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Lorenzo’s Oil Effective Treatment for Genetic Disorder
Reported July 12, 2005

(Ivanhoe Newswire) -- A new study gives hope for patients with a debilitating genetic disorder. Certain patients with adrenoleukodystrophy (ALD) could benefit from a treatment called Lorenzo's oil.

ALD patients accumulate high levels of saturated, very long-chain fatty acids in their brains. Patients with ALD progress differently. For example, those with rapidly progressive cerebral ALD (CERALD) typically are diagnosed between ages four and eight and progress rapidly to total disability within a few years. An adult form of this disease progresses slowly and is far less disabling.

In 1989, a new treatment called Lorenzo's oil was developed to normalize the levels of saturated very long-chain fatty acids within four weeks in most patients with ALD. But clinical trials showed the treatment did not change the rate of progression of the disease in patients who already had neurological symptoms.

The new study was done at the Kennedy Kreiger Institute in Baltimore. Researchers treated, 89 boys with ALD who had no neurological symptoms with Lorenzo's oil. Most of the boys were under age 7. They were followed for about seven years. Fatty acids blood levels were checked throughout the study, and MRIs were also done every six to 12 months.

Researchers report 74 percent of the patients were doing well at the final follow-up. The patients who did show MRI abnormalities also had an increase in the levels of the saturated very long chain fatty acid C26:0. This suggests Lorenzo's oil decreased the C26:0 level to protect against cerebral disease. Researchers recommend that Lorenzo's oil be offered to boys with ALD who are neurologically asymptomatic, have normal brain MRI results and are at risk of developing CERALD. They say patients younger than 7 years old are prime candidates for this treatment.

In an accompanying editorial, doctors from the University of Washington, Seattle, point out successful implementation of this recommended treatment means identifying children who are at risk. They say neonatal screening would help in identifying more at-risk patients at a very early age.

SOURCE: Archives of Neurology, 2005;62:1073-1080