FDA cancer concern over CAR-T gene therapy

Recently, the US Food and Drug Administration (FDA) has expressed that it is investigating cases of blood cancer in CAR-T therapies. Currently, the FDA have not placed restrictions on the use of CAR-T therapies but the move is a significant step in understanding the risks of gene therpay. The US regulator has recieved a number of reports of T cell malignancies in patients who have rceived autologous treatments using these cells and has determined the risk is applicable to all approved BCMA directed and CD19 directed modified CAR T cell immunotherapies. The use of integrative vectors can result in insertional mutagenesis due to gene disruption. While the benfits ofgene therpay are immense to patient quality of life, the need to understand the risk associated with these vectors and reduce these are imperative in moving the field forward.

FDA Investigating Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies | FDA