A Kennedy Krieger Researcher Announces Groundbreaking Results of 10-Year, International Study of Lorenzo's Oil'

September 28, 2002
Dr. Hugo Moser, director of Neurogenetics at Kennedy Krieger Institute in Baltimore, will present the findings of a major, 10-year study of Lorenzo's Oil' therapy in children with adrenoleukodystrophy (ALD).

GHENT, Belgium - Dr. Hugo Moser, director of Neurogenetics at Kennedy Krieger Institute in Baltimore, will present the findings of a major, 10-year study of Lorenzo's Oil' therapy in children with adrenoleukodystrophy (ALD) today at a scientific symposium at Ghent University.

The multi-site, international clinical trial, headed by Dr. Moser, found that, in a significant number of cases, carefully supervised Lorenzo's Oil therapy in children with early-stage ALD reduced the chance of neurological damage that can later devastate the child and his family. The study is the first to examine the preventive effect of the treatment. Previous studies on the effect of Lorenzo's Oil in ALD patients who are already symptomatic reported only limited benefit.

"It's not an absolute preventive. It reduces the chances of developing the symptoms, but it does not eliminate the chance," Dr. Moser said. "The need to pursue other treatments remains critical. But in the meantime, it's important that we let families and the scientific community know that this positive response has been observed."

Adrenoleukodystrophy, a rare disorder affecting one in 17,000 people worldwide, causes the breakdown of myelin, the fatty substance that acts as insulating material around nerve fibers. Symptoms typically appear by age 5 or 6, and in the worst cases, rapid neurological deterioration ensues, including lost verbal communication, loss of strength and coordination and, eventually, complete breakdown of bodily function.

Lorenzo's Oil, named for the child with ALD made famous in the 1992 movie of the same name, has the remarkable property of correcting, at least in part, one of the main biochemical abnormalities in ALD, the accumulation of very long chain fatty acids.

The study involved 69 boys in the United States and 36 boys in Europe between 1989 and 1999. All of the boys were between the ages of 18 months and 6 years of age and had normal neurological examinations and brain MRIs at the time of enrollment. All of the children in the study were treated with Lorenzo's Oil.

The study found that the boys who successfully followed a strict treatment/diet regimen for two or more years were two-thirds less likely to later develop neurologic or structural brain abnormalities during the two-year to four-year follow-up period when compared to those who, for a variety of reasons, did not successfully follow the regimen. Delay in initiating the therapy increased the hazard of neurologic involvement. The study concludes that Lorenzo's Oil therapy in neurologically uninvolved boys delays the onset of neurological and MRI abnormality, provided that it is accompanied by substantial and prolonged adherence to a strict treatment regimen that must be supervised by a multi-disciplinary team.

The results of the study are being presented today at a scientific symposium entitled, "Peroxisomal Disorders and Regulation of Genes," at Ghent University. The organizers of this symposium have a long-standing interest in therapy and ten years ago initiated the European component of the study. The American component was supported by a grant from the Office of Orphan Drug Products of the Food and Drug Administration. The study results have been submitted to the FDA for review.

Kennedy Krieger has been at the forefront of ALD research for decades. In 1981, Ann Moser, Dr. Moser's wife, developed a diagnostic test for the disorder, and since then they have diagnosed more than 6,000 patients all over the world. In 1993, Dr. Moser was part of the team of researchers that identified the gene that causes the disorder.

Kennedy Krieger Institute is dedicated to helping children and adolescents with disabilities resulting from disorders of the brain achieve their potential and participate as fully as possible in family, community and school life.

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