Once a skeptic, Dr. Hugo W. Moser of the Kennedy Krieger Institute has recommended that all children predisposed to a deadly neurological illness take Lorenzo's oil, a treatment found by desperate parents hoping to save their ailing son.
He said the oil, made famous by the 1992 movie of the same name, should be taken by boys who carry blood factors that place them at risk for adrenoleukodystrophy (ALD) but have not yet developed symptoms.
Preliminary data suggest that the treatment is safe and may be capable of delaying or preventing the onset of symptoms, Dr. Moser said. The disease, passed on by mothers, afflicts only males.
"My strong recommendation is that these persons ought to be informed about the diet and then be placed on it if they choose," Dr. Moser said. The "diet" consists of oral doses of Lorenzo's oil, which is derived from olive and rapeseed oils, and an array of foods that includes those low in saturated fats.
Dr. Moser said his study of 53 symptom-free boys, reported this week in the journal International Pediatrics, gave strong but not yet conclusive evidence that Lorenzo's oil forestalls the onset of ALD.
In its most severe form, the disease attacks boys between the ages of 4 and 10, robbing them of the ability to walk, see and speak in as little as two years, and eventually killing them.
For Dr. Moser, the recommendation may mark the beginning of a wider shift toward accepting the oil as a weapon against ALD. He said another arm of his research suggests that Lorenzo's oil ** slows the degeneration of some boys who began taking it after their symptoms emerge.
Dr. Moser said he was still analyzing the data and cautioned that it was premature to draw solid conclusions. Even if the oil proves effective in slowing the decline of afflicted boys, he said, it is unlikely that the oil by itself will substantially improve the lives of afflicted children.
The 69-year-old doctor, who directs mental retardation research at the Baltimore hospital, said the oil may prove more effective against advanced ALD if combined with other drugs. Last week, he received government approval to administer Lorenzo's oil alongside beta interferon, a new drug used by people afflicted with multiple sclerosis.
Meanwhile, Augusto Adone, the former World Bank economist who pioneered use of the oil for his ailing son, said the research so far vindicates the position he has taken for several years.
The movie, "Lorenzo's Oil," depicts the struggle waged by Augusto and Michaela Adone to save the life of their son, Lorenzo, a gifted boy stricken with ALD in 1984. Lacking any medical training, they pored over scientific journals and within two years pieced together the theory that a refined oil may halt or slow the disease.
After seven years of treatment, the Adones contend that therapy stopped Lorenzo's decline and partly reversed it.
The disease, which afflicts one or two people in 100,000, is caused by the buildup of substances called long-chain fatty acids in the bloodstream. The acids destroy protective myelin that covers nerve fibers in the brain, hindering their ability to control such functions as speech, movement and even swallowing.
Lorenzo's oil is thought to reduce levels of the fatty acids to normal. Its effects on symptoms are still under study.
Dr. Moser, once Lorenzo's doctor, was initially skeptical about the oil's benefits. He feared that it was toxic and suspected that reductions in fatty acids were temporary fluctuations that occur naturally.
Nonetheless, he has been conducting studies on Lorenzo's oil since the late 1980s in an effort to gauge its effectiveness, testing the oil on more than 300 children so far. Families from across the country obtain the oil from Dr. Moser, who has government approval to write prescriptions for research purposes.
Thirteen years ago, he developed a blood test to identify people with the biochemical markers for the disease. With this, he started testing the oil on boys who were predisposed to ALD but had not yet developed symptoms -- as well as on boys who were already sick.
In his study of 53 boys without symptoms, two boys died and three others developed symptoms of the severe disease after taking Lorenzo's oil. In contrast, 32 boys were well, while some others had developed mild symptoms. The boys had taken the oil an average of 38 months when the results were compiled.
Dr. Moser said the results are difficult to analyze because the study lacked a control group of untreated boys for comparison purposes. But he said historical data indicate that at least 31 percent of predisposed boys develop symptoms of the severe disease by age 10.
In the United States, families wishing to administer Lorenzo's oil to their sons can do so only by enrolling in the study centered at the Kennedy Krieger Institute. Patients living outside Baltimore can obtain it through their doctors, who in turn contract Dr. Moser.