Thuốc Lorenzo’s Oil

Thuốc Lorenzo's Oil
Thuốc Lorenzo's Oil

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Common Name(s): Erucic acid, Glyceryl trierucate, Glyceryl trioleate, Lorenzo’s oil, Oleic acid

Medically reviewed by Last updated on Sep 18, 2019.

Clinical Overview


Open-label studies in asymptomatic boys (average age at diagnosis, 7 years) with negative magnetic resonance imaging (MRI) and in adults with “pure” adrenomyeloneuropathy (AMN) (ie, noncerebral) suggest that the administration of daily Lorenzo’s oil can prevent or slow the progression of the disease. However, there have been no controlled clinical trials.


Information is limited and dosage adjustment may be necessary to avoid adverse thrombocytopenia. Lorenzo’s oil 2 to 3 mL/day in children 8 years of age (average) has been used in a clinical study. Case studies in Zellweger syndrome report using Lorenzo’s oil 2 g/kg daily.


None well documented.


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


None well documented.

Adverse Reactions

Thrombocytopenia has been reported following treatment with Lorenzo’s oil.


Information is lacking.


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

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

Animal data

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

Clinical data

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

Limited case reports of patients with Zellweger syndrome exist with conflicting results.Arai 2008, Tanaka 2007


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.

Adverse Reactions

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

Purpura, petechiae, bleeding, and depression of natural killer cells have been reported.Chai 1996, Pour 2000 A case report of lipoid pneumonia exists.Majori 2014


Information is lacking.


