CAR T-cell therapy eradicates metastatic liver cancer in 3-year-old boy

September 15, 2026 at 09:28 AM2 min

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A 3-year-old boy with metastatic hepatoblastoma achieved complete remission after receiving an experimental CAR T-cell therapy targeting the GPC3 protein. The case, reported in The New England Journal of Medicine, marks the first human trial of this approach. The child remained disease-free 12 months after treatment.

Key Facts

The boy's initial liver tumor measured 11.2 cm x 9.6 cm x 7.1 cm and had metastasized to the lungs and bones.
He received two infusions of GPC3-CAR T cells engineered to recognize glypican-3, a protein highly expressed in liver cancers.
After the first infusion, CT scans and falling alpha-fetoprotein levels showed a partial response; eight weeks later, follow-up scans showed no remaining signs of disease except residual scarring.
At 12 months post-treatment, the child remained disease-free, according to the report in The New England Journal of Medicine.
The CARE trial is being conducted at Baylor College of Medicine, with a similar trial called IMPACT underway at Seattle Children's Hospital.

Case Background

The child was initially diagnosed with hepatoblastoma, a type of liver cancer, with a mass measuring 11.2 cm x 9.6 cm x 7.1 cm in the left lobe of the liver. The cancer had metastasized to the lungs and showed signs of spreading to the bones. He underwent three sessions of chemotherapy and two surgeries to remove the primary liver tumor and lung metastases. Despite these interventions, the cancer rapidly returned, and a new tumor appeared in his lungs.

Experimental Treatment

Following the rapid relapse, the child was enrolled in the CARE study, the first human trial of an experimental CAR T-cell therapy for this cancer type. He received two doses of GPC3-CAR T cells made from his own T cells and engineered to recognize glypican-3 (GPC3), a protein highly expressed in liver cancers. Researchers also added genes expressing the immune proteins interleukin-15 and interleukin-21 to enhance the CAR T cells' longevity and tumor-killing ability. After the first infusion, the patient showed a partial response, indicated by CT scans and falling levels of alpha-fetoprotein, a marker of tumor activity. Eight weeks later, he received the second dose, and follow-up scans showed no remaining signs of disease except some residual scarring.

Outcome and Next Steps

At 12 months, the child remained disease-free, a result described as remarkable given the severity of his disease before trial enrollment. Co-author Andras Heczey, a pediatric oncologist at Seattle Children's Hospital and the University of Washington, said the study provides evidence that these new CAR T cells may be a safe and effective modality for hepatoblastoma. He emphasized the need for further evaluation in patients with GPC3-positive solid tumors. Testing continues in the CARE study at Baylor College of Medicine and in a similar clinical trial called IMPACT conducted through Seattle Children's Hospital.