The cysteine-reactive small molecule ebselen facilitates effective SOD1 maturation

Journal: Nature Communications

Published: 2018-04-27

DOI: 10.1038/s41467-018-04114-x

Affiliations: 7

Authors: 11

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Research Highlight

New drug lead for motor neuron disease

© koto_feja/Getty

© koto_feja/Getty

An antioxidant drug could offer a new treatment for amyotrophic lateral sclerosis (ALS), the neurodegenerative disorder commonly known as motor neuron disease.

A team that included University of Wollongong scientists screened for drugs that interact with SOD1, a pathogenic protein responsible for some familiar forms of ALS. They found that ebselen — a drug with anti-inflammatory and antioxidant properties that has already been tested in patients with other brain disorders — helped correct SOD1 misfolding and prevented the protein from aggregating.

Notably, ebselen binding did not interfere with SOD1’s normal function in human cells, and the drug stabilized different mutant forms of the protein.

Given the drug’s track record of safety in people, clinicians could now quickly evaluate ebselen as a therapeutic to treat familial ALS caused by SOD1 mutations.

Supported content

  1. Nature Communications 9, 1693 (2018). doi: 10.1038/s41467-018-04114-x
Institutions FC
Institute of Integrative Biology, University of Liverpool, United Kingdom (UK) 0.36
Centro di Ricerca di Risonanze Magnetiche (CERM), UNIFI, Italy 0.23
Illawarra Health and Medical Research Institute (IHMRI), UOW, Australia 0.18
Department of Chemistry, University of Liverpool, United Kingdom (UK) 0.09
Consorzio Interuniversitario Risonanze Magnetiche di Metallo Proteine (CIRMMP), Italy 0.05
Department of Biomedical, Experimental and Clinical Sciences "Mario Serio", UNIFI, Italy 0.05
Department of Chemistry, UNIFI, Italy 0.05

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