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. 

Last updated February 2025.

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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.

Last updated March 2025.

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A National Centre of Excellence in Speech Genetics: Transforming Patient Care

Even with intense therapy, children with apraxia have severely impaired speech development, which has lifelong impacts on literacy, educational and employment outcomes. 

Thanks to funding provided by the Good Friday Appeal, clinician researchers from The Royal Children’s Hospital and the Murdoch Children’s Research Institute are establishing a speech genetics clinic for speech apraxia.

The new clinic will draw on expertise in speech pathology, neurology, neuropsychology and clinical genetics.

The centre will transform standard practice for children with debilitating speech apraxia, moving from the current ‘watch and wait’ surveillance, or ‘trial and error’ symptom-based therapies, toward precision medicine, with a focus on genetic testing, advancing detection, diagnosis, genetic counselling, earlier targeted intervention and even prevention. 

This will improve life outcomes for children at the Royal Children’s Hospital and across the country.

Posted June 2020

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Children’s Cancer Centre Tissue Bank

The Children’s Cancer Centre (CCC) Tissue Bank collects solid tumour, blood and bone marrow samples from oncology patients to learn more about cancer in children.

The tissue bank staff work in collaboration with hospitals, laboratories and researchers both locally and internationally to translate those findings into potentially life-saving treatments.

“Samples are preserved with the utmost care and dignity for future clinical and research purposes. Over 50 staff including consultants, surgeons, haematologists, oncologists, specialist pathologists, researchers and research enablers work together as part of the team,” said Louise Ludlow, CCC Tissue Bank Coordinator.

“Together we are committed to improving the survival rate and long term health outcomes for children diagnosed with cancer.”

In addition to the processing of samples for clinical trials, the CCC Tissue Bank also prepares clinical samples for further molecular testing (testing for certain genes, proteins, or other molecules in a tissue sample) as standard of care.

Thanks to donor support, the CCC Tissue Bank has also participated in important research projects outside of the Melbourne Children’s campus, including research conducted by St. Jude Children’s Research Hospital in the Memphis USA, The Hospital for Sick Children in Toronto, Canada, and the Kid’s Cancer Centre, Sydney Children’s Hospital.

“The Tissue Bank is an invaluable resource which allows us to collaborate in adequately powered international studies through sample and knowledge sharing, ensuring that we remain at the forefront of cutting edge paediatric cancer research,” Louise said.

Posted June 2020

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The Kidney Flagship

Bringing together researchers from the Hospital and the Murdoch Children’s Research Institute (MCRI), the Kidney Flagship aims to reduce the burden of genetic kidney disease on patients and their families by improving diagnosis and treatment, and the development of new targeted therapies.

The new clinical pipeline will see kidney patients with suspected genetic disease undergo a genomic test that can easily be added to routine blood tests. In many cases this test will be able to find the genetic change and identify how or why kidney disease occurred.

It will also reduce the need for unnecessary biopsies and mean less time in hospital for patients.

Following this test, researchers will be able to grow or ‘model’ the disease in a lab and aim to identify targeted treatments for the patient.

Ultimately, this could lead to the development of a drug or treatment that could improve outcomes for all kidney patients.

Posted May 2020

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The CLARITY Juvenile Arthritis Research Platform

With support from the Good Friday Appeal, The Royal Children’s Hospital (RCH) Department of Rheumatology has established the CLARITY Juvenile Arthritis Research Platform to better understand and treat the disease.

The RCH and the Murdoch Children’s Research Institute are home to Australia’s only paediatric rheumatology research program, and the new CLARITY study is set to help translate research findings into practice, improving clinical care for children both locally, nationally and internationally. With the aim of enrolling 250 participants annually, the study will enable new research that aims to identify biomarkers for diagnosis, prognosis and treatment of children with juvenile arthritis.

Photo: RCH Melbourne – Creative Studio Photography
Posted June 2018

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Brain Tumour Clinical Trials

To advance the care, treatment and diagnosis of these patients at the RCH, the Children’s Cancer Centre (CCC) identified the need for a greater enrolment in international clinical trials.

The CCC now has two Clinical Trial Coordinators administering a new clinical trials program aimed at improving overall survival and ongoing quality of life for children with cancer. Though often confused with drug trials, clinical trials are aimed at developing new or improved methods of care. This includes things like:

  • innovative drug therapies,
  • the best combination of procedures to provide more effective, and
  • less toxic treatments for better long-term quality of life.

The launch of the clinical trials programs has allowed for more individualised care for paediatric brain tumour.

Clinical trials are the backbone of careThey provide access to the best level of international care. By participating in them, children at the RCH get the same care as those in big medical centres like New York.”

– Michael Sullivan, Head of Neuro-Oncology and Solid Tumour Programs

Thanks to your generous support, funds from the Good Friday Appeal and the Cancer Crusaders Auxiliary have supported brain tumour clinical trials at The Royal Children’s Hospital, Melbourne.

Posted May 2018

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Melbourne Genomics Health Alliance

Genomic medicine and exploring how it can help young patients is a new research initiative pivotal to contemporary research at The Royal Children’s Hospital.

Genomic medicine is the sequencing and analysis of a person’s genes. By looking at an individual child’s genes, diagnosis and treatment can be tailored to each child.

The Royal Children’s Hospital is a founding member of the Melbourne Genomics Health Alliance, which brings leading local healthcare, research and academic organisations together to conduct further research.

Their vision is for Victoria to be a world leader in the use of genomics in healthcare.

The Good Friday Appeal is pleased to fund the hospital’s membership to the Alliance, providing an expanded world-wide networks to the hospital’s researchers and clinicians.

Posted November 2016

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Murdoch Children’s Research Institute

The Good Friday Appeal supports the research capability and output of the Murdoch Children’s Research Institute (MCRI) and The Royal Children’s Hospital (RCH).  

MCRI is Australia’s leading child health research organisation and ranks among the top three children’s medical research institutes globally. MCRI aims to improve the prediction, prevention, diagnosis, and treatment of rare, complex diseases and common conditions affecting children and young people.  

MCRI’s mission is for all children to live healthy and fulfilling lives. Their vital research is making a life-changing difference to children not just in Australia, but around the world. From developing new treatments for inherited and chronic kidney disease to bioengineering prosthetic heart valves, the MCRI is transforming the health and lives of a generation of children and their families. 

More than 1,900 MCRI researchers are working to find answers to rare and common childhood conditions. This research allows cures, new treatments and medications to be developed for the biggest health problems facing children.

mcri

“The support we receive through the Good Friday Appeal helps us achieve great things at MCRI. With your help, we can continue to find cures and prevent illnesses and disease in babies, children and adolescents so they can live long, healthy and fulfilling lives”

– MCRI Director, Professor Kathryn North AM

Thanks to the Good Friday Appeal, MCRI can undertake significant research projects and access cutting-edge technology.

For example, the MCRI runs life-changing clinical trials to improve childhood cancer survival rates and reduce the risk of scabies in countries like Fiji and more.

Additionally, the MCRI is developing new treatments for inherited and chronic kidney disease using stem cells and bioengineering prosthetic heart valves.

The MCRI’s commitment to improving the lives of children by shaping government policy, developing clinical guidelines for diagnosing and treating children, and informing best practice is helping children live healthier and more fulfilling lives.

Sharing a campus with The Royal Children’s Hospital means patients have ready access to clinical trials and research, helping to quickly translate research into great care. Many MCRI researchers are also clinicians, which allows them to use their research to solve problems they see in their daily work with children and families.

Last updated March 2025.

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