Neuroblastoma is the most common solid tumour in early childhood, with average age of diagnosis of one to two years old.

It's critical that we progress our discoveries into actual treatments for kids as quickly as possible.

96-well plate in lab for testing

Neuroblastoma is a childhood cancer of specialised cells involved in the development of the nervous system and other tissues. These tumours are most commonly found in the adrenal glands (above the kidneys) and in tissues around the spinal cord in the abdomen, pelvis, neck and chest.

Neuroblastoma is the most common solid tumour in early childhood, with average age of diagnosis of one to two years old. It is rare in children over 10 and very rare in adults. Most patients are diagnosed with advanced disease that responds poorly to conventional therapies.

Neuroblastoma claims more lives of children under the age of five than any other cancer. The survival rate for high-risk neuroblastoma is about 50%.

About 20% of neuroblastomas have tumour cells that contain many, even hundreds of copies of the cancer gene (oncogene) called MYCN. MYCN has critical roles in cell growth and differentiation, metabolism, and genome stability. Multiple copies of the gene, called ‘MYCN amplification’, represent a powerful marker of aggressive, high-risk neuroblastoma.

For more information about neuroblastoma, see the websites of:

Please note that, while Children’s Cancer Institute conducts medical research into childhood cancer and works closely with clinicians, we do NOT treat patients and are unable to provide treatment advice. For questions or concerns about an individual child’s cancer treatment, or to find out about relevant clinical trials, please contact your treating oncologist (cancer specialist).

Violet’s story

Violet had been complaining of tummy upsets and aches and pains for several months, but everyone thought it was just growing pains. In November 2015, an ultrasound picked up the real cause – a tumour the size of a fist near her left kidney. Her official diagnosis was Stage 4 neuroblastoma. Read Violet’s story.

Our neuroblastoma research

Examples of our neuroblastoma research teams, achievements and projects include:

  • Research led by Professor Michelle Haber in our Experimental Therapeutics program showed that neuroblastoma formation and growth is reduced when a biochemical pathway called the polyamine pathway is inhibited with a drug called DFMO combined with chemotherapy. An international Phase I clinical trial of DFMO in children with relapsed neuroblastoma is underway. This is led by Sydney Children’s Hospital, Randwick, and the cost of the DFMO is being supported by The Kids Cancer Project and Lions Childhood Cancer Research Foundation.
  • CBL0137 is a new anti-cancer compound developed by our long-standing, close research collaborators at Roswell Park Cancer Institute in the USA. A clinical trial of CBL0137 in adult cancer patients is underway. When we tested CBL0137 in a laboratory model of neuroblastoma, we observed unprecedented cure rates. A Phase I clinical trial of CBL0137 is planned, that will be led by Sydney Children’s Hospital in children with high-risk neuroblastoma and other aggressive childhood cancers. The trial will be run through the North American Children’s Oncology Group (COG) Phase I consortium, and involve the largest paediatric oncology centres in the US, and Sydney Children’s Hospital, Randwick.
  • Researchers in Professor Glenn Marshall’s Molecular Carcinogenesis program are using a new technique called single cell profiling to investigate how neuroblastoma tumours begin. They are also exploring ways to target MYCN-related molecules and pathways as a potential neuroblastoma treatment strategy.
  • Our Tumour Biology and Targeting researchers led by Professor Maria Kavallaris are investigating a chemically-modified natural compound called dextran-catechin as a future neuroblastoma treatment. This molecule targets copper metabolism, an emerging target for anti-cancer drug design.
  • Our Histone Modification researchers led by A/Professor Tao Liu discovered a new long, non-protein coding RNA found in large amounts in high-risk neuroblastoma. We are working to define its role in modifying DNA structure to promote neuroblastoma tumour growth, and will test new combination therapies to target it.
  • The Zero Childhood Cancer national personalised medicine program, led by Children’s Cancer Institute in partnership with Sydney Children’s Hospitals Network, opened a national clinical trial in late 2017. It includes children with the most serious cases of infant, childhood and adolescent cancer, including neuroblastoma.