The mouse version of multiple sclerosis can be sent into complete remission and prevented with a new gene therapy technique that stops the disease’s immune response. University of Florida Health researchers say the gene therapy technique they used on mice gives them a great deal of hope that the therapy could work to completely reverse multiple sclerosis in humans. This is stunning news that could give hope to around 2.3 million people around the world who have multiple sclerosis, a debilitating and progressive autoimmune disorder. Additionally, researchers say that the new gene therapy also has significant potential to treat other autoimmune disorders.
Multiple sclerosis is the most common neurological disease among young adults, Science Daily reports. It has been incurable, but perhaps not for long. This disorder begins when a person’s own immune system begins to attack the myelin sheath that surrounds nerve fibers. Once that happens, the nerves misfire and people begin to experience vision disturbances, speech problems, and muscle weakness. The disease can lead to paralysis and has the potential to significantly impact a person’s quality of life.
“Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis” was published in Molecular Therapy this month and details the research that uncovered this promising, potential therapy for MS.
They report that they used an adeno-associated virus, believed to be harmless, in order to get a gene into the livers of animal models. They targeted the liver because the liver is an organ that is able to induce immune tolerance. The gene that was delivered into the livers of the mice is known to be responsible for a particular brain protein. Myelin oligodendrocyte glycoprotein, the one delivered into the liver by way of a reportedly harmless virus, showed so much promise that seven months after the therapy was introduced, the animal models still showed no signs of autoimmune encephalomyelitis, the mouse version of multiple sclerosis.
“Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,” reported Dr. Brad E. Hoffman of the University of Florida College of Medicine.
When this gene therapy was combined with an existing drug, the treatments produced near-complete remission and prevented the development of the rodent version of multiple sclerosis, Science Daily reported. When the therapy was used alongside a drug that has been used on heart stents and to prevent transplant rejection, the new therapy was reportedly even more impressive. With this drug, among test groups, 71 percent and 80 percent of the subjects experienced near complete remission of symptoms after they had paralysis in their hind limbs.
There is still more research that will need to be done before clinical trials on humans can begin, but Hoffman and his team are extremely optimistic that this new therapy will be able to provide “long-term remission for people and a long-term quality of life” for people who have been suffering from multiple sclerosis and possibly for other autoimmune diseases as well.
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