Saar Gill, M.D., Ph.D., Assistant Professor in the Perelman School of Medicine, and Dr. Miriam Kim, M.D., Ph.D., a postdoctoral researcher in the Gill Lab, have co-invented a new chimeric antigen receptor (CAR) T cell therapy for the treatment of acute myeloid leukemia (AML). Targeted CAR T cell therapies for AML have been difficult to develop in the past because shared expression of targetable antigens on the tumor and normal tissue typically result in unacceptable toxicity.
The new therapy developed in the Gill Lab involves genetically removing the AML target (CD33) from normal hematopoietic stem and progenitor cells to create a hematopoietic system resistant to CD33-targeted therapy, thus enabling specific targeting of AML with CD33-directed CAR T cells without the previously observed toxicity. This novel approach could lead to an effective therapy for otherwise incurable AML once fully clinically developed.
A patent application has been filed and a clinical trial is pending.