Mom's Story

A discussion about Mom's Story and MS…

Archive for the tag “treatment”

The Latest on Stem Cell Treatment

Recent media reports have featured news about a clinical trial involving harvesting a person’s own stem cells to treat aggressive multiple sclerosis.
• This treatment, called autologous haematopoietic stem cell transplant (HSCT), attempts to “reboot” the immune system, which is believed to launch attacks on the brain and spinal cord in people with MS.
• HSCT is under investigation in clinical trials in Canada, the United States, Europe and elsewhere. Clinical trials are needed to fully understand the benefits and risks of HSCT in MS, and who might benefit most from this approach, since it does not seem to be effective in all types of MS.
• In HSCT, stem cells from a person’s own bone marrow or blood are stored, and the rest of the individual’s immune cells are depleted usually by chemotherapy. Then the stored stem cells are reintroduced and over time they produce new cells that repopulate the body with immune cells.
• There is exciting progress being made through innovative research related to the potential of many types of stem cells both for slowing MS disease activity and for repairing damage to the nervous system.
• At present, there are no approved stem cell therapies for MS. Stem cell therapy is in the experimental stage, and it’s important for people to have the best available information to understand this exciting area of research and make decisions related to this complex issue.
• In November 2015, the International Conference on Cell-Based Therapy for Multiple Sclerosis was convened by the National MS Society and the European Committee for Treatment and Research in Multiple Sclerosis, bringing leading researchers and clinicians together to confer 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. A summary and consensus on next steps will be published by the conference organizers, with recommendations to help speed the development of new cell-based treatment solutions.
• With the urgent need for more effective treatments for MS, particularly for those with more progressive forms of the disease, we believe that the potential of all types of cell therapies must be explored. The Society is currently supporting 12 research projects exploring various types of stem cells, including cells derived from bone marrow, fat and skin, and has supported 68 stem cell studies over the past 10 years.

Small Pilot Trial Suggests High-Dose Vitamin D is Safe and Regulates Immune Responses in People with MS

Summary
• High-dose vitamin D supplementation increased vitamin D levels in the blood, was safe and tolerable, and reduced the proportion of immune cells that are thought to drive disease, in a small study of 40 people with relapsing-remitting MS.
• The trial was too small to detect differences in disease activity, but a larger Society sponsored trial of vitamin D supplementation is currently recruiting participants.
• The team (Elias S. Sotirchos, MD, Pavan Bhargava, MD, Peter A. Calabresi, MD, and colleagues, Johns Hopkins University School of Medicine, Baltimore) has published results in Neurology. Dr. Bhargava was funded by a Sylvia Lawry Physician Fellowship from the National MS Society.

Background: Multiple sclerosis involves immune attacks on the brain and spinal cord. A number of genetic and environmental factors influence whether a person will develop MS. These factors may also impact the severity of the disease. There is growing scientific evidence that low levels of vitamin D in the blood are a risk factor for developing MS. In lab mice, vitamin D can reduce the effects of EAE, an MS-like disease, and some evidence suggests it may impact ongoing disease activity in people who have MS.

An important initial step to pursuing this lead was to determine whether taking large doses of vitamin D was safe and provides any hints of impact against the immune activity that is associated with MS. A team at Johns Hopkins University undertook this preliminary step to determine whether a larger-scale clinical trial was warranted.

The Study: Investigators randomly assigned 40 people with MS to receive either 800 IU of vitamin D, or 10,400 IU, daily for six months (nutritional supplementation is typically 600 IU). Participants were maintained on standard disease modifying treatment throughout the course of the study. Blood tests were done at three and six months to determine whether the dose increased the levels of vitamin D in the blood, and immune system effects. Blood and urine were assessed for calcium levels, since an excess of calcium can be a side effect of high-dose vitamin D supplementation. The primary goals of this study were to determine safety and effects on immune activity markers.

The investigators reported a few adverse events that did not differ between the groups, and they were all minor.

Vitamin D levels increased more in the high-dose group, to a level that has been suggested as the optimal target for people with MS. Immune cells known as Th17 cells – which have been suggested to be major players in the immune attack on the brain and spinal cord in MS – were reduced in the high-dose group, but not in the low-dose group. Investigators also found that the higher the levels of vitamin D in the blood, the greater the reduction of Th17 cells.

Results were published in Neurology (published early online, December 30, 2015).

