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Great Strides Moved MS Research Forward During 2008

Great Strides Moved MS Research Forward During 2008

The year saw rapid research progress in virtually every field of science and medicine that impacts our understanding of the unpredictable neurological disease of multiple sclerosis. Thanks to its generous contributors, in 2008 the National MS Society invested nearly $50 million to support over 440new and ongoing MS research projectsas part of its international effort to prevent, treat and cure MS.

Significant advances have been made in both clinical and laboratory studies in MS. In addition, more than130 clinical trials are underway around the world– with over one dozen final-phase trials of new therapies including some taken by mouth. Key highlights of the last year include:

Treatment/Pipeline

• Walking speed improved significantly in a clinical trial of 240 people with all types of MS takingFampridine-SR(MS-F204, Acorda Therapeutics, Inc.) compared with those taking inactive placebo. Fampridine-SR is a sustained-release formula of 4-aminopyridine, which temporarily enhances nerve signaling. The company is planning to file in 2009 for approval of this drug to treat mobility issues in MS. • Positive results were published from at least three phase 2 trials of drugs in the pipeline for relapsing-remitting MS. Two are the experimental oral therapieslaquinimod(Teva Pharmaceutical Industries) andBG00012(Biogen Idec, Inc); the other isalemtuzumab(Genzyme Corporation and Bayer Healthcare), given by yearly infusion. Each was found to reduce MS disease activity by modulating the immune attack. Larger phase 3 trials are ongoing for each of these agents. • One course of the IV drug rituximab (Rituxan®, Genentech and Biogen Idec) was shown toreduce MS diseaseactivity for 48 weeks in people with relapsing-remitting MS. Rituximab depletes immune B cells, which may play a role in the immune attack on brain and spinal cord tissues in MS. A clinical trial ofrituximab in people with primary progressive MSbrought disappointing results, with no apparent benefit, but researchers are still analyzing data from that study for possible signs of impact on the disease. •Fast Forward, the National MS Society’s initiative aimed at translating promising laboratory discoveries into effective new treatments for MS, got off to a flying start during its first year of operation. Fast Forward entered talks with several companies with the aim of developing novel therapies or repurposing existing drugs for the treatment of MS, and closed its first deal, details of which will soon be announced.

Nervous System Protection/Repair

• University of Rochester researchers funded in part by the National MS Society showed for the first time that human glial progenitor cells – immature myelin-making cells –restored the myelininsulation on nerve fibers as well as neurological function when transplanted into the brains of some mice born without the ability to form myelin. Myelin is a key target of the immune attack in MS.• Researchers from the Salk Institute for Biological Sciences and other institutions reported thatadult stem cells in micethat were on their way to becoming nerve cells could be reprogrammed by changing a single gene to turn into cells that make myelin. Further research is needed to translate these findings in people and to determine their significance to myelin repair in MS. • Astudy of brain tissueobtained from people with MS indicated that, while many areas of damage showed expected loss of myelin and nerve cells, a few older lesions showed a 72% increase in nerve cells when compared with neighboring brain regions. The findings, by Society-funded investigators at the Cleveland Clinic Foundation, support the possibility that nerve cells in the white matter of the brain can be replaced after they are destroyed by MS. • Brigham and Women’s Hospital researchers supported by the National MS Society reported thatan experimental compound(a derivative of fullerene, a form of carbon molecule) reduced disease progression, as well as damage to nerve fibers and their myelin insulation, when administered to mice with a progressive MS-like disease.  •Two teams of researchersfunded by the National MS Society reported findings on nerve tissue injury and repair that add important information needed to stop MS progression and develop nervous system repair strategies. One team (from the Mayo Clinic) found two enzymes that may serve as markers of progressive MS and nerve fiber injury, and the other team (from Mount Sinai School of Medicine) reported that another enzyme is essential for replenishing myelin-making cells that have been depleted by MS. Both teams hope to identify targets for the development of new therapies for MS.

