Eleven children were alive when the three-year study ended, including some who only partially responded to treatment and got repeat doses of the modified cells.
“Those kids were all destined to die without that therapy,” said University of Pennsylvania’s Dr Carl June, a pioneer of CAR T-cell therapy who was not involved in the new research.
“No one’s ever had patients responding like this before, so we just don’t know what it’s going to look like a decade from now,” June said. “For sure, there are going to be more trials now based on these exciting results.”
CAR T-cell therapy harnesses the immune system to create “living drugs” able to seek and destroy tumours. T-cells from the patient’s blood are collected and strengthened in the lab, then returned to the patient through an IV, where they continue to multiply.
Six CAR T-cell therapies have been approved by the US Food and Drug Administration for blood cancers. Some early patients have been cured.
But success in solid tumours has been elusive. The latest study was done by researchers at the Vatican’s Bambino Gesu pediatric hospital in Rome.
“They seem to have found a unique combination” to get the modified cells to multiply initially, then last a long time to continue their cancer-killing work, said Dr Robbie Majzner of the Stanford University School of Medicine, who was not involved in the new study.
Study co-author Dr Franco Locatelli said they also added a safety switch to eliminate the cells if a patient had a severe reaction. When one patient had problems, they flipped the safety switch, showing that it worked, although later, they determined the patient’s problem was caused by a brain bleed unrelated to the CAR T-cells.
Many of the children had a side effect that is common with CAR T-cell therapy — an immune overreaction called cytokine release syndrome. It can be serious, but was mild in most, the researchers reported.
They concluded that CAR T-cell therapy was “feasible and safe in treating high-risk neuroblastoma”.
