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

by Molly Chiu, Baylor College of Medicine

edited by Lisa Lock, reviewed by Robert Egan

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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.

The immunotherapy was engineered from the patient's own cells at the Good Manufacturing Practices laboratories at the Center for Cell and Gene Therapy using two vectors: one encoding the second-generation GPC3-CAR and the other encoding IL15, IL21 and the inducible caspase 9 safety switch, which Center for Cell and Gene Therapy investigators previously showed controlled CAR T-cell expansion.

Two infusions were administered eight weeks apart in an outpatient setting. A partial response was observed after the first infusion, and imaging showed complete resolution of the metastatic disease after the second infusion. No dose-limiting toxicities or cytokine release syndrome occurred. Complete regression continues one year after treatment.

"This case demonstrates that a durable complete response in a chemotherapy-resistant solid tumor can be achieved entirely in the outpatient setting without systemic toxicity," said first author Dr. David Steffin, associate chief of cellular therapy and bone marrow transplant at Texas Children's and associate professor of pediatrics—hematology and oncology in the Center for Cell and Gene Therapy at Baylor.

"This study provides evidence that these novel CAR T cells may be a safe and effective modality for hepatoblastoma and highlights the need for further assessment in patients with GPC3+ solid tumors," said corresponding author Dr. Andras Heczey, principal investigator at Seattle Children's and professor of pediatrics in hematology-oncology at the University of Washington School of Medicine. Heczey was at Baylor and Texas Children's at the time of the research.

Publication details

David Steffin et al, Complete Regression of Hepatoblastoma after Interleukin-15– and Interleukin-21–Coexpressing CAR T-Cell Therapy, New England Journal of Medicine (2026). DOI: 10.1056/nejmc2605958

Journal information: New England Journal of Medicine

Key medical concepts

HepatoblastomaCytokine Release Syndrome

Clinical categories

OncologyPediatrics Provided by Baylor College of Medicine Who's behind this story?

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