Mom's Story

A discussion about Mom's Story and MS…

New research strengthens genetics, MS link

 

In a large-scale, genome-wide analysis of more than 110,000 samples, researchers identified 200 genetic loci associated with multiple sclerosis. The study authors said that while the research highlights the role of several different immune cells that contribute to the initiation of this inflammatory disease, the mechanisms that lead this inflammatory disease to target the brain and spinal cord remain unclear.

By comparing the genomes of people with and without MS, the researchers identified 200 variants that were significantly more common among those with the disease. Most of these variants implicate genes that are associated with immune cells and immune system function, including a few potentially specific to brain-related functions.

Interestingly, many of the genes identified were known to also be involved in other autoimmune diseases, such as rheumatoid arthritis, Type I diabetes, and ulcerative colitis. This raises intriguing questions about why these diseases target different organs and have different clinical manifestations.

The findings were presented at the American Society of Human Genetics (ASHG) 2016 annual meeting in Vancouver, B.C.

World’s Largest MS Research Conference Highlights Advances in Progressive MS, Gut Microbiome, Managing Symptoms, and New Approaches to Restoring Function

Results from clinical trials, including new approaches to treating progressive MS, lifestyle and wellness research and myelin repair strategies were among more than 2,000 presentations made at the European Committee for Treatment and Research in MS (ECTRIMS) meeting held in London, England in September.
The world’s largest gathering of MS researchers convened more than 9,000 scientists and clinicians and industry representatives from across the globe, including many National MS Society-funded researchers, meeting and presenting on cutting-edge MS research progress. In addition, the European Rehabilitation in MS network met jointly with ECTRIMS this year.

During the conference, the International Progressive MS Alliance announced new investments of over $14 million US dollars to support three Collaborative Network Awards. These international teams were selected to accelerate the pace of research in key areas to speed new therapies for progressive MS.

Below are highlights of presentations focused on stopping MS, restoring function, and ending MS forever. In most cases, studies presented are considered preliminary. Many will be analyzed more thoroughly, and likely published in peer-reviewed journals.

STOPPING MS

Many presentations showed continued benefits of available therapies and longer-term safety information, as well as more evidence that early and ongoing treatment with a disease-modifying therapy has long-term benefits for controlling disease activity, delaying accumulation of disability, and protecting quality of life.

Siponimod in secondary progressive MS: More details were presented from a 60-month, phase 3 clinical trial of the experimental oral therapy siponimod (Novartis Pharmaceuticals AG) involving 1,651 people with secondary progressive MS. The trial met its primary endpoint, with those on active treatment showing a modest 21% reduced risk of disability progression compared to those on placebo. Secondary endpoints suggested that those on active therapy had 23.4% lower average change in brain volume and reduced MRI-detected lesion volume. The medication showed a similar safety profile to others that work by preventing white blood cells from entering the central nervous system. (Abstract #250)

More details from trial of lipoic acid in secondary progressive MS: Dr. Rebecca Spain and colleagues (Oregon Health & Science University) presented results from a small, controlled clinical trial on the oral anti-oxidant supplement called lipoic acid in people with secondary progressive MS. The lipoic acid group had 66% less brain tissue shrinkage, or atrophy, than the group taking inactive placebo pills. This pilot study suggests potential benefits if they hold up in a larger trial. (Abstract #222)

New results on gut bacteria: Efforts are advancing to pinpoint bacteria in the gut that may drive inflammatory immune system activity in MS and others that can suppress it, which may open the door to novel probiotic or other therapeutic approaches to treating MS.

  • Drs. Yan Wang, Lloyd Kasper and colleagues (Dartmouth Medical School and Eastern Washington University) reported that treating mice with the gut-related molecule called polysaccharide A (PSA) expanded a type of immune cells called “Regulatory B cells” (Bregs) which promote an immune response that prevents mice from getting MS-like disease. (Abstract #181) Members of this team also reported that PSA had positive effects in mice with progressive MS-like disease. (Abstract #P465)
  • Dr. Sergio Baranzini (University of California, San Francisco) and other collaborators in the National MS Society-supported MS Microbiome Consortium are analyzing gut bacteria to unearth clues about MS susceptibility and progression. They analyzed bacteria in stool samples from 64 people with MS who had received treatment for MS, and 68 people without MS. Certain bacteria were increased in people with MS, and those bacteria increased immune cells (T helper 1 cells) that are major players in MS immune attacks. Another type of bacteria that could suppress the immune attack was reduced. (Abstract #179)

Disappointing results for nerve-protection approaches: A small two-year clinical trial of fluoxetine (same compound as the anti-depressant Prozac) did not meet its goal of improving walking speed in people with progressive MS. The multi-center team from Belgium is still analyzing other results, such as changes in MRI and cognition. (Abstract #253) Likewise, a trial conducted at the University of Oxford tested the ability of amiloride to protect against nerve damage in people with acute optic neuritis (often an early sign of MS) failed to show any neuroprotective benefit. (Abstract #102) Additional trials of neuroprotective approaches to MS are ongoing.

