Lorenzo’s oil is 4 parts of glyceryl trioleate and 1 part glyceryl trierucate, which are the triacylglycerol forms of oleic acid and erucic acid and are prepared from olive oil and rapeseed oil. It is used in the investigational treatment of asymptomatic patients with 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, with the actual production initially developed by retired British scientist Don Suddaby (formerly of Croda International). Suddaby and his colleague, Keith Coupland, received U.S. Patent No. 5,331,009 for the oil. The royalties received by Augusto were 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 film Lorenzo’s Oil.
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Lorenzo Odone died on May 30, 2008, at the age of 30, after suffering from aspiration pneumonia, caused by food getting stuck in his lungs.
Treatment costs
Lorenzo's oil costs approximately US $440 for a month's treatment.
In the U.S., Lorenzo's oil is currently available to only the patients taking part in a clinical trial under the direction of the Kennedy Krieger Institute. Thus, Lorenzo's oil can be obtained only through prescription by Kennedy Krieger Institute-authorized physicians. A 500-ml bottle costs approximately $56.00. Some insurance companies will provide coverage for the oil, but others do not because it is still considered an experimental drug by the Food and Drug Administration (FDA).
Proposed mechanism of action
The mixture of fatty acids purportedly reduces the levels of very long chain fatty acids (VLCFAs), which are elevated in ALD. It does so by competitively inhibiting the enzyme that forms VLCFAs.
Effectiveness
Lorenzo's oil, in combination with a diet low in VLCFA, has been investigated for its possible effects on the progression of ALD. Clinical results have been mixed and the use of Lorenzo's oil has been controversial due to uncertainties regarding its clinical efficacy and the clinical indications for its use.
Hugo Moser played a prominent role in both the treatment of Lorenzo Odone and the scientific evaluation of Lorenzo's oil. In 2005, Moser published a controlled study concluding that Lorenzo's oil does not alter the course of the illness in symptomatic patients, but asymptomatic patients had a reduced risk of developing ALD while on the dietary therapy. Moser appraised Lorenzo's oil again in a 2007 report.
Moser's findings, that Lorenzo's oil did not help symptomatic ALD patients, are consistent with prior studies published in 2003 and 1999.
A study by Poulos published in 1994 found that Lorenzo's oil is of limited value in correcting the accumulation of saturated VLCFAs in the brain of patients with ALD. Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.
Side effects
The oil has been shown to cause a lowered platelet count, which can lead to thrombocytopenia and lymphopenia.
There are no reports of toxicity from dietary consumption of erucic acid.
Current state
Dietary manipulation using Lorenzo's oil has been shown to lower blood levels of very-long-chain fatty acids, but it is ineffective in symptomatic ALD. However, studies by Dr. Hugo Moser have found evidence that use of the oil by asymptomatic patients may slightly delay the onset of symptoms.