Generation Victoria (GenV): Establishment and Campus Value
The primary objective of GenV is to enroll every child born in Victoria, along with their parents, into this study. This ambitious initiative aims to create a state-based research platform to accelerate the ability of researchers in addressing complex health and wellbeing challenges affecting children and young people by 2035.
Led by the Murdoch Children’s Research Institute, GenV looks at how children grow and develop, how people age, and how health changes across generations. GenV is the only birth and parent cohort launched internationally during the COVID-19 pandemic, and it includes families who are often underrepresented in research, such as those living in regional and rural communities, culturally and linguistically diverse families, and First Nations families.
Made possible through philanthropy, including support from the Good Friday Appeal, the Victorian Government, and the Paul Ramsay Foundation, this project aims to improve the prediction, prevention, and treatment of various child health issues such as preterm birth, allergies, and anxiety. GenV is helping researchers, communities and policymakers improve health and wellbeing of families at the RCH, throughout Victoria and across Australia in the long term.
To date, over 115,000 participants have joined the study, which includes families and babies from every birthing hospital in Victoria.
Through research and testing, GenV supports a wide range of conditions, both common and rare, affecting children and adults today. GenV is involved in 16 active research studies and supports a wide range of collaborative observational and interventional research.
From reducing the unprecedented rate of chronic adult diseases to developing improved predictive tools, this initiative is helping to ensure the next generation is as healthy as possible.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
The RCH Neuroscience Advanced Clinical Imaging Service (NACIS)
Over the last decade, The Royal Children’s Hospital (RCH) and the Murdoch Children’s Research Institute (MCRI) have worked to deliver an advanced neuroimaging program that has revolutionised neurosurgical practice and improved surgical outcomes for children with epilepsy and selected brain tumours.
This resulted in the establishment of Neuroscience Advanced Clinical Imaging Service (NACIS) under the Department of Neurosurgery at the RCH.
The Neuroscience Advanced Clinical Imaging Service (NACIS) is a one-of-a-kind service at The Royal Children’s Hospital (RCH). Through advanced brain imaging technology and image processing techniques, this service has been able to help make the invisible appear visible, going further than commercial magnetic resonance imaging.
Thanks to the support of the Good Friday Appeal, NACIS is improving long term outcomes and quality of life for young patients with brain tumours and epileptic disorders.
The NACIS team can help to identify particular lesions in the brains of patients with epilepsy, which were unable to be accurately detected before the establishment of the service. Across 2024, NACIS has helped clinicians deliver personalised and safer brain surgery to over 120 children.
NACIS uses sophisticated image processing techniques to produce a detailed map of a child’s brain vessels, functional brain regions and the underlying nerve fibre connections, which control functions like language, vision and movement.
They also use the technology to assist surgeons in identifying safe “surgical road maps” for precise brain mapping and surgical execution, as well as assessing patients’ post-surgery to ensure the best possible outcomes The map is used by surgeons to precisely plan and perform their operations, allowing them to avoid injuring these critical functional brain structures, thus ensure the best possible functional outcomes.
NACIS is a one of kind service, working to perform more high-risk and complex surgical cases, and in selected cases, offering surgery as a treatment to children previously considered inoperable.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
The Melbourne Children’s Trials Centre
The Royal Children’s Hospital (RCH) already conducts many high quality clinical trials and with a dedicated centre on campus, better co-ordination and an improved facility, the RCH is performing more clinical trials, more often. Some of these trials include screening, early treatment and new important therapeutics or improved clinical management and medical procedure.
Thanks to the Good Friday Appeal, the Melbourne Children’s Trial Centre (MCTC) has been able to increase the capacity to support clinical trials across The Royal Children’s Hospital (RCH) and Murdoch Children’s Research Institute (MCRI), also known as the Melbourne Children’s Campus. This is helping to drive healthcare innovation and support sick children all across the hospital.
Importantly, the centre has also helped support and oversee close to 400 clinical trials across last year alone. Professor Andrew Davidson, the Medical Director of the MCTC, knows first-hand the impact this program has had campus wide.
Professor Andrew Davidson
“The MCTC ensures that we have the best environment to support and perform trials across campus. This ensures that our trials are more powerful and efficient,” said Andrew.
The MCTC supports all types of trials, including commercially sponsored trials which aim to test the success of a new drug or medical device. Since the MCTC began, the centre has helped support over 250 of these types of trials, helping change countless lives.
Andrew was especially proud to share the recent impact this type of trial has had on a three-year-old boy living with a rare metabolic condition called phenylketonuria. This condition typically requires a lifelong restrictive diet, as a sudden increase in protein can risk neurological impairment and intellectual disability.
