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.

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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.

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TOTAL XVII – TOTAL Therapy Study

Paediatric acute lymphoblastic leukaemia (ALL) is the most common childhood cancer. Yet, despite the latest in clinical advances, it still remains the leading cause of cancer related death in young people. 

Thanks to the Good Friday Appeal Run for the Kids, newly diagnosed patients with 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, 61 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, with patients enrolled continuing with follow up 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.  

As well, 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 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.  

“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.  

“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. 

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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.

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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.

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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, Project Lead for the Precision Newborn Health Project, explained that the project 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,” said Jeanie.  

In the first year of the project, Jeanie and her team made steady progress. A steering committee and working groups were established to oversee and lead the project.  

The team has also begun refining data analysis and modelling programs to better predict health and developmental outcomes for babies born extremely preterm. They have also started the same research for 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 project 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.” 

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GenV research project into health problems

At The Royal Children’s Hospital (RCH), clinicians and researchers are tackling the biggest health problems facing children, from mental health, to obesity, autism and allergies.

Thanks to the Good Friday Appeal and Run for the Kids, a new digital platform will help collect information for a large-scale population health study to provide the big picture on the current health challenges facing children.

Led by the Murdoch Children’s Research Institute (MCRI) and Scientific Director Professor Melissa Wake, the ambitious Generation Victoria (GenV) research project will ask all Victorian families with babies born in 2021 – 2022 to be involved.

“GenV offers the possibility of a better future for us all. It aims to gather information from around 150,000 babies born over the next two years and their parents, and to follow them into the future. This will give us a research platform to better understand problems such as asthma, food allergies, obesity and mental illness, and give us the opportunity to better treat and prevent common and complex conditions,” said Melissa.

GenV research team from MCRI, including Melissa (front centre)

Health, development and wellbeing information from both parents and their newborns will be collected over a number of years via the Personize Digital Platform. Measuring characteristics on such a large scale will inform targeted research to change the future of children’s health.

“The Personize data platform is vital to GenV’s success. It solves research roadblocks of size, speed, cost and burden – not just for GenV, but for other studies too. Families can contribute their research data via ultra-short and engaging digital sessions. This means that everyone, no matter who or where they are, can take part easily from home,” said Melissa.

“GenV truly is a collaborative study and a partnership of many. We are profoundly grateful to supporters of Run for the Kids and the Good Friday Appeal for making the Personize data platform possible.”

Published: 3 December 2021

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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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