PA Medical News about Niemann-Pick Disease, Type C

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Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C
Journal of Child Neurology, 10/22/09

Patterson MC et al. – Miglustat stabilized neurological disease progression in pediatric patients with Niemann–Pick disease type C, with comparable safety and tolerability to that observed in adults and juveniles.

Exclusive Author Commentary
Marc C. Patterson, 10/26/09

Only two therapeutic studies have been performed in human Niemann-Pick disease, type C (NPC). The first (Neurology 1993;43:61-64) examined the effect of cholesterol-lowering agents on hepatic and plasma cholesterol levels. Although the levels of cholesterol were reduced to varying degrees, subsequent follow up showed no evidence of therapeutic benefit. The second study in NPC was the trial of miglustat, which was the first to suggest that a drug could modify the course of human NPC as had been shown in animal models. The pediatric arm of this study was uncontrolled, but showed similar trends in outcome measures to the controlled study which enrolled patients over 12 years of age. Thus, miglustat may slow disease progression in NPC. In the short to medium term, combination therapy with miglustat and agents acting via different mechanisms may yield the best results. Curative therapy for NPC will require approaches that correct the primary defect, and must be considered a long term goal at present.

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