For the first time, scientists have shown they can correct gene mutations in human embryos — a breakthrough with implications for multiple sclerosis (MS) and several other diseases. This landmark study was a collaboration involving scientists at the Salk Institute in La Jolla, California; the Oregon Health & Science University in…

My multiple sclerosis (MS) brings fatigue, pain, and instability into my life, but surprisingly, it also makes me more aware of my life and surroundings. For me, that means being more aware in the present moment and focusing on the good in my life right now. Living in…

Women who breastfeed for 15 months or longer may have a lower risk of developing multiple sclerosis (MS) than those who breastfeed for shorter periods or not at all, according to a recent study. The study also suggests that women who had their first…

Advertising for stem cell therapies not supported by clinical research — often made directly to patients and sometimes promoted as a "cure" for diseases like multiple sclerosis or Parkinson's — is a growing problem that needs to be addressed and regulated, a team of leading experts say, calling such "stem cell tourism" potentially unsafe. Stem cell tourism is the unflattering name given to the practice of encouraging patients to travel outside their home country to undergo such treatment, typicaly at a private clinic. The article, titled "Marketing of unproven stem cell–based interventions: A call to action" and recently published in the journal Science Translational Medicine, was co-authored by scientists with universities and hospitals in the U.S., Canada, U.K., Belgium, Italy, Japan, and Australia. It focuses on the global problem of the commercial promotion of stem cell therapies and ongoing resistance to regulatory efforts. Its authors suggest that a coordinated approach, at national and international levels, be focused on "engagement, harmonization, and enforcement in order to reduce risks associated with direct-to-consumer marketing of unproven stem cell treatments." Treatments involving stem cell transplants are now being offered by hundreds of medical institutions worldwide, claiming efficacy in repairing tissue damaged by degenerative disorders like MS, even though those claim often lack or are supported by little evidence . They also noted that the continued availability of these treatments undermines the development of rigorously tested therapies, and potentially can endanger a patient's life. The researchers emphasize that tighter regulations on stem cell therapy advertising are needed, especially regarding potential clinical benefits. They support the establishment of international regulatory standards for the manufacture and testing of human cell and tissue-based therapies. "Many patients feel that potential cures are being held back by red tape and lengthy approval processes. Although this can be frustrating, these procedures are there to protect patients from undergoing needless treatments that could put their lives at risk," Sarah Chan, a University of Edinburgh Chancellor’s Fellow and report co-author, said in a news release. Chan and her colleagues are also calling for the World Health Organization to offer guidance on responsible clinical use of cells and tissues, as it does for medicines and medical devices. "Stem cell therapies hold a lot of promise," Chan said, "but we need rigorous clinical trials and regulatory processes to determine whether a proposed treatment is safe, effective and better than existing treatments." According to the release, the report and its recommendations followed the death of two children at a German clinic in 2010. The clinic has since been shut down. Certain stem cell therapies — mostly involving blood and skin stem cells – have undergone rigorous testing in clinical trials, the researchers noted. A number of these resulted in aproved treatments for certain blood cancers, and to grow skin grafts for patients with severe burns. Information about the current status of stem cell research and potential uses of stem cell therapies is available on the website EuroStemCell.

New analyses of how Merck’s Mavenclad (cladribine tablets) act to treat relapsing multiple sclerosis (MS) give researchers an entirely new picture of immune processes leading to the disease. Data showed that the drug lowers both immune B-cells and, to a lesser degree, T-cells. But the numbers of both cell…

