title: Gene-modified T-cell therapy using chimeric antigen receptors for pediatric hematologic malignancies.
abstract: Chimeric antigen receptor (CAR) is the generic name for synthetic T cell receptors redirected to tumor-associated antigens. Most CARs consist of an ectodomain (scFv or ligand), a hinge region, a transmembrane domain, and signaling endodomains derived from one or two co-stimulatory molecules (CD28, 4-1BB, etc) and from a CD3-ζ chain. CD19-targeted CAR T cell therapy has achieved major success in the treatment of B cell malignancies. CD19 CAR-T cells elicited complete remission in 70-90% of adult and pediatric patients with relapsed/refractory acute lymphoblastic leukemia (ALL). CD19 CAR T cell therapy from allogeneic donors including third party donors is a potential option for B-cell malignancies. CAR T cell therapies for myeloma, acute myeloid leukemia, and T-cell leukemia are still under development. Our group is currently preparing a phase I study of CD19 CAR T cell therapy in pediatric and young adult patients with ALL using a non-viral gene transfer method, the piggyBac-transposon system.
authors.affiliation: Shinshu University School of Medicine, Department of Pediatrics.
authors.forename: Yozo
authors.initials: Y
authors.lastname: Nakazawa
mesh_terms: D002648:Child; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D019337:Hematologic Neoplasms / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D006801:Humans; D007167:Immunotherapy; D011948:Receptors, Antigen, T-Cell / Q000235:genetics / Q000627:therapeutic use*; D013601:T-Lymphocytes / Q000276:immunology*
chemical_list: D011948:Receptors, Antigen, T-Cell