research

Alkermes and Biogen have begun working together on a compound known as ALKS 8700 as a potential treatment for relapsing forms of multiple sclerosis. Under the agreement, Alkermes will be responsible for obtaining regulatory approval of the drug, while Biogen will handle its marketing. ALKS is taken orally. The body quickly transforms it into a compound known as monomethyl fumarate that can counter MS. Aikermes designed it to have better features than Tecfidera (dimethyl fumarate) — in particular, fewer gastrointestinal side effects. The partnership gives Biogen worldwide marketing rights to ALKS 8700. Alkermes will receive a royalty on global sales. Aikermes is evaluating ALKS 8700's safety and effectiveness in what it has dubbed the EVOLVE-MS clinical trial program. It includes two Phase 3 trials that are comparing ALKS 8700 with Tecfidera in patients with relapsing-remitting MS, or RRMS. Preliminary results of the EVOLVE-MS-1 trial, which involved 580 patients, showed few gastrointestinal side effects from ALKS 8700. The most common adverse events in the first month of treatment were flushing, diarrhea, and a rash known as pruritus. Aikermes discussed the treatments safety, and patients' ability to tolerate it, at the 7th Joint ECTRIMS-ACTRIMS Meeting in Paris in October. The company is still recruiting participants for a second trial that will compare ALKS 8700 and Tecfidera's effect on the gastrointestinal system. The EVOLVE-MS-2 study will  be conducted at locations in several U.S. states and six sites in Poland. Alkermes expects to release initial findings from the trial in the first half of 2018.  

Immune cells that destroy myelin in multiple sclerosis (MS) access the brain and spinal cord via two different routes, a new mouse study shows. This suggests that therapies which target these entry routes may shield the brains of MS patients from further damage. The study, “Caveolin1 Is Required for…

A diet rich in vegetables and low in protein reduced inflammation in multiple sclerosis (MS) patients by modulating the gut microbiome and promoting bacteria that helps control a hyper-reactive immune system. The study reporting the findings, “Immunological and Clinical Effect of Diet Modulation of the Gut…

So-called silent brain lesions in patients with early-stage relapsing-remitting multiple sclerosis (RRMS) may, in fact, not be silent at all, according to a French study that linked such lesions to cognitive decline in early MS. This link has likely been missed since the major tool for measuring disability in MS…

Smoking changes a DNA-based mechanism that influences multiple sclerosis patients’ gene activity, a Swedish study confirms. Another finding was that MS appears to aggravate the harmful effects of smoking. The study dealt with DNA methylation, the process by which the body adds methyl groups to a DNA molecule. Methylation can change…

Exposure to certain gut bacteria at a young age may cause multiple sclerosis (MS) and fuel its progression, a new mouse study shows. The study, “Gut dysbiosis breaks immunological tolerance toward the central nervous system during young adulthood,” appeared in the journal Proceedings of the National…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

MMJ Hires Lead Investigator for Phase 2 Trials of Medicinal Cannabis to Treat Progressive MS The real news here is what hiring a lead investigator means. It means that Phase 2 trials of a medical marijuana product to treat MS pain and spasticity are closer to beginning.

An estimated 947,000 people in the U.S. have multiple sclerosis (MS) — more than double the long-accepted figure of 400,000 — according to a newly completed study organized and funded by the National Multiple Sclerosis Society (NMSS). “This is definitely not what we expected,”  Nicholas G. LaRocca, vice…

Available long-term data on Fampyra (fampridine; 4-aminopyridine) suggest the treatment may improve walking speed in patients with multiple sclerosis (MS) for up to one year, but more research is needed, a French study reports. The study “Multiple Sclerosis and Clinical Gait Analysis before and after Fampridine: A Systematic Review”…

U.S. nurses and physicians’ assistants prescribe antibody-based disease-modifying therapies to their multiple sclerosis patients more than neurologists do, a survey indicates. The trend has been for the doctors to stick with interferon therapies, the study said. Antibody-based disease-modifying therapies are also known as monoclonal antibodies. They are designed to harness the…

