The movie Lorenzo's Oil was based on a true story of a couple in Washington, who in a desperate attempt to save their son, Lorenzo, from a debilitating nerve disease, developed their own treatment for it, which they called "Lorenzo's oil."
The movie has inspired thousands of families affected by the disease, Adrenoleukodystrophy, or ALD, a rare genetic disorder that strikes mainly boys and, less frequently, young men. The victim progressively loses the ability to move, hear, speak and breathe.
Lorenzo Odone was 5 years old when he started showing symptoms.
Diagnosed with ALD, he was given two years to live. But his parents refused to accept the prognosis. They devoured neurology textbooks, learned complex biochemistry, and came up with their own therapy to prevent nerve damage: a mix that essentially consisted of olive oil and rape seed oil.
Since the movie was released 10 years ago, the scientific community has remained highly skeptical. Several small studies showed little, if any, benefit from Lorenzo's oil. Some doctors suggested the treatment was quackery.
But two teams of researchers, one led by Hugo Moser of the Kennedy Krieger Institute in Baltimore, Md., funded by the Food and Drug Administration, and another team based in Europe found that Lorenzo's oil is beneficial if given to children before they display any symptoms. It is the largest study of its kind.
Oil Prevented Symptoms From Developing
Children with a family history of the disease can get a blood test for ALD in the first few years of life. If those who test positive start taking daily doses of the oil, the study found, they were 66 percent less likely to develop symptoms or any visible changes in the brain.
"In adrenoleukodystrophy there is a buildup of unusual fats in the body that eventually lead to harm," said Dr. Gerald Raymond of the Kennedy Krieger Institute. "They don't cause problems right away but over time they lead to problems in the nervous system. Lorenzo's oil prevents the buildup of these fats."
Michael Benton was diagnosed with ALD when he was 7, before he showed any symptoms. "We were looking at total debilitation: inability to eat, inability to speak, walk," said Michael's mother, Patty. "It was a very negative prognosis."
Benton took two swigs of the oil daily throughout elementary school, high school and college. He's now 22, and still taking the oil.
"I feel great," Michael Benton told ABCNEWS. "I'm very active. I like to ride my bike, go down to the beach, play volleyball, go surfing."
Lorenzo Odone has not been helped as much by the oil bearing his name because he didn't start taking it until after symptoms developed. Today he is unable to walk and is being cared for at home.
How long it will continue to help patients such as Benton is not known, but this simple treatment is giving boys with ALD the chance to become healthy young men.