Research Impact

Our research has a significant impact on the lives of children with cancer in the past, present and future.

Back in the 1960s, the overall survival rate for childhood cancer was a dismal 30% [1]. Today, it has risen to over 80% in Australia and other high income countries [2]. For some types of cancer, such as acute lymphoblastic leukaemia, the rate is even higher.

There can be no doubt that, without research, survival rates would not have reached these levels. Of course, changes to clinical care have also played a major role − for example, the move towards treatment of children by multidisciplinary teams in specialised cancer care centres.

At Children’s Cancer Institute, our focus has always been on translational (bench to bedside) research, and close collaboration with our clinical partners has been a critical ingredient in our success. Today, just as in the past, it is the combined effort of scientists and clinicians that leads to real progress.

Professor Maria Kavallaris AM, Head of Theme, Head of Research Engagement

Now, more than ever, we are working with others in a global partnership to fight this fight together.”

- Professor Maria Kavallaris AM, Head of Theme, Translational Cancer Nanomedicine, Head of Research Engagement, Children's Cancer Institute

Research today

The critical role of basic research

To work out how to treat a disease, we must first understand the nature of that disease. This is where basic research comes to the fore. Countless hours of laboratory research is needed to build up a picture of each type of childhood cancer… its key characteristics, what drives its growth and spread, and how it might best be tackled.

A good example is our research into the deadly brain cancer, diffuse intrinsic pontine glioma (DIPG). We were the first in Australia to establish a dedicated DIPG research team, and the first to establish a DIPG tumour bank which enabled DIPG cells to be grown in the lab for experimental work.

Our impact in action

Drug development

Without research, there would be no new cancer treatments. To develop a new treatment, scientists must first discover a weakness in the cancer that can be targeted − a chink in its armour. This may be a critical gene, for example, or another type of molecule in the cancer cell. Next, a drug must be found that is capable of inhibiting (cancelling the effect of) that molecule.

Over the years, our researchers have worked on a number of such ‘targeted treatments’ for use in children with cancer. A recent example is a potent drug combination for the treatment of children with neuroblastoma.

Before a new treatment can be trialed in children with cancer, it must first be thoroughly tested in ‘preclinical models’ – cancer cells growing in culture, and animal models that represent the human cancer as closely as possible.

Developing relevant and useful preclinical models is a major research undertaking. At Children’s Cancer Institute we have leading experts on the job, and over the years we have developed some of the best models of childhood cancer in the world.

Our impact in action

Improved diagnosis and monitoring

A significant factor in rising survival rates for childhood cancer has been improved diagnostics. The successful treatment of a cancer relies on its accurate detection and classification. And once treatment has begun, the growth and spread of the cancer must be carefully monitored to assess how well the child is responding to that treatment.

One of the biggest advances in treating acute lymphoblastic leukaemia in recent years resulted from the development of a new method of detecting and measuring leukaemic cells, called minimal residual disease (MRD) testing. Here at the Institute, our scientists developed a unique MRD technique which is today used by clinicians throughout Australia to predict relapse in children with this disease.

Professor Michelle Haber Executive Director

We are focused on attacking the Achilles heel of the most aggressive, difficult to treat cancers. Those that are still claiming too many children’s lives."

- Professor Michelle Haber AM, Executive Director, Children's Cancer Institute

Remaining challenges

There are major challenges that must be overcome if we are to further improve survival rates for childhood cancer, as well as improve quality of life for survivors.

For some types of cancer, such as certain brain tumours, there are still no effective treatment options. For other cancers, the only treatments available cause serious problems, sometimes for the remainder of the child’s life. Even when a child is successfully treated, there is the ever-present threat of relapse, and the wait to see if there will be ‘late effects’ that become apparent as the child matures.

These challenges require novel solutions, and it is research that will provide these.

Research tomorrow

Looking to the future, there is every reason to feel optimistic about where childhood cancer research is going and what it will achieve.

Technology is advancing in leaps and bounds, continually bringing new possibilities to light. For example, in the field of nanotechnology, our scientists are working on creating nanoparticles (chemical structures on the tiniest scale) that can package and deliver therapeutic drugs directly to cancer cells, even in the most difficult to reach places.

A growing focus on personalised medicine – such as in the Zero Childhood Cancer program − is giving children diagnosed with even the most aggressive cancers fresh hope for survival, at the same time as teaching scientists more and more about individual childhood cancers and what drives them.

With increasing global cooperation in cancer research, progress is accelerating at an unprecedented rate. International collaboration on research projects is now commonplace, while online data sharing – for example, data generated by the genomic analysis of individual children’s tumours − is giving scientists all over the world access to information never before available.

Perhaps most exciting of all, as we grow our understanding of the causes of childhood cancer, it may become possible to prevent cancer in some children.


  1. Cancer Epidemiology (2020); doi: 1016/j.canep.2020.101733
  2. Australian Institute of Health and Welfare: Australia’s children (2020)

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