Lorenzo's oil
Encyclopedia
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.
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 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.
.
s 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.
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. Moser appraised Lorezo's oil again in a 2007 report.
Moser's findings, that Lorenzo's oil did not help symptomatic ALD patients, are consistent with prior studies published in 2003 and 1999.
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. Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.
count.
Erucic acid is considered to be a toxin, and its presence in the food supply is regulated.
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. Moser's team, Dr. Gerald Raymond and Ann Moser continue the trial and his work with the leukodystrophies.
Triglyceride
A triglyceride is an ester derived from glycerol and three fatty acids. There are many triglycerides, depending on the oil source, some are highly unsaturated, some less so....
forms of oleic
Oleic acid
Oleic acid is a monounsaturated omega-9 fatty acid found in various animal and vegetable fats. It has the formula CH37CH=CH7COOH. It is an odorless, colourless oil, although commercial samples may be yellowish. The trans isomer of oleic acid is called elaidic acid...
and erucic acid
Erucic acid
Erucic acid is a monounsaturated omega-9 fatty acid, denoted 22:1 ω-9. It has the formula CH37CH=CH11COOH. It is prevalent in rapeseed, wallflower seed, and mustard seed, making up 40-50% of their oils...
), prepared from olive oil
Olive oil
Olive oil is an oil obtained from the olive , a traditional tree crop of the Mediterranean Basin. It is commonly used in cooking, cosmetics, pharmaceuticals, and soaps and as a fuel for traditional oil lamps...
and rapeseed
Rapeseed
Rapeseed , also known as rape, oilseed rape, rapa, rappi, rapaseed is a bright yellow flowering member of the family Brassicaceae...
oil.
It is used in the investigational treatment of asymptomatic patients with adrenoleukodystrophy
Adrenoleukodystrophy
Adrenoleukodystrophy is a rare, inherited disorder that leads to progressive brain damage, failure of the adrenal glands and eventually death. ALD is a disease in a group of genetic disorders called leukodystrophies, whose chief feature is damage to myelin...
(ALD). The oil was formulated by Augusto and Michaela Odone
Augusto and Michaela Odone
Augusto Daniel Odone and Michaela Teresa Murphy Odone are the parents of Lorenzo Odone , a child afflicted with the illness adrenoleukodystrophy...
after their son Lorenzo
Lorenzo Odone
Lorenzo Michael Murphy Odone was an American adrenoleukodystrophy patient whose parents, Augusto and Michaela Odone, sought a treatment for the disease and invented Lorenzo's oil....
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
The Myelin Project
The Myelin Project is an international scientific research organization aimed at accelerating clinical and translational research focused on myelin repair and to regenerate the nerve's myelin sheath, — a process called remyelination — destroyed in a host of diseases such as multiple...
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
Lorenzo Odone
Lorenzo Michael Murphy Odone was an American adrenoleukodystrophy patient whose parents, Augusto and Michaela Odone, sought a treatment for the disease and invented Lorenzo's oil....
died on May 30, 2008, after suffering from aspiration pneumonia
Aspiration pneumonia
Aspiration pneumonia is bronchopneumonia that develops due to the entrance of foreign materials into the bronchial tree, usually oral or gastric contents...
, caused by food getting stuck in his lungs.
Treatment costs
Lorenzo's oil costs approximately US$440 for a month's treatment. Most insurance companies in the United States will not pay for it since it is an Investigational New DrugInvestigational New Drug
The United States Food and Drug Administration's Investigational New Drug program is the means by which a pharmaceutical company obtains permission to ship an experimental drug across state lines before a marketing application for the drug has been approved...
.
Proposed mechanism of action
The mixture of fatty acidFatty acid
In chemistry, especially biochemistry, a fatty acid is a carboxylic acid with a long unbranched aliphatic tail , which is either saturated or unsaturated. Most naturally occurring fatty acids have a chain of an even number of carbon atoms, from 4 to 28. Fatty acids are usually derived from...
s purportedly reduces the levels of very long chain fatty acids (VLCFAs), which are elevated in ALD
Adrenoleukodystrophy
Adrenoleukodystrophy is a rare, inherited disorder that leads to progressive brain damage, failure of the adrenal glands and eventually death. ALD is a disease in a group of genetic disorders called leukodystrophies, whose chief feature is damage to myelin...
. It does so by competitively inhibiting
Competitive inhibition
Competitive inhibition is a form of enzyme inhibition where binding of the inhibitor to the active site on the enzyme prevents binding of the substrate and vice versa.-Mechanism:...
the enzyme
Enzyme
Enzymes are proteins that catalyze chemical reactions. In enzymatic reactions, the molecules at the beginning of the process, called substrates, are converted into different molecules, called products. Almost all chemical reactions in a biological cell need enzymes in order to occur at rates...
that forms VLCFAs.
Effectiveness
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.Hugo Moser
Hugo Moser (scientist)
Hugo Moser, M.D. , was a research scientist and Director of the Neurogenetics Research Center at the Kennedy Krieger Institute. Dr. Moser was also University Professor of Neurology and Pediatrics at Johns Hopkins University...
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. Moser appraised Lorezo's oil again in a 2007 report.
Moser's findings, that Lorenzo's oil did not help symptomatic ALD patients, are consistent with prior studies published in 2003 and 1999.
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. Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.
Side Effects
The oil has been shown to cause a lowered plateletPlatelet
Platelets, or thrombocytes , are small,irregularly shaped clear cell fragments , 2–3 µm in diameter, which are derived from fragmentation of precursor megakaryocytes. The average lifespan of a platelet is normally just 5 to 9 days...
count.
Erucic acid is considered to be a toxin, and its presence in the food supply is regulated.
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.In the U.S., Lorenzo's oil is currently only available to patients taking part in a clinical trial at the Kennedy Krieger Institute
Kennedy Krieger Institute
The Kennedy Krieger Institute is an internationally recognized organization improving the lives of children and adolescents with learning disabilities, disorders of the brain, spinal cord, and musculoskeletal system...
. This trial was formerly held under the direction of Dr. Hugo Moser
Hugo Moser (scientist)
Hugo Moser, M.D. , was a research scientist and Director of the Neurogenetics Research Center at the Kennedy Krieger Institute. Dr. Moser was also University Professor of Neurology and Pediatrics at Johns Hopkins University...
, until Moser's death in 2007. Moser's team, Dr. Gerald Raymond and Ann Moser continue the trial and his work with the leukodystrophies.