New Gene Therapy Could Reverse Paralysis in Multiple Sclerosis

The researchers were able to prove that they could prevent and reverse the effects of EAE using the hepatic gene therapy along with the AAV vector and the full DNA coding sequence for MOG. This therapy also caused the production and expansion of the antigen-specific FOXP3+ Tregs. The researchers took a group of mice that had mild to moderate neurological deficits of MS and gave them the gene therapy. The mice showed extensive signs of improvement and protection from EAE. Furthermore, the vector therapy caused a reversal in the pathological and clinical signs of MS. The researchers were also able to effectively protect the mice from the end-stage fatal EAE with a short course of AAV therapy and immunosuppressants along with showing the possibility of restoring mobility and function after severe paralysis.

The researchers believe that they have proven the effectiveness of AAV therapy along with a short course of the immunosuppressant drug, rapamycin, in preventing the debilitating symptoms of MS and reversing the existing symptoms. The effectiveness of the therapy may require that it be administered during the early stages of MS. Researchers also believe that the findings in this study may point to the therapy being effective in treating other autoimmune diseases as well.