Royal Marsden leads as first UK site for new paediatric clinical trial, offering new hope for children and young people with cancer who have relapsed

Children and young people in the UK with cancers that have come back can now get rapid access to new personalised treatments quicker than ever before thanks to a national tumour biopsy sequencing platform and clinical trial.

The Royal Marsden is the first hospital in the UK to open as an ESMART centre, working with The Institute of Cancer Research (ICR) to test the benefits of not one but multiple targeted drugs and treatment combinations not previously available for children and young people.

Patients may be placed on ESMART within just a few weeks of having their tumours sequenced through the Stratified Medicine Paediatrics (SMPaeds) programme, and the molecular information is used to match patients to treatments on the ESMART trial. SMPaeds, which is led by The Royal Marsden’s academic partner the ICR, is the first platform of its kind in the country, and now routinely analyses biopsies from all children with solid tumours who relapse in the UK.

While survival rates for the more common and, generally, more curable children’s cancers is high, this is sadly not the case for rarer, more aggressive cancers such as neuroblastomas, and cases where cancer spreads or comes back after initial treatment. For these children, survival rates can be much lower and treatment options very limited.

For the last two years in the UK, when a young person’s cancer comes back, their tumour is routinely sequenced through SMPaeds. The findings are discussed by an expert panel of clinicians and scientists who recommend potential treatment options based on the specific gene faults identified in the tumour. But treatments have been very limited, and clinical trials often only test combinations of existing chemotherapy drugs, radiotherapy or single molecularly targeted drugs.


The knowledge that we're building up about paediatric cancers and targets and helping to drive future treatments and future trials is really exciting and will really help patients.

Dr Lynley Marshall, Head of the Oak Paediatric and Adolescent Drug Development Unit

The new ESMART clinical trial is now open in the UK, funded by Cancer Research UK, and has been developed to radically widen the scope of treatments available for children and young people. Currently, the trial has 10 treatment arms (plus 5 more that are waiting regulatory approval) that are testing an array of therapies on their own or in combination, including targeted drugs, immunotherapies, radiotherapy and chemotherapy. And as researchers develop promising new drugs, they are quickly rolled into the study.

Dr Lynley Marshall, Oak Foundation Consultant in Paediatric and Adolescent Oncology Drug Development at The Royal Marsden  is chief investigator and UK lead of the ESMART trial. She said:

“We’ve spent years trying to get a more targeted approach to children’s cancers in place, and we’re really proud to have helped develop ESMART and to have it available in the UK. The knowledge that we're building up about paediatric cancers and targets and helping to drive future treatments and future trials is really exciting and will really help patients.

“More importantly, it brings hope for families that may have lived through multiple relapses. The fact of just knowing that there’s something else that they can try with the real possibility of benefit makes all the difference.”

Dr Lynley Marshall is Head of the Oak Paediatric and Adolescent Drug Development Unit which is generously funded by Oak Foundation and other supporters of The Royal Marsden Cancer Charity.

The European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumours (ESMART) has been designed collaboratively within the Innovative Therapies for Children with Cancer (ITCC) European paediatric early phase clinical trial consortium in which UK centres and investigators play key roles. It started in France in the summer of 2017, with the first UK arm opening at The Royal Marsden in December 2019, led by Dr Lynley Marshall.

Following opening The Royal Marsden, the ESMART trial is now open at the Royal Manchester Children’s Hospital with further sites due to open in Birmingham, Newcastle and Great Ormond Street Hospital over the next few months.

Supported by The Royal Marsden Cancer Charity 

Supporters of The Royal Marsden Cancer Charity have enabled the Charity to help improve the patient experience for children and their families. The Charity contributed £16 million to the cost of the Oak Centre for Children and Young People when it was built and have since funded the creation of break-out spaces such as the Pandora parents’ lounge and outdoor play areas.

The Charity continues to fund a number of different services within the centre including a team of play specialists who use their understanding of child development, therapeutic play activities and distraction techniques to help young patients prepare for and cope with painful or invasive procedures, scans and treatment and to ensure the service runs smoothly. The Charity also supports the pain management team, who work closely with the physiotherapists and psychologists to help children and young people improve their pain management and find ways of coping.