This New Experimental Therapy Eliminated Cancer
A three-year-old boy diagnosed with hepatoblastoma, the most common form of liver cancer in children, had a primary tumor measuring more than four inches across in the left lobe of his liver, with cancer that had spread to his lungs and shown indications of spreading to his bones. He underwent three rounds of chemotherapy, surgery to remove the primary liver tumor, and two additional operations to remove cancer from his lungs. The cancer came back anyway, with a new tumor appearing in his lungs after the final surgery. When standard treatment options had been exhausted, the boy was enrolled in the CARE study at Baylor College of Medicine and Texas Children's Hospital in Houston, the first human clinical trial of an experimental form of CAR T cell therapy for this type of cancer. Researchers engineered a treatment from his own T cells, modifying them to recognize and target glypican-3, a protein expressed at high levels on the surface of liver cancer cells, and also equipped the modified cells with two immune signaling proteins, IL-15 and IL-21, to help them survive and multiply after being infused back into his body. The treatment was given as two outpatient infusions eight weeks apart, meaning the boy did not need to be hospitalized for either dose.
After the first infusion, imaging showed a partial response. After the second infusion, imaging showed complete resolution of all detectable metastatic disease. The results were published in the New England Journal of Medicine and described by the researchers at Baylor and Texas Children's Hospital as a complete radiographic regression, meaning no cancer could be found anywhere in the boy's body on imaging. He has been in remission for one year. No cytokine release syndrome occurred, a serious inflammatory reaction that is one of the main safety concerns associated with CAR T cell therapy, and no dose-limiting toxicities of any kind were observed during or after either infusion. The treatment was manufactured at the Center for Cell and Gene Therapy's Good Manufacturing Practices laboratories, and included a safety switch called inducible caspase 9 that allows researchers to deactivate the engineered cells if serious side effects emerge. Hepatoblastoma has a survival rate of approximately 50 to 80 percent depending on how far the cancer has spread at diagnosis, and children whose cancer returns after initial treatment have historically had very limited options. This boy had run out of standard options entirely. He received two outpatient infusions. A year later, imaging shows nothing.
