Promising new treatment for rare cancer in children

Promising new treatment for rare cancer in children

A new treatment using 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 blood cancers. This is the first time that researchers have obtained such encouraging results in solid tumors, experts in the field have said, and raises hopes that it could be used against other types of cancers.

It’s too early to call it a cure for neuroblastoma, a cancer of nerve tissue that often begins in early childhood in the adrenal glands near the kidneys in the abdomen.

Standard treatment can be intense, involving chemotherapy, surgery and radiation therapy, 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 at the end of the three-year study, some of whom responded only partially to treatment and received repeated doses of the modified cells.

“These children were all destined to die without this therapy,” said Dr. Carl June of the University of Pennsylvania, a pioneer in CAR-T therapy who was not involved in the new research.

“Nobody’s ever had patients responding like this before, so we just don’t know what it will look like in a decade,” June said. “Of course there will now be more trials based on these exciting results. »

CAR-T cell therapy harnesses the immune system to create “living drugs” that can seek out and destroy tumors. T cells from the patient’s blood are collected and strengthened in the laboratory, then returned to the patient intravenously where they continue to multiply.

Six CAR-T cell therapies have been approved by the United States Food and Drug Administration for blood cancers. Some the first patients were cured.

But success in solid tumors has been elusive. The latest study was carried out by researchers at the Vatican’s Bambino Gesu Pediatric Hospital in Rome.

“They seem to have found a unique combination” for the modified cells to initially multiply and then last a long time to continue their cancer-killing work, said Dr. Robbie Majzner of 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 kill cells if a patient had a severe reaction. When a patient had problems, they flipped the safety switch, showing it worked, although they later determined that the patient’s problem was caused by brain bleeding unrelated to CAR-T cells. .

Many children have had a common side effect with CAR-T therapy – an immune overreaction called “cytokine release syndrome”. It can be severe, but mild in most cases, the researchers reported.

They concluded that CAR-T therapy was “feasible and safe in the treatment of high-risk neuroblastoma.”


The Associated Press Health and Science Department is supported by the Howard Hughes Medical Institute Science and Education Media Group. The AP is solely responsible for all content.

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