• E-mail
• Print
• Comment
• Font Size
• Digg
• del.icio.us
• Discuss article

Lorenzo's Oil Prevents Rare Disease in Boys

Posted on: Tuesday, 12 July 2005, 06:45 CDT

WASHINGTON -- A treatment called Lorenzo's Oil can prevent the onset of a rare but devastating neurological disease in young boys, U.S. researchers reported on Monday.

The study is the first to use validated scientific methods to test whether the substance, a combination of two fats extracted from olive oil and canola or rapeseed oil, actually works, the researchers said.

They tested the oil on 89 boys who had been diagnosed with X-linked adrenoleukodystrophy or X-ALD, but who had not yet begun to show symptoms.

Writing in the Archives of Neurology, Dr. Hugo Moser of the Kennedy Krieger Institute in Baltimore and colleagues said it prevented symptoms from ever beginning in most of the boys.

"This clinical study clearly demonstrates that the use of Lorenzo's Oil can prevent the onset of the rapidly progressive and devastating form of the brain disease that affects 50 percent of boys with X-ALD," Moser said in a statement.

His team tested 89 boys age 7 or younger who had an abnormal gene that identifies X-ALD.

For seven years the boys got daily oral doses of the substance, the basis for the 1992 movie "Lorenzo's Oil," and moderated what kinds of fat they ate.

Subsequently, 74 percent of the boys showed no signs of disease progression, the researchers found.

"The results of this trial offer clinical support for treating the many young boys identified at high-risk for the disease," said Dr. Gary Goldstein, chief executive officer of the Kennedy Krieger Institute.

X-ALD affects 16,000 patients in the United States.

Patients cannot metabolize big fat molecules called long-chain fatty acids -- either made inside the body or eaten in food. The condition causes the breakdown of myelin, the fatty substance that coats and insulates nerve fibers.

The most dangerous type is the childhood cerebral form, in which brain cells are destroyed. Up to 40 percent of cases are this type, which usually appears between 4 and 8 years of age.

Symptoms include a loss of the ability to speak, reduced strength and coordination and, eventually, complete breakdown of bodily function and death.

There is no cure, but potential treatments include the cholesterol drug lovastatin and bone marrow transplants.

Then there is Lorenzo's Oil, a treatment invented by Augusto and Michaela Odone after their son, Lorenzo, was diagnosed with ALD.

Lorenzo's Oil normalizes the concentrations of very long chain fatty acids. The Odones said the treatment stopped the progression of Lorenzo's disease and their son, now 27, is alive though severely disabled.

More News in this Category