Adrenoleukodystrophy (ALD)

What is adrenoleukodystrophy?

Adrenoleukodystrophy (ALD) is a rare genetic disorder, affecting about 1 in 15,000 people worldwide (MedlinePlus Genetics (opens in a new tab)). Changes in the ABCD1 gene stop the body breaking down very long-chain fatty acids. These fats build up and damage the brain, spinal cord and adrenal glands. Because it is passed down on the X chromosome, it mostly causes severe disease in boys and men, though women who carry it can also develop symptoms (MedlinePlus Genetics (opens in a new tab)).

The main forms

• Childhood cerebral ALD — the most severe form. Learning and behavior problems usually begin between ages 4 and 10 and can look like ADHD, followed by rapid neurological decline (MedlinePlus Genetics (opens in a new tab)).
• Adrenomyeloneuropathy (AMN) — signs usually appear between early adulthood and middle age, with progressive stiffness and weakness in the legs (MedlinePlus Genetics (opens in a new tab)).
• Some people have adrenal insufficiency only, and some have no symptoms (MedlinePlus Genetics (opens in a new tab)).

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Conventional medicine (AMA)

For the cerebral form, an allogeneic stem cell transplant has long been the main treatment that can arrest the loss of myelin in the brain, and only if it is done very early; results are better the earlier it is started (Engelen et al., 2012 (opens in a new tab)). Other care is supportive (NINDS (opens in a new tab)):

• Stem cell transplant may halt the disease in children if ALD is found and treated early. It carries a significant risk, so it is a decision for a specialist team (Engelen et al., 2012 (opens in a new tab)).
• Adrenal insufficiency treatment: boys and men with ALD are advised to have regular adrenal testing by an endocrinologist, and steroid replacement therapy can be started if it is needed (Engelen et al., 2012 (opens in a new tab)).
• Medicines to help manage muscle tone, seizures and spasticity, which are used for many leukodystrophies (NINDS (opens in a new tab)).
• Physical, occupational and speech therapy to help improve mobility and function (NINDS (opens in a new tab)); adult men with AMN often need a referral to a rehabilitation physician (Engelen et al., 2012 (opens in a new tab)).
• Gene therapy: in a clinical trial of 32 boys with early cerebral ALD, none of the 29 who completed the first 24 months had a major functional disability, and about six years on, most still had none. Gene therapy carries a risk of blood cancer, so long-term safety is still being studied (Eichler et al., 2024 (opens in a new tab)).

Because ALD can affect the brain, adrenal glands and spinal cord, care often brings together neurology, endocrinology and rehabilitation teams (Engelen et al., 2012 (opens in a new tab)).

What it costs: unknown — it depends heavily on the treatments needed and your insurance. What to expect: successful care focuses on managing symptoms, except where early transplant can halt progression.

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MBHA Wellness Coach

A 2025 review of the research found that diet is one of the pillars of managing ALD, alongside medicines, gene therapy and stem cell transplant (Nutrients, 2025 (opens in a new tab)):

• Cutting very long-chain fats from food alone has limited effect, because the body also makes them itself.
• “Lorenzo’s Oil” slows the body’s production of these fats, but its clinical benefits are inconsistent.
• Newer approaches — such as the “Bambino Diet” and new supplements similar to Lorenzo’s Oil with antioxidants — show promising effects on blood fat levels.
• Malnutrition is more likely in people with ALD, so nutrition should be planned individually with the care team.