Trial results

Early and continuous treatment with Ocrevus (ocrelizumab) leads to a greater and more durable slowing of disability progression — seen for up to 6.5 years — in people with primary progressive multiple sclerosis (PPMS), according to long-term data on its use in PPMS patients in a Phase 3…

Mayzent (siponimod) reduces the risk of people with secondary progressive multiple sclerosis (SPMS) becoming dependent on a wheelchair, a new analysis of the EXPAND study shows. These findings further corroborate prior trial data demonstrating that Mayzent use delays disability progression and cognitive decline in SPMS patients. The results…

Using newer disease-modifying therapies (DMTs) as an initial treatment for children and adolescents with multiple sclerosis (MS) or clinically isolated syndrome (CIS) is associated with fewer relapses and brain lesions compared to the use of older and injectable DMTs, according to a real-world study in the U.S.

Oxcarbazepine, an anti-epileptic medicine, given in combination with a disease-modifying therapy (DMT) may help to stop disability progression in multiple sclerosis (MS) patients, results of Phase 2 trial suggest. Monica Marta, PhD, with Queen Mary University of London and Barts Health NHS Trust/The Royal London Hospital presented the data at the…

Transplanting mesenchymal stem cells (MSCs) is safe and can delay disease progression in people with active, progressive multiple sclerosis (MS), according to results from a single-center clinical trial conducted in Israel. Six months after the transplant, a considerable proportion of patients showed no signs of disease activity, compared to…

Use of plasma exchange (PLEX) is not effective for treating progressive multifocal leukoencephalopathy (PML), a dangerous brain infection that has been associated with using the multiple sclerosis (MS) medicine Tysabri (natalizumab), a real-world study contends. The findings highlight the importance of  closely monitoring Tysabri users to detect…

Treatment with a potential remyelinating agent called liothyronine was safe and well-tolerated by people with multiple sclerosis (MS) in a Phase 1b trial. Preliminary results also suggested benefits in cognition, motor function, and fatigue. The study, “A Phase 1b, open-label study to evaluate the safety…

New 10-year data from the Phase 3 ENDORSE trial confirms the long-term benefits of Biogen’s Tecfidera for patients with relapsing-remitting multiple sclerosis (RRMS), the most common form of this disease. Real-world data from another study also showed Tecfidera to be superior to several other disease-modifying therapies for relapsing MS,…

Oral ozanimod is more effective at reducing the frequency of relapses than Avonex (interferon beta-1a) in patients with relapsing forms of multiple sclerosis (MS), and does so with reasonable safety, results of Phase 3 trial show. These findings were described in the study, “Safety and efficacy of ozanimod…

Using smartphones and smartwatches to monitor disease course via the FLOODLIGHT app leads to high adherence and satisfaction among patients with multiple sclerosis (MS), results from a pilot study show. The research, “Adherence and Satisfaction of Smartphone- and Smartwatch-Based Remote Active Testing and Passive Monitoring in People With Multiple Sclerosis: Nonrandomized Interventional Feasibility Study,” was published in the Journal of Medical Internet Research. Assessing MS progression commonly requires periodic in-clinic visits, and doctors may miss subtle changes occurring between such visits. Smartphones may be a solution for real-time data collection and better patient monitoring outside the clinic. The Roche-sponsored FLOODLIGHT study assessed the feasibility of using smartphones and smartwatches to assess MS symptoms, including hand function, gait and posture, mood, and cognitive impairment. FLOODLIGHT primarily evaluated adherence and feedback to the smartphone- and smartwatch-based assessments, as well as the participants’ satisfaction, as determined by their impact on daily activities. The study included 76 MS patients (ages 20 to 57; 53 were women) and 25 healthy controls, who were recruited at the Multiple Sclerosis Centre of Catalonia in Barcelona, and the University of California, San Francisco. Most patients (91%) had relapsing-remitting MS. The mean time since symptom onset was 11.3 years, and the mean Expanded Disability Status Scale score was 2.4. The test battery included active tests performed daily, weekly, every two weeks, or on demand for 24 weeks, and passive monitoring, which analyzed sensor-based gait and mobility. Participants were instructed to complete the active tests at roughly the same time every day, and to carry the smartphone and smartwatch with them all the time. Adherence was assessed via the proportion of weeks with at least three days of completed testing, and four daily hours of passive monitoring. Satisfaction was determined through a questionnaire. Clinical evaluations were conducted at the study's start, after 12 weeks, and at the study's end. Brain magnetic resonance imaging scans were taken at the enrollment visit and at week 24 (study's end). Most participants finished the study – 92% of MS patients and 64% of the controls. Reasons for discontinuation included the burden of continuous monitoring and lack of availability for the three required in-clinic visits. Over an 18-month period, from November 2016 to April 2018, most participants performed five to seven active tests per week, including the Two-Minute Walk Test (2MWT). Adherence to both active tests and passive monitoring for the 24 weeks was good, and remained stable over time after week six. In the final week, participants had a minimum of four hours of passive monitoring data over an average of four days. Adherence was higher for passive monitoring than the tests. Data showed that 70% of participants completed all active tests, 75% did all active tests except the 2MWT, and 79% had data collected via smartphone- or smartwatch-based passive monitoring. The longer a person's disease duration, the lower was their adherence. In contrast, disease severity was not seen to impact adherence. Overall scores reflecting satisfaction was 73.7 out of 100 at week 24, having remained stable since week 12. Sixty-one patients reported that the test battery had an at least an acceptable impact on daily activities. Thirty-two had no issue with the active tests, while one-third would rather avoid the 2MWT. A majority, 60%, of the MS patients said they would like to continue using the FLOODLIGHT app to better understand their disease and improve its management. “Persons with multiple sclerosis were engaged and satisfied with the FLOODLIGHT test battery,” the scientists wrote. As such, “FLOODLIGHT sensor-based measures may enable continuous assessment of multiple sclerosis disease in clinical trials and real-world settings.” FLOODLIGHT tests are being assessed in long-term studies with larger patient groups in the CONSONANCE Phase 3b trial of Ocrevus (ocrelizumab, by Roche-owned Genentech) in people with progressive MS still enrolling, contact information is here), and in a global study open to all MS patients with a mobile device, called FLOODLIGHT Open.

