In 1989, one of the authors of this study, Augusto Odone, pioneered a treatment (Lorenzo's oil), which was shown to normalise the levels of saturated very long-chain fatty acids within four weeks in most patients with ALD.

Previous clinical trials led to the conclusion that Lorenzo's oil did not alter the rate of progression of the disease in patients who already had neurological symptoms. Hugo W. Moser, M.D., of the Kennedy Kreiger Institute, Baltimore, and colleagues treated 89 boys with ALD who had no neurological symptoms and normal brain MRIs with moderate dietary fat restriction and Lorenzo's oil between 1989 and 2002.

Sixty-four of the patients were younger than seven years old when they began treatment and all were followed up for an average of approximately seven years. Because of the devastating nature of cerebral ALD, and the hope that the striking reduction of very long chain fatty acid levels would lead to clinical benefit, none of the boys were given placebo. Fatty acids blood levels were assessed every month for the first six months after enrolment in the study and every three to six months thereafter. Neurological examinations and MRIs were scheduled every six to 12 months.

Sixty-six patients (74 percent) were well at last follow-up. Twenty-one patients (24 percent) developed MRI abnormalities and 10 patients (11 percent) developed neurological abnormalities. The researchers found a significant association between the development of MRI abnormalities and an increase in the levels of the saturated very long chain fatty acid C26:0.

"Patients who had a neurological abnormality had significantly higher weighted average C26:0 levels than those who did not have an abnormality, suggesting that an LO-induced decrease in the C26:0 level could protect against the inflammatory cerebral disease,” the authors report.; Source: American Medical Association (AMA)