EFFECT OF GLYCEROL TRIERUCATE ON CLINICAL COURSE OF ADRENOLEUKODYSTROPHY

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. A published report from our group has provided evidence that Lorenzo's oil (a 4:1 mixture of glyceryl trioleate and glyceryl trierucate), when administered to boys with X-ALD less than 6 years old, were neurologically asymptomatic and had a normal brain MRI, had a reduced risk of developing brain MRI and neurological abnormalities. However, the follow-up period for this study was relatively short and the method of assessing the changes of plasma VLCFA levels was not optimal. Because of these limitations we carried out additional studies of 89 patients with longer follow-up (6.9+/-2.7 years) and used a more sensitive method (length adjusted area under the curve) to assess the alterations in plasma very long chain fatty acid levels.