and Well CRC, Director of Smiling Mind. Director from 2006.

Mr Steven Casper

Mr James Craig

Dr Charles Day

Mr Bruce Grey

Non-Executive Director Partner, DLA Piper and Chairman of the Murdoch Childrens Research Institute’s Development Board. CoChair of the Jewish National Fund (Victoria Division). Director from February 2013.

Non-Executive Director Chairman of Macquarie Capital Advisers and Director of other public and non-profit organisations. Chairman of the Murdoch Childrens Research Institute’s Investment Committee. Member of the Audit, Finance and Risk Committee since December 2013. Director from 2009.

Non-Executive Director Project Director at the University of Melbourne and Director of Bionic Vision Technologies. Member of the Murdoch Childrens Research Institute’s Translation and Commercialisation Committee. Director from 2011.

Non-Executive Director Managing Director at Advanced Manufacturing CRC Limited. Chairman of the Murdoch Childrens Research Institute’s Translation and Commercialisation Committee. Chairman of Advanced Braking Technology Limited, Director of CAP-XX Limited. Chairman of the Expert Advisory Panel for the Victorian Government’s Technology Voucher Program. Director from August 2013.

Mrs Suzi Carp

Professor Christine Kilpatrick Non-Executive Director Chief Executive Officer of The Royal Children’s Hospital. Director from 2008.

Mrs Sarah Murdoch Non-Executive Director Ambassador of the Murdoch Childrens Research Institute, Deputy Chair and International Ambassador of the Australian Ballet, Director of the Australian Research Alliance for Children and Youth, Patron of the National Breast Cancer Foundation, Chair of the National Breast Cancer Foundation’s Women’s Advisory Network. Director from February 2014.

The Hon Rob Knowles AO

Mr Ian Miller

Professor Paul Monagle

Non-Executive Director Chairman of The Royal Children’s Hospital, Director of Silverchain Group of Companies, Drinkwise Australia Ltd, IPG Pty Ltd, Brotherhood of St Laurence, and Annex. Director from 2012.

Non-Executive Director, Deputy Chairman Executive Chairman of the Burnham Group. Non-Executive Director of Macquarie Investment Management Limited and Avea Insurance Limited. Chairman of the Murdoch Childrens Research Institute’s Audit, Finance and Risk Committee and Member of the Investment Committee. Director from 2007, Appointed Deputy Chairman February 2010.

Non-Executive Director Stevenson Chair of Paediatrics and Assistant Dean, The Royal Children’s Hospital Academic Centre, the University of Melbourne. Director from 2008.

Professor Kathryn North AM

Mrs Judy Paterson

Mr Jason Yeap OAM

Non-Executive Director Director of Ace Radio Broadcasters. NonExecutive Director of the Victorian Clinical Genetics Services. Director from 1996.

Non-Executive Director Chairman of Mering Corporation Pty Ltd and Herbaceutic Holdings Ltd, China, Trustee of the National Gallery of Victoria, Chairman of the National Gallery of Victoria Foundation. Director from 2011.

Executive Director Director, Murdoch Childrens Research Institute, David Danks Professor of Child Health Research, the University of Melbourne. Chair NHMRC Research Committee. Member NHMRC Council. Chair of the Victorian Clinical Genetics Services Board. Director from February 2013.

annual report 2013

executive committee Invited to attend:

Professor Kathryn North AM

Professor Andrew Sinclair

Professor John Bateman

Professor Andy Giraud

Director

Associate Director Laboratory Services

Professor Katie Allen

Professor Sheena Reilly

Dr MaryAnne Aitken

Professor Vicki Anderson

Mr John Dakin

Dr Leanne Mills

Associate Director Clinical & Public Health

Audit, Finance & Risk Committee

Head of Research Strategy

Monitors the systems and controls established to safeguard the Institute’s assets. Chairman, Mr Ian Miller Mr Viren Abeyasinghe Mr Martin Armstrong (to October 2013) Mr Lachlan Armstrong (from December 2013) Mr James Craig (from December 2013) Mr John Dakin (from May 2013) Mr David Gillespie Ms Lisa Keam (to March 2013) Professor Kathryn North AM (from February 2013)

Population Health

Chief Operating Officer

Clinical Sciences

Translation & Commercialisation Committee

Advises the Board on business development opportunities for the biotechnology and pharmaceutical industries and on related issues including patent protection. Chairman, Mr Bruce Grey Mr Martin Armstrong (to October 2013) Mr John Dakin (from May 2013) Dr Charles Day Dr David Lester Ms Sue Michelmore Professor Kathryn North AM (from February 2013) Dr Andrew O’Brien Dr George Raitt Professor Sheena Reilly Assoc Professor Howard Slater Professor George Werther Invited to attend Ms Michelle Baker Dr James Dromey Professor Barrie Finnin

Cell Biology

Development Board

Provides advice on fundraising, marketing and communications activities to engage donors and supporters. Chairman, Mr Steven Casper Ms Anna A’Beckett (from February 2014) Mrs Ariane Barker Ms Lisa Bond Ms Genevieve Brammall Mr Anthony Coops Mr Andrew Cox Ms Susannah Fairley (to December 2013) Mrs Tanya Hamersfeld Mr Clark Kirby Ms Marisa Leone Mr Peter Mastos Mrs Kate Mohr Mrs Sarah Murdoch (to December 2013) Mr Adrian Redlich (to December 2013) Mrs Dahlia Sable

Infection & Immunity

Professor David Thorburn Genetics

r Viren Abeyasinghe M Chief Financial Officer A ssoc Professor David Amor Director VCGS M s Lyn Amy Head of Engagement & Development Professor John Carlin Quantitative Sciences A /Professor Andrew Davidson Medical Director of Melbourne Children’s Trials Centre Dr James Dromey Head of Commercialisation & Legal Mr Wayne Mather Chief Information Officer

Head of Office of the Director

Investment Committee Advises on the financial investments of the Institute.

Chairman, Mr James Craig Mr Viren Abeyasinghe Mrs Ariane Barker Mr John Dakin (from May 2013) M r Ben James (to August 2013) Ms Lisa Keam (to March 2013) Mr Dominic Leary (to February 2013) Mr Paul Martin (to August 2013) Mr Ian Miller Mr John Nickson (to August 2013) Professor Kathryn North AM (from February 2013)

Remuneration Committee M r Leigh Clifford AO M r Martin Armstrong (to October 2013) M r Ian Miller

Victorian Clinical Genetics SERVICES Board (VCGS)

Chair, Professor Kathryn North AM (from February 2013) Mr John Dakin (from May 2013) Mr David Gillespie Professor Stephen Roger Holdsworth Ms Lisa Keam (to March 2013) Professor Finlay Macrae Mrs Judy Paterson Invited to attend: Mr Viren Abeyasinghe Assoc P rofessor David Amor (VCGS Director)

annual report 2013

cell biology KEY PROTEIN LINKED TO CANCER IDENTIFIED

MATERNAL BMI PREDICTS TYPE 2 DIABETES IN CHILDREN

Researchers have made landmark discoveries studying the IGFBP-2 protein. Not only is this protein important in the normal insulin-like growth factor (IGF) pathway, but it is also involved in cancer cell growth. This work is likely to translate into more effective cancer therapies.

A team of international researchers, including Dr Matt Sabin (left) from the Institute, found a mother’s body mass index (BMI) can be used as a predictor for the later development of type 2 diabetes in her children, and is a stronger predictor than genetic data.

The Hormone Research group, led by Dr Vincenzo Russo and Professor George Werther, is internationally recognised for its leading work in the insulin-like growth factor field. The IGF system is ubiquitous during human embryonic life, early postnatal development and in adulthood. It controls cellular maintenance and a number of essential metabolic functions in most organs and tissues. Impairment of the IGF system occurs in disease state and malignancies, and elevation of the IGFBP-2 gene is consistently reported in major aggressive cancers. These include paediatric brain tumours and other common tumours affecting the Australian population, such as prostate and breast cancer. Researchers were able to demonstrate the pathways whereby IGFBP-2 could dramatically increase the rate of cancer cell growth and accelerate their spread to other sites where they normally wouldn’t grow. The study was the first to identify a specific region of the protein that is required for entry in the cell nucleus and for binding to genes that regulate cancer cell growth. When the team introduced changes to this region, the protein could not enter the nucleus and was rendered unable to activate the cancerpromoting genes. The team is now working toward the development of anti-cancer therapeutic molecules targeting IGFBP-2 activity in cancer cells. The study was published in the prestigious journal Oncogene.

Researchers studied more than 1800 children for more than 20 years and found not only did maternal BMI predict the later development of type 2 diabetes in offspring, but that the association was independent of other childhood factors, including genetics. Researchers also found a risk predication model based on both the child and mother’s BMI status was more accurate in predicting adulthood type 2 diabetes compared with an approach including only the child’s BMI data. They found no association between the father’s BMI and the later development of type 2 diabetes in the offspring. The study showed maternal BMI at recruitment of the child to the study, as well as the child’s own BMI, systolic blood pressure and genetic risk score for type 2 diabetes were all significantly associated with increased risk. Interestingly, fruit consumption showed inverse associations. The study was published in the Journal of Pediatrics.

EPIGENETICS CHANGE RAPIDLY IN FIRST YEAR OF LIFE The way our genes are activated changes rapidly when we are infants, a study by the Institute found. Researchers studied the epigenetic “switches” that regulate gene activity in 15 sets of twins at birth and at 18 months of age. The study, which was published in Genome Biology, found widespread epigenetic change in the genome within the first 18 months of life. Epigenetic gene switches help to direct development and ageing but can also be influenced by the environment, particularly in early life. Focusing on DNA from cheek cells, researchers found that one third of the locations they looked at had changed their epigenetic state between birth and 18 months of age, with the regions furthest from genes changing the most.

< TWINS REBECCA & ZOE WITH MUM VICKI KOUTS

This supports the idea that our genomes are most susceptible to the environment in the first 1000 days of life. Researchers also found twins can become more epigenetically different with age, but unexpectedly that some twins can become more epigenetically similar. Researchers say this may be because some twins have very different environments in the womb, but very similar environments after birth.

Hirschsprung disease is a disorder of the bowel in which the last part of the large intestine lacks a nervous system. The absence of the nervous system means that the bowel is unable to coordinate its normal muscular activity, called peristalsis, which propels food along the intestinal tract. Without treatment the affected portion of the bowel forms a physiological block to the movement of food.

The researchers’ next focus will be on specific diet and lifestyle factors of mums and babies, which may have the capacity to change their epigenetics and possibly influence future health.

The novel treatment developed and trialled by researchers involves reintroducing a new enteric nervous system in the non-functioning part of the bowel. Nerves can be obtained from nerve stem cells, but the question is: how do you get them into the bowel? The study, published in the Biomaterials journal, described a successful method of introducing nerve stem cells into the bowel of mice. In this treatment the nerve stem cells are grown on a biodegradable mesh, made by Monash Universitybased polymer chemists, and introduced into the bowel by wrapping the outside of the bowel. The next step for our researchers is to see if the finding translates when using human nerve stem cells in the patient’s own tissue.

NOVEL TREATMENT POSSIBILITY FOR HIRSCHSPRUNG DISEASE Our researchers are working on an innovative treatment for Hirschsprung disease, which could mean children would not have to undergo surgery for the condition. While the current surgical treatment is life-saving, many children have ongoing problems which persist into adolescence and sometimes into adulthood.

annual report 2013

clinical sciences GLOBAL BURDEN OF DISEASE STUDY Our researchers were collaborators on the international Global Burden of Disease, Injury and Risk Factor Study. The study brought together a large community of experts and leaders in epidemiology, economics, statistics and other disciplines to measure levels and trends in all major diseases, injuries and risk factors. The study produced new and comprehensive sets of estimates and easy-to-use data visualisation tools for research and teaching. In total, the study generated nearly one billion estimates of health outcomes. A total of seven articles from the Global Burden of Disease Study (GBD) 2010 study were published in The Lancet. The GBD 2010 is the largest ever systematic effort to describe the global distribution and causes of a wide array of major diseases, injuries and health risk factors. The results show that infectious diseases, maternal and child illness, and malnutrition now cause fewer deaths and less illness than they did 20 years ago. As a result fewer children are dying every year, but more young and middle-aged adults are dying and suffering from disease and injury as noncommunicable diseases, such as cancer and heart disease, become the dominant causes of death and disability worldwide. Since 1970, men and women worldwide have gained slightly more than 10 years of life expectancy overall but they spend more years living with injury and illness.

