Lorenzo's oil

Lorenzo's oil is a 4:1 mixture of glycerol trioleate and glycerol trierucate (the triglyceride forms of oleic and erucic acid), prepared from olive oil and rapeseed oil.[1] 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. Augusto received U.S. Patent No. 5,331,009[2] for the oil. The royalties he now receives are 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.

Lorenzo Odone died on May 30, 2008, after suffering from aspiration pneumonia, caused by food getting stuck in his lungs.[3][4]


Treatment costs

Lorenzo's oil costs approximately US$440 for a month's treatment.[citation needed] Most insurance companies in the United States will not pay for it since it is an Investigational New Drug.[citation needed]

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.[5]


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.[6]

Hugo Moser played a prominent role in both the treatment of Lorenzo Odone and the scientific evaluation of Lorezo'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.[7] Moser appraised Lorezo's oil again in a 2007 report.[8]

Moser's findings, that Lorenzo's oil did not help symptomatic ALD patients, are consistent with prior studies published in 2003[9] and 1999.[10][6]

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.[11] Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.[12]

Side Effects

The oil has been shown to cause a lowered platelet count.[13] Erucic acid is considered to be a toxin, and its presence in the food supply is regulated.[14][15]

Current state

In summary, 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.[7]

In the U.S., Lorenzo's oil is currently only available to patients taking part in a clinical trial at the Kennedy Krieger Institute.[citation needed] This trial was formerly held under the direction of Dr. Hugo Moser, until Moser's death in 2007. Moser's team, Dr. Gerald Raymond and Ann Moser continue the trial and his work with the leukodystrophies.

External links


  1. ^ Shankar Vedantam. "A Real-Life Sequel to 'Lorenzo's Oil' - washingtonpost.com". Washington Post 2007-01-28. pp. A01. http://www.washingtonpost.com/wp-dyn/content/article/2007/01/27/AR2007012701542_pf.html. Retrieved 2007-12-10. 
  2. ^ U.S. Patent 5,331,009: Pharmaceutical compositions for treating adrenoleukodystrophy. Issued July 19, 1994.
  3. ^ Lorenzo's Oil boy is dead at 30, BBC News; retrieved 2008-06-02.
  4. ^ Subject of 'Lorenzo's Oil' dies at 30, CNN; retrieved 2008-07-12
  5. ^ http://www.myelin.org/attachments/contentmanagers/11/scishow.swf
  6. ^ a b Berger J, Pujol A, Aubourg P, Forss-Petter S (July 2010). "Current and Future Pharmacological Treatment Strategies in X-Linked Adrenoleukodystrophy". Brain Pathol. 20 (4): 845–56. doi:10.1111/j.1750-3639.2010.00393.x. PMC 2967711. PMID 20626746. http://www.pubmedcentral.nih.gov/articlerender.fcgi?tool=pmcentrez&artid=2967711. 
  7. ^ a b Moser, HW; Raymond GV, Lu S-E, Muenz LR, Moser AB, Xu J, Jones RO, Loes DJ, Melhem ER, Dubey P, Bezman L, Brereton NH, Odone A (2005-07). "Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's Oil". Archives of Neurology 62 (7): p. 1073–80. doi:10.1001/archneur.62.7.1073. PMID 16009761. 
  8. ^ Moser HW, Moser AB, Hollandsworth K, Brereton NH, Raymond GV (September 2007). ""Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: rationale and current assessment of efficacy". J. Mol. Neurosci. 33 (1): 105–13. doi:10.1007/s12031-007-0041-4. PMID 17901554. 
  9. ^ Aubourg P, Adamsbaum C, Lavallard-Rousseau MC, Rocchiccioli F, Cartier N, Jambaqué I, Jakobezak C, Lemaitre A, Boureau F, Wolf C (September 1993). "A two-year trial of oleic and erucic acids ("Lorenzo's oil") as treatment for adrenomyeloneuropathy". N. Engl. J. Med. 329 (11): 745–52. doi:10.1056/NEJM199309093291101. PMID 8350883. 
  10. ^ van Geel BM, Assies J, Haverkort EB, Koelman JH, Verbeeten B, Wanders RJ, Barth PG (September 1999). "Progression of abnormalities in adrenomyeloneuropathy and neurologically asymptomatic X-linked adrenoleukodystrophy despite treatment with "Lorenzo's oil"". J. Neurol. Neurosurg. Psychiatr. 67 (3): 290–9. PMC 1736534. PMID 10449548. http://jnnp.bmj.com/cgi/pmidlookup?view=long&pmid=10449548. 
  11. ^ Poulos A, Gibson R, Sharp P, Beckman K, Grattan-Smith P (1994). "Very long chain fatty acids in X-linked adrenoleukodystrophy brain after treatment with Lorenzo's oil". Ann. Neurol. 36 (5): 741–6. doi:10.1002/ana.410360509. PMID 7979219. 
  12. ^ Magnhild Rasmussen, Ann B. Moser, Janet Borel, Surinder Khangoora and Hugo W. Moser (Aug, 1994). "Brain, liver, and adipose tissue erucic and very long chain fatty acid levels in adrenoleukodystrophy patients treated with glyceryl trierucate and trioleate oils (Lorenzo's Oil)". Neurochemical Research (Springer Netherlands) 19 (8): 1073–1082. doi:10.1007/BF00968719. PMID 800117. http://www.springerlink.com/content/j062517w8v068541. 
  13. ^ Crowther MA, Barr RD, Kelton J, Whelan D, Greenwald M (February 1995). "Profound thrombocytopenia complicating dietary erucic acid therapy for adrenoleukodystrophy". American Journal of Hematology 48 (2): 132–3. PMID 7847331. 
  14. ^ Food Standards Australia New Zealand (June 2003) Erucic acid in food : A Toxicological Review and Risk Assessment . Technical report series No. 21; Page 4 paragraph 1; ISBN 0 642 34526 0, ISSN 1448-3017
  15. ^ "Food Standards Agency - Agency issues warning on erucic acid". 2 September 2004. http://www.food.gov.uk/news/newsarchive/2004/sep/erucic. Retrieved 2007-11-02. 

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