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Royal Marsden leads as first UK site for new paediatric clinical trial following ground-breaking research

A new drug called Fadraciclib will be available to eligible young patients with aggressive neuroblastoma.

06 October 2020

Dr Lynley Marshall, Oak Foundation Consultant in Paediatric and Adolescent Oncology Drug Development at The Royal Marsden, and UK ESMART study lead investigator

A new drug that has passed safety tests in adults will offer new hope to children with aggressive neuroblastoma, according to ground-breaking laboratory research carried out by our academic partner The Institute of Cancer Research.

It is believed a new targeted drug called fadraciclib could be a less toxic treatment than existing therapies and a game changer for aggressive forms of neuroblastoma. The findings, recently published in The Journal of Clinical Investigation, explain the drug targets a gene called N-Myc which occurs in aggressive forms of neuroblastoma.

Professor Louis Chesler and his team at The ICR worked with international collaborators at the Baylor College of Medicine, Houston and found that fadraciclib is effective at blocking N-Myc activity while minimising damage to healthy cells. Up until now the structure of the N-Myc protein makes targeting it extremely challenging, and efforts to date have been unsuccessful.

Fadraciclib will be available to eligible young patients recruited to the international ESMART clinical trial, led in the UK at The Royal Marsden’s Oak Centre for Children and Young People. The drug is expected to be available to patients entering the trial by the end of the year.

“This drug discovery is a perfect example of the importance of our collaboration with the ICR and the speed we can get new treatments from labs into the clinical setting. Fadraciclib will be available in two new combination arms of the ESMART trial.

Dr Lynley Marshall, Oak Foundation Consultant in Paediatric and Adolescent Oncology Drug Development at The Royal Marsden, and UK ESMART study lead investigator

The European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumours (ESMART) trial 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. Funded in the UK by Cancer Research UK, trial management is by the Children’s Cancer Trials Team at the University of Birmingham Cancer Research Clinical Trials Unit (CR-CTU). It started in France in the summer of 2017, with the first UK arm opening at The Royal Marsden in December 2019. Following opening The Royal Marsden, the ESMART trial is now open at the Royal Manchester Children’s Hospital and Birmingham Children’s Hospital with further sites due to open in Newcastle and Great Ormond Street Hospital over the next few months.

Supported by The Royal Marsden Cancer Charity

The Royal Marsden Cancer Charity raises money solely to support The Royal Marsden, a world-leading cancer centre.  It ensures Royal Marsden nurses, doctors and research teams can provide the very best care and develop life-saving treatments, which are used across the UK and around the world. From funding state-of-the-art equipment and ground-breaking research, to creating the very best patient environments, The Royal Marsden Cancer Charity will never stop looking for ways to improve the lives of people affected by cancer.