Vitamin D deficiency and smoking linked to progression: Dr. Maria Isabel Zuluaga and team (Vall d’Hebron University, Barcelona) explored the independent impacts of smoking and vitamin D deficiency in a large group of people followed over time. They found that those with severe vitamin D deficiency (defined as blood levels at less than 8 ng/ml) showed an increased risk for MS disability, and active smokers also had an increased risk for disability progression. (Abstract #252) Graduate student Ms. Eva Rosa Petersen (Danish MS Center, Copenhagen) also found that smoking intensity was linked with higher frequency of relapses among people taking interferon beta. Smoking one pack of cigarettes per day increased relapse rates by 25%. (Abstract #178)

Vitamin D added to Rebif: A large international trial did not show a statistical difference between treatment groups after adding vitamin D (14,000 IU [350 µg] vitamin D3 daily) or placebo to Rebif therapy in relapsing MS, in terms of the percent of participants who were free from disease activity after 48 weeks. Dr. Raymond Hupperts (Orbis Medical Centre, Sittard-Geleen, The Netherlands), who presented results, noted that both groups were stable, which likely contributed to the inconclusive results. (Abstract #166)

Biomarkers under development: Teams are making headway toward having a simple test that can predict a person’s disease course, progression and response to therapy. Dr. Bibiana Bielekova (National Institute of Neurological Diseases and Stroke) and team examined proteins in the spinal fluid of people with neurological diseases, including all types of MS, and identified a “signature” of markers that distinguished MS from other diseases, and also differentiated relapsing MS from progressive MS. (Abstract #219). Other investigators also reported progress in this area, including advances using “neurofilament light chain” as a biomarker. (Such as Abstracts #183, #249) These early results need further development but indicate that  sensitive biomarkers for predicting disease course and response to therapy may become useful tools for the clinical management of MS.

RESTORING FUNCTION – WELLNESS, LIFESTYLE, SYMPTOMS

Home-based rehabilitation can work: With funding from the National MS Society, Dr. Gabriel Pardo (Oklahoma Medical Research Foundation) and colleagues compared the benefits of three approaches to rehabilitation for gait and balance in a small study: unsupervised home-based exercise 5 times/week; home-based exercise supervised remotely by a physical therapist 2-3 times per week via audio and visual conferencing; and home-based exercise plus in-person physical therapy 2-3 times/week. They found that all participants improved, and that the telerehabilitation program worked as well as the onsite program to improve gait and balance. Further research in larger trials could make telerehabilitation a cost-effective and more accessible alternative for people with MS. (Abstract #120)

Tackling fatigue: Dr. Vincent de Groot (VU University Medical Center, Amsterdam) reported results from three clinical trials testing different strategies over 16 weeks to lessen fatigue, in 90 people with MS: aerobic training, cognitive behavioral therapy, and energy conservation management. Only cognitive behavioral therapy effectively reduced severe fatigue in this short-term study. This is a commonly available type of psychotherapy. (Abstract #142) Read more about managing fatigue

Pain more common than previously reported: Dr. Carolyn Young (University of Liverpool) and colleagues found that nearly 66% of over 700 people with MS reported nerve pain. Higher levels were found in those who had MS for a longer time, had more severe disability, or were not working. (Abstract #P337Read more about addressing pain in MS

New trial confirms Ampyra (fampridine) benefits: Dr. Jeremy Hobart (Plymouth Hospitals NHS Trust) presented results from a large clinical trial of fampridine, a twice-a-day oral therapy that was previously approved for its ability to improve walking.. This trial wanted to show evidence that its benefits include meaningful functional improvements for people. The results over 6 months showed that 43% of those on active therapy had significantly better self-reported walking ability, mobility, and balance than those on placebo, with no new safety issues reported. (Abstract #254)

Cognitive rehabilitation enhances brain connections: Several studies showed that rehabilitation to improve cognition goes hand-in-hand with changes in brain connectivity (how areas of the brain interact). While many of these treatments are still experimental, some are available from rehabilitation specialists such as speech pathologists or neuropsychologists. Discuss options with your MS doctor:

