Study proves Lorenzo's Oil prevents rare disease in boys

U.S. researchers have confirmed that a treatment called Lorenzo's Oil can prevent the onset of a rare but devastating neurological disease in young boys, X-linked adrenoleukodystrophy or X-ALD.

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

The oil was the subject of a movie in 1992 called 'Lorenzo's Oil'.

The researchers say 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, does actually work.

In their study 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.

The boys were age 7 or younger and had been identified as having the abnormal gene.

Over a period of seven years the boys got daily oral doses of the substance, and moderated what kinds of fat they ate.

The researchers found that subsequently, 74 percent of the boys showed no signs of disease progression.

X-ALD affects 16,000 patients in the United States; sufferers are unable to metabolize the 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 form is the childhood cerebral form, in which brain cells are destroyed, and this accounts for up to 40 percent of cases, which usually appear between 4 and 8 years of age.

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

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

Lorenzo's Oil appears to normalise the concentrations of the very long chain fatty acids.

The Odones say the treatment halted the progression of Lorenzo's disease and their son, now 27, is alive though severely disabled.

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

He says the 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.

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

The study is published in the Archives of Neurology.