Boston Children's Hospital’s Post

Two studies in The New England Journal of Medicine last year reported exciting long-term follow-up results of gene therapy for cerebral adrenoleukodystrophy (CALD), the degenerative neurological disease portrayed in the movie “Lorenzo’s Oil”: 80 percent of boys were free of major disabilities. But they also highlight a risk: About 10 percent of the boys developed cancer, and one passed away from complications of cancer treatment. Our care team is continuing to help families weigh the risks and benefits, while our researchers seek ways to improve safety. ⤵️