Fortunately, however, the toddler was able to participate in a clinical trial at the RCH, leading to a breakthrough in his treatment. This allowed the sick child to enjoy something many take for granted – his first birthday cake.
“Often these rare diseases have no treatment option other than a clinical trial – and most of the time, these trials are lifesaving, and it completely changes the child and family’s life,” Andrew explained.
The MCTC also supports investigator initiative trials. These trials respond to academic or clinician research questions, helping change the way that children are cared for globally.
“With investigator initiative trials, we want to continue to be at the forefront of innovative ways of doing them. We’re already very successful as a campus and we want to continue to be very successful,” Andrew said.
With over 100 investigator initiative trials running at any time, the MCTC is helping promote the advancement of medical knowledge, all while changing the lives of young people.
Looking forward, the MCTC aims to further advance paediatric healthcare innovation on both a regional, national and global scale.
“This funding has allowed us to work more closely with the Sydney Children’s Hospital, Monash Children’s Hospital and the Queensland Children’s Hospital. This has been particularly important for rare diseases, where we needed a national approach to trials,” Andrew shared.
The MCTC is revolutionising healthcare for children and young people across the world. Thanks to the Good Friday Appeal, the centre will be able to further establish themselves as leaders in enabling efficiency and high-quality paediatric trials.
“The Good Friday Appeal’s support is helping provide an environment so that children at the RCH and sometimes around Australia can get access to whatever treatments they need that can completely change their lives,”
Professor Andrew Davidson, the Medical Director of the Melbourne Children’s Trials Centre
“The MCTC makes a huge difference to children all over,” Andrew concluded.
Example Trials
Positive end-expiratory pressure levels during resuscitation of preterm infants at birth (POLAR) trial
Almost all premature babies receive help with their breathing using a treatment called positive end-expiratory pressure (PEEP). The device used can give air and oxygen to the lungs between each breath to stop their lungs from collapsing.
Currently, due to a lack of evidence, doctors around the world give different amounts of PEEP to premature babies.
The POLAR trial explores the right amount of PEEP to give at birth.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
TOTAL XVII – TOTAL Therapy Study
Thanks to the Good Friday Appeal’s Run for the Kids initiative, newly diagnosed patients with acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma at The Royal Children’s Hospital (RCH) have been able to access the latest in personalised medicine.
The trial, known as the TOTAL Therapy Study XVII or TOTAL 17, is an international collaboration led by St Jude Children’s Research Hospital in America.
Over the past two years, 62 patients at the RCH have been enrolled in the innovative program, far exceeding the initial aim of enrolling 20 patients per year. Recruitment for the trial ceased in mid-2023 due to reaching the trials capacity limit, with patients enrolled continuing with follow up therapy and treatments including neurocognitive assessments.
Once enrolled, the child’s blood and bone marrow samples were sent to St Jude. There, clinicians used state-of-the-art tools and gene mapping to identify the specific cancer-causing gene combinations, providing patients with the opportunity to receive treatment that is specifically designed to fight their type of cancer.
Additionally, the study offers an opportunity to identify the likelihood of cancer impacting patients and their families again in the future, while also providing information about the suitability of different drugs to help minimise side effects.
Currently, the RCH is the only hospital in Australia participating in the TOTAL Therapy Study XVII, providing access to the best outcomes globally.
This would not be possible without support of the Good Friday Appeal and Run for the Kids, something Dr Di Hanna, a Paediatric Oncologist at the RCH Children’s Cancer Centre, is grateful for.
Dr Di Hanna
“The Good Friday Appeal fundraising is critical for every piece of the cancer journey. We need the latest research, diagnostic tools, best clinical trials, and patient and family support networks, and the Good Friday Appeal has been a cornerstone for each part of that.
“Importantly, thanks to the Good Friday Appeal, we are also able to provide children with leukaemia and lymphoma access to one of the best clinical trials in the world,” said Dr Hanna.
One of the patients enrolled in the trial is five year old Bobby, who was diagnosed with B-cell ALL when he was three.
For patients like Bobby, the long term impacts of the TOTAL Therapy Study XVII are life changing.
Bobby Marr in hospital
“One of the best things the trial has offered us is the additional supports, including the neurocognitive and physio assessments. It has been invaluable for us when considering things like getting Bobby ready for school, including any adjustments we need to make,” said Trudy, Bobby’s mum.
“Additionally, the idea that we could be part of that larger pool of research is significant to us. Being able to contribute to future developments to benefit other families made our decision an easy one,” she added.