Risk Factors/Disease Activity

• Investigators from the University of Queensland, funded by the National MS Society and MS Australia,published resultssuggesting that a person’s set of immune-related genes may help determine which parts of the brain and spinal cord are attacked by the immune system during the course of their MS, and may explain why individuals with MS experience tissue damage, and corresponding symptoms, differently.  • In a major step toward discovering molecules that may be used as “markers” to predict MS disease activity and progression, researchers at the University of California, San Franciscoidentified a pattern of gene expression(i.e., patterns of genes being turned on or off) within immune cells that was associated with quick conversion to MS in a study of 37 people with CIS (clinically isolated syndrome, a first event suggestive of MS). This study was funded in part by the National MS Society. • University of Toronto researchers investigated 117children at high risk for MS– youngsters who have had one neurologic episode. They reported at the World Congress of MS Research that vitamin D levels were significantly lower in the 16% of the children who went on to develop definite MS. Future studies are needed to determine whether vitamin D supplementation alters disease susceptibility or course. • An international collaboration of investigators founddifferences in genetic materialbetween people who respond to interferon beta treatment and those who don’t, in a study of 287 people with relapsing-remitting MS. If confirmed by larger studies and explored further, these results are an important step toward using genetic information that may one day guide treatment decisions for patients and their doctors. • Researchers at Stanford University and other institutions conductedhigh-tech analysesof different types and stages of MS brain lesions to uncover hundreds of proteins that may be active at different stages of the disease. To validate the approach, which was funded in part by the National MS Society, they narrowed in on two of the proteins and blocked their activity using existing drugs in mouse models of MS, and were able to improve symptoms.

Impacts of MS on People

People with MS who see neurologists are more likely than those who see other providers to receive treatment with disease-modifying agentsand to see rehabilitation specialists and urologists, according to a project using data from the Sonya Slifka Longitudinal MS Study (funded by the National MS Society’s Promise 2010 research and care initiative). The study suggests that those who only consult non-neurologists for their MS care may not be receiving the latest advice or widest spectrum of treatments that have been shown to improve outcomes. • TheAtlas of MSwas unveiled by the MS International Federation at the World Congress on MS. This international survey reached people in 112 World Health Organization member states, area and territories, representing 88% of the world’s population, to gather data on resources available to diagnose, treat, and support people with MS. The Atlas highlights the need for improved MS care. • Thesymptoms of MS that affect mobility have a significant impact on quality of life, safety, and financial and emotional health among many people living with MS, according to the results of two 2008 surveys conducted by Harris Interactive on behalf of Acorda Therapeutics, Inc. and the National MS Society. The findings provide new data related to the impact of mobility loss and walking difficulty on different aspects of daily life for people with MS.

Driving Research and Collaboration

• For the first time,$5 million has been awarded for MS researchwithin the Congressionally Directed Medical Research Programs, thanks in large part to efforts by MS activists across the country. This line-item allocation for investigator-initiated research projects is funded through the Department of Defense.  • The internationalteams focusing on nervous system repair and protectionin MS, funded through the Society’s Promise: 2010 initiative, published or presented over 60 reports in medical journals and scientific meetings over the last year to share their discoveries with the research community. The teams will convene in January 2009 to further collaborations toward the goal of testing repair and protection in MS. • The network ofPediatric Centers of Excellenceestablished by the National MS Society chose a data coordinating center that will expedite efforts to investigate MS in children. Although the centers have already begun conducting research investigations, this data center is establishing the infrastructure necessary for larger, more comprehensive studies. • The National MS Society’sfirst Tykeson Fellows Conferencewas held to spur new ideas and collaborations among young scientists and physicians across North America and to offer insights into ways they can pursue successful careers as MS researchers. • Over 5,000 clinicians, clinical researchers and basic scientists from around to world convened in Montreal, Quebec in September to share findings at thefirst World Congress on Treatment and Research in Multiple Sclerosis. Research on nervous system repair, pediatric MS, new therapies in the pipeline and much, much more was reported in nearly 1,000 presentations at this exciting meeting. These and many other advances this year helped move us closer to a world free of MS.