Why is this research important?
Around 100 children are diagnosed with neuroblastoma each year in the UK. Most children with low-risk neuroblastoma will be cured, but the outlook is less certain for children with high-risk neuroblastoma.
These children need extremely intensive treatment that can last for years and despite the intensity of treatment, only 40 to 50 per cent survive for five or more years.
Research into more effective treatments for children with high-risk neuroblastoma is urgently needed.
The research team at UCL Great Ormond Street Institute of Child Health, led by Professor Anderson, aim to develop and refine a new form of targeted immunotherapy for children with neuroblastoma. It would have fewer side-effects and provide lifelong protection against this disease, saving more children’s lives.
“Our ultimate aim is to design a safe new treatment that not only destroys a child’s cancer but also prevents it returning, providing a cure for life,” explains Professor Anderson.
Immunotherapy uses substances made by the body or in a laboratory to strengthen the body's own immune system to fight cancer. It represents the most promising new cancer treatment approach since the development of the first chemotherapies in the late 1940s.
Professor Anderson and his team have been working on a type of immunotherapy called cellular or adoptive immunotherapy. The treatment involves collecting the patient's own immune cells (T cells) and genetically modifying them in the laboratory before being injected back into the patient.
The modified T cells, known as CAR (chimeric antigen receptor) T cells, are programmed to find and kill neuroblastoma cells which have a specific molecule, GD2, on their cell surface.
Progress to date
The researchers have already run a previous clinical trial in children with relapsed neuroblastoma but there were concerns that targeting the GD2 molecule might cause long term side-effects as it can also be found on some healthy cells.
With Action’s funding, the researchers are refining ways of producing and delivering CAR T cells without having unwanted effects on healthy cells. This could give children more chance of a cure. If successful, this new technique will be tested in a follow up clinical trial.