Impact Milestones
2023 – 2024
By the end of April 2023, 48 out of the 62 enrolled patients completed baseline tests to determine how the brain works in terms of thinking, learning, remembering and problem solving.
The tests provided valuable data and insights for early interventions and referrals to relevant departments.
Samples from patients were sent to St Jude Children’s Hospital for comprehensive genomic profiling of leukemia cells. Out of the 52 patients profiled, 16 received targeted therapy.
Thanks to the new and targeted therapies, some patients who entered the trial avoided having a bone marrow transplant.
Based on sampling, St Jude’s has also helped identify two patients with a variant that may influence cancer predisposition.
Patients and their families have been linked with RCH Genetic Counselling services.
Beyond immediate treatment, patients were also offered assessments that look at how treatments affect the brain and thinking abilities.
As of September 2024, the study remained close to recruitment, with 45 patients still participating in the study. Meanwhile, three have completed the study and are in follow-up therapy.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Type 1 Diabetes Immunotherapy Program at MCRI/RCH
Type 1 diabetes is diagnosed in two to three children per week at The Royal Children’s Hospital (RCH) and is currently incurable. However, thanks to the support of the Good Friday Appeal, a science and research program conducted by the Murdoch Children’s Research Institute (MCRI) and the RCH is offering new hope.
Now in its fourth year, the program which also works closely with the Australasian Type 1 Diabetes Immunotherapy Collaborative, is investigating immune-based therapies to both prevent and treat type 1 diabetes in existing patients. This important trial aims to improve developmental outcomes, quality of life and mental health outcomes while reducing suffering and mortality rates.
Type 1 diabetes is one of the largest disease groups within the hospital. It is also currently incurable. Despite current therapies, it predisposes affected individuals to adverse kidney, nerve, eye, heart and brain outcomes and is also associated with a high mental health burden for patients and their parents.
The RCH and Murdoch Children’s Research Institute (MCRI) are working together to develop the Type 1 Diabetes Immunology Program, in a collaborative effort with the Australasian Type 1 Diabetes Immunotherapy Collaborative.
This program will involve a specialist research centre, designed to understand and treat type 1 diabetes.
It will leverage immune-based therapies and recent breakthroughs in the field, to implement new therapies that come from the area of stem cells, immunology or a combination of both. The program has the potential to radically change the outcomes of children and adolescents with the type 1 diabetes. It can reduce morbidity and mortality rates and improve quality of life and mental health outcomes as well as developmental outcomes.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Healthy Trajectories: A Child and Youth Disability Research Hub
Children with disability are among the most complex patients at The Royal Children’s Hospital (RCH). This means it is important to develop an innovative and research-based approach when it comes to the management of disability related health and social issues. Thanks to the support of the Good Friday Appeal, the RCH has been able to develop Healthy Trajectories; a child and youth disability research hub at the Melbourne Children’s Campus.
This hub harnesses the strengths of the campus partners to put in place a new vision for children with disability, their families and carers, with the potential for significant Victorian, national and international impact. It aims to increase inclusion and participation for children and young people with disability by targeting potential barriers and inequalities and addressing the research priorities they identify as crucial.
Themes like building a sense of belonging for students with disability and designing learning spaces for diversity, inclusion and participation have been explored through hub research. This interdisciplinary research addresses practice and policy gaps, helping to build a more sustainable National Disability Insurance Scheme and making a life-changing difference to patients.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Creating evidence for telehealth-delivered neurodevelopmental assessments
Thanks to the Good Friday Appeal, The Royal Children’s Hospital (RCH) and the Murdoch Children’s Research Institute (MCRI) are coming together to undertake research to provide clinicians with guidance and evidence-based information on how to best deliver telehealth developmental assessments.
Patients accessing healthcare via telehealth appointments have increased following the COVID-19 pandemic.
It is vital children and young people with complex neurodevelopmental needs are given accurate and timely diagnoses and appropriate plans to manage their symptoms. This funding will ensure the quality and accuracy of assessments, so patients receive the best possible care.
For most children, the gift of learning, communicating with others, developing friendships, and engaging in day-to-day activities comes naturally. Yet for many children with a developmental disability, this is not the case.
Effective diagnosis and treatment plans for developmental disabilities traditionally rely on a series of in-person assessments that look at social and communication skills, speech and language abilities, and behaviour and brain function. Clinicians have shifted parts of these assessments to telehealth to treat vulnerable or disadvantaged patients.
Currently, the reliability of telehealth developmental assessments is not known. This makes a study in the area crucial in preventing incorrect diagnoses, unsafe clinical practices, and substandard clinical outcomes.
