title: Will novel agents for ALL finally change the natural history?
abstract: Pediatric acute lymphoblastic leukemia (ALL) cure rates have markedly improved over the past years to approximately 85%, but remain at 40%-50% in adults. Redefining current adult chemotherapy regimens is likely to improve the natural course of the disease, but new agents are needed. Immunotherapy approaches for pre-B ALL are in the forefront of research on novel agents; in particular, advances are being made in manipulating autologous T cells either by infusion of a bifunctional antibody (eg, blinatumomab) or by ex vivo genetic modification of chimeric antigen receptors (CARs). The natural course of Philadelphia positive ALL has already improved by targeting ABL/BCR1. Other mutated genes are being discovered and novel small molecules that target their products are being studied in clinical trials. Finally, ALL is a heterogeneous disease and novel agents are likely to impact the natural course of smaller populations of biologically defined ALL subtypes.
authors.affiliation: Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College, 1275 York Ave, New York, NY 10065, USA. Electronic address: douerd@mskcc.org.
authors.forename: Dan
authors.initials: D
authors.lastname: Douer
mesh_terms: D000293:Adolescent; D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016503:Drug Delivery Systems / Q000379:methods*; D005260:Female; D016044:Fusion Proteins, bcr-abl / Q000037:antagonists & inhibitors / Q000235:genetics; D006801:Humans; D017710:Lymphocyte Transfusion; D008297:Male; D015452:Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / Q000235:genetics / Q000628:therapy*; D013601:T-Lymphocytes / Q000637:transplantation*
chemical_list: D000970:Antineoplastic Agents; D016044:Fusion Proteins, bcr-abl
keywords: ALL; CAR T cells; acute lymphoblastic leukemia; immunotherapy; targeted agents