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Lorenzo's oil - more than just a great movie.

Not much of Hollywood's output these days can be described as "great," but even rarer is the film that also accurately presents a real-life medical problem and the incredible means by which those involved have dealt with it.

The story, for those who missed the TV debut of the movie, is about Lorenzo Odone, a beautiful, gifted child who at age six began exhibiting the distressing signs and symptoms of a rare genetic nerve disorder, adrenoleukodystrophy (ALD)--imbalance, slurring of speech, blindness, and deafness. Typically, death occurs in months or years. Yet eight years later, during which time he has not spoken a word or taken a step, Lorenzo lives.

The cynic might question the quality of that kind of life, but to Lorenzo's loving and singularly dedicated parents--and to the parents of hundreds of other children with the dread disease--it has indeed been a life worth preserving.

Refusing to accept the fact that ALD is progressive and incurable, the Odones have devoted their total energies for the past eight years to a worldwide search for a cure for this and such other demyelinating diseases as multiple sclerosis. Neither knew anything whatsoever about the field of human lipid metabolism--Michaela Odone was an editor, her husband Augusto an economist with the World Bank in Washington.

ALD is characterized by abnormal blood levels of complex saturated fatty acids known as VLCFAs (very-long-chain fatty acids). Formed from ordinary dietary fats, they are broken down in normal individuals by an enzyme lacking in ALD patients. In such patients, they destroy the insulating myelin sheath around nerve fibers, causing blindness, speech loss, muscular paralysis, and death.

The Odones exhaustively reviewed the world medical literature on ALD and conferred with the world's ALD experts. A theory of their own grew out of these investigations. VLCFAs are manufactured by a particular enzyme. If that enzyme could be fooled into producing harmless compounds by providing it with a substance that would divert the enzyme away from the production of VLCFAs, perhaps it would show down or stop the demyelinating process.

The substance they sought and for which a manufacturer was eventually found is a dietary supplement prepared from an extract of rapeseed and other vegetable oils--"Lorenzo's Oil." Currently being taken by 240 patients being studied by Dr. Hugo Moser of the Kennedy Krieger Institute in Baltimore, it has brought the VLCFA level of nearly all of them under control.

Not all patients, however, have benefited clinically from the therapy. Some have done very well, their disease having stabilized in the early stages, permitting them to live essentially normal lives. Those with advanced symptoms have not done so well, and most continue to go downhill. This suggests that there may be other aspects of the disease not yet understood that are not affected by the oil therapy.

Dr. Moser, together with some French and German scientists, recently identified the gene that causes ALD after a 30-year search that brought with it, he says, "many zig-zags." In 1963, it was discovered that this gene lived on the human X chromosome; 18 years later, scientists pinpointed the gene to a more specific location on that chromosome.

Moser states that the publication of the results of this joint effort in the February 25 issue of Nature "has immediate benefits for carrier identification and provides long-term hope for therapy." In two to three years, scientists hope to be better able to understand how the disease comes about. In the meantime, Moser and his French colleagues are studying the possibility of gene therapy and hope within the next five to 10 years to be able to introduce a normal gene into the patient's own bone marrow.

The Odones' current effort is the Myelin Project, supporting the research of a small number of scientists around the world with funds raised mostly in the Odones' native Italy. Because damaged myelin does not repair itself, patients rarely improve. The current research seeks to find a way to do that.

Meanwhile, Lorenzo is still paralyzed, able to use only blinking and finger wiggling as his means of communication while his mother reads and talks to him. Nevertheless, he is still alive, and some patients with ALD who would have been expected to die years ago are not only alive but doing well because of the Odones' love and total dedication to their son.
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Publication:Medical Update
Date:Apr 1, 1993
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