JUPITER, Fla. - Researchers at Scripps Florida say they've found a way to stop multiple sclerosis in mice.
In a study published this week in the journal Nature, Scripps scientists say they've developed a compound that stops MS by knocking out TH17, a type of white blood cell that malfunctions in patients with MS and other autoimmune diseases such as rheumatoid arthritis and lupus.
If it works in humans, the new treatment, known as SR1001, would have a couple of advantages over existing MS drugs, said Tom Burris, a professor in the Department of Molecular Therapeutics at Scripps Florida.
First, it could be taken as a pill rather than injected. Second, the compound would attack only TH17 cells while sparing other disease-killing cells.
"Right now, the treatments that are out there suppress the entire immune system, and that comes with a lot of side effects," Burris said.
It's unclear whether the treatment is a cure or simply stalls the disease, he said.
An estimated 400,000 Americans have MS, according to the Multiple Sclerosis Foundation in Fort Lauderdale. MS, which prevents nerve cells in the brain and spine from communicating, is most commonly diagnosed in women between 20 and 40.
"The hunt for a new MS drug is very urgent," said Kasey Minnis, director of operations at the Multiple Sclerosis Foundation. "There's no medication that's super-effective in all people with MS."
The new treatment is promising enough that it has garnered interest from drug companies, Burris said.
"We have a lot of interest from biotech and pharma companies, and we're trying to strike a deal with someone," he said.
Scripps could license SR1001 to one of those companies within a few months, Burris said.
TH17 cells produce interleukin-17, a natural molecule that can cause inflammation.
"In these autoimmune diseases, the body is tricked into attacking itself," Burris said.
When scientists blocked signals from the TH17 cells in mice, their MS disappeared.
Dr. Daniel Kantor, an MS specialist in Ponte Vedra Beach, said the treatment could work, but he also urged caution. MS is so much easier to treat in mice than in humans, he said, that researchers sometimes joke that rattling the cage will cure the disease in laboratory animals.
"Even if it's really going to work in the end, it will take years before it's used in humans," Kantor said. "Animal data is different from human data. Until you have large human studies, you never know."
Scripps' work was supported by National Institutes of Health grants totaling $3.6 million.
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