The Royal Children’s Hospital (RCH) and the Murdoch Children’s Research Institute (MCRI) are working together to develop evidence-based telehealth assessments.
The study will provide clinicians with guidance and guidelines on how to deliver high-quality telehealth assessments. This will result in positive health outcomes across the fields of neuropsychology, clinical psychology, developmental paediatrics and speech pathology.
The outcome of this study will be published in journals, discussed at national and international conferences, and shared in workshops to allow clinicians globally to deliver greater care and provide best practices.
This research can lead to high-quality telehealth-delivered assessments. It can also increase access to accurate assessments for vulnerable patients, such as those with immune deficiency, reduced mobility, mental health and neurodevelopmental problems, and those in regional or rural areas.
In 2024, the project team focused on the groundwork. This includes ongoing consultations with RCH staff to ensure the success of the project, creating recruitment and workflow protocols, obtaining preliminary data and establishing assessment protocols.
As of January 2025, the team’s protocols have been fully optimised, and recruitment has commenced—setting the stage for an active and productive year ahead.
“Thanks to the Good Friday Appeal, we will be able to create a new level and higher standard of care for neurodevelopmental assessments delivered via telehealth. This has the potential to remove barriers and ensure more accurate and timely diagnoses and clinical results, helping change children and young people’s lives.”
Associate Professor Jonathan Payne, Principal Research Fellow at the Murdoch Children’s Research Institute
Impact Milestones
2024
Actively collaborated with RCH clinical leads to align the study protocols with the needs and demands of clinical practice. This is important so the methods the team are aiming to validate will be feasible for RCH clinicians to put into everyday practice.
Results of the consultation have guided detailed assessment protocols and the procedural manual for the study so that telehealth assessments will be readily implementable into daily clinical practice.
Recruitment strategies and workflows have been created.
Preliminary data from all study arms have been obtained and are proving very valuable:
In the neuropsychology arm, the methods we are using for telehealth assessment of learning challenges and intellectual ability appear to be produce accurate results and have been acceptable to clinicians and families.
In the autism arm, preliminary assessments revealed that our adapted telehealth procedure, based on current best practices, required a minor modification to gather additional information from the child. This adjustment ensures the assessment process is more comprehensive and clinically meaningful.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Cardiology Research
Thanks to support from the Good Friday Appeal, researchers at the Murdoch Children’s Research Institute (MCRI) are helping to ensure a brighter future for children with cardiac conditions.
Associate Professor Jonathan Mynard is a bioengineer and cardiovascular researcher at the MCRI, specialising in paediatric hypertension, medical devices, cardiovascular modelling, blood pressure and flow dynamics (haemodynamics), and congenital heart disease. Jonathan’s current research project is looking at the difference in blood pressure measurements between a child’s arms, and the impact this may have on a child’s diagnosis. It is the first study worldwide to determine the size and frequency of inter-arm blood pressure differences in children and adolescents.
Initial findings revealed that even a small difference in blood pressure measurements between one arm and the other could lead to a wrong diagnosis. “Children with high blood pressure, many of whom appear to be healthy, have a greater risk of developing hypertension in adulthood, a major risk factor for cardiovascular disease,” said Jonathan.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Precision Newborn Health
These babies, born early or with complicated medical conditions, are often at high risk of having major problems with their health and development throughout their life, with many needing ongoing paediatric care and then adult care.
Thanks to support from the Good Friday Appeal, the RCH has been able to establish a dedicated research program, known as the Precision Newborn Health Program.
This innovative research program commenced in 2022 and is working towards improving precision in predicting infants at high risk of health and developmental problems. The program also aims to create personalised intervention programs targeted at those most at risk to improve their outcomes.
Professor Jeanie Cheong, Program Lead for the Precision Newborn Health Program, explained the program would help build a healthier future for babies cared for on Butterfly.
“We aim to predict neurodevelopment, feeding and motor problems, and severe lung disease, which are common outcomes across all high-risk infant groups, and carry significant burden to these children and their families.
“Through better prediction of which infants are at highest risk of long-term poor health and developmental outcomes, we will be able to target support to those who are most at risk, which means more streamlined care and better outcomes.”
In the first two years of the program, Jeanie and her team made steady progress.
The team has refined data analysis and modelling programs to better predict developmental outcomes for babies born extremely preterm. They have also started the same research for preterm babies who are at risk of lung disease and babies who have had surgery. Additionally, they have partnered with parents and health practitioners to design an early intervention program targeted at children from age two.
Jeanie is grateful for the community’s support through the Good Friday Appeal in making this program possible.
“On behalf of the babies, children and families, I thank all the supporters of the Good Friday Appeal for their support in the Precision Newborn Health initiative. This will make a substantial contribution to improving their lives.”
