Four of 33 Children Survive Years After Experimental T-Cell Therapy in Early Safety Trial

Four children with traditionally incurable brain cancers remain alive years after an experimental immune therapy, New Scientist reported. Three of them show no evidence of disease. The treatment, called tumour-associated antigen T-cell therapy, was tested in 33 children and young adults.

One participant with diffuse intrinsic pontine glioma survived more than two years. Three others with recurrent glioblastoma, astroblastoma or medulloblastoma lived two to five years without detectable disease after prior treatments failed. Researchers collected T-cells from each patient’s blood and trained them in the laboratory to recognize three protein markers common in pediatric brain tumors.

The cells were then returned intravenously. The method does not involve genetic engineering. Most patients experienced only fatigue or headache. Two developed temporary tumor swelling. The trial, published in Nature Medicine, was a phase-I safety study without a control group.

The team at Children’s National Hospital has started two new trials. One pairs the therapy with ultrasound to open the blood-brain barrier. The other sequences each tumor to train T-cells against patient-specific antigens.