An experimental cancer therapy has reached a potentially significant milestone in the treatment of solid tumors.
Chimeric Antigen Receptor (CAR) T-cell therapy has produced impressive results against blood cancers, including certain leukemias and lymphomas. Its effectiveness against solid tumors, however, has remained limited — until now.
A 3-year-old boy with chemotherapy-resistant cancer that had spread from his liver to his lungs and possibly his bones was declared cancer-free after receiving just two doses of the experimental treatment.
Researchers from Texas Children’s, Baylor College of Medicine and Seattle Children’s detailed the case in a report published this month in The New England Journal of Medicine.
The child had been diagnosed with hepatoblastoma, a common type of liver cancer in young children. At the time, doctors found a large tumor measuring 4.4 × 3.8 × 2.8 inches.
Although he underwent several rounds of chemotherapy and surgery to remove the tumor, the cancer returned. It later spread to his lungs, while tests also indicated possible involvement of his bones.
Doctors then turned to CAR T-cell therapy, an approach that removes a patient’s T cells, genetically modifies them to recognize cancer and returns them to the body to attack malignant cells.
The boy received two doses of the experimental GPC3-CAR treatment over an eight-week period. Afterward, doctors found no remaining evidence of disease, and he remained cancer-free one year later — a result indicating complete regression.
“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,” study co-author and pediatric oncologist Andras Heczey said.
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Liver cancer is the third-leading cause of cancer-related deaths worldwide. Hepatoblastoma is generally treated with surgery and chemotherapy, but those approaches do not always eliminate the disease.
CAR T-cell therapy is an innovative cancer treatment, but the FDA has approved it only for certain forms of leukemia, lymphoma and multiple myeloma.
Treating solid tumors with CAR T cells has proved especially challenging because the tumors can create a hostile chemical environment that blocks or destroys attacking immune cells.
Cancer cells may also be difficult to distinguish from healthy tissue. Tumors can recruit nearby healthy cells, including fibroblasts and regulatory immune cells, to form a protective barrier around them.
The latest development follows research suggesting that T-cell therapy may also help treat deadly brain tumors in children.
Because the liver cancer treatment was tested in only one patient during an early-phase trial, researchers caution that additional studies are needed to determine its safety and overall effectiveness. Still, the outcome offers an encouraging signal.
Further testing of GPC3-CAR therapy is underway through the CARE study at Baylor College of Medicine. The trial is the first human study of an experimental CAR T-cell treatment for children with recurrent solid tumors.