Woman in cancer remission for record 19 years after CAR-T immune treatment

CAR-T-cell therapy treated a girl with a rare childhood cancer, raising hopes for future recipients of the approach

A CAR-T cell (orange) has attacks a cancer cell (green), which is starting to contract.Credit: Eye Of Science/Science Photo Library

The girl was four years old when she arrived at Texas Children’s Hospital in Houston to receive a highly experimental therapy for nerve-cell cancer. Standard treatments had been unable to hold the cancer back. It had spread to her bones, and the prognosis was poor.

Nineteen years later, she is cancer-free and the mother of two children. The remarkable success story, published on 17 February in Nature Medicine¹, is the longest reported cancer remission following treatment with engineered immune cells called CAR T cells.

In the years since the girl’s treatment in 2006, CAR T cells have produced stunning results in some blood cancers such as leukaemia. Seven CAR-T-cell therapies have been approved by the US Food and Drug Administration since 2017, and some early recipients of CAR-T therapy have been cancer-free for more than a decade.

But researchers have struggled to repeat that success against solid tumours such as those caused by neuroblastoma, a nerve-cell cancer that is typically diagnosed in young children. That makes the latest results particularly good news, says Sneha Ramakrishna, a paediatric oncologist and cancer researcher at Stanford University School of Medicine in California, who was not involved in the study.

“This provides me with a lot of hope,” she says. “We’re going to unlock CAR T cells for people with solid tumours.”

Cell target

CAR T cells are immune cells that have been engineered to make a protein called a chimeric antigen receptor (CAR). This protein is designed to latch onto a target found on a cancer cell, triggering the immune cell to attack and destroy it.

When the Texas neuroblastoma study began in 2004, CAR-T-cell therapy was still a bit of a wild idea, says Helen Heslop, an immunotherapy researcher at Baylor College of Medicine in Houston, and a member of the team that ran the trial. “This is some kind of weird synthetic biology,” she remembers thinking. “Will it actually work?”

The CAR-T-cell therapies Heslop and her colleagues tested then are now considered first-generation editions of the molecules. Later versions, including those that are now approved medicines, contain extra modifications to bolster their power. Heslop calls her first CAR-T-cell study “a vintage trial”.