New Clinical Trials on the Way for Adrenoleukodystrophy

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Tomasz_Mikolajczyk / Pixabay

Persistence is a tricky thing. In Hollywood films, persistence pays off. The dorky kid keeps asking the cheerleader out while he’s helping her with her homework until she finally agrees to go on a date with him. On the date, she realizes that he’s everything she was looking for, but she had been looking in the wrong places. In real life, the persistence our dorky hero showed would have gotten him a beating from the cheerleader’s football player boyfriend. However, persistence is not only in the realms of love.

Dr. Tom Scanlan of Oregon Health & Science University (OHSU) exemplifies persistence with his crusade to develop a treatment for adrenoleukodystrophy (ALD).

The genetic disorder causes damage to the protective layers of fat around nerve cells in the central nervous system, i.e. brain and spinal column. Adrenoleukodystrophy became a topic of conversation in the early 1990s with the release of Lorenzo’s Oil, a true story about a family dealing with the condition. Males are more affected than females because the gene that causes the disorder is present only on X-chromosomes. There are two types of the disorder: a severe version that presents early in life and often results in death and a milder version that presents later in life and may result in numbness or paralysis of the legs.

Dr. Scanlan originally synthesized a molecule 1996 while trying to develop a new treatment for high cholesterol when he was conducting research at the University of California, San Francisco. Later, the company intending to take the formula to clinical trials stopped the process. By the time the rights lapsed and returned to Dr. Scanlan in 2010, he had moved on from cholesterol. An idea struck him that it might be used in arresting the progress of ALD.

Testing with mice showed some astounding results. They were impressive enough to press further with human trials. Those trials are ongoing at the time of this article’s publication.

Dr. Scanlan’s persistence to do something with his synthesized molecule may soon help the thousands of people with ALD in the United States.

Click here to read an article about Dr. Scanlan on the OHSU website.


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