Stephen Gottschalk Lab

Our laboratory focuses on translational immuno-oncology and immunotherapy for pediatric cancer. Adoptive immunotherapy with T cells, genetically engineered to express chimeric antigen receptors (CARs), has been highly effective for B-cell acute lymphoblastic leukemia (ALL) and lymphoma, leading to approval by the US Food and Drug Administration (FDA). However, CAR T cell therapy approaches have shown limited antitumor activity for solid tumors and brain tumors. 

This lack of efficacy is most likely multifactorial, including a limited array of targeted antigens, and the inability of CAR T cells to sustain their effector function in the tumor microenvironment. Most existing CAR T cell therapy approaches have only targeted cancer cells—not cells of the tumor stroma—which play a critical role in promoting tumor growth and therapeutic resistance. 

A wide array of approaches, including 2D and 3D cell culture systems, flowcytometry, advanced imaging, and singe cell analysis allows us to evaluate the function of engineered immune cells. For our in vivo studies, we take advantage of cell line xenograft, patient derived xenograft (PDX), and immune competent syngeneic models.

Once we select a cell therapy approach for clinical evaluation, we work closely with our Clinical Research Office, the Regulatory Affairs Office, the Experimental Cellular Therapeutics Laboratory, and the Children’s GMP to translate our approach into early phase clinical testing. Our expansive and collaborative research strategy ensures our laboratory can advance translational immune-oncology and immunotherapy for pediatric cancer.