The first in-human clinical trial of EHP-101, an investigational treatment for multiple sclerosis (MS) and systemic scleroderma (SSc), has been successfully completed, Emerald Health Pharmaceuticals (EHP) announced. EHP-101 demonstrated it was safe and well-tolerated in healthy volunteers, supporting further clinical development of EHP-101 in future Phase 2 clinical trials. The…

I knew nothing about multiple sclerosis (MS) before 2012, including the cause, the cure, or any other details. I had so many unanswered questions about how I would live with MS when I was diagnosed. I had never heard of treatment for MS and was unsure of the…

Though known mainly for killing tumor and virally infected cells, a T-cell subtype may restrain immune responses and be protective in autoimmune diseases such as multiple sclerosis (MS), according to new research. The study, “Opposing T cell responses in experimental autoimmune encephalomyelitis,” was published recently in…

A Phase 1 clinical trial found T20K, Cyxone’s investigational plant protein-derived treatment for multiple sclerosis (MS), to be safe and well-tolerated in healthy men, and raising the possibility that it might work at low doses, the company announced. A next step is to develop an oral formulation for the…

3D Imaging of Brain Lesions May Spot Those Most Likely to Heal, Guiding Treatment These scientists are working to create a new diagnostic tool that would allow doctors to use an MRI to look at brain lesions in 3D. This would allow them to see the shape and surface…

Fewer and less severe gastrointestinal (GI) side effects were evident in relapsing-remitting multiple sclerosis (RRMS) patients taking the investigational oral treatment Vumerity (diroximel fumarate) twice a day compared to those using Tecfidera (dimethyl fumarate), topline data from the EVOLVE-MS-2 trial show. Vumerity is a fumarate compound being developed…

Janssen has announced positive, top-line results from its Phase 3 OPTIMUM study, testing the effectiveness and safety of ponesimod tablets, compared to Sanofi‘s Aubagio (teriflunomide), in adults with relapsing forms of multiple sclerosis (MS). The study met its primary goal — a reduction in the…

An antiviral medication called amantadine and ondansetron, which treats chronic nausea, both work to reduce fatigue in people with multiple sclerosis (MS), but amantadine may be more effective, a single-site trial in Iran reports. The study, “Comparison of the effects of amantadine and ondansetron in treatment…

Treating people with relapsing forms of multiple sclerosis (MS) with opicinumab and Avonex (interferon beta-1a) for 72 weeks did not lead to a dose-dependent reduction in disability,  results of a Phase 2 trial show. However, an ongoing study is evaluating opicinumab in a subgroup with better clinical responses.

A Phase 3 trial is planned to confirm the safety and efficacy of oral ibudilast (MN-166) in treating people with secondary progressive multiple sclerosis (SPMS) without relapses, or those whose disease is not active, MediciNova announced. Data from this single Phase 3 study may be used to request…

Targeting a protein found in immune T-cells called Oct1 may help prevent the misguided immune response seen in autoimmune diseases like multiple sclerosis (MS), a study shows. Most importantly, a lack of Oct1 does not influence the immune system’s ability to fight viral infections. The study “T cell-selective…

Cyxone launched the first-in-human Phase 1 clinical trial assessing the effects of T20K, its new therapeutic candidate for the treatment of multiple sclerosis (MS), in healthy volunteers. The announcement came after the company received approval from the Dutch Ethics Committee and Central Commission on Research Involving Human…

