Meeting the complex needs of young patients

The ICR and The Royal Marsden are leaders in the research and development of new drugs to tackle childhood cancers

1,800

children aged 14 or under are diagnosed with cancer each year

In the UK, about 1,800 children aged 14 or under are diagnosed with cancer each year. The partnership between the ICR and The Royal Marsden is therefore crucial, as it increases young patients’ access to the latest treatments and clinical trials for particularly hard-to-treat paediatric cancers. Our researchers, with the support of the BRC and Oak Foundation, are also UK leaders in developing personalised treatment strategies, helping to improve survival rates and reduce side effects from treatment for children and young people. Ultimately, we want to empower families by providing patients with the widest possible range of options that can make all the difference.

The BRC increases young patients’ access to the latest treatments and clinical trials

Personalised treatments

Professor Louis Chesler leads the Paediatric Solid Tumour Biology and Therapeutics Team in the ICR’s Clinical Studies Division, and is an Honorary Consultant Medical Oncologist at The Royal Marsden. He leads an ambitious precision-medicine programme to match each child diagnosed with cancer in the UK to clinical trials of targeted cancer medicines.

This is guided by individualised genomic testing, which Professor Chesler developed together with Dr Mike Hubank, Joint Head of Clinical Genomics in the NIHR Centre for Molecular Pathology at The Royal Marsden and the ICR.

The number of Phase I and II paediatric drug trials conducted at The Royal Marsden increased from seven in 2009 to 23 in 2016

Thanks to funding from the BRC, the ICR and the UK charity Christopher’s Smile, Professor Chesler and his team are trialling this approach in several hundred children with cancer, through innovative clinical trials conducted at The Royal Marsden’s Oak Centre for Children and Young People. The idea is to use next-generation sequencing technologies on blood and tumour tissues to rapidly obtain a genetic profile of the key changes occurring in each patient’s tumour.

In 2016, a national pilot began in 21 hospitals in the Children’s Cancer and Leukaemia Group network, and approximately 275 children have been tested for molecular changes.

A much larger national trial of multi-platform genomic testing will begin in 2018 and run for three years through the Cancer Research UK Experimental Clinical Medicines Centre Network, funded by Cancer Research UK and Children with Cancer UK.

Professor Louis Chesler

Innovative trials

As well as developing tests that can guide precise treatment, Professor Chesler also works with the Oak Paediatric and Adolescent Oncology Drug Development Team at The Royal Marsden. Dr Lynley Marshall leads this team, which has been generously supported by Oak Foundation for more than 15 years. Together, Professor Chesler and Dr Marshall conduct innovative clinical trials of drugs discovered by the ICR, and of drugs initially developed for adult cancers, to help find new treatments for hard-to-treat childhood cancers.

Increasing the number of clinical trials available is key to the team’s ambition to offer children the same opportunities for personalised cancer treatment as adults have. Combined with advanced molecular testing, targeted molecular medicines maximise the chance of successful treatment and avoid the use of more toxic chemotherapies.

The European ITCC-eSMART trial is the first paediatric trial of this type, using a multi-armed ‘basket trial’ approach, with a patient’s treatment based on molecular testing. One of the drugs being tested for the first time in children as part of this trial is vistusertib, identified by Professor Chesler’s laboratory as a drug that targets the MYCN gene, which drives poor outcome in several children’s cancers. The trial is being led in the UK by Dr Marshall.

"I hope that through this initiative, we can drive forward the best possible use of targeted drugs in children"

Professor Chesler is also the UK lead of the USA-EU trial of lorlatinib, which targets ALK – another important gene that is altered in childhood cancers.

“Children deserve the very best treatments for cancer, but for too long there have been delays in applying molecular techniques and biomarker testing to clinical trials, to personalise their treatment and maximise the chance that these drugs can work,” he says.

“We face major barriers in obtaining targeted cancer drugs for children and using them in advanced clinical trials. But I hope that through this initiative, we can drive forward the best possible use of targeted drugs in children and make the case very clearly that they should be more widely available, as they have been for adults.”

Increasing access to treatments

Through comprehensive clinical and academic collaboration between the ICR and The Royal Marsden in paediatric cancer research, more children treated at the hospital are enrolled in Phase I and II clinical trials than at any other centre in the UK. Many also participate in larger international Phase III trials that compare new treatments with existing standard options.

“We have a highly committed team who go to great lengths to provide the best possible clinical care, while offering the most promising new treatment options for our young patients,” says Dr Marshall.

“Our centre has contributed to the development of new drugs in increasingly sophisticated studies, with real impacts on the lives of our patients and their families. “One-third of our patients now come from outside our catchment area, which is mainly due to the fact that we offer more new drug trials than any other centre, while offering high-quality clinical care, hope and support to families.

“It’s incredibly rewarding when a patient has a good response to a new drug. Like in adult drug development, we can have patients who move from trial to trial with an excellent quality of life over long periods of time.”