Why does brain cancer kill more children than any other disease?

August 2, 2020

Brain cancer kills more children in Australia than any other disease. The question that springs to mind after processing this appalling statistic is, why?

Research blog

It’s a good question. The simple answer is threefold:

  • We don’t know what causes brain cancer
  • Current treatments are not good enough
  • We need more focus and funding

In addition, there are specific issues to deal with in paediatric or childhood brain cancers.

Let’s start with the cause. The fact is, we don’t know what causes children to develop brain cancer, or adults for that matter. There are various theories, from genetic mutations to epigenetic or environmental factors, to viral infections, and research is continually making new discoveries, which improve our understanding of the disease.

But the more we discover about brain cancer, the more we realise there is still so much more to find out. Take medulloblastoma, for example, which is a common paediatric brain tumour. Because of research and a greater understanding of how brain cancer operates on a molecular level, what was previously thought of as one type of brain cancer is now known to have at least four genetic sub-types, which all require different treatments. As we discover more about the disease, we discover there are even more questions to answer regarding how to treat it.

Cure Brain Cancer Foundation has long supported research into medulloblastoma, including the work of Associate Professor Nick Gottardo, a paediatric brain cancer specialist. Most recently, A/Prof Gottardo recieved a Cure Brain Cancer Foundation Innovation Grant to further his research into medulloblastoma.

His research aims to find approaches that enhance existing treatments, prove the new methods work using laboratory techniques, then translate them into clinical trials.

Current radiation treatment works by damaging the DNA inside cancer cells, causing the cells to die. However, cancer cells often repair the DNA damage, survive, and multiply, leading to treatment failure and cancer regrowth. He has evidence radiation therapy can be improved if DNA repair is stopped.

Sadly, current treatments simply aren’t effective enough and cause considerable harm to children with the disease. In fact, the internationally recognised expert in childhood brain cancer, A/Prof Nick Gottardo, describes current treatments as “woefully ineffective”.

Brain cancer is different in children than in adults. There are forms which more commonly affect kids, such as medulloblastoma and DIPG (Diffuse Intrinsic Pontine Glioma). But even when typically adult tumours, such as high grade gliomas, do occur in children, they present very differently on a molecular level.

“They look the same under the microscope, but molecularly they are very distinct diseases….Having more information on these tumours can only benefit us in being able to choose more rational therapies in the near future.”

– A/Prof Nick Gottardo

Again, we don’t know why children manifest their own versions of this disease. Plus, children’s brains are still developing, so the standard treatments for brain cancer, which include surgery, radiotherapy and chemotherapy, can result in more substantial and permanent side effects than they would for an adult. This is a massive problem with childhood cases; applying treatment is harder than in the case of adults. Hence a major consideration when developing new treatments for paediatric brain cancer is how to provide quality of life as well as increasing survival.

“We’re all aiming for 100%, that’s our goal – to cure all children. But we also want to cure them, leaving them with a good quality of life in the long term.”

– A/Prof Nick Gottardo

A trial run by the European International Society for Paediatric Oncology found that hyperfractionated radiotherapy (radiation treatment in which the total dose of radiation is divided into small doses and treatments are given more than once a day) had less of an impact on children’s memory, planning, and organisational skills, than conventional radiotherapy. The team says this is an example of how adjusting radiation dose can help reduce side effects while still treating the tumour. In a separate study the researchers looked into different tumour types and observed that different tumours have an impact on quality of life. Furthermore, tumour sub-type is important, with children with medulloblastoma tumours containing the ‘sonic hedgehog’ gene experiencing a better quality of life after treatment than those with other medulloblastoma sub-types. This is the first time that tumour biology has been related to quality of life in medulloblastoma sub-types.

“What we want to do is find new therapies that will be more specific against the tumour and with fewer side effects, so that the children are cured and also cured with excellent quality of life in the future”.

– A/Prof Nick Gottardo

Research into paediatric brain tumours has come a long way already. Yes, there is a long way to go; survival rates for brain cancer have hardly improved in more than 30 years. But that’s not because nothing has been done. However, the number of clinical trials for children and access to these trials is limited; with a disease of such low incidence, the clinical research has historically tended to follow what has been done in adult trials, but this is changing. After years of investment in world-class research, impactful advocacy and widespread awareness, we are now seeing results.

In 2015 Cure Brain Cancer Foundation provided the $1.3 million of early funding needed to get the Zero Childhood Cancer program off the ground. Four years on, Zero is a nation-wide, world-class personalised medicine program for Australian children with deadly and hard to treat cancers, with 81 brain cancer patients enrolled so far.

Twelve-year-old Jack is one of those patietns. The reality is, four years ago, children like Jack had very little hope.

Jack and his mum, Vivian

But Jack wasn’t diagnosed with brain cancer four years ago, he was diagnosed in 2017 and suffered an aggressive, and likely deadly recurrence in early-2018.

