Neuroblastoma is a major focus for research led by our childhood cancer experts here at the ICR. A devastating cancer which often affects very young children, it’s due for a wave of publicity over the coming months as a new storyline in Coronation Street will see character Fiz Brown’s daughter Hope diagnosed with the disease. This spotlight on neuroblastoma will be welcomed by our researchers.
As well as leading scientific programmes and major clinical trials in neuroblastoma, for around 18 months we have been calling for changes to current EU rules that will make it easier for children with cancer – including some with more serious forms of neuroblastoma – to gain access to drugs through clinical trials.
What is neuroblastoma?
Neuroblastoma is a solid tumour of nervous tissue which usually starts in the abdomen, and can grow to a large size and spread widely before it’s spotted. Around 100 children each year are diagnosed in the UK.
Most children with low or intermediate risk forms of neuroblastoma respond well to treatment – and tantalisingly, very young children can sometimes cure themselves of it without medical help. But about 45% of children have high-risk forms driven by particular genetic mutations, which is very difficult to cure and requires very intensive treatment.
What research are we doing?
Much of our neuroblastoma research, often carried out in close partnership with our hospital colleagues at The Royal Marsden, is aimed at creating new and more effective modern treatment options for children who fail to respond to treatment. For example, our researchers are looking for new cancer-driving genes and developing new ways to offer drugs to the children who might benefit, based on genetic profiling.
One example of how this ‘personalised’ approach to treatment can work is Sophie, whose story was featured on a major BBC Panorama documentary about research at the ICR and The Royal Marsden, ‘Can You Cure My Cancer?’, earlier this year. Sophie didn’t have neuroblastoma, but benefited from a programme of research in neuroblastoma to explore the potential of new drugs that target a gene called ALK – often mutated in high-risk neuroblastoma. Sophie had an ALK mutation, spotted through genetic sequencing, and was able to receive an ALK-targeted drug as a result.
How can changes in regulation help?
Although there are some exciting research advances in children’s cancer, current EU rules are preventing some children from accessing genetically targeted drugs that could help them, including drugs that could treat certain forms of neuroblastoma. Pharmaceutical companies that make these drugs are currently able to seek an exemption from trialling them in children by classifying them as ‘adult’ drugs – even if the science shows they could work in childhood cancers too.
Last year we launched a new policy on this issue, leading to widespread coverage in the media. Since then, it’s remained on the news agenda and recently we wrote a letter to the Daily Telegraph – co-signed by more than 10 other charities – calling for change. We’ve been working hard to raise awareness of the issue with politicians too, who have tabled questions to the national and European Parliaments to ask for change.
It’s good news that Coronation Street will raise awareness of neuroblastoma. Childhood cancers do not always have the highest profile in the news, and we hope that Fiz’s and Hope’s story will help lead to more widespread understanding about the effect this disease can have on families.
Even better, it could shed light on the passion and dedication of scientists working to find new treatments for neuroblastoma – and perhaps draw attention to the need for a change in the regulations, to make it easier to do this kind of live-saving research.
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