Dietary erucic acid therapy for X-linked adrenoleukodystrophy

William B Rizzo, R. T. Leshner, A. Odone, A. L. Dammann, D. A. Craft, M. E. Jensen, S. S. Jennings, S. Davis, R. Jaitly, J. A. Sgro

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Abstract

We investigated the biochemical and clinical efficacy of dietary erucic acid (C22:l) therapy for X-linked adrenoleukodystrophy (ALD). In a double-blind crossover study of patients who were on chronic oleic acid (C181) 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 C260 to normal in ALD patients, and may prevent further demyelination in some mildly affected boys.

Original languageEnglish (US)
Pages (from-to)1415-1422
Number of pages8
JournalNeurology
Volume39
Issue number11
StatePublished - Jan 1 1989

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Adrenoleukodystrophy
Oleic Acid
Diet
Therapeutics
Leukoencephalopathies
Sphingomyelins
Brain
Demyelinating Diseases
Adrenal Glands
Phosphatidylcholines
Double-Blind Method
Cross-Over Studies
erucic acid
Fatty Acids
Magnetic Resonance Imaging
Liver

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Rizzo, W. B., Leshner, R. T., Odone, A., Dammann, A. L., Craft, D. A., Jensen, M. E., ... Sgro, J. A. (1989). Dietary erucic acid therapy for X-linked adrenoleukodystrophy. Neurology, 39(11), 1415-1422.

Dietary erucic acid therapy for X-linked adrenoleukodystrophy. / Rizzo, William 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.

In: Neurology, Vol. 39, No. 11, 01.01.1989, p. 1415-1422.

Research output: Contribution to journalArticle

Rizzo, WB, Leshner, RT, Odone, A, Dammann, AL, Craft, DA, Jensen, ME, Jennings, SS, Davis, S, Jaitly, R & Sgro, JA 1989, 'Dietary erucic acid therapy for X-linked adrenoleukodystrophy', Neurology, vol. 39, no. 11, pp. 1415-1422.
Rizzo WB, Leshner RT, Odone A, Dammann AL, Craft DA, Jensen ME et al. Dietary erucic acid therapy for X-linked adrenoleukodystrophy. Neurology. 1989 Jan 1;39(11):1415-1422.
Rizzo, William 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. / Dietary erucic acid therapy for X-linked adrenoleukodystrophy. In: Neurology. 1989 ; Vol. 39, No. 11. pp. 1415-1422.
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