Gene-engineered T cell therapies are soon to be United States Food and Drug Administration (FDA) approved for at least two types of B cell malignancies in pediatric and adult patients, in the form of CD19 targeted chimeric antigen receptor T (CAR T) cell therapy. This represents a triumph of a true bench to bedside clinical translation of a therapy that was conceived of in the early 1990s. Clinical results have demonstrated efficacious responses in patients with the CD19 positive diseases B cell acute lymphoblastic leukemia and diffuse large B cell lymphoma. However, significant challenges have emerged, including worrisome immune-related toxicities, therapy resistance, and understanding how to administer CD19 CAR T cells in clinical practice. Although much remains to be learned, pioneering clinical trials have led to foundational insights about the clinical translation of this novel therapy. Here, we review the "lessons learned" from the pre-clinical and human experience with CAR T cell therapy. Stem Cells 2018;36:36-44.
Keywords: Acute lymphoblastic leukemia; CD19; Chimeric antigen receptor T; Chronic lymphocytic leukemia; Diffuse large B cell lymphoma.
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