3-year-old’s cancer disappeared after two doses of experimental CAR-T therapy

Baylor College of Medicine researchers achieved full remission lasting 12 months in a child with chemotherapy-resistant hepatoblastoma after two outpatient infusions of GPC3-targeted CAR-T cells. The case, reported in NEJM, is the first sustained complete cure of a solid tumor with this method without systemic toxicity.

• Liver tumor 11.2×9.6×7.1 cm with lung metastases vanished after two infusions
• CARE T-cell therapy enhanced with IL-15 and IL-21 and built-in safety mechanism
• Full remission persists 12 months, with no cytokine release syndrome
• Method is still in phase 1 trials and not FDA approved