A gene mutation may explain the uncontrolled, inflammatory immune response seen in autoimmune and chronic inflammatory diseases like multiple sclerosis, scientists at the Research Institute of the McGill University Health Centre (RI-MUHC) report. It's a discovery that, they said, appears to be "a big step in the right direction." According to the study, published in the journal Science Immunology, alterations in the FOXP3 gene affect specific immune cells called regulatory T-cells, or Tregs. Those mutations hamper Tregs in performing a crucial regulatory role, leading to a loss of control over the immune system’s response to a perceived threat. “We discovered that this mutation in the FOXP3 gene affects the Treg cell’s ability to dampen the immune response, which results in the immune system overreacting and causing inflammation,” Ciriaco Piccirillo, the study's lead author and an immunologist in the Infectious Diseases and Immunity, Global Health Program, at the RI-MUHC, said in a news release. Tregs are known to be the immune system players responsible for keeping other immune cells under control, preventing them from attacking the host’s own tissues, while maintaining a proper immune response against harmful agents. The normal activity of Treg cells is essential for preventing excessive immune reactions. The FOXP3 gene is also well-known, and documented, to be essential for proper Treg cell function. However, the mechanisms by which FOXP3 gene is involved in Treg cell activities are still poorly understood. In the study, “Suppression by human FOXP3+ regulatory T cells requires FOXP3-TIP60 interactions,” the research team — in collaboration with researchers at University of Pennsylvania, University of Washington School of Medicine, and Teikyo University School of Medicine in Japan — evaluated the impact of a FOXP3 gene mutation in autoimmunity response. Taking advantage of cutting-edge technology, the team studied samples from two patients carrying a common FOXP3 gene mutation, which caused a genetic immune disorder called IPEX. Interestingly, the researchers found that this genetic variant did not reduce the number of Treg cells or the levels of FOXP3 protein. Instead, the mutation altered the way Tregs could suppress other immune cells to prevent overactivation. “What was unique about this case of IPEX was that the patient’s Treg cells were fully functional apart from one crucial element: its ability to shut down the inflammatory response,” said Piccirillo. “Understanding this specific mutation has allowed us to shed light on how many milder forms of chronic inflammatory diseases or autoimmune diseases could be linked to alterations in FOXP3 functions,” added Khalid Bin Dhuban, the study's first author and a postdoctoral fellow in Piccirillo’s laboratory. The team developed a compound capable of restoring Treg cells' ability to control the immune system in the presence of this specific FOXP3 gene mutation. Tested in animal models of colitis and arthritis, two chronic inflammatory diseases, the compound reduced inflammation and restored normal Treg function. Researchers now plan to develop similar drugs that may be of use in other diseases where Treg cells are known to be defective, including multiple sclerosis, type 1 diabetes, and lupus. "Currently, we have to shut down the whole immune system with aggressive suppressive therapies in various autoimmune and inflammatory diseases," said Piccirillo. “Our goal is to increase the activity of these Treg cells in certain settings, such as autoimmune diseases, but we want to turn it down in other settings, such as cancer.” “This discovery gives us key insights on how Treg cells are born and how they can be regulated,” Piccirillo added. “With this discovery, we are taking a big step in the right direction.”