A pregnancy hormone called estriol may be effective in controlling relapses in women with multiple sclerosis (MS), says a researcher at the University of California, Los Angeles (UCLA). In clinical trials, estriol has also lowered fatigue and improved thinking — work that originated in Rhonda Voskuhl’s quest to understand…

A physiotherapist-supported exercise program using Nintendo Wii may be a feasible and cost-effective way of helping  people with multiple sclerosis (MS) be more physically active, researchers reported after performing a small pilot study. While findings showed some evidence that people improved — both in terms of self-reported health, gait and balance measurements — researchers underscored that more data needs to be gathered on the intervention’s effectiveness, as the study mainly intended to determine if such a program was feasible. Researchers at the Bournemouth University and Poole Hospital NHS Foundation Trust, both in the U.K., argued that a physical activity intervention using active gaming at home may overcome the many challenges MS patients face when attempting to be active. Barriers to it could be physical, but psychological factors, such as fear, embarrassment, or lack of confidence, can also prevent patients from attempting to increase their activity levels. Moreover, practical aspects — such as transport and cost — can hinder people from joining interventions. In the report, “Mii-vitaliSe: a pilot randomised controlled trial of a home gaming system (Nintendo Wii) to increase activity levels, vitality and well-being in people with multiple sclerosis,” researchers explained they used data generated in earlier Nintendo Wii studies to design an improved intervention program. Earlier studies showed that behavior change techniques, including motivational interviews and problem solving, would likely improve the impact of an intervention. These early studies also highlighted the importance of considering the functional levels, environment, and preferences of individual patients when prescribing a Wii-based program. The study (ISRCTN49286846), described in the journal BMJ Open, shows that among the 30 people who signed up, only two dropped out because of medical reasons. Patients either received the 12-month intervention, called Mii-vitaliSe, directly or after a six-month waiting period. Those on the waiting list group were given six months of intervention. Patients, who had low levels of physical activity when the study began, were instructed as to the benefits of physical activity and on how to use the Wii. During the personalized intervention, participants had access to regular support from a physiotherapist and were provided with a personal activity workbook, which aided participants in setting goals and monitoring progress, among other things. They were also asked to keep a log to track their activity, which showed an average use of the Wii two times per week, for 27 minutes each day. Results showed that patients who started the intervention immediately tended to report better physical activity levels, and better physical and psychological well-being. They also had numerical improvements in gait and balance. While no severe adverse reactions were seen, participants reported pain and worsening of scar tissue after some exercises, for which they received follow-up advice and care. The team also identified several problems or difficulties, including wrongly completed questionnaires, that will allow them to improve measurements once they launch a larger study. "Our study is the first to report on home-based use of the Wii for people with MS in the UK. Overall, findings from this study are promising and support proceeding to a full-scale trial of effectiveness and cost-effectiveness. We will refine the trial design, aspects of the intervention and finalize outcome measures in the light of our experiences from this pilot study" the researchers wrote.

MMJ BioScience, an affiliate of medical cannabis research company MMJ International Holdings, has hired a principal investigator to lead clinical trials exploring potential therapeutic applications of cannabinoids in progressive multiple sclerosis (MS). Dr. Bianca Weinstock-Guttman, a neurology professor at the State University of New York at Buffalo, is executive director…

Myelin-producing Brain Cells Regenerated Using Stem Cells in Early Study We know that when the myelin coating of our nerve axons is destroyed, MS symptoms result. So a process that halts or reverses that destruction is the goal of a lot of MS research. This is a…

