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Composition
Oleo-erucic acid (Lorenzo's oil) is a 4:1 mixture of oleic and erucic acid extracted from olive oil and rapeseed oil respectively. It has been used in the preventative treatment of adrenoleukodystrophy (ALD). The oil was formulated by Augusto Odone, father of Lorenzo Odone. He received US Patent No. 5,331,009 for the oil. What royalties he now gets are paid to the Myelin Project which he founded to further research treatments for ALD and similar disorders. This therapy obtained widespread publicity in 1992 because of the movie Lorenzo's Oil.
Cost and reimbursement
It is expensive and is sold for US $ 440 for around 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 short chain 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 efficiency is lessened once symptoms start.
Studies
In an early study, Duchesne studied 8 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 in indicated that there was only a slight but statistically significant slowing of clinical progression and delay of death.
However 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, 5 symptomatic heterozygous women, and 5 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 although some parents of ALD patients feel otherwise. In addition recent studies by Dr. Hugo Moser have found there is evidence that use of the oil by asymptomatic patients may delay the onset of symptons significantly.
In the U.S., Lorenzo’s Oil is currently only available to patients taking part in a clinical trial under the direction of Dr. Hugo Moser of the Kennedy Krieger Institute.
References
"Lorenzo's oil"