  • Dr. Brian Sandroff (Kessler Foundation, West Orange, NJ) and colleagues showed that treadmill training improved information processing speed and brain connectivity in a small pilot study funded by the Society. (Abstract #P796)
  • Dr. Pietro Iaffaldano (University of Bari, Italy) and colleagues showed that a home-based computerized training program that targeted specific cognitive issues improved overall cognitive function significantly more than a non-specific program. Also, those who had less function in certain brain areas showed greater improvement after cognitive training. (Abstract #145)
  • Oiane Rilo (University of Deusto, Bilbao, Spain) and colleagues showed that a three-month, group-based cognitive rehabilitation program improved working memory, information processing speed, verbal memory and executive function (which is important in problem solving and planning), and altered brain connectivity. (Abstract #144)

Emerging treatment for muscle spasticity: Dr. Daniel Kantor (Kantor Neurology, Ponte Vedra Beach, FL) and colleagues report that in a trial of 354 people with relapsing-remitting or secondary progressive MS, Arbaclofen Extended Release Tablets (Osmotica Pharmaceuticals) significantly reduced spasticity compared to baclofen. The extended-release tablets caused significantly less sleepiness, drowsiness and dizziness than baclofen. (Abstract #128) The company reports that it has filed for FDA approval of Arbaclofen.

RESTORING FUNCTION – NERVOUS SYSTEM REPAIR

More Anti-LINGO Results: In June 2016 Biogen announced that its phase 2 clinical trial of anti-LINGO (proposed name opicinumab), an approach to repair myelin, did not meet its primary endpoint of improvement in physical function, cognitive function, or disability. The trial involved 418 people with relapsing MS who were taking interferon beta-1a (Avonex) plus one of several doses of intravenous opicinumab or placebo for 72 weeks. Dr. Diego Cadavid from the company described ongoing evaluations from the extensive testing and monitoring during the trial, which are helping to pinpoint the patient population, dosage and outcome measures that would inform the design of any future trials of anti-LINGO.  (Abstract #192)

Myelin repair in pediatric and adult MS: Dr. Sabine Pfeifenbring (University of Göttingen, Germany) and an international team analyzed brain biopsies from children who had been diagnosed with MS and compared the extent of damage and natural myelin repair against those of adults with MS. They found that children showed less damage to myelin-making cells and more evidence of myelin repair than adults. However, some myelin repair was found to occur at virtually all ages in MS. (Abstract #194)

Exercise enhances myelin repair in mice: To investigate some reasons why exercise promotes benefits in people with MS, Drs. S. Jensen and Wee Yong (University of Calgary) did a study where mice with myelin damage in their spinal cords used running wheels soon after the injury. They reported finding more evidence of generation of myelin-making cells and myelin repair in the active mice than those that did not use the running wheels after injury. (Abstract: #P1210)

Emerging approaches to protection and repair:  Dr. Martin Sanders (Io therapeutics) presented results from mice suggesting that the compound IRX4204 promotes repair of damaged myelin in mice. He noted that previous studies suggested that IRX4204 also showed signs of reducing immune attacks and protecting against nerve loss. This work was supported in part by a National MS Society’s Fast Forward investment. (Abstract #193)

Drs. Sarah Starossom, Samia Khoury and team (Brigham and Women’s Hospital, Boston) reported on studies of Chi3l3, a naturally occurring molecule in the brain that can stimulate the transformation of resident stem cells into myelin-making cells. The team noted that it plays an important role in recovery from the MS-like disease in mice, and may have potential for development as a new treatment approach in MS. (Abstract #195)

Positive Results Announced from Clinical Trial of BAF-312 (Siponimod) in Secondary Progressive MS

Summary

Results presented at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) provided additional details from a 60-month, phase III clinical trial of the experimental oral therapy siponimod (BAF312, Novartis Pharmaceuticals AG) involving 1,651 people with secondary progressive MS.

The trial met its primary endpoint of reducing the risk of disability progression compared with inactive placebo. Those on active treatment had a 21% reduced risk of disability progression compared to those on placebo. Secondary endpoints suggested that those on active therapy had 23.4% lower average change in brain volume and reduced lesion volume.

The therapy was generally well tolerated and similar to adverse events reported for similar compounds.

Details

Background: Siponimod (BAF312) is an experimental immune system-modulating therapy that was designed to be a more selective sphingosine 1-phosphate receptor modulator than Gilenya® (fingolimod, Novartis International AG). Gilenya, was approved in 2010 for adults with relapsing forms of MS to reduce the frequency of clinical relapses and to delay the accumulation of physical disability. Siponimod previously demonstrated safety and efficacy on MRI scans in a phase II study in people with relapsing-remitting MS (The Lancet Neurology, 2013 Aug;12(8):756-67).  Siponimod is thought to act by retaining certain white blood cells in the body’s lymph nodes, keeping them out of circulation and from entering the central nervous system. Siponimod also distributes effectively to the central nervous system (brain and spinal cord) where it may have direct anti-inflammatory or other effects.

