The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie “Lorenzo’s Oil.”
Fifteen of the 17 boys treated in a study had no major disability two years later. Doctors say that is remarkable for a disease that often causes swift decline and kills within a decade.
Until now, the only effective treatment has been a stem cell transplant from a sibling, but few patients have a suitable donor. The experimental therapy, made by Bluebird Bio, may help kids who lack a donor.
Study results were published Wednesday by the New England Journal of Medicine and discussed at a medical conference in Kansas City, Missouri.