Genetic Variants Linked to CAR T-Cell Efficacy and Safety

Scientists have identified variants in three genes that influence both the efficacy of CAR T-cell therapy and the likelihood of severe side effects. Published today in Science Immunology , these findings represent a first step toward adopting a precision medicine approach in an increasingly used form of cancer immunotherapy.

CAR T-cell therapies have undeniably transformed the treatment of blood cancers, with a rapidly growing number of approvals over the past decade and progressively expanding toward solid tumors. While genetic variants associated with immune-related side effects have been identified for other forms of cancer immunotherapy, such as checkpoint inhibitors, the role of human genetics in CAR T-cell therapy has remained largely unexplored. However, this is particularly important given that most CAR T cells are manufactured from a patient’s own immune cells, meaning the therapy itself carries the patient’s unique genetic makeup.