Lorenzo's oil really does save lives
New Scientist vol 176 issue 2363 - 05 October 2002, page 7

THE controversial do-it-yourself medicine that inspired the heart-rending Hollywood movie Lorenzo's Oil has finally been proved to work, ending years of uncertainty about the treatment and demolishing the claims of experts who repeatedly said it was worthless.
Hugo Moser, the neurologist and doctor played by Peter Ustinov in the film, last week unveiled the conclusions of a 10-year study into the oil's effects on a group of boys with the same genetic condition as Lorenzo.
Carriers of the defect progressively lose the ability to move, hear, speak and - in the end - breathe. Some victims get the childhood form of the disease, known as adrenoleukodystrophy, which usually kills within just two years. Others get the adult form, which strikes people in their late twenties and acts more slowly.
It was 17 years ago that Lorenzo Odone started bumping into furniture. His parents were told he had just two years to live. Racing against the clock, Augusto Odone and his wife pestered scientists, devoured neurology textbooks and learned the language of biochemistry. The result was Lorenzo's oil, a mixture of oleic and erucic acids.
Lorenzo is still alive today but unable to move and being cared for at the family home. The medical establishment has been very sceptical about whether the oil actually has any beneficial effects.

But last week, Moser - himself once a sceptic - told an international meeting at the University of Ghent that the oil should be routinely given to boys with the genetic defect.
Between 1989 and 1999, two teams, one led by Moser at the Kennedy Krieger Institute in Baltimore, Maryland, and the other based in Europe, followed 104 young boys with the condition. At the outset all were under six years of age and none had begun to develop symptoms. Their movement, hearing and MRI brain scans were normal.
The study shows that the boys whose parents scrupulously gave them the oil - despite the sceptism of many doctors - were much less likely to develop symptoms in childhood than boys who did not get the oil on a regular basis.
When it ended, 76 per cent of the boys getting the oil were still healthy, and their brains appeared normal. But the same was true for only about a third of the boys who did not regularly get the oil. There was no control group of boys given only a placebo, as it was felt to be unethical.
The protection offered by the oil - though not complete - is dramatic and will come as a shock to the medical community, Lorenzo's father told New Scientist.
After years trying to persuade doctors and scientists to take it seriously, Odone says he feels vindicated.
A big remaining uncertainty is whether those boys in the study who have escaped the childhood version of the disease will also be spared the adult form. As the boys are all still in their late teens, this may not be known for years.
The devastating symptoms of the disease are caused by the progressive loss of myelin, the fatty sheath that insulates nerve fibres enabling them to conduct impulses efficiently. Boys with the defect have very high levels of very long chain saturated fatty acids in their blood. The oil brings these levels back down to normal by blocking the enzymes that make the acids. But it's not clear how this prevents the onset of symptoms in boys.

Parents of affected boys should give them the oil as soon as they learn their son carries the genetic defect, Odone says. "If you wait, the symptoms might come and then you are in a different ballpark. We are not sure the oil is useful after symptoms have developed."

David Concar

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