New clinical trials for children with high risk neuroblastoma- Children’s Cancer Foundation

Neuroblastoma, a solid tumour, almost exclusively occurs in children aged 0-5 years. High-risk neuroblastoma, the most aggressive form, has poor survival rates and continues to cause a disproportionate number of children’s cancer deaths.

The Children’s Cancer Foundation is proud to announce funding to open a suite of international neuroblastoma clinical trials offering optimised treatment for around 180 patients over the next 4 years. Together with our funding partners the Steven Walter Children’s Cancer Foundation and Hyundai Help for Kids, a grant of $531,000 has been awarded to open these trials targeting high risk, low and intermediate risk and infant neuroblastoma at six hospitals across Australia and New Zealand.

Leanne Shaw, mother of Ava diagnosed with Stage 4 high-risk neuroblastoma at age 2, shares the painful details of this aggressive and rare cancer that continues to take a devastating toll on her family.

The painful diagnosis of our little girl’s neuroblastoma

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In March 2016 our beautiful little daughter Ava was born. Around her second birthday, Ava, a bright, bubbly and active toddler became unwell: she was lethargic, crying often and didn’t want to play with any of her toys or interact with others. We knew something wasn’t right.

We took her to a doctor several times to be told it was a virus and then an infection, which we treated with antibiotics.

The day Ava screamed for over 1.5 hours inconsolably, we realised she hadn’t touched her toys for 4 days. She began to limp later that same day. This was the turning point; we decided to take Ava straight to The Royal Children’s Hospital in Melbourne.

Initially, the doctors suspected she had septic arthritis. They proceeded with an ultrasound, X-ray and MRI. While waiting for my husband James to return to the hospital, five doctors walked into Ava’s room and closed the door. My gut instinct said ‘cancer’. An oncologist explained they had found tumours on Ava’s MRI scans. When James arrived, they detailed the extent of cancer in Ava’s tiny little body. It was bad. Very bad.

At just 2 years of age, Ava had Stage 4 high-risk neuroblastoma – the biggest killer of childhood cancer patients has the lowest survival rate, is incredibly tough to treat and has a high rate of relapse for those that survive.

Ava had a primary mass so big, in her adrenal gland above her left kidney; it was squashing her kidney completely.

She also had tumours in the bones in her legs and pelvis. The cancer was in her bone marrow, blood, lymph nodes and lungs. It was sickening to see cancer hotspots in her eye sockets and skull.

Treatment began almost immediately – we were admitted for 3.5 weeks at first and she received more scans, a central line to her heart, a nasogastric tube for food and medication, and her first cycle of chemotherapy. From that point onwards, our lives changed.

Fighting for outcomes after treatment cycles

James, who was a stay at home dad and working casual shifts on weekends, stopped work. We had moved to Australia (from the UK) in 2015, I was the main earner in our family and now had to take significant time off work or work remotely. In September 2018. I gave birth to our second child, Angus. Today, I work full time to support my family while James remains the primary carer for both Ava and Angus.

Ava’s life went from days filled with crèche, dancing, swimming and GymbaROO to hospital visits, blood transfusions, chemotherapy and suffering.

Ava has received eight cycles of chemotherapy, major surgery to remove the primary tumour, and two stem cell transplants (SCTs). She is still recovering from the second stem cell transplant in hospital for which the stay has already been 9 weeks with no confirmed date of release yet.  Despite all of this treatment, Ava still has such a long journey ahead – 1 month of radiotherapy and 6 months of immunotherapy.

At this stage, we are awaiting scans to see the impact of the SCTs and we hope they have indeed have wiped out all signs of disease.

Ava meanwhile, has been exposed to double the amount of chemotherapy and two-times the number of transplants, which causes long-term side effects such as hearing loss, heart defects, kidney and liver failure, infertility, growth issues. She has experienced a reduction in her hearing and in her kidney function, she is unable to walk properly, she is suffering significant neuropathic pain requiring daily pain relief. Her gut is also not functioning causing Ava to require hospitalisation for IV nutrition.

Ava-Wake-_-Ballerina_WEB_READY.jpgThroughout all this, Ava has been a model of hope, strength and motivation. She lives each day as it comes, drawing the happiness from any given situation. She keeps us strong.

It’s certainly the biggest challenge we have faced. We hope that soon, Ava will be able to travel to New York to receive treatment of a trial drug not funded in Australia, which has promising results in stopping relapse of neuroblastoma. We are determined to give our little girl the best and longest life possible.

By sharing our story, I hope to raise awareness of this childhood cancer to ensure better support. Ava and other kids with neuroblastoma deserve to have their stories told.


Extending the clinical trial through to translational research

The Children's Cancer Foundation will also be providing a further $671,000 for a linked translational research program for the SIOPEN high-risk neuroblastoma clinical trials through the Children’s Cancer Institute in Sydney. This program will focus on understanding relapse from this aggressive disease, improve residual disease detection and develop testing models for high-risk neuroblastoma.


Photo Credit: Leanne Shaw, Mother of Ava

Ava Wake, High-risk Neuroblastoma patient