News and research
This page documents breaking multiple sclerosis news and research stories as they occur during the month.
For news and research for the previous 12 months please use the links on the menu on the left.
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New grant to fund multiple sclerosis trials, research(22/05/13)
A new Commonwealth grant for researching multiple sclerosis (MS) is expected to advance existing research collaborations and enable new ones.
Multiple Sclerosis Research Australia chief executive Jeremy Wright says the $1 million grant will also help fund infrastructure.
"It's very targeted towards expanding what we call platform projects and those projects provide the infrastructure for much more research to happen," he said.
The chronic disease, which attacks the central nervous system, affects more than 2 million people worldwide, including 23,000 Australians.
Mr Wright cited a new trial looking to expand and recruit enough patients to get a statistical bearing on whether Vitamin D might slow the progress of MS.
MS research often tries to uncover the role of the autoimmune system in the development and advance of the disease.
"We've got collaborative projects looking at the way different proteins influence the immune system so it's not overactive like it is in MS, where the immune system is pedalling too hard and damaging the nerves in the brain," Mr Wright said.
"Universities and clinicians are collaborating at 19 or 20 sites around Australia to recruit enough patients to get a statistical bearing on the outcome of providing Vitamin D and seeing if that slows the progress of MS."
The grant represents about one-third of the research group's annual spending. It comes before a World MS Day on May 29.
Source: ABC News © 2013 ABC (22/05/13)
Application submitted to FDA for approval of Plegridy™ (peginterferon beta-1a) in multiple sclerosis (21/05/13)
Biogen Idec has announced it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of Plegridy™ (peginterferon beta-1a), the company’s pegylated subcutaneous injectable candidate for relapsing forms of multiple sclerosis (RMS).
“We believe that based on the efficacy and safety Plegridy has demonstrated, in addition to its less frequent dosing schedule, it has the potential to become a preferred interferon treatment option.”
This regulatory submission was based on the results from the first year of the two-year global Phase 3 ADVANCE study. The data demonstrated that Plegridy met all primary and secondary endpoints by significantly reducing disease activity including relapses, disability progression and brain lesions compared to placebo, and showed favorable safety and tolerability profiles at one year.
“This filing demonstrates our dedication to the treatment of MS, both through the discovery of new medications and the development of innovative solutions that enhance treatment for people living with this disease,” said Douglas E. Williams, Ph.D., Biogen Idec’s executive vice president of Research and Development. “We believe that based on the efficacy and safety Plegridy has demonstrated, in addition to its less frequent dosing schedule, it has the potential to become a preferred interferon treatment option.”
In addition to the BLA filing with the FDA, Biogen Idec plans to submit a Marketing Authorisation Application (MAA) for Plegridy to the European Medicines Agency (EMA) in the coming weeks.
Biogen Idec has been the leader in the development of MS therapies for three decades and its robust treatment portfolio and development pipeline provides options that may help manage the disease from its earliest signs through its later stages.
The company anticipates hearing from regulatory authorities regarding the status and acceptance of these submissions within the next couple of months.
About Plegridy
Plegridy is a new molecular entity in which interferon beta-1a is pegylated to extend its half-life and prolong its exposure in the body, enabling study of a less frequent dosing schedule. Plegridy is a member of the interferon class of treatments and, if approved, would be a new addition to this class, which is often used as a first-line treatment for MS.
About ADVANCE
The two-year Phase 3 ADVANCE clinical trial is a global, multi-center, randomized, double-blind, parallel-group, placebo-controlled study designed to evaluate the efficacy and safety of Plegridy in 1,516 patients with relapsing-remitting MS.
The study investigates two dose regimens of PLEGRIDY, 125 mcg administered subcutaneously every two weeks or every four weeks compared to placebo. The analysis for all primary and secondary efficacy endpoints occurred at one year. After the first year, patients on placebo are re-randomized to one of the PLEGRIDY arms for the duration of the second year of the study. After completing two years in the ADVANCE study, patients have the option of enrolling in an open-label extension study called ATTAIN and will be followed for up to four years.