The Study: Participants were randomly assigned to take siponimod or placebo capsules daily for up to 60 months. The primary endpoint of the study was reducing the risk of disability progression, as measured by the EDSS scale at three months. Secondary endpoints included reducing the risk of disability progression as measured by the EDSS at six months versus placebo, the risk of worsening mobility as measured by the timed 25-foot walk test, disease activity as observed on MRI scans, relapse rate, and safety/ tolerability.

Results:  Results were presented at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) on September 17, 2016. The trial met its primary endpoint of reducing the risk of disability progression compared with inactive placebo. Those on active treatment had a 21% reduced risk of disability progression (confirmed at 3 months) compared to those on placebo. Secondary endpoints suggested that those on active therapy had at 26% reduced risk of disability progression (confirmed at 6 months), a 23.4% lower average change in brain volume, and reduced MRI-detected brain lesion volume. There was no significant difference seen between groups in the timed 25-foot walk. Relapse rates were significantly lower in those taking siponimod.

Safety: The therapy was generally well tolerated and similar to adverse events reported for similar compounds. Serious adverse events occurred in 16.7% of participants. The serious adverse events reported to be more likely for those taking siponimod included nervous system disorders and infections.

Comment:
“These results suggest a modest benefit for people with secondary progressive MS, which is a positive step forward in the global effort to speed solutions for people living with this chronic form of the disease,” said Timothy Coetzee, PhD, Chief Advocacy, Services and Research Officer at the National MS Society. “We look forward to learning additional details about its potential benefit and safety.”

Low-fat, plant-based diet in multiple sclerosis: A randomized controlled trial

Publication History

Published Online: July 06, 2016

http://www.msard-journal.com/article/S2211-0348(16)30100-6/fulltext#s0005

The role of diet in ameliorating the severity of multiple sclerosis (MS) has been long debated, but there remains a paucity of relevant research. Observational studies by Dr. Roy Swank, published between 1953 and 2003, suggested significantly reduced MS disease activity and disability progression and longer survival in people following a diet low in total and saturated fat compared with those who did not (Swank, 1953, Swank and Goodwin, 2003, Swank, 1970). Swank’s diet book, last published in 1987, remains popular among patients with MS. However, this approach to treating MS has never been subjected to a well-controlled clinical trial.

The supposed large clinical effect of the Swank low fat diet led to our hypothesis that a very-low-fat, plant-based diet might have a large effect on MRI activity. We conducted a pilot study to explore the tolerability and potential benefits of a very-low saturated fat, plant-based diet followed for 12 months by people with relapsing-remitting MS (RRMS) with the primary endpoint being brain MRI disease activity.

 

Study suggests antibody may have therapeutic effect on MS

Researchers have developed an antibody with potential therapeutic effects against multiple sclerosis. The discovery opens up a new strategy for controlling the disease.

For the cells of the immune system circulating in the bloodstream to reach the central nervous system, they must penetrate the blood-brain barrier and blood-spinal cord barrier. During previous work, the authors studied a factor involved in opening the blood-brain barrier, the NMDA receptor. They found that blocking the interaction of this receptor with tPA has beneficial effects linked with maintaining the integrity of the barrier.

Scientists at the Institut National de la Santé et de la Recherche Médicale, in France, developed a monoclonal antibody (Glunomab) directed against the specific site on the NMDA receptor to which tPA binds. In cellular models of the human blood-brain and blood-spinal cord barriers, the use of this antibody prevented opening of the barrier under inflammatory conditions, limiting the entry of lymphocytes. The team then tested the therapeutic effects of the antibody in an experimental mouse model of MS. After intravenous injection of Glunomab, the progression of partial or total paralysis of the limbs – as assessed by a clinical score – was blocked. In these treated mice, this effect was linked with reduced infiltration of lymphocytes into the nervous tissue, and reduced demyelination.

Results of mouse model studies sometimes do not translate to humans and may be years away from being a marketable treatment. However, the authors argue that by preventing myelin destruction by the cells of the immune system, this strategy might represent a promising therapy for the control of MS.

The study was published in the journal Brain.

MS Trial Alert: Researchers Recruiting People with Relapsing MS for Antibody Study

Summary: Investigators at seven sites in the United States are recruiting at least 24 people with relapsing MS for a study of ublituximab (TG Therapeutics, Inc.), an experimental monoclonal antibody administered via intravenous infusion. At most, 100 people will be enrolled.

