FDA Approves Dysport for Pediatric Limb Spasticity
August 9, 2016
Key clinical point: Gene therapy using autologous hematopoietic stem cell transfers appears safe and effective for the treatment of cerebral adrenoleukodystrophy.
Major finding: Gene therapy involving transduced autologous hematopoietic stem cell transfers is associated with stabilization of lesion progression and no major functional disability in a majority of patients with cerebral adrenoleukodystrophy.
Data source: Open-label, phase 2-3, STARBEAM trial in 17 patients with early-stage cerebral adrenoleukodystrophy.
Disclosures: Bluebird Bio, the National Institutes of Health, the Patient-Centered Outcomes Research Institute, the Great Ormond Street Hospital, and the University College London Great Ormond Street Institute of Child Health Biomedical Research Centre supported the study. Nine authors declared financial ties to Bluebird Bio, and four authors were employees of Bluebird Bio. One author declared personal fees from a pharmaceutical company involved with lentiviral vectors and a patent related to enhanced lentiviral vector expression, and another two authors declared fees and funding from other pharmaceutical companies unrelated to the study.
These results suggest that transplantation with autologous hematopoietic stem cells transfected with Lenti-D is at least as effective as conventional allogeneic transplantation for the treatment of cerebral adrenoleukodystrophy, and it is possibly safer. Lenti-D therapy certainly has potential but some concerns remain. Careful surveillance to assess long-term outcomes is therefore essential.
For many years, gene therapy has shown great promise, but clinical applications have always seemed just beyond the horizon. Today, Lenti-D therapy appears to be poised as a real treatment option for cerebral adrenoleukodystrophy, and it might even become the first gene therapy approved by the Food and Drug Administration.
Marc Engelen, MD, PhD, is at the Academic Medical Center – Emma Children’s Hospital, Amsterdam. His comments are taken from an editorial (). Dr. Engelen declared grant support from Minoryx Therapeutics and personal fees from Vertex Pharmaceuticals, outside the submitted work.
Gene therapy using autologous hematopoietic stem cells appears safe and effective as a treatment for early-stage cerebral adrenoleukodystrophy, according to an interim analysis of results from the STARBEAM study.
X-linked adrenoleukodystrophy is characterized by a defect in the ABCD1 gene, leading to progressive demyelination that most commonly presents as learning and behavioral changes in boys aged 3-15 years. The only effective treatment to date has been allogeneic hematopoietic stem cell transplants, but these come with considerable risks, including graft-versus-host disease and graft failure.
August 9, 2016