Children with most aggressive cancers to benefit from Australian-first personalised medicine clinical trial

18 Sep 2017

Personalised medicine for childhood cancers in Australia is a step closer thanks to the Zero Childhood Cancer program’s national clinical trial launched today.

Personalised medicine for childhood cancers in Australia is a step closer thanks to the Zero Childhood Cancer program’s national clinical trial launched today. Zero Childhood Cancer is one of the world’s most comprehensive child cancer personalised medicine studies, and is led by Children’s Cancer Institute and the Kids Cancer Centre at Sydney Children’s Hospital, Randwick.

In an Australian first, scientists from thirteen leading Australian and international research institutes and doctors from all eight of Australia’s kids’ cancer centres will work together to identify and recommend new treatment options. These will be specifically tailored to suit the individual cancers of children with the highest risk of treatment failure or relapse and give their families hope.

The Zero Childhood Cancer program recognises that each child’s cancer is unique, so they respond differently to anti-cancer treatment. Detailed laboratory analysis of tumour samples will help identify the drugs most likely to kill each child’s specific cancer.

The national clinical trial builds on a successful NSW pilot study of nearly 60 children begun in late 2015 for children with the most aggressive cancers whose chance of survival on standard treatments was less than 30%. The pilot study proved the program’s feasibility, successfully putting in place the complex logistics and laboratory testing needed to analyse patient tumours and get meaningful results back to doctors in real-time.

The clinical trial expands the program to give hope to families across the country and will enrol more than 400 Australian children over the next three years, bringing the most advanced diagnostic technologies close to home. The clinical trial is open in Sydney with other cities set to open in a staged roll-out over coming months.

Professor Michelle Haber AM, Executive Director of Children’s Cancer Institute and Research Lead for Zero Childhood Cancer, said the pilot study showed the urgent need for personalised medicine.

“Originally this pilot study was planned for 12 young patients. However nearly 60 children have been enrolled in the program due to the high demand by clinicians and parents.

“We’re thrilled to broaden the Zero Childhood Cancer program nationally, in partnership  with each of Australia’s eight child cancer treatment centres and leading national and international research centres, in order to deliver child cancer personalised medicine to every child at highest risk of treatment failure, wherever in the country they may live,” she said.

A/Professor Tracey O’Brien, Director of the Kids Cancer Centre at Sydney Children’s Hospital, Randwick said cancer is the biggest killer of children by disease in Australia.

“Despite the dramatic increase in childhood cancer survival rates over the last sixty years from virtually 0 to 80%, three children and adolescents die every week in Australia from cancer.

“The challenge in curing every child is that each child’s cancer is unique. Every day on our wards we face the challenge of trying to find the best possible treatment for each child with cancer, especially those with the most aggressive cancers.  It is a balance finding specific treatments that will kill the cancer cells but minimise harm to the child.

“I truly believe the Zero Childhood Cancer program is a potential game-changer in how we treat high-risk cancer. As the Zero Childhood Cancer program is implemented, and as we gather more information, we will improve our capacity to identify the most effective treatment for each child’s cancer,” she said.

Professor Haber said personalised treatment gives kids with the most aggressive cancers the best chance of surviving their disease because it is based on reliable scientific information, such as individual genetic mutations, unique to that child’s cancer.

“Using the latest molecular profiling techniques and laboratory testing of patient cancer cells with anti-cancer drugs, Zero Childhood Cancer will give the most detailed diagnosis possible in Australia to date for children with the most aggressive cancers. It is one of the most complex and comprehensive personalised medicine programs in the world,” she said.

Of the over 950 Australian children and adolescents diagnosed with cancer each year, 150 are diagnosed with cancer types with less than a 30% survival rate, and a further 60 relapse and then have less than a 30% chance of cure. It’s these children – including those suffering from aggressive brain tumours, sarcomas, infant leukaemias and neuroblastomas – who will benefit from the Zero Childhood Cancer Program. The trial will be open to every Australian child with high-risk childhood cancer regardless of the underlying type/diagnosis.

Sherie Polzin says the program will offer children like her one-year-old daughter Ava with high-risk leukaemia the best chance of success.

“As a mother of a childhood cancer patient, the Zero Childhood Cancer clinical trial is really exciting. The collaboration of national and international hospitals and medical research institutes working together, striving for the same result, to give high risk patients the best chance of success is inspiring. It is so uplifting to know that we are one step closer to potentially curing childhood cancers.

This will make such a difference to families by giving them hope and encouragement knowing that their child is receiving the best possible treatment that is tailored to their individual requirements,” she said.

While Ava is currently not enrolled in the clinical trial as she undergoes her own treatment plan, she is an example of the types of patients diagnosed with aggressive childhood cancer who will benefit from the Zero Childhood Cancer program.

A significant group on the trial to benefit will be children with relapsed cancer, whose survival rates are usually much lower than when first diagnosed and with limited future treatment options. About half of the children on the NSW pilot study had relapsed cancer.

Cancers evolve so a child’s cancer at relapse can be quite different at the molecular level to the cancer at diagnosis.

With Zero Childhood Cancer, where possible, relapsed cancers will be biopsied, which is not currently the standard of care for most cancers. Relapsed cancer cells will be analysed in unprecedented detail to give doctors much more information to guide targeted treatment decisions. This gives children with high-risk relapsed cancers the best hope yet.

Another benefit of personalised medicine is the potential to refine or change an individual child’s cancer subtype. Cancer diagnoses may be changed once detailed genetic and other molecular tests are done, opening up new treatment options. Several children on the pilot study had changed diagnoses as a result of detailed testing.

A/Professor O’Brien said targeted therapies such as those identified through Zero Childhood Cancer will allow a much more sophisticated approach.

“The information we gather will benefit children on the program first and foremost but will also be incorporated into future frontline treatments. The knowledge gained is likely to unlock further scientific discoveries that will also ultimately benefit future patients.  Most of all, it will bring us a step closer to our vision of one day curing all children of cancer.”

“This is a very exciting initiative that has the potential to revolutionise the way in which treatment decisions about childhood cancer will be made.”

“We believe this will improve survivorship whilst reducing the impact of drug toxicity. The scale and sophistication of translating this type of discovery directly to the patient’s bedside in real time, wherever they are in Australia, is unprecedented, it is tomorrow’s care today” she said.

The Zero Childhood Cancer national clinical trial will run until at least 2019. The data gathered will enable evidence-based treatment options in the present, and build a powerful research repository for the future. Data from the program will be shared with all clinical and research partners around Australia, in Europe and USA.

The program is free to children who meet the clinical trial enrolment criteria and enrolment is through their treating oncologist. The trial is sponsored by the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG).