There is a new development in treating a typically untreatable and terminal nerve disease commonly known as Lorenzo's Oil.
The rare genetic disorder was highlighted in the 1992 movie Lorenzo's Oil, which told the story of Lorenzo Odone, a victim of the disease. In the quest for a treatment for his son, Lorenzo's father combined two fats extracted from olive and rapeseed oils – hence, the disease's common name. It impacts mostly boys, and typically they don't live long beyond age 10. Lorenzo lived to age 30.
The Child Neurology Society has released a study on the new technique that Dr. David Prentice of the Charlotte Lozier Institute says is exciting.
"[These researchers] took the patients' own adult stem cells from bone marrow," he explains, "and then they added the correct gene to these cells, grew up a nice big batch of these corrected cells, and then gave them back to these little boys."
Seventeen boys suffering the disease received the treatment, and 15 of them showed stabilization.
"That doesn't mean it's a cure," Prentice cautions. "We don't know how long it will last, but using the patient's own adult stem cells here to kind of carry this functional gene in is a very clever way to be able to get it there, to get it to stay and not have to worry about transplant rejection."
While it shows tremendous prospects for treating patients, it could be a while before it's widely available. Federal health authorities generally want scientists to monitor treatments for 15 years before assessing the technique and making a decision on additional patients.