A child who received Car-T cancer therapy is still without illness 18 years later

About 18 years ago, a 4-year-old girl with a rare nerve cell cancer received an infusion of immune cells that were genetically created to fight the disease. Since then, she has been left without cancer, perhaps making her the longest survivor patient with cancer who received this adapted treatment, researchers report February 17 Nature medicine.

As part of a clinical test, the girl received CAR-T cell therapy, a treatment that requires the removal of some of a patient’s immune cells and their programming to target and kill cancer cells before returning them. Since 2017, seven CAR-T cell therapies have been approved by the US food and drug administration for some blood cancers. But solid tumors, like the girl’s neuroblastoma, have been more difficult to treat with this technology.

This is because solid tumors, which make up approximately 90 percent of all cancers, are tougher to penetrate and are equipped with molecules that can hinder engineering cells, says Helen Heslop, a scientist in Baylor College of Medicine and the Hospital of Texas Children in Houston.

“Neuroblastoma is the first solid tumor where it seems to have curative effects with CAR-T cells,” says Carl June Cancer immunotherapist, which was not involved in the study. “Really really exciting, I think, to see that this happens.”

The Heslop team recruited 19 children with neuroblastoma – 11 with growing active cancer and eight at high risk of relaxation. From 2004 to 2009, all 19 were injected with CAR-T cells. Within seven years of treatment, 12 patients withdrew and died. Of the seven survivors, five were at risk of relapse when treated and were not disease 10 to 15 years later. The other two had increased active cancer at the time they were injected with treatment. One was still in prayer eight years later, but stopped participating in the study at that point; The other is 18-year-old survivors.

“I think now we need to know why some people progressed and some people not,” says June, who works at the University of Pennsylvania Perelman Medical School. Maybe in patients who did not continue to benefit, or engineering cells did not climb so long, or the tumor lost the protein the cells were targeting.

Since this study was done, Heslop says, she and other investigators have added special molecules to CAR-T cells to make them last longer and trace tumors better. In 2023, researchers at Bambino Gesù Children’s Hospital in Italy published a study in which nine out of 27 patients with neuroblastoma had no signs of cancer six weeks after receiving the next CAR-T cells. Five of those patients were without cancer about one to two years later. Long -term results should come out soon.

“We hope that those patients too will have sustained benefit and survive long -term,” Heslop says. While it would take much more research, she says this is a glow that neuroblastoma, and perhaps other solid tumors, can be curable with CAR.