Adrenoleukodystrophy (ALD)

The University of Minnesota is the world’s most experienced center in the treatment of patients with cerebral ALD.

We have several clinical trials for ALD patients, and we transplant almost as many patients with cerebral ALD as the rest of the world combined. We have published extensively on the clinical aspects of ALD, including biomarkers, oxidative stress, neuropsychology, and imaging.

Adrenoleukodystrophy (ALD) is a rare genetic disorder that affects males. It’s characterized by the inability of the body to break down "very long chain fatty acids" (VLCFA). The resulting build-up of VLCFA can cause dysfunction of the adrenal glands, testes and the central nervous system by breaking down the myelin sheaths that protect nerves. Normally, nerve endings are covered with a myelin sheath which helps speed the communication of nerve impulses. If myelin is lost, the underlying nerve cell will become dysfunctional. When this breakdown occurs in the brain, it’s known as cerebral ALD (cALD), which often causes rapidly progressive neurologic deterioration and eventual death.

Parents of children with cALD typically begin to notice behavioral changes, such as hyperactivity, poor performance in school, and short attention span.

Nearly 40 percent of ALD cases are the cerebral form of ALD which begins during childhood, usually between the ages of 4 and 8. The only effective treatment for cALD is a blood and marrow transplant (BMT), which can halt progression of myelin loss.