Lorenzo's Oil -- Hope and Disappointment

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Abstract
The rational development of therapy for a disease depends critically on knowledge about its pathogenesis. In the absence of such insight, physicians are vulnerable to the prevailing winds of opinion and practice, each liable to shift from one moment to another. Even when a therapeutic approach seems logical, it must still be proved effective -- not by anecdotal reports, surrogate responses, or public opinion, but by the scientific method and the performance of clinical trials. Such is the case for dietary therapy of adrenoleukodystrophy, a rare and devastating neurodegenerative disease. A recent popular film, Lorenzo's Oil, prematurely implied that dietary . . .