New Gene Therapy Could Reverse Paralysis in Multiple Sclerosis

Previous researchers have found that when they administer Tregs to a murine model of MS, EAE, they can reduce or halt the neurological symptoms of MS, although the results are temporary. Researchers have also found that they could administer Treg cell injections as a safe treatment method to patients with other autoimmune disorders, such as graft-versus host disease and type 1 diabetes. One of the major challenges of this treatment approach is the inability to generate enough of the cells.

Dr. Hoffman's team of researchers and scientists used the full coding DNA sequence for MOG to deliver the liver-targeting AAV vector and then delivered the therapy to the EAE mouse models. This approach has been attempted in the past, leveraging the tolerogenic nature of the liver using hepatic gene transfer to successfully include robust transgene tolerance in both small and large animals with diseases. MOG was the chosen Treg-stimulating antigen due to the potency of the T-cell response that it induces in patients with MS. 

When Dr. Hoffman's researchers prophylactically administered the gene therapy to the EAE mouse models, they discovered that the mice were protected from developing the disease through an immunosuppressive effect. While the control mice began to show neurological deficits within 10 days following the induction of EAE, the mice who were given the AAV vector showed no clinical signs of EAE.