Impact Milestones
April 2022 – March 2023
In the first year of the program, a steering committee and working groups were established to oversee and lead the program.
Staff were appointed, including two program managers.
A web page has been developed to keep stakeholders updated with the progress of the program.
The team began refining data analysis and modelling programs to better predict health and developmental outcomes for babies born extremely preterm. They started the same research for babies who have had surgery.
They partnered with parents and health practitioners to design an early intervention program targeted at children from age two.
April 2023 – December 2024
The steering committee and working groups continue to meet regularly to oversee and lead the program.
The program team is now well established and is strengthening collaborations with the Department of Electrical and Electronic Engineering at the University of Melbourne, the Centre for Health Analytics at the RCH, and the Royal Women’s Hospital.
The team have developed data modelling to better predict developmental outcomes for babies born extremely preterm, with similar work underway for preterm babies at risk of lung disease and babies who have had surgery.
They have partnered with parents and health practitioners to design an early intervention program targeted at children from age two. They have identified a lack of easy to access reputable information for families of high-risk babies and will fill this gap by developing a digital platform.
Everything Good Friday Appeal, straight to your inbox. Get more information about your impact and how to get involved.
Clinician Scientist Fellowships
The Royal Children’s Hospital (RCH) attracts the best medical minds from across the world to create a brighter future for children’s healthcare. Thanks to the Good Friday Appeal, the Clinician Scientist Fellowship Program offers clinicians protected and funded research time to find new cures, treatments and medications for the biggest health problems facing children. In any given year there are approximately 20 fellows at various stages of their five-year fellowship.
Professor Rick Leventer is one of the Clinician Scientist Fellows. As a paediatric neurologist, Rick sees patients with all types of neurological disorders and has particular research interests in genetic diseases of the central nervous system that affect how the brain develops during pregnancy and in the first years of life.
“Before the fellowship, I was doing most of my research in my spare time and after hours as it was a struggle to find the time to do it effectively whilst trying to balance all of my clinical duties,” said Rick.
The face of the 2021 Appeal Malu has epilepsy
Thanks to your support of the Good Friday Appeal, Rick was able to further his work as both a clinician and scientist with protected time to focus on his research. Through the Clinician Scientist Fellowship, Rick is able to offer patients and families seen in his neuro-genetic clinic, including those with epilepsy, the opportunity to be part of research on early brain development, making direct impact on his patient’s care.
Part of Rick’s research involves analysing the brain tissue collected during a hemispherectomy – a surgery to disconnect or remove parts of the brain that are causing seizures – to find the cause of epilepsy.
“With the parent’s permission, some of the brain tissue that is removed is saved and snap frozen in the operating theatre. We then take it directly to the laboratory for genetic and microscopic analysis where we try and understand why the brain may have developed abnormally to cause seizures.”
Through the fellowship, Rick and his team have already made some exciting breakthroughs, including identifying specific nerve cells that carry a genetic error which trigger epileptic seizures. This has been a significant finding not only for medical professionals, but also to help families understand the cause of their child’s condition.
Rick’s discoveries are also important for the future of children’s health, creating opportunities for further research into precision medicine to target the genetic error and guide the best surgical approaches aimed at the smallest amount of tissue necessary to control seizures.
Rick is one of 20 fellows at the RCH who are able to mentor younger clinicians interested in research.
“The funds put in to support one clinician scientist are actually multiplied many times by the influence we can have on other researchers on campus,” said Rick.
Thanks to generous support from the Good Friday Appeal, the Clinician Scientist Fellowship program offers clinicians across the Melbourne Children’s Campus protected and funded research time to find new cures, treatments and medications for the biggest health problems facing children. It also nurtures the next generation of clinician researchers.
The program is unique. Few centres across the world can provide the same protected time for clinicians to engage in research. It supports individuals who are fluent in both science and medicine to ensure the translation of research into clinical care.
In 2024, six new fellows were appointed with their research studies spanning improving care for children with sepsis – a common and devastating condition that can result in organ damage and death, demonstrating the cost effectiveness of precise surgical intervention for drug-resistant epilepsy, and improving support for parents and carers to navigate end of life decision making.
Over the past two years, 25 fellows have also been supported with their research spanning new cancer treatments, reducing hospital admissions for young cancer patients with a fever, and understanding brain microstructures in child development to guide tailored treatments for mental health and attention difficulties, such as injury prevention.
Since 2016, fellows have written over 2,000 publications and secured over $70 million in further grant funding, thanks to support from the Clinician Scientist Fellowship program.
