Translating CAR T-cells for tumours

Tomorrow

Despite promising technologies like proton beam, they won’t work for all.

Brain tumours are one of the most common causes of cancer death in children. One particularly devastating brain tumour is diffuse intrinsic pontine glioma (DIPG) – a brainstem cancer diagnosed in around 30 children each year in the UK. Because it grows diffusely within vital brain tissue it’s usually impossible to treat, even with techniques like proton beam.

Despite significant research into better treatments, the average survival for a child after diagnosis with DIPG is just nine to 15 months. Dr Karin Straathof is developing a pioneering yet familiar new treatment for children with lifethreatening tumours: CAR T-cell therapy. “We believe it could be just as powerful for treating childhood tumours, like DIPG and neuroblastoma,” says Dr Straathof. “We just need to adapt the approach. It’s trickier when the cancer isn’t in the blood or bone marrow, where T-cells can more easily find tumour cells.

“We’re developing CAR T-cell therapy, to save more of these young lives. But also, because it’s so targeted, we hope it can ensure children live happier and healthier lives afterwards, free of side effects of treatment.

“Some of our challenges are the same, such as cancer cells losing their unique marker. Others are unique to solid cancers, such as ensuring CAR T-cells can reach tumours and stick around long enough to shrink them.”

2016 GOSH researchers launch the UK’s first clinical trial of CAR T-cell therapy for childhood neuroblastoma. Early results are promising but suggest higher doses may be required compared with CAR T-cell treatments for blood cancer.

Dr Karin Straathof.

We’ve come a long way since the Hospital for Sick Children opened in 1852.

The hospital’s dedicated and passionate staff have pioneered many new and better ways of treating children with some of the most life-threatening conditions. But we are yet to find a cure and kinder and more effective treatments for all childhood cancers.

Throughout the hospital’s history, it has been the continued and generous support of our donors that has helped to fund the research that provides new breakthroughs. With your ongoing generosity, we seek to give hope to all children that needs specialist help.

Great Ormond Street Hospital Children’s Charity. Registered charity number 1160024. LBGDM2020