Compound Stops MS Progression In Animal Model

Researchers from The Scripps Research Institute in Florida have developed a new class of compounds that have shown to effectively halt the progression of MS in animal models. This new class of highly selective compounds may just lead the way for the researchers to find other more effective therapeutic routes for multiple sclerosis in humans. The findings were published in the advanced online edition of the Nature journal.

Most of the current treatments for MS focus on the entire immune system of an MS patient in order to control the symptoms associated with the disease. Such treatments may also leave the patients more vulnerable to other adverse side effects due to the weakened immune system. The new compound, known as SR1001, only suppresses the action of a specific cell type that plays a significant role in autoimmunity, leaving the other functions of the immune system working normally. In this way, many of the adverse side effects that are associated with autoimmunity suppression when using current MS therapies may be prevented.

According to Tom Burris, Ph.D., a professor in the Department of Molecular Therapeutics at Scripps Florida and lead author of the study, “This is a novel drug that works effectively in animal models with few side effects.” Burris and his colleagues have long been investigating small molecule compounds that may affect particular receptors that bind with other molecules to trigger certain effects on cells. The researchers have been particularly interested in a pair of orphan nuclear receptors called called RORĪ± and RORĪ³ which are involved in both autoimmune and metabolic diseases.

These receptors play a critical role in the development of TH17 cells, a form of T helper cells that have recently been implicated in the pathology of several autoimmune diseases which include multiple sclerosis. The TH17 cells produce Interleukin-17, a natural molecule in the body that induces inflammation.

“If you eliminate TH17 cell signals, you basically eliminate the disease in animal models,” Burris added. “Our compound is the first small-molecule orally active drug that targets this specific cell type and shuts it down. Once SR1001 is optimized, chances are it will be far more potent and effective,” he further stated. Although this might prove to pave the way for the development of other therapeutic routes for treating MS, a lengthy process of more studies and review may be needed to ensure the safety of the drug on humans. This may be required before any new drug treatment for MS may finally reach the market.


Scripps Research Institute (2011, April 17). Compound effectively halts progression of multiple sclerosis in animal model. ScienceDaily. Retrieved April 19, 2011, from

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