Moser HW et al. (2005) Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil. Arch Neurol 62: 1073–1080

Asymptomatic patients with X-linked adrenoleukodystrophy (ALD) can improve their clinical outcome by consuming Lorenzo's oil, according to a recent prospective study by Moser et al. from the Kennedy Krieger Institute. Lorenzo's oil (4:1 glyceryl trioleate–glyceryl trierucate) has previously been shown to normalize plasma levels of fatty acids such as hexacosanoic acid (C26:0) within 4 weeks in most ALD patients, and it appears that the biochemical effect can translate into a clinical benefit.

The researchers followed the progress of 89 asymptomatic boys (mean age 4.8 years) with ALD, all of whom were treated with Lorenzo's oil and moderate fat restriction following measurement of baseline plasma C26:0 levels. Follow-up continued for an average of 6.9 years—neurological examination and brain MRI scans were undertaken at 6–12-month intervals, in addition to measurement of plasma C26:0 levels. A proportional hazards model was used to assess any association between C26:0 levels and development of neurological and MRI abnormalities.

Twenty-four percent of patients developed MRI scan abnormalities, and 9% developed both neurological and MRI abnormalities. Interestingly, abnormalities developed only in patients aged ≤7 years at baseline. There was a significant association between abnormal MRI scan development and increase in plasma C26:0 levels for patients of all ages.

Concluding that C26:0 reduction by Lorenzo's oil is associated with a reduced risk of MRI abnormality development, the authors recommend that asymptomatic boys with ALD who are at risk of developing the rapidly progressive cerebral ALD should be treated with Lorenzo's oil therapy.