GLP-1R agonists demonstrate potential to treat Wolfram syndrome in human preclinical models – published online 30/03/2023

Vyron Gorgogietas, Bahareh Rajaei, Chae Heeyoung, Bruno J. Santacreu, Sandra Marín-Cañas, Paraskevi Salpea, Toshiaki Sawatani, Anyishai Musuaya, María N. Arroyo, Cristina Moreno-Castro, Khadija Benabdallah, Celine Demarez, Sanna Toivonen, Cristina Cosentino, Nathalie Pachera, Maria Lytrivi, Ying Cai, Lode Carnel, Cris Brown, Fumihiko Urano, Piero Marchetti, Patrick Gilon, Decio L. Eizirik, Miriam Cnop, Mariana Igoillo-Esteve

Wolfram syndrome is an autosomal recessive disorder caused by mutations in the WFS1 gene. Individuals affected by Wolfram syndrome develop diabetes mellitus, optic nerve atrophy, hearing loss and other neurological problems. There are currently no treatments to prevent or delay the disease. However, glucagon-like peptide 1 receptor (GLP-1R) agonists have been shown to preserve glucose tolerance and reduce neuroinflammation and vision loss in Wfs1-deficient mice and rats. In this issue, Gorgogietas et al (https://doi.org/10.1007/s00125-023-05905-8) report that GLP-1R agonists also improve the function and survival of WFS1-deficient human pancreatic beta cells and neurons. The authors conclude that these data provide a strong preclinical basis to test GLP-1R agonists in individuals with Wolfram syndrome in clinical trials.

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