Treatment

Infusible disease-modifying treatment — that is, therapies given intravenously — might have greater benefits for younger people with multiple sclerosis (MS) than oral ones, new research suggests. The research was presented at the ongoing American Academy of Neurology (AAN)’s annual meeting (May 4-10) by Brandi Vollmer,…

Robert Scola Jr. is a federal judge in Miami, and he’s just recused himself from a case involving the UnitedHealthcare (UHC) insurance company. The case is one of two filed in Miami over the last two months claiming that UHC has improperly denied coverage for a specific cancer…

A new clinical application prototype that uses machine learning to help physicians predict the best treatment options for patients with multiple sclerosis (MS) will be unveiled at the American Academy of Neurology’s 2019 annual meeting May 4–10, in Philadelphia, Pennsylvania. The prototype is called PIN Population Data Platform. It has been…

Giving estrogen to two different adult mouse models of multiple sclerosis (MS), including the experimental autoimmune encephalomyelitis (EAE) model, promoted remyelination, a new study shows. Exposure to the hormone affected gene activity in oligodendrocytes, tricking them into producing myelin (the fatty substance that protects nerve cells, and that is destroyed…

Study Will Explore Benefits of Tai Chi, Meditation on MS Patients’ Balance I’ve tried yoga to help with my balance and flexibility and found it useful. But tai chi scares me a little — it seems too difficult. Maybe, if the results of this study are very positive, I’ll…

High levels of satisfaction with the efficacy and convenience of Aubagio (teriflunomide), an oral treatment for relapsing multiple sclerosis (MS), were reported by patients across the U.S. and 13 other countries, a post-hoc analysis of data from a real-world Phase 4 study found. The study “Teriflunomide real-world evidence: Global…

In clinical practice, relapse events dropped by roughly half over a four-year period in relapsing-remitting multiple sclerosis (RRMS) patients treated with Aubagio (teriflunomide), a real-world study reports. The study, “Real-life outcomes of teriflunomide treatment in patients with relapsing multiple sclerosis: TAURUS-MS observational study,” also examined patients’ perspectives in…

Researchers have developed a compound based on the thyroid hormone T3 that is able to repair damaged myelin in the brain of mice, a discovery that holds promise for healing myelin loss in patients with multiple sclerosis (MS), results of an early study reveal.

The U.S. Drug Enforcement Administration (DEA) has determined that the cannabidiol-based active ingredient of EHP-101, an investigational oral treatment for multiple sclerosis (MS) under development by Emerald Health Pharmaceuticals (EHP), should not be classified…

Mavenclad (cladribine) tablets stand out as a treatment for relapsing forms of multiple sclerosis (MS), providing two years of proven efficacy with a maximum of 20 days of oral treatment, a top executive with EMD Serono says. After being approved in more than 50 countries worldwide, including the European Union, Australia,…

I spend a great deal of time in my head. I think. A lot. Perhaps I do so more than I should, but then again, it is a haven at times. My thoughts run the gamut from the serious to the inane. Today my thoughts…

The European Medicines Agency (EMA) is reviewing safety data for Sanofi Genzyme‘s Lemtrada (alemtuzumab) following new reports of serious treatment side effects. Lemtrada is a humanized monoclonal antibody used to slow disease progression in adult patients with relapsing-remitting multiple sclerosis (RRMS). It works by blocking the activity of…

A collaboration between DNAnexus, the Sutter Health network, and the University Pittsburgh Medical Center (UPMC) aims to improve personalized medicine in multiple sclerosis (MS) through the first large-scale clinical genomic research study, the companies announced. The network has the goal of improving screening, diagnosis, and treatment of…

Progressive multifocal leukoencephalopathy, more commonly referred to as PML, is a brain infection that’s frequently fatal. PML is particularly dangerous to people with MS, which is why I’m interested in a report about a medication that’s showing promise as a PML treatment. The concern for people with MS is…

A clinical trial testing a computer program called Deprexis as a home-based therapeutic strategy for multiple sclerosis (MS)-related depression is enrolling participants with all types of MS  at three sites in the U.S. and two in Germany. The U.S. study locations are the Cedars-Sinai Medical Center,…

Last weekend a mother brought 4,000£ ($5,200 U.S.) of medicinal cannabis from Holland into the U.K. for her daughter with severe epilepsy. It was confiscated by customs. Last year medicinal cannabis was partially legalized in the U.K. when a similar incident happened. Doctors can now issue prescriptions.

