“In two days, we went from having a child that we thought was just slightly off to one that was going to die with 100 per cent certainty.” – Amanda Walker
Amanda Walker’s daughter, Abbie, had been diagnosed with an inoperable and incurable cancer known as DIPG. The extremely aggressive cancer took Abbie just five months after her diagnosis. She was six years old.
When you hear Abbie’s story, and that of so many others, the urgent need to improve treatment for children with cancer could not be clearer. Researchers here at The Institute of Cancer Research, London, and elsewhere, are trying to better understand the enormous complexity that is specific to childhood cancer. That science is central to getting new and better treatments.
And much of that progress towards new treatments is being driven by parents determined to ensure no other child or family has to go through what they’ve been through.
Tailoring treatment using a genetic test
One of the most inspiring advances from last year was the development of a genetic test for children with cancer, with funding from the parent-led charity Christopher’s Smile.
The test, developed by researchers at the ICR and The Royal Marsden NHS Foundation Trust, works by sequencing 81 different cancer genes. This allows doctors to understand specific mutations in each child’s tumour and personalise treatment accordingly.
Christopher’s Smile was set up in 2008 after Kevin and Karen Capel’s son died aged five from a brain tumour called medulloblastoma. Karen and Kevin explain why the test is so important to them: “When our son died there was no biological information available to clinicians about individual children’s tumours. The test is a first for children.”
And since the development of the test, the ICR has received additional funding from Children with Cancer UK to fund the initial phase of a programme for rapid DNA sequencing of every UK child diagnosed with a solid tumour.
We are working to better understand the complexities specific to childhood cancers. This knowledge is enabling us to improve treatments.
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Better treatment for neuroblastoma
In June a major European trial showed that two ICR-discovered drugs – busulfan and melphalan – are more effective in treating children with neuroblastoma than the current standard treatment.
The trial was a major collaborative effort across Europe and involved nearly 130 centres in 18 different countries – making it the largest-ever trial in children with high-risk neuroblastoma. The disease is one of the deadliest childhood cancers.
The research was led by the SIOPEN group of neuroblastoma researchers, which included the now-retired ICR Professor, Andrew Pearson, who devised the trial.
Enormous collaborations of this kind are vital in childhood cancer research due to the rarity of the conditions. Professor Pearson stressed the importance of such research: “This practice-changing clinical trial has only been possible because of a huge and co-ordinated effort across Europe, led by researchers, clinicians and families who are determined to find more effective treatments for children with neuroblastoma.”
Helping families affected by Wilms tumour
In May this year scientists from the ICR worked with colleagues in the Netherlands at the Hubrecht Institute-KNW to identify a new genetic cause of the childhood kidney cancer Wilms tumour.
Wilms tumour affects about 1 in 10,000 children, but fortunately is curable in about 90 per cent of cases. The researchers hope the study will help parents whose child has been diagnosed with the condition.
Professor Nazneen Rahman, who led the research at the ICR, said: “This study has been of immediate use to families in providing a reason for why their child developed cancer, and information about risks to other children, which is very rewarding.”
Closing a loophole in childhood cancer regulations
While making progress in the labs and in trials is important, it’s also vital that children can access any new treatments in clinical trials and that’s only possible if the regulations surrounding them work effectively.
Unfortunately, not enough cancer medicines are being trialled in children or licensed for paediatric use. And we believe a major cause is the European regulations governing children’s medicines – they allow drug companies to opt out of running clinical trials of new cancer drugs in children, even where there is good evidence that the drugs could be beneficial.
Alongside many parent-led charities, The Royal Marsden, and UK cancer charities, we have been campaigning to close this loophole in the 2006 Paediatric Regulation. Our analysis of European Medicines Agency data showed that, between 2012 and 2016, pharmaceutical companies were granted waivers so they did not have to trial cancer drugs in children for 33 of 53 cancer treatments (62 per cent), which were ultimately approved.
The European Commission has run a consultation on the Paediatric Regulation and hopefully concerns raised by the ICR and others will be addressed so more new cancer treatments are trialled in children.
Abbie’s Army, set up by Abbie’s mum Amanda and her dad Ray, is one of the parent-led charities supporting our call to the European Commission, as well as funding our research.
Amanda said: “It’s heart-breaking to know that there might be treatments out there for children with rare cancers, like Abbie’s, but that they aren’t being trialled because of a loophole. The current system is failing children with cancer. On behalf of all the families experiencing what we did, we ask the European officials to act on these recommendations.”
Families fighting for children’s futures
Driven by tragedy, parent-led charities, like Abbie’s Army, are improving the chances for children diagnosed with cancer in the future. And fortunately we’ve come a long way since the 1960s when only about 3 in 10 children with cancer were treated successfully. Now 8 in 10 children diagnosed with cancer live for at least five years and most of these children will be cured.
This month is an opportunity to appreciate all of the work that the ICR and other researchers have contributed to improve the chances for children with cancer but it is also a chance to reflect on the extraordinary people who make this research possible. Our family charity partners have a huge impact on the childhood cancer research conducted at the ICR and we’re enormously grateful for their support.
And while parents like Amanda, Ray and so many others, are still losing their children to cancer, we will continue our efforts to defeat it.
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