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Multiple Sclerosis

Description
Multiple sclerosis (or MS) is a chronic, often disabling disease that attacks the central nervous system (CNS), which is made up of the brain, spinal cord, and optic nerves. Symptoms may be mild, such as numbness in the limbs, or severe, such as paralysis or loss of vision. The progress, severity, and specific symptoms of MS are unpredictable and vary from one person to another. Today, new treatments and research are giving new hope to people affected by the disease.

Source: National Multiple Sclerosis Society

Who is at Risk?
MS is not contagious and is not directly inherited, although heredity is a factor. People from different ethnic groups have different tendencies to develop MS. It is most common among people of Northern European ancestry, although African Americans and Hispanics develop MS as well. Other ethnic groups – Intuits, African blacks, and Southeast Asians – are much less likely to have MS. The average person in the United States has about one chance in 750 of developing MS. But relatives of people with MS, such as children, siblings or nonidentical twins, have a higher chance – ranging from one in 100 to one in 40. In addition to genes, other factors – perhaps exposure to germs or viruses – play a part in causing MS. That is why scientists say that MS is not directly inherited.

Source: National Multiple Sclerosis Society

A Potential New MS Treatment's Long and Winding Road

In a remarkable turn of events, a 20-year-old treatment pioneered by Scripps Research scientists for an exceedingly rare form of leukemia appears to be on the verge of becoming the first effective oral therapy for multiple sclerosis (MS), a disease that affects an estimated 2.5 million people worldwide. The drug, cladribine, which is currently marketed under the name LEUSTATIN® by Ortho Biotech, Inc. (an affiliate of Johnson & Johnson) for the treatment of hairy cell leukemia, was initially identified and developed by Dennis Carson, a Scripps Research scientist working in collaboration with Ernest Beutler, the late Scripps Research professor and chair of the Department of Molecular and Experimental Medicine. In January of 2009, primary data from a two-year Phase III trial of cladribine tablets for MS sponsored by Merck Serono, a division of Merck KGaA, Darmstadt, Germany, was announced and showed that the drug significantly reduced the relapse rate of MS patients with the relapsing-remitting form of the disease.

The potential importance of the drug cannot be overstated, according to Associate Professor Jack Sipe, M.D., the Scripps Research scientist.who first suggested that the drug might have potential as a treatment for MS. Currently, there are six FDA-approved medications for the treatment of MS, all require injection, and all are expensive and difficult for patients to tolerate. Many of Sipe’s MS patients have been on them for five to ten years and they're very tired of injections. This new treatment, if it's approved, has the potential to make a significant difference in the quality of life for people who suffer from MS.

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Scripps Research Study Describes Powerful New Tool in the Fight Against Autoimmune Diseases, Blood Cancers
A study led by a Scripps Research Institute scientist describes a new, highly pragmatic approach to the identification of molecules that prevent a specific type of immune cell from attacking their host. The findings add a powerful new tool to the ongoing search for potential treatments for autoimmune diseases, such as multiple sclerosis (MS), as well as blood cancers, such as myeloid leukemia. The study was led by Thomas Kodadek, Ph.D., a professor in the Chemistry and Cancer Biology Departments at Scripps Florida. In the new study, Kodadek and his colleagues used samples from an animal model of multiple sclerosis to screen for T cells—a type of white blood cell that plays a central role in the immune system—with a heightened presence in the disease. The screen also identified molecules that interfere with these T cells' "autoreactivity," in other words, their attack on the body itself rather than a foreign invader such as virus or bacteria.

Their technique simultaneously uncovers and isolates autoreactive T cells as well as inhibitors to them. It's a double whammy. At the heart of this is a comparative screening process of normal T cells versus disease-causing T cells. While the process is technically complicated and difficult, the thinking behind it is not. Kodadek and his colleagues wanted to simplify the process of identifying compounds that could inhibit autoreactive T cells with exceptional specificity, and they succeeded. The scientists used a model of MS, an autoimmune inflammatory disease affecting the brain and spinal cord, for the study. In MS, the immune system attacks the myelin sheath covering and protecting nerve cells, leading to a variety of symptoms depending on which part of the nervous system is affected. Common symptoms of the condition include fatigue; numbness; walking, balance, and coordination problems; bladder and bowel dysfunction; vision problems; dizziness and vertigo; sexual dysfunction; pain; cognitive problems; emotional changes; and spasticity.

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Study Reveals Unexpected Mechanism of New Multiple Sclerosis Treatment

Patients suffering from multiple sclerosis (MS) recently received the welcome news that the U.S. Food and Drug Administration (FDA) had approved a promising new drug for their condition called Gilenya. Now, a team from The Scripps Research Institute has discovered that this drug's success may involve an unexpected biological mechanism acting within the central nervous system. This difference may mean that Gilenya offers even more benefits than previously realized and would represent the first MS therapy with direct central nervous system activities.

The work also reveals potential new strategies targeting the central nervous system for research into better MS treatments.   The drug could make a big difference to MS patients.  Jerold Chun, M.D., Ph.D., a professor in the Department of Molecular Biology and member of the Dorris Neuroscience Center at Scripps Research, made the discovery.  These results are going to make doctors and scientists consider central nervous system mechanisms in MS therapies, as they follow patients being treated with Gilenya.

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Scientists Develop Compound that Effectively Halts Progression of Multiple Sclerosis

Scientists from the Florida campus of The Scripps Research Institute have developed the first of a new class of highly selective compounds that effectively suppresses the severity of multiple sclerosis in animal models. The new compound could provide new and potentially more effective therapeutic approaches to multiple sclerosis and other autoimmune diseases that affect patients worldwide. Current treatments for autoimmunity suppress the patient's entire immune system, leaving patients vulnerable to a range of adverse side effects. Because the new compound, known as SR1001, only blocks the actions of a specific cell type playing a significant role in autoimmunity, it appears to avoid many of the widespread side effects of current therapies.

Tom Burris, Ph.D., a professor in the Department of Molecular Therapeutics at Scripps Florida led the study. Burris and his colleagues have been involved in several discussions with both pharmaceutical and biotechnology firms who are very interested in developing it further.  A lengthy process of drug development and review is required to ensure a new drug's safety and efficacy before it can be brought to market.

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