Mixture of modified vegetable oils used in treating adrenoleukodystrophy
Lorenzo's oil
is a liquid solution made of 4 parts
glycerol trioleate
and 1 part glycerol trierucate, which are the
triacylglycerol
forms of
oleic acid
and
erucic acid
.
[1]
It is prepared from
olive oil
and
rapeseed oil
.
[2]
It is used in the investigational treatment of asymptomatic patients with
adrenoleukodystrophy
(ALD), a nervous system disorder.
The development of the oil was led by
Augusto and Michaela Odone
after their son
Lorenzo
was diagnosed with the disease in 1984, at the age of five. Lorenzo was predicted to die within a few years. His parents sought experimental treatment options, and the initial formulation of the oil was developed by retired British scientist Don Suddaby (formerly of
Croda International
).
[3]
Suddaby and his colleague, Keith Coupland, received a U.S. patent (since expired) for invention of the oil.
[4]
The
royalties
received by Augusto were paid to
The Myelin Project
which he and Michaela founded to further research treatments for ALD and similar disorders.
[
citation needed
]
The Odones and their invention obtained widespread publicity in 1992 because of the film
Lorenzo's Oil
.
Research on the effectiveness of Lorenzo's oil has seen mixed results, with possible benefit for asymptomatic ALD patients but of unpredictable or no benefit to those with symptoms, suggesting its possible role as a preventative measure in families identified as ALD dominant.
Lorenzo Odone
died on May 30, 2008, at the age of 30; he was bedridden with paralysis and died from
aspiration pneumonia
, likely caused by having inhaled food.
[5]
[6]
Treatment cost
[
edit
]
In 2012, Lorenzo's oil cost approximately $400 USD for a month's treatment.
[7]
Proposed mechanism of action
[
edit
]
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.
[8]
Effectiveness
[
edit
]
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.
[9]
Hugo Moser
played a prominent role in both the treatment of Lorenzo Odone and the scientific evaluation of Lorenzo'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.
[10]
Moser appraised Lorenzo's oil again in a 2007 report.
[11]
Moser's findings, that Lorenzo's oil did not help symptomatic ALD patients, are consistent with prior studies published in 2003
[12]
and 1999.
[9]
[13]
A study by Poulos published in 1994 found that Lorenzo's oil is of limited value in correcting the accumulation of saturated VLCFAs in the brain of patients with ALD.
[14]
Comparative autopsies showed that treatment enriched erucic acid in plasma and tissues, but not in the brain.
[15]
Side effects
[
edit
]
The oil has been shown to cause a lowered
platelet
count,
[16]
which can lead to
thrombocytopenia
and
lymphopenia
.
[17]
: 646?657
There are no reports of toxicity from dietary consumption of erucic acid.
[17]
[18]
[19]
Current state
[
edit
]
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. Moser's 2005 study has found "strongly suggestive, albeit not fully definitive, evidence of a preventive effect" of Lorenzo's oil on the onset of symptoms when used by asymptomatic patients.
[10]
References
[
edit
]
- ^
"Page Not Found"
.
www.lowproteinconnect.com
. 20 October 2020. Archived from
the original
on 27 June 2020
. Retrieved
13 July
2022
.
- ^
Shankar Vedantam.
"A Real-Life Sequel to 'Lorenzo's Oil' ? washingtonpost.com"
. Washington Post 2007-01-28. pp. A01
. Retrieved
2007-12-10
.
- ^
"Lorenzo Odone"
. 1 June 2008 – via www.telegraph.co.uk.
- ^
U.S. Patent 5,331,009: Pharmaceutical compositions for treating adrenoleukodystrophy
.
[
dead link
]
Issued July 19, 1994.
- ^
"Lorenzo's Oil boy is dead at 30"
.
BBC News
. 31 May 2008
. Retrieved
2008-06-02
.
- ^
"Subject of 'Lorenzo's Oil' dies at 30"
.
CNN
.
Associated Press
. 2008-05-30. Archived from
the original
on July 17, 2008
. Retrieved
2008-07-12
.
- ^
"Lorenzo's Oil ? The Oil"
. The Myelin Project
. Retrieved
3 November
2012
.
[
dead link
]
- ^
"Archived copy"
.
www.myelin.org
. Archived from
the original
on 4 November 2013
. Retrieved
17 January
2022
.
{{
cite web
}}
: CS1 maint: archived copy as title (
link
)
- ^
a
b
- ^
a
b
Moser HW, Raymond GV, Lu SE, Muenz LR, Moser AB, Xu J, Jones RO, Loes DJ, Melhem ER, Dubey P, Bezman L, Brereton NH, Odone A (July 2005).
"Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's Oil"
.
Archives of Neurology
.
62
(7): 1073?80.
doi
:
10.1001/archneur.62.7.1073
.
PMID
16009761
.
- ^
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
.
S2CID
21333247
.
- ^
Aubourg P, Adamsbaum C, Lavallard-Rousseau MC, Rocchiccioli F, Cartier N, Jambaque 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
.
- ^
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. Psychiatry
.
67
(3): 290?9.
doi
:
10.1136/jnnp.67.3.290
.
PMC
1736534
.
PMID
10449548
.
- ^
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
.
S2CID
41340913
.
- ^
Magnhild Rasmussen; Ann B. Moser; Janet Borel; Surinder Khangoora; 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
(8). Springer Netherlands: 1073?1082.
doi
:
10.1007/BF00968719
.
PMID
7800117
.
S2CID
11658824
.
- ^
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.
doi
:
10.1002/ajh.2830480217
.
PMID
7847331
.
S2CID
29556389
.
- ^
a
b
Luger CL et al. Food Safety and Foodborne Toxicants. Chapter 14 in Hayes' Principles and Methods of Toxicology, Sixth Edition. Eds A. Wallace Hayes, Claire L. Kruger. CRC Press, 2014
ISBN
9781842145371
. Quote: "In humans. however. although the long-term use of Lorenzo's oil (oleic acid and erucic acid) in the treatment of adrenoleukodystrophy or adrenomyeloneuropathy leads to thrombocytopenia and lymphopenia (Unkrig et al. 1994), adverse effects from dietary consumption of erucic acid have not been reported."
- ^
Food Standards Australia New Zealand (June 2003)
Erucic acid in food
Archived
2008-12-03 at the
Wayback Machine
:
A Toxicological Review and Risk Assessment .
Technical report series No. 21; Page 4 paragraph 1;
ISBN
0-642-34526-0
, ISSN 1448-3017
- ^
"Food Standards Agency - Agency issues warning on erucic acid"
. 2 September 2004
. Retrieved
2007-11-02
.
External links
[
edit
]