Liver Cancer Disappears in 3-Year-Old After GPC3-CAR T Treatment; No Recurrence for 1 Year

A new immune cell therapy completely eliminated liver cancer in a 3-year-old child whose disease had not responded to chemotherapy.

According to a study published on the 15th (local time) in the New England Journal of Medicine (NEJM), researchers from Baylor College of Medicine, Texas Children’s Hospital and Seattle Children’s Hospital reported results of GPC3-CAR T-cell therapy in one child with hepatoblastoma, a type of pediatric liver cancer.

The child initially had a large primary liver tumor and lung metastases. Before enrolling in the clinical trial, the child received three rounds of chemotherapy and underwent resection of the primary tumor and two lung metastases, but the disease no longer responded to chemotherapy, and new lung metastases recurred after surgery.

The researchers engineered immune cells from the child’s own cells using two vectors. One encoded a second-generation GPC3-CAR, while the other encoded interleukin-15 (IL15), interleukin-21 (IL21) and an inducible caspase 9 safety switch.

The treatment was administered in two infusions 8 weeks apart in an outpatient setting. A partial remission was observed after the first infusion, and imaging after the second showed complete disappearance of metastatic disease. No dose-limiting toxicity or cytokine release syndrome occurred. The complete response was maintained 1 year after treatment.

First author David Steffin, associate director of cellular therapy and bone marrow transplantation at Texas Children’s Hospital, said, “This case shows that durable complete remission can be achieved in chemotherapy-resistant solid tumors in an outpatient setting without systemic toxicity.”

Corresponding author Andras Heczey, principal investigator at Seattle Children’s Hospital, said, “This study provides evidence that these novel CAR T cells may be a safe and effective treatment for hepatoblastoma,” adding that “further evaluation in patients with GPC3-positive solid tumors is needed.”