Next Steps: This team is now conducting a larger trial at several centers nationwide, in which they are recruiting 172 people with relapsing-remitting MS to compare the effectiveness of 600 IU of vitamin D supplementation versus 5000 IU vitamin D supplementation at reducing MS disease activity, when added to standard therapy with glatiramer acetate (Copaxone®, Teva Pharmaceutical Industries). The study is funded by a research grant from the National MS Society, with support from the Society’s Greater Delaware Valley Chapter.

Further research in the laboratory also is suggesting that vitamin D’s capabilities go beyond immune regulation. Read more

Read more about the larger, ongoing study
Read more about research on vitamin D and MS

New Lab Studies Add Evidence That High Salt Diets Increase Inflammation and May Have Implications for MS

Summary
• The results from two recently published laboratory studies suggest that high levels of salt shift the balance of the immune system toward inflammation, and that salt alters the function of several types of immune cells pertinent to MS.
• These two studies, which were both published in the Journal of Clinical Investigation, were led by Dr. David Hafler (Yale University) and Dr. Dominik Müller (Max-Delbruck Center, Berlin, Germany).
• Dr. Hafler is funded by the National MS Society to study the impact of high salt on the immune system, and the Yale team is also conducting a pilot clinical trial to explore the impact of high- and low-salt diets on MS disease activity.

Background: Eating high levels of salt, which is part of the typical Western diet, has been linked to heart disease, chronic inflammation, and cancer. Recent lab reports have also suggested that dietary salt can speed the development of the immune attack in an MS-like disease in mice, and that the mouse disease responds differently to salt depending on the gender and genetic makeup of the mice. One small study in people found a possible link between dietary salt levels and relapses in people with MS, but this study suggested a link, which is not the same as establishing an actual cause. So far, laboratory findings related to the effects of salt have been stronger than the few studies that have been reported in people. Understanding whether high dietary salt is a risk factor for developing MS or for worsening disease activity is an active area of research.

The Studies: Two studies recently published in the Journal of Clinical Investigation suggest that high dietary salt affects two types of immune cells in a way that increases inflammation, a state that is generally considered harmful in MS. A study by National MS Society-supported researchers at Yale University and Harvard Medical School led by David Hafler, MD, investigated the effects of high salt on regulatory immune cells called “Tregs.” Tregs normally suppress immune responses by other immune cells, but in people with MS Tregs have been shown to be less able to perform this helpful function to turn off attacks. The team showed in mice and in cells in lab dishes that high salt blocks the ability of Tregs to suppress potentially harmful immune cells, and shifts Tregs toward activity that increases inflammation.

The other study, by an international team led by Dominik N. Müller at the Max-Delbruck Center in Berlin, Germany, investigated immune cells called “macrophages.” This study showed that high salt blocks the activation of a subset of macrophages, reducing their ability to suppress inflammatory cells and creating an imbalance in the immune system. In mouse models, high salt diets also delayed wound healing.

Comment: Taken together, these laboratory studies add new evidence that high levels of dietary salt may increase inflammation and autoimmunity, and decrease the ability of regulatory cells and processes to limit harmful immune cell activity. More studies are needed to determine the possible role of a high-salt diet in the risk of developing MS and whether reducing salt intake may be helpful for reducing disease activity in people with MS. Dr. Hafler is funded by the National MS Society to study the impact of high salt on the immune system, and the Yale team is also conducting a pilot clinical trial to explore the impact of high- and low-salt diets on MS disease activity.

Read more about dietary factors that may play a role in MS
Read more about research on the immune system in MS

Case of PML Reported in Person Taking Tecfidera®

In December 2014, important label changes were made to the prescribing information for Tecfidera® (dimethyl fumarate, Biogen Idec) including information regarding an individual who developed PML. Most recently, Biogen has confirmed report of a second case of PML (progressive multifocal leukoencephalopathy, a viral infection of the brain that often leads to death or severe disability) that occurred in a person taking Tecfidera. According to the company, the 64-year-old patient has primary progressive MS and experienced severe and prolonged lymphopenia (decreased white blood cells) during treatment with Tecfidera. Severe and prolonged lymphopenia is a known risk factor for PML and Consideration should be given to interrupting treatment if lymphocyte counts are low for more than six months. The patient is stable and is not hospitalized. Biogen has reported the case to the U.S. Food and Drug Administration (FDA).
PML is caused by the re-activation of a virus called the JC (John Cunningham) virus, a common virus to which many people have been exposed. PML has emerged in people using other medications, including the MS treatment Tysabri® (natalizumab, Biogen), and the MS treatment Gilenya® (fingolimod, Novartis AG).
It is not possible at this point to determine a person’s risk for developing PML because there have been so few cases in people taking Tecfidera. There have been two reported cases of PML in people with MS among the more than 155,000 individuals who have been treated with Tecfidera to date.
The symptoms of PML are diverse and can be similar to MS symptoms. For this reason, individuals should be alert to any new or worsening symptoms and report them promptly to their MS healthcare provider. Learn more about the risk factors and symptoms of PML from the web site of The PML Consortium. Individuals who have concerns about this report should discuss it with their MS healthcare providers.
If and when the FDA or Biogen provide additional information or recommendations for people taking Tecfidera or other MS medications, the National MS Society will share it as soon as possible.

