Immuno-gene therapeutics are transforming the therapeutic landscape of hematological malignancies. The recent approvals of two chimeric antigen receptor (CAR)-T cell therapies—tisagenlecleucel (marketed as Kymriah™) and axicabtagene ciloleucel (marketed as Yescarta™)—mark the beginning of the next revolution in cancer treatment. However, along with demonstrated efficacy in hematologic malignancies, CAR-T cells have the capacity to elicit serious toxicities.
Safety considerations related to CAR-T cells may impact both trial design and trial management, as the adverse events (AEs) associated with immuno-oncology agents differ from those associated with cytotoxic therapies. Consequently, anticipating, preventing and managing toxicity is a key component of clinical studies involving CAR-T cells.