The Clinical Use of N-Acetylcysteine in Other Medical Disorders

Frye, Richard E.

doi:10.1007/978-981-10-5311-5_18

Cited by 1 publication

(2 citation statements)

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“…Oxidative stress mechanisms have also been implicated in affective disorders [5]. It is not surprising that NAC has been studied extensively in neurodegenerative and psychiatric disorders, albeit with contradicting results [6,7]. Redox imbalance; characterized by antioxidant depletion and the accumulation of ROS and manifested into common themes such as neuronal damage, neurodegeneration, or inflammation, is implicated in the pathophysiology of many rare (e.g., amyotrophic lateral sclerosis (ALS), Huntington's disease), and common diseases (e.g., Alzheimer's disease (AD), Parkinson's disease (PD)) [3,8,9].…”

Section: Introductionmentioning

confidence: 99%

“…Thus, it is hypothesized that the treatments that can directly or indirectly enhance the antioxidant defenses may provide health benefits in these seemingly divergent yet connected diseases [9]. Although it is reasonable to expect that supplementation of cysteine or GSH as such would serve to enhance the antioxidant pool; precursors and derivatives such as NAC are preferred due to their ease of formulation, stability, and superior attributes relevant to palatability and odor [7].…”

Section: Introductionmentioning

confidence: 99%

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N-acetylcysteine Pharmacology and Applications in Rare Diseases—Repurposing an Old Antioxidant

2023

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N-acetylcysteine (NAC), a precursor of cysteine and, thereby, glutathione (GSH), acts as an antioxidant through a variety of mechanisms, including oxidant scavenging, GSH replenishment, antioxidant signaling, etc. Owing to the variety of proposed targets, NAC has a long history of use as a prescription product and in wide-ranging applications that are off-label as an over-the-counter (OTC) product. Despite its discovery in the early 1960s and its development for various indications, systematic clinical pharmacology explorations of NAC pharmacokinetics (PK), pharmacodynamic targets, drug interactions, and dose-ranging are sorely limited. Although there are anecdotal instances of NAC benefits in a variety of diseases, a comprehensive review of the use of NAC in rare diseases does not exist. In this review, we attempt to summarize the existing literature focused on NAC explorations in rare diseases targeting mitochondrial dysfunction along with the history of NAC usage, approved indications, mechanisms of action, safety, and PK characterization. Further, we introduce the research currently underway on other structural derivatives of NAC and acknowledge the continuum of efforts through pre-clinical and clinical research to facilitate further therapeutic development of NAC or its derivatives for rare diseases.

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