In a significant development for pediatric oncology, an experimental CAR T-cell therapy has successfully treated a 3-year-old boy with a rare and aggressive form of liver cancer. The child, diagnosed with hepatoblastoma, had his cancer spread from the liver to his lungs and potentially his bones, and it had become resistant to chemotherapy.
After receiving just two doses of the novel GPC3-CAR T-cell treatment over an eight-week period, the boy showed no remaining signs of the disease. A year later, he remains cancer-free, indicating a complete regression of the illness. This case marks a potential breakthrough in treating solid tumors with CAR T-cell therapy, a treatment previously more limited in its application to blood cancers.
The findings were published this month in The New England Journal of Medicine by researchers from Texas Children’s, Baylor College of Medicine, and Seattle Children’s. The toddler's initial diagnosis involved a large tumor measuring 4.4 × 3.8 × 2.8 inches. Despite multiple rounds of chemotherapy and surgical removal of the tumor, the cancer recurred and metastasized.
CAR T-cell therapy involves genetically modifying a patient's own white blood cells, known as T cells, to specifically target and destroy cancer cells. While effective against certain leukemias and lymphomas, its success against solid tumors has been hindered by the complex tumor microenvironment, which can create a protective shield against immune cells. Tumor cells can also mimic healthy cells, further complicating the immune response.
"This study provides evidence that these novel CAR T-cells may be a safe and effective modality for hepatoblastoma and highlights the need for further assessment in patients with GPC3+ solid tumors," stated study co-author and pediatric oncologist Andras Heczey.
Hepatoblastoma is a common childhood liver cancer, though liver cancer overall is the third leading cause of cancer-related deaths globally. Traditional treatments for hepatoblastoma often include radiation and surgery, which are not always successful.
Currently, CAR T-cell therapy is only FDA-approved for specific types of leukemias, lymphomas, and multiple myeloma. However, this recent success with a solid tumor follows previous research suggesting CAR T-cell therapy's effectiveness against deadly brain tumors in children.
Further research is ongoing to determine the full efficacy and safety of this GPC3-CAR treatment for liver cancer and other GPC3-positive solid tumors. The CARE study, a first-in-human trial for pediatric patients with recurrent solid tumors, is continuing at Baylor College of Medicine to evaluate this experimental form of CAR T-cell therapy.