Gc. Korenke et al., PROGRESSION OF X-LINKED ADRENOLEUKODYSTROPHY UNDER INTERFERON-BETA THERAPY, Journal of inherited metabolic disease, 20(1), 1997, pp. 59-66
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.