PROGRESSION OF X-LINKED ADRENOLEUKODYSTROPHY UNDER INTERFERON-BETA THERAPY

The cerebral phenotype of X-linked adrenoleukodystrophy (ALD) is a rap idly progressive neurodegenerative disorder characterized by a cerebra l inflammatory response and elevated very long-chain fatty acids (VLCF A). Interferon-beta (INFB) is known to suppress the synthesis of tumou r necrosis factor alpha and interferon-gamma, which have been reported to be elevated in the margin of the areas of demyelination in ALD bra ins. We report on treatment with interferon-beta in 8 patients with ce rebral ALD, who additionally received glycerol trioleate/glycerol trie rucate. INFB-1a (Rebif, Serono, Switzerland) was given subcutaneously once a week, 3 million units for the first 3 months and 6 million unit s for the next 9 months. All patients showed an unimpeded progression of neurological symptoms during INFB therapy. Therapy was stopped with in 6 months in 4 patients because of the fast neurological deteriorati on with loss of walking. In all patients the MRI demonstrated a progre ssion of demyelination with a qualitatively unchanged gadolinium enhan cement. Further studies are needed to elucidate the pathomechanism of demyelination in ALD in order to find an effective therapy for cerebra l ALD patients.