Lorenzo's Oil Recommended for Genetic Disorder

NEW YORK (Reuters Health) - Treatment with Lorenzo's oil may help prevent neurologic abnormalities in boys with a rare genetic disorder called x-linked adrenoleukodystrophy (ALD), new research suggests. As such, the authors recommend that they receive this therapy.

Lorenzo's oil was invented in 1989 by Augusto Odone as a treatment for his son Lorenzo who had ALD. The oil contains unsaturated fatty acids that bind to an enzyme and prevent the build-up of very long-chain fatty acids, which plays a key role in ALD.

Treatment with Lorenzo's oil has been shown to reduce levels of these fatty acids, but it was unclear if such therapy provided actual clinical benefits. "After its introduction, there were a number of studies showing that the Oil was not particularly useful in symptomatic patients. So, the clinical community lost interest," lead author Dr. Hugo W. Moser, from the Kennedy Krieger Institute in Baltimore, told Reuters Health.

Instead of abandoning this therapy, the researchers hypothesized that Lorenzo's oil may be useful only if initiated during the earliest stages of the disease.

To test this, "we devised a method by which we can diagnose asymptomatic ALD patients," Moser noted. "We then used this method to screen the relatives of ALD patients and that's how we came up with the current cohort."

Moser's team assessed the outcomes of 89 asymptomatic boys with ALD who were treated with Lorenzo's oil and moderate fat restriction and were followed for about 7 years. The findings appear this month in Archives of Neurology.

Twenty-four percent of the subjects showed brain abnormalities on magnetic resonance imaging (MRI) and 11 percent developed MRI and neurologic abnormalities. "Sixty-four patients (74 percent) remained free of neurological involvement," the researchers report.

Consistent with previous reports, MRI abnormalities correlated with increases in very long-chain fatty acid levels, the authors state.

In an editorial, Dr. Raymond Ferri and Dr. Phillip F. Chance, from the University of Washington in Seattle, call the current study "remarkable," but note that it is often difficult to identify patients at an early asymptomatic stage. One possible way to increase identification of at-risk patients is through neonatal screening, they add.

"My main priority now is to develop a neonatal screening test for ALD," Moser said. "We know that plasma fatty acids are already increased on the day of birth in boys with ALD. We'd like to come up with a test that could" be incorporated into the existing neonatal assay that screens for 31 metabolic disorders, he added.

SOURCE: Archives of Neurology July 2005.