bluebird bio Announces Publication of Interim Data from Starbeam Study of Lenti-DTM Drug Product in Patients with Cerebral Adrenoleukodystrophy (CALD) in The New England Journal of Medicine
bluebird bio Announces Publication of Interim Data from Starbeam Study of Lenti-D TM Drug Product in Patients with Cerebral Adrenoleukodystrophy (CALD) in The New England Journal of Medicine
– Additional follow-up data to be included in poster presentation at Child Neurology Society (CNS) Annual Meeting –
– 15/17 (88%) of the patients infused with Lenti-D drug product remain alive and free of major functional disabilities (MFDs), the primary efficacy endpoint of the trial –
– No engraftment failure, graft versus host disease (GVHD) or life-threatening infections occurred; no evidence of insertional oncogenesis –
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational gene therapy in boys with cerebral adrenoleukodystrophy (CALD) were published in the New England Journal of Medicine (NEJM).
“I have seen firsthand the devastation that CALD can inflict on these young boys and their families,” said David A. Williams, M.D., chief scientific officer and senior vice-president for research at Boston Children’s Hospital and president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and the principal investigator of the Starbeam study. “However, when cerebral disease is detected early, it is possible to slow or stop the progression of disease. Currently, allogeneic hematopoietic stem cell transplant is the only available therapy – but one that presents challenges for patients without a matched sibling donor. These data suggest that Lenti-D may also be a viable option for patients, and one that, being autologous, could potentially overcome some of the challenges associated with allogeneic stem cell transplantation. The results of this study represent the continued development of effective gene therapy approaches to human disease treatments.”
The New England Journal of Medicine: Hematopoietic Stem Cell Gene Therapy for Cerebral Adrenoleukodystrophy
The Starbeam Study is a global, multi-center study assessing the efficacy and safety of an investigational gene therapy in boys up to 17 years of age with CALD. As of the April 25, 2017 data cut off for the publication, 16 of the 17 patients had completed the primary analysis period (24 months or discontinuation).