Lorenzo's oil

Lorenzo's oil is a 4:1 mixture of glycerol trioleate and glycerol trierucate (the triglyceride forms of oleic and erucic acid) used in the preventative treatment of adrenoleukodystrophy (ALD). The oil was formulated by Augusto and Michaela Odone after their son Lorenzo was diagnosed with the disease in 1984, at the age of five. Augusto received U.S. Patent No. 5,331,009 for the oil. The royalties he now receives are paid to the Myelin Project which he and Michaela founded to further research treatments for ALD and similar disorders. The Odones and their invention obtained widespread publicity in 1992 because of the movie Lorenzo's Oil.

Cost and reimbursement
Lorenzo's oil is expensive, costing approximately US$440 for a month's treatment. Most insurance companies will not pay for it since it is considered an experimental treatment by the FDA.

Mechanism of action
This mixture of fatty acids reduces the levels of very long chain fatty acids (VLCFA) known to cause ALD. It does so by competitively inhibiting the enzyme that forms VLCFAs.

Effectiveness
Lorenzo's oil has not been clinically proven to be effective against the progression of ALD. Of five published studies, four have failed to demonstrate benefit in symptomatic patients. It does, however, reduce the probability of developing symptoms if its administration is started before they begin. Its efficacy is lessened once symptoms start.

Studies
In an early study, Duchesne studied eight males and reported improvements in follow-up magnetic resonance imaging of the brain.

In the only clinical study with positive findings, Moser tried Lorenzo's oil in 53 asymptomatic patients with ALD. Analysis of the effect of the oil indicated that there was only a slight but statistically significant slowing of clinical progression and delay of death.

A controlled study by Moser concluded that Lorenzo's oil does not alter the course of the illness in symptomatic patients; however, dietary therapy started before the development of symptoms may reduce the frequency and severity of subsequent neurological disability.

A study by Poulos found that Lorenzo's oil is of limited value in correcting the accumulation of saturated VLCFAs in the brain of patients with ALD.

In the best study so far, Aubourg reported in the New England Journal of Medicine the results of an open trial of treatment with Lorenzo's oil in 14 men with adrenomyeloneuropathy, five symptomatic heterozygous women, and five boys (mean age, 13 years) with preclinical ALD. Over a mean follow-up of 33 months, none of the 14 men with ALD improved, one of the five asymptomatic boys developed signs of myelopathy, and there were no changes in the symptomatic heterozygous women. The authors concluded that this open trial found no evidence of a clinically relevant benefit from dietary treatment with Lorenzo's oil in patients with ALD.

Current state
In summary, although dietary manipulation using Lorenzo's oil has been shown to change blood test results, it appears to be ineffective for the symptomatic ALD even though some parents of ALD patients feel otherwise. However, recent studies by Dr. Hugo Moser have found evidence that use of the oil by asymptomatic patients may delay the onset of symptoms significantly.

In the U.S., Lorenzo’s oil is currently only available to patients taking part in a clinical trial at the Kennedy Krieger Institute. This trial was formerly held under the direction of Dr. Hugo Moser, until Moser's death in 2007. Moser's team continues the trial.