The multiple sclerosis therapy Tysabri could trigger melanoma, the Southern Network on Adverse Reactions (SONAR) has warned. Although its investigation failed to demonstrate that melanoma is more common among Tysabri-treated MS patients than in the general population, unusual features among the patients raise concerns about a possible link, the organization said. Contending that current monitoring efforts are inadequate, it suggested improvements that could generate a better understanding of the relationship between Tysabri treatment and cancer. The organization's report, published in the journal Cancer Medicine, was titled “Melanoma complicating treatment with natalizumab for multiple sclerosis: A report from the Southern Network on Adverse Reactions, also known as SONAR." SONAR is an organization that was formed in the Southern United States in 2010 to investigate adverse drug reactions that regulators might not be aware of. Its goal is to reduce the time it takes between detecting an adverse reaction and have regulators act on it. A case that a SONAR investigator came across led to the group investigating possible links between Tysabri and melanoma. A 43-year-old woman developed melanoma in her urethra, the tubing that drains urine from the bladder, after being treated with Tysabri for about two years. Melanoma is most often a skin cancer that is related to sun exposure, but the woman had no skin lesions. After extensive surgery, she relapsed and died when the cancer spread to other parts of her body. She had declined anti-cancer treatment. The case prompt SONAR to look for similar cases. Its investigators found seven studies that involved Tysabri-treated MS patients developing melanoma. In addition, they looked through the U.S. Food and Drug Administration’s Adverse Event Reporting System (FAERS) and the Tysabri Safety Surveillance Program. The surveillance program is part of the Tysabri Outcomes Unified Commitment to Health (TOUCH) database run by Tysabri's developer, Biogen, The research team found 137 cases in the FAERS database through April 1, 2014. The patients' average age was 45. Seventeen percent of the group developed tumors in locations not exposed to the sun, and nine died. The researchers said the database contained only about half the information it should have, such as tumor site, patients' family history of cancer, and earlier immunosuppressive treatment. Fifteen percent of the cases in the FAERS database were based entirely on information from the TOUCH database. Seventy-three percent were cases initially reported to FAERS but with TOUCH information added. Thirteen percent of the FAERS cases contained no additional information. Importantly, there was even less patient information in the TOUCH database than in the FAERS database. Out of eight items researchers believe a database should contain, TOUCH had information on two, on average. “The existence of the TOUCH Safety Surveillance Program, an FDA-mandated program, did not improve melanoma reporting,” the team wrote. This shortage of data stymies research into possible links between Tysabri treatment and melanoma, the researchers said. As an example, although the death rates in the databases were low, there was no information about survival in many cases, which could lead to flawed survival estimates. The investigation noted that patients received a wide range of Tysabri doses before they were diagnosed with melanoma. While some received only one or a few injections, others had been treated for a long time. These observations do not seem to support a link between Tysabri and melanoma, the team said. “A longer therapy duration would be expected if natalizumab caused melanoma via an immunologic pathway, unless existing nevi [lesions of the skin or mucus tissue] were already premalignant lesions,” the researchers wrote. But other information the team found seemed to suggest a Tysabri-melanoma link. For example, the average age of melanoma patients was much lower than that reported in the National Cancer Institute’s Surveillance, Epidemiology and End Results (SEER) database. The average age in the institute's database is 63, compared with 45 in the FAERS database and 41 in cases in academic journals. In addition, many patients developed tumors in unusual places not exposed to sunlight. Finally, the low melanoma death rate in Tysabri-treated patients differed from that seen in the general population. All these factors suggest that melanoma after Tysabri treatment could differ from other types of melanoma, the researchers argued. While the molecular workings of Tysabri might promote melanoma growth, studies so far have not found a relationship between the drug and this cancer. In fact, some studies suggest that MS patients, in general, have a lower risk of melanoma than others. The team said more information on patients could give researchers a better understanding of the potential relationship between Tysabri and melanoma. The implication was that the standard of reporting in the FAERS and TOUCH databases could improve. To minimize the risk of patients who receive Tysabri developing melanoma, the researchers offered a number of suggestions for IV centers, physicians, patients, and educational programs. For instance, they suggested that all patients should have a skin examination before the start of treatment, and regular physical and skin exams while receiving Tysabri. While noting that risk of infection and the development of tumors can occur with all immunosuppressive treatments, the team said more studies are needed to explore the risk of Tysabri-treated patients developing melanoma.

AXIM Biotechnologies has secured an additional $4 million in institutional financing to advance clinical trials of its cannabinoid therapies for multiple sclerosis (MS) and irritable bowel syndrome. Some of the trials involve a chewing-gum therapy delivery system. The financing will let the company continue developing a patented chewing-gum-delivered therapy for pain and spasticity. AXIM…

In case you missed them, here are some news stories that appeared in MS News Today that caught my eye over the past week. **** Air Pollution May Trigger Relapses in MS Patients, French Study Finds Attention if you live in location where the air…

A stressful microenvironment, characterized by low metabolites and low oxygen levels, triggers the generation of immune cells directly implicated in a variety of inflammatory diseases, such as multiple sclerosis (MS). The study, “Cellular Stress in the Context of an Inflammatory Environment Supports TGF-β-Independent T Helper-17 Differentiation,” was…

The recent annual meeting of the Consortium of Multiple Sclerosis Centers (CMSC) brought researchers and healthcare professionals to New Orleans to discuss advances — and obstacles to advances — in multiple sclerosis (MS) research. Clinical trials, preclinical studies, basic research, and health interventions were among the May meeting’s focus. Multiple Sclerosis News…

Many people with MS experience symptoms related to digestion. According to the Pittsburgh Institute for MS Care and Research, “Nearly two-thirds of MS patients have at least one GI symptom that persists for 6 months or more.” Some of the most common problems are dysphagia (trouble swallowing), heartburn, nausea,…

Recently approved, Ocrevus (ocrelizumab) should now be available nationwide for patients prescribed the therapy. But as with any new treatment, concerns about safety and practical issues are on many patients’ minds. Multiple Sclerosis News Today asked Dr. Jack Burks — a neurologist and researcher who serves as chief…

Smoking can kill off the immune cells that commonly protect people from multiple sclerosis (MS) and other autoimmune diseases, say researchers at the University of Copenhagen — a finding that may lead to new ways of treating such illnesses. Their study, “Smoking reduces circulating CD26hiCD161hi MAIT cells in healthy…

