Adrenomyeloneuropathy is an X-linked recessive disorder characterized by peripheral neuropathy and spastic paraparesis with or without Addison's disease, resulting in significant limitation of daily activities and function. Considerably elevated levels of very long chain fatty acids (VLCFA) can be detected in peripheral blood, cultured skin fibroblasts and cultured amniocytes. The disorder is uniformly progressive and is untreatable. Since earlier reports of the use of VLCFA-restricted diet enriched with oleic acid had suggested that this novel dietary therapy might hold promise in AMN patients, obligate heterozygotes, and asymptomatic at-risk individuals, we undertook collaborative research with investigators at the John F. Kennedy Institute, Baltimore, MD.
The aim of this study was to see whether normalization of elevated levels of VLCFA through dietary VLCFA-restriction, and supplementation with glycerol trierucate (GTE) and glycerol trioleate (GTO) oils, will significantly improve peripheral nerve function and prevent the progression of disability from spinal cord involvement. One patient was included in this protocol from our center. The serum VLCFA level, which was four-times the control value at baseline, normalized after 1 year of dietary therapy. The patient's neurological status, as assessed by clinical examination and MRI brain scan was relatively unchanged after up to 4 years of treatment. The lack of rise in serum cortisol level following ACTH stimulation was unaffected after 2 years of therapy. There were no adverse effects after up to 4 years of therapy as determined by routine blood studies, body weight monitoring, serial EKG's and echocardiography. The patient voluntarily discontinued therapy after 4 years because of financial reasons.
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