Promising treatment for rare cancer in children gives hope to experts

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A novel treatment using supercharged immune cells works against tumors in children with a rare type of cancer, researchers reported Wednesday.

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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 cancers in the blood. It is the first time researchers have obtained such encouraging results in solid tumors, experts in the field said, and expressed hope that it could be used against other types of cancer.

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

Standard treatment can be intense, including chemotherapy, surgery, and radiation, depending on the stage of the cancer and other factors. The children in the study had cancer that had come back or was particularly difficult to treat.

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

“All of those kids were destined to die without that therapy,” said Dr. Carl June of the University of Pennsylvania, a pioneer of the CAR-T therapy who was not involved in the new research.

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

CAR-T cell therapy harnesses the immune system to create “living drugs” capable of finding and destroying tumors. T cells are collected from the patient’s blood 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 cancer. Some initial patients have recovered.

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

“It looks like they’ve found a unique combination,” said Dr. Robbie Mazner of the Stanford University School of Medicine. The modified cells have been shown to multiply initially, then last long enough to continue their cancer-killing work. . Study.

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

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

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


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

Promising treatment for rare cancer in children gives hope to experts

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