Rationale: Ublituximab is a new monoclonal antibody that binds to a molecule (CD20) on the surface of immune cells called B cells, and depletes them from circulation. B cells have several functions including making antibodies, and evidence suggests they play a role in immune-system mediated damage to brain and spinal cord tissues in MS. Other therapies targeting B cells (rituximab, ocrelizumab) have shown some benefit in clinical trials. Ublituximab binds to CD20 in a unique way, and thus may have greater B cell depletion capabilities than similar agents. Clinical trials are ongoing in people with blood cancer as well.

Eligibility and Details: Participants should be aged 18 to 55, have a diagnosis of relapsing MS, and have had more than one relapse in the previous two years. Further enrollment criteria are available from the contact below.

Participants are initially randomly assigned to receive either ublituximab or placebo infusions (infusions range from 1 to 4 hours).  After 28 days, participants receiving placebo will receive ublituximab.

The primary outcomes being measured are the levels of B cell depletion, and the number of participants who experience adverse events. Secondary outcomes include monitoring relapses and MRI-detected disease activity.

Contact: To learn more about the enrollment criteria for this study, and to find out if you are eligible to participate, please contact Koby Mok, PhD, via e-mail at kmok@tgtxinc.com, or by phone at 949-422-2468.

Sites are enrolling in the following cities:
Fort Collins, CO
Lexington, KY
San Antonio, TX
Knoxville, TN
Columbus, OH
Phoenix, AZ
Round Rock, TX

Download a brochure that discusses issues to think about when considering enrolling in an MS clinical trial (PDF).

Finding Solutions for the Advanced Care Needs of People with MS

While researchers are working to identify new and better strategies to stop MS, restore function and end MS forever, people whose MS has become more disabling—and their family members and friends—need information right now about how to manage the challenges they face. With these goals in mind, the National MS Society convened a group of key stakeholders – including people with MS, support partners, Society staff and clinicians from the fields of neurology, primary care, rehabilitation medicine, psychology, nursing, physical therapy and speech pathology– to help inform the Society’s role in finding solutions for individuals and families who are facing advanced care needs.

“At the Society, when we face a challenge, we get the brightest minds together and put the problems on the table,” said Cyndi Zagieboylo, President & CEO of the National MS Society. “We need to pursue every opportunity to support people with advanced MS in living their best lives.”

What It’s Like

People living with MS lent a vital voice to the process. “It’s going to be very important as you think about this that you understand our lived experience,” urged Lisa Iezzoni, MD, a health services researcher who has MS. “It takes me about 10 times longer to do the most basic task.”
Karen Jackson, who lives with primary progressive MS, agreed. “Having advanced MS means I have lost the ability to be spontaneous,” she said. “I am forced to plan every minute of every day. The only thing more exhausting than planning my day, is not planning. It takes an annoying sequence of action steps to achieve even the smallest goal, like buying gas or parking the car.”

Resilience, however, rang through despite the challenges of advanced care needs, which for both of these women includes wheeled mobility. “When people ask me how I feel about my MS, I tell them that I’m not sick,” insisted Dr. Iezzoni. “I just can’t walk.” Ms. Jackson added, “Explain to people what your needs are. They want to help.” It’s worth the effort, she says. “Not participating in life is not an option.”

If I Have to Use a Wheelchair…
Getting a wheelchair was noted to be a “line in the sand” for many people living with MS, who often view the choice to use one as a loss of independence.  Meanwhile, by trying to stay on their feet, people might be curtailing activities because of increased fatigue, or concerns about stumbling or falling.

“One of our challenges is that the wheelchair is used to symbolize disability,” said physical therapist Jean Minkel (Independence Care System. New York). “The wheelchair should not be considered a failure of therapy.”

Dr. Iezzoni heartily agrees. “When I finally started using a wheelchair 14 years after my first MS symptom, it was like spring after a housebound winter,” she said. “Silliness – that I was afraid people wouldn’t think I was strong because I was using a wheelchair.” Ms. Jackson agreed. “I’m learning to navigate a new normal,” she said. “My goal when I meet you is to have my chair disappear in 10 minutes, so that you only see me!”

Evaluating the home environment is key to determining the type of mobility device needed. “A picture is worth a thousand words and a home visit is a narrative,” said Ms. Minkel.  “To understand the need, we need to see the environment. For example, show me what the front door looks like.”