BRAIN INJURY HAS IMPLICATIONS ON LANGUAGE AND VERBAL SKILLS Research by the Institute found preschool-aged children with moderate and severe traumatic brain injury (TBI) suffer greater verbal and language impairments than children with mild brain injury, but those with mild brain injury perform similar to uninjured children. The study found that language appears vulnerable to brain injury and should be investigated as a matter of course in clinical assessments of TBI recovery. Lead researcher Dr Louise Crowe said that while previous research has suggested TBI places children at risk of language impairments, the majority of these studies have been conducted with school-aged children. “We know that TBI occurs frequently in young children, with some research showing that children younger than three years have the highest rates of brain injury. So it was important for us investigate the impact of brain injury during this pivotal time in language development.” In a follow-up study, researchers will now track children every two years to clarify whether language skills in children with moderate-severe TBI decline or remain stable over time. The study will include the use of imaging technology to enhance the understanding of brain injury in childhood and language development. The paper was published in the Journal of Head Trauma Rehabilitation.

REPORT HIGHLIGHTS DISPARITIES FOR PEOPLE LIVING WITH CEREBRAL PALSY Social, financial and educational disparities of people living with cerebral palsy (CP) were highlighted in a report that was led by the Institute. Improvements in paediatric care have increased the survival rates of children with CP, such that 90 per cent of children are now expected to live into their adulthood. The Institute’s study investigated the quality of life, educational and social outcomes of 335 young adults with CP and compared this with more than 2000 people living without disability in Victoria. The study found that although two thirds of young adults with CP received their education in mainstream schools, only 50 per cent completed secondary school. The highest educational level achieved by participants with CP varied according to functional ability. Those who had the most severe functional impairment were 25 times less likely to have completed secondary school. Employment rates were also much lower for CP patients, with only 36 per cent of young adults with CP being employed. The proportion of those never having sought employment (54 per cent) was nearly four times of that of their peers. The majority of people living with CP also had greater financial inequalities, with nearly 80 per cent having an annual income of less than $20,000. Lead researcher Professor Dinah Reddihough said that while intellectual impairment experienced by some individuals with CP would inevitably be a barrier to educational achievement, independent living and employment options, the study highlighted the indisputable disadvantage of those living with CP. “This study really highlights the need for efforts to improve self-care independence and strategies for increasing educational levels, participation in employment, and social opportunities.”

PREMMIE BABIES STILL LAGGING BEHIND WHEN THEY START SCHOOL A study by the Institute and the Royal Women’s Hospital found babies born extremely early or small are still lagging behind their term-born counterparts by the time they reach school age. The study, which included 189 children born extremely early or small (prior to 28 weeks or weighing less than 1000g) and 173 term-born children, found 71 per cent of the children born premature experienced a cognitive, educational or behavioural impairment at eight years of age, and worryingly, 47 per cent exhibited multiple areas of concern. Children in the study were assessed on their intellectual ability, spelling, reading, mathematics and on a range of behavioural outcomes including emotional symptoms, hyperactivity and peer relationship problems. Despite improved care and recent changes in obstetric and neonatal management, the study found the children who were born extremely premature performed significantly below their term-born peers on all cognitive and academic assessments, with the rates of reading and spelling impairment approximately double the rate of the term-born group. Lead author Dr Esther Hutchinson said the research highlights two important messages. “The good news is most babies born extremely early or small now survive, and the vast majority of the surviving babies escape major disabilities such as cerebral palsy, deafness and blindness. The concerning news however is that nearly half of these children will have multiple intellectual, educational and behavioural issues, and as such are disadvantaged when they reach school.” Senior author Associate Professor Peter Anderson said despite medical advances in obstetric and neonatal care, the findings reinforce our need to persist with efforts to enhance the quality of life of these children. The study was published in Pediatrics.

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< hamish & his family

FONTAN FOR LIFE Scans while mum Jodie was pregnant showed doctors that her little boy had a rare and very severe heart defect called hypo-plastic left heart syndrome. This means the left side of his heart did not form correctly as he developed in utero. Hamish was born in April 2008, and he was on the operating table having the first of many open-heart surgeries before the day was out. Surgeons told Jodie and her husband, Eddy, that Hamish was in a critical condition with a slim chance of pulling through. After nine and a half months spent at The Royal Children’s Hospital and many weeks in intensive care on life support, his parents were finally able to take their tiny boy home to Hobart, but his journey wasn’t over. At two years old Hamish was Australia’s youngest-ever Fontan patient. The Fontan procedure is a life-saving medical technique used to treat children born with certain serious heart defects. The procedure involves restructuring the heart so that it can operate effectively using only one ventricle, or pumping chamber, as opposed to two. Hamish is one of more than 900 children and adults who are taking part in the largest heart study of its kind in the world, the Fontan Registry, which is led by the Institute. The procedure is relatively new in medical terms; it has only been around for 40 years. The registry was established two years ago so researchers and surgeons could follow-up patients living in Australia and New Zealand, and improve their health outcomes and quality of life. Already the registry is seeing results; new research has found that doomsayers, who said the Fontan operation would last less than 20 years, have been proven wrong. In the largest follow-up study of its kind, and the only one based on entire populations, researchers found the best possible results in terms of survival for this group of heart patients. “We are now proving that the vast majority of these patients will survive for three decades,” Murdoch Childrens Associate Professor and heart surgeon Yves d’Udekem said. “It is therefore likely a lot of these patients will see their fourth and even fifth decade post-surgery.”

continue to grow and in 30 years will grow to more than 3000 patients in Australia “The registry is already improving the care of these patients,” Yves said. “However; I want to be able to tell my patients what kind of life they can expect and what effect this procedure will have on their heart, lungs, kidneys, liver and even their brain.” Hamish is five now, but as Jodie says: “Our little Hamish is a fighter, but ultimately, the medical complications he is faced with means that his life ahead has more battles to overcome which we truly hope and pray he will get through - although the journey is uncertain.” There will be constant monitoring, but the research done by the Fontan Registry gives hope for Hamish’s future. “The doctors haven’t been able to figure out why some babies’ hearts don’t develop properly genetics or just bad luck, but they are making progress and that’s good news for Hamish and the other precious and amazing children who are and have been on similar journeys. If he’d been born a decade ago, it’s doubtful he’d have made it.” Researchers are now undertaking a study that will investigate the liver and kidney function of patients who have undergone the Fontan procedure. The project is important in determining how these two major organs tolerate the higher vein pressures generated after Fontan surgery, and findings from this research will ultimately improve the care Fontan patients receive. In the future the registry will also be used to initiate large-scale trials for potential new medications and treatments. The collaborative work is the first of its kind in the world, with all cardiac specialists in Australia and New Zealand working jointly on the project.

> ASSOCIATE professor yves d'udekem

Further research from the registry has found the Fontan population is increasing rapidly. While this used to be a rare procedure, the population will

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genetics

STUDY SUGGESTS SLEEP-DISORDERED , BREATHING COMMON IN FRIEDRICH S ATAXIA

LINK BETWEEN DNA COMPLEX AND DISEASE UNCOVERED

KEY GENE FOUND FOR MITOCHONDRIAL DISORDER

Researchers at the Institute have conducted a world-first study into the condensin complex, which shapes our genetic material (DNA).

A team of researchers identified a new gene responsible for mitochondrial complex III deficiency, a devastating disorder that impairs energy generation. Complex III deficiency can lead to variable symptoms such as developmental regression, seizures, kidney dysfunction and frequently, death.

The study, which was published in Nature Communications, is the first to identify how the chromosome condensin complex binds to sites in the vertebrate genome, providing a vital missing link between chromosome structure abnormalities and diseases. These findings are particularly important as discovering where condensin binds in our DNA is essential to understanding these complex processes and also provides vital clues as to why mutations in condensin complex members are increasingly associated with disease. Previously research focused on understanding the role of condensin in the separation of DNA into daughter cells; however the new findings show that condensin is primarily located in gene rich regions. The team, led by Dr Damien Hudson, has contributed greatly to knowledge in this area and their work has resulted in the field moving from understanding how condensin segregates DNA, to concentrating on how it regulates our genes.

This work stemmed from earlier investigation, published in 2012, in which the team sequenced over 1000 genes in 42 patients with severe infantile mitochondrial disease. Led by Dr Elena Tucker, the new study investigated one patient from a consanguineous family that had a severe complex III deficiency. Using a combination of massively parallel sequencing and computational analyses, the team uncovered the two key proteins required for complex III, UQCC1 and UQCC2. The genetic basis of complex III deficiency is not fully understood with around half of cases having no known cause. This lack of genetic diagnosis is partly due to an incomplete understanding of the genes required for complex III assembly and function. Prior to this study, there were only three known factors required for human complex III assembly, but this work expands the number to five. As a result, researchers now have an improved understanding of complex III biogenesis. Establishing molecular diagnoses is important for patients and their families because it ends the long diagnostic journey, as well as the ongoing, invasive and risky investigations that children are often subjected to. Currently there are no effective treatments or cures for complex III deficiency. The discovery of new assembly factors, such as UQCC1 and UQCC2, enables a better understanding of this important enzyme which will hopefully aid the development of effective treatments in the future.

Results from a study conducted by our researchers show that a night time respiratory problem called obstructive sleep apnea occurs more frequently in people with Friedreich’s ataxia than in the general population. Friedreich’s ataxia is a neuromuscular disease that mainly affects the nervous system and the heart. People with the condition often die prematurely, around 40 years of age, due to heart complications. Obstructive sleep apnea is a disorder in which the muscles of the throat intermittently relax and block the upper airway during sleep, causing pauses in breathing. This can lead to a reduction in the amount of oxygen that makes it to vital organs and to irregular heart rhythms. Results from the study showed that 20 per cent of people with Friedreich’s ataxia suffered from some form of sleep disordered breathing which was compared with seven per cent of the general population. The presence of obstructive sleep apnea was significantly correlated with having had the disease for a longer period of time and with more severe symptoms. Researchers recommended that people with Friedreich’s ataxia undergo regular screening for obstructive sleep apnea to see whether they need subsequent treatment. The paper was published in Neurology.

MICROARRAY TESTING Genetics testing for chromosome abnormalities has undergone dramatic improvements over the last 10 years due to the development and use of microarrays. These are collections of hundreds of thousands of small stretches of DNA arrayed on a bead; their sequences and positions in the genome are known and are used to determine whether their matching sequences are present or absent in a patient’s DNA. These microarray tests use higher resolution which is at least ten times better than old tests and are therefore able to detect much smaller abnormalities. One of the main discoveries in genetics over the last 10 years has been that no two individuals, other than identical twins, have the same genome structure; specifically we all have hundreds of sequences that differ in copy number. When a microarray analysis is performed on a patient’s DNA, it is important to be able to distinguish what are harmless copy number changes from any that have a role in causing the patient’s

medical problem. These tests are commonly requested to investigate intellectual disability, developmental abnormalities, neurological disorders and cancer. This type of analysis has improved the diagnostic success rate from about 3.5 per cent using microscopy to about 15 per cent using microarrays. It has therefore been very attractive to provide this type of testing prenatally to improve the diagnosis of fetal abnormalities. However, when a rare variant is found it is difficult if not impossible to assess its significance, as the amount of detailed information on the fetus’ wellbeing is very limited beyond what can be surmised from ultrasound examination.

DR DAMIEN BRUNO & ASSOCIATE PROFESSOR HOWARD SLATER

In a study published in the British Journal of Obstetrics and Gynaecology, our researchers investigated whether using microarrays with very high resolution would be more suitable for prenatal diagnosis than arrays with lower resolution. Researchers concluded from the study and from their wealth of data on studying DNA from children, that almost all pathogenic copy number abnormalities can be detected with a low resolution array and thus the recurrent dilemma of interpreting new and incidental findings could be avoided. Victorian Clinical Genetics Services has been in a unique position to perform prenatal diagnosis using microarray analysis as its scientists and geneticists were the first to adopt this technology in Australia and have the most experience of this type of work.

