Lorenzo's Oil: Can It Really Treat ALD?

Learn about the ALD treatment inspired by Lorenzo Odone

Lorenzo's oil is a potential treatment for some people with adrenoleukodystrophy (ALD). ALD is a rare and sometimes fatal genetic disorder that affects white matter in the nervous system. It also affects the adrenal glands.

The oral treatment is a mixture of unsaturated fatty acids. While some people with ALD may benefit from Lorenzo's oil, it's not a miracle treatment. It does not repair existing damage or cure ALD. This article discusses some potential benefits and downsides of Lorenzo's oil.

A healthcare provider examines a child who is sitting in their parent's lap

ljubaphoto / Getty Images

What Is Lorenzo's Oil?

The oil is named for Lorenzo Odone, a 5-year-old who started showing signs of ALD in 1983. His parents, Augusto and Michaela, were frustrated by the lack of treatment options.

Despite having no medical training, they set out to find a treatment. They developed the oil in 1986. The 1992 movie Lorenzo's Oil tells the somewhat controversial story of their journey.

Even with the treatment, Lorenzo lived with severe neurological impairments. He died in 2008 at the age of 30, having lived far longer than expected.

Lorenzo's oil is a combination of unsaturated fatty acids consisting of:

  • 4 parts glyceryl trioleate (triolein), which comes from oleic acid (extracted from olive oil)
  • 1 part glyceryl trierucate, which comes from erucic acid (extracted from rapeseed oil)

Most studies to date have been rather small, and results mixed. Larger-scale studies are needed, but this is difficult with rare conditions such as ALD.

The Food and Drug Administration (FDA) has not approved Lorenzo's oil as a medical treatment. It's still considered an experimental drug, though it may be available through clinical trials.

Which Diseases Does Lorenzo's Oil Treat?

Lorenzo's oil is formulated specifically for ALD.

ALD is one of a group of neurological conditions called leukodystrophies. These are rare disorders that affect white matter in the brain and spinal cord. ALD, which is also called X-linked adrenoleukodystrophy or X-ALD, is caused by a gene mutation (change) on the X chromosome.

You can inherit this gene mutation from one or both parents. ALD is more severe in people with one X and one Y chromosome (often assigned male at birth) than in people with two X chromosomes (often assigned female at birth).

If you have ALD, your body has trouble breaking down very long-chain fatty acids (VLCFAs). VLCFAs build up in the nervous system and adrenal gland, causing inflammation that damages myelin (the protective layer that forms around nerves). The main signs of ALD are loss of myelin and adrenal gland dysfunction.

Lorenzo's oil has been shown to lower VLCFA levels in a study of people with ALD and in a mouse model study. However, it's not an effective treatment for other demyelinating diseases, such as multiple sclerosis (MS), which are caused by other factors.

Potential Benefits

Lorenzo's oil may stop VLCFAs from building up, which reduces inflammation. This helps prevent damage to myelin and slows disease progression in those who are still asymptomatic (without symptoms of the condition).

It's important to note that Lorenzo's oil should not replace other treatments for ALD. These include medications, physical therapy, and stem cell transplants.

Potential Downsides

Lorenzo's oil does not repair damaged myelin. It doesn't alleviate symptoms. It doesn't slow or stop progression in children or adults who already have neurological damage or adrenal dysfunction.

Lorenzo's oil appears to be safe. However, 30% to 40% of people taking it develop a reversible, moderately lower platelet count without significant bleeding. Platelets are blood cells active in clotting. About 55% have a slight increase in liver enzymes (which can indicate liver damage), but without major damage to the liver.

It's not an FDA-approved prescription drug. You must find a clinical trial to take it. It also requires close monitoring.

Daily Dosage

Lorenzo's oil is still an experimental treatment, so there are no clear dosing guidelines.

In one clinical trial, children assigned male with ALD took 2 to 3 milliliters (mL) of Lorenzo's oil per 1 kilogram (kg) (about 2.2 pounds) of body weight (mL/kg) per day. They also limited fat from other sources to 10% to 15% of total daily calories.

In general, you take a dose that amounts to about 20% of your total daily calories.

Summary

Lorenzo's oil is a mixture of unsaturated fatty acids. It's intended to treat ALD, an inherited, progressive disease that is often fatal. The treatment may help delay disease progression in people who haven't developed symptoms yet.

It works by preventing a certain type of fatty acid from building up and causing inflammation that damages myelin. It does not affect existing damage. It doesn't slow progression in people who already have symptoms.

Lorenzo's oil shows promise, but much more research is needed. The FDA has not approved Lorenzo's oil to treat ALD or other conditions. It's only available through clinical trials. You can learn more about Lorenzo's oil from a neurologist or other ALD specialist.

9 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
  1. National Organization for Rare Disorders. X-linked adrenoleukodystrophy.

  2. Lerner BH. Complicated lessons: Lorenzo Odone and medical miracles. The Lancet. 2009;373(9667):888-889. doi:10.1016/S0140-6736(09)60534-1 Allowing this older citation as it is supporting the history of the treatment

  3. Sassa T, Wakashima T, Ohno Y, Kihara A. Lorenzo's oil inhibits ELOVL1 and lowers the level of sphingomyelin with a saturated very long-chain fatty acid. J Lipid Res. 2014;55(3):524-530. doi:10.1194/jlr.M044586

  4. United Leukodystrophy Foundation. Lorenzo's oil.

  5. National Institute of Neurological Disorders and Stroke. Adrenoleukodystrophy information page.

  6. Johns Hopkins Medicine. Adrenoleukodystrophy (ALD).

  7. Ahmed MA, Kartha RV, Brundage RC, et al. A model‐based approach to assess the exposure–response relationship of Lorenzo’s oil in adrenoleukodystrophy. Br J Clin Pharmacol. 2016;81(6):1058-1066. doi:10.1111/bcp.12897

  8. Morita M, Honda A, Kobayashi A, et al. Effect of Lorenzo’s oil on hepatic gene expression and the serum fatty acid level in abcd1-deficient mice. JIMD Rep. 2017;38:67-74. doi:10.1007/8904_2017_32

  9. Adrenoleukodystrophy News. Lorenzo's oil.

By Ann Pietrangelo
Ann Pietrangelo is a freelance writer, health reporter, and author of two books about her personal health experiences.