CARLSBAD, Calif., Aug. 1, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (ISIS) announced today that new preclinical data evaluating antisense compounds to treat myotonic dystrophy type 1 (DM1) were published in the journal Nature. These new findings show that antisense targeting of a toxic RNA, the disease causing component in DM1, resulted in reversal of disease symptoms that were sustained up to one year after treatment in a mouse model of DM1. "DM1 is a progressive disease that leads to the gradual loss of muscle function. Although DM1 is estimated to effect approximately 150,000 patients worldwide, there are no treatments available that could delay the onset of symptoms or slow down the progression of this debilitating disease. We are encouraged by these early results, which build on the groundbreaking work conducted by Dr. Charles Thornton at the University of Rochester Medical Center and others elucidating the mechanism of DM1. Using our antisense technology, we and our collaborators were able to target the toxic RNA, remove it and restore normal cell function," said Frank Bennett, Ph.D., Senior Vice President, Research at Isis. In the published study, Isis and collaborators from the University of Rochester evaluated antisense compounds in a mouse model of … Continue reading →

Researchers have successfully reversed symptoms of the most common form of adult muscular dystrophy in mice, pointing toward a possible treatment for humans. In a paper published in the journal Nature, scientists from the University of Rochester Medical Center and two drug companies -- Isis Pharmaceuticals and Genzyme -- describe how they attacked the disease by targeting "toxic" genetic messengers. "For 20 years, we studied myotonic dystrophy, hoping that someday we would learn enough to spot its Achilles heel," University of Rochester neurologist and senior author Charles Thornton said in a statement Wednesday. "This work comes close to doing that." Myotonic muscular dystrophy affects about 35,000 people in the U.S. Patients suffering from the disease find their muscles wasting away, leaving them weak. They also often cannot relax their muscles due to prolonged muscle contractions, meaning it can be hard for them to release their grip on a handle or move their jaw. The disease is caused by a genetic mutation that causes abnormal messenger RNA -- the genetic courier that's transcribed from DNA -- to accumulate in the nuclei of cells. That buildup causes an intracellular traffic jam that interferes with multiple proteins, including MBNL1, which is involved in … Continue reading →

ScienceDaily (Aug. 1, 2012) Scientists have reversed symptoms of myotonic muscular dystrophy in mice by eliminating a buildup of toxic RNA in muscle cells. The work, carried out by scientists at the University of Rochester Medical Center, Isis Pharmaceuticals Inc. and Genzyme, is published in the August 2 issue of Nature. After experimental antisense compounds were administered to mice twice a week for four weeks, symptoms of the disease were reduced for up to one year -- a significant portion of a mouse's lifespan. The investigators say that while the work is an encouraging step forward against myotonic dystrophy, one of the most common forms of muscular dystrophy, it's too soon to know whether the approach will work in patients. But they are cautiously optimistic, noting that the compound is extremely effective at reversing the disease -- whose genetic underpinnings make it particularly vulnerable to an antisense approach -- in a mouse model. "These results give us strong encouragement about the possibility of developing a treatment that could fundamentally alter the disease. It's an important step on a long path," said senior author Charles Thornton, M.D., a neurologist at the University of Rochester Medical Center who has been pursuing new … Continue reading →

Symptoms of a form of muscular dystrophyhave been reversed in mice, a research team including scientists from Isis Pharmaceuticals Inc.reported Wednesday. Chemicals injected into the mice rapidly reduced levels of toxic molecules that cause the disease, the scientists said. They reported their findings in the scientific journal Nature. It will take much more research before the treatment can be tried in humans, said an accompanying news article in Nature. "However, the path to success now seems clearly visible," the article stated. The chemicals work through Isis' gene-blocking antisense technology, which stops production of disease-causing proteins. The technology represents Isis' bid to become a powerhouse in discovering new drugs. Antisense drugs can theoretically work on a vast number of diseases. Shares of Isis, which is based in Carlsbad, closed Wednesday at $11.80, a loss of 32 cents for the day. That's still about 63 percent higher than Isis' close of $7.25 on Jan. 3, the first trading day of 2012. The team included Frank Bennett, senior vice president of research at Isis, and two other Isis scientists. Others are scientists at the University of Rochester Medical Center andGenzyme.Charles Thornton, a University of Rochester neurologist, was senior author. The disease is called … Continue reading →

Public release date: 1-Aug-2012 [ | E-mail | Share ] Contact: Emily Boynton emily_boynton@urmc.rochester.edu 585-273-1757 University of Rochester Medical Center Scientists have reversed symptoms of myotonic muscular dystrophy in mice by eliminating a buildup of toxic RNA in muscle cells. The work, carried out by scientists at the University of Rochester Medical Center, Isis Pharmaceuticals Inc. and Genzyme, is published in the August 2 issue of Nature. After experimental antisense compounds were administered to mice twice a week for four weeks, symptoms of the disease were reduced for up to one year a significant portion of a mouse's lifespan. The investigators say that while the work is an encouraging step forward against myotonic dystrophy, one of the most common forms of muscular dystrophy, it's too soon to know whether the approach will work in patients. But they are cautiously optimistic, noting that the compound is extremely effective at reversing the disease whose genetic underpinnings make it particularly vulnerable to an antisense approach in a mouse model. "These results give us strong encouragement about the possibility of developing a treatment that could fundamentally alter the disease. It's an important step on a long path," said senior author Charles Thornton, M.D., a … Continue reading →