A protein that promotes nervous system repair through remyelination — the creation of myelin, the protective sheath around nerve cells — in mice also is found in remyelinating plaques in brains of multiple sclerosis (MS) patients, new research shows. This protein potentially represents a new therapeutic target in demyelinating…

Rituximab-treated multiple sclerosis (MS) patients who take vitamin D supplements have less inflammatory activity, a study reports. Increased levels of vitamin D were associated with beneficial treatment outcomes, such as better self-perceived health and reduced levels of the inflammation marker C-reactive protein…

Tecfidera (dimethyl fumarate) is more efficient at preventing relapses, and has a lower discontinuation rate than Aubagio (teriflunomide), according to a Danish study. The study “Comparative effectiveness of teriflunomide and dimethyl fumarate: A nationwide cohort study” was published in the journal Neurology. Aubagio (marketed by Sanofi Genzyme) and Tecfidera…

Nearly 18% of new multiple sclerosis (MS) patients referred to two speciality clinics in Los Angeles, California, had been misdiagnosed with the disease, according to a recent study. Most of them actually were affected by migraines, among other…

The effectiveness of Mayzent (siponimod) in both the brain and the body make it an oral therapy tailored for people with early secondary progressive multiple sclerosis (SPMS), according to Dan Bar-Zohar, MD, top executive with Novartis, the treatment’s developer. Mayzent was recently approved by the U.S. Food and Drug Administration…

  It’s tough paying for medications. Whether they’re for multiple sclerosis (MS) or another illness, Americans are having a hard time coming up with the cash needed to cover the cost of their meds. Many people are developing workarounds and compromises to deal with the problem. And in some…

Gabapentin and pregabalin now have more restrictive rules for prescriptions in the United Kingdom. Because of concerns regarding the medications’ misuse, both were classified as class C controlled medicines. It is now illegal to supply, sell, or possess these medicines without a prescription, according to a news release by…

When the U.S. Food and Drug Administration approved the disease-modifying therapy Mayzent for relapsing types of multiple sclerosis, it specified in its label that the treatment was for people with clinically isolated syndrome, relapsing-remitting MS, and — importantly — secondary progressive MS provided they have "active" disease. The approval is good news, an MS researcher and physician said to Multiple Sclerosis News Today in an interview, but "surprising" in that the FDA's decision was largely based on a trial that didn't involve CIS patients and wasn't focused on responses among particular types of SPMS. “It's the first time that I've seen in the MS field that regulators made an approval designation — active secondary progressive MS — based on an underpowered subgroup analysis,” said Robert Fox, MD, a neurologist at the Mellen Center for Multiple Sclerosis at the Cleveland Clinic. Novartis' medication, as a first oral therapy approved in the U.S. for a form of SPMS, is a big step forward in MS treatment, he said. But details of the FDA's decision caught him off guard. Fox served on the steering committee for the EXPAND Phase 3 clinical trial , on which the FDA decision was largely based. His clinic was also one of the sites treating and evaluating patients in this pivotal study. Results of the EXPAND trial showed that Mayzent could reduce the risk of disability progression at three months (the trial’s primary endpoint, or goal) by 21% in treated SPMS patients, compared to those given a placebo. Among those with active SPMS (meaning with relapses), a 33% reduction was observed. The treatment, an S1P modulator that works in part to keep lymphocytes from entering the brain to trigger inflammation, also decreased the annualized relapse rate by 55% and improved cognitive processing speed in all treated patients.  “What was found, and I think quite clearly found in a large-size study, was that siponimod in patients with secondary progressive MS clearly slowed the progression of clinical disability over the course of the trial,” Fox said. “It's a statistical concept — obviously patients either progress or they don't progress — but on an overall basis there was a 21% slowing in the rate of progression of clinical disability.” The FDA’s decision is particularly important for SPMS patients. While Ocrevus (ocrelizumab) also treats all relapsing MS forms and people with primary progressive disease (PPMS), it's an intravenous therapy given every six months. Mavenclad (cladribine), approved for relapsing patients in the U.S. just days after Mayzent, is another oral and active disease therapy. To Fox, Mayzent seemed to reach beyond only those secondary progressive patients with clinically active disease. “Really, this is the only drug that's been found to be effective in secondary progressive MS," he said. “To that degree, it stands alone.” That's why two points in the FDA's decision surprised him. The first is the label's specific mention of clinically isolated syndrome. CIS is defined as the first clinical presentation of this disease — a neurological episode that lasts at least 24 hours, and is characterized by inflammatory demyelination (the loss of myelin, the protective coat surrounding neurons).   For clinicians like Fox, CIS is a first manifestation of MS — a kind of "mono sclerosis." Since there’s only one documented attack, it can’t yet be considered multiple sclerosis, “as the multiple hasn't happened,” Fox said, but many "in the field consider CIS to be … an early stage of MS." “If the patient has a whole bunch of lesions on their brain [as seen on an MRI scan] and they had a single clinical event, ah, probably, they have MS,” he said. Regulatory bodies like the FDA, however, have historically considered CIS to be its own separate entity. That makes this decision doubly surprising, according to Fox, since the EXPAND trial only enrolled patients with SPMS, not CIS.   “It's the first time I've seen them approve for CIS specifically when there wasn't a trial in CIS,” Fox said. “I agree with it — I don't have a problem with it — it just surprised me that the regulators were so progressive in their appreciation of MS.” The second — and far more unsettling — surprise was the FDA’s decision to only approve Mayzent for “active” SPMS patients, instead of all SPMS patients. This decision didn’t come out of nowhere, he noted, but it remains puzzling in the context of the EXPAND trial.  In compiling trial results, investigators did a subgroup analysis — as they often do, almost as an aside for research reasons — and found more favorable responses to Mayzent treatment in patients with active inflammation before the trial's start, those it determined to be with "active" disease.   “There was a third of patients who had a relapse in the two years prior to enrollment, and those patients actually had a 30% slowing in disability progression, compared to the 21% overall,” Fox said. This certainly does suggest that Mayzent can be more effective in people with active disease — but there's a catch. The trial itself was not designed to make such a distinction. It enrolled SPMS patients regardless of activity, and its priority goal was changes in disease progression across all who were treated with Mayzent or given a placebo.   “What's important is that the trial was powered for the overall outcome. It was not powered for subgroup analysis,” Fox said, considering this a crucial point.  In clinical studies, being “powered” refers to the enrolling of whatever specific number of participants a study needs to ensure its results will reach statistical significance. More people are redundant and, as such, an unnecessary cost; fewer could mean that trial's conclusions cannot be supported by rigorous scientific measures.  In other words, Fox said, the only conclusions that can be drawn from the EXPAND study reliably — with rigor — are based on data drawn from all its SPMS patients, not a subgroup with active disease. This trial “followed over 1,600 patients for the clinical disability. These are purposely powered so that you're not following twice as many people as you need to … you're powered for that primary outcome,” he said. “So, how could they [the FDA] look at a subgroup analysis and make an approval decision based on a subgroup analysis that was underpowered?” The neurologist gave as examples other subgroup differences found in trial analyses that didn't affect regulatory approval — but to his mind, equally could have. One was an analysis finding female SPMS patients responded to the therapy better than males, showing lesser disease progression. "So why didn't they just approve it for the females and not the males?" Fox asked. But, when asked, Fox did not think the label to necessarily be an error. "My point is the absurdity of it," he said. "How could they make the regulatory approval based on a subgroup analysis that wasn't powered for conclusions?" He was also particularly troubled because the FDA “didn't define what ‘active’ means — is it just a relapse, or is it MRI disease activity?"  For many clinicians, “active” SPMS refers to ongoing inflammation that can be observed on MRI (magnetic resonance imaging) scans. In EXPAND, however, the active subgroup was defined as patients with clinical relapses within two years of being enrolled in the trial. Fox worries about this apparent lack of a regulatory definition of "active" SPMS, since “obviously, the insurance companies are going to seize upon that, and they're going to look for every way they can to avoid covering it for patients.” Mayzent, Fox agreed, is likely to be expensive. The therapy is reported to carry a U.S. list price of $88,500 a year. “I always have a concern about the cost of these drugs. They're all fearfully expensive,” he said, noting he treats SPMS patients. His focus now is on working to ensure that possible regulatory and financial hurdles won’t pose too much of an obstacle for patients, especially those with SPMS. “I don't know what the insurance companies are going to do with this, but I'm hoping that it is available for my patients, and I say that as their clinician,” Fox concluded.

