Researchers have used gene therapy to treat boys with adrenoleukodystrophy, the New York Times reports.
Boston Children's Hospital's David Williams and his colleagues conducted a small study involving 17 boys with the disease. ALD is caused by a mutation in the ABCD1 gene, which resides on the X chromosome, and leads to a loss of nerve cells, neurological function, and death within a few years, though it can be treated, if caught early, with bone marrow transplants.
All of the participants in this study received the treatment, an infusion of autologous hematopoietic stem cells and a lentiviral vector containing ABCD1 cDNA. As reported in the New England Journal of Medicine this week, 15 of the 17 boys were alive and free of functional disability at a median 2.5 years after the infusion. One boy had major functional disabilities and the other withdrew from the study.
"To me, it seems to be working," Jim Wilson from the University of Pennsylvania, who was not involved in the study, tells the Times.
The therapy was developed by Bluebird Bio, and the company says it is speaking with regulators in both the US and Europe on seeking approval for the treatment, FierceBiotech adds.