In a world-first precision medicine study of children enrolled in Zero Childhood Cancer Program (ZERO), Australian researchers and clinicians have shown that precision medicine – where treatment is tailored to an individual child’s cancer – leads to significantly improved outcomes in children with high risk cancer.

Luminesce Alliance funded several components of the ZERO program*, which is jointly led by two of Luminesce Alliance partners: Children’s Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital, Randwick.

In the study, published 6 June 2024 in the leading international journal Nature Medicine, the researchers found that precision medicine (also called personalised medicine) was shown to be superior to standard or non-guided therapy, both in terms of clinical response and survival.

Associate Professor Loretta Lau, a paediatric molecular oncologist at Kids Cancer Centre, researcher at Children’s Cancer Institute and first author on the paper, and Professor Glenn Marshall AM, Clinical Lead of ZERO and co-senior author on the paper, said the world-first study shows that precision medicine has the potential to change the model of care for children with cancer.

“Our study provides important new evidence that response to precision-guided therapy translates into improved survival,” said A/Prof Lau and Prof Marshall.

“This work provides some hope to families where none previously existed in a new model of treatment for high-risk childhood cancer which is changing national and international clinical practice.”

The study involved more than 100 scientists and clinicians working together across 9 child cancer centres. Over a minimum follow-up period of 18 months, the study included 384 children with high-risk cancers (with a very low chance of cure).

To determine the impact on survival, the researchers measured ‘progression-free survival’, the length of time that a patient lives without their cancer getting any worse. The results showed that children who received a ZERO-recommended treatment did significantly better than those who did not. In fact, their 2-year progression-free survival was more than double that of children who received standard therapy (26% vs 12%), and five times higher than that of children who received an unguided treatment (a novel agent not chosen on the basis of molecular findings).

Importantly, the study found that children who received their recommended therapy early on in their treatment pathway did significantly better than those who received it after their disease had progressed, suggesting that the sooner a personalised treatment strategy can be implemented, the better the chance of preventing relapse and death.

*Luminesce Alliance funded the following components of the ZERO program, mentioned in the Nature Publication:

“More recently, the INFORM study showed improved survival outcomes limited to patients with high-evidence targets2.”

• INFORM2: An exploratory multinational phase I/II combination study of Nivolumab and Entinostat in children and adolescents with high risk Malignancies – Luminesce Alliance
• The INFORM2 Clinical Trial opens in Australia – Luminesce Alliance

“The ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial used whole-genome sequencing (WGS) (paired tumor-germline), and transcriptomic sequencing and DNA methylation, to identify molecular targets in high-risk cancers.”

• The Luminesce Alliance Computational Biology Program developed, benchmarked, and optimised tools for genome and transcriptome analysis for more than 970 patients enrolled in the Zero Childhood Cancer Program (ZERO) PRISM trial. These tools are now being used in subsequent expansion of the ZERO Childhood Cancer Program.

For more information

• Children’s Cancer Institute Australia announcement
• The full manuscript online at https://www.nature.com/articles/s41591-024-03044-0

About Luminesce Alliance

Luminesce Alliance is a not-for-profit cooperative joint venture between the Sydney Children’s Hospitals Network, the Children’s Cancer Institute, the Children’s Medical Research Institute, the University of Sydney and UNSW Sydney. It has been established with the support of the NSW Government to coordinate and integrate paediatric research.

 

On Tuesday 18 June 2024, Treasurer the Hon Daniel Mookhey MLC delivered the 2024-25 NSW State Budget.

This Budget includes $13.4 billion to upgrade hospitals across the State and building housing for key health workers; $480.7 million to relieve pressure on emergency departments; $274.7 million Essential Health Services Fund to help recruit 250 new healthcare workers to new hospitals across the state; and $130.9 million Family Start Package to improve access to care for families including early intervention programs to boost lifelong maternal and child health.

Investment in hospital infrastructure

$3.4 billion in 2024-25 to upgrade hospitals and health facilities across the State.

The Building Better Hospitals Package commits $265.0 million for a critical upgrade of Port Macquarie Hospital and an additional $395.3 million to deliver ongoing hospital redevelopments at Eurobodalla, Ryde, Temora, Mental Health Complex at Westmead, Liverpool, Moree, Nepean, Cessnock and Shellharbour.

• A further $250.0 millionwill be invested across NSW hospitals as part of the Critical Asset Maintenance Program. Development also continues for the new Single Digital Patient Record system, a next generation system which will consolidate and make it easier for clinicians to access patient information.

Relieving pressure on our emergency departments

The NSW Government’s Emergency Department Relief Package invests $480.7 million to help to avoid an estimated 290,000 visits to emergency departments each year once fully implemented.

