UF Researchers Make Breakthrough in Multiple Sclerosis Research

Sep 21, 2017

Researchers at the University of Florida have developed a new therapy that they hope could lead to a cure for multiple sclerosis.

The process uses a gene therapy, which researchers have been testing on mice. Between 71 percent and 81 percent of the mice had a near-complete remission of the disease, said Dr. Brad E. Hoffman, an associate professor in the departments of pediatrics and neuroscience at UF’s College of Medicine.

Hoffman said the results surprised researchers as did the fact that the mice continued to show no signs of the disease seven months after treatment.

“We were very impressed with the results,” Hoffman said.  “We expected there to be a reversal or at least a dampening of the disease. We were quite surprised when we had the level and magnitude and the robustness of the reversal and how long the reversal and protection lasted in these mice.”

Multiple sclerosis is an incurable neurological disease that affects about 2.3 million people worldwide. The immune system causes nerve fibers to misfire, causing muscle weakness and problems with vision, speech and muscle coordination.

The therapy suppresses the immune response that causes the disease. Using a gene in brain protein and a drug used in organ transplant recipients, the researchers caused a near-complete remission of the disease in the mice. Their findings were published today in the journal Molecular Therapy.

Most of the technologies involved in the process have been used in the treatment of other diseases. Still, the process involves injecting a protein from the brain into the liver, which could delay its use in clinical trials on humans until other tests are completed.

Hoffman expects clinical trials to start in three to five years and if everything goes well, the treatment could go to market in 10 years.

“If we can provide a long-term treatment for these patients that has very little side effects and they have a higher quality of life, that would be a successful result for me,” Hoffman said.