Drug may block death of brain cells in MS

June 14, 2018
Researchers at the University of Alberta discovered a unique process of brain cell death that affects the cells that are most vulnerable in multiple sclerosis. After identifying the process called pyroptosis, or fiery death, the researchers were able to block the enzyme in the brain that is responsible for it, using a drug that could potentially treat MS.

Pyroptosis is a type of programmed cell death that is associated with inflammation, but its role in MS was previously unknown. Importantly, the study’s authors were able to show pyroptosis in both brain tissues from MS patients and in lab models of MS. The researchers found that the drug known as VX-765 protected oligodendrocytes, the cells that insulate nerves in the brain and are susceptible to damage in MS. VX-765 is currently in clinical trials for epilepsy.

The study’s authors said this could be a game changer, because they discovered a fundamental mechanism by which brain cells are damaged in MS that couples inflammation with neurodegeneration and that the drug is already known to be safe in humans. They believe identifying this mechanism also opens the doors to new indicators for monitoring disease progression of MS, which has been challenging because symptoms can vary widely between patients.

The researchers said that their findings identify a clinically relevant novel pathway that opens the doors to new therapeutic targets that prevent cell damage.

The study was published in the journal PNAS.

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