October 10, 2002 BALTIMORE (AP) -- Results of a 10-year study suggest that Lorenzo's oil, portrayed as a miracle treatment in the movie of the same name, appears to delay the symptoms of a rare genetic disease in young boys if they take it early and follow a low-fat diet.
"This is such a terrible disease that the results we have are very favorable," said Dr. Hugo Moser, who has searched for years for a treatment for adrenoleukodystrophy, or ALD.
Those who have the disease become weak, lose their ability to communicate and eventually die.
Moser, director of neurogenetics at Baltimore's Kennedy Krieger Institute, said that boys without symptoms of the disease who took the oil and rigidly stuck to a low-fat diet were two-thirds less likely to develop brain abnormalities than those who didn't strictly follow the treatment.
The boys remained symptom-free for two to four years, he said.
"It's not an absolute preventive. It reduces the chances of developing the symptoms, but it does not eliminate the chance," Moser said. "The need to pursue other treatments remains critical."
The oil used as a treatment was developed by Augusto and Michaela Odone, a Virginia couple whose search for a cure for their ALD-stricken son, Lorenzo, inspired the 1992 movie "Lorenzo's Oil."
The Odones said their mixture of derivatives of natural cooking oils, like canola and olive oil, stopped the decline of their son and partly reversed it.
A 1993 study by French researchers showed the oil was ineffective for adults with symptoms of the disease.
The childhood version of ALD hits boys between the ages of 4 and 10, quickly stealing their ability to see and speak and eventually killing them. The adult form progresses more slowly.
Moser said his research - the first attempt to test the oil as a preventive - involved 105 boys in Europe and the United States who began treatment before age 6. The study was conducted from 1989-99 with participants joining over the years.
A control group was not used for ethical reasons because that would have required some of the children with the disease to be given placebos. The study has not been published in a scientific journal.
However, Dr. William Rizzo, a pediatrician at the University of Nebraska Medical Center and Children's Hospital of Omaha, said the preliminary findings provide hope.
"The study is flawed by its very nature," said Rizzo, who has also studied the oil. "But it's still really terrific information for those families with children who have inherited the gene and haven't showed symptoms yet."
ALD is caused by a genetic defect that destroys the sheath that covers nerve fibers. It is characterized by the buildup of substances in the blood called long-chain fatty acids.
The theory is that the fatty acids enter the brain and cause inflammation that causes the disease's symptoms. Lorenzo's oil is believed to return acid levels to normal.
"This study shows the oil I invented has therapeutic value," said Augusto Odone. "In most cases, it prevents the symptoms before they start."
Today, Lorenzo, who turned 24 on May 29, is deprived of most of his functions. He communicates through blinking his eyelids and wiggling his fingers, according to a Web site promoting research into a cure.
Michaela Odone died of cancer in June 2000.
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