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Emerging Therapies, Risk Factors and Other MS Research News from American Academy of Neurology Meeting

April 10, 2013

Over 12,000 neurologists and other researchers convened at the American Academy of Neurology’s (AAN) annual meeting in San Diego in March to share progress in understanding and treating neurological diseases like MS. In most cases, studies presented are considered preliminary. Many of the results will be analyzed more thoroughly, and usually published in peer-reviewed science and medical journals. Confidence in a study’s findings grows when it is repeated by others, with similar results.

Read blogs from the AAN meeting
Read scientific summaries (abstracts) on the AAN’s Website

Here are a few highlights from more than 500 MS-related presentations focusing on stopping MS, restoring function, and ending MS forever.


Many studies were presented showing continued benefit and safety of available therapies, and additional findings from therapies proceeding through the development pipeline. Other studies looked at factors that may drive MS progression and relapses, opening possibilities for stopping MS in its tracks.

Emerging Therapies

Amiloride in progressive MS: This oral blood pressure medicine was tested in a small study of 14 people with primary progressive MS because it had been shown previously to have nerve-protecting (neuroprotective) properties. Dr. Tarunya Arun of Oxford University, with colleagues in the UK and Netherlands, reported that after 3 years, treatment with amiloride resulted in a reduction of brain shrinkage associated with progressive disease, compared to what was experienced before treatment. Further testing is getting underway in a larger study in the UK, which the National MS Society is helping to support through the UK MS Society. (Abstract S31.002)

Large trial in progressive MS: A poster presentation by Dr. David Miller of University College London and an international team suggested that good progress is being made in a large trial of Gilenya® being supported by Novartis Pharmaceuticals Corporation. The study involves more than 1000 participants with primary-progressive MS, and it’s designed to determine if Gilenya can slow down MS disability compared to inactive placebo after 3 to 5 years of treatment. This is one of several large studies like this underway in progressive MS. (Abstract P07.116)

New form of existing therapy for relapsing MS: Dr. Peter Calabresi of Johns Hopkins University presented results of an international, phase 3 trial of peginterferon beta-1a in relapsing MS, a new form of Avonex® designed to stay in the body longer than the standard form. The results over one year suggest peginterferon injected under the skin every two or four weeks was effective in reducing relapse rates and also reduced the risk of progression of disability. This study is continuing into a second year. Trial sponsor Biogen Idec has announced plans to apply for regulatory approval in 2013. (Abstract S31.006)

Copaxone® in fewer doses: Dr. Omar Khan of Wayne State University, Michigan, presented results of a one-year phase 3 trial supported by Teva Pharmaceuticals suggesting that under-the-skin injections of twice the standard dose of Copaxone, taken three times per week, were effective in reducing relapses and MRI-detected disease activity, and revealed no unexpected safety issues. (Abstract S01.005)

Switching therapies: A large study in France called ENIGM tracked the impact of switching from therapy with Tysabri® to Gilenya.® Among 200 people who switched after an average of 30 months on Tysabri, there was a “washout” interval of 3 to 6 months in which no therapy was given, during which 32% experienced a relapse. The researchers concluded that switching increases the likelihood of disease reactivation, and that the washout period should not be longer than 3 months. (Abstract 41.002)

More results on Tecfidera™: An evaluation of two phase 3 trials of oral dimethyl fumarate (Tecfidera, Biogen Idec), recently approved by the FDA for relapsing MS, suggested that the treatment began to take full effect against MS disease activity after 3 months’ use, and the effect was sustained over the two years of the trials. (Abstract S41.005)