Pamela Arterbridge noticed something was wrong back in 2013 when she woke up one morning, and her legs and feet were tingling. Two years later, Lorraine Lee knew she had a problem when her right leg became extremely fatigued after every workout. Neither realized their eventual diagnosis would be multiple…

For those who read my column, I am certain you can see I am an emotional writer. This is a difficult column to write. I have been thinking about end-of-life decisions for months. I am reminded of my mortality each time I visit the hospital and I’m asked…

New data on the recently approved multiple sclerosis (MS) drug Ocrevus (ocrelizumab) will be presented at the upcoming American Academy of Neurology (AAN) Annual Meeting 2017, which will take place April 22-28 in Boston. The meeting is the first scientific conference focusing on neurology since the U.S.

It was my 2015 annual check-in, and my neurologist and I were in the exam room. I was sitting on one of those little stools with wheels, he was leaning across the exam table. We had just finished discussing my steadily worsening symptoms and treatment plan, which consisted…

While many multiple sclerosis patients celebrated the recent approval of Ocrevus (ocrelizumab), others argued that the drug is largely a rebranded version of rituximab. Rituximab — sold as Rituxan for indications like non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis — is used off-label to treat relapsing MS. In online forums and social media,…

There are a few moments when life changes. In my day, it was meeting your partner across a crowded room. Now it’s sadly the flick of an app. What hasn’t changed is that only later do you realize this really was a moment. This isn’t a story about romance…

(Editor’s note: Tamara Sellman continues her occasional series on the MS alphabet with the first in a three-part series of terms starting with the letter “C.”) When it comes to multiple sclerosis, mastering an understanding of the disease means you need to mind your Ps and Qs, dot your…

Nektar Therapeutics has started a Phase 1 clinical trial of its biologic therapy NKTR-358 for inflammatory disorders and autoimmune diseases like multiple sclerosis. NKTR-358 is a first-in-class regulatory T-cell stimulator designed to correct the immune system dysfunction associated with these disorders. It targets regulatory T-cells, or Tregs. Other immunosuppressant therapies suppress the…

Dr. Stephen Hauser, chair of the neurology department at the University of California San Francisco, was instrumental in the early research and later clinical trials that ultimately led to Ocrevus (ocrelizumab), the first therapy approved by the U.S. Food and Drug Administration (FDA) for both relapsing MS (RMS) and primary progressive multiple sclerosis…

Here is a transcript of the Multiple Sclerosis News Today interview about Ocrevus (ocrelizumab) with Dr. Robert Lisak of the Consortium of Multiple Sclerosis Centers (CMSC). Lisak, a professor of neurology and of immunology and microbiology at Wayne State University School of Medicine, is a former…

Interest in  Ocrevus (ocrelizumab), the first FDA-approved treatment for both relapsing and primary progressive multiple sclerosis, is running high among patients and the organization representing them — as, arguably, are expectations of its use. But how do physicians involved in MS care view the newcomer? Dr. Robert Lisak (Photo courtesy…

History has been made because of a major breakthrough for multiple sclerosis sufferers everywhere. Ocrevus (ocrelizumab), by Genentech, has been finally approved by the U.S. Food and Drug Administration. It is the first drug treatment that includes my type of MS, primary progressive. This is what I’ve…

With the recent approval of Ocrevus (ocrelizumab) for both primary progressive and relapsing multiple sclerosis (MS), interest in the medication is peaking. To help readers of Multiple Sclerosis News Today better understand this new medication and how it works, as well issues dealing with access, use, and potential side effects, here is a summary…

The historic approval of Ocrevus (ocrelizumab), the first-ever treatment for primary progressive multiple sclerosis (MS), set off ripples in the relatively calm waters of MS news reporting. The drug, which was also approved Tuesday as an unusually effective and safe treatment for relapsing MS, is viewed as a game-changer…

At long last, and for the first time in medical history, people with both relapsing and primary progressive forms of multiple sclerosis have reason to celebrate. The U.S. Food and Drug Administration (FDA) today approved Ocrevus (ocrelizumab)  as a disease-modifying therapy for both forms of MS, a chronic autoimmune disease.

CGEN-15001, which could become the first tolerance-inducing therapy for multiple sclerosis (MS) and other autoimmune conditions, is on the agenda of a scientific conference in Canada that is going on now and another conference in May. The first CGEN-15001 presentation that Compugen is delivering is at the Keystone Symposia: Immune Regulation in Autoimmunity and…