Dec 08, 2006
2006 A Very Good Year in MS Research
The year 2006 saw rapid research progress in the fields of science and medicine that impact our understanding of the unpredictable neurological disease of multiple sclerosis. Thanks to its generous contributors, the National MS Society was able to invest over $42 million this year into over 350 new and ongoing MS research projects as part of its international effort to spur momentum in cutting-edge research to cure, treat and better understand MS.
Significant advances have been made in both clinical and laboratory studies in MS. In addition, more than 130 clinical trials are underway around the world, and still other experimental drugs are in the pipeline. Key highlights of the year include:
- Acorda Therapeutics (Hawthorne, NY) announced positive results of a Phase 3, placebo-controlled clinical trial of Fampridine-SR, an oral drug designed to provide symptomatic relief by compensating for lost nerve conduction. In 301 patients with all types of MS, those on active treatment showed an average increase in walking speed of 25% versus those on inactive placebo. The company is expected to meet with the U.S. Food and Drug Administration (FDA) to determine next steps needed to apply for marketing approval.
- The FDA approved the return to market of Tysabri® (natalizumab, produced by Biogen Idec and Elan Pharmaceuticals) to delay the accumulation of physical disability and reduce the frequency of relapses (clinical exacerbations) in those with relapsing MS. There is now in place a mandatory registration program for patients and prescribing physicians to minimize the risk of PML (progressive multifocal leukoencephalopathy), caused by a common virus called the JC virus. The drug is dispensed at registered infusion centers across the country.
- The National MS Society launched a new postdoctoral fellowship program in MS rehabilitation research. The immediate goal is to recruit and train talented clinician-scientists in rehabilitation research specific to MS; the ultimate goal is to get more hands and minds working on ways to help people with MS maximize their abilities.
- Researchers from the University of California, Los Angeles reported that administering Androgel® (testosterone gel applied to the skin) to 10 men with relapsing-remitting MS significantly improved cognitive function and slowed brain tissue loss. This small study was funded by the National MS Society’s initiative on Gender Differences in MS. Further study involving larger numbers of patients and control groups is necessary to confirm these early results.
- In another offshoot from the Society’s initiative on Gender Differences, UCLA investigators began the first large-scale trial of a sex hormone for the treatment of MS. The two-year, controlled clinical trial of estriol involves 130 women with early relapsing-remitting MS. If successful, this clinical trial will lay the groundwork for a larger, definitive trial that could lead to a new treatment option for women with MS. Its results may also have implications for women with other autoimmune diseases, such as rheumatoid arthritis.
- How many people in the U.S. have MS? Although the National MS Society estimates that about 400,000 people have the disease, no one knows for certain. To address this and other important questions about MS, the Society formed a Task Force on Epidemiology that is determining the feasibility of a nationwide tracking system for MS, collaborating closely with the CDC/Agency for Toxic Substances and Disease Registry, which is creating a proposal for setting up such a system.
- Members of the four Nervous System Repair teams funded by the National MS Society through its Promise: 2010 campaign met for the first time in November to share data and foster collaborations. Their efforts are paving the way for clinical trials focused on protecting the nervous system and restoring function in people with MS.
- Several oral therapies continued to progress through the MS pipeline:
- a phase II controlled clinical trial of oral fingolimod (FTY720, Novartis Pharmaceuticals Corp.) in 255 people with active, relapsing MS found that up to 77% of those taking fingolimod remained free of relapses over two years; a large phase III trial is now underway.
- oral cladribine (an immune-modulating drug by Serono), now being tested in an international Phase 3 clinical trial, has been designated by the FDA as a “Fast Track Product,” which should expedite its future review;
- a multicenter, phase II controlled clinical trial of oral BG00012 (an oral fumarate, Biogen Idec) led to a 69% reduction in active inflammation on MRI scans in 257 people with relapsing-remitting MS;
- in an open-label, 144-week extension study of oral teriflunomide (an agent that may modulate T cells), those on placebo during the original trial who switched to teriflunomide experienced up to an 85% decrease in new, active areas of disease activity seen on MRI at week 144.
- a phase II controlled clinical trial of oral fingolimod (FTY720, Novartis Pharmaceuticals Corp.) in 255 people with active, relapsing MS found that up to 77% of those taking fingolimod remained free of relapses over two years; a large phase III trial is now underway.
- Harvard investigators reported that individuals who showed signs of significant exposure to the Epstein-Barr virus, which causes infectious mononucleosis and other disorders, were twice as likely to develop MS up to 20 years later. The study, funded in part by the National MS Society, adds to previous evidence linking the virus to the risk of developing MS, but does not prove that EBV actually causes MS. Other studies suggest that smoking cigarettes may contribute to the risk of MS and MS progression, and that higher vitamin D intake may help protect against developing MS.
- For the first time, an international panel of experts developed consensus on proposed definitions of acquired childhood neurological disorders, including pediatric MS. The International Pediatric MS Study Group, established and supported by the National MS Society, has proposed specific ways that doctors can differentiate among childhood neurological diseases that often mimic each other. This effort should help doctors make critical diagnosis and treatment decisions that could eliminate months of uncertainty and improve care of children with these potentially devastating disorders.
- The needs of children who develop MS-like symptoms are at the heart of the National MS Society’s nationwide network of Pediatric MS Centers of Excellence, launched early this year. The 6 centers have committed to sharing resources and best practices such as MRI protocols and neuropsychological evaluations and to collecting standardized medical data so that families can benefit from the collective knowledge of the network. In addition to providing optimal care and support, these centers will build a framework for research into this patient population, which may also provide clues to adult MS.
- In a first, Johns Hopkins University researchers reported that nerve cells derived from mouse embryonic stem cells that were transplanted into rats with spinal cord injury were able to connect with muscles and partially restore function. While this work was done in a model of spinal cord injury, it has relevance to the potential use of cell replacement to repair damage in MS.
- Two genes that may contribute to making a person susceptible to MS have been identified by a group of European researchers known as the “GAMES” Collaborative Group. MS involves an immune-system attack on the body’s own brain and spinal cord, and many genes are thought to contribute to susceptibility. The two candidate genes fit this picture because they encode for a brain tissue component and an immune component. This work was supported by the Wellcome Trust, with additional funding from the National MS Society (USA), the MS Society of the UK and Northern Ireland and Applied Biosystems.
- A study by an international team including those funded through the National MS Society’s “MS Lesion Project” suggested that a substantial amount of natural repair can occur to the myelin coating on nerve fibers which is damaged by immune forces in people with MS. While previous studies had shown that myelin repair occurs, this study found evidence of repair in a proportion of patients’ tissues not only in those with the more common relapsing course, but also in those with progressive disease.
- Society-supported researchers at Stanford University uncovered evidence they believe may explain the role of a protein, osteopontin, in stimulating repeated relapses and disease progression. This could lead to new therapeutic approaches that target osteopontin’s effect in the MS disease process.
These and other leaps forward have made 2006 a momentous year in the fight against MS.