Compassionate usage of Lorenzo's Oil in peroxisomal disorders

Principal Investigator: Gerald Raymond

Lorenzo's oil, a mixture of glyceryl trioleate and glyceryl trierucate, lowers very long chain fatty acids in X-linked adrenoleukodystrophy. There is preliminary data that lowering very long chain fatty acids may prevent the development of demyelination. The use in other peroxisomal disorders has not been evaluated although there may be potential ability to reduce some of the very long chain fatty acids. Given the rarity in these other disorders, a true study is unlikely, but periodically the compassionate usage of the agent may be warranted, as there are no alternative therapies.