Sanofi Genzyme and Principia Biopharma have entered into a license agreement to advance the clinical development of PRN2246, an oral drug candidate for the treatment of multiple sclerosis and other diseases of the central nervous system. PRN2246 is an inhibitor of the Bruton’s tyrosine kinase, an enzyme encoded by the BTK gene that plays a crucial role in B-cell development and the B-cell signaling pathway. B-cells are known to be involved in the development of autoimmune diseases that affect the nervous system, including multiple sclerosis. PRN2246 is an orally available therapy designed to easily access the central nervous system (brain and spinal cord) by crossing the blood-brain barrier, and impact the signaling of immune cells and brain cells involved in autoimmunity and inflammatory processes. The drug is designed to safely and effectively modulate B-cell function without depleting these cells. A Phase 1 clinical trial is now testing the drug's safety in healthy volunteers. Under the agreement, which is expected to close shortly, Principia will grant Sanofi an exclusive, worldwide license to develop and commercialize PRN2246. Principia, in return, will receive $40 million in upfront payments from Sanofi, and future milestone payments could reach $765 million. Principia will retain the option to co-fund the treatment's Phase 3 development in exchange for other royalties in the United States. Principia has developed a novel way to design and develop better and safer therapies based on oral small molecules. The company uses its proprietary Tailored Covalency technology to develop its drug candidates, which are, according to the company's website, safer, and more selective, potent and durable than other available treatments. The terms of this licensing agreement are still subject to customary regulatory approval.

Editor’s note: Tamara Sellman continues her occasional series on the MS alphabet with this first of two columns about terms starting with the letter M. Symptoms of MS Memory problems Memory loss is a common complaint for people with MS. Even for those with no physical or…

Longevity Biotech has received a $316,384 grant from the National MS Society to see if LBT-3627, the nerve cell-protecting therapy it has tested in Parkinson’s, can work in multiple sclerosis as well. The company designed the therapy to protect and repair damaged nerve cells and restore balance to the out-of-whack immune response associated…

The CXCR7 receptor present on mature monocytes — a type of white blood cell — may be a therapeutic target to alleviate the inflammation seen in multiple sclerosis (MS) and similar disorders, a new study shows. The study, “Frontline Science: CXCR7 mediates CD14+CD16+ monocyte transmigration across the blood…

Have you ever thought about stopping whatever MS treatment you’re using? I have. So has John Corboy. Corboy’s not an MS patient. Rather, he’s a researcher at the University of Colorado’s medical school. And he’s studying whether older patients, if they haven’t had a relapse for several…

A blood-clotting protein called fibrinogen prevents myelin production and blocks the neuron remyelination repair process in mice, a study finds. The study, “Fibrinogen Activates BMP Signaling in Oligodendrocyte Progenitor Cells and Inhibits Remyelination after Vascular Damage,” appeared in the journal Neuron. Its conclusions offer new insights and…

In the future, a blood test may make diagnosing multiple sclerosis (MS) much easier, thanks to newly identified biomarker patterns that distinguish between MS patients and healthy people. The test could also correctly detect primary progressive MS (PPMS) in patients who also had relapsing-remitting disease (RRMS). Australian researchers suggest that…

#MSParis2017 – Trial to See if Disease-modifying Therapies Not Necessary in Older MS Patients This tops my list this week because, at age 69, I certainly fit the definition of an “older” MS patient. The study is hoping to enroll 300 MS patients in the U.S. who…

The National Multiple Sclerosis Society (NMSS) has officially announced its collaboration with Corrona on the launch of the Corrona Multiple Sclerosis Registry to compare the safety and effectiveness of approved therapies in multiple sclerosis (MS). Corrona, based in Cambridge, Massachusetts, conducts observational cohort studies, offering analytic expertise…

The generation of a thin myelin sheath during remyelination — one that continues to protect nerve cells over time — is indicative of the long-term health and activity of the central nervous system (CNS) in demyelinating diseases such as multiple sclerosis (MS), a new study shows. These findings, which aim…

Research teams from Canada, Portugal and the United States, each with projects focused on predicting and defining characteristics of multiple sclerosis (MS) , will share this year’s 1 million euro ($1,165,700) Grant for Multiple Sclerosis Innovation (GMSI), announced by Merck at the 7th Joint ECTRIMS-ACTRIMS meeting in Paris, France,…