• $171.4 million to establish a ‘Single Front Door’ – a single point of advice, assessment, triage and referral for people in NSW with urgent non-life threatening conditions, backed with the introduction of three additional virtual care services and helping 180,000 people avoid a trip to the ED •
• $100.0 million to back in urgent care services, a key instrument of the health system that will provide a pathway to care outside of hospitals for an estimated 114,000 patients •
• $70.1 million to expand emergency department short stay units to improve patient flow and reduce ED wait times by nearly 80,000 hours •
• $15.1 million for an Ambulance Matrix that enables paramedics to transport patients to emergency departments with greater capacity and reduces wait times by providing real time hospital data • $31.4 million to increase Hospital in the Home across the State allowing over 3,500 additional patients each year to be cared for in their home rather than a hospital bed
• $53.9 million to improve patient flow and support discharge planning by identifying patients early on that are suitable to be discharged home with the appropriate supports in place.

 Supporting primary healthcare through GPs

• $188.8 million as part of the Bulk[1]Billing Support Initiative to ensure that primary health care services remain accessible to families and households and to protect the cost of seeing a GP for families and households, reduce the strain on emergency departments

 investing in our frontline health workers

• $274.7 millionEssential Health Services Fund, to help recruit 250 new healthcare workers to new hospitals across the state including at Prince of Wales Hospital, Tweed Hospital, Bowral, Sutherland, Wentworth, Cowra, Cooma, Glen Innes, and Griffith.
• $200.1 million expansion of the NSW Health Key Worker Housing Accommodation program to house health workers in regional NSW to better recruit and retain essential health workers across the state. Early assessment has identified a number of possible future locations such as Lismore, Eurobodalla, Tweed Heads and Lake Cargelligo.
• delivering an extra 500 regional paramedics
• abolishing the wages cap and delivering the highest wage increases for health workers in over a decade.

For full details:

• Media Release: 2024-25-nsw-budget-park-a-plan-to-build-a-better-health-system-for-nsw-communities.pdf
• Budget papers: 2024-25 NSW Budget Papers

 

A new research centre focused on improving the care and wellbeing of cancer survivors will be established as part of a new 10-year partnership between UNSW Sydney and Cancer Council NSW (CCNSW).

The launch of the Australian Research Centre for Cancer Survivorship is the first of its type focused entirely on transforming the lives of cancer survivors through an evidence-based approach and world-class researchers.

Created through a joint investment of $40 million from UNSW and Cancer Council NSW, the centre will leverage the expertise of each organisation as well as building new capacity to drive research, education and advocacy in cancer survivorship. Critically, the centre will partner with consumers and industry to address critical issues for cancer survivors. A major focus of the work will be breaking down barriers to access in regional and rural settings and greater engagement with primary care.

For full details visit UNSW website

This article and image have been reproduced with the gracious permission of UNSW Sydney.

Luminesce Alliance paediatric precision medicine clinician researchers, Professor Sue Woolfenden and Dr. Alan Ma, feature in Research Australia’s latest report, Clinician Researchers: Research Activating the Health System. The report highlights the breadth and variety of the clinician researcher role, by profiling more than 30 clinician researchers from across Australia at different stages of their careers.

Professor Russell Gruen, Dean of the College of Health and Medicine at Australian National University and a Research Australia Director said, ‘Clinician researchers bring both science and service to healthcare and translate the findings to benefit patients. They are motivated by truth and inspire the same in others.

‘Such careers should be valuable and rewarding. Yet those that choose this path often face many challenges.’

We encourage you to read the report to discover the invaluable role played by clinician researchers and hear their feedback on their challenges, barriers, and critical areas for change, to ensure they can continue to serve our community.

To find out more about the report visit Research Australia.

Professor Sue Woolfenden is Professor of Community Paediatrics at the University of Sydney and Director of Community Paediatrics within the Clinical Services Integration and Population Health Directorate at Sydney Local Health District. Prof. Woolfenden is integral to the development of Luminesce Alliance Psychosocial Platform and associated research.

Dr Alan Ma is a Clinical Geneticist at Sydney Children’s Health Network, Research Translation Fellow and Senior Lecturer at the University of Sydney. Dr Ma’s Luminesce Alliance funded research has contributed to improve the patient journey for children with rare genetic diseases.

 

With programs like Sydney Children’s Hospital Network’s (SCHN) Kids Advanced Therapeutics (KAT) program, we now have the potential to treat and improve the quality of life of children with rare diseases, who previously had no options.

We invite you to view the introductory webinar to discover how clinicians and researchers are preparing for a new wave of therapies that will revolutionise the future of paediatric health care. Read more

At the recent ZERO Symposium, supported by Luminesce Alliance, the Zero Childhood Cancer Program (ZERO) announced the adoption of the Beacon protocol to launch a new portal that enables the sharing of large genomic data sets across the research community, to strengthen analysis of childhood cancers. The Beacon platform does not require specialised skills to extract the data and is built to the GA4GH standard for sharing genomic and health data. ZERO is encouraging collaborators to host similar Beacons so that all childhood cancer data can be cross-shared through a single interface. Read more 

A study published in the latest issue of Cancer Medicine conducted interviews with surgeons involved in the Luminesce Alliance supported PRISM national precision medicine trial for poor prognosis paediatric cancer patients. The study examines the perspectives of surgeons, delving into their attitudes, experiences, confidence and knowledge of genetic concepts. Read more