Neuroblastoma is the most common extracranial solid tumor in children and the most common malignancy in infants. It begins in immature nerve cells and most often starts in the nerve tissue of the adrenal glands. However, it may also arise in nerve tissue of the neck, spinal cord, chest, or abdomen.
Neuroblastoma accounts for roughly 7% of all childhood cancers and 15% of childhood cancer mortality. It usually occurs in children under the age of five, and nearly half of all cases are diagnosed in children younger than two years old.
New advances published by Nature Portfolio describe several long-term survivors from a follow-up of a phase 1 clinical trial for a genetically engineered CAR T cell therapy developed at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital, and Houston Methodist Hospital.
The trial (NCT00085930) was the first study launched at the Center for Cell and Gene Therapy. Nineteen children with relapsed or refractory neuroblastoma were treated with CAR T cells engineered to target GD2, an antigen highly expressed on the surface of neuroblastoma cells.
Of the 11 patients with active disease at the time of treatment, two experienced sustained complete responses — one lasting more than 18 years. Of the eight patients with no evidence of active disease at the time of treatment, five remained disease-free at their last follow-up between 10 and 15 years after infusion.
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