Novel immunotherapy leads to complete regression of chemotherapy-resistant liver cancer in 3-year-old

A new report in the New England Journal of Medicine by researchers at Baylor College of Medicine, Texas Children's Hospital and Seattle Children's describes a complete regression of hepatoblastoma, the most common pediatric liver cancer, in a 3-year-old treated with a novel immunotherapy.

The patient is enrolled in the CARE study (NCT04715191), a first-in-human, phase 1 trial evaluating glypican-3-specific chimeric antigen receptor (GPC3-CAR) T cells armed with interleukin-15 and -21 (IL15 and IL21).

The child initially presented with a large primary liver tumor and metastases in the lungs. Before enrollment in the CARE study, the patient was treated with three lines of chemotherapy and underwent complete resection of the primary liver tumor and two lung metastases. His cancer was no longer responsive to chemotherapy, and a new lung metastasis recurred after surgery when he was enrolled in the CARE study.

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