Overview of Chimeric Antigen Receptor T-cell (CAR-T) Therapy

Chimeric antigen receptor (CAR) T cells are genetically modified T cells, where autologous T cells are manipulated ex vivo to express the antigen-binding domain from a B cell receptor that is fused to the intracellular domain of a CD3 T cell receptor. Thus, recognition of a specific cell surface antigen activates T cell response. Various modifications can enhance CAR effector function.

CAR T cells have been studied most extensively in hematologic malignancies. They have shown remarkable success but also have significant side effects including signs of cytokine release syndrome such as fever, hypotension, altered mental status, and seizures, with some patients requiring intensive care.

Presently FDA approved CAR T cell therapies include:

Tisagenlecleucel for pediatric ALL.

Axicabtagene ciloleucel for certain types of adult NHL.

Many others are likely to get approved, prior to the talk.