3-year-old boy declared cancer-free after CAR T-cell therapy

A significant advancement in cancer treatment has been reported. Chimeric Antigen Receptor (CAR) T-cell therapy, which has been effective in treating certain blood cancers, has shown potential in treating solid tumors. A 3-year-old boy diagnosed with hepatoblastoma, a type of liver cancer, was declared cancer-free after receiving two doses of an experimental CAR T-cell therapy. The boy's cancer had spread from his liver to his lungs and possibly his bones, and despite multiple rounds of chemotherapy and surgery, the cancer returned. Doctors administered the GPC3-CAR treatment, which involves genetically modifying the patient's T cells to target cancer cells. Following the treatment, the boy showed no signs of the disease after one year, suggesting complete regression. The findings were published in The New England Journal of Medicine by researchers from Texas Children’s Hospital, Baylor College of Medicine, and Seattle Children’s Hospital. Pediatric oncologist Andras Heczey, a co-author of the study, noted that this research indicates the potential of CAR T-cells as a safe and effective treatment for hepatoblastoma and emphasizes the need for further studies on GPC3+ solid tumors. Liver cancer is the third leading cause of cancer-related deaths worldwide, and hepatoblastoma typically requires radiation and surgery, which are not always successful. While CAR T-cell therapy is currently FDA-approved for certain blood cancers, its application for solid tumors has been challenging due to protective barriers created by tumor cells. This breakthrough follows previous findings that T-cell therapy can effectively treat aggressive brain tumors in children. The GPC3-CAR treatment is being further evaluated in the CARE study, the first human trial of this CAR T-cell therapy for pediatric patients with recurrent solid tumors at Baylor College of Medicine.