As you might have heard, a disease-modifying therapy (DMT) for patients with active secondary progressive multiple sclerosis (SPMS) was approved a few days ago. That’s great news. A medication targeting SPMS is overdue, but it could be better. The DMT is Mayzent (siponimod), a tablet that’s taken…

NeurologyLive has launched a series of educational videos intended to provide an overview of the diagnositic methods and treatment options available to multiple sclerosis (MS) patients. The video series, “Advances in the Diagnosis and Management of Multiple Sclerosis,” also will include future perspectives on upcoming MS therapies,…

MS Patient Groups React Favorably to Mayzent Approval, But Question Therapy’s Price Tag The approval of this medication is very good news. However, as the headline indicates, it comes with a relatively hefty cost. It’s approved for active secondary progressive multiple sclerosis (SPMS), and many of us have SPMS…

A shortened DNA molecule showed an increased ability to bind myelin in human cells, and may boost the development of remyelination approaches for multiple sclerosis (MS) treatment, according to a study. The study, “Optimization of a 40-mer Antimyelin DNA Aptamer Identifies a 20-mer with Enhanced Properties…

The U.S. Food and Drug Administration (FDA) has approved Mavenclad (cladribine) tablets for the treatment of adults with relapsing forms of multiple sclerosis (MS), including relapsing-remitting MS (RRMS) and active secondary progressive disease (SPMS). Up to 85 percent of people with MS are initially diagnosed…

National organizations that represent patients with multiple sclerosis (MS) welcome the U.S. Food and Drug Administration’s March 26 approval of Novartis’ oral therapy Mayzent (siponimod) — but they complain that, at $88,500 per year, the treatment is overpriced. The Multiple Sclerosis Society of America (MSSA) is clearly upbeat about the…