CD123-specific CAR T-cell therapy (St. Jude Children's Research Hospital)

This is an investigational second-generation chimeric antigen receptor (CAR) T-cell therapy developed by St. Jude Children's Research Hospital for the treatment of pediatric and young adult patients with relapsed or refractory CD123-positive hematologic malignancies. The therapy utilizes a lentiviral vector to engineer T cells—derived from either the patient (autologous) or a previous transplant donor—to express a CAR targeting the interleukin-3 receptor subunit alpha (CD123), a marker highly expressed on acute myeloid leukemia (AML) blasts and leukemic stem cells. The CAR construct features a CD28 costimulatory domain for enhanced T-cell activation and survival. For improved safety, the cells are engineered to co-express the CD20 antigen, which serves as a 'safety switch' allowing the cells to be selectively eliminated via the administration of rituximab in the event of severe treatment-related toxicity. It is currently being evaluated in clinical trials as a bridge to allogeneic hematopoietic cell transplant.

Other names

CD123-CAR T cellsCD-123-CAR T cellsCD 123-CAR T cellsCD123 CAR-T with CD20 safety switchCD-123 CAR-T with CD20 safety switchCD 123 CAR-T with CD20 safety switchCAR123-CD20CAR-123-CD20CAR 123-CD20