RedHill Biopharma has received a Notice of Allowance for a new patent on RHB-104 its potential therapy for patients with relapsing-remitting multiple sclerosis (RRMS). Once granted by the United States Patent and Trademark Office (USPTO), this patent will be valid until 2032. RHB-104 is a proprietary, orally-administered antibiotic combination with potentially potent intracellular, antimycobacterial…

Nearly half of multiple sclerosis patients do not always report their relapses to healthcare providers, two surveys indicate. Mallinckrodt sponsored the surveys to better understand patients’ experience with relapses, which are sudden episodes of new symptoms or worsening of existing symptoms. The company presented the survey results at the 7th joint meeting…

Researchers, using two different kinds of stem cells in rats, were able to regenerate oligodendrocytes — myelin-producing brain cells that are defective in multiple sclerosis (MS). They were also able to grow adult neural stem cells in laboratory cultures and prod them to develop into oligodendrocytes. The exact cause of MS is unknown — including what triggers attacks on myelin — but the loss of oligodendrocytes seen in the disease is known to play a role in its progression. Nerve cells in the brain send their signals through their axons, long arm-like structures that extend out from the centers of the nerve cells. The signals are electrical pulses transmitted along the length of an axon. Oligodendrocytes provide the insulation — called myelin — that wraps around axons, speeding up the transmission of electrical signals through the nerve cells. Loss or malfunction of oligodendrocytes means that signaling in the brain is impaired. It is this slowing of signaling that is thought to cause MS symptoms. Researchers from the Heinrich-Heine-University, Germany, with support from British and Chilean colleagues, designed a novel approach to regenerate oligodendrocytes, according to a press release. Stem cells are immature cells that give rise to differentiated cells — cells with a specific function, such as oligodendrocytes. Adult neural stem cells can divide and produce nerve cells and other brain cells, including oligodendrocytes. However, in normal circumstances, the regeneration of cells that take place in the human brain is not enough to repair the damage seen in MS. The researchers set out to find conditions that would promote the differentiation of adult human NSCs into oligodendrocytes. They discovered that another type of stem cell, mesenchymal stem cells (MSCs), could provide the signals required. First they tested their system in rats, and found that by using factors produced by human MSCs, they could induce the growth of new oligodendrocytes in the animals. Then they grew adult NSCs in the laboratory, and using the same factors from human MSCs were able to promote the establishment of oligodendrocytes in the cultured cells.

Preliminary data from the Phase 3 EVOLVE-MS-1 trial shows that ALKS 8700 — an investigative therapy developed by Alkermes to treat relapsing forms of multiple sclerosis — has a good safety and tolerability profile. ALKS 8700 is an oral compound. Once inside the body, it is rapidly transformed into the therapeutic compound monomethyl fumarate (MMF). Although similar, this drug candidate was designed to offer features different than those achieved with the commercially available Tecfidera (dimethyl fumarate). Alkermes is currently assessing the safety and efficacy of ALKS 8700 in the EVOLVE-MS program, which includes two Phase 3 clinical trials in patients with relapsing-remitting MS. The EVOLVE-MS-1 is a two-year study being conducted in 107 U.S. and European research sites. It will evaluate the long-term safety of ALKS 8700 in some 930 RRMS patients. Interim data collected during the first month of treating 580 participants showed low incidence of GI adverse events, with no reports of serious events. The most common adverse side effects associated with the treatment were flushing, pruritus and diarrhea. Alkermes, which is based in Ireland, said additional results from the initial three months of treatment further supported the positive safety data of ALKS 8700, with only 2.3 percent of patients reporting serious adverse events and 3.7 percent having to stop treatment. The EVOLVE-MS-2 trial, being conducted at 48 U.S. sites, will compare the safety and efficacy of ALKS 8700 versus Tecfidera in RRMS patients. The study is still recruiting participants. Recent data of EVOLVE-MS-2 was also subject of a poster presentation at the ECTRIMS-ACTRIMS Meeting.