Source: Business Wire ©2013 Business Wire (21/05/13)
XenoPort to drop multiple sclerosis drug after trial failure(20/05/13)
XenoPort Inc said it will stop developing its experimental multiple sclerosis treatment after data from a late-stage trial showed the drug did not improve patients' condition significantly compared with a placebo.
The company said it would shut down all activities related to the drug, arbaclofen placarbil, and planned to provide an update on the impact of the expected savings.
The drug did not show statistical significance compared to the placebo on two clinical scales - severity of symptoms and response to treatment, and spasticity.
The drug, which was intended to treat spasticity, stiffness and involuntary multiple spasms, was tested on 228 multiple sclerosis patients in the United States.
Dosages of 30 mg and 45 mg of the drug administered twice a day were compared to the placebo group.
XenoPort said seven patients experienced adverse events, none of which were related to the treatment.
The company spent about $13.2 million in 2012 on the development of the drug, according to a regulatory filing.
XenoPort had stopped the development of arbaclofen placarbil as a heartburn drug in March 2011, after it failed in a mid-stage trial.
Source: Reuters © Thomson Reuters 2013 (20/05/13)
Scientists detect novel molecular target for multiple sclerosis(20/05/13)
Working with lab mice models of multiple sclerosis (MS), UC Davis scientists have detected a novel molecular target for the design of drugs that could be safer and more effective than current FDA-approved medications against MS.
The findings of the research study, published online today in the journal EMBO Molecular Medicine could have therapeutic applications for MS as well as cerebral palsy and leukodystrophies, all disorders associated with loss of white matter, which is the brain tissue that carries information between nerve cells in the brain and the spinal cord.
The target, a protein referred to as mitochondrial translocator protein (TSPO), had been previously identified but not linked to MS, an autoimmune disease that strips the protective fatty coating off nerve fibers of the brain and spinal cord. The mitrochronical TSPO is located on the outer surface of mitochondria, cellular structures that supply energy to the cells. Damage to the fatty coating, or myelin, slows the transmission of the nerve signals that enable body movement as well as sensory and cognitive functioning.
The scientists identified mitochondrial TSPO as a potential therapeutic target when mice that had symptoms of MS improved after being treated with the anti-anxiety drug etifoxine, which interacts with mitochondrial TSPO. When etifoxine, a drug clinically available in Europe, was administered to the MS mice before they had clinical signs of disease, the severity of the disease was reduced when compared to the untreated lab animals. When treated at the peak of disease severity, the animals' MS symptoms improved.
"Etifoxine has a novel protective effect against the loss of the sheath that insulates the nerve fibers that transmit the signals from brain cells," said Wenbin Deng, principal investigator of the study and associate professor of biochemistry and molecular medicine at UC Davis.
"Our discovery of etifoxine's effects on an MS animal model suggests that mitochondrial TSPO represents a potential therapeutic target for MS drug development," said Deng.
"Drugs designed to more precisely bind to mitochondrial TSPO may help repair the myelin sheath of MS patients and thereby even help restore the transmission of signals in the central nervous system that enable normal motor, sensory and cognitive functions," he said.
Deng added that better treatments for MS and other demyelinating diseases are needed, especially since current FDA-approved therapies do not repair the damage of immune attacks on the myelin sheath.
The UC Davis research team hopes to further investigate the therapeutic applications of mitochondrial TSPO in drug development for MS and other autoimmune diseases. To identify more efficacious and safer drug candidates, they plan to pursue research grants that will enable them to test a variety of pharmacological compounds that bind to mitochondrial TSPO and other molecular targets in experimental models of MS and other myelin diseases.
The journal paper is entitled, "A TSPO ligand is protective in a mouse model of multiple sclerosis."
Source: News Medical (20/05/13)
Mechanism found for Alemtuzumab side effect(20/05/13)
Researchers think they may have figured out why a drug that is highly effective against one autoimmune disease can sometimes trigger another.
