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Blood, 1 June 2005, Vol. 105, No. 11, pp. 4255-4257.
Prepublished online as a Blood First Edition Paper on February 1, 2005; DOI 10.1182/blood-2004-12-4837.
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GENE THERAPY
Brief report
Failure of SCID-X1 gene therapy in older patients
Adrian J. Thrasher,
Salima Hacein-Bey-Abina,
H. Bobby Gaspar,
Stephane Blanche,
E. Graham Davies,
Kathryn Parsley,
Kimberly Gilmour,
Douglas King,
Steven Howe,
Joanna Sinclair,
Christophe Hue,
Frédérique Carlier,
Christof von Kalle,
Geneviève de Saint Basile,
Françoise le Deist,
Alain Fischer, and
Marina Cavazzana-Calvo
From the Molecular Immunology Unit, Institute of Child Health, London, United Kingdom; Department of Biotherapy, Unité d'Immunologie et d'Hématologie Pédiatriques, Institut National de la Santé et de la Recherche Médicale (INSERM) Unit 429, and Laboratoire d'Immunologie Pédiatrique, Hôpital Necker, Paris, France; Immunology Unit, Great Ormond Street Hospital for Children, London, United Kingdom; and Cincinnati Children's Research Foundation, Cincinnati, OH.
Gene therapy has been shown to be a highly effective treatment for infants with typical X-linked severe combined immunodeficiency (SCID-X1,  c-deficiency). For patients in whom previous allogeneic transplantation has failed, and others with attenuated disease who may present later in life, the optimal treatment strategy in the absence of human leukocyte antigen (HLA)–matched donors is unclear. Here we report the failure of gene therapy in 2 such patients, despite effective gene transfer to bone marrow CD34+ cells, suggesting that there are intrinsic host-dependent restrictions to efficacy. In particular, there is likely to be a limitation to initiation of normal thymopoiesis, and we therefore suggest that intervention for these patients should be considered as early as possible.
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