Research backs 'Lorenzo's Oil' treatment of disease

New research offers scientific vindication for the parents whose fight to save their ailing son inspired the 1992 movie ''Lorenzo's Oil." It comes too late to help the real-life Lorenzo, who is nearly totally paralyzed, but suggests that the oil could help save future generations of boys from the same, fatal nerve disorder.

The paper, published yesterday in the Archives of Neurology, found that the oil was often effective at fending off symptoms in young boys who had the genetic disease that has devastated Lorenzo Odone. Among the 89 boys who started taking the oil before symptoms appeared, only 11 percent went on to develop them, compared with the usual 35 percent.

In a prototypical clash between activist parents and resistant doctors, the medical establishment has been extremely skeptical about the effectiveness of Lorenzo's oil. Among those skeptics was Dr. Hugo W. Moser, a professor at Johns Hopkins University and the lead author of the new paper.

''Even though many people -- and I must say, myself not excluded -- maligned the oil, this study, which as far as I can tell is scientifically sound, showed that it does have an effect on certain types of ALD," Moser said. ''It suggests to me that it's more than snake oil."

The paper does not address whether Lorenzo's oil, which is extracted from olive and rapeseed oil and must be combined with a low-fat diet, can slow the disease once its symptoms have already appeared. Previous studies had found that it did not seem to help.

But the new research does raise the possibility that early tests -- which Moser is working to develop -- could pick up Lorenzo's disease in babies and that it could then be stopped before it causes damage. Its authors also recommend that the oil become standard treatment for young boys with the disease who have not yet shown symptoms.

The disease, X-linked adrenoleukodystrophy, or ALD, affects about 16,000 Americans, generally striking boys between ages 4 and 10, damaging their nerves, and often progressing quickly to paralysis then death. The problem stems from abnormal fatty acids that damage myelin, the protective sheathing around nerves.

In some cases, the disease leads to rapid deterioration, while in others it remains relatively benign, a variable path that complicated researchers' attempts to determine whether the oil made a difference.

''One of the tragic ironies of this story," said Lorenzo's half brother, Francesco, ''is that because of my father's and stepmother's efforts, so many young boys will be spared the symptoms of this evil disease. But unfortunately, for Lorenzo, it came too late."

When Lorenzo Odone was diagnosed in 1984, doctors told his parents, Augusto and Michaela Odone, that the then-6-year-old was doomed. They plunged into research and developed the oil, which seemed to stop his degeneration.

Lorenzo is still alive today, but, at 27, is almost totally paralyzed, and it is unlikely, his half brother said, that he would be able to understand the news about Moser's paper.

His family's medical activism has evolved into The Myelin Project, a nonprofit group that aims to spur researchers to find ways to repair myelin, in hopes of restoring Lorenzo's nerves and helping millions of others with myelin-related diseases such as multiple sclerosis. His mother died of cancer in 2000; Augusto is a coauthor on the new paper.

The Archives of Neurology study, funded by the federal government, Johns Hopkins, and The Myelin Project, followed 89 boys who had ALD but no symptoms for an average of seven years. It found the boys by screening members of families with a tendency toward ALD, and once they entered the study, the boys were given about three-quarters of an ounce of the oil per day.

Moser, who directs neurogenetics research at Kennedy Krieger Institute in Baltimore, is now concentrating on developing a test to screen newborns for ALD.

Several other metabolic diseases are already caught by newborn screening tests. When they are, and treatment by dietary regime or medication follows, children who would otherwise have ended up severely disabled or mentally retarded can lead normal lives.

''I wake up at night and say, 'What the hell are you doing, why don't you have that yet?' " Moser said. ''I think it's a terrible tragedy when people are diagnosed on the basis of symptoms. We have to identify it before the symptoms."

Carey Goldberg can be reached at goldberg@globe.com.