The most his team could offer was an experimental treatment to buy a little time, says Dr. Christian Seitz, head of the Cell and Gene Therapy Program at Hopp Children's Cancer Center in Heidelberg. Seitz and his colleagues found that the boy’s cancer carried a set of markers that could be targets for immune cells, called T-cells, that are specially programmed to hunt cancer, and although this type of solid tumor is not usually treatable this way, the team decided to proceed.
The study suggests that doctors and scientists may be able to tailor immune cell treatments even for certain cancers that have been difficult to tackle with earlier forms of engineered immune cell therapy.
Not every cancer is vulnerable to these attacks, though. “The dogma over the past 10 years was, basically, if you have a large solid tumor, it's not going to work,” says Seitz. Something about the T-cells produced by most approaches makes them unsuitable for broaching the defenses of these tumors, which produce their own little microenvironment full of molecules that protect them from the immune system. The approach is typically more successful in blood cancers.
The patient receiving T-cell therapy against PRAME in July 2025. —Courtesy of Hopp Children’s Cancer Center
The role of genetics in T-cell therapy
The boy’s cancer carried a protein called PRAME, which scientists had already been investigating as a potential target for T-cell treatments for melanoma and sarcoma, and which seems to be present in many pediatric cancers as well. Seitz and colleagues worked with Immatics Biotechnologies in Germany, which is running clinical trials of various PRAME-focused treatments, to produce T-cells for the boy’s infusion.
Eight weeks after the treatment, the boy and his mother came in for his follow-up. After the scans, Seitz recalls, “I was looking at the images, and I was like, ‘This is not possible.’” All of the liver metastases were gone. Tumors elsewhere had shrunk enormously, and when doctors checked the tumor tissue for signs of live cancer cells, they saw none. The boy is now cancer-free, with no detectable signs of disease. What’s more, his blood still contains T-cells that hunt down PRAME, suggesting that his body may now be able to eliminate further recurrences.
As with many engineered T-cell discoveries, this particular approach, if it reaches the clinic, is unlikely to work for every patient or every cancer. Still, with many of these approaches, says Orentas, “just a few patients benefit, but when they benefit, they really benefit. I think that's where we're headed with this.”
Seitz, who is now planning a clinical trial of the treatment with 18 pediatric cancer patients who all have PRAME in their tumors, just saw his recovered patient this week. Over the weekend, the boy had been part of an extreme cycling event. “Apparently, they drive uphill, and then they go nuts downhill between trees and rocks,” Seitz says. “And I was like, ‘Oh my God...please don't crash into a tree! It's not worth it!’ But he really loves it”—and Seitz feels honored to have helped him reclaim his life.
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