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infection & immunity WORKING TOWARDS ZERO TUBERCULOSIS DEATHS

PREVENTING LUNG DISEASE IN CYSTIC FIBROSIS PATIENTS

Researcher Professor Steve Graham was a lead author on an ambitious international strategy that aims to reduce the burden of tuberculosis (TB) in children worldwide. Steve is currently supporting child TB research in a number of countries in the Asia-Pacific region as well as having provided technical assistance to many others including the African region.

Our researchers, in collaboration with the Queensland Children’s Medical Research Institute and the Telethon Institute for Child Health Research, are one step closer to preventing serious lung disease which is the main cause of suffering in cystic fibrosis.

The report, The Roadmap for Childhood TB: Toward Zero Deaths was co-authored by a number of health and advocacy groups, including the World Health Organization (WHO), Stop TB Partnership, UNICEF, USAID and the Treatment Action Group. The Roadmap is the first ever comprehensive plan to address TB in children which is ‘a hidden epidemic that is both avoidable and unacceptable’. The WHO estimates that there are about 500,000 TB cases in children each year and at least 74,000 deaths, though estimates are difficult due to problems of confirming diagnosis in children and common clinical overlap with other common diseases in young children such as pneumonia, malnutrition and HIV. A key step in the Roadmap is for countries in high burden settings to ‘know your epidemic’. The Roadmap lists 10 priorities or steps, including implementation of screening of children that are in close contact with TB cases – a policy that has been universal for decades but rarely implemented in high burden settings. It highlights the importance of much stronger collaboration and cooperation between the child health sector and the TB control programmes than currently exist in most settings to improve epidemiological data, implement policy, provide training and conduct operational research. The Roadmap was officially launched in October in Washington DC, USA.

The study sought to determine risk factors for the onset of bronchiectasis. Bronchiectasis is a progressive and persistent form of lung disease. Researchers studied 127 infants diagnosed with cystic fibrosis and found that they could determine which children were at most risk of developing bronchiectasis by three years of age. The study showed children with cystic fibrosis who have neutrophil elastase in their lungs at three months are seven times more likely to develop bronchiectasis by 12 months, and four times more likely by three years. Neutrophil elastase is one of the body’s mechanisms for destroying bacteria in the body. The levels of neutrophil elastase in the body is normally controlled, however in cystic fibrosis excess neutrophil elastase can cause breakdown of the lung structure. This leads to irreversible lung damage like bronchiectasis. Researchers say if they have a mechanism for mopping up neutrophil elastase then they could potentially stop or delay the start of damage to the lungs. The research was published in the world’s most prestigious clinical medicine journal, The New England Journal of Medicine.

VACCINE COULD REDUCE THE RATE OF ALLERGY & INFECTION IN CHILDREN A vaccine that was routinely used in Australia until the 1980s to prevent tuberculosis could reduce the rate of allergies and infections in children. In 2013, researchers from the Institute, the University of Melbourne and Mercy Hospital for Women launched a trial that will study the effect of giving babies Bacillus Calmette-Guérin (BCG) vaccine at birth. They believe the vaccine’s beneficial non-specific effects could boost children’s immunity to help protect against allergic disease and infections.

The Melbourne Infant Study: BCG for Allergy & Infection Reduction (MIS BAIR) will recruit 1400 newborn babies and give half of them the BCG vaccine. Researchers hope the study will show whether the vaccine could help reduce the rapidly increasing rate of allergic disease in Australia and other developed countries. Professor Nigel Curtis, from the Institute, who is leading the trial, says the vaccine is known to have a beneficial effect on immunity. “It could be a simple and safe once-off preventative measure to reduce the chance of children suffering infections and allergies in later life.” >

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< Dr andrew steer & Dr pierre Smeesters

DEVELOPING GUIDELINES FOR PNEUMONIA Researcher Dr Catherine Satzke’s work on the Pneumococcal bacteria has led her to arguably the world’s peak body on global health. In 2003, the World Health Organization (WHO) convened a working group to publish a set of standard methods for studies measuring nasopharyngeal carriage of the pneumococcal bacteria. A decade after the original guidelines were published, Catherine co-organised a meeting at the WHO in Geneva, leading the reconvened working group to update the consensus standard methods. Published in 2013, these guidelines set the standard for conducting studies to assess pneumococcal carriage and are important in ensuring that any findings are valid across different geographical settings. The methods describe the collection, transport and storage of nasopharyngeal samples.

DEVELOPING BEST WAY TO IDENTIFY DISEASE IN DEVELOPING COUNTRIES Our researchers are continuing their work in developing the best surveillance program for Group A Streptococcus ‘strep’ (GAS) in developing countries, where mortality from GAS disease is high. GAS-related disease, including rheumatic fever, skin infection and scabies, affects up to 18.1 million people worldwide, with over 500,000 people dying from these diseases each year. Developing a safe and effective vaccine against GAS will be the best way to prevent this disease. To be able to do so, researchers are trying to understand who develops strep and what strains of strep are the most common to cause childhood illness.

> PROFESSOR nigel curtis & kaya gardiner

The World Health Organization currently recommends that BCG vaccine be given at birth to all infants in most countries to protect against tuberculosis. It is given to about 120 million of the 133 million infants born every year, making it one of the most widely-used vaccines. This means that the safety profile is clearly established. As a result of the declining prevalence of tuberculosis, routine vaccination with BCG was

stopped in Australia and a number of other developed countries in the mid-1980s. “If giving the BCG vaccine at birth is found to be effective, it’s reintroduction into the routine Australian immunisation programme could lead to a reduction in allergic disorders in the population, and would result in a significant increase in quality of life for children and their families.”

The group, which is led by Dr Andrew Steer, has been working in Brazil and in the Asia Pacific region and has been awarded a National Health & Medical Research Council project grant to further develop their research line. These investigations into GAS are among the most detailed in developing countries to date and are of great significance to international efforts in developing new diagnostics and an effective global vaccine to control the disease.

Streptococcus pneumoniae (the pneumococcus) is the most common cause of pneumonia, the leading cause of death of children under five years old worldwide. The pneumococcal conjugate vaccine is being widely introduced in developed countries, and new vaccines are under development. Researchers at Murdoch Childrens play a pivotal role in understanding the vaccine effectiveness and the long-term impact of vaccines in these settings. The Pneumococcal Research group has an expanding program of work on vaccine trials and introduction studies globally, and are currently leading projects in five other countries. “By taking a global approach, we hope to make a real difference in reducing childhood deaths from pneumonia,” Catherine said. > dR catherine satzke

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population health LOW VITAMIN D LINKED TO FOOD ALLERGIES New research by the Institute has found a lack of vitamin D increases the likelihood of food allergy in infants. Researchers found infants who are vitamin D insufficient were three times more likely to have a food allergy. Those with vitamin D insufficiency were also more likely to have multiple food allergies, with the odds increasing to 10 times more likely among those with two or more food allergies. Researchers found no link with lack of vitamin D and eczema. Interestingly, the study showed the link was only evident for infants with vitamin D insufficiency with Australian-born parents, but not for infants of parents who were born overseas. Researchers hypothesise the different effect of vitamin D on food allergy depending on the parents’ country of birth may be related to skin colour or other genetic, epigenetic or environmental reasons. The study, which was published in the Journal of Allergy and Clinical Immunology, involved more than 5000 12-month-old infants. Infants underwent a skin prick test to common allergic foods and an oral food challenge to confirm allergy. Researchers then examined the blood of 780 infants within the study, and measured their serum level of vitamin D.

PROFESSOR katie allen

Lead researcher Professor Katie Allen said the rising prevalence of vitamin D insufficiency over the past 20 years has paralleled the rise in food allergy. “Food allergy is on the rise, and Australia has some of the highest reported prevalence in the world with more than 10 per cent of infants having food allergy. There has also been a rise in vitamin D insufficiency, with up to 30 per cent of Melbourne pregnant mothers now vitamin D insufficient.”

< ALLERGY PARTICIPANT BENJAMIN

SOCIO-ECONOMIC DIFFERENCES IN WEIGHT SEEN BY AGE FOUR Children from disadvantaged families are far more likely to be overweight and obese by the age of 10. Our research found the socio-economic differences in high body mass index (BMI) were already present at age four. The study also found these differences not only persisted but more than doubled by 10 years of age. Researchers studied more than 4000 children from the age of four until they were 11 years old. The national study showed that the average BMI was markedly higher for children of low socioeconomic families at every age. This suggests that childhood has now become the critical time when socioeconomic inequalities and BMI association develop and strengthen. Researchers found disadvantaged children who were already overweight or obese by preschool had the most rapid increase in their weight. However, they were also overrepresented in a second group of concern – those who rapidly developed overweight from middle childhood. This study is the first Australian study to follow children longitudinally, since they were four years old, so researchers could see for the first time in Australian children how overweight and obesity develop and track through their life course. Lead researcher Professor Melissa Wake said the study highlighted that the risks of children developing or being persistently overweight or obese was significantly higher in the most disadvantaged children as opposed to socially advantaged children.

“This study provides the first direct evidence that vitamin D sufficiency may be an important protective factor for food allergy in the first year of life. This adds supporting evidence for medical correction of low vitamin D levels.”

“Clearly targeting children with early overweight and low socioeconomic background, particularly those from socially disadvantaged families, must be a top intervention priority. However, this alone won’t suffice, because vulnerability to unhealthy weight gain increases incrementally with each quintile further from the most advantaged. Only whole-society approaches are going to eliminate overweightassociated morbidity.”

This research was done in collaboration with the University of Melbourne.

The study was published in PLOS One and was part > of the Longitudinal Study of Australian Children.

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,, SHARED CARE OBESITY TRIAL UNSUCCESSFUL Researchers found a “shared care” obesity treatment trial, in which hospital-based obesity specialists and local GPs shared the management and treatment of obese children, was ineffective. The study, which was published in the British Medical Journal, showed that the shared-care obesity management program, provided to children aged three to 10 years old, did not improve children’s body mass index (BMI) relative to those who received no treatment for their obesity.

SLEEP PATTERNS TRACKED FOR FIRST TIME Sleep patterns of children and how many hours may be optimal for their health and wellbeing has been tracked and recorded for the first time by researchers at the Institute.

The trial was developed as a “best of both worlds” type approach, as it combined easy-to-access GPs with harder-to-access specialists. Only a small minority of obese children can access hospital-based weight management clinics due to location or the overwhelming need for those services (some states only have one specialist weight management clinic). Researchers were hopeful this approach would alleviate some of the stress that is currently on specialist weight management services, and would help those children who were unable to access specialist clinics. Interestingly, the study showed that 20 per cent of children reduced their BMI over the 12 month study, regardless of intervention. Researchers are now interested in investigating what biological and behavioural discoveries can be made from these children, and if there are unique periods in life when reducing BMI and maintaining this lower weight is easier to achieve. >

Until now, optimal childhood sleep parameters have been based on opinion rather than empirical evidence. To provide accurate data on age-specific sleep patterns in Australian children, researchers studied 10,000 children involved in the Longitudinal Study of Australian Children. From the age of four months to nine years, the researchers looked at duration of sleep, number and length of night wakes and number of sleep episodes. The study found a striking range in sleep patterns. Researchers found a huge variation at all ages in sleep duration, bedtime and wake time. From four months to nine years, 24-hour sleep duration fell from an average peak of 14 hours at four to six months to 10 hours at nine years. This was mainly due to a progressively later bedtime, shifting from 8pm to 9pm with age, and a declining length of sleep during the day from three hours to less than half an hour. The number and duration of wakes during the night also fell. Lead researcher Dr Anna Price said the study highlighted the wide range in “normal” child sleep across early childhood. In future research, these parameters will be used to empirically determine optimal child sleep patterns for child and parent outcomes such as mental and physical health.

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EARLY POOR MENTAL HEALTH LINK TO EARLY PUBERTY Children who have an earlier onset of puberty have poorer mental health from as early as preschool age, a study by the Institute found. The study, which followed almost 3500 children from age four through to 11, found boys with an onset of puberty by eight to nine years of age had greater behavioural difficulties and poorer emotional and social adjustment from early childhood (four to five years of age). This pattern continued through to early adolescence. Girls with early puberty had more difficulties in emotional and social adjustment from early childhood, but not the behavioural problems found in boys. Lead researcher Dr Fiona Mensah said the study provides new evidence of pre-existing and persistent early childhood differences in socio-

emotional wellbeing among children who experience early puberty. Professor George Patton says the study supports a ‘life course’ hypothesis, being that differences in pubertal timing and childhood adjustment may at least, in part, be the result of genetic and environmental factors early in life. “Understanding what lies behind early puberty may also tell us much about the origins of emotional and behavioural problems of children and adolescents.” The study was published in the Journal of Adolescent Health.

