UF researchers prevent, reverse multiple sclerosis in mice

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GAINESVILLE, Fla -- In the novel "Flowers for Algernon," a man undergoes an experimental medical treatment that has been tested on mice.

That testing hasn't reached the human stage yet in a unique treatment discovered by University of Florida researchers who think they could someday help treat patients suffering from a currently incurable disorder.

Dr. Brad Hoffman, a clinical immunologist at UF, spent his Thursday afternoon answering questions over the phone from reporters around the world.

He sat at a conference table in a room at the Cancer Genetics Research Complex on campus.

Multiple Sclerosis (MS) is the most common auto immune disorder affecting the Central Nervous System.

More than a decade in the making, Dr. Hoffman and his team discovered a unique gene therapy technique that prevented and reversed MS in mice.

"In MS you have these rogue cells that go and attack the body. They're attacking a very particular brain protein that during this attack strips away a protective protein around the neuron," Hoffman said.

The team's technique works by putting a piece of DNA into a harmless virus and then injecting the virus into a mouse's liver. The researchers harness the liver's natural ability to induce tolerance and suppress immune responses.

The organ can then target the rogue cells that cause muscle paralysis and other damage. That alone was a good start, but when combined with another drug for organ transplant recipients called rapamycin -- the results were astonishing.

"We could take a mouse that was almost completely paralyzed and within just two weeks we could completely reverse the disease."

With no recurrence. Before the therapy can be tested in humans during a clinical trial, further research involving other preclinical models will be needed.

"If everything goes well and the testing, are we looking at something that could be out to market in 10 years, 15 years?" asked a reporter.

"It'd be speculative, but I would hope that would be a realistic timeline," Hoffman responded.