The CD52-targeted biologic drug alemtuzumab (Lemtrada) is in late-stage development for multiple sclerosis, with long-term efficacy shown with just two annual courses of treatment. But, in a few patients, the agent appears to prompt an autoimmune attack on the thyroid or other organs. The mechanism has been unclear.
A new study from researchers in Australia and China, published in Nature Immunology, suggests a possible answer. They found that T cells expressing CD52 downregulated other T cells in vitro. Also, examination of samples from human patients with type 1 diabetes (the autoimmune form) showed low counts of these CD52+ cells as well as impaired function in the few that did exist.
In another set of experiments in diabetes-prone young mice, injecting the animals with lymphocyte populations lacking the CD52+ subset resulted in accelerated development of diabetes.
The researchers suggested that depletion of CD52 T cells -- as occurs after alemtuzumab treatment -- may unleash other T cells to attack host tissues, resulting in autoimmune disease.
Source: MedPage Today © 2013 MedPage Today, LLC (20/05/13)
Has Iran found a "cure" for MS? (16/05/13)
Reports are coming out of Iran that a so-called "cure" has been found for Multiple Sclerosis.
Iran Multiple Sclerosis Society Deputy Chairman, Mohammad Ali Saraian said, that Iran will be releasing a new oral Multiple sclerosis treatment on the local markets in October of 2013.
The treatment has been developed by Iranian doctors, and according to Saraian, is being tested on real patients, before being released to the open markets.
In January 2013, Iranian television reported that scholars from the Tabriz Medical Research University have prepared an herbal substance which can completely cure multiple sclerosis.
The treatment is called "MS Nut" (Nutrition) and made of special oil plants. It is delivered in syrup form.
The scholars have been working to prepare the treatment for two and a half years. The medicament was tested on 100 multiple sclerosis patients. 65 of them completed the treatment. Most patients fully recovered, but those with more advanced forms of the disease were unable to.
According to the information, the period of treatment with "MS Nut" lasts from six months to a year. The treatment has been already licensed in Iran.
It should be noted that at present this discovery has not been reported in scientific papers or journals.
In related news, Fars news agency reported that Saraian also has reported that multiple sclerosis is developing among youth in Iran.
"Judging by the people who come for treatments, we see that the multiple sclerosis has increased in the country, compared to 10 years ago," he said. "Most of them are young people."
Saraian noted that mostly the cases of multiple sclerosis have increased in such cities as Tehran, Shiraz and Isfahan.
Despite the fact that some Iranian sources claim there are some 50,000 multiple sclerosis patients in Iran, Saraian noted that there are no exact statistics on MS in Iran.
Source: TREND © 2013 Trend News Agency (16/05/13)
Smartphones as memory aids may help those with multiple sclerosis(14/05/13)
Smartphone apps to store your grocery list, find your car, and remind you to take your medication can help combat memory loss caused by multiple sclerosis.
Multiple sclerosis (MS) patients often struggle to keep track of doctor's appointments, children's school trips, and other elements of a busy, 21st-century schedule. But a new slate of digital tools can help patients with this neurological condition control the chaos and keep helpful reminders at their fingertips.
According to MS specialist Dr. Daniel Kantor, "One of the toughest symptoms for people with MS, their loved ones, and their neurologists, is memory loss. This can range from difficulty with multi-tasking and the tip-of-the-tongue phenomenon ('what’s that word I’m looking for?') to difficulty with remembering important life events."
When measuring and treating memory loss, one size doesn't necessarily fit all.
"Your neurologist may do the memory testing themselves or they may send you to a neuropsychologist...who specializes in doing hours of memory testing to help your neurologist better characterize what types of memory problems you are having [and] best figure out what part of the brain is involved," Kantor said in an interview with Healthline. "Some people do well with visual hints, and a neuropsychologist can help with making suggestions, such as using Post-It notes and reminders."
Now, with the addition of a few select applications, or “apps”, MS sufferers can quickly type—or even speak—these reminders into their cell phones.
