DIETARY THERAPY OF ADRENOLEUKODYSTROPHY - PRELIMINARY-RESULTS OF A LONG-TERM STUDY

Adrenoleukodystropy (ALD) is a X-linked recessive disorder of peroxiso mal metalbolism with several distinct clinical manifestations. The chi ldhood form is characterized by extensive inflammatory demyelination i n the central nervous system and/or adrenal insufficiency. Patients wi th the later onset form (adrenomyeloneuropathy) typically develop a sl owly progressive peripheral neuropathy and/or adrenal insufficiency in adulthood. As a consequence of the underlying biochemical defect in p eroxisomal beta-oxidation, very long chain fatty acids (VLCFA) accumul ate in the plasma of patients, asymptomatic carriers, and heterozygote s. The combination of a diet low in VLCFA with the oral application of GTE/GTO-oil (glycerotrioleate and -trierucate, lorenzo's oil) decreas es or even normalizes plasma VLCFA levels in greater than 90% of patie nts. One goal of dietary therapy in ALD is to assess if diet treatment can prevent, postpone or lessen subsequent neurological disability in currently asymptomatic patients. Preliminary results indicate that af ter at least 12 month of diet about 60% of our patients showed no chan ges in their clinical and neuroradiological status. This group remaine d stable over a total cumulative period of 763 months. 14 patients had signs of at least mild progression on neurological examination and/or neuroimaging studies. The disease progressed significantly in only 6 patients (17%).