WASHINGTON, July 11 (UPI) -- More than a decade after the film "Lorenzo's Oil" depicted the true story of a father who develops a compound to treat his son, who has been afflicted with a devastating mental disorder, science finally has caught up with Hollywood.
A new study indicates the oil, a compound comprised of two fats from olive oil and rapeseed oil, may prevent the more severe symptoms of the disease, formally known as X-linked adrenoleukodystrophy or ALD.
As depicted in the 1992 film, Augusto Odone painstakingly developed the compound for his son, Lorenzo, despite the position of mainstream medicine at the time that nothing could be done to halt the progression of ALD. Odone is a co-author of the new study, which appears in the July issue of Archives of Neurology.
Subsequent research has shown the compound is ineffective for reversing the progression of the disease once symptoms start, but the current study suggests it may prevent symptoms from developing in the first place if it is initiated early in the course of life.
"We feel this can have a preventive effect," Dr. Hugo Moser, lead author of the study, told United Press International.
"We think that the data we present are sufficiently strong that we recommend it for all boys who are asymptomatic," added Moser, who is director of the neurogenetics research center at the Kennedy Krieger Institute in Baltimore and a professor at Johns Hopkins University.
ALD predominantly strikes boys. Approximately 15,000 children and adults are afflicted with the disease in the United States and 200,000 worldwide. The inherited disorder can begin either during adulthood or childhood between the ages 4 and 10. The childhood-onset form of the disease is typically more severe, and boys afflicted with it usually die at a very young age.
Drs. Raymond Ferri and Phillip F. Chance, of the University of Washington in Seattle, write in an accompanying editorial that the findings "may establish new standards for the treatment of this degenerative disorder."
They noted, however, that 20 percent of boys with this condition who could benefit from treatment may go undetected. This is because the victims either show no symptoms or have symptoms of other disorders.
Moser's team identified the patients in the study through genetic screening tests that are not usually conducted.
"We are obviously going to miss some people, so we are working very hard to try to develop neonatal screening for ALD" that could be conducted along with other tests routinely done after birth, Moser said.
ALD is caused by a genetic mutation in the ALD gene. Due to the mutation, people with this condition cannot break down specific fats called very-long-chain fatty acids. These fatty acids accumulate in the brain, ultimately destroying the myelin sheath, the fatty covering on the brain's nerve fibers. In the childhood version of the disease, this leads to the gradual loss of all mental and physical function and usually death occurs within several years.
In the study, Moser's team treated 89 boys with ALD between 1989 and 2002 using Lorenzo's oil and a modified diet in an attempt to reduce the levels of the fatty acids to normal. At the time treatment was started, most of the boys were under 7 years old, and none was exhibiting symptoms and all their brains appeared normal on MRI scans.
The team monitored all of the boys closely for seven years, and 74 percent, or 66 boys, appeared healthy at last follow-up. In addition, 40 more boys have been given the oil and diet regimen since the study was completed and "virtually all have achieved normal fatty acid levels," Moser said.
Moser's team reported that 21 of the 89 boys developed brain abnormalities that were detected on MRI and 10 developed neurological problems. The brain abnormalities were associated with an increase in the levels of the very-long-chain fatty acids.
Moser said the therapy should be continued until boys are at least 8 -- when the risk of developing the severe symptoms drops dramatically -- and perhaps beyond that.
"We don't really know how long treatment should be continued," Moser said. It also is not known whether the treatment will prevent the boys from developing symptoms of the adult disease. That would take another 15 years of following them to find out, Moser said.
Boys with signs of inflammation in the brain that can be detected on an MRI scan should not be started on the oil and instead should receive bone-marrow transplants, which have been shown to be very effective.
"We have more than a dozen boys (who received bone-marrow transplants) who have finished college and are working, so the results can be lasting," Moser said.
Although the adult symptoms are not as severe, they can be severely disabling. Moser's team launched a study in adults in March to determine if Lorenzo's oil could be effective at that age.
Odone still gives the oil to Lorenzo, who is now 27. Although severely disabled, Lorenzo has exceeded experts' predictions of his lifespan by 17 years, which his father attributes to the oil.
That is something Moser used to disagree with, but considering Lorenzo's relative longevity, he said he may be changing his position.
"I used to say in the past I know of other patients not treated who have lived this long, but that's not really true anymore," Moser said. "We have certainly seen untreated patients live to their mid-teens and one in their early 20s, but Lorenzo has outlived them all."
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