PRESCHOOLERS WHO STUTTER DO JUST FINE EMOTIONALLY & SOCIALLY Stuttering may be more common than previously thought, but preschool stutterers fair better than first thought, a study by the Institute, the University of Sydney and the University of Melbourne found. The study of more than 1600 children, which followed the children from infancy to four years old, found the cumulative incidence of stuttering by four years old was 11 per cent, which is more than twice what has previously been reported. However, the study refutes the long-held view that suggests developmental stuttering is associated with a range of poorer outcomes in the preschool period. The study found the reverse was true, with stuttering associated with better language development, nonverbal skills with no identifiable effect on the child’s mental health or temperament at four years old. But researchers found that recovery from stuttering was low; 6.3 per cent, 12 months after onset. Rates of recovery were higher in boys than girls, and in those who did not repeat whole words at onset than those who did. The study found boys were more likely to develop stuttering. Lead researcher Professor Sheena Reilly said parents could be happy in knowing that they could take a “watch and wait” approach to their child’s stuttering and it wouldn’t be causing harm to their child’s language skills or social and emotional development. Due to the low rates of recovery in the study, researchers were unable to determine what predicts which kids will recover from stuttering, but say this will be the focus of research moving forward.

MEET THE RESEARCHER: DR ANGELA MORGAN Dr Angela Morgan began her career as an acute care speech pathologist and says she has seen her work come full circle now that she is also a esearcher. Angela says she is now able to answer the questions she was unable to provide information on without research. “Families would ask me when their brain-injured child would eat and talk again, and I didn’t have the answers to what seemed like the most basic, but critical questions.” This thirst to know more led Angela into paediatric research, and she was determined to understand predictors of outcomes for children with brain injury and associated speech and swallowing disorders. In a world first, Angela and a team of researchers from the Institute identified prognostic markers in a child’s brain, which they say can accurately predict their speech outcome following a brain injury. These neurological predictors could help doctors to identify early who may need intensive speech therapy following a severe brain injury. Working in collaboration with the University College

London Institute of Child Health, the team used a novel approach by examining brain structure and function using magnetic resource imaging (MRI) on children at least a year post-injury. The study of brain structure revealed that the corticobulbar tract in the left hemisphere of the brain was crucial for good speech outcome. Angela said the research highlighted the importance of brain imaging techniques for providing an accurate prognosis of speech deficits. The study was published in leading scientific journals Neurology and Brain. Angela’s decorated career is rewarding. “When I see my research findings translated into clinical practice, it’s satisfying to know my work is improving health outcomes for children with speech or swallowing conditions.” Her work has been cited in several key clinical practice documents and guidelines internationally. Angela’s contributions were recognised nationally by her peers in 2012, when she received the Elizabeth Usher Speech Pathology Australia Award. In a personal highlight associated with her research career, Angela met the Queen during her postdoctoral work at University College London.

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research highlights The Institute is leading eight national Centres of Research Excellence (CRE), which are prestigious five-year grants awarded annually by the National Health & Medical Research Council (NHMRC). They provide support for researchers who pursue collaborative research to develop capacity in clinical population health and health services research. CHILD LANGUAGE

PNEUMOCOCCAL VACCINOLOGY

The CRE in Child Language unites 10 existing longitudinal studies and two large omnibus studies, which are based in Australia and the UK. Established in 2012, an important project within the CRE is to develop a short-form language measure that will enable researchers to gauge a child’s language ability using a suite of short tests. The team has also begun work on a project to collect DNA samples from children, providing an extremely rich data source that will enable researchers to advance the science of how language develops, what goes wrong and when and how to intervene.

International pneumococcal vaccine research has become a major program within Murdoch Childrens. This is appropriate as pneumonia, mainly pneumococcal in origin, continues to be the largest cause of child illness and death in the world. The Pneumococcal Research team has been involved in the field for 12 years across the world.

A Policy Practice and Implementation committee was established to ensure effective translation of research into health policy and practice. The committee focuses on knowledge exchange throughout the research generation phase into the practical and real world policy, practice and program mechanisms, primarily focusing within the state of Victoria.

CEREBRAL PALSY Using the globally recognised Cerebral Palsy Registers as a framework, the objectives of the Cerebral Palsy Centre will be achieved by undertaking targeted research to determine best treatments. The knowledge generated will then be made available to parents, to those working with individuals with cerebral palsy and to the worldwide community. Currently the number of researchers working in this field is small. The Centre aims to address this problem by providing training scholarships to increase the research workforce. The team will foster international collaborations to ensure that the most up-to-date information is available for Australian families and for medical and allied health professionals. The Centre lays the foundations for significantly improving the care of children with disabilities now and into the future.

In Vietnam, a Pneumococcal Vaccine Trial will for the first time allow researchers to compare two newgeneration pneumococcal vaccines head to head while exploring innovative dosage regimens. This ground-breaking trial has been established in Ho Chi Minh City. The team continues to build on a decade of work in Fiji, leading to the introduction of pneumococcal, rotavirus and HPV vaccines. This work makes Fiji a pioneer in new vaccine introduction in the Asia-Pacific region. In 2013 the team, led by Professor Kim Mulholland, also continued projects in Indonesia, Laos and Mongolia. In addition, the Institute has active pneumococcal research in Gambia, and planned new projects in India, Ethiopia and the Philippines.

BIOSTATISTICS ViCBiostat, which involves researchers from the Melbourne Children’s campus in collaboration with Monash University, aims to build the discipline of biostatistics in Victoria and Australia, with a focus on methodological research and high-level training of professional biostatisticians and a strong emphasis on collaboration and translation with the ultimate aim of improving the population’s health. Dr Ian White of the Medical Research Council Biostatistics Unit, Cambridge, who is a leading international researcher in biostatistics, visited the team in 2013, presenting lectures and leading a short course on “handling missing outcome data in randomised trials” as well as discussing collaborative

research with the VicBiostat team. In 2013, ViCBiostat researchers were Chief Investigators on NHMRC Project Grants worth a total of $6.2 million.

EMERGENCY MEDICINE The Paediatric Emergency Medicine CRE (PEM-CRE) brings together the leading paediatric emergency medicine researchers in Australia and New Zealand. The group is investigating the best methods to transfer knowledge into practice in all emergency department settings in Australasia. Under the umbrella of the Paediatric Research in Emergency Departments International Collaborative (PREDICT) these researchers aim to initially provide evidence for the acute management of head injuries, a study still in progress, and prolonged seizures, which will commence in mid-2014.

> Emergency CRE Head dr Ed Oakley

The PEM-CRE will partner with mixed emergency departments and experts from other fields as it moves to improve care for all children in emergency departments in Australia and New Zealand. The group is planning to work with an international knowledge translation expert to assist in the implementation of their research. >

annual report 2013

NEWBORN MEDICINE

FOOD & ALLERGY

KIDS PLAY ON DESPITE CONCUSSION

The CRE in Newborn Medicine aims to improve physical, cognitive, psychological and other health outcomes for newborn babies and their families. The team works to ensure effective treatments are introduced efficiently into clinical practice and ineffective or harmful treatments are abandoned. In a major highlight for 2013, the trial results of Brett Manley’s PhD were published in the New England Journal of Medicine.

The Centre for Food & Allergy Research had a busy year in 2013. Since the group’s establishment in February it has had a strong presence at national and international conferences. A group of researchers was invited to present at the European Academy of Allergy and Clinical Immunology/ World Allergy Organization (WAO) Congress in Milan, as well as the Australasian Society of Clinical Immunology and Allergy (ASCIA) annual conference in Perth. Collaboration has been of key importance, with researchers working alongside Allergy & Anaphylaxis Australia during Food Allergy Awareness Week and the World Allergy Organization during Allergy Awareness Week.

On-field management of sport-related head injuries in children does not follow international guidelines, a study by the Institute found. Many children continued to play on despite signs of concussion. The study looked at children who presented to the emergency department at The Royal Children’s Hospital with sport-related head injury.

During the year, the Victorian Infant Brain Study was awarded its third NMHRC project grant to conduct a 13-year follow-up of this unique study. This research group looks at brain injury and early brain development in premature and sick infants using state-of-the-art neuroimaging techniques.

Overall, more than 60 peer-reviewed papers were published, with almost one-quarter of these in the top-ranked journals in the allergy discipline. A submission was made to Food Standards Australia New Zealand (FSANZ) on minimum age labelling of infant foods and the team have a number of other translational activities in progress. In 2014, the team will host its inaugural research symposium and research training day, featuring international experts in the field of food allergy research.

NEUROMUSCULAR DISORDERS The CRE in Neuromuscular Disorders (CRENMD) represents a critical step in the care and treatment of people with neuromuscular disorders. The Centre’s research program aims to translate ‘best evidence’ from clinical and laboratory-based research into ‘best clinical practice’ under two themes of diagnosis and treatment. The CRE-NMD will instigate a national collaborative diagnostic network to ensure that all patients have access to an accurate genetic diagnosis to inform prevention through prenatal diagnosis and to guide prognosis and surveillance. The establishment of an Australasian Neuromuscular Network (ANN) underpins the successful translation of clinical and research outcomes from the CRE-NMD into practice, using a unified and coordinated framework. The ANN has grown to more than 350 members in 2013, ranging from clinicians and researchers to advocacy and patient-support organisations. The network is increasingly recognised as a peak body representing neuromuscular disorders.

The study, which is ongoing, found for organised sports, overall 41 per cent did not comply with internationally recommended concussion guidelines, 27 per cent were not immediately assessed by qualified personnel and 28 per cent were allowed to return to play on the same day of their injury. Also, 91 per cent of parents and 96 per cent of patients were unaware of any concussion or return-to-play guidelines from their organisations. At follow-up, 72 per cent had followed a return-to-play process as recommended based on international guidelines. Injured children should slowly proceed from rest, to mild exercise to full-contact game play over a minimum of one week.

INDONESIA & NEW ZEALAND PLAY MAJOR ROLE IN DEVELOPMENT OF NEW ORAL ROTAVIRUS VACCINE The rotavirus team at the Institute celebrated a major milestone in the development of a new oral rotavirus vaccine candidate, RV3, in 2013 with the launch of phase two of clinical trials in Indonesia. The vaccine candidate has the potential to save the lives of half a million children who die from rotavirus ‘gastro’ diarrhoeal disease every year worldwide. The RV3 Rotavirus Vaccine candidate has been developed from a unique strain of rotavirus that was found naturally in healthy asymptomatic newborn babies, who were then protected from severe rotavirus diarrhoea in the first two years of life. The aim is that RV3 will be an effective and affordable vaccine, designed to be given orally to babies from birth to provide the earliest possible protection. This world-first approach has enormous potential to reduce disease and deaths of the most vulnerable children around the world. The RV3 vaccine has already been shown to be well tolerated in a single dose to adults, children and babies in Melbourne, Australia, and a clinical trial has been successfully running since 2011 in New Zealand, administering the vaccine to newborns. The vaccine candidate is the culmination of almost four decades of research in Australia by the Institute, the Royal Children’s Hospital Melbourne and the University of Melbourne, following the discovery of rotavirus by a team of staff led by Professor Ruth Bishop in 1973.

