A 3-year-old with chemotherapy-resistant metastatic hepatoblastoma entered complete regression after two infusions of his own T cells engineered to target glypican-3. The outpatient infusions were eight weeks apart; the response has lasted one year, with no dose-limiting toxicity or cytokine release syndrome reported. The result comes from one patient in a phase 1 study and requires evaluation in a broader group.
The three-year-old entered treatment with a large liver tumour and lung metastases. After three lines of chemotherapy, surgeons completely removed the primary tumour and two lung metastases, but a new lung metastasis developed and the cancer no longer responded to chemotherapy. The child then entered CARE, a first-in-human phase 1 study of glypican-3-specific CAR T cells.[1], [2]
The treatment used the child's own T cells, engineered to recognise glypican-3 and carry interleukin-15 and interleukin-21. Two infusions were delivered in an outpatient clinic eight weeks apart. A partial response followed the first; after the second, imaging showed complete resolution of metastatic disease. Complete regression continued through one year of follow-up, with no dose-limiting toxicity or cytokine release syndrome reported.[2], [1]
The result rests on one patient's experience, reported in the New England Journal of Medicine as a letter. CARE is an early-stage safety study, and this case does not show that other patients with hepatoblastoma will have the same outcome. Researchers say broader assessment may be needed in glypican-3-positive solid tumours. The one-year response is notable, but effectiveness and less common harms require more patients and longer follow-up.[1], [2]