Extension studies: Several presentations focused on results from extension phases of completed clinical trials in relapsing-remitting MS, including the following. These often involve open-label periods where participants who were on placebo during the original trial are switched to active treatment and others participants continue on therapy and are evaluated for a period of time.
  • Extension of a phase 2 study of infrequent infusions of ocrelizumab (Hoffmann-La Roche Ltd.) showed continued effectiveness at week 144 in most who continued in the study and no new serious adverse events. Ocrelizumab is in further testing for both relapsing and progressive MS. (Abstract S31.004)
  • A one-year extension of a phase 3 study of oral laquinimod (Teva Pharmaceuticals) showed that the risk of disability progression was significantly reduced for those who started on active therapy in the original trial versus those who started on placebo and then switched to therapy during the extension phase. As in the original trial, the most common adverse event was elevated liver enzymes. (Abstract S41.004)
  • A one-year extension of two phase 3 alemtuzumab trials (Genzyme, a Sanofi company and Bayer Healthcare Pharmaceuticals) showed there was a durable benefit against relapses and progression, often without additional IV infusions. Risks continued for adverse events including thyroid disorders and autoimmune disorders, and one person died from sepsis. Alemtuzumab is currently being evaluated by the FDA for marketing approval. (Abstract 41.001)
  • A one-year extension of a phase 2 trial of daclizumab high-yield process (DAC HYP, Biogen Idec and Abbott Biotherapeutics) showed that monthly under-the-skin injections continued to reduce relapses, MRI disease activity, and disease progression. There was one death due to autoimmune hepatitis and the incidence of serious infections, skin events and liver function test abnormalities were similar to those found in the original trial. (Abstract S01.001)

Exploring Disease Activity

Can vaccinations trigger MS attacks? Dr. Mauricio Farez of Universidad del Salvador, Buenos Aires, Argentina reported on his analysis examining whether common vaccinations are linked to MS. Among his findings, he reported that vaccination against yellow fever may substantially increase the risk of MS relapse, suggesting that someone with MS who is planning a trip to a country where there’s increased risk for yellow fever should discuss the risks and benefits of vaccination with an MS doctor. (Abstract S10.001)

Sugar and progression: We don’t know yet why some people’s MS progresses slowly and others experience rapid progression, but a small study from Drs. Wael Richeh, Jesus Lovera and colleagues at Louisiana State University gives food for thought. They asked whether blood sugar is linked to levels of MS disability, and found that people with higher levels of glucose were more likely to have higher levels of disability. This important lead needs more study to prove a role for blood sugar in MS progression. (Abstract P04.130)

Genes and kids: Drs. Emmanuelle Waubant and Jennifer Graves at the University of California at San Francisco and collaborators described preliminary results of a gene study in 117 children with MS, showing that one particular gene (rs4648356) was associated with lower rates of relapse, while another (rs11154801) was associated with higher rate of relapses. The study, which requires confirmation, was supported by the National MS Society and the Consortium of MS Centers. (Abstract P05.133)

Predictors/Disease Tracking Tools: Tool to track progression: Dr. Nicholas LaRocca of the National MS Society described efforts of the newly formed MS Outcome Assessments Consortium to accelerate development of more effective treatments for MS, particularly for progressive forms of MS. MSOAC is a coalition of industry, academia, patient representatives, regulatory and other government agencies, and the National MS Society. In collaboration with the Critical Path Institute, they will analyze data from completed MS clinical trials and other studies and work with regulatory agencies to qualify a new clinician-reported outcome measure that can be used to more sensitively track the impact of therapy on disease disability and progression for future MS trials. (Abstract S31.001)

Predicting who will respond to Copaxone®: Drs. Francisco Quintana, Howard Weiner and team at Harvard’s Brigham and Women’s Hospital described a study that analyzed serum samples from people with MS who were taking glatiramer acetate (Teva Pharmaceuticals). They were able to find antibody profiles that could detect those who responded to therapy and those who did not. Further work to confirm these findings could lead to a method to know early on whether a person is benefiting from this treatment or not. (Abstract S11.002)


The broad area of research to restore function encompasses efforts to repair the nervous system and also stimulate recovery of lost function through exercise, rehabilitation and other means.

“Rewiring” the brain: Invited speaker Dr. Maria Assunta Rocca of San Raffaele Hospital in Milan, Italy presented compelling data for how the brain reorganizes to adapt to MS damage. In one study, the team looked at the impacts of a 12-week computer-assisted course that focused on training to increase memory and attention. They had previously reported improved attention and executive thinking abilities. Using functional MRI, which allows a real-time glimpse of the brain at work, they also found indicators that brain circuitry and activity had increased in specific areas, which appeared to persist at least 6 months after the training was completed. (Abstract P04.030)

