Researchers at the University of Alberta have discovered a unique process of brain cell death that affects the cells that are most vulnerable in multiple sclerosis (MS).
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.
“This could be a game changer, because we discovered a fundamental mechanism by which brain cells are damaged in MS that couples inflammation with neurodegeneration,” said Chris Power, a neurologist, lead author of the study and co-director of the UAlberta MS Centre. “The drug is already known to be safe in humans.”
MS is a common disease of the brain and spinal cord that affects people in the prime of their life. There is currently no curative treatment for MS and its cause remains unknown. On average, 11 people with MS are diagnosed daily and Alberta has one of the highest rates of the disease in the world.
The publication of the study in PNAS marks the first molecular analysis of pyroptosis in the human brain. Pyroptosis is a type of programmed cell death that is associated with inflammation, but its role in MS was previously unknown. Importantly, Power’s lab was able to show pyroptosis in both brain tissues from MS patients and in lab models of MS.
“The study’s findings make a key contribution to the MS field in identifying a novel mechanism that contributes to progression in MS,” said Karen Lee, vice president of research at the MS Society of Canada. “The MS Society of Canada is encouraged by the results of this study and what it means for people living with MS—hope for another avenue through which treatment options can be explored to stop MS in its tracks.”
Power’s lab 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.