The MS Hug???

The MS Hug: What Is It? How Is It Treated?
http://www.healthline.com/health/multiple-sclerosis/ms-hug#WhatIsMS1

Study Uncovers Gene Variation Linked to Response to MS Therapy; May Open Up New Treatment Approaches

Collaborating researchers in the U.S. and Italy have uncovered a gene variant that appears to influence whether a person responds well to interferon beta, a commonly used therapy for relapsing forms of MS. More broadly, the gene may regulate immune activity in unexpected ways, and its discovery may lead to new approaches to stopping inflammation and immune attacks in MS. Drs. Federica Esposito and Filippo Martinelli Boneschi (San Raffaele Scientific Institute, Milan), Philip L. De Jager (Brigham and Women’s Hospital, Boston) and colleagues have published their results in the Annals of Neurology (Early online May 14, 2015). The study was supported by the National MS Society and several other agencies.

Background: For reasons that are unclear, some people with relapsing forms of MS do not respond well to therapy and continue to experience disease activity despite being on a disease-modifying therapy. Previous genetic studies in MS have uncovered over 159 genetic variations that contribute to making people susceptible to developing MS, but these studies haven’t identified genetic variations that influence how a person responds to treatment. Finding a way to identify early in the disease course the best therapy for an individual – a “personalized medicine” approach – is likely to improve outcomes of treatment and quality of life for people living with MS. One of the lead authors of this study, Dr. De Jager, recently won the Barancik Prize for Innovation in MS Research for tackling critical questions like this with the goal of developing personalized treatments and prevention of MS.

This Study: Trying a different approach to search for genetic influences on treatment responses, the investigators first studied a group of individuals with MS who were taking interferon beta or glatiramer acetate. The individuals were classified as being responders, partial responders, or non-responders to their medication based on specific criteria. Then the researchers analyzed their full complement of genes (genome-wide association study) and found one genetic variant that was consistently associated with lack of response to interferon beta. When the researchers repeated this in three other groups of people with MS from Italy, France and the U.S., this finding held up.

The genetic variant (rs9828519) is near a gene (SLC9A9) that controls pH levels (acidity) within cells. The team explored functions of this gene, and found that its activity was diminished in people more likely to have MS relapses. They also conducted laboratory work, finding suggestions that the gene appears to play a role in regulating immune cell activity, and that its loss leads to damaging immune reactions. This suggests the gene may play a broader role in regulating immune activity.

Comment: Although the results of this study are not yet ready for applying to the management of MS, this discovery may lead to new approaches for stopping inflammation and immune attacks in MS. In addition, this study is an important step toward the goal of personalized medicine. The researchers point out that additional research is warranted to confirm their findings and to determine whether the genetic variant is relevant to how well people respond to other MS medications.

New molecule may lead to inflammation inhibitor

Scientists have developed a new drug-like molecule that can inhibit inflammation. The find has shown promise in preventing the progression of multiple sclerosis.
Walter and Eliza Hall Institute scientists have developed a small drug-like molecule called WEHI-345 that binds to and inhibits a key immune signaling protein called RIPK2. This prevents the release of inflammatory cytokines. Examining WEHI-345’s potential to treat immune diseases in experimental models of MS, it was found that WEHI-345 prevented further progression of the disease in 50 percent of cases after symptoms of MS first appeared.
Results of mouse model studies sometimes do not translate to humans and may be years away from being a marketable treatment. Calling the results extremely important, researchers said WEHI-345 had potential as an anti-inflammatory agent.
The study’s lead author, Dr Ueli Nachbur, said institute scientists would use WEHI-345 to further investigate the signaling pathway that produced inflammatory cytokines and to develop a better, stronger inhibitor of RIPK2 for treating inflammatory disease. “This signaling pathway must be finely balanced, because WEHI-345 only delayed signaling rather than blocked it. Nevertheless, this delay is enough to completely shut off cytokine production,” he said.
The research was published in the journal Nature Communications.

