Prognosis and Treatment

The prognosis for X-ALD is extremely grim. Most patients with this genetic disease die within 1 to 10 years of the onset of symptoms. Those with Childhood Cerebral ALD typically die between the ages of 5 to 20 years. AMN sufferers can possibly have a normal life expectancy unless they develop symptoms of cerebral demyelination.

There is currently no cure for adrenoleukodystrophy. Patients with Addison’s disease must take potentially life-saving adrenal steroid replacement therapy, although it doesn’t affect the progression of neurological symptoms. In a small study of young, pre-symptomatic patients with early signs of cerebral demyelination, hematopoietic stem cell transplantation (HSCT) proved to be successful in halting the progression of adrenoleukodystrophy. Lorenzo’s Oil is likely the most well known treatment method for ALD, but it only lowers the levels of VLCFA and does not stop the progression of symptoms once they begin. The medication Lovastatin may also lower VLCFA levels, but its use in different experiments with ALD patients demonstrates conflicting results.

Gene therapy successfully treated two 7-year old boys who displayed premature signs of cerebral ALD at Saint Vincent de Paul Hospital in Paris, France, stopping further development of the disease after a period of 14-16 months. Physical therapy, psychological support, and special education are a few symptomatic and supportive treatments available for ALD patients. Although it risks the chance of death, boys with Childhood Cerebral ALD in its early stages can potentially benefit from bone marrow transplants; this treatment is not recommended for those with the adult-onset form of this disease or for those who already have severe symptoms.

Scientists and researchers, in cooperation with volunteers at the Myelin Project, have made great research strides toward finding a cure for adrenoleukodystrophy and similar disorders. Remyelination of the human central nervous system is coming within reach and may be accomplished in our lifetime.

This little boy lost his fight with ALD at the age of 8.