N-Acetylcystein (NAC) bei Retinitis pigmentosa

Zusammenfassung

Retinitis pigmentosa (RP) ist eine genetisch bedingte degenerative Netzhauterkrankung, die zum fortschreitenden Untergang der Photorezeptoren und letztlich zur Erblindung führen kann. Oxidativer Stress spielt eine zentrale Rolle in der Pathogenese der RP. In der vorliegenden narrativen Übersichtsarbeit wird die Wirkung von N-Acetylcystein (NAC), einem Antioxidans und Glutathionvorläufer, auf den Krankheitsverlauf bei RP-Patienten zusammengefasst. Das NAC kann oxidativen Stress in der Netzhaut reduzieren und zeigt in präklinischen Modellen potenziell neuroprotektive Effekte. Erste klinische Studien deuten auf eine Verbesserung der Sehschärfe und makulären Funktion unter NAC-Therapie hin. Die derzeit laufende multizentrische Phase-III-Studie „NAC Attack“ zielt darauf ab, die langfristige Wirksamkeit und Sicherheit von NAC bei RP zu überprüfen.

Abstract

Retinitis pigmentosa (RP) is a genetically mediated degenerative retinal disease that leads to progressive photoreceptor degeneration and, ultimately, blindness. Oxidative stress plays a central role in the pathogenesis of RP. This study examines the effect of N-acetylcysteine (NAC), an antioxidant and glutathione precursor, on the disease progression in RP patients. NAC can reduce oxidative stress in the retina and has shown potential neuroprotective effects in preclinical models. Initial clinical studies indicate improvements in visual acuity and macular function with NAC therapy, though no structural changes have yet been demonstrated. The ongoing multicentre Phase III trial “NAC Attack” aims to evaluate the long-term efficacy and safety of NAC in RP.

Schlüsselwörter

Retina - Retinitis pigmentosa - Netzhautdegeneration - Antioxidanzien - genetisch bedingte Netzhauterkrankungen

Keywords

retina - retinitis pigmentosa - retinal degeneration - antioxidants - inherited retinal diseases

Publication History

Received: 09 November 2024

Accepted: 25 January 2025

Accepted Manuscript online:
26 January 2025

Article published online:
24 March 2025

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