Two doses of engineered CAR T cells eliminate child's metastatic liver cancer

A 3-year-old boy with chemotherapy-resistant hepatoblastoma that had spread to his lungs and bones achieved complete remission after two doses of an experimental CAR T-cell therapy. The case, published in The New England Journal of Medicine, is the first time CAR T cells have eliminated a solid tumor in a child. Liver cancer is the third leading cause of cancer-related death globally.

Bottom line — The NEJM case marks the first complete remission of a chemotherapy-resistant solid tumor using CAR T cells.

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• The boy's primary liver tumor measured 11.2 × 9.6 × 7.1 cm at diagnosis, with metastases in the lungs and suspected bone involvement, per ScienceAlert.
• After three rounds of chemotherapy and multiple surgeries, the cancer rapidly returned, the researchers reported.
• The experimental GPC3-CAR T cells were engineered to recognize glypican-3, a protein highly expressed in liver cancers, and included genes for interleukin-15 and interleukin-21 to boost persistence, according to the study.
• The patient received two infusions in an outpatient setting without systemic toxicity, said pediatric oncologist David Steffin of Baylor College of Medicine.
• Follow-up scans 12 months after treatment showed no detectable disease apart from residual scarring, the team reported.
• The findings are from a single patient in the early-phase CARE trial; further testing is ongoing at Baylor and in the IMPACT trial at Seattle Children's Hospital, per the researchers.

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