The FDA grants CD33-specific CAR+ T cell Investigational New Drug Application clearance for treatment of R/R AML

On 2^nd May 2017, the U.S. Food and Drug Administration (FDA) accepted the Investigational New Drug (IND) application and granted clearance for  CD33-specific Chimeric Antigen Receptor (CAR) +  T-cell therapy for patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML).¹

CD33-specific CAR+ T-cell is a lentiviral construct that co-expresses CD33-specific CAR and a kill switch based on a tag derived from the epidermal growth factor receptor, which allows for the conditional elimination of CAR-T cells in vivo. CD33 is a transmembrane protein expressed on normal and malignant myeloid-derived cells. In AML blasts, CD33 is broadly expressed and high levels of CD33 expression is associated with poor survival outcomes in AML patients. ² Preclinical analysis of CD33 CAR-T cells in CD33+ AML cell lines have demonstrated specific cytotoxicity.³

The IND approval granted by the FDA allows CD33-speciifc CAR T-cell to be evaluated in phase I clinical trial (NCT03126864) in R/R AML patients which is now active. In this study, the safety and tolerability of three different doses of CD33-CAR T-cell would be evaluated in CD33+ patients with R/R AML. Treatment of the first patient enrolled in this study is anticipated to begin in June 2017.¹