Timely Reminders Could Save Your Life
There's no substitute for preparedness, and patients with MS know the unpredictable nature of their disease. Even those who do not suffer from memory loss can panic and forget critical information during a crisis.
In Case of Emergency, or ICE, is an app that can be used to store vital data about a patient's condition, allergies, medications and emergency contacts on his or her mobile device. First responders are trained to look for these emergency apps. Having this information organized in one place, where EMTs can easily retrieve it, can shave critical minutes off time otherwise spent sorting through a purse or a wallet—time that could be spent saving a life.
Memory loss can also impact the daily routine of taking medications. A majority of MS patients are prescribed one of a slate of disease-modifying drugs. Remembering to take their pill or perform their injection each day is necessary to ensure the efficacy of the treatment.
MediSafe, a virtual pillbox app released in November 2012, not only reminds you to take your meds, but also syncs data with your family members' devices, alerting them to missed medications so they can provide a gentle reminder as well.
“Medication adherence is a persistent and elusive problem, interrupting patients’ wellbeing, costing health providers and insurers billions annually, and causing preventable deaths,” said MediSafe Project CEO Omri ‘Bob’ Shor in a press release. “MediSafe Project’s involvement of patients’ loved ones and caretakers is proving itself a breakthrough in reducing the harm that comes from medication non-adherence.”
MediSafe reports that after eight weeks, 81 percent of its users were taking their medications on time. That’s a 31 percent jump from the World Health Organization’s estimated average medication adherence rate of only 50 percent.
Day-to-day Struggles
Memory loss impacts other aspects of an MS sufferer’s life as well. Simply remembering their parking spot at the mall can be a daunting task.
MyCar Locator, released in December 2011, utilizes your cell phone's GPS to set coordinates when you press the “park” button. When you want to return to your car, the app will lead you back to your vehicle with a simple directional arrow and approximate distance.
Out of Milk is another daily routine essential. The app allows users to create, sort, and share shopping and to-do lists, and also displays coupons and promotions from local grocery stores.
"We have been contacted by grateful users suffering from a range of issues including cognitive memory loss, patients suffering through chemotherapy, multiple sclerosis, and more who all tell us the same thing: Out of Milk has helped them and their families manage their shopping lists and save money quickly and easily," said Paul Sheaffer, co-founder of Capigami and developer of Out of Milk in an interview with Healthline.
"One of the core features of Out of Milk is it's ability to share lists in real-time," Sheaffer added. "We've found that many users going through medical hardship use this feature to share their needs with friends or family who also use the app who can then do their shopping for them."
The apps above are all available through the Google Play store for Android phones, but there are thousands more apps in the Amazon Appstore and on iTunes to help patients take charge of their daily and long-term goals.
"We think of our memory and the way we think as what makes us uniquely human," Kantor said. "Feeling like you are not thinking as well as you used to can make you feel like you have lost something. Some people feel depressed when they've lost a piece of themselves and that's why it is so important to alert the people around you about how you are feeling and not let yourself fall into depression, Remember, you are not alone!"
Source: Healthline Copyright © 2005 - 2013 Healthline Networks, Inc (14/05/13)
Blocking protein expression delays onset of multiple sclerosis in mice(10/05/13)
Blocking the expression of just one protein in the brain delays the onset of paralysis in mice with a form of multiple sclerosis, say researchers at the School of Medicine.
Exactly why this happens is still unclear. It may be, in part, that blocking expression of the protein, SIRT1, enhances the production of cells that make the insulating myelin sheath necessary for the transmission of nerve signals. This myelin coating is damaged in autoimmune diseases such as multiple sclerosis and Guillain-Barre syndrome.
Although much more research is needed, the findings suggest that it may one day be possible to induce the brains of patients with myelin-associated diseases or injuries to heal themselves by selectively interfering with the activity of SIRT1.
“We are excited by the potential implications our study has on demyelinating diseases and injuries,” said Anne Brunet, PhD, an associate professor of genetics. “It’s intriguing because activating SIRT1 is typically considered to be beneficial for metabolism and health, but in this case