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Gene Therapy in a Patient with Sickle Cell Disease. 2017
Jean-Antoine Ribeil, and
Salima Hacein-Bey-Abina, and
Emmanuel Payen, and
Alessandra Magnani, and
Michaela Semeraro, and
Elisa Magrin, and
Laure Caccavelli, and
Benedicte Neven, and
Philippe Bourget, and
Wassim El Nemer, and
Pablo Bartolucci, and
Leslie Weber, and
Hervé Puy, and
Jean-François Meritet, and
David Grevent, and
Yves Beuzard, and
Stany Chrétien, and
Thibaud Lefebvre, and
Robert W Ross, and
Olivier Negre, and
Gabor Veres, and
Laura Sandler, and
Sandeep Soni, and
Mariane de Montalembert, and
Stéphane Blanche, and
Philippe Leboulch, and
Marina Cavazzana
From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unité de Technologies Chimiques et Biologiques pour la Santé, Centre National de la Recherche Scientifique Unité Mixte de Recherche 8258, INSERM Unité 1022, Faculté de Pharmacie de Paris, Université Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, Hôpital Kremlin-Bicêtre, AP-HP, Le Kremlin-Bicêtre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative Therapies, Imagine Institute, Université Paris Descartes, Sorbonne Paris Cité University (M.S., B.N., L.W., M.C.), Mère-Enfant Clinical Investigation Center, Groupe Hospitalier Necker Cochin (M.S.), Université Paris Diderot, Sorbonne Paris Cité University, INSERM Institut National de Transfusion Sanguine, Unité Biologie Intégrée du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and Laboratoires de Virologie, Hôpital Cochin (J.-F.M.), Paris, Atomic and Alternative Energy Commission, Université Paris-Sud, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Institut Mondor de Recherche Biomédicale, Equipe 2, Centre de Référence des Syndromes Drépanocytaires Majeurs, Centre Hospitalier Universitaire Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Créteil (P. Bartolucci), and Université Paris Diderot, Sorbonne Paris Cité University, INSERM Unité 1149, Hôpital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P., T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (P.L.).
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addition of an antisickling β-globin gene into autologous hematopoietic stem cells. Adverse events were consistent with busulfan conditioning. Fifteen months after treatment, the level of therapeutic antisickling β-globin remained high (approximately 50% of β-like-globin chains) without recurrence of sickle crises and with correction of the biologic hallmarks of the disease. (Funded by Bluebird Bio and others; HGB-205 ClinicalTrials.gov number, NCT02151526 .).
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