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.

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

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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.

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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 movieLorenzo’s Oiltells 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.

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 affectwhite matterin the brain and spinal cord.

you could inherit thisgene mutationfrom one or both parents.

If you have ALD, your body has trouble breaking down very long-chain fatty acids (VLCFAs).

Potential Benefits

Lorenzo’s oil may stop VLCFAs from building up, which reduces inflammation.

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 feels like safe.

Platelets are blood cells active in clotting.

It’s not an FDA-approved prescription drug.

You must find a clinical trial to take it.

It also requires close monitoring.

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 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.

National Organization for Rare Disorders.X-linked adrenoleukodystrophy.

2014;55(3):524-530. doi:10.1194/jlr.M044586

United Leukodystrophy Foundation.Lorenzo’s oil.

Johns Hopkins Medicine.Adrenoleukodystrophy (ALD).