When a child receives a diagnosis of diffuse intrinsic pontine glioma or relapsed brain tumour, families often hear that survival beyond a year is rare. Yet four children who took part in an early trial of a new cell therapy remain alive years after treatment, three of them with no detectable disease.
The approach, known as tumour-associated antigen T-cell therapy, was given to 33 children and young adults. Researchers extracted each patient's own T-cells from blood, trained them in the laboratory to recognise three protein markers commonly found on paediatric brain tumours, multiplied the reactive cells and returned them by intravenous infusion. The phase 1 trial met its goals for safety and feasibility.
Three of the four long-term survivors had recurrent glioblastoma, astroblastoma or medulloblastoma. They show no evidence of disease between two and five years after receiving the cells. The fourth child, who had diffuse intrinsic pontine glioma, has lived more than two years since treatment. These outcomes stand in sharp contrast to the typical prognosis for such tumours.
These children are getting to grow up – it’s truly awesome.
Gene Hwang of Children's National Hospital captured the human weight of the data. The results appeared in Nature Medicine on 30 June 2026, with trial identifier NCT03652545.
The therapy does not involve genetic engineering of the sort used in CAR-T treatments. Side effects were mostly mild, such as fatigue and headache, though some patients experienced temporary tumour swelling. Its success in a handful of cases has prompted two new trials. One will combine the cells with ultrasound to open the blood-brain barrier. The other will use a personalised method that sequences each child's tumour to find unique antigens before training the T-cells.
Catherine Bollard, also of Children's National Hospital, described the gratitude of one family still in touch years later.