Exercise and the brain: A small study from Society-supported scientists Drs. Victoria Leavitt, John DeLuca and others at the Kessler Foundation in New Jersey tested whether aerobic exercise impacts the brain. Using MRI scans and memory tests, they found hints that aerobic exercises (30 minute sessions, 3 times a week, over 3 months) improved memory and increased the volume of the hippocampus, a part of the brain involved with memory and other functions. These preliminary results require additional follow-up. (Abstract P04.034)

Exercise and fatigue: German researchers Drs. Stephan Schmidt of Bonn, and Marc Wonneberger of Cologne reported on the impacts of longer-term aerobic exercise to build endurance in people with MS. This study involved 60 people split into two groups: those with fatigue and those without fatigue. Both groups did individualized endurance exercise on treadmills for 12 months. After 6 months of exercise, both groups had improved oxygen consumption, but those who started out with fatigue didn’t show improvement in their fatigue scores until 9 months into the program. (Abstract P04.042)

Brain power: Researchers from Milan and from Kessler also reported that people with MS with more “brain reserve” (larger brain size) and more “cognitive reserve” (higher levels of cognitive leisure activities when they were in their 20s) were at lower risk for cognitive changes associated with MRI lesions. Even when brain size was accounted for, those with more cognitive reserve appear to have lower risk for cognitive changes. Research is ongoing to see whether enrichment activities can help build cognitive reserve. (Abstract P04.109)

CCSVI treatment in MS: First results from a controlled endovascular treatment trial (percutaneous transluminal venous angioplasty) were presented by Drs. Robert Zivadinov, Adnan Siddiqui and the team from State University of New York at Buffalo. In this blinded study, 9 people had the angioplasty and 10 had a sham treatment. At six months, the team did not detect adverse events from the treatment, but also found that it failed to provide sustained improvement in venous outflow. They also found that those whose veins increased in outflow tended to have increased MS disease activity seen on MRI. (Emerging Science Poster P04.273)

Prevalence of CCSVI in MS: Dr. Robert Fox, Claude Diaconu and a team at Cleveland Clinic and Case Western Reserve reported preliminary results from a National MS Society-supported study of CCSVI in 61 people with different types of MS and 20 people without MS. They used ultrasound techniques that included technicians trained in CCSVI assessment who were unaware of the participants’ disease status. Although changing the interpretation of CCSVI criteria produced substantial differences in the proportion of participants meeting those criteria (20% to 40% of non-MS met criteria versus 21.3% to 36.1% of MS participants), there was no significant difference between the non-MS and MS groups. (Abstract P05.177)


There were several reports focusing on risk factors that may contribute to making a person more likely to get MS, including recaps of National MS Society-supported studies related to salt and MS immune activity. This line of research could eventually lead to ways to prevent people from getting MS.

Dystel Prize: This year’s winner of the John Dystel Prize for MS Research, given jointly by the National MS Society and the AAN, was Professor George Ebers of Oxford University, UK. He presented his work on the influences of genes and the environment in relation to who develops MS. Read more

Reproduction and risk of MS: Drs. Melinda Magyari, Per Solberg Sorensen and colleagues from the Danish MS Center in Copenhagen looked at potential factors that may account for an increased incidence of MS in women over several decades. Using the Danish MS Registry, which captures info on most people in their country who have MS, they found that pregnancy and childbirth offered significant protection against developing MS, lasting up to 5 years. This and other studies can offer more clues to the influence of hormones and other factors in MS. (Abstract IN8-1.001)

Environmental exposures: Drs. Ellen Mowry at Johns Hopkins University, Lisa Barcellos at University of California, Berkeley and colleagues asked a proportion of women enrolled in Kaiser Permanente Northern California health care about their possible exposures to specific environmental factors. They adjusted results for some known risk factors such as genes already known to increase MS risk, vitamin D status, prior mononucleosis, and smoking in order to seek new possible factors. Results, which require further study, show that women exposed to small pets and reptiles may have been protected from developing MS, while those exposed to hair permanent solution may have had increased risk for developing MS. (Abstract P05.138)

Avonex is a registered trademark of Biogen Idec
Copaxone is a registered trademark of Teva Pharmaceutical Industries
Gilenya is a registered trademark of Novartis
Tecfidera is a trademark of Biogen Idec
Tysabri is a registered trademark of Biogen Idec and Elan Pharmaceuticals

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.


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