Multiple Sclerosis Gene Therapy Being Tested By Researchers

Multiple sclerosis (MS) is a disease whereby the immune system attacks myelin (fat wrappings) around axons, leaving them exposed.  Destruction of myelin generates poor electrical conduction of ions and a lack of communication in the nervous system. Imagine if researchers could find a way to stop the immune response before the disease becomes debilitating.  Researchers at the University of Florida have received a $40,000 grant from the National Multiple Sclerosis Society to test a gene therapy protocol in mice.  This therapy has been developed to help the body not treat itself as a foreign entity, a process referred to as immune tolerance.  This therapy is meant to treat the earliest stages of MS.  If immune tolerance can be re-established, the immune attack could be stopped.  This may prove useful for a wide number patients.

According to Brad E. Hoffman, Ph.D., assistant professor of pediatrics at the University of Florida College of Medicine, “In previous years, we have learned a lot about how to manipulate tolerance using gene therapy.  Tolerance is your body’s way of not responding to substances that would otherwise induce an immune response so you don’t have an immune response to everything. In multiple sclerosis, the body loses that ability to distinguish between self and not-self so it starts to attack its own nervous system cells.”

The National Multiple Sclerosis Society reports approximately 2.3 million people have MS globally.  These patients suffer from vision loss, fatigue, speech slurring, odd sensations and lack of mobility.  Late stage MS can lead to blindness and paralysis.

Gene therapy is often used to correct a bad gene.  In gene therapy, researchers will deliver a gene that codes for a brain protein into the liver by way of the Adeno-associated virus (AAV).  Researchers believe that this gene will be able to induce production of regulatory T cells which will suppress the immune function.

Continue Reading