How a decade of research progress offers hope to children with neuroblastoma – The Institute of Cancer Research

Posted: September 22, 2020 at 2:04 pm

Image: Kevin and Karen Capel with their son Christopher

In 2006, Karen and Kevin Capels son, Christopher, was diagnosed with medulloblastoma a type of brain tumour accounting for 15-20 per cent of all childhood brain cancers. Two years later, Christopher passed away, nine days before his sixth birthday.

Kevin and Karens devastating loss is sadly a tragedy endured by countless families. With the aim of changing the outlook for children with cancer and opening doors to potential new treatments, Karen and Kevin Capel set up the charity Christophers Smileto help fund research into childhood cancers like medulloblastoma.

Since their charity was established, Christophers Smile has raised more than 1 million for our childhood cancer research, including funding for five of our research scientists here at The Institute of Cancer Research. Their efforts and generosity have been vital in helping identify and develop kinder, smarter treatments for children with cancer.

One of the toughest things for Karen and Kevin was to see their little boy struggle with the many side effects that go along with surgery and chemotherapy post-surgery mutism, sickness, hair loss, infections, ulcers, extreme fatigue, loss of appetite, loss of hearing.

Side effects of treatments like chemo and radiotherapy are particularly bad for children as they are still growing and their organs are more susceptible to damage. Having witnessed their sons tough treatment journey, Karen and Kevin decided that sparing children from devastating side effects was something they should prioritise in their funding efforts.

One of the teams at the ICR that they have generously supported is that of Professor Louis Chesler. Professor Cheslers Paediatric Solid Tumour Biology and Therapeutics Teamworks to understand the underlying biology of childrens cancers. The team is striving to make the biggest difference to children with cancer by developing new personalised therapies that are not only more effective but also kinder to children.

The teams research focuses on the two most common solid childhood tumours neuroblastoma, a nerve tissue cancer and medulloblastoma, the type of brain tumour that Christopher suffered from.

Image: Professor Louis Chesler in the lab

Over the last decade, Professor Cheslers team has been striving to make personalised medicine a reality for children. In this respect, one of their greatest milestones to date is his work, with colleagues at The Royal Marsden NHS Foundation Trust, to develop and implement a test that can analyse many cancer genes in childrens tumours simultaneously and rapidly.

The test has demonstrated how powerful genetic testing can be for younger patients, not just adults as it can spot specific gene alterations and help doctors pick out the best targeted drugs for children with cancer based on this genetic information.

Around 400 children at 21 leading hospitals across the UK have now started receiving the test, and NHS has incorporated it into standard practice. Commenting on the development, Karen Capel said:

Our proudest moment working with the ICR has been the introduction of a genetic sequencing panel specifically for children into NHS England test directory. The panel was developed by the ICR, funded by Christophers Smile, and the result means that children with solid tumours may now be sequenced at diagnosis and relapse across England.

The team also showed that around half of children with solid tumours meaning lumps of abnormal cells found in muscles, bones or organs, but not in the blood could benefit from targeted drugs that are already available for adults. However, their study showed that only seven per cent of kids were able to access these drugs.

Professor Cheslers work has therefore exposed the limitations and frustrating barriers that children face when it comes to receiving innovative, targeted treatments. For this reason, Kevin and Karen have been highly active in campaigning for children to have better access to cancer drugs.

The aim of Professor Cheslers team is to treat more children whose tumours have these targetable genetic defects with better drugs. The test will help them achieve their goal but, at present, they are also working on gathering the necessary data to guide the use of the most appropriate drug for each child.

It is crucial to repurpose existing targeted drugs used in adults so that children can benefit from them too but developing drugs specifically targeted at childrens cancers is also a priority for Professor Chesler and his team.

By uncovering various molecular mechanisms and interactions involved in childhood cancers and providing new ways to study the disease, the team has shed light on the drivers and mechanisms of tumour formation and progression. Thanks to this, the team is leading the way in the field of childhood cancer. Their ultimate aim is to extend the lives of children with cancer by being able to target genes that sustain the growth of childhood tumours.

Commenting on the research at the ICR made possible by Christophers Smile over the past years, Kevin Capel said:

Research is key to getting new and better treatments. When our son was diagnosed, there was no biological information available to doctors about individual childrens tumours. Our funding contributions have helped change that, as researchers at the ICR have already made discoveries that have improved our understanding of various childhood cancers.

Back in 2015, Professor Chesler discovered ways to target the unique genetic paths that medulloblastoma follows when relapse occurs and a childs disease comes back. The team showed that certain genetic changes were responsible for the cancer becoming more aggressive and identified drugs that could help to target relapse.

Since then, Professor Chesler and his team have identified new gene targets and developed animal models to better study and understand various childhood cancers, as well as to help them test new drugs before they are ready to be trialled in patients.

The hope has always been that their research will produce models and data that will form the basis of new clinical trials for children and this is already happening. For example, based on their promising findings throughout the last decade, a new drug known as fadraciclib is now ready to enter clinical trials in children with the childhood cancer neuroblastoma.

Image: Karen and Kevin Capel

While we have seen lots of progress over the last decade, survival of children with medulloblastoma, neuroblastoma and rhabdomyosarcoma especially after the disease relapses and comes back remains extremely low, and there is still a pressing need for greater understanding and new therapies.

Christophers Smile are committed to continue their work to increase the survival of children diagnosed with cancer by supporting our life-saving research. The charity also campaigns to improve access to new targeted drugs for children.

Reflecting on the achievements of the past ten years, Professor Chesler said:

I couldnt be prouder of the work we have carried out, which would have not been possible without the Capels and their charity, Christophers Smile.

I have had the pleasure of working with them for over a decade and their funding has been instrumental in bringing new developments and treatments closer to children and their families. I am extremely grateful for their unwavering dedication and Im inspired by how theyve been able to take their grief and turn it into something capable of changing childrens lives.

We have delivered really exciting discoveries and started new and fruitful developments in these past years. Im excited about how far weve come - but there is still a long way to go and we continue to work so that, hopefully one day, we can defeat childrens cancer.

More here:
How a decade of research progress offers hope to children with neuroblastoma - The Institute of Cancer Research

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