The wheeled device is not the only crucial factor – so is choosing the proper cushion to sit on. Some cushions can relieve pressure, thus preventing pressure sores (sites of damaged skin that can cause serious infections). “Thirty percent of our clients are at risk for pressure sores,” said Minkel. “Only two percent get them because they have pressure-relieving wheelchair cushions.”

The National MS Society provides guidance for people with MS and healthcare providers to navigate the process of choosing and obtaining coverage for a wheeled device.

Finding Solutions
Participants considered other key issues related to the advanced care needs of people with MS, naming some difficult problems and suggesting solutions.

  • Breathing easier — “Respiratory dysfunction begins very early in the disease process,” noted physical therapist Donna Fry, PhD (University of Michigan-Flint). But, she said, respiratory exercises can improve strength in respiratory muscles even late in the disease. Dr. Fry’s team has shown these improvements using “threshold inspiratory muscle trainers,” inexpensive devices that can help breathing muscles to get stronger. “Most clinicians are not aware of the potential early involvement of the respiratory system in people with MS and of accessible, inexpensive equipment that can enhance muscle strength,” she added.
  • Muscle spasticity — “Quite a few people with MS are experiencing significant problems from spasticity,” said neurologist Francois Bethoux, MD (Cleveland Clinic). Spasticity may be as mild as the feeling of tightness of muscles or may be so severe as to produce painful, uncontrollable spasms in the extremities, usually the legs. Dr. Bethoux believes spasticity can often be managed without specialized care. “Optimal care would involve an early diagnosis, setting realistic goals, and re-evaluation,” he said. Plus, stretching is vital, even if mobility is impaired
  • Swallowing — “We all swallow 400-500 times a day, often without knowing,” said speech-language pathologist Alex Burnham (The Boston Home). “But 30-40% of people with MS can have problems with swallowing.” The consequences can be serious – breathing in food or fluids, choking, malnutrition, dehydration, and not taking medicine. Especially later in the disease, says Mr. Burnham, swallowing and feeding issues can have dramatic effects on quality of life, especially if it limits enjoying a meal with friends and family or prevents someone from eating favorite, culturally-significant foods. Mr. Burnham advocated for screening for these problems during regular visits. “Ask patients, have you had any trouble eating? Swallowing your pills?” Burnham also mentioned novel therapies that may prove helpful, such as the “free water protocol,” in which patients are allowed to have water by itself to improve hydration. Another method is neuromuscular electrical stimulation, applied in low doses to the neck
  • Speech — Swallowing disorders can occur hand-in-hand with speech difficulties. “It’s never too early to start thinking about assistive technology, especially for people with a wide fluctuation of symptoms,” noted Mr. Burnham. “They might be fine in the morning, but then if they don’t get a nap, fatigue makes it hard for them to speak intelligibly later in the day.” Give people with MS an opportunity to use as many different modes of communication as possible, he advised. “Miscommunication can lead to frustration, social isolation, and a loss of independence,” said Mr. Burnham. “Maintaining any form of communication is critical for empowerment, relationships, and appropriate disease management.”  , including the use of smartphone applications.
  • Thinking and mood problems – “Cognitive changes are among the most prevalent reasons that people with MS are admitted to nursing homes,” said Rosalind Kalb, PhD, Vice President, Healthcare Information and Resources at the Society. “We need to be providing strategies to help people compensate for cognitive changes, and we need to speak to family members, since families may help to pick these changes up earlier.” With mood, it’s vital to understand that although depression in common in MS, some mood changes may be a natural consequence of the process of an advancing chronic disease. “People may be grieving over changes,” said Dr. Kalb. “We need to treat depression when it is present and also be respectful and comfortable with talking with people who are not depressed about how they want to live the rest of their lives.”

Achieving Optimal Care
The group discussed how to achieve optimal care for people with advanced MS.  Nicholas LaRocca, PhD, Vice President of Healthcare Delivery and Policy at the Society, noted that health care concerns are changing as the MS population gets older. “The average age of people with MS has increased by over 30 years since 1984,” he said. “Coexisting conditions, such as hypertension, increase with age and appear to be increasing in MS. Furthermore, people with MS who have some of these conditions at diagnosis reach the most severe level of mobility impairment faster than those who don’t.”

The group agreed that education is needed on both ends of this spectrum. Primary care providers need to be educated about MS so that they can distinguish MS symptoms from conditions that require primary care. And people with MS need to be educated about the importance of watching out for their own health. “A person with a disability still needs their cholesterol checked,” said Ms. Minkel. ”They still need their blood pressure checked.” Neurologists and primary care providers need to communicate, collaborate and coordinate their care of a person with MS.

