New gene therapy for cerebral adrenoleukodystrophy


Eichler et al. recently published an article entitled "Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy" in New England Journal of Medicine ( They enrolled boys with cerebral adrenoleukodystrophy who have early-stage disease and gadolinium enhancement on magnetic resonance imaging at screening. They performed an investigational therapy with the infusion of autologous CD34+ cells transduced with the elivaldogene tavalentivec lentiviral vector. Patients were assessed for the occurrence of graft-versus-host disease, death, and major functional disabilities, as well as changes in neurologic function and in the extent of lesions after the therapy. They conlcuded that such gene therapy may be a safe and effective alternative to allogeneic stem-cell transplantation in boys with early-stage cerebral adrenoleukodystrophy.