Common Name(s): Erucic acid, Glyceryl trierucate, Glyceryl trioleate, Lorenzo's oil, Oleic acid
A widely publicized 1992 film (Lorenzo's Oil) about a triglyceride mixture used in the treatment of adrenoleukodystrophy (ALD) made the general public aware of the product.Hudson 2000
Lorenzo's oil is a combination of glyceryl trierucate (an ester of erucic acid, a 22-carbon monounsaturated fatty acid) and glyceryl trioleate (an ester of oleic acid, an 18-carbon monounsaturated fatty acid), which are generally combined in an approximate ratio of 1:4 (glycerol trierucate:glycerol trioleate) when used for clinical evaluation.Moser 2004
Uses and Pharmacology
As a component of medical nutrition therapy for patients with type 2 diabetes, the American Diabetes Association Standards of Care (2014) recommend higher quality dietary fat intake, as an alternative to decreased fat intake, by replacing saturated and/or trans fats with mono- and poly-unsaturated fatty acids in the diet. This Mediterranean-style approach to eating may improve glycemic control and cardiovascular disease risk factors (moderate-quality evidence).ADA 2014
Peroxisomal disorders are a group of inherited metabolic disorders that result in demyelination, progressive brain damage, and premature death.
In ALD, a deficiency in a transporter protein results in a lack of the enzyme required to oxidize very long chain fatty acids (VLCFA) with a consequent accumulation of these fatty acids in body fluids and tissues. This X-linked recessive disorder is characterized by demyelination of cerebral nerves resulting in a variety of neurological symptoms, including peripheral neuropathy, blindness, and spastic tetraplegia. The disease is also associated with adrenocortical insufficiency and hypogonadism.Moser 2004, Moser 2005, Moser 2006 Treatment includes adrenal steroid replacement, bone marrow transplantation, and Lorenzo's oil. Alternative therapies based largely on animal studies include lovastatin, phenylbutyrate, arginine butyrate, and gene therapy.Moser 2004, Pai 2000
Distinct forms of ALD exist. The most common form of ALD is adrenomyeloneuropathy (AMN), a slowly progressing, non-inflammatory form occurs mostly in adults. Childhood cerebral ALD, another of the most common forms, progresses rapidly, while adolescent cerebral ALD affects a small number of patients and progresses more slowly. AMN is estimated to affect about 1 in 40,000 men and 1 in 30,000 women Clinical Trials 2017; while the incidence of cerebral ALD is estimated at 1 in 17,000.Moser 2005 Addison disease can be an early indicator of ALD, and screening for ALD is recommended.Moser 2006
Zellweger syndrome is a related disorder in which an absence of peroxisomes in the kidney, liver, and brain results in the accumulation of VLCFA with consequent damage to these organs.Arai 2008
In vitro studies (using cells obtained from patients affected by ALD) and animal studies show that mono-unsaturated fatty acids inhibit the synthesis of toxic, saturated VLCFA and reduce their accumulation in cells.Moser 2004, Moser 2006, Rizzo 1993 Researchers evaluating the biochemical responses of Lorenzo's oil have shown an absence of effect on oxidative stress processes and positive effects on plasma levels of hexacosanoic acid and other long-chain fatty acids.Deon 2006, Deon 2008 Mitochondrial abnormalities are being evaluated with a potential role for coenzyme Q10.Rare Diseases 2008
Reviews of ALD and AMN studies are becoming more consistent in their recommendations about the place of Lorenzo's oil in therapy, although the number of researchers in this field is limited.Ahmed 2016, Moraes 2013, Moser 2006, Prieto 2013, Sasso 2014, Semmler 2008 A few long-term studies (up to 13 years) are available.Moser 2006, Moser 2005, Restuccia 1999
Open-label studies in asymptomatic boys (average age at diagnosis, 7 years) with a negative MRI, and in adults with "pure" AMN (ie, noncerebral), suggest that the daily administration of Lorenzo's oil can prevent or slow the progression of the disease.Deon 2008, Moser 2006, Moser 2007 Single-group trials consistently show a lack of effect in disease progression among those patients who are already symptomatic.Moser 2006, Semmler 2008, Suzuki 2001
Double-blind, placebo-controlled, randomized clinical trials are lacking; a study meeting these criteria to investigate the effect of Lorenzo's oil in pure AMN was terminated by the data and safety monitoring board.Clinical Trials 2017 Alternative methods of modelling data in order to conduct Phase II clinical trials in rare conditions such as adrenoleukodystrophy have been suggested.Basu 2016
Information is limited, and dosage adjustment may be necessary to avoid adverse thrombocytopenia.Moser 2006 Lorenzo's oil 2 to 3 mL per day in children 8 years of age (average) has been used in a clinical study.Deon 2008 Case studies in Zellweger syndrome have reported using Lorenzo's oil 2 g/kg daily.Arai 2008
Pregnancy / Lactation
Information regarding safety and efficacy in pregnancy and lactation is lacking.
None well documented.
Thrombocytopenia has been reported following treatment with Lorenzo's oil, but the condition can be managed by a dosage adjustment. No evidence of abnormal bleeding has been reported.Moser 2006
Information is lacking.
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