Children and young people with cancer who have relapsed can quickly access new treatments, thanks to new study from Cancer Research UK

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Children and young people in the UK with cancers that have come back now have faster than ever access to new personalized treatments thanks to a national tumor biopsy sequencing platform and a clinical trial funded by Cancer Research UK*.

The genetic code of tumors is sequenced through the Stratified Medicine Pediatrics** program, and the molecular information is used to match patients to treatments in the ESMART*** trial, which is testing multiple new targeted drugs, and treatment combinations previously were not available for children and young people.

Patients can be placed on ESMART within weeks of their tumors being sequenced, giving children and youth with cancer with limited treatment options quick access to the targeted drugs that are likely to work for them.

The Stratified Medicine Pediatrics program is led by: The Institute of Cancer Research, London, and the ESMART is a European trial currently being rolled out in the UK. It has been opened in the leading center of the process, The Royal Marsden NHS Foundation Trust in London, and the Royal Manchester Children’s Hospital. Additional branches will open in Birmingham, Newcastle and Great Ormond Street Hospital in the coming months.

Cancer in children and adolescents is rare, and when it happens, more than 80% of diagnosed patients survive their cancer for 5 years or more****. However, this includes more common and, in general, more curable cancers. For more aggressive cancers such as neuroblastomas and cases where cancer spreads or comes back after the first treatment, survival can be much lower and there are fewer treatment options.

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

ESMART’s new clinical trial is now open in the UK and is designed to radically expand the scope of treatments available to children and young people. Currently, the study has 10 treatment arms (plus 5 others pending regulatory approval) that are testing a range of therapies on their own or in combination, including targeted drugs, immunotherapies, radiotherapy and chemotherapy. And when researchers develop promising new drugs, they are quickly rolled into the study*****.

Professor Louis Chesler, who leads the Stratified Medicine Pediatrics program at the Institute of Cancer Research, London, said: “Our Stratified Medicine Pediatrics program goes beyond traditional DNA sequencing of tumors, as the platform also gives us valuable information about specific characteristics of the tumor that can be targeted by precision medicine This was a big step forward and it’s great to now also have access to the latest treatments that we know can work for our patients.

“In conjunction with the ESMART trial, the Stratified Medicine Pediatrics program provides us with an excellent array of treatments and combinations to choose from to give children and young people the best possible treatment options for them. Combined with our ongoing work to improve cancer diagnosis in young people and better understand the cause of treatment resistance and relapse, the future looks brighter for our young patients, which is great news for them and their families.”

dr. Lynley Marshall, UK principal investigator of the ESMART study and Oak Foundation Consultant in Pediatric and Adolescent Oncology Drug Development at the Royal Marsden Hospital, said: “We have spent years trying to get a more targeted approach to childhood cancer, and we are very proud to have helped develop ESMART and have it available in the UK.”

“But more importantly, it brings hope to families who may have experienced multiple relapses. Just knowing there is something else they can try with the real possibility of benefit makes all the difference.”

Michelle Mitchell, chief executive of Cancer Research UK, said: “Cancer in children and young people is different than in adults, and research into these cancers faces its own challenges that can prevent progress. Our research strategy for cancer in children and young people, launched last year is specifically designed to address these unique challenges and drive progress in this area.”

“The Stratified Medicine Pediatrics program and ESMART are wonderful examples of the work we are funding that is revolutionizing the way children and young people with cancer are treated in the UK, and I look forward to seeing the ways they evolve in the future. will develop.”


* The money used for both works was given to Cancer Research UK as a philanthropic donation from Children with Cancer UK. The ESMART study has also recently received additional funding from the Jon Moulton Charity Trust.

** The Stratified Medicine Pediatrics program is the first platform of its kind in the country and now routinely analyzes biopsies of all children with solid tumors (including brain tumors) relapsing in the UK, which was previously not a standard process for most pediatric cancers.

*** The European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (ESMART) was developed in collaboration with the Innovative Therapies for Children with Cancer (ITCC) European Pediatric Early Phase Clinical Trial Consortium.

**** 82% of children survive cancer for five years or more, as shown by the 2006-2010 actuarial survival of children diagnosed with cancer in the UK. Source: National Cancer Intelligence Network. National Register of Progress Reporting of Tumors in Children, 2012. Oxford: NRCT; 2013

According to data from 2001-2005 in the UK, more than 80% of young people with cancer (including all benign/uncertain brain tumors, other CNS tumors and intracranial tumours) survive their disease for at least five years. Source: Five-Year Relative Survival, Ages 15-24, UK, 1991-2005 Data was provided on request by North West Cancer Intelligence Service (NWCIS) in 2013. Similar data can be found here:

***** Details of current ESMART arms and treatments being trialled:



Arm A (completed)

Ribociclib + TOTEM

Arm B (completed)

Ribociclib + Everolimus

Arm C (completed)

Adavosertib + Carboplatin

Arm D (open)

Olaparib + irinotecan

Arm E (closed)

Vistusertib (AZD2014)

Arm F (closed)

Vistusertib (AZD2014) + TOTEM

Arm G (completed)

Nivolumab + Cyclophosphamide+/-radiotherapy

Arm H (cancelled)

Vistusertib + Selumetinib

Arm I (open)


Arm J (open)

Lirilumab + Nivolumab

Arm K (new, in adjustment)

Fadraciclib + Temozolomide

Arm L (new, in preparation)

Fadraciclib + Cytarabine

Arm M (new, in adjustment)

Ribociclib + Everolimus

+/- Dexamethasone

Arm N (new, in line)

Ceralasertib (AZD6738) + Olaparib

Arm O (new, in line)

Futibatinib (TAS-120)

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