Gene Therapy Promises Breakthrough for Boys with Lorenzo’s Oil Disease

Published Date : Oct 05, 2017

Gene therapy has been seemed to achieve success as an experimental treatment for boys inheriting a nerve disease that featured in the “Lorenzo’s Oil” movie. For a disease that commonly kills and leads to a swift decline within a decade, the achievement of having no major disability in 15 of the 17 boys treated in a study has been remarkable. New York couple Paul and Liliana Rojas could find it a bittersweet news as one son has received the treatment and the other has not. Their father has explained that the eldest son was way far along in the disease to be qualified for the study, but thankfully, the younger was not.

The disease is caused by a single gene and occurs in one in every 20,000 boys worldwide. It just about exclusively attacks boys having only a single X chromosome copy, which bears the gene that causes the disease.

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The New England Journal of Medicine had published the study’s results Wednesday, which have been discussed at a Kansas City, Missouri medical conference. Cambridge, Massachusetts therapy maker, Bluebird Bio, Inc. has paid for the study with some support from government grants. The U.S. witnesses the sale of just one gene therapy, an August approved leukemia treatment, whereas a handful are sold elsewhere.

As a matter of fact, Lorenzo’s oil has not proved to be a cure. Stem cell transplant has been considered to be the only effective treatment. However, just about one in five boys affected with the disease have a donor required to perform the transplant. Gene therapy, on the other hand, helps such boys to serve as their own donors, and could be “possible safer” than and “as good as stem cell transplant,” as Emma Children’s Hospital’s Dr. Marc Engelen commented in the journal.