His brain cancer had not only returned, but it had spread throughout his brain and spine. Before long, the once athletic junior tennis player was wheelchair-bound and the fluid on his brain was beginning to affect his eyesight.

Jack was enroled in Zero, which works by taking a truly personalised approach to treatment, attempting to match the patients’ specific tumour with a potential therapy.

In Jack’s case, tests showed his tumour contained a mutation known as BRAF V600E. Doctors believed this was driving the rapid growth of his cancer and personalised therapies were developed to target the mutation. They worked.

After just 45 days of receiving treatment with a simple oral pill, Jack was out of his wheelchair and back playing tennis again. All with minimal side effects. He’s now back at school and his doctors believe he has made a full recovery. Jack is a tangible example of how funds raised through events like Walk4BrainCancer really do make a huge difference. This incredible work being carried out at Children’s Cancer Institute, the Kid’s Cancer Centre, the Sydney Children’s Hospital at Randwick and collaborators across the country would not have be possible without the earlier pilot study, funded with the help of Walk4BrainCancer.

Hearing Jack’s story was a hugely proud moment for us at Cure Brain Cancer Foundation, as we can genuinely say to everyone who has donated to the Foundation that they have helped save Jack’s life. Four years ago, Jack probably would have died. In 2019, he is thriving.

Zero project lead, Professor Michelle Haber, along with her inspirational team, deserve special praise for their incredible work, as do the other funders who have backed Zero.

We were proud to stand alongside both the Federal Government and Minderoo Foundation at the launch of game-changing initiatives like the Australian Brain Cancer Mission in 2017, and we will continue to partner with them and other foundations who share our desire to find a cure for brain cancer.

So much has changed in the brain cancer landscape in recent years, but unfortunately stories like Jack’s are still the exception rather than the norm. We won’t stop investing in innovative programmes like Zero until every Australian diagnosed with brain cancer has an outcome like Jack’s.

Over the coming years, Zero will collect molecular, biological, drug response and clinical data will from hundreds of children diagnosed with the worst types of cancer in Australia. This will provide an unprecedented, comprehensive and unique dataset that will help identify new treatments for patients now based on their specific tumour type, accelerate paediatric clinical trials access to new drugs for children, and direct innovative research to accelerate translation from the laboratory to the clinic in the future.

Zero Childhood Cancer launch

It is anticipated that 35 to 50% of all patients enrolled on Zero will be diagnosed with CNS (brain and spinal cord) tumours, creating a wealth of knowledge that does not currently exist. Importantly, this information will be shared globally, increasing the existing data available and expanding the body of knowledge. A strong relationship between Children’s Cancer Institute and the German Cancer Research Centre (DKFZ) in Heidelberg are examples of this international collaboration that will undoubtedly benefit children diagnosed with these tumours in future.

And last year, Cure Brain Cancer Foundation and The Isabella and Marcus Foundation also dedicated $400,000 to BIOMEDE – a clinical trial for Australian children with DIPG. This enables newly diagnosed Australian children with DIPG as to receive cutting-edge, experimental treatment for their deadly tumour.

Previously, the only DIPG treatment available in Australia was very limited, with patients surviving an average of just nine months. Consequently, many children and families travelled overseas at great cost to access treatments.

BIOMEDE is the first adaptive clinical trial in Australia to analyse a child’s tumour to identify which drugs approved for the trial are most likely to be effective. This means the majority of trial participants will receive tailored treatment, based on the genetic profile of their tumour. The trial’s flexible ‘adaptive’ nature also means that new drugs can be added to the trial if they show increased promise.


Cure Brain Cancer Foundation is also funding Dr Ryan Cross at the Walter and Eliza Hall Medical Research Institute in Melbourne. Dr Cross is testing the effectiveness of Chimeric Antigen Receptor T (CAR-T) cell therapies for children and adults with gliomas, particularly DIPG. T cells are white blood cells which recognise and kill tumour target cells. This immunotherapy project will arm these T cells with genetically engineered ‘weapons’ to kill cancers in children and adults, with the aim to rapidly translate any successful findings into clinical trials.


Professor Stewart Kellie, a paediatric neuro-oncologist and oncologist at The Children’s Hospital at Westmead, says there have already been huge advances, but says global collaboration is key to success when it comes to paediatric clinical trials and research. By pooling resources, answers can be arrived at more quickly.

“I think the biggest change that I’ve seen in my professional career has been the incorporation of research – and particularly clinical trials – into the frontline treatment of children”.

– Prof. Stewart Kellie

As you can see, progress is being made, but many questions remain regarding childhood brain tumours. We don’t know why brain cancer occurs. But, crucially we need to answer the question ‘how?’ How do we treat this disease? How do we improve survival, as investment in research has done for other diseases such as leukaemia and breast cancer? How do we give children diagnosed with brain cancer a more hopeful prognosis?

Together, we will find answers to those questions and change the world for children with brain cancer, both now and in the future.

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