Give-away

The Giveaway for Mom’s Story on Amazon has ended. Thanks for participating. Mom’s Story, a Child Learns about MS is available on Amazon in paper: http://www.amazon.com/Moms-Story-Child-Learns-About/dp/147835819X/ref=tmm_pap_title_0?ie=UTF8&qid=1427210007&sr=1-1

and as an ebook on Kindle: http://www.amazon.com/Moms-Story-Mary-Nickum-ebook/dp/B00C0BO9TA/ref=tmm_kin_swatch_0?_encoding=UTF8&sr=1-1&qid=1427210007

Also Available:
http://www.barnesandnoble.com

Single-Payer Healthcare

Lets take this seriously:

How Common Is It To Have MS With Other Medical Conditions? First Results from the MS “Comorbidities” Project

Unfair as it seems, MS doesn’t keep other disorders away. It’s possible to have MS and “something else” at the same time. A new international initiative is being launched to understand how common it is for people with MS to have other conditions and how these other conditions may impact the course and treatment of an individual’s MS. The first stage of this project is now published, in preparation for an international scientific workshop jointly supported by the National MS Society and the European Committee for Treatment and Research in MS (ECTRIMS) to focus attention on comorbidities and determining next steps to finding solutions for people with MS.
Background: In scientific terms, having two chronic medical conditions at once is called “comorbidity.” There is growing recognition that comorbidities may complicate the diagnosis of MS and also influence disease progression, as well as an individual’s wellness and quality of life. In addition MS some other disorders may have risk factors in common. For these reasons, the MS Comorbidities Project is seeking to characterize the types and frequencies of comorbidities in MS in advance of a scientific meeting to map out next steps for research strategies to address this gap area. This project is being undertaken by the International Advisory Committee on Clinical Trials in MS, a committee comprised of international leaders in MS research and clinical care that is jointly supported by the National MS Society and ECTRIMS.
The first phase of this project was a systematic review of existing published studies related to specific medical conditions in people who have MS. Ruth Ann Marrie, MD, PhD (University of Manitoba) and colleagues* in Denmark, Italy and the U.S., now report their findings in seven papers published in the MS Journal. (Read overview and companion papers; no subscription is needed.) The review was supported in part by the National MS Society (U.S.A.) and a Don Paty Career Development Award from the MS Society of Canada.
Review Results: The authors identified more than 7,000 studies on a variety of comorbidities and MS, and narrowed these down, completing a full-text review of 249 studies that were conducted between 1905 and 2012. Most were conducted in North America or Europe, leading the authors to comment that little is known about comorbidities that occur with MS in Central or South America, Asia, or Africa. In addition, the quality and design of the studies were so variable that it was difficult to compare results. Nevertheless, their extensive research yielded these highlights, among many others:
• The five most prevalent disorders occurring alongside MS were depression, anxiety, high blood pressure, high cholesterol, and chronic lung disease.
• The most prevalent autoimmune diseases occurring with MS were thyroid disease and psoriasis.
• The types of cancer that occurred most often in people with MS were cervical, breast, and digestive system cancers. There appeared to be a higher than expected risk of meningiomas and urinary system cancers, and a lower than expected risk of pancreatic, ovarian, prostate and testicular cancer, compared to the general population.
• Some disorders were found more often than expected by the investigators based on previous research, such as heart disease, congestive heart failure, stroke, arthritis, inflammatory bowel disease, irritable bowel syndrome, seizure disorders, bipolar disorder, sleep disorders, and alcohol abuse.
Comment: The authors suggest that further work is necessary to develop data sources that examine MS comorbidities worldwide, and that are specific to individuals of different ages, genders, and ethnicities. They also conclude that efforts should be coordinated so that methodologies are similar and results can be compared.
To this end, the Society and ECTRIMS are convening a workshop that will move this research forward. The International Advisory Committee on Clinical Trials in MS and other experts in MS research will meet in spring 2015 to discuss next research steps, such as available data that may facilitate further research and which comorbidities demand more immediate focus.

Post Navigation