News

Oral Cannabidiol, PTL101, Meets Goals of Phase 1 Study as Possible Spasticity Treatment

Results of a Phase 1 clinical trial in healthy volunteers show that PTL101, an oral cannabidiol compound, is a safe and effectively delivered potential treatment of spasticity in multiple sclerosis (MS) and for conditions like epilepsy, Harvest One Cannabis announced. These findings were published in the journal Clinical Pharmacology in Drug Development, in the study…

Nurses, Physicians’ Assistants Prescribe Antibody-based Therapies More Than Neurologists, Survey Shows

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…

Cannabinoid, Dronabinol, Seen as Long-term Treatment Option for Neuropathic Pain in Phase 3 Study

Multiple sclerosis (MS) patients being treated with dronabinol, a cannabinoid, do not show signs of drug abuse or dependency, leading researchers to conclude it has potential to be a long-term and safe treatment option for neuropathic pain. The issue of pain management, specifically central neuropathic pain (CNP), in patients with autoimmune disorders…

Physical Exercise Using Nintendo Wii May Improve Balance and Gait in MS Patients, Study Says

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.

Europe’s CHMP Urges Approval of Ocrevus in EU to Treat Relapsing, Primary Progressive MS

Europeans with relapsing multiple sclerosis (MS) and early primary progressive MS are one step closer to accessing Ocrevus, now that the European Medicines Agency has urged the European Union to approve the therapy. The positive opinion — announced in a press release issued Nov. 10 by the EMA’s Committee for Medicinal Products for Human Use — is an intermediary step required in the regulatory pathway to allow patient access to a new drug. The European Commission will now make a final decision on whether Ocrevus should be granted marketing authorization in all 28 EU member states. This decision will take the CHMP recommendation into consideration. If approved, Ocrevus will become the first disease-modifying medicine available throughout Europe for patients with PPMS. Once this happens, any decisions on price or insurance reimbursements will be the responsibility of each member state. Ocrevus won U.S. approval earlier this year. It was also recently approved in Switzerland for both relapsing MS and PPMS. Ocrevus is an anti-CD20 antibody developed by Genentech, a division of Roche. It blocks immune B-cells, preventing them from attacking nerve cells and their myelin protective sheath, as well as inhibiting other pro-inflammatory immune signals involved in MS. CHMP based its positive recommendation on data from three pivotal Phase 3 clinical trials: the OPERA I and II trials in relapsing MS patients, and the ORATORIO trial in PPMS patients. Results from the OPERA clinical studies demonstrated that treatment with Ocrevus for up to 96 weeks could reduce the annualized relapse rate by 46.4 percent compared with EMD Serono’s approved drug Rebif (interferon beta-1a) in relapsing MS patients. The ORATORIO trial showed that Ocrevus could reduce by 24 percent the risk of 12-week confirmed disability progression compared to placebo in PPMS patients. Data from the trial further supported the drug's therapeutic benefit in early-stage PPMS patients. Additional studies are warranted to better evaluate the therapeutic potential of Ocrevus for patients with more advanced stages of the disease. The most common treatment-associated adverse effects reported wee infusion-related reactions and infections.

MMJ Hires Lead Investigator for Phase 2 Trials of Medicinal Cannabis to Treat Progressive MS

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…

Sanofi and Principia Join to Develop Potential B-Cell-targeting Oral MS Treatment

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.

Targeting Blood-clotting Protein Can Restore Brain’s Potential to Repair Myelin Layer, Study Shows

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…

#MSParis2017 – Merck Announces Winners of 1 Million Euro Grant for Innovative Multiple Sclerosis Research

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 Awaits US Patent for Antibiotic Combo, RHB-104, as Potential RRMS Therapy

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…

Myelin-producing Brain Cells Regenerated Using Stem Cells in Early Study

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.

#MSParis2017 – EVOLVE-MS-1 Interim Trial Data Shows Alkermes Therapy Safe for Treating Relapsing MS

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.