“Supplement put together by couple with ailing child appears to delay rare, deadly illness
By ALEX RAKSIN
Los Angeles Times
Lorenzo’s Oil — a dietary supplement concocted by Virginia parents in a last-ditch effort to save their son’s life — appears to delay the effects of a rare genetic disease in boys if taken early and in concert with a low-fat diet, according to the first long-term study of the treatment.
The Archives of Neurology study followed 89 boys with the genetic mutation for childhood cerebral adrenoleukodystrophy and found that 74 percent of them remained healthy after 13 years.
The results “are very favorable,” said Hugo W. Moser, the pediatric neurologist at Baltimore’s Kennedy Krieger Institute who led the study. “In the general population, half of the boys with the genetic mutation develop severe neurological abnormalities by age 7.”
The disease, which strikes about one child in every 30,000, develops when long-chain saturated fatty acids accumulate in the blood.
The acids gnaw away at the protective myelin sheaths that cover the brain’s nerve fibers, depriving patients of the ability to speak, see and move within five years. It usually leads to death within seven years.
Kitchen creation
Most physicians wait for symptoms to appear before treating the disease. There have been no effective therapies.
The study “provides real hope that Lorenzo’s Oil can delay the onset of symptoms,” said Raymond Ferri, a pediatric neurologist at the University of Washington, who was not connected with the research. “That means a lot, because if the symptoms start older, we know, then the course of the disease is much slower and people do much better.”
Researchers have known since 1989 that Lorenzo’s Oil can reduce dangerous fatty acids. The oil, a combination of fats from ordinary kitchen items such as olive oil and rapeseed oil, was first developed by Augusto and Michaela Odone, whose search for a cure for their son Lorenzo inspired the 1992 movie Lorenzo’s Oil.
Lorenzo, now 27, is alive, although he can’t move or speak.
The study began in 1989, when doctors began administering the oil in pill form to boys, then ages 4 and 5, with the mutation on their X chromosome that leads to the disease.
When the boys were examined in 2002, 66 had normal neurological results and brain MRIs.
Based on the evidence, Moser said, “we are recommending that doctors administer it to all boys with biochemical evidence of the disorder until the child reaches early adolescence, when the risk of developing it declines tenfold.”
‘May not be a miracle’
Moser acknowledged that the study wasn’t as definitive as a randomized clinical trial. There was no control group, because Moser did not want to give some patients a placebo.
Based on the results of the study, he has started 40 more boys on the treatment and is recommending that doctors administer the oil as a prophylactic because it has “no serious adverse side effects and probably dramatic benefits.”
“Clearly it’s superior to the present situation in which doctors treat the disease only when its symptoms have become evident and, tragically, irreversible,” he said.
Augusto Odone said the study validated years of work he and his wife had done.
She died in 2000 of lung cancer.
“The new research gives me the vindication I’ve long sought,” he said from his home in northern Virginia, where he is recovering from a bone fracture.
“Lorenzo’s Oil may not be a miracle for everyone with the disease, but for many, it works.”
“