Overview
- A New England Journal of Medicine case report published Aug. 12 describes an adolescent with widely metastatic, treatment‑resistant kidney cancer who has no detectable disease one year after a single compassionate‑use infusion of PRAME‑specific TCR‑engineered T cells.
- The treatment used the patient’s own T cells, reprogrammed at NCT Heidelberg to recognize the intracellular tumor antigen PRAME with a genetic vector supplied by Immatics, which lets the cells target peptides presented on HLA molecules rather than surface proteins.
- Early signs of activity included massive T‑cell infiltration into tumors nine days after infusion and biopsies at three months that showed no viable tumor cells, but the patient required about two weeks of intensive care for acute toxicity after treatment.
- Building on the case, KiTZ Heidelberg and partners are preparing the PRAMEtime Phase I/II trial to start in 2027 to enroll up to 18 PRAME‑positive children and adolescents, with roughly €1.8 million in funding from the Dietmar Hopp Foundation and Immatics providing the vector for cell manufacture.
- Tumor sequencing from the INFORM program shows PRAME occurs in many high‑risk pediatric cancers, making it a strategic target, but researchers caution this is a single‑patient result that requires formal trials to confirm how safe and broadly effective the approach will be.