New genetic testing advances targeted treatments for childhood cancer in the UK

• New research has looked into a comprehensive and structured approach to genetic testing matching children and young people with cancer to targeted treatments. The study aimed to advance precision medicine in the UK to help young cancer patients receive more targeted cancer treatments, an important step towards increasing survival rates and reducing the side-effects of treatment.
  
  
• Researchers looked at tumours of relapsed childhood cancers to see how they adapt and change in response to treatment.
  
  
• Researchers showed that a ctDNA blood test, a less invasive approach to track tumour evolution, can add valuable information and in some patients detect additional DNA mutations in the tumour that were missed by tumour biopsy.
  
  
• Co-funded by Children with Cancer UK and Cancer Research UK, the first phase of the Stratified Medicine Paediatrics research programme is led by Institute of Cancer Research. The paper is available on ACCR Journal Cancer Discovery.
  
  

The first phase of the Stratified Medicine Paediatrics (SMPaeds1) programme is advancing precision medicine in the UK to help young cancer patients receive more targeted and less toxic treatments.

SMPaeds1 aimed to advance precision medicine for children and young people whose cancer has returned. The project focused on analysing childhood cancer tumours at diagnosis and relapse stages of the cancer journey, to help better understand how the tumours evolve.

The research team from The Institute of Cancer Research, London introduced the use of ctDNA — DNA that is released into the bloodstream circulation by cancer cells — as a potential tool for tracking cancer progression and identifying new treatment targets. The ctDNA test offers a less invasive way to monitor how tumour mutations change over time and can add additional information that is complimentary to tissue biopsy.

The first phase of the ground-breaking precision medicine research project was completed in October 2023 and was led by, Professor Louis Chesler, Professor of Paediatric Cancer Biology at The Institute of Cancer Research, London, and Consultant in Paediatric Oncology at The Royal Marsden NHS Foundation Trust, together with author, Dr Sally George, Group Leader of the Developmental Oncology group at the Institute of Cancer Research, London and an Honorary Consultant Paediatric Oncologist at The Royal Marsden NHS Foundation Trust.

Dr Sally George explains:

We showed that ctDNA analysis can add valuable information and that in some patients it can detect additional DNA mutations that are in the tumour but were missed by tumour biopsy.

SMPaeds1 is the largest study with matched ctDNA and tissue sequencing to date and shows the value of ctDNA testing for children with cancer. We are working with colleagues across Europe to transition ctDNA analysis from being a research test to being available clinically.

The project also identifies DNA mutations that become enriched at relapse. This will help us prioritise future research to understand why those mutations are enriched and if we can develop new treatments to target cancers with those mutations.

The second phase of the research programme, SMPaeds2 – also co-funded by Children with Cancer UK and Cancer Research UK – is currently underway and aims to develop and study new tests that will build upon the success of the first phase of the programme. SMPaeds2 will investigate blood cancers and solid tumours in children and young people, including in the brain, muscle and bone, which can be more difficult to access, diagnose and treat.

Children with Cancer UK CEO, Amar Naher said:

We’re proud to be involved in a research project leading the way in advancing precision medicine in the UK. Children with Cancer UK’s mission is to create a world where every child and young person survives their cancer diagnosis. We strive to meet this mission through funding impactful research. The outcomes from the SMPaeds programmes could help in the development of targeted treatments and could lead to improved, less invasive ways of monitoring and treating childhood cancers. By identifying genetic changes that drive relapse, the project helps advance personalised treatments, supporting the charity’s aims to improve survival rates and quality of life for young cancer patients.

Dr Laura Danielson, children’s and young people’s research lead at Cancer Research UK, said:

We’re excited about this new research from the SMPaeds programme revealing the potential use of less invasive blood tests to better understand solid tumours in children and young people. These data demonstrating that analysing ctDNA could lead to a more complete picture of the tumour and how it is changing over time are incredibly important. This will pave the way for better understanding of what drives relapse or lack of response to treatment, and with it the hope of developing better, more targeted treatments. We’re proud to fund innovative research like this, which is unlocking new ways to study and potentially treat childhood cancers to give young cancer patients the best possible chance to live long, healthy lives.

Co-funded by Children with Cancer UK and Cancer Research UK, the SMPaeds1 paper can be read online.

Notes to Editors:

About Children with Cancer UK:
Children with Cancer UK is the leading children’s cancer charity in the UK. Our vision is a world where every child and young person survives their cancer diagnosis. Our mission is to improve survival rates and the quality of survival in young patients, and to find ways to prevent cancer in the future. We are dedicated to raising and investing funds into vital research that helps us understand childhood cancers and improves treatments. We support families as they navigate treatment and life beyond cancer. What began as a small memorial charity in 1988, has evolved into a leading childhood cancer charity raising more than £300 million and investing in over 300 pioneering research projects.

About Cancer Research UK for Children & Young People:

Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research, influence and information.

Cancer Research UK for Children & Young People is the part of Cancer Research UK dedicated to driving progress for 0-24-year-olds with cancer.

Cancer Research UK is the biggest charitable funder of research into children’s and young people’s cancers in the UK.

Together with its partners and supporters, Cancer Research UK for Children & Young People is driving forward progress to ensure more children and young people can live longer, better lives, through more effective and less toxic treatments.

In the last 50 years, Cancer Research UK has helped double children’s cancer survival in the UK. Today, more than 8 in 10 children and young people (aged 0-24) diagnosed with cancer in the UK now survive for at least ten years.

Money raised through Cancer Research UK for Children & Young People will be used to fund research into cancers affecting children and young people (aged 0-24).

For more information, please visit www.cruk.org/children-and-young-people or follow us on Facebook.

About The Institute of Cancer Research, London:
The Institute of Cancer Research, London, is one of the world’s most influential cancer research organisations.

Scientists and clinicians at The Institute of Cancer Research (ICR) are working every day to make a real impact on cancer patients’ lives. Through its unique partnership with The Royal Marsden NHS Foundation Trust and ‘bench-to-bedside’ approach, the ICR is able to create and deliver results in a way that other institutions cannot. Together the two organisations are rated in the top four centres for cancer research and treatment globally.

The ICR has an outstanding record of achievement dating back more than 100 years. It provided the first convincing evidence that DNA damage is the basic cause of cancer, laying the foundation for the now universally accepted idea that cancer is a genetic disease. Today it is a world leader at identifying cancer-related genes and discovering new targeted drugs for personalised cancer treatment.

The ICR is a charity and relies on support from partner organisations, funders and the general public. A member institution of the University of London, it is one of the UK’s leading higher education institutions, placing first for biological sciences and second overall in the definitive ‘REF2021’ rankings of UK university research quality, impact and environment, and provides postgraduate higher education of international distinction.

The ICR’s mission is to make the discoveries that defeat cancer.

For more information visit ICR.ac.uk

Press Enquiries:

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