Novel treatment shows promise against rare cancer in kids


Massachusetts, April 6 (BNA): A new treatment using supercharged immune cells appears to work against tumors in children with a rare type of cancer, researchers said Wednesday.

Nine of the 27 children in the Italian study showed no sign of cancer after six weeks of treatment, although two children later relapsed and died.

A treatment called CAR-T cell therapy is already used to help the immune system fight leukemia and other cancers in the blood. This is the first time researchers have achieved such encouraging results in solid tumors, experts in the field said, which raises hopes that they could be used against other types of cancers, according to the Associated Press (AP).

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 intensive, including chemotherapy, surgery, and radiation, depending on the stage of the cancer and other factors. The children in the study had cancers that came back or were particularly difficult to treat.

Eleven children were alive when the three-year study ended, including some who had only partially responded to treatment and received repeat doses of the modified cells.

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

“Nobody’s had patients respond like this before, so we don’t know what it’s going to look like a decade from now,” John said. “Certainly, there will be more trials now based on these exciting results.”

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

Six CAR-T cell therapies have been approved by the US Food and Drug Administration for blood cancers. Some patients were treated early.

But success in treating solid tumors has been elusive. The latest study was conducted by researchers at the Vatican City’s Bambino Gesu Children’s Hospital in Rome.

said Dr. Robbie Magsner of Stanford University School of Medicine, who was not involved in the new study.

They also added a safety switch to get rid of the cells if a patient had a severe reaction, said Dr. Franco Locatelli, co-author of the study. When a patient had problems, they flipped the safety switch, showing that it was working, though they later determined that the patient’s problem was caused by a brain hemorrhage unrelated to CAR-T cells.

Many children had a common side effect with CAR-T therapy which was an overactive immune reaction called “cytokine release syndrome.” Researchers reported that it could be serious, but it was mild in most cases.

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

HF






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