Experimental CAR-T cell therapy eliminates liver cancer signs in child
A genetically engineered CAR-T cell therapy achieves unprecedented response in a child with treatment-resistant liver cancer.
Key Takeaways
AIAn experimental therapy using chimeric antigen receptor T cells (CAR-T) has shown remarkable progress against liver cancer, successfully eliminating signs of the disease in a three-year-old child, despite the cancer’s spread and resistance to previous treatments.
A study published in the New England Journal of Medicine reported that the child had hepatoblastoma, a type of liver cancer affecting children. The tumor was large and had metastasized to the lungs and bones. Despite undergoing three rounds of chemotherapy and surgeries to remove tumors from the liver and lungs, the cancer returned and a new tumor appeared.
As part of the “CARE” study at Baylor College of Medicine, the child received two doses of an experimental treatment called “GPC3-CAR.” The therapy was created from the child’s own T cells, which were genetically modified to recognize the glypican-3 (GPC3) protein, associated with certain liver cancers.
Notable response to therapy
Scans after the first dose showed a partial response, with tumor shrinkage and a decrease in blood levels of alpha-fetoprotein. After the second dose, given eight weeks later, there were no clear signs of remaining disease except for some residual scarring, and the child remained free of disease markers after 12 months.
David Stephen, lead author of the report and a pediatric oncologist at Baylor College of Medicine, said the case demonstrates the potential for a complete and sustained response in a patient with a solid tumor resistant to chemotherapy, without causing significant systemic toxicity.
Andras Heczey, a co-investigator in the study, noted that these results provide preliminary evidence that CAR-T cells could be used to treat hepatoblastoma, but further studies are needed to assess safety and effectiveness.
Ongoing clinical trials
The researchers cautioned that this outcome is based on a single patient in an early-stage trial, so the overall safety and efficacy of the therapy in a larger group of patients remain unknown. The “CARE” and “IMPACT” trials are ongoing to study this approach in other patients with solid tumors.