Adrenoleukodystrophy, or ALD, strikes one in 17,000 people. (In America, that’s one baby every 36 hours.) ALD is an x-linked metabolic disorder, characterized by progressive neurologic deterioration due to demyelination of the cerebral white matter. Brain function declines as the protective myelin sheath is gradually stripped from the brain’s nerve cells. Without that sheath, the neurons cannot conduct action potentials. In other words, the neurons cannot stop telling the muscles and other elements of the central nervous system what to do.

This sequence of events appears to be related to an abnormal accumulation of saturated very-long-chain fatty acids (VLCFA) in the serum and tissues of the central nervous system, which sets off an abnormal immune response that leads to demyelination. It is unclear exactly how this chain of events works, but scientists do know that it has its roots in genetics.

ALD is caused by a genetic abnormality, commonly referred to as a “genetic mutation”, affecting the X chromosome, otherwise known as an “x-linked” condition. Everyone has two sex chromosomes: women have two X chromosomes and men have an X and a Y chromosome. If a woman inherits the abnormal X chromosome, she still has a normal, second X chromosome to help balance out the affects of the mutation. Boys and men do not have a second X chromosome, so if they inherit this genetic abnormality, they will most likely get the disease.

ALD takes several forms, which can vary widely in their severity and progression. They include:

Childhood Cerebral Demyelinating ALD. This is the most common form of ALD, representing about 45% of all ALD cases. It is characterized by an inflammatory process that destroys the myelin, causing relentless progressive deterioration to a vegetative state or death, usually within five years.

There is no cure for ALD. But there are successful treatments


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Treatment: Pre-Onset ALD
Treatment: Onset ALD
Film: Lorenzo’s Oil

Treatment: Onset ALD

Once onset the only treatment for ALD is surgery: a bone marrow transplant. But parents of these boys, when diagnosed with ALD at birth, can begin search for a matching donor immediately. The sooner the transplant, the higher its chances of success (and a normal life for their boy).

Treatment: Pre-Onset ALD

The parents of these boys can delay onset of the disease for years by giving their boys an extract of rapeseed and olive oil – Lorenzo’s Oil. Their parents know to get bi-annual MRI of their boy’s brains to scan for lesions. They also know what clinical symptoms to look for, and when. So do their doctors.  

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“Lorenzo’s Oil” is a 1992 film starring Susan Sarandon and Nick Nolte based on the true story of Augusto and Michaela Odone, parents who discovered what they hoped would be a cure for their son Lorenzo’s adrenoleukodystrophy (ALD). Sarandon was nominated for an Academy Award for her portrayal of Michaela.

The film depicts the Odones controversial discovery of a drinkable extract of rapeseed and olive oil (Lorenzo’s Oil). It reduces levels of very long chain fatty acids (VLCFAs), which are elevated in patients with ALD. Although Loenzo’s Oil did not cure Lorenzo of ALD, it is widely believed it benefitted his health and prolonged his life (he lived to be 30). Today, Lorenzo’s Oil is a treatment for pre-onset ALD, often delaying symptoms of the disease for years.

The Odones later founded the Myelin Project, an ally in today’s fight to pass Aidan’s Law.