Dietary erucic acid therapy for X‐linked adrenoleukodystrophy
Abstract
We investigated the biochemical and clinical efficacy of dietary erucic acid (C22:1) therapy for X-linked adrenoleukodystrophy (ALD). In a double-blind crossover study of patients who were on chronic oleic acid (C18:1) therapy, addition of erucic acid to the diet led to a further reduction in plasma hexacosanoic acid (C26:0) concentration. We treated 12 newly diagnosed ALD patients with a diet enriched with erucic acid and oleic acid for 2 to 19 months. Mean plasma C26:0 concentration decreased to normal by 4 weeks, and the C26:0 composition of plasma sphingomyelin and phosphatidylcholine became normal by 4 months on therapy. Fatty acid analysis of postmortem tissues from 1 boy treated for 10 months suggested that dietary erucic acid entered the heart, liver, adrenal gland, and brain. Eight patients remained on treatment long enough (mean, 12 +/- 3 months) to evaluate their clinical response; 6 of these patients with moderate to advanced disease deteriorated neurologically or showed progression of white matter disease on brain magnetic resonance imaging whereas 2 mildly affected patients remained clinically stable after 10 and 19 months. No adverse effects of the diet occurred. We conclude that dietary erucic acid therapy is effective in lowering plasma C26:0 to normal in ALD patients, and may prevent further demyelination in some mildly affected boys.
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Dietary therapy with 'Lorenzo's Oil' normalizes the levels of VLCFA in plasma within four weeks.
“This therapy normalizes the levels of VLCFA in plasma within four weeks [Rizzo et al., 1989]”
From ABCD1 mutations and the X-linked adrenoleukodystrophy mutation database: Role in diagnosis and clinical correlations (2001) · cites this paper as Rizzo et al., 1989
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- Authors
- Rizzo, W. B., Leshner, R. T., Odone, A., Dammann, A. L., Craft, D. A., Jensen, M. E., Jennings, S. S., Davis, S., Jaitly, R., & Sgro, J. A.
- Affiliations
- Virginia Commonwealth University (US)
- ORCID
- Rizzo, W. B.
- Venue
- Neurology
- Published
- 1989-11
- Volume
- 39
- Issue
- 11
- Pages
- 1415-1415
- ISSN
- 0028-3878 (ISSN-L)
- 1526-632X
- OpenAlex
- W2164071022
- Cited by
- 207
- References
- 0
- Citations by year
- 2026: 6 · 2025: 2 · 2024: 2 · 2023: 4 · 2022: 2 · 2020: 4 · 2019: 4 · 2018: 3 · 2017: 5 · 2016: 6 · 2015: 1 · 2014: 3 · 2013: 4 · 2012: 2
- FWCI
- 7.93
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Neurology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institute of Diabetes and Digestive and Kidney Diseases (DK-33914)
- National Center for Research Resources (RR 00065)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Neuroscience › Neurology › Neurological diseases and metabolism
- Keywords
- X-linked adrenoleukodystrophy, hexacosanoic acid, plasma fatty acid composition, sphingomyelin, phosphatidylcholine, demyelination, white matter disease, dietary therapy
- MeSH terms
- Adrenoleukodystrophy, Diffuse Cerebral Sclerosis of Schilder, Clinical Trials as Topic, Diet, Erucic Acids, Fatty Acids, Fatty Acids, Monounsaturated, Humans, Genetic Linkage, Lipids, Magnetic Resonance Imaging, Nervous System, Oleic Acids, Time Factors, X Chromosome, Oleic Acid
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