Adrenoleukodystrophy (ALD) is a rare genetic brain disorder. It is caused by mutations in the ABCD1 gene that lead to an accumulation of saturated very long-chain fatty acids (VLCFAs) and cause the demyelination of nerve fibers. Myelin is a protective sheath that insulates nerve cells in the brain.

Available treatments can arrest the demyelination of nerve cells, but no treatment exists that can restore myelination, which would be required for disease regression.

Corticosteroid replacement therapy

Most patients with ALD have adrenal insufficiency, a condition where the adrenal glands do not produce sufficient amounts of steroid hormones. Adrenal insufficiency causes Addison’s disease, which can be treated with corticosteroid replacement therapy.

Stem cell therapy

Stem cell therapy is the standard of care for patients with childhood cerebral ALD (CALD). It requires bone marrow stem cells from a donor that are transplanted into a patient’s body, where they develop into nerve cells in the brain. Stem cell therapy can stop disease progression in early stages of CALD.

A major problem with this approach is that it requires the severe weakening of the patient’s immune system. The immune system normally recognizes the donor cells as foreign and would reject them if not weakened. The attack of the donor’s stem cells by the recipient’s immune system can cause graft-versus-host disease, a life-threatening complication of stem cell therapy.

Lorenzo’s Oil

Lorenzo’s Oil is a 4:1 mix of unsaturated fatty acids — oleic acid and erucic acid. These two fatty acids interact with the same enzyme that executes the elongation of saturated fatty acids and thereby inhibit the formation of VLCFAs. The efficacy of Lorenzo’s Oil has never been demonstrated in randomized clinical trials.

Experimental treatments

There are also a number of experimental treatments that are being developed to treat ALD.


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