Potential treatment for MS shows neuroprotective properties

A novel treatment for multiple sclerosis (MS) blocked nerve cell damage in experimental models, which suggests the promise of preventing disability from this disease, according to results of a study published online September 4, 2015, in Journal of Neuroinflammation.

In this study, scientists targeted an enzyme, granzyme B, which cytotoxic cells release as a “lethal hit” against neurons in patients with MS. To combat this enzyme, the scientists developed a granzyme B inhibitor, serpina3n, which they tested in human in vitro and mouse in vivo experiments. They found that serpina3n reduced inflammatory-mediated neurodegeneration without significantly suppressing the immune system response.

“We can interfere with some of the weapons these cytotoxic cells use to induce damage to the nerve cells in the brain, but without disrupting the other positive functions that these cells have,” said senior study author Fabrizio Giuliani, MD, Associate Professor of Neurology at the University of Alberta, Edmonton, Canada. “This molecule, serpina3n, will block the damage caused by granzyme B that induces the neurodegeneration in this disease, and the neurodegeneration strongly correlates with the disability.”

Interfering in the early stages of inflammation in the brains of patients with MS could at least slow progression of the disease. “In our models, we haven’t seen that the disease disappears,” Dr. Giuliani said. “The disease is still there, the inflammation is still there, but there’s not as much damage in the nerve cells that would induce a permanent disability.”

The researchers are now preparing experiments using human analogues of serpina3n that will further test the impact of inhibiting granzyme B in patients with multiple sclerosis.

“This could eventually open the door to a new stream of treatments,” Dr. Giuliani said. “If we can induce neuroprotection, there is a good possibility we can decrease the rate of disability that is associated with inflammation in the brain. If it works as we think, this will make an impact on the treatment of MS patients.”