Aubagio (teriflunomide), an approved medicine for relapsing forms of multiple sclerosis (MS), specifically targets highly metabolic and more autoreactive T-cells, analysis of the Phase 3 TERI-DYNAMIC clinical trial data shows. The findings, contrary to expectations, support a selective effect of Aubagio on different T-cell populations. The study “Teriflunomide treatment for multiple sclerosis modulates T cell mitochondrial respiration with affinity-dependent effects” was published in the Science Translational Medicine journal. In MS, immune cells, or lymphocytes known as T-cells, attack and destroy myelin, the fat-rich substance that wraps around nerve fibers (axons). Myelin loss creates lesions that affect nerves of the brain and spinal cord. Previous evidence suggested that T-cells, depending on their active or resting state, rely on specific ways of energy production or metabolism. Aubagio, marketed by Sanofi Genzyme, is a well-known inhibitor of a mitochondrial enzyme called dihydroorotate dehydrogenase (DHODH), that is crucial for the activity of T-cells. However, how Aubagio selectively targets the autoreactive T-cells is poorly understood. To shed light on this matter, an international group of researchers used data from the TERI-DYNAMIC clinical trial that tested Aubagio in patients with relapsing form of MS to better understand how the therapy inhibited the patients' self-immune responses. The Phase 3, open-label TERI-DYNAMIC trial (NCT01863888) included 70 patients from Belgium, Germany, and The Netherlands, aged 18 to 56. Participants received Aubagio as a 14 milligram (mg) once-daily, oral dose, and researchers assessed the changes in immune cells' profile up to 24 weeks. Results showed that, contrary to what was expected, Aubagio was not generally decreasing T-cell levels in treated patients. Instead, it significantly reduced a particular subset of T-cells, called "Th1 helper cells." Moreover, researchers found that the diversity of T-cell receptors — the surface proteins that can recognize a particular antigen (a protein that can elicit an immune response) — making T-cells specific to a certain target was reduced in MS patients after treatment with Aubagio. These findings suggested that some T-cells were particularly susceptible to Aubagio. Using a mouse model for MS, the experimental autoimmune encephalomyelitis (EAE) model, researchers showed that the CD4+ T-cells (helper T-cells) and CD8+ T-cells, those that reacted most strongly against self-antigens, were the most sensitive to DHODH inhibition by Aubagio. Moreover, researchers saw that Aubagio was not affecting the production of pro-inflammatory molecules — called cytokines — at the cell level, but their overall decrease probably was due to the reduction in T-cell numbers. In line with these findings, CD4+ T-cells that produced the cytokine interferon gamma were significantly reduced with Aubagio treatment, whereas CD4+ T-cells that produced interleukin 17A were unchanged. This suggests that Aubagio is able to interfere with specific sub-types of immune cells. When the team compared the metabolic profile of T-cells from healthy subjects with that from patients with relapsing-remitting MS (RRMS) in both remission and in relapse phases, they found that the metabolism of T-cells from the last group was significantly altered, and thus targetable. Altogether, the results suggested that T-cells with a high-affinity to self-antigens are more susceptible to inhibition of the DHODH enzyme by Aubagio. “Therapeutic targeting of metabolic alterations might represent an attractive concept in MS, and might represent an as yet unrecognized key mechanism of teriflunomide-mediated immune modulation in this disease,” the researchers concluded.

Exosomes — tiny vesicles secreted by cells — collected from bone marrow stem cells and injected into a mouse model of multiple sclerosis (MS) helped to treat the disease, a study reports. Specifically, this treatment eased myelin loss and neuroinflammation in the mice, and improved motor function, the…

Vumerity (diroximel fumarate), taken as a 462 milligram (mg) tablet twice daily, significantly decreases disease activity in patients with relapsing-remitting multiple sclerosis (RRMS), and leads to low rates of gastrointestinal side effects, new interim data of Phase 3 trial EVOLVE-MS-1 show. The findings were presented at the 2019 Consortium…

Vesicles in the blood that contain a protein called fibrinogen important for blood clotting were found to be “sufficient and required” for the occurrence of spontaneous disease relapses in mouse models of multiple sclerosis (MS), a study reports. In mice, these vesicles were associated with immune cell infiltration, inflammation,…

Oral Gilenya (fingolimod) taken daily at a 0.5 mg dose is superior to Copaxone (glatiramer acetate) injections at lowering relapses and disease activity over one year in patients with relapsing-remitting multiple sclerosis (RRMS), according to results of a Phase 3b trial. The research, “Efficacy and Safety…

Adamas Pharmaceuticals announced the study design and baseline characteristics of patients enrolling in the company’s ongoing Phase 3 trial testing the efficacy and safety of ADS-5102 (amantadine) extended-release capsules in patients with multiple sclerosis (MS) who have difficulty walking. The data were presented in a poster,…

Treatment with Mayzent (siponimod) provided sustained improvements and prevented deterioration of cognitive processing speed in patients with secondary progressive multiple sclerosis (SPMS), regardless of their cognitive function prior to therapy, according to results of a Phase 3 clinical trial. The data were presented at the recent 2019 American…

Treatment with Tysabri (natalizumab) lowers the levels of the biomarker serum neurofilament light chain (sNfL) in patients with secondary progressive multiple sclerosis (SPMS), according to data from a Phase 3 trial. Findings also revealed that higher levels of sNfL correlated with MS lesions and disease activity prior to starting the…