Personalized T-cell therapy clears metastatic kidney cancer in teen

Researchers at the Hopp Children's Cancer Center Heidelberg have achieved complete remission of advanced, treatment-resistant kidney cancer in an adolescent using a personalized T-cell therapy targeting the PRAME antigen, according to results published August 12 in the New England Journal of Medicine.medicalxpress+1

The case involved a 17-year-old patient who had battled nephroblastoma since age 7, enduring multiple relapses over a decade. By the time of treatment, the disease had spread extensively, with large tumor lesions in the abdominal cavity and metastases in the lungs, liver, pelvis, and brain. No curative options or clinical trials remained available.medicalxpress

Engineering an Immune Attack

The patient's own T cells were genetically modified at the National Center for Tumor Diseases Heidelberg to recognize PRAME, a tumor antigen identified through the INFORM tumor sequencing program. The genetic vector used to reprogram the cells was provided by Immatics ↘ 1.56%, a Tübingen-based biotechnology company that has achieved treatment successes with PRAME-specific T cells in adult clinical trials.linkedin+1

Christian Seitz, the treating physician and medical director of stem cell transplantation, cell and gene therapy at KiTZ and Heidelberg University Hospital, said the decision to proceed despite the advanced disease was based on "highly promising clinical data from our collaboration partner Immatics".medicalxpress

The patient received the locally manufactured cells as an infusion in July 2025 after a seven-day manufacturing process. Just nine days later, a tumor biopsy showed massive T-cell infiltration and cancer cell death. Three months after treatment, no living tumor cells could be detected.medicalxpress

From One Patient to a Clinical Trial

"Currently, almost one year after the infusion of the T cells, our patient is in excellent condition. He trains regularly, takes part in cycling races, was able to successfully complete his vocational training and now plans to obtain his Abitur," Seitz said.medicalxpress

The case has laid the groundwork for a phase I/II clinical trial called "PRAMEtime," planned for 2027, which will enroll up to 18 children and adolescents ages 8 to 17 with PRAME-positive solid tumors. Immatics will provide the vector, while the Center for Innovative Therapies in Heidelberg, founded in June 2026, will manufacture the cells for future projects.medicalxpress

Olaf Witt, co-lead of the planned trial and director of KiTZ, noted that data from more than 2,500 patients in the INFORM study show PRAME occurs in many high-risk pediatric tumors, "and therefore represents a promising target for new therapies".news-medical+1