Experimental CAR T therapy induces remission in child with metastatic liver cancer

A 3-year-old boy with metastatic hepatoblastoma has achieved complete remission following experimental GPC3-targeted CAR T cell therapy, according to a case report published in The New England Journal of Medicine.

The patient initially presented with a large liver tumor and cancer that had spread to the lungs and potentially the bones. Despite undergoing three rounds of chemotherapy and multiple surgeries to remove the primary tumor and lung metastases, the cancer recurred.

As part of the Phase 1 CARE study, researchers at institutions including Texas Children’s Hospital and Baylor College of Medicine administered two doses of modified T cells. These cells were genetically engineered to target glypican-3 (GPC3), a protein highly expressed in hepatoblastoma, and were further enhanced with interleukin-15 and interleukin-21 to improve survival and activity.

Following the second infusion, imaging showed complete remission. The patient remained disease-free for 12 months without experiencing severe systemic toxicities, such as cytokine release syndrome. Experts note this result is promising for treating solid tumors, though the method remains in the experimental stage.