Experimental CAR T-cell therapy induces remission in child with liver cancer

A 3-year-old boy with advanced, metastatic liver cancer has entered complete remission following an experimental immunotherapy treatment. Researchers from the Baylor College of Medicine in Texas detailed the results in the New England Journal of Medicine, noting that the remission has lasted for at least one year.

The treatment utilized a novel form of chimeric antigen receptor (CAR) T-cell therapy. Unlike traditional CAR T therapies that have seen success in blood cancers like leukemia, this experimental approach targets solid tumors. The engineered cells were designed to target glypican-3, a protein frequently found on the surface of cancer cells but absent from healthy cells.

The strategy involved a two-fold engineering process: the cells were modified to express glypican-3 receptors and two immune-related signaling proteins, interleukin-15 and interleukin-21, to enhance anti-cancer efficacy and cell survival. Additionally, the cells included a safety switch that allows doctors to eliminate them using a specific drug if necessary. The boy is part of an ongoing phase I clinical trial.