Neuroblastoma: Our battle against this rare but deadly childhood cancer
May 2, 2014
“While it’s tough knowing there are children out there suffering with the disease, staying positive isn’t that hard. The courage of children, and their parents, is an incredible inspiration.” - Dr Daniel Carter
Neuroblastoma accounts for 15% of all paediatric cancer deaths in Australia and 50% of these children won’t survive. Children’s Cancer Institute is determined to put an end to this devastating disease.
Neuroblastoma can have devastating consequences for the patients and families such as four-year-old Lulu, who was diagnosed with neuroblastoma at just eight months old and has spent the majority of her short life in hospital.
However, Children’s Cancer Institute researcher, Dr Daniel Carter, is helping pave the way to a better future for children like Lulu. His project goal is to uncover information that can be used to design a new treatment for neuroblastoma, the most common solid cancer found in infants.
Dr Carter aims to uncover the early changes in cells that lead to neuroblastoma by examining two particular metabolites – glutamine and glutathione — that are present in very high levels at the start of neuroblastoma.
Although Lulu continues to fight her battle with amazing bravery, determination and resilience, she has not responded to conventional treatment. This means her mum, Josi, is playing an agonising waiting game and living day to day with no idea of the next steps in Lulu’s journey.
It also means that it is critical that we continue our research into not only beating this disease for good, but also ensuring the best quality of life for the patient once treatment is over.
“While it’s tough knowing there are children out there suffering with the disease, staying
positive isn’t that hard,” says Dr Carter. “The courage of children, and their parents, is an incredible inspiration.”
A new treatment approach for neuroblastoma
Our research has shown that a drug called DFMO, previously used for curing sleeping illness, can help combat neuroblastoma.
When used in combination with modern chemotherapy, DFMO slows tumour progression much more effectively than chemotherapy drugs alone.
One of our own researchers, who is also a paediatric oncologist at Sydney Children’s Hospital, is leading a major new international clinical trial based on this research – which if successful, could one day change neuroblastoma treatment forever.
This trial could have huge implications for children like Nathan, who was diagnosed with stage 4 neuroblastoma and passed away in his parents’ arms at the age of just two-and-a-half.
Read Nathan’s story.
About Children’s Cancer Institute
Originally founded by two fathers of children with cancer in 1976, Children’s Cancer Institute is the only independent medical research institute in Australia wholly dedicated to research into the causes, prevention and cure of childhood cancer. Forty years on, our vision remains unchanged – to save the lives of all children with cancer and to eliminate their suffering. The Institute has grown to now employ more than 220 researchers, operational staff and students, and has established a national and international reputation for scientific excellence.
Our focus is on translational research, and we have an integrated team of laboratory researchers and clinician scientists who work together in partnership to discover new treatments which can be progressed from the lab bench to the beds of children on wards in our hospitals as quickly as possible. These new treatments are specifically targeting childhood cancers, so we can develop safer and more effective drugs and drug combinations that will minimise side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life.
We are currently leading the establishment of the Zero Childhood Cancer national child cancer personalised medicine program for children with the most aggressive cancers, in partnership with the Sydney Children’s Hospitals Network. This program will revolutionise the way treatment decisions are made, with the aim of improving survivorship for those children at highest risk of treatment failure from their disease.