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2. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Author

Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Pajno, Roberta, Barzaghi, Federica, Giannelli, Stefania, Dionisio, Francesca, Brigida, Immacolata, Bonopane, Marco, Casiraghi, Miriam, Tabucchi, Antonella, Carlucci, Filippo, Grunebaum, Eyal, Adeli, Mehdi, Bredius, Robbert G., Puck, Jennifer M., Stepensky, Polina, Tezcan, Ilhan, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Ciceri, Fabio, Roncarolo, Maria Grazia, and Aiuti, Alessandro

Published
2016
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13. Insulin-like growth factor 1 regulates developing brain glucose metabolism

Author

Cheng, Clara M., Reinhardt, Rickey R., Lee, Wei-Hua, Joncas, George, Patel, Sonya C., and Bondy, Carolyn A.

Subjects
Mental retardation -- Research, Brain research -- Analysis, Growth factors -- Research, Science and technology
Abstract

The brain has enormous anabolic needs during early postnatal development. This study presents multiple lines of evidence showing that endogenous brain insulin-like growth factor 1 (Igf1) serves an essential, insulin-like role in promoting neuronal glucose utilization and growth during this period. Brain 2-deoxy-D- [1-[sup.14]C]glucose uptake parallels Igf1 expression in wild-type mice and is profoundly reduced in Igf1-/- mice, particularly in those structures where Igf1 is normally most highly expressed. 2-Deoxy-D-[1-[sup.14]C]glucose is significantly reduced in synaptosomes prepared from Igf1-/- brains, and the deficit is corrected by inclusion of Igf1 in the incubation medium. The serine/threonine kinase Akt/ PKB is a major target of insulin-signaling in the regulation of glucose transport via the facilitative glucose transporter (GLUT4) and glycogen synthesis in peripheral tissues. Phosphorylation of Akt and GLUT4 expression are reduced in Igf1-/- neurons. Phosphorylation of glycogen synthase kinase 3[Beta] and glycogen accumulation also are reduced in Igf1-/- neurons. These data support the hypothesis that endogenous brain Igf1 serves an anabolic, insulin-like role in developing brain metabolism. mental retardation | glycogen synthase kinase | glucose transporter | GLUT4 | Akt

Published
2000

14. Efficacy and safety of once-weekly GLP-1 receptor agonist albiglutide (HARMONY 2): 52 week primary endpoint results from a randomised, placebo-controlled trial in patients with type 2 diabetes mellitus inadequately controlled with diet and exercise

Author

Nauck, Michael A., primary, Stewart, Murray W., additional, Perkins, Christopher, additional, Jones-Leone, Angela, additional, Yang, Fred, additional, Perry, Caroline, additional, Reinhardt, Rickey R., additional, and Rendell, Marc, additional

Published
2015
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15. Biochemical and Morphometric Analyses Show that Myelination in the Insulin-like Growth Factor 1 Null Brain Is Proportionate to Its Neuronal Composition

Author

Cheng, Clara M., primary, Joncas, George, additional, Reinhardt, Rickey R., additional, Farrer, Robert, additional, Quarles, Richard, additional, Janssen, Jeremy, additional, McDonald, Michael P., additional, Crawley, Jacqueline N., additional, Powell-Braxton, Lynn, additional, and Bondy, Carolyn A., additional

Published
1998
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16. Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Author

Jonathan Appleby, Francesca Ferrua, Katie Rolfe, Maria Grazia Roncarolo, Laura Castagnaro, Erika H. De Boever, Rickey R. Reinhardt, Maria Pia Cicalese, Alessandro Aiuti, Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, and Aiuti, Alessandro

Subjects
0301 basic medicine, Male, Opportunistic infection, Adenosine Deaminase, opportunistic infection, Immune deficiency, Gene Expression, Autoimmunity, Gastroenterology, Adenosine deaminase, Agammaglobulinemia, Drug Discovery, Transgenes, retroviral vector, Child, biology, Hematopoietic Stem Cell Transplantation, immune reconstitution, gene therapy, Combined Modality Therapy, Phenotype, Treatment Outcome, Child, Preschool, Bronchitis, Molecular Medicine, Original Article, Safety, safety, medicine.medical_specialty, Genetic Vectors, Neutropenia, 03 medical and health sciences, Gene therapy, Genetic, Internal medicine, Genetics, medicine, Humans, Enzyme Replacement Therapy, Adverse effect, Molecular Biology, Pharmacology, Severe combined immunodeficiency, business.industry, Drug Discovery3003 Pharmaceutical Science, Infant, Genetic Therapy, Immune reconstitution, immune deficiency, medicine.disease, Adenosine deaminase deficiency, 030104 developmental biology, Upper respiratory tract infection, ADA-SCID, biology.protein, Retroviral vector, Severe Combined Immunodeficiency, business, Follow-Up Studies
Abstract

Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are impaired. Gene therapy (GT) with an autologous CD34+-enriched cell fraction that contains CD34+ cells transduced with a retroviral vector encoding the human ADA cDNA sequence leads to immune reconstitution in most patients. Here, we report short- and medium-term safety analyses from 18 patients enrolled as part of single-arm, open-label studies or compassionate use programs. Survival was 100% with a median of 6.9 years follow-up (range, 2.3 to 13.4 years). Adverse events were mostly grade 1 or grade 2 and were reported by all 18 patients following GT. Thirty-nine serious adverse events (SAEs) were reported by 15 of 18 patients; no SAEs were considered related to GT. The most common adverse events reported post-GT include upper respiratory tract infection, gastroenteritis, rhinitis, bronchitis, oral candidiasis, cough, neutropenia, diarrhea, and pyrexia. Incidence rates for all of these events were highest during pre-treatment, treatment, and/or 3-month follow-up and then declined over medium-term follow-up. GT did not impact the incidence of neurologic/hearing impairments. No event indicative of leukemic transformation was reported., In 2016 the gene therapy, Strimvelis was approved for the treatment of patients with ADA-SCID for whom there is no suitable bone marrow donor. In this issue of Molecular Therapy, Aiuti et al. provide detailed safety data for treated patients. All patients are alive and without evidence of leukemic transformation.

Published
2018

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