NEW GENETIC CARRIER SCREENING OFFERED PUBLICLY FOR THE FIRST TIME In 2013, for the first time in Australia, genetic carrier screening for three common inherited conditions was made accessible to the public via a simple blood test. Until this point the genetic carrier test for cystic fibrosis, fragile X syndrome and spinal muscular atrophy had only been available to families who had a direct link, such as an immediate family member, with the condition. However, genetic experts pushed for the test to be available more broadly because many people are carriers of CF, FXS or SMA even though they do not have anybody in their family who has the condition. Currently in Australia one in 25 people are carriers for cystic fibrosis, one in 150 are carriers for fragile X syndrome and one in 40 are carriers for spinal muscular atrophy. Associate Professor David Amor, Director of Victorian Clinical Genetics Services, which is based at Murdoch Childrens, said they are offering screening for the three conditions because the conditions have a big impact on people and their families.

annual report 2013

, vcgs director s report 2013 Victorian Clinical Genetics Services is a subsidiary of Murdoch Childrens and provides not-for-profit clinical and laboratory genetics services, with an emphasis on innovation and translating the latest research discoveries into clinical care. Our services are provided through six diagnostic laboratories based at the Institute and through clinical services located at multiple sites across Victoria, Tasmania and a new agreement to provide clinical genetics services to the Northern Territory that will commence in 2014.

his is an era of unprecedented change in genetictesting technologies, which continues to impact all parts of our service. In 2013 this impact was greatest in the area of prenatal diagnosis, with the availability for the first time in Australia, of NonInvasive Prenatal Testing (NIPT) for Down syndrome. The NIPT test uses new gene-sequencing technology called Next Generation Sequencing, to measure foetal DNA that is circulating in the maternal blood. In 2013 VCGS started providing NIPT in partnership with US company Natera, and in 2014 we hope to commence NIPT testing in our own laboratories. Despite the absence of any government funding, NIPT is being rapidly adopted by pregnant women in Australia and is widely regarded as the future of prenatal testing. The other major clinical use of Next Generation Sequencing is as a method of examining large numbers of genes, or even an entire genome, in a single test. VCGS has piloted the use of this technology by focussing inherited forms of heart disease, including genetic causes of sudden cardiac death, and is now providing a clinical service that includes more than 100 cardiac genes. In 2013 this test became the first Next Generation Sequencing test to receive formal accreditation through the National Association of Testing Authorities (NATA), and VCGS is now receiving requests for this test from across Australia and overseas. Most importantly, results from these tests are changing the management for families affected by genetic heart disease and sudden cardiac death. In 2013 our laboratories also launched the new VCGS Reproductive Genetic Carrier Screen, a test designed for women who are planning a pregnancy or who are in early pregnancy. The Reproductive Genetic Carrier Screen combines testing for three serious genetic conditions: cystic fibrosis, spinal

muscular atrophy and Fragile X syndrome, and is backed up by support from our counselling team. This new testing program has received strong support from the obstetric community and is well on the way to becoming part of standard pregnancy care. A major milestone for VCGS laboratories occurred in September when we switched on our new Laboratory Information Management System (LIMS). LIMS is an IT system that provides the backbone for data management in our laboratories, uniting all six VCGS laboratories under one comprehensive shared reporting system. The new LIMS is the culmination of more than two years work and will greatly enhance communication and productivity between all VCGS laboratories and clinicians. Our clinical services continue to provide a full range of clinical and metabolic genetic services. In 2013, some consolidation of our metropolitan clinic locations has seen the end of formal involvement of VCGS at Monash Health, where we have played a major role in developing the service since its inception. VCGS will continue to take pride in this achievement, and in the knowledge the Monash Genetics service is now sufficiently strong to function independently. VCGS will maintain ongoing close working relationships with genetics staff at Monash Health. VCGS also strengthened its partnership with the Genetic Support Network of Victoria (GSNV), which is housed within the Institute. GSNV represents and supports individuals, families and support groups affected by or working with genetic/rare conditions, and receives operational support from VCGS to help achieve these goals. GSNV also provides assistance for setting up condition-specific support groups and on-going in-service training for the volunteers

involved. As a peak body, GSNV provides a respected voice and opinion on behalf of Victorian consumers and aims to contribute to the development of effective state and national policies. The latest version of POSSUMweb, our dysmorphology database of genetic syndromes, is receiving very positive acceptance worldwide. Feedback has noted the flexible format, ease of use, and the ability to access the platform from many different devices, which has greatly enhanced its performance. POSSUM has been a trusted source of specialist genetic information and has been used worldwide for more than 20 years, testimony to the calibre and quality of VCGS researchers involved in this ongoing project. VCGS continues to be a leader in genetic education and research, training the next generation of genetic scientists, counsellors and clinicians. The Master of Genetic Counselling continued to attract strong applications from within Australia and internationally. Graduates from 2012 and 2013 are already employed in a variety of positions throughout Australia and overseas. In 2013 VCGS staff made a number of exciting research discoveries and published a total of 75 scientific publications, many in prestigious international journals. With the appointment of new Murdoch Childrens Director Kathryn North, 2013 saw an increased focus on genetics and genomics at the Institute, highlighting the importance of VCGS to the strategic vision and generating an atmosphere of excitement within VCGS. I thank our Board, Executive Committee and all our staff for their ongoing support and commitment to providing the highest standard of clinical and laboratory genetics services and patient care. Associate Professor David Amor Director, VCGS

annual report 2013

research support & operations The role of Research Support and Operations is to improve the efficiency and effectiveness of our researchers through support and excellent research resources, systems and processes.

2013

was truly a transformative year for the Research Support and Operations team. It was a great pleasure to join as Chief Operating Officer at the beginning of the year and I was impressed by the commitment of the Institute to establish more efficient resources, systems and processes. Our key goal of “supporting great research” necessitated a change of name to Research Support and Operations (RS&O) in April, which preceded exciting changes to our structure and processes, including the appointments of new members to the management team. Of key importance has been equipping our researchers with the resources and infrastructure to support our strategic initiatives and to create large genomic and patient databases. Under the management of new Chief Information Officer Wayne Mather, the Information Technology group underwent a significant review and restructure. This has enabled us to expand our current services and systems in line with the huge expansion in amounts and flow of data required by our researchers, especially those in the bioinformatics and imaging areas. Now that we are settled in our impressive office and research facilities, we have been able to focus on maximising our space and modernising equipment. In 2013 the Biobanking storage strategy commenced, which will give

us a facility capable of storing more than 200,000 samples. This state-of-the-art resource includes ultra-cold storage units and robotic sample handler. Scientific Services continues to play an important role in maintaining core equipment and services across campus. The team has commissioned a new cuttingedge cell sorting instrument to complement our existing Flow Cytometry facilities, which will increase researchers’ efficiency and capability to simultaneously sort up to six cell types at high speed. 2013 also marked the establishment of a dedicated genomics facility with the aim of incorporating genomic analysis into a “personalised medicine” framework. We made great advances in our translation and commercialisation area, including the formalisation of our relationship with Curve Tomorrow, a technology development and innovation company. The Curve Tomorrow team is working closely with our researchers to develop new mobile and tele-health applications for use by clinicians, researchers, patients and families. Aacorn, an augmentative assistive communication app for kids developed in collaboration with Aacorn LLC, was launched on the iTunes store. The Commercialisation team also launched the Translational fund – an internal fund established to support translation of the Institute’s most promising research into outcomes that will benefit society. Other highlights include the commencement of phase two of clinical trials in Indonesia and New Zealand of the rotavirus vaccine and the growing of our intellectual property portfolio to 18 patents. The Institute is committed to providing a safe and supportive working environment to our researchers and staff. Under new leadership, the Human Resources team leads positive changes to our culture and leadership development. The team ran 11 different leadership development workshops in 2013, with more than 350 staff and students participating across the year. A DNA of Management Program was extremely popular as a way of upskilling leaders. The Engagement and Development team expanded, allowing us to pursue more innovative and novel

approaches to reach out to existing donors and the general public. Our internal and external communications functions have been brought together to further strengthen our media, online and social media presence. Events such as Discovery Day offered the opportunity for us to connect with the community, and our various campaigns were once again pivotal in retaining the support of long-time donors. Step-athon was launched as national campaign for primary school children to help teach them the importance of physical activity and to raise awareness for the Institute’s work. We look forward to this event growing in the coming years. Due to the efforts of our dedicated team, the year’s fundraising activities proved highly-successful. More than $6.9 million was raised through support from donors, corporate support and trusts and foundations in 2013. Although the Institute receives support from governments and grant-making bodies, the support we receive from philanthropic and corporate funds are needed to bring our research ideas to life. Thank you to each one of

our donors for their support over the year. All in Research Support and Operations should be proud of the huge achievements made in 2013. Our ongoing dedication to supporting the Institute’s researchers is vital to improving the health and wellbeing of children. Mr John Dakin Chief Operating Officer

annual report 2013

core groups QUANTITATIVE SCIENCES Bioinformatics

The Bioinformatics research group focuses on the analysis of data from high-throughput genetic technologies in collaboration with a diverse range of medical research groups. By being embedded in a medical research environment, the group produces highly relevant methods and analysis for a range of projects. In 2013, the Bioinformatics group continued to fulfil a pivotal role in several collaborations throughout the Institute, cementing its position at the forefront of bioinformatics research methodology. The group now comprises eight members including post-docs, PhD students and research assistants. The team was involved in more than 15 journal publications and presented at several national and international conferences. Dr Nadia Davidson was awarded the Harold Mitchell Foundation travel fellowship and also received a grant from the Swiss National Science Foundation to fund a research trip to Zurich.

Clinical Epidemiology & Biostatistics (CEBU)

The Clinical Epidemiology and Biostatistics Unit (CEBU) provides expertise in biostatistics, study design and data management to a wide range of research at the Institute, with a primary focus on clinical and population health studies. The team collaborates with many research groups and also conducts its own research to evaluate and extend biostatistical methods. In what was a very busy year, CEBU researchers were co-authors on 84 publications, including seven as first authors. The team was successful as co-investigators on six new National Health & Medical Research Council (NHMRC) Project Grant applications. Four members of the CEBU team presented their research at the annual conference of the International Society for Clinical Biostatistics (ISCB) in Munich, Germany, including PhD student Cattram Nguyen who received a Student Conference Award from the ISCB to attend. In October, CEBU moved to spacious new quarters in the refurbished South Building, as the first phase of development of the Melbourne Children’s Trials Centre, in which it will play a key role in 2014 and beyond.

Statistical Genetics

The Statistical Genetics group conducts research in statistical population genetics with a major focus on statistical methods development. In particular, the group works on the typing of immune system genes, detecting and controlling for population stratification, and detecting complex associations between genes and disease. These methods are also applied to large cohort studies of disease and natural genetic variation. The team is a small but successful one. In 2013, group leader Dr Stephen Leslie was awarded an NHMRC project grant to support his method-ological research on controlling for fine-scale population structure in genomic data. His work was published in journals such as Stem Cell and PLOS Computational Biology. Dr Leslie, along with the team’s other members, Dr Damian Vukacevic and Dr Alan Motyer, presented at various seminars in Australia and abroad.

The Statistical Genetics group will continue to collaborate with research groups nationally and internationally, both on methods development and the application of their methods to data.

Australian Paediatric Pharmacology Research Unit (APPRU)

Clinical Research Development Office (CRDO)

Headed by Associate Professor Noel Cranswick, the Australian Paediatric Pharmacology Research Unit (APPRU) performs high-quality, timely, clinical trials in children. The team provides clinical trial services and personnel who are highly experienced in phase I to phase IV trials and issues relating to good clinical practice (GCP), Case Report Form (CRF) management and subject recruitment.

The Clinical Research Development Office (CRDO) was established by Associate Professor Andrew Davidson to facilitate and increase capacity for clinical and public health research across the campus. It provides training, advice and guidance for clinical research staff on protocol development through to publishing their research findings.

APPRU also provides clinical pharmacology, research and development, clinical trial design and protocol development, GCP compliance, pharmacokinetic modelling and regulatory affairs strategy. 2013 was a very busy year for APPRU. The team worked on more than 25 clinical trials involving clinical specialties such as haematology, metabolics, dermatology, gastroenterology and neurology. A number of new nursing staff joined the team and their contribution has been vital in ensuring the successful operation of APPRU.

