Lorenzo's oil

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For the film, see Lorenzo's Oil (film).

Lorenzo's oil is a 4:1 mixture of glycerol trioleate and glycerol trierucate (the triglyceride forms of oleic and erucic acid) used in the preventive treatment of 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 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.[1]

Contents

[edit] Cost and reimbursement

Lorenzo's oil costs approximately US$440 for a month's treatment. Most insurance companies will not pay for it since it is considered an experimental treatment by the FDA.

[edit] Mechanism of action

This mixture of fatty acids reduces the levels of very long chain fatty acids (VLCFA) known to cause ALD. It does so by competitively inhibiting the enzyme that forms VLCFAs.

[edit] Effectiveness

Main article: Adrenoleukodystrophy#Treatment

Lorenzo's oil, in combination with a diet low in VLCFA, has been used with limited success, especially before disease symptoms appear. A 2005 study shows positive long-term results with this approach.[2] A 2007 report also appraises "Lorenzo's oil".[3] See also the Myelin Project.[4]

[edit] Studies

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Please help improve this article by adding reliable references. Unsourced material may be challenged and removed. (December 2007)

In an early study, Duchesne studied eight males and reported improvements in follow-up magnetic resonance imaging of the brain.

In the only clinical study with positive findings, Moser tried Lorenzo's oil in 53 asymptomatic patients with ALD. Analysis of the effect of Lorenzo's oil indicated that there was only a slight but statistically significant slowing of clinical progression and delay of death.

A controlled study by Moser concluded that Lorenzo's oil does not alter the course of the illness in symptomatic patients; however, dietary therapy started before the development of symptoms may reduce the frequency and severity of subsequent neurological disability.

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.[5]

Patrick Aubourg reported in the New England Journal of Medicine the results of an open trial of treatment with Lorenzo's oil in 14 men with adrenomyeloneuropathy, five symptomatic heterozygous women, and five boys (mean age, 13 years) with preclinical ALD. Over a mean follow-up of 33 months, none of the 14 men with ALD improved, one of the five asymptomatic boys developed signs of myelopathy, and there were no changes in the symptomatic heterozygous women. The authors concluded that this open trial found no evidence of a clinically relevant benefit from dietary treatment with Lorenzo's oil in patients with ALD.[6]

A 2005 study by Moser et al in the journal Archives of Neurology examined the effectiveness of Lorenzo's oil and moderate fat restriction on 89 asymptomatic boys at risk for ALD. After 7 years of treatment 24% of the boys developed MRI abnormalities and 11% developed both neurological and MRI abnormalities. The study demonstrated a significant association between the development of MRI abnormalities and a plasma hexacosanoic acid increase. The researchers concluded that Lorenzo's oil therapy be given to asymptomatic boys with X-linked adrenoleukodystophy who have normal brain MRI results to delay the onset of disease [7]. Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.[8]

[edit] Current state

In summary, although dietary manipulation using Lorenzo's oil has been shown to change blood test results, it appears to be ineffective for the symptomatic ALD even though some parents of ALD patients feel otherwise. However, recent studies by Dr. Hugo Moser have found evidence that use of the oil by asymptomatic patients may delay the onset of symptoms significantly.

In the U.S., Lorenzo’s oil is currently only available to patients taking part in a clinical trial at the Kennedy Krieger Institute. This trial was formerly held under the direction of Dr. Hugo Moser, until Moser's death in 2007.[9] Moser's team, Dr. Gerald Raymond and Ann Moser continue the trial and his work with the leukodystrophies.

[edit] External links

[edit] References

  1. ^ Lorenzo's Oil boy is dead at 30, BBC News; retrieved 2008-06-02.
  2. ^ 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. 
  3. ^ Moser, HW; Moser AB, Hollandsworth K, Brereton NH, Raymond GV (2007-09). ""Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: rationale and current assessment of efficacy.". Journal of Molecular Neuroscience 33 (1): p. 105–13. doi:10.1007/s12031-007-0041-4. PMID 17901554. 
  4. ^ Weis, Margaret (2008-05-07). A review of recent progress. The Myelin Project. Retrieved on 2008-05-31.
  5. ^ 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. 
  6. ^ Aubourg P, Adamsbaum C, Lavallard-Rousseau MC, et al (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. 
  7. ^ Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil. Arch Neurol. 2005;62:1073-1080.
  8. ^ 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 19: 1073–1082. Springer Netherlands. doi:10.1007/BF00968719. 
  9. ^ Shankar Vedantam. "A Real-Life Sequel to 'Lorenzo's Oil' - washingtonpost.com", Washington Post 2007-01-28, pp. A01. Retrieved on 2007-12-10.