ALSUntangled #82: N-acetylcysteine

N-acetylcysteine is a thiol-containing compound and a precursor of glutathione, with mechanistic plausibility for ALS, including reducing oxidative stress, regulating neuroinflammation, and mitigating mitochondrial dysfunction. Preclinical studies have yielded conflicting results on whether N-acetylcysteine can delay the onset of motor impairment and prolong survival in ALS mouse models. Several case studies of oral or subcutaneous administration of N-acetylcysteine in patients with ALS did not demonstrate convincing benefits. Clinical trials to date have also failed to demonstrate efficacy in slowing ALS progression. While N-acetylcysteine shows theoretical promise, further research is needed to clarify its therapeutic role in ALS. At present, ALSUntangled does not support the use of N-acetylcysteine as a treatment to slow ALS progression.

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Publication Details

Journal
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Published
2026-03-04
DOI
https://doi.org/10.1080/21678421.2026.2638590
Primary Topic
Amyotrophic Lateral Sclerosis Research
Type
article
Field-Weighted Citation Impact
0.00
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article

ALSUntangled #82: N-acetylcysteine

E. Pioro, Bhavya Bakshi, W Paul, David Shirilla et al.
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Amyotrophic Lateral Sclerosis Research
article

ALSUntangled #82: N-acetylcysteine

E. Pioro, Bhavya Bakshi, W Paul, David Shirilla, Kara A. Dyckman, Juliette Foucher, Sarah R. Breevoort, Vincent K. Chang, Sandeep Yerraguntla, Carlayne E. Jackson, Olimpia Carbunar, G. H. Carter, Andrew Brown, Richard Bedlack, G. I. Nathaniel, Bertorini Te, Morgan Beauchamp, Olivia Wang, Ethan Greenstein, Elia Tito, Sabrina Paganoni, Gary L. Pattee, Natasha J Olby, Dylan Ratner, Yuyao Sun, Keshav Chandran, Anusha Rao, Xiaoyan Li, Nicholas Maragakis, Michael Benatar, Basil Elsharif, Olivia Allen, Christopher McDermott, Nan Jiang
article en

Abstract

N-acetylcysteine is a thiol-containing compound and a precursor of glutathione, with mechanistic plausibility for ALS, including reducing oxidative stress, regulating neuroinflammation, and mitigating mitochondrial dysfunction. Preclinical studies have yielded conflicting results on whether N-acetylcysteine can delay the onset of motor impairment and prolong survival in ALS mouse models. Several case studies of oral or subcutaneous administration of N-acetylcysteine in patients with ALS did not demonstrate convincing benefits. Clinical trials to date have also failed to demonstrate efficacy in slowing ALS progression. While N-acetylcysteine shows theoretical promise, further research is needed to clarify its therapeutic role in ALS. At present, ALSUntangled does not support the use of N-acetylcysteine as a treatment to slow ALS progression.

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Tulane University (US), Campbell University (US), Karolinska University Hospital (SE), Harvard University (US), North Carolina State University (US), University of British Columbia (CA), University of Kentucky (US), Johns Hopkins University (US), University of Miami (US), Eastern Virginia Medical School (US), University of Tennessee Health Science Center (US), The University of Texas at San Antonio Health Science Center (US), Duke University (US), Medical College of Wisconsin (US), Augusta University (US), University Medical Center (US), University B.T. & Evening College (IN), University of Alabama at Birmingham (US), Massachusetts General Hospital (US), Compumedics (Australia) (AU), Goucher College (US), Digital Health Cooperative Research Centre (AU), Marymount University (US), Old Dominion University (US), University of Sheffield (GB), The University of Texas Health Science Center at Houston (US), Georgia Highlands College (US)
Openalex Percentile: Top 40%
Amyotrophic Lateral Sclerosis Research
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