A key achievement for the CRDO during the year was the introduction of a new format of workshops; interactive, activity-based classes covering topics such as Good Clinical Practice and Ethical Principles. These were extremely well received and imparted lasting knowledge to the attendees. In another new initiative, the CRDO continued to broaden its scope into community and population health research through popular Research Coordinators Forums, hosted monthly. The CRDO also supported the successful grant application for the new Melbourne Children’s Trials Centre, which will launch in 2014.

annual report 2013

financials at a glance of consolidated entry

statement of comprehensive income for the year ended 31 december 2013

where the money comes from government & other peer reviewed grants

other research income

where the money goes research

49%

58%

22%

finance income

contract research & clinical trials

2012

2013

2012

Revenue from research & clinical activities

93,877,155

85,027,134

70,435,550

63,358,981

Depreciation & amortisation

(5,635,262)

(4,535,606)

(4,817,210)

(4,110,184)

Other expenses for research & clinical activities

(95,409,072)

(90,070,625)

(73,650,670)

(68,760,259)

(7,167,179)

(9,579,097)

(8,032,330)

(9,511,462)

6,996,756

11,874,318

6,996,756

11,874,318

(1,310,832)

(1,041,916)

(1,310,832)

(1,041,916)

Net surplus obtained from Fundraising Activities

5,685,924

10,832,402

5,685,924

10,832,402

Finance income

3,678,667

5,877,173

3,525,440

5,598,621

3,678,667

5,877,173

3,525,440

5,598,621

2,197,412

7,130,478

1,179,034

6,919,561

1,806,561

(820,576)

1,806,561

(820,576)

4,003,973

6,309,902

2,985,595

6,098,985

7% technical & scientific services

donations, fundraising & bequests

administration

Donation, Estates & Bequest & Fundraising income Fundraising expense

1% fundraising

REVENUE 2012 ($M)

2013 ($M)

Government & other peer reviewed grants

46.98

51.53

Clinical services

21.67

23.44

Finance expenses Net Finance income / (expense)

Donations, fundraising & bequests

6.07 (11.87*)

7.00

Finance income

5.88

3.68

Contract research & clinical trials

9.09

8.90

Other research income

7.29

10.00

Other comprehensive income:

96.98

104.55

Net change in available for sale financial assets

Total Revenue

The Company

2013

Surplus/(Deficit) from research & clinical activities

12% 4%

Consolidated

clinical services (VCGS)

10%

clinical services (VCGS)

murdoch childrens research institute and its controlled entities

*$11.87M includes $5.8M campaign funds

Operating surplus for the period

Total comprehensive surplus for the period

EXPENDITURE

2012 ($M)

2013 ($M)

Research expenditure

53.11

59.22

Clinical services

21.74

22.58

Fundraising

1.04

1.31

Administration

6.90

7.21

Facility, technical & scientific services

12.86

12.05

Total Expenditure

95.65

102.36

annual report 2013

murdoch childrens research institute and its controlled entities

balance sheets as at 31 december 2013

statements of cash flows for the year ended 31 december 2013

Consolidated

The Company

2013

2012

2013

2012

2013

2012

2013

2012

Patient fees received

16,537,981

14,194,882

Government and other grants received

63,693,281

54,924,398

55,272,200

47,042,523

7,003,088

11,886,775

6,996,756

11,874,318

911,563

1,215,000

791,563

1,215,000

15,899,723

15,725,654

17,779,728

17,557,941

(94,929,194)

(92,671,244)

(74,280,692)

(71,794,267)

(67,073,043)

9,116,442

(61,797,578)

6,559,555

(61,177,528)

411,693

278,552

398,845

(527,302)

1,111,308

6,577,632

22,838,981

6,577,632

22,838,981

Acquisition of property, plant and equipment

(3,964,319)

(6,019,225)

(3,020,574)

(3,507,798)

Proceeds from redemption of term deposits

69,468,653

Acquisition of investments

(8,136,005)

(26,745,785)

(8,136,005)

(26,745,785)

NET CASH FROM/ USED IN INvesting activities

5,110,999

59,821,176

(4,707,404)

63,165,359

Repayment of borrowings

NET CASH USED IN FINANCING ACTIVITIES

4,005,443

(1,976,402)

1,852,151

1,987,831

13,709,229

15,685,631

10,233,110

8,245,279

17,714,672

13,709,229

12,085,261

10,233,110

CURRENT ASSETS Cash and cash equivalents

17,714,672

13,709,229

12,085,261

10,233,110

Trade receivables and other assets

11,619,861

8,029,109

9,261,847

5,107,130

Other investments

20,448,472

18,867,087

19,898,336

18,337,330

Total current assets

49,783,005

40,605,425

41,245,444

33,677,570

Cash flows from operating activities

Donations received Interest received

NON-CURRENT ASSETS

Other receipts

Trade receivables and other assets

64,390,121

Cash paid to suppliers and employees Payment of right of occupancy

63,048,660

64,390,121

63,048,660

Other investments

38,826,385

34,928,614

38,826,385

34,928,614

Property, plant & equipment

17,766,105

18,095,587

14,246,447

14,701,622

119,641,150

117,414,322

116,121,492

114,020,357

169,424,155

158,019,747

157,366,936

147,697,927

Trade and other payables

28,741,666

21,939,770

27,427,830

20,982,325

Employee benefits

10,082,282

9,526,305

6,082,819

5,867,463

Total current liabilities

38,823,948

31,466,075

33,510,649

26,849,788

Total non-current assets TOTAL ASSETS CURRENT LIABILITIES

NON-CURRENT LIABILITIES Employee benefits

1,421,402

1,378,840

1,170,855

1,148,302

Total non-current liabilities

1,421,402

1,378,840

1,170,855

1,148,302

40,245,350

32,844,915

34,681,504

27,998,090

129,178,805

125,174,832

122,685,432

119,699,837

Total liabilities NET ASSETS

MEMBERS’ FUNDS Accumulated funds

127,621,166

95,164,927

121,167,338

89,729,477

1,518,094

(288,467)

1,518,094

(288,467)

Capital reserve

400,000

Permanent investment funds

26,258,827

Building development fund

2,600,000

Fellowships & scholarships fund

1,000,000

39,545

Fair value reserve

Translational Research Fund TOTAL MEMBERS’ FUNDS

The Company

129,178,805

125,174,832

122,685,432

net cash provided FROM/ used in OPERATING ACTIVITIES cash flows from investing activities Investment income received Advances to (from) related parties Proceeds on sale of investments

CASH FLOWS FROM FINANCING ACTIVITIES

NET INCREASE/ (DECREASE) IN CASH AND Cash Equivalents cash AND CASH EQUIVALENTS AT 1 JANUARY

cash AND CASH EQUIVALENTS AT 31 DECEMBER

119,699,837

annual report 2013

students STUDENT IN PROFILE: MATT BIRD Researcher Matt Bird discovered his love of science by chance during high school work experience at St Vincent’s Institute for Medical Research. “In those two weeks I had a ball, I was sold,” says the third year PhD student. Since then Matt has developed a particular interest in mitochondria. “I have a drive to understand how all the machinery in cells works, and what happens when it breaks down. As a side interest, I’m also getting into learning how the human body develops. It’s mind-blowing to think that it all comes together from a single cell.”

Mitochondrial research at Murdoch Childrens, led by Professor David Thorburn, extends from being the Australasian referral centre for diagnosis of resulting disorders in children through to identifying the genetic basis and the specific mechanisms causing disease. The goal is to improve reproductive options for families and to develop new approaches for effective therapy. Under David’s guidance, Matt is working to understand why mitochondrial disease in children disproportionately affects the brain. This was achieved by looking at the function of patient’s brain cells made from skin, and brain cells from a mouse with mitochondrial disease, and the work is currently being reviewed for publication. Matt has been selected to attend the 64th Nobel Laureate Meeting in Germany in 2014, giving him the chance to meet Nobel Prize winners in the fields of medicine and biology. He will join about 600 young researchers to share knowledge, establish new contacts and discuss relevant topics such as global health, the challenges to medical care in developing countries or future research approaches to medicine. In the future Matt hopes to move into a role that helps facilitate better communication about medical research. “I think what scientists do is valuable. However, I think scientists should be encouraged to go beyond the lab bench and engage with the community and policy makers. We have much to share with these groups who fund our research, and much to learn from them to guide us.”

STUDENT IN PROFILE: ASHLEY DI BATTISTA PhD graduate Ashley Di Battista is motivated by her fascination with how the brain works. Originally from Toronto, Ashley was encouraged by a mentor at Toronto Sick Kids to pursue a career in clinical neuropsychology, which saw her head to Melbourne where she completed the combined Masters/PhD in Clinical Neuropsychology under the tutelage of experts, including Professor Vicki Anderson. But it was Ashley’s own experience with a chronic health condition that led her into a career in medical research. “I was exposed to hospitals and research at a very young age, and I started asking questions about medicine and wellbeing. Thankfully I had very encouraging healthcare providers and parents, who fostered my excitement to ask tough questions and get answers about the care I was receiving.” Ashley’s PhD aimed to understand how adolescents define their own quality of life after a traumatic brain injury. Her previous research had found that the majority of the available information about quality of life after brain injury in adolescents wasn’t actually from

young people but from their parents. The subsequent paper was published in The Journal of Neurotrauma. “I thought it was especially important to ask adolescents how life was for them after injury, using tools commonly available and reported on in the broader psychological literature, as well as using an interview I developed,” said Ashley, who is now back in Canada undertaking a postdoctoral fellowship at the Hospital for Sick Kids in Toronto. In her relatively short career Ashley has been featured in numerous journals and invited to talk at several conferences and seminars. She has been the recipient of prestigious awards and scholarships in both her native country and Australia, including the Canadian Institutes of Health Research Doctoral Research Award. “I consider myself very lucky to have a job I love, which continues to encourage me to ask difficult questions and find creative solutions, which can ultimately help to improve the lives of children and adolescents with chronic health conditions and diseases.”

annual report 2013

grants Murdoch Childrens was awarded $26.2 million by the National Health & Medical Research Council (NHMRC) in 2013 for new research projects commencing in 2014. This will fund 23 new research projects, 14 fellowships and scholarships and three Centres of Research Excellence.

FEDERAL > Department of Education, Employment and Workplace Relations > Department of Health and Ageing > Australian Department of Families, Housing, Community Services and Indigenous Affairs

STATE > Department of Education and Early Childhood Development > Department of Health

INTERNATIONAL COMPETITIVE FUNDING In 2013 Murdoch Childrens received $6.9 million in grants from international funding bodies. A large amount of support was from the Bill & Melinda Gates Foundation supporting our rotavirus and pneumococcal vaccine programs for children in developing countries. > Curesearch for Children’s Cancer > European Huntington Disease Network > Freidreich Ataxia Research Association, USA > Bill & Melinda Gates Foundation > March of Dimes > Marie Curie International Fellowship > Muscular Dystrophy Association, USA > National Institute of Allergy and Infectious Disease, USA > Parkinson’s Disease Foundation International Research Grants Program > Thrasher Research Fund > World Health Organization

COMPETITIVE GRANTS Our research was generously supported by many charitable foundations, which provided peer-reviewed funding for innovative research. Alfred Felton Bequest ANZ Trustees Australian Cystic Fibrosis Research Trust Australian Research Council Arthritis Australia Australian Rotary Health Bone Health Foundation Bone Marrow Donor Institute Lions Club Foundation Brain Foundation Cass Foundation Cerebral Palsy Alliance Cerebral Palsy Institute Clifford Craig Medical Research Trust Cooperative Research Centre for Poultry Cure Kids Fiji Financial Markets Foundation for Children Garnett Passe and Rodney Williams Memorial Foundation Harold Mitchell Foundation Hearing Cooperative Research Centre Heart Foundation Heart Kids Australia Helen Macpherson Smith Trust Ian Potter Foundation Jack Brockhoff Foundation Juvenile Diabetes Research Foundation Marian and E.H. Flack Trust Mason Foundation Menzies Foundation Muscular Dystrophy Association of Australia National Health & Medical Research Council National Stroke Foundation Preston and Loui Geduld Trust Fund R.E Ross Trust Royal Australian College of Physicians Foundation Royal Australian College of Surgeons Shepherd Foundation Sidney Myer Fund Sleep Health Foundation Sylvia and Charles Viertel Charitable Foundation Victorian Medical Insurance Agency Victorian Neurotrauma Initiative

ACHIEVEMENT AWARDS , The Institute s staff awards formally acknowledge and reward high achievers, those who have excelled in their research endeavours or for their contribution to the Institute and commitment to its values.