Early and ongoing evaluation of symptoms also is key. “We need to educate people with MS and their caregivers about advocating for chronic care issues,” said Ruth Whitham, MD (Oregon Health& Science University). “Perhaps we can develop an advanced MS care checklist that would include symptoms to think about and what to do if you notice them.” The goal is to help people with MS to advocate for early and ongoing assessment, and for healthcare providers to ask routinely about changes that may be occurring throughout all bodily systems.
Importantly, people with MS need to know they have the right to advocate for care, regardless of how advanced their MS. “We don’t ever want a person to hear, ‘There’s nothing more we can do for you,’” added Dr. Kalb.

Caring for Caregivers
Speakers paid careful attention to how advanced care needs can affect caregivers.
“Families can become isolated,” said psychologist David Rintel, EdD, whose father lived with MS. “You feel pretty different from everyone else, and that isolation is harmful to your physical and mental health.” He advised that healthcare providers should see the caregiver occasionally along with the patient, if the patient grants permission, to get their perspective, and also see how the caregiver themselves are doing. “We need to learn the signs of burnout, such as depression, and increased use of alcohol,” he said. “Caregiver burden is real.”

There also is much that a caregiver needs to learn – navigating the healthcare system, how to transfer people safely, and management of bladder and bowel problems. “Dealing with bowel/bladder issues is actually a leading cause of caregiver burnout,” added nurse Cindy Walsh (The Boston Home).

“Families have to learn how to ask for help,” said Dr. Rintel. “They have to ask in a way where they say what, where, when and how long. Most people would help if they understood specifically what you need.”

Next Steps
The group identified the highest priority research questions that need to be answered concerning the care and support of people with advanced care needs and their families, pinpointing questions in the areas of assistive technology; comorbidities and primary care; health care system issues (e.g., insurance coverage); long-term care; symptoms and complications; skin care; speech, swallowing, and pulmonary functions; and the benefits of wellness/lifestyle interventions. They are now formulating a prioritized list of these questions to help inform the Society’s next steps.

A white paper describing the meeting’s discussion highlights and recommendations regarding the Society’s response to the needs of those affected by advanced MS will be posted on the Society’s web site, and a similar paper will be submitted for publication in a peer-reviewed journal.

Help is Available Now
Individuals nationwide may contact the Society’s MS Navigator® program via the Society’s toll-free help line 1-800-344-4867 (1-800-FIGHT MS) or via email (contactusNMSS@nmss.org). The MS Navigator Program connects people to resources,, helps people access optimal healthcare and understand benefits such as health insurance, face financial challenges and planning for the future, and find support when MS progresses.

Right now, MS activists are engaged on a number of fronts to improve quality of life and access to care. Among these is advancing home modification tax credit legislation, to provide financial relief for home modifications to promote safety and mobility.
The National MS Society provides support to people living with advanced MS, including care guides for families, information about symptom management, a guide to financial planning, and information on advanced directives. Read more

The Society also provides support for healthcare professionals who are seeking to help people with MS obtain care at home, in nursing homes, assisted living facilities, or adult day homes. Read more

 

Study Shows Expansion of Stem Cell Clinics in the U.S. and the Need for Better Oversight

Researchers have published a paper describing the proliferation of stem cell clinics in the United States and ethical issues and regulatory concerns that come with marketing unproven treatments for many conditions. Their study shows that many different types of unproven stem cell treatments are being offered, and highlights concerns for the safety of people who undergo these treatments.

There is exciting progress being made through innovative research related to the potential of many types of stem cells for slowing MS disease activity and for repairing damage to the nervous system. At present, there are no approved stem cell therapies for MS. People need the best available information to understand this exciting area of research and make decisions related to this complex issue.

The paper’s findings support the need for stem cell therapy to be explored in the context of carefully conducted clinical trials that can determine what the optimal cells, delivery methods, safety and actual effectiveness of cell therapies might be for people with MS.

Positive Results from Study of Bone Marrow-Derived Stem Cells in People with Aggressive, Relapsing MS

Summary

  • Researchers in Canada have published results of a long-term trial of an individuals’ own (autologous) hematopoietic (blood cell-producing) stem cell transplantation. The study involved 24 people with aggressive relapsing-remitting MS whose disease was not controlled with available therapies.
  • Three years after the procedure, 70% remained free of disease activity, with no relapses, no new MRI-detected inflammatory brain lesions, and no signs of progression.
  • None of the surviving participants, who were followed for 4 to 13 years after the procedure, experienced clinical relapses or required MS disease-modifying therapies to control their disease, and 40% experienced reductions in disability.
  • One of the participants died and another required intensive hospital care for liver complications. All participants developed fevers, which were frequently associated with infections, and other toxicities.
  • Additional research is focusing on figuring out who might benefit from this procedure and how to reduce its risks.

