Experimental CAR-T therapy cures deadly liver cancer in 3-year-old boy

A three-year-old boy in the United States, suffering from the rare liver cancer hepatoblastoma that had metastasized to his skeleton and lungs, was deemed terminal after three ineffective chemotherapy cycles. He was treated with an experimental CAR-T therapy that harvested his own T-cells, edited them to target cancer cells and to produce interleukin-15 and interleukin-21, and then reinfused them in two doses eight weeks apart.

The first infusion triggered a response, and after the second the disease appeared to vanish. One year after the treatment the child is cancer-free, with no signs of relapse, says researcher Else Marit Inderberg. The therapy, previously successful only against blood cancers, is in a phase-I study and will need phase-II and III trials before possible approval, likely first in the United States and later in Norway.