Outstanding Contributions Acknowledges the contribution of a staff member in research or research support and operations who consistently goes above and beyond their role. > Blanche Dekker, Scientific Services

Outstanding Manager/Supervisor Recognises excellence in leadership. > Dr Catherine Satzke

Rising Star Award Recognises up-and-coming researchers for research excellence, for a researcher up to 10 years postdoctoral. > Dr Angela Morgan

Director’s Award Recognises excellence in research achievement for senior researchers, 10 or more years postdoctoral. > Professor Melissa Wake

NATIONAL HEALTH & MEDICAL RESEARCH COUNCIL AWARDS

Gustav Nossal Scholarship Highest ranked applicant in medical and dental post graduate scholarship > Dr Katherine Howell

Elizabeth Blackburn Fellowship Highest ranked female in the public health category > Professor Melissa Wake

Research Fellow Top ranked applicant > Professor Melissa Wake

annual report 2013

special events KIDS STEP UP IN SUPPORT OF CHILD HEALTH In 2013 the Institute launched a new campaign called Step-a-thon for Kids, which is about getting kids active, healthy and having fun, whilst at the same time supporting other kids by raising money for child health research. In its inaugural year over 19,000 primary school students right across the country participated, slapping on a slap band pedometer and stepping up during the first week of September to raise over half a million dollars for the Institute. Ambassador Sarah Murdoch, along with Director Professor Kathryn North, Dr Andrew Rochford and Stephanie Rice helped kick off the campaign with a launch held at The Hills Grammar School in Sydney in July.

> students from the Hills Grammar school

At the launch event, both Sarah and Kathryn spoke about the importance of creating and encouraging a healthy community, to help address health problems before they begin. They said instilling the simple message of getting active while kids are young is vital, and the fact that they can take simple steps to live a healthy lifestyle is an important message to convey. Founder of Step-a-thon and Senior Manager of Engagement, Matthew Hannan, said in the lead up and during the event researchers and staff at the Institute were inspired by the response from parents about their children who were supporting siblings or friends that had or were suffering from illness, disease or disability. “The feedback from many parents was that Step-athon had a positive impact on their child’s behaviour and that their children felt empowered that they could get active and help kids across Australia at the same time, which was really inspiring.” Such stories included Western Australia youngster Piper, who raised $6,500 and was inspired to do so by her little sister Ivy, who has Global Developmental Delay. Another example is Erin, who raised over $1000 in honour of her little brother Dion, who passed away not long after his third birthday from a Mitochondrial Disease called Leigh’s Disease. All money raised from Step-a-thon for Kids will support our research to help find cures and treatments and also help prevent common and rare childhood conditions. The event wouldn’t have been possible without the help and support of our Ambassador Sarah > Murdoch and our sponsors.

> director professor kathryn north, sarah murdoch & stepahnie rice

principal sponsor

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events

Discovery Day The late Dame Elisabeth Murdoch’s Cruden Farm once again provided the perfect backdrop for the Institute’s ninth annual Discovery Day. Over 2,200 people attended the event, raising $100,000 for child health research.

Biennale Murdoch Childrens, along with Sarah and Lachlan Murdoch, hosted a spectacular evening of wonder and generosity in late October for the inaugural Biennale event. The evening featured an intimate dinner giving supporters the opportunity to learn about the Institute’s work and meet key researchers.

As the event was the first celebrated without the late Dame Elisabeth, a bench was presented in her honour as a way of saying thank you for opening up her gardens for children and their families to enjoy each year. Adults and children alike were treated to an energetic program of entertainers, including Playschool’s Justine Clarke. Other activities included massages, crafts, rides and face painting, as well as cooking demonstrations with Coda restaurant’s owner and head chef, Adam D’Sylva.

The dinner was followed by a cocktail party and auction, with items including a red carpet experience with Tom Cruise in Los Angeles raising over $30,000. Special guests included fashion designer Alex Perry, chef George Calombaris, TV’s Catriona Rowntree, and performers Tottie Goldsmith and Ms Murphy.

Supporters: Vaalia, Herald Sun, Leader Community Newspapers, SPC Committee: Sharon Bassat, Susannah Fairley, Eliza Mantello, Dimity Millear, Kate Mohr, Monique Rajch, Dahlia Sable, Camille Sunshine

Supporters: AZB Creative, Bright Young Thing Culinary Event Makers, Harry the Hirer, The Greatest Show on Earth

< sarah murdoch & alex perry

< kids enjoying discovery day

Ballet Last year, The Australian Ballet hosted an exclusive preview performance of Alexei Ratmansky’s Cinderella with all proceeds going towards child health research at the Institute. Pre-performance drinks were held in The Arts Centre’s Commonwealth Bank Lounge, where some of our donors and friends were joined by our Ambassador Sarah Murdoch and the inimitable Artistic Director at the Australian Ballet, David McAllister. Supporters: The Australian Ballet, Arts Centre Melbourne

annual report 2013

corporate partners & major supporters Our corporate partners and major corporate supporters provide significant financial support for our research as well as helping to promote the Institute to a new audience. FOXTEL is one of the Instituteâ&#x20AC;&#x2122;s longest corporate supporters and have been providing significant financial and pro bono support since the Foxtel Lap began in 2005. The corporate treadmill challenge raised over $3 million for the Institute and introduced a number of supporters to Murdoch Childrens. In 2013 Foxtel was a founding sponsor of Step-a-thon for Kids, helping 19,000 kids get active and raising over half a million dollars. As well as sponsoring this key event, Foxtel filmed the Step-athon advertisement featuring our Ambassador, Sarah Murdoch, and also helped promote the event to their staff and across the Foxtel TV network. Foxtelâ&#x20AC;&#x2122;s ongoing commitment is helping the Institute to continue its life saving child health research.

News Corp Australia is another organisation that has been supporting the Institute in a range of ways for countless years. In 2013 News Corp provided significant pro bono advertising and editorial support for our campaigns throughout the year, including substantial national promotion for Step-a-thon for Kids. Murdoch Childrens was also the beneficiary of the News Corp Australia 2013 Young Lions Competition. The best and brightest young advertising creatives developed inspiring campaigns to help increase the awareness and fundraising for our research. Vaalia, who has been a corporate partner for the past three years and in that time has contributed $450,000 to our research, have committed to supporting the Institute for another three years. As well as providing financial support Vaalia has contributed to the Institute in a number of ways including sponsoring events such as Discovery Day and Step-a-thon. The Institute would also like to thank Johnsonâ&#x20AC;&#x2122;s Baby and SPC Ardmona who supported the Institute in 2013. Researchers and staff at the Institute are extremely thankful of the support of corporates and funders; without this support many of our new innovative research projects would not get off the ground. Together we share the same objective; to give kids the best chance at a healthy, happy life.

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< MATTHEW hannan, dr david ELLIOT from murdoch childrens & tracey berkeley from vaalia.

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donors

A UNIQUE CONTRIBUTION Sophie Pennington’s decision to start her online auxiliary shop ‘Give a Little Bit’, which supports stroke research at the Institute and The Royal Children’s Hospital, is one that is close to her heart.

BABY ZARA INSPIRED FUNDRAISING Lauren Copping and Devro Matekuare’s lives changed forever in August 2012 when Lauren was in the later stages of her first pregnancy. “I was seven months pregnant and couldn’t feel our baby moving,” Lauren recalls, “We went to the hospital in Kalgoorlie, they put a monitor around my belly and tried to get our baby to move, but she was barely responsive.”

and chronic lung disease, a terminal illness with no cure.”

Lauren had an emergency caesarean and Zara was born. The following day they were flown to Perth, where Zara spent the next three months in hospital before being discharged at 38 weeks old.

After Zara’s death, Lauren and Devro wanted to support research into Pulmonary Hypertension, so that other families would not have to endure the pain they have been through.

“Our celebration was short-lived. After returning from hospital, Zara wouldn’t settle at nights and was always vomiting and not breathing well. She had contracted a viral infection and had also developed Pulmonary Hypertension

Knowing the value of community fundraising, they held a BBQ in their home town, and raised over $1000 for research at the Institute. Lauren’s workplace Nickel West matched the funds two for one, increasing the total raised to over $3000.

After spending more than six months in hospital, in March 2013 Zara passed away in their arms, on life support. “She had fought so hard for so long but her little body couldn’t fight any longer. Saying goodbye to our baby girl was the saddest day of our lives.”

Sophie’s daughter Harriet suffered from a stroke just six weeks shy of her third birthday. Following the stroke Harriet spent a month in hospital, but thankfully is now a happy and healthy eight year old.

products available on the online store are handmade by either Sophie or donated by one of a number of local artists with all of the profits directed to our research, which is looking into risk factors, causes, treatments and outcomes following childhood stroke. The store was launched in September 2013 and has raised over $4000.

To give back to the doctors and researchers who cared for Harriet, Sophie came up with the idea of Give a Little Bit, an online auxiliary. All the

> l auren & devro with baby zara

> sophie penniNgton & her daughter harriet

annual report 2013

donors Major Donors Angior Family Foundation Annamila ANZ Trustees Australian Camps Association Australian Communities Foundation Graham Burke CAF Community Fund Mr John Calvert-Jones AO & Mrs Janet Calvert-Jones AO Children’s Cancer Centre Foundation CIKA Collier Charitable Fund Consolidated Press Holdings Mr & Mrs James & Claudia Craig Creswick Foundation Cripps Foundation Crocka Foundation DHB Foundation Equity-One Mortgage Fund Fight Cancer Foundation Fragile X Alliance Mr & Mrs Graeme & Pamela Fraser Gandel Philanthropy George & Freda Castan Families Charitable Foundation Mr David Jenkins & Ms Fran H Lefroy Dr & Mrs George & Joan Lefroy Miss Marj Lefroy Mr Sam Riggall & Ms Nichola Lefroy Limb Family Foundation National Muscular Dystrophy Research Centre P&S Bassat Foundation Mr & Mrs Rowly & Judy Paterson Michael Peterson Anthony Pratt & Claudine Revere RCH Anaesthesia Research Trust RCH1000 The Royal Children’s Hospital The Royal Children’s Hospital Foundation Mrs Pamela Sargood Scanlon Foundation Spotlight Charitable Foundation The Cameron Family Foundation The Fox Family Foundation The Greek Conference The Invergowrie Foundation The Isabel & John Gilbertson Charitable Trust The Kimberley Foundation The Miller Foundation The Ponting Foundation The Pratt Foundation The Scobie & Claire Mackinnon Trust

Donors

The Sunraysia Foundation Tour de Cure Ms Faye Williams Mr & Mrs Ron & Joan Wilson The Wolf Foundation UHG Foundation Victorian Medical Insurance Agency

Event Sponsors & Pro Bono Ace Radio Adnate Street Art AFL Akarba Alex Perry All Sportz Antler Luggage Atlantic Group The Australia Times AZB Creative Bean Media Bijoux BodyCare Physiotherapy Bright Young Things Briner Buzz Products Canaccord Central Equity Justine Clarke Colonial First State Global Asset Management Cotton On Kids Creative Fairies Croc Media Lucy Desbordes The Design Depot Alanna Deutrom Adam D’Sylva e.Motion21 Eastern Press The European Fehily Contemporary Five of the Best FizzKids Food & Desire Four Seasons Resorts Bali FOXTEL Gallery Gabrielle Pizzi Greatest Show on Earth Guy Grossi Hagens Organic Meat Harry the Hirer Hatched Media Ice Events IOOF Paul Jamieson

Kay & Burton Lake House Daylesford Life Like Touring Life Lounge Agency Macakizi Hotel, Bodrum Maiyet McClelland Galleries Melbourne Festival News Corp Australia Noah’s Creative Juices Norton Rose Fulbright Australia Nova Entertainment Phil & Ted Police Band Centre PRB Design The Press Club Proactivity Alexandra Pyke Qantas Rathdowne Fabrics & Remnants Raw Materials Royce Hotel S&J Media Group Sandhurst Scott Newett Photography Soccerwise Soho Grand South Melbourne Dance Centre Spencer Travel Splitrock Spotlight Studio Bauhaus Sweeney Research Sweet Studio Christian Thompson Turtle Island Village Roadshow Limited Virgin Australia

Major Corporate Philanthropy The Australian Ballet Grays Online News Corp Australia Swisse Vitamins Macquarie Group Foundation Russell Investment Group FOXTEL Novo Nordisk Big W