“These results suggest that aggressive MS may be stopped with an effective but risky procedure, for a subset of people,” said Dr. Bruce Bebo, Executive Vice President, Research, at the National MS Society. “Additional research by investigators around the world is focusing on figuring out who might benefit from this procedure and how to reduce its risks, which can include death.”

Details
Background: An experimental procedure that has been explored for several years in MS is called “autologous hematopoietic (blood cell-producing) stem cell transplantation” – or HSCT. This procedure has been used in attempts to “reboot” the immune system, which launches attacks on the brain and spinal cord in people with MS.

In HSCT, the stem cells (derived from a person’s own bone marrow or blood) are stored, and the rest of the individual’s immune cells are depleted by chemotherapy. Then the stored stem cells are reintroduced by infusion into the vein. The new stem cells migrate to the bone marrow and over time produce new blood cells, including immune cells. The goal of this currently experimental procedure is to establish a new immune system that no longer recognizes myelin and other nervous system tissue as dangerous. In theory, this should stop the attacks that lead to tissue damage and disability.

There are a number of laboratories around the world testing variations of HSCT for the treatment of autoimmune diseases, including MS. Preliminary findings suggest this is a promising, but potentially risky strategy for the treatment of MS.

The Study: Drs. Harold Atkins, Mark Freedman and team at the Ottawa Hospital, University of Ottawa and other institutions in Canada conducted a Phase 2 trial of HSCT that involved 24 people with aggressive relapsing-remitting MS whose disease was not controlled with available therapies. No control group was used which would have enabled comparison against the results found in the treatment group. The procedure used by this group included complete destruction of bone marrow cells and an additional step that enriched the transplanted cells for stem cells.

Results – Safety: One of the participants died of transplantation-related complications that caused liver failure and another required intensive hospital care for liver complications. The treatment regimen was modified over the course of the study to reduce toxicity, but all participants still developed fevers, which were frequently associated with infections.

Results – Effectiveness: Three years after the procedure, 70% of the participants remained free of disease activity, meaning they had no relapses, no new MRI-detected inflammatory brain lesions, and no signs of progression. The remaining 30% experienced progression of disability. In addition, for the entire follow-up period ranging from 4 to 13 years after the procedure, of the 23 survivors:

  • None experienced clinical relapse, had new active inflammatory MRI brain lesions, or required MS disease-modifying therapies to control their disease.
  • The average rate of brain atrophy (shrinkage), a measure that has been linked to MS progression, returned to levels associated with normal aging.
  • 40 percent experienced some lasting reversal of disability such as vision loss, muscle weakness and balance problems.
  • Some were able to return to work or school.

The results were published online on June 9, 2016 in The Lancet.  Major funding for the study came from the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Foundation.

Next Steps: Rigorous clinical trials of stem cell therapies are needed to determine their safety and effectiveness in people with MS. Trials of this and other stem cell therapy approaches are taking place in Canada, the United States, Europe and elsewhere. To help explore the potential of stem cell therapy, in November 2015, the International Conference on Cell-Based Therapy for Multiple Sclerosis was convened in Lisbon, Portugal under the auspices of the International Advisory Committee on Clinical Trials in MS (a group jointly sponsored by the National MS Society and the European Committee for Treatment and Research in Multiple Sclerosis). Seventy leading researchers and clinicians conferred on clinical trials needed to provide answers about which types of cells, which route of delivery, and which types and stages of disease, would be the most promising approach for treating MS. Read more about this meeting

Read more about stem cells and MS

Canadian Researchers Uncover Rare Gene that Increases Risk of Progressive MS

Researchers at the University of British Columbia have uncovered a rare gene mutation that appears to dramatically increase the risk, in some individuals, of developing a severe form of progressive multiple sclerosis. While the cause of MS is not known, scientists believe several different factors, including susceptibility genes, may interact to trigger the disease. The gene was discovered in two unrelated families that had multiple members with MS. The researchers also determined that the gene (NR1H3) contains instructions for a protein called LXRA, which is thought to be a control switch for genes involved in many functions, including some that help control inflammation and integrity of nerve-insulating myelin in the brain and spinal cord. This type of discovery can provide crucial clues to biological pathways that underlie MS, and may lead to new approaches for stopping MS and restoring function. The study, by Drs. Carles Vilariño-Güell, Weihong Song, A. Dessa Sadovnick and others, was funded in part by the MS Society of Canada and appeared in the journal Neuron on June 1, 2016.

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