"Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: Rationale and current assessment of efficacy

Hugo W. Moser, Ann B. Moser, Kim Hollandsworth, N. Hong Brereton, Gerald V. Raymond

Research output: Contribution to journalArticle

Abstract

X-linked adrenoleukodystrophy (X-ALD) is a genetic disorder that damages the nervous system and is associated with the accumulation of saturated very long chain fatty acids (SVLCFA). Oral administration of "Lorenzo's oil" (LO), a 4:1 mixture of glyceryl trioleate and glyceryl trierucate, normalizes the SVLCFA levels in plasma, but its clinical efficacy and the clinical indications for its use have been controversial for more than 15 years. We review the biochemical effects of LO administration and the rationale for its use and present a current appraisal of its capacity to reduce the risk for the childhood cerebral phenotype when administered to asymptomatic boys and to slow progression of adrenomyeloneuropathy in patients without cerebral involvement. We also present current efforts to provide definitive evaluation of its clinical efficacy and discuss its possible role in the new therapeutic opportunities that will arise if newborn screening for X-ALD is validated and implemented.

Original languageEnglish (US)
Pages (from-to)105-113
Number of pages9
JournalJournal of Molecular Neuroscience
Volume33
Issue number1
DOIs
StatePublished - Sep 2007

Fingerprint

Adrenoleukodystrophy
Fatty Acids
Neurology
Screening
Inborn Genetic Diseases
Plasmas
Nervous System
Oral Administration
Therapeutics
Newborn Infant
Phenotype
Lorenzo's oil
trierucate

Keywords

  • Adrenoleukodystrophy
  • Clinical trials
  • Erucic acid
  • Fatty acids
  • Glyceryl trierucate
  • Glyceryl trioleate
  • Lorenzo's oil
  • Newborn screening
  • Therapy
  • Very long chain fatty acids

ASJC Scopus subject areas

  • Neuroscience(all)
  • Biochemistry
  • Genetics

Cite this

"Lorenzo's oil" therapy for X-linked adrenoleukodystrophy : Rationale and current assessment of efficacy. / Moser, Hugo W.; Moser, Ann B.; Hollandsworth, Kim; Brereton, N. Hong; Raymond, Gerald V.

In: Journal of Molecular Neuroscience, Vol. 33, No. 1, 09.2007, p. 105-113.

Research output: Contribution to journalArticle

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