Lenti-D Hematopoietic Stem Cell Gene Therapy to Arrest Progression of Cerebral Adrenoleukodystrophy: Interim Results of an International Phase 2/3 Trial
Year: 2018
Type: article
Study design: Clinical Trial
Abstract: In X-linked adrenoleukodystrophy (ALD), mutations in ABCD1 lead to loss of function of the peroxisomal membrane half-transporter ALD protein and toxic accumulation of very long chain fatty acids. Cerebral ALD (CALD) is characterized by demyelination and neurodegeneration with generally fatal outcome. Disease progression, leading to loss of neurologic function and death, can only be halted with allogeneic hematopoietic stem cell transplantation (allo-HSCT). We enrolled boys ≤ 17 years of age in STARBEAM, a single-arm, open-label, phase 2/3 study of the safety and efficacy of Lenti-DTM gene therapy for CALD.
Authors: Christine Duncan, Florian Eichler, Patricia L. Musolino, Paul J. Orchard, Satiro De Oliveira, Adrian J. Thrasher, Myriam A. Armant, Colleen Dansereau, Troy C. Lund, Weston P. Miller, Gerald V. Raymond, Raman Sankar, Ami J. Shah, Caroline Sevin, H. Bobby Gaspar, Paul Gissen, Hernan Amartino, Drago Bratkovic, Nicholas J.C. Smith, Asif M. Paker, Esther Shamir, Tara O'Meara, Mohammed Asmal, David Davidson, Patrick Aubourg, David A. Williams
Institutions: Dana-Farber Cancer Institute, Massachusetts General Hospital, University of Minnesota, University of California, Los Angeles, University College London, Boston Children's Hospital, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Lucile Packard Children's Hospital, Stanford University, Inserm, MRC Laboratory for Molecular Cell Biology, Hospital Universitario Austral, South Australia Pathology, Women's and Children's Hospital, Bluebird Bio (United States), Cincinnati Children's Hospital Medical Center
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2791502543