A Mr Bruce Abbott AEG Aidan & Oliver Biggar Cystic Fibrosis Fund Allen & Lee Real Estate The American Fund for Charities Mr Andrew Alston Mr Clint Anderson Mr & Ms Christopher & Melissa Archibald Arthritis SA & Osteoporosis SA Arthritis Victoria A S K Aluminium Fabrications B Mr & Mrs John & Rosemary Barr Mr & Mrs Michael & Judy Begg Dr Bligh Berry Mr Dean Berry Big W Broadmeadows Big W Fountain Gate Big W Social Club Big W Werribee Mr Grant Biggar Mr Peter Bird & Ms Cherie Zanette Mr & Mrs Brian & Helen Blythe Mr David Briskin & Elizabeth Ross Brittle Bones Mr Daniel Broadbent Mr & Mrs Malcolm & Janet Brodie C Ms Eve Casper Mr Steven Casper & Ms Ilana Wald Mr & Mrs Trevor & Heather Cohen Mr & Mrs John & C Collingwood Mrs Helen M Collis Mrs Julie Conti Mr & Mrs Anthony & Melanie Coops Lauren Copping & Devro Matekuare Mr & Mrs Tom & Elana Cordiner Mr Laurence G Cox AO & Mrs Julie Ann Cox Kate Cranage D Mrs June Danks J Dart Joanne Davies Mr Cliff Dawson Dr Charles Day & Ms Elise Everest Dennis Family Corporation Dr Annette Domanti Mr Denis Dowty Ms Susan Drummond Mr George Dyer E Mr Michael Egan England Obstetric Services Mr Peter Ewen

F Mr & Mrs Ken & Lisa Fehily Mrs Joan Fell Mr Robert Fiani The Flew Foundation Kerry Flynn Mr Michael Forsdick Mr & Mrs Lindsay & Paula Fox Mr Peter Fraser Freedom Dental Dr & Mrs John & Diana Frew Mr Rodney Frost G Ms Neilma Gantner Mr & Mrs Ross & Judith Gardner Ms Marilyn Garnam Mrs Sylvia Gelman AM Mr & Mrs Bruce & Robyn Gibbons Mr & Mrs John & Suzanne Gibson Give a Little Bit The Good Guys Foundation Mr & Mrs Stuart & Marion Gooley Mrs Louise M Gourlay OAM Mrs Elaine Greenhall Mr & Mrs Peter & Terry Griffin H Mrs & Dr Joanne & John Hagiliassis Mr Maurice E Hall Mr & Mrs Brian & Natalie Hamersfeld Mr & Mrs Colin & Carolyn Harris Mr Wesley Harris Mr Barry Hastie Matt Hawkins Mr & Mrs Codie & Ellie Hearfield Hearts of Hope Australia Mr & Mrs Geoffrey & Dorothy Heeley Mr & Mrs W H & Rea Hodgson Mr Matthew Hogg Mr David Holckner Mrs Debra Holmes Hong Kong Business Association Victoria Mr & Ms John & Belinda Hope Mr Timothy Hovey Mr & Mrs Russell & Kate Howcroft Ms Gillian Hund Mr Davell M Hutchinson Professor John Hutson I In Line Logistics Mr Tony Isaacson ITW Buildex Marija Ivkovic Mr Jayaram Iyengar J Mrs Margaret James Dean Jenkins

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donors

In Memory of Catherine Martin

In celebration of Graham & , Deborah Goldsmith s 30th Wedding Anniversary

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Mr Raoul Salter

In celebration of , Jade Swiatlo s Bat mitzvah

In lieu of Birthday Gifts for Henry McGregor Anderson

Mrs Tanya Barnett

Mrs Bethany Dickins Helen Blazek

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In lieu of Birthday Gifts James Behr

Mrs Denise Young

Mr & Mrs Peter & Rachel Neil

In Memory of Dame Elisabeth Murdoch AC DBE

, In celebration of Hudson s Barmitzvah

Dr Elan Kaplan Mrs Gloria Prossomariti

Mr Heath Jones K Ms Julie Kantor Mr Azzem Kassem Mr Daniel Keegal Mr & Mrs Peter & Elizabeth Kelly Mr Ian Kennedy AM & Dr Sandra Hacker AO Mr Adam Kerr KMS Financial Solutions Ms Lynette Korn Mr & Mrs Larry & Sophie Kornhauser Dean & Rebecca Koutsoumidis L Mr John Landy AC & Mrs Lynne Landy Lauriston Girls’ School Mr & Mrs David & Jeannette Lewis Mr & Mrs Josh & Karen Liberman Listamere Mr John B Little Ms Emma Longmore Ms Jocelyn Lovegrove Mr Hogan Lovells Lower Kindergarten Purple Room M Ms Christine MacGill Mr & Mrs Alasdair & Prudence MacLeod Magistrates’ Court of Victoria Mr & Mrs Mick & Amanda McMahon Ms Tina McMeckan Ms Alison McNab Mr & Mrs Ian & Libby McNaughton Mr & Mrs John & Julie McPhee Methodist Ladies’ College Michael & Andrew Buxton Foundation MLC Community Foundation Mr & Mrs Dean & Kate Mohr Miss Simone Myers N Nelson Alexander Real Estate Charitable Fund Rachel Nicholls Mr John Nolan O Mr & Mrs Simon & Merryn Olive Mr Tim Olsen Operation Newstart Australia Ms Denise O’Reilly Ms Carmel Origlia John O’Rourke The Outdoor Education Group

P Paediatric Integrated Cancer Service Mr & Mrs Arthur & Heather Paikos Nando Pellicano Peter Isaacson Foundation Mr & Mrs Justin & Edwina Portelli Portland House Group Lady Primrose Potter AC Precision landscapes Mr & Mrs Patrick & Maureen Purcell Q Mr Justin Quill Mr & Mrs Michael & Adrian Quilter R Mr Fudge Raco RACV Club Dr Janahan Rajakulendran Mrs Donna Ravenscroft Mrs Shirley Reeder Ms Anita Reilly Michael Ridler Mrs Sue Ritchie River Capital Foundation Ms Judy Roach Judith & Michael Robinson AO Mrs B. J. Roper Mr Michael Rose Rotary Club of Canterbury Mr Adam Ryan

Mr & Ms Peter & Debbie Thomas Mr Dino Tsimaras U UBS Foundation Uncle Bobs Club V Mr & Mr Blain & Saverio Valmorbida W Mr Andrew Wallace-Barnett Ms Rachel Walsh Judith Ward Dr Jenny Webb Westpac Mrs Nola White The Willaura Quilter Friends Mrs Joanne Williams Mr Steven Williams Mr Ray Wilson Wingate Group Y Mr Jason Yeap OAM & Ms Min Lee Wong Mr & Mrs Edward & Mandy Yencken

T Sean Taylor Mr Andrew Templar Terumo Corporation Mr Ion Teska

Mr Christopher Griffin Ms Margery Embury Mr Hogan Lovells Ms Claire Russell The Willaura Quilter Friends Mr & Mrs Patrick & Maureen Purcell Mr Peter Graham Mr Davell M Hutchinson Mr Gavan Woinarski Mrs Jennifer Armstead The Mt. Eliza Morning Garden Club Mr & Mrs David & Sharona Brott Mr & Mrs Michael & Elizabeth Richards News Corp Mr & Mrs Graeme & Pamela Fraser The American Fund for Charities In memory of Elizabeth Ham

Z Christine Chrysoula Zafirakis Donations of $500 and more are listed.

S Mr & Mrs Nathan & Dahlia Sable Mr Ash Saward SCT Logistics Perth Silberscher Family Foundation Mr Jason Silver Mr Leon Skaliotis Mr & Mrs Colin & Jan Smith Patrick Smith Mr Oscar Sokolski Mr Martin Solomon Team Southland Mr Neil Spitzer Mr Michael Spurr STAUFF Corporation Steeline Case Steel Mr & Mrs Grant & Terri Stephenson Ms Shani Stevens Sarah Swanston

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Mr & Mrs Ross & Judith Gardner

Ms Kerrie County

In memory of James John Hansen-Naarden

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Ms Mary O’Dea Mr Geoff Farrugia Ms Sandra Foxcroft Mr Vince Carpenzano Maxine Andrews Ms Wilma Blanksby Ms Tracy Farrugia Ms Joy Barclay Raj Sonogan Ms Marcia Clohesy Mr Chris Slattery Ms Rosemary Scarlett F Renehan Mr & Mrs Voyer Mr Tom Sammann Mr & Mrs Steve & Helen Burke Ms P McMaster Mr Pete Bugden John & Aaron Farrugia In celebration of John G Rogers AM Queens Birthday award

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annual report 2013

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Mr & Mrs Adam & Samana Trayer Ms Rosslyn Bancroft Mrs & Mr Carrie & Jamie Flinkier Dr & Mrs Daniel & Sandi Sable Ms Rebecca Zwier Ms Fay Miller Mr Greg Monahan Dr Aaron Walton & Dr Deb Friedman Mr & Mrs Ronald & Pam Sackville Mr & Mrs Paul & Sharon Bassat Ms Rachel Bloom Ms Eve Casper Mr & Mrs Syd & Sara Robenstone Mr & Mrs Eric & Jan Upton Mr & Mrs Alex & Elizabeth Furman Mr & Mrs Henri & Leah Buchbinder Mr & Mrs Barry & Natalie Knobel Mr Brad Akkerman Mr & Mrs Jack & Evelynne Gance Mr & Mrs Barry & Suzi Carp Mr & Mrs Jacob & Debbie Weinmann Mr Brian Hamersfeld & Mrs Natalie Hamersfeld Mr & Mrs Rodney & Vanessa Brott Mr & Mrs Barry & Lorraine Bloom Mr & Mrs Barry & Kaye Fink Ms Lynn Trayer Mr & Mrs Joe & Glenda Flinkier Mr & Mrs David & Rhonda Jacobson Mr & Mrs Charles & Karyn Akkerman Mr & Mrs Henry & Pnina Douek

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Miss Jade Gesell Mrs Nicole Garretty Mr & Mrs Codie & Ellie Hearfield In celebration of Nick Wong

Mr & Mrs Daniel & Rebecca Harford Dr Ann Frazier Mr & Mrs Alex & Elizabeth Furman Dr Marguerite Evans-Galea Dr Nick Wong Miss Greta Gillies Origin Energy Ms Fiona Gosschalk Mr James Tseng Mr Nandor Roczo Dr David Martino Dr Shoo Peng Siah

In memory of Phema Lhamo Davis Gibbons

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Mr & Ms Dave & Chanie Curtis Mr & Mrs Jeff & Lisa Wise Mr & Ms Jonathan & Michelle Mushin Mr & Mrs Craig & Roxanne Harris Vanessa & Ashley Levin Mr & Mrs Wayne & Helena Lippe Redman Family Mr & Mrs Brian & Natalie Hamersfeld Ms Liz Pullos Mr Lou Bosancic Mr & Ms Simon & Georgina Raik-Allen Mr Craig Howard Dr Julie Lustig Mr Steven Casper & Ms Ilana Wald Mr Justin & Angie Perelberg Mr & Mrs Eddie & Lindy Tamir Mr & Mrs Mark & Shellie Jelinek Mr & Mrs Timothy & Jane Jackson Mr Damian Abrahams Ms Danielle McKeown Mr & Mrs Nathan & Anne Moshinsky Mr & Mrs Richard & Gail Swasson Mr & Mrs Tony & Kate Beaconsfield Mr & Mrs Morry & Pauline Wrobel Ms Emma Rosenberg Mr & Mrs Dean & Shareen Joel Mr & Mrs Bori & Helen Liberman Mr Alan Synman In celebration of , Sophie Davis s 40th birthday

Mr Steven Casper & Ms Ilana Wald

Thank you to the following organisations which generously assisted in the production of this report. Production & Design

In memory of Suzanne Theresa Brown

Mrs & Mr Irene & Jack Brown In lieu of Birthday Gifts Viren & Vandana

TOMORROW S CURES NEED YOUR DONATIONS TODAY

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You have the power to help the , Murdoch Childrens Research Institute save kids lives. With your support, our researchers can continue to discover cures for conditions including diabetes, cancer, allergies, premature birth, obesity and genetic conditions. Donate today. Phone 1300 766 439 or visit www.mcri.edu.au

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Paper stock Edited by Simone Myers & Caitlin Moore (pr@mcri.edu.au) The text of this Annual Report is printed on Spicers

In Memory of Zara Matekuare

Lauren Copping & Devro Matekuare

Pacesetter 148 gsm and the cover on Spicers Pacesetter Satin 350 gsm