A novel treatment that uses supercharged immune cells appears to work against tumors in children with a rare type of cancer, researchers reported Wednesday.
Nine of the 27 children in the Italian study had no signs of cancer six weeks after treatment, although two later relapsed and died.
The treatment, called CAR-T cell therapy, is already used to help the immune system fight leukemia and other types of blood cancers. This is the first time researchers have achieved such encouraging results in solid tumors, experts in the field said, raising hopes that it could be used against other types of cancer.
It’s too early to call it a cure for neuroblastoma, a cancer of nerve tissue that often begins in childhood in the adrenal glands near the kidneys in the abdomen.
Standard treatment can be intense and include chemotherapy, surgery, and radiation, depending on the stage of the cancer and other factors. The children in the study had cancers that had come back or were particularly difficult to treat.
Eleven children were alive when the three-year study ended, including some who only partially responded to treatment and received repeat doses of the engineered cells.
“All of those children were destined to die without that therapy,” said Dr. Carl June of the University of Pennsylvania, a pioneer of CAR-T therapy who was not involved in the new research.
“No one has ever had patients respond like this before, so we don’t know what it will be like a decade from now,” June said. “There will certainly now be more trials based on these exciting results.”
CAR-T cell therapy harnesses the immune system to create “living drugs” capable of seeking out and destroying tumors. T cells from the patient’s blood are collected and strengthened in the laboratory, 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 of the first patients have been cured..
But success in solid tumors has been elusive. The latest study was conducted 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 and then last a long time to continue their work against cancer, said Dr. Robbie Majzner of Stanford University School of Medicine, who did not participated in the study. new study
Study co-author Dr. Franco Locatelli said they also added a safety switch to kill the cells if a patient had a severe reaction. When a patient had problems, they flipped the safety switch and demonstrated that it worked, only to later determine that the patient’s problem was due to a brain hemorrhage not related to CAR-T cells.
Many of the children had a side effect that is common with CAR-T therapy: an immune overreaction called “cytokine release syndrome.” It can be severe, but was mild in most, the researchers reported.
They concluded that CAR-T therapy was “feasible and safe in the treatment of high-risk neuroblastoma.”
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