40 children are diagnosed with neuroblastoma each year in Australia.
Neuroblastoma claims the lives of more children under 5 than any other cancer.
Neuroblastoma is usually advanced by the time it is diagnosed, making it difficult to treat.
AMXT1501/DFMO (polyamine inhibition) therapy
Update - 2 August 2024
Regarding the use of AMXT1501/DFMO (polyamine inhibition) therapy to treat cancer, we have recently learnt of the possibility of developing severe cardiac toxicity resulting in cardiac arrest.
Consequently, a decision has been taken to pause development of the planned clinical trial of AMXT1501/DFMO in children with high-risk neuroblastoma and DIPG (diffuse intrinsic pontine glioma) until further information is available.
While AMXT1501/DFMO remains a potentially effective treatment approach, trialling this drug combination in children must be done safely and with all data to hand.
We will therefore be carrying out extensive laboratory testing to explore a potential link between this drug combination and cardiac toxicity.
We are looking for any evidence of cardiac toxicity in our living models of disease, and collaborating with cardiac experts to understand the mechanism of toxicity, and to investigate if any such toxicity can be avoided by changes in drugs, dosages or scheduling.
We also await the results of the AMXT1501/DFMO Phase 1 clinical trial currently underway in adult cancer patients.
What is neuroblastoma?
Neuroblastoma is a cancer that develops in specialised cells of the nervous system, called neuroblasts. The cells grow out of control, forming a tumour, most often in the adrenal glands above the kidneys, or in the tissues around the spinal cord.
How common is neuroblastoma in children?
Neuroblastoma is the most common solid tumour found in children under five, with most cases diagnosed at around two years of age. Each year in Australia, about 40 children are diagnosed with neuroblastoma.
What happens when a child gets neuroblastoma?
Children with neuroblastoma can suffer a wide range of symptoms, depending on where the tumour is growing. For example, if the tumour is pressing on nerves or the spinal cord, symptoms could include weakness or paralysis. If it is growing in the bladder or bowel, the child may have problems going to the toilet.
Unfortunately, neuroblastoma is usually advanced by the time it is diagnosed, making it difficult to treat. Although the overall survival rate is about 76%, neuroblastoma actually claims more lives of children under the age of 5 than any other cancer.
There are many different types of neuroblastoma. The worst of these grow very aggressively and have a cure rate of just 50%. These cases are referred to as ‘high-risk’ neuroblastoma.
For more information about neuroblastoma, see Neuroblastoma Australia or the Cancer Australia website.
Please note that, while Children’s Cancer Institute works closely with clinicians, we do NOT treat patients and are unable to provide treatment advice. For questions about an individual child’s cancer treatment, or to find out about relevant clinical trials, please contact your treating oncologist.
“High-risk neuroblastoma has a survival rate of just 1 in 2. This is why it’s so critical that we progress our discoveries into actual treatments for kids as quickly as possible.”
- Professor Michelle Haber, Executive Director, Children’s Cancer Institute
Research into neuroblastoma
At Children’s Cancer Institute, a large part of our research effort is aimed at improving the survival rate of children with high-risk neuroblastoma. We have been researching this disease for many years and have made a number of world first discoveries.
We are attacking neuroblastoma on a number of fronts.
- We are developing new targeted therapies and taking these to clinical trial in children with high-risk and relapsed neuroblastoma.
- We are using tiny particles called nanocells to deliver anti-cancer agents directly to neuroblastoma cells. This is cutting-edge technology that we hope will solve the problem of side effects and late effects caused by chemotherapy.
- We are identifying new targets for therapy – molecules in neuroblastoma cells that can be targeted with new cancer therapies.
- We are investigating the origins of neuroblastoma – how these tumours begin in a child – so we can try to prevent them.
All statistics cited are from the Australian Institute of Health and Welfare.
