Anavex Life Sciences Corp (AVXL)

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“The findings are very important and also quite exciting and interesting,” said Fumihiko Urano, a Wolfram Syndrome clinical researcher from Washington University School of Medicine who was not involved in this study. “It’s one of the first articles showing that targeting MAM can improve behavior phenotypes in a mouse model of Wolfram Syndrome.”

The team behind the new study didn’t pick an S1R agonist to test out of thin air. They previously showed that the S1R agonist blarcamesine protected mice against Alzheimer’s disease, a neurodegenerative condition partially caused by mitochondrial dysfunction and increased ER-mitochondrial contact (2). The drug, produced by Anavex Life Sciences, is currently undergoing a phase III clinical trial to test its efficacy in patients with Alzheimer’s disease and a phase II trial in patients with Parkinson’s disease, another neurological condition with etiology linked to misbehaving mitochondria.

Delprat and his team demonstrated that using another S1R agonist, PRE-084, to activate S1R restored the lost contacts between the ER and mitochondria in fibroblasts taken from patients with Wolfram Syndrome. The wolframin-mutation-carrying fibroblasts had fewer contact points between the ER and mitochondria than control fibroblasts, resulting in underactive mitochondria. After treatment with PRE-084, the mitochondria in the Wolfram Syndrome patient-derived fibroblasts had more contact points with the ER and produced more energy as measured by ATP production.

https://www.drugdiscoverynews.com/a-drug-that-protects-against-alzheimer-s-disease-may-help-with-wolfram-syndrome-15484