Lorenzo's Oil

Common Name(S): Erucic acid and oleic acid; Lorenzo's Oil

History: A widely publicized movie about the use of Lorenzo's oil to treat a devastating neurological syndrome recently catapulted this compound into the public spotlight. The oil has been touted for the treatment of a rare genetic disease known as adrenoleukodystrophy, when it appears in children, and adrenomyeloneuropathy when it takes a more insidious path in adults. This X-linked recessive disorder is characterized by demyelinization of cerebral nerves resulting in a variety of neurologic symptoms ranging from peripheral neuropathy to blindness, spastic tetraplegia and death.

The primary metabolic abnormality in this disease is the accumulation of saturated very-long-chain fatty acids, which occurs because of a genetically impaired ability to degrade them by normal oxidation. In the absence of normal cellular oxidation of these fatty acids, saturated very-long-chain fatty acids accumulate and are believed to be responsible for the neurologic symptoms associated with the disorder.

Uses of Lorenzo's Oil

Lorenzo's oil has been used to treat certain rare diseases, without verified success.

Side Effects of Lorenzo's Oil

Thrombocytopenia has been reported following treatment.

Toxicology: Thrombocytopenia has been reported following treatment with Lorenzo's oil. In the study by Aubourg et al, the platelet count declined in 23 of 24 patients, but this was not correlated with plasma levels of erucic acid or other metabolites. None of the patients had abnormal bleeding or hematoma. Some of the patients had asymptomatic neutropenia. Dietary supplements of safflower and fish oils were given to the patients during this study, and while a 30% decrease in plasma docosahexaenoic acid levels occurred in all patients, none reported symptoms of essential fatty acid deficiency.

Summary: Lorenzo's oil represents a combination of oleic and erucic acids, which are long-chained monounsaturated fatty acids. The use of this preparation in adrenomyeloneuropathy, a genetically transmitted disease, has resulted in generally poor effectiveness. While the use of this combination appears logically based on the pharmacology of the genetic defect, clinical studies to date have not shown a valid clinical effect.