Trial testing innovative cancer treatment on children and young people launches

A first-of-its-kind trial has been launched to explore if a personalised cancer treatment that uses the body’s own immune system to attack the disease is effective in children and young people.

Up to 60 patients with difficult-to-treat solid tumours will be recruited in the UK and US for the study, known as Mighty.

They will be treated with Car T-cell immunotherapy, which engineers a patient’s own immune cells to recognise and fight cancer.

Mighty is being led by scientists on Cancer Grand Challenge’s NextGen team, with the UK arm headed by experts at University College London (UCL).

The first patient, who is in their 20s, has been enrolled on to the study.

The trial will include patients with rhabdomyosarcoma, a soft tissue cancer that develops in voluntary muscles and is most common in children under 10, Ewing sarcoma, which affects bones or soft tissue in teenagers and young adults, and soft tissue sarcoma, which primarily affects teenage boys and young men.

All three cancers are rare and aggressive.

Dr Karin Straathof, associate professor in tumour immuno-oncology at UCL, member of the NexTGen team and lead investigator for the Mighty trial, said: “Cancers in children and young people are fundamentally different from those in adults.

“They are unique in how they develop, how they resist treatment, and where their vulnerabilities lie.

“So, the treatments should be different too.

“While current chemotherapy-based treatments work well for some patients, in other patients the tumour does not respond or comes back.

“We urgently need new treatments that kill cancer cells without harming healthy ones – T-cell immunotherapy has the potential to do just that – not only improve survival but also protect their long-term quality of life.

“We’re looking forward to seeing the results of this important study.”

Patients in the UK will be recruited from University College London Hospital (ULCH) and Great Ormond Street Hospital (Gosh), while US patients will be recruited from the Children’s National Hospital in Washington and the Dana-Faber Cancer Institute in Boston.

Cancer Grand Challenges is a global initiative founded by Cancer Research UK and the National Cancer Institute in the US.

The trial is also being co-funded by The Mark Foundation for Cancer Research.

Ryan Schoenfeld, chief executive at The Mark Foundation for Cancer Research, said: “For far too many children living with these types of cancer, current treatments are not good enough.

“This trial offers a real sense of hope.

“By harnessing the power of Car T-cell therapy in innovative ways, the NexTGen team is pushing the boundaries of what’s possible for childhood cancer treatment.”

Dr David Scott, director of Cancer Grand Challenges, added: “We launched Cancer Grand Challenges to find solutions to the toughest challenges in cancer, and we recognised the urgent need to develop more effective treatments for these children.

“The launch of this clinical trial represents collaboration between the best scientists from around the world, who we have brought together to develop this cutting-edge innovation.”