Ahmed MA, Kartha RV, Brundage RC, et al. A model-based approach to assess the exposure-response relationship of Lorenzo’s oil in adrenoleukodystrophy. Br J Clin Pharmacol. 2016;81(6):1058-1066.26836218American Diabetes Association. Standards of medical care in diabetes–2014. Diabetes Care. 2014;37(suppl 1):S14-S80.24357209Arai Y, Kitamura Y, Hayashi M, Oshida K, Shimizu T, Yamashiro Y. Effect of dietary Lorenzo’s oil and docosahexaenoic acid treatment for Zellweger syndrome. Congenit Anom (Kyoto). 2008;48(4):180-182.18983586Basu C, Ahmed MA, Kartha RV, et al. A hierarchical Bayesian approach for combining pharmacokinetic/pharmacodynamic modeling and Phase IIa trial design in orphan drugs: Treating adrenoleukodystrophy with Lorenzo’s oil. J Biopharm Stat. 2016;26(6):1025-1039.27547896Chai BC, Etches WS, Stewart MW, Siminoski K. Bleeding in a patient taking Lorenzo’s oil: evidence for a vascular defect. Postgrad Med J. 1996;72(844):113-114.8871463Deon M, Garcia MP, Sitta A, et al. Hexacosanoic and docosanoic acids plasma levels in patients with cerebral childhood and asymptomatic X-linked adrenoleukodystrophy: Lorenzo’s oil effect. Metab Brain Dis. 2008;23(1):43-49.18026827Deon M, Wajner M, Sirtori LR, et al. The effect of Lorenzo’s oil on oxidative stress in X-linked adrenoleukodystrophy. J Neurol Sci. 2006;247(2):157-164.16750542Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Raymond GV. A phase III trial of Lorenzo’s Oil in adrenomyeloneuropathy. In: [Internet]. Bethesda (MD): National Library of Medicine (US). 2005-[cited 2010 Jun 7]. Available from: Accessed September 2017.Hudson Jones A. Medicine and the Movies: Lorenzo’s Oil at Century’s End. Ann Intern Med. 2000;133(7):567-571.11015184Majori M, Scarascia A, Anghinolfi M, et al. Lipoid pneumonia as a complication of Lorenzo’s oil therapy in a patient with adrenoleukodystrophy. J Bronchology Interv Pulmonol. 2014;21(3):271-273.24992138Making rare diseases a public-health and research priority. Lancet. 2008;371(9629):1972.18555895Moraes CT. Adrenoleukodystrophy and the mitochondrial connection: clues for supplementing Lorenzo’s oil. Brain. 2013;136(Pt 8):2339-2341.23842565Moser H, Dubey P, Fatemi A. Progress in X-linked adrenoleukodystrophy. Curr Opin Neurol. 2004;17(3):263-269.15167059Moser HW. Therapy of X-linked adrenoleukodystrophy. NeuroRx. 2006;3(2):246-253.16554262Moser HW, Mahmood A, Raymond GV. X-linked adrenoleukodystrophy. Nat Clin Pract Neurol. 2007;3(3):140-151.17342190Moser HW, Raymond GV, Dubey P. Adrenoleukodystrophy: new approaches to a neurodegenerative disease. JAMA. 2005;294(24):3131-3134.16380594Moser HW, Raymond GV, Lu SE, et al. Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo’s oil. Arch Neurol. 2005;62(7):1073-1080.16009761Pai GS, Khan M, Barbosa E, et al. Lovastatin therapy for X-linked adrenoleukodystrophy: clinical and biochemical observations on 12 patients. Mol Genet Metab. 2000;69(4):312-322.10870849Pour RB, Stöckler-Ipsiroglu S, Hunneman DH, et al. Enhanced lymphocyte proliferation in patients with adrenoleukodystrophy treated with erucic acid (22:1)-rich triglycerides. J Inherit Metab Dis. 2000;23(2):113-119.10801052Prieto Tenreiro A, Penacho Lázaro MÁ, Andrés Celda R, et al. Dietary treatment for X-linked adrenoleukodystrophy: is “Lorenzo’s oil” useful? Endocrinol Nutr. 2013;60(1):37-39.22622158Restuccia D, Di Lazzaro V, Valeriani M, et al. Neurophysiologic follow-up of long-term dietary treatment in adult-onset adrenoleukodystrophy. Neurology. 1999;52(4):810-816.10078732Rizzo WB. Lorenzo’s oil—hope and disappointment. N Engl J Med. 1993;329(11):801-802.8043042Sassa T, Wakashima T, Ohno Y, Kihara A. Lorenzo’s oil inhibits ELOVL1 and lowers the level of sphingomyelin with a saturated very long-chain fatty acid. J Lipid Res. 2014;55(3):524-530.24489110Semmler A, Köhler W, Jung HH, Weller M, Linnebank M. Therapy of X-linked adrenoleukodystrophy. Expert Rev Neurother. 2008;8(9):1367-1379.18759549Suzuki Y, Imamura A, Shimozawa N, Kondo N. The clinical course of childhood and adolescent adrenoleukodystrophy before and after Lorenzo’s oil. Brain Dev. 2001;23(1):30-33.11226726Tanaka K, Shimizu T, Ohtsuka Y, Yamashiro Y, Oshida K. Early dietary treatments with Lorenzo’s oil and docosahexaenoic acid for neurological development in a case with Zellweger syndrome. Brain Dev. 2007;29(9):586-589.17418516


This information relates to an herbal, vitamin, mineral or other dietary supplement. This product has not been reviewed by the FDA to determine whether it is safe or effective and is not subject to the quality standards and safety information collection standards that are applicable to most prescription drugs. This information should not be used to decide whether or not to take this product. This information does not endorse this product as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about this product. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this product. This information is not specific medical advice and does not replace information you receive from your health care provider. You should talk with your health care provider for complete information about the risks and benefits of using this product.

This product may adversely interact with certain health and medical conditions, other prescription and over-the-counter drugs, foods, or other dietary supplements. This product may be unsafe when used before surgery or other medical procedures. It is important to fully inform your doctor about the herbal, vitamins, mineral or any other supplements you are taking before any kind of surgery or medical procedure. With the exception of certain products that are generally recognized as safe in normal quantities, including use of folic acid and prenatal vitamins during pregnancy, this product has not been sufficiently studied to determine whether it is safe to use during pregnancy or nursing or by persons younger than 2 years of age.

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