2003 World Record Set:
18 days, 4 hours, 51 minutes!

The Mississippi River Challenge for
Rett Syndrome & Leukodystrophy

- May 10, 2003 6 AM to May 28, 10:51 AM 2003-
A 2,348 Mile Marathon to Support a Marathon of Research

L to R: Bradford & Eid, near Lock & Dam 24

Adrenoleukodystrophy (ALD)

What Is Adrenoleukodystrophy (ALD)?

ALD is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, the fatty covering, on nerve fibers in the brain and leads to progressive neurological disability and death. It is probably best know to Americans as the disease that afflicts the young boy Lorenzo Odone, whose story is told in the 1993 film 'Lorenzo's oil'.

Who Gets ALD?

ALD does not recognize racial, social, economic, or religious boundaries. It does, however, manifest preferentially in males.

Diagnosing ALD

For ALD, very accurate diagnostic testing is available. However, there is a tremendous lack of awareness among both laypersons and physicians in terms of requesting these studies.  An ALD diagnosis can be done on a blood sample by either looking at the level of very long chain fatty acids (VLFCAs) in the blood, or by DNA analysis.

There are several forms of ALD

Onset of the classic childhood form, which is the most severe and affects only boys, may occur between ages 4 and 10. Features of this form may include visual loss, learning disabilities, seizures, dysphagia, deafness, disturbances of gait and coordination, fatigue, intermittent vomiting, and progressive dementia. The most common symptoms are usually behavioral changes such as abnormal withdrawal or aggression, poor memory, and poor school performance.

In the milder adult-onset form, which typically begins between ages 21 and 35, symptoms may include leg stiffness, progressive spastic paraparesis of the lower extremities, and ataxia. Although adult-onset ALD progresses more slowly than the classic childhood form, it can also result in deterioration of brain function.

Another form of ALD is occasionally seen in women who are carriers of the disorder. Symptoms are mild and may include spastic paraparesis of the lower limbs, ataxia, hypertonia, mild peripheral neuropathy, and urinary problems.

What Happens When A Child Has ALD?

ALD affects the brain with active demyelination. Demyelination is the stripping away of the myelin sheath which acts as insulation for the nerves. This process is an inflammatory response that destroys the nerve cells. In ALD, boys develop normally until after early symptoms that are often mistaken for attention deficit disorder, there appear to be signs of serious neurological involvement. Impaired auditory discrimination, visual disturbances, impaired coordination, dementia or seizures. The disease worsens over several years, and usually in less than two years from ALD diagnosis, the child will lose all cognitive, mental, and physical functions, and deteriorate into a vegetative state leading to death.

Treatment and Therapies

Once an ALD diagnosis has been made, treatment options are limited.

The most uniformly accepted and recommended therapy is bone marrow transplant and the newer thearpy is a cord blood transplants. Both of these treatments are only suitable for afflicted boys during a relatively narrow window, are are risky (up to 25% mortality).

The most common form of dietary therapy that has been implemented is a 4:1 mixture of glycol trioleate and glycol trioleate. This oil (Lorenzo's Oil) has demonstrated a rapid reduction of very long chain fatty acids in plasma. New research suggests early treatment with Lorenzo's oil, low-fat diet can delay onset of ALD. Please see New research suggests early treatment with Lorenzo's oil, low-fat diet can delay onset of ALD - 10/9/2002 for more information.

ALD is a deadly disease that is often not diagnosed in a timely manner that would allow for a chance at successful treatment. As this is a genetic disease, the defect could also be discovered in the prenatal stage. In addition, many children die or become mentally and physically disabled due to complications related to both the course of the disease and the nature of the therapy. Even today's best case scenario cannot be described as a cure. Many adults are severely handicapped and can only look forward to a long-term downward spiral with no hope of relief.

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Leukodystophy Research!

All donations to research for Leukodsytrophy research for this charitable event are being directed to the United Leukodystrophy Foundation (ULF), a 501(c)3 non-profit group.

Click here to find out more about the ULF and information on Leukodystrophies

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