The Royal Marsden

Targeted drug approved for young patients

A new class of cancer drug that was trialled in the Oak Paediatric and Adolescent Drug Development Unit at The Royal Marsden has been approved for use in Europe.
Two-year-old Charlotte Stevenson received larotrectinib as part of the SCOUT trial

Two-year-old Charlotte Stevenson received larotrectinib as part of the SCOUT trial

The Royal Marsden was the only UK centre to trial larotrectinib, a ‘tumour-agnostic’ drug that targets a specific genetic abnormality called an NTRK gene fusion. Dr Julia Chisholm, Consultant in Paediatric and Adolescent Oncology at The Royal Marsden, is the Principal Investigator for the ongoing SCOUT study, which tests the safety and efficacy of the drug for the treatment of tumours with NTRK gene fusion in children.

She said: “The beauty of this drug is that it targets the abnormality in the tumour, and it is a step towards treating cancers based on genetics rather than site of origin in the body.”

NTRK gene fusions can be found in sarcomas and some brain, kidney, thyroid and other cancers. Two-year-old Charlotte Stevenson was diagnosed with infantile fibrosarcoma at 14 weeks old at her local hospital in Belfast. After a year of standard treatment, she was recruited onto the trial at The Royal Marsden.

Her mum, Esther, said: “When we were told about the drug trial, it seemed very daunting, but we knew that our options were limited. We decided to give it a try and are so glad that we did.

“We have been able to watch Charlotte develop at a rapid rate, making up for lost time and amazing us all with her energy and enthusiasm for life. She can now live a relatively normal life and, best of all, the drug has had an incredible impact on the tumour.”

The Oak Paediatric and Adolescent Drug Development Unit is funded by The Royal Marsden Cancer Charity.

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.