Lorenzo's Oil

Scientific Name(s):Mixture of glyceryl trierucate, and glyceryl trioleate

Common Name(s): Lorenzo's oil , glyceryl trierucate with glyceryl trioleate , erucic acid , and oleic acid 1

Clinical Overview

Uses of Lorenzo's Oil

Lorenzo's oil has been used to treat certain rare genetic diseases such as adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN), without verified success.

Lorenzo's Oil Dosing

Lorenzo's oil is a combination of the triglycerides of erucic acid and oleic acid in a 1:4 ratio. Patients typically are placed on a low-fat diet; one clinical trial used oleic acid or glycerol trioleate at 1.7 g/kg and 0.3 g/kg of erucic acid or glycerol trierucate. Due to lack of clinical trial data, use is best avoided during pregnancy and lactation.

Contraindications

Lorenzo's oil should be avoided in patients who already have neurological deficits due to the side effect profile of the oil and likely neurological progression of the disease. Avoid use in patients with know hypersensitivity reactions to any component of the oil.

Pregnancy/Lactation

Information regarding safety and efficacy in pregnancy and lactation is lacking.

Lorenzo's Oil Interactions

None well documented.

Lorenzo's Oil Adverse Reactions

Purpura, petechiae, bleeding, and depression of natural killer cells have also been reported.

Toxicology

Thrombocytopenia and lymphocytopenia have been reported following treatment.

History

A widely publicized movie about the use of Lorenzo's oil to treat a devastating neurological syndrome catapulted this product into the public spotlight.

Chemistry

Lorenzo's oil is a combination of glyceryl trierucate (a component of erucic acid, a 22-carbon monounsaturated fatty acid) and glyceryl trioleate (a component of oleic acid, an 18-carbon monounsaturated fatty acid). These generally are combined in an approximate ratio of 1:4 (glycerol trierucate:glycerol trioleate) and have been used for the clinical evaluation of Lorenzo's oil.

Lorenzo's Oil Uses and Pharmacology

The oil has been touted for the treatment of a rare genetic disease known as adrenoleukodystrophy (ALD) when it appears in children, and adrenomyeloneuropathy (AMN) when it takes a more insidious path in adults. This X-linked recessive disorder is characterized by demyelination of cerebral nerves resulting in a variety of neurological symptoms including peripheral neuropathy, blindness, spastic tetraplegia, and death. 2 , 3

The primary metabolic abnormality in this disease is the accumulation of saturated very long chain fatty acids in body fluids and tissues, which occurs because of a genetically impaired ability to degrade them by normal oxidation. Hexacosanoic acid (C26:0) is the most consistently elevated fatty acid. In the absence of normal cellular oxidation of these fatty acids, the saturated very long chain fatty acids accumulate and are believed to be responsible for the neurological symptoms (ie, from demyelination within the CNS) associated with the disorder and with adrenocortical insufficiency and hypogonadism. 4

Theoretically, treatment of these diseases should involve reducing the level of saturated very long chain fatty acids present in the blood and nerves. In vitro, mono-unsaturated fatty acids have been shown to inhibit the synthesis of saturated very long chain fatty acids and to reduce their accumulation in cells obtained from patients affected by ALD. 2 Therefore, the saturated very long chain fatty acids that are believed to be toxic even at low concentrations appear to be exchanged for nontoxic mono-unsaturated fatty acids.

Research reveals no animal data regarding the use of Lorenzo's oil for adrenoleukodystrophy/adrenomyeloneuropathy treatment.

Results of clinical trials of patients with diets enriched with monounsaturated fatty acids, such as oleic acid, have shown a partial reduction in the plasma levels of saturated very long chain fatty acids. Peripheral nerve function has improved in some patients with AMN. 5

Although the addition of erucic acid to oleic acid (the combination used in Lorenzo's oil) was found to have led to complete normalization of plasma levels of very long chain fatty acids and promised to represent an even more effective therapy, 6 there is little to no evidence that this improves or delays progression of ALD or AMN. 4

Preliminary investigations of the efficacy of the oil mixture in the childhood form of the disease generally found no consistent effect, presumably because the fulminant childhood form did not permit sufficient time for the lipid-modifying effects of therapy. 2 Additionally, a case report described a 5-year-old boy who responded to 5 months of therapy with the oil mixture 7 as evidenced by an increased ability to swallow and an improvement in cerebral structure. In contrast, another case report described the failure of the oil to prevent clinical deterioration, and the child developed progressive visual loss and spastic tetraparesis despite dietary changes, steroid therapy, and gamma globulin treatment. 8

Well-designed studies of the oil suggested that treatment with Lorenzo's oil offers little evidence for a cessation or remission of the disease in adults.

In one study, in which 24 patients were treated for up to 48 months, the plasma levels of very long chain fatty acids declined to nearly normal within the first 10 weeks of treatment. However, none of the patients improved and 9 of 14 adult males had functional deterioration (eg, new cerebral lesions on MRI). Because of ethical considerations, this was not a placebo-controlled study. It is not clear if treatment may have slowed disease progression without resulting in a measurable improvement. 1

In an analogous but larger open trial of 108 patients, 9 treatment with the oil for up to 1 year demonstrated no improvement in nerve transmission as determined by assessment of visual evoked potentials. There was no correlation between the plasma levels of very long chain fatty acids and the rate of deterioration of visual function. Nerve transmission was examined in another study involving 8 patients on Lorenzo's oil dietary therapy for 3 years; Lorenzo's oil had no effect on modifying the natural course of the disease. 3 , 10 The disappointing results of dietary therapy for ALD may be associated with the failure of erucic acid to enter the brain. 11 , 12 However, therapy with lovastatin reduces plasma concentrations of very long chain fatty acids, particularly hexacosanoic acid (C26:0). 13

Dosage

Lorenzo's oil is a combination of the triglycerides of erucic acid and oleic acid in a 1:4 ratio. Patients typically are placed on a low-fat diet; one clinical trial used oleic acid or glycerol trioleate at 1.7 g/kg and 0.3 g/kg of erucic acid or glycerol trierucate.

Pregnancy/Lactation

Information regarding safety and efficacy in pregnancy and lactation is lacking.

Interactions

None well documented.

Adverse Reactions

Purpura, petechiae, bleeding, and depression of natural killer cells have also been reported. 14 , 15

Toxicology

Long-term hematological side effects must be considered. 16 , 17 Thrombocytopenia has been reported following treatment with Lorenzo's oil. 16 In one study, it was noted that the platelet count declined in 23 of 24 patients, but this was not correlated with plasma levels of erucic acid or other metabolites. None of these patients had abnormal bleeding or hematoma. Some of the patients had asymptomatic neutropenia. Dietary supplements of safflower and fish oils were given to the patients during this study, and while a 30% decrease in plasma docosahexaenoic acid levels occurred in all patients, none reported symptoms of essential fatty acid deficiency.

One author suggests avoiding therapy with Lorenzo's oil in patients who are already symptomatic, due to the side effect profile of the oil and because of the continued neurological progression of the disease in most patients treated with the oil. 18

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