An experimental T-cell therapy has allowed four children with aggressive, typically fatal brain cancers to survive for years after treatment in a clinical trial. Three show no signs of disease.
The children received tumour-associated antigen T-cell therapy at Children’s National Hospital in Washington, D.C. One child with diffuse intrinsic pontine glioma remains alive more than two years later, while three others with recurrent glioblastoma, astroblastoma or medulloblastoma have lived between two and five years without evidence of disease.
Gene Hwang, who worked on the trial, said: “These children are getting to grow up – it’s truly awesome.” Catherine Bollard noted that the phase I study showed a signal of efficacy despite its small size and lack of a control group.
The therapy trains a patient’s own T-cells to target multiple tumour antigens without genetic modification. It produced fewer side effects than CAR T-cell treatments, mainly fatigue and headache.
Researchers have now started two follow-up trials, one combining the therapy with ultrasound to improve brain access and another using personalised tumour sequencing for antigen targeting.
