Eighteen years ago, a four-year-old girl was given a futuristic cancer treatment that sounded like something out of a biomedical thriller—genetically engineered immune cells designed to annihilate her disease. The therapy, known as CAR-T cell treatment, was an experimental shot in the dark against her rare nerve cell cancer. Nearly two decades later, she’s still here, still cancer-free, possibly the longest-surviving patient to ever receive this kind of tailor-made cellular warfare.
Her treatment wasn’t some off-the-shelf chemo cocktail. CAR-T therapy involves extracting a patient’s T cells, reprogramming them into ruthless cancer assassins, and then sending them back in to finish the job. The FDA has since approved multiple CAR-T therapies for blood cancers, but solid tumors—like neuroblastoma—have remained stubbornly resistant, as if they were designed by an evil AI hell-bent on outlasting humanity.
Solid tumors make up about 90% of all cancers, and they’re a nightmare to crack. They come armored with defenses that neutralize immune attacks and keep engineered T cells from doing their work. “Neuroblastoma is the first solid tumor where there looks like there could be curative effects with CAR-T cells,” says Carl June, a cancer immunotherapy expert who wasn’t involved in the study but still sounds impressed. “It’s really exciting, I think, to see this happen.” In science-speak, that’s basically a standing ovation.
Between 2004 and 2009, researchers treated 19 children with neuroblastoma using CAR-T therapy. It was a brutal test: 12 relapsed and died within seven years. But among the survivors, something astonishing happened—five high-risk patients showed no signs of disease 10 to 15 years later. Two had actively growing cancer when they got the treatment. One stayed in remission for eight years before disappearing from the study. The other? She’s still cancer-free after 18 years.
Why did some survive while others didn’t? That’s the billion-dollar question. Carl June suspects either the engineered cells didn’t last long enough in some patients, or the tumors evolved, shedding the protein that made them vulnerable. Cancer, after all, is nothing if not adaptive—like a biological Hydra, always growing back stronger.
Researchers aren’t sitting still. Heslop’s team and others have been upgrading CAR-T cells with molecular enhancements designed to make them tougher, smarter, and more persistent. In 2023, an Italian research group dropped another hopeful update—nine of 27 neuroblastoma patients showed no signs of cancer just six weeks after receiving next-gen CAR-T cells. A year or two later, five were still cancer-free.
The war against cancer is far from over, but genetic engineering is starting to tip the scales. If today’s CAR-T cells can take down solid tumors, we might be witnessing the beginning of a new era—one where cancer is no longer an unbeatable villain, but just another enemy to outsmart.
Five Fast Facts
- CAR-T therapy was originally inspired by HIV research, as scientists studied how viruses manipulate immune cells.
- Neuroblastoma, the cancer in this case, primarily affects children and is responsible for about 15% of all childhood cancer deaths.
- The first-ever CAR-T therapy approved by the FDA was Kymriah, greenlit in 2017 for a type of leukemia.
- Bambino Gesù Children’s Hospital, which contributed to the latest CAR-T research, was founded in 1869 and is known as the “Pope’s Hospital.”
- T cells, the immune cells used in CAR-T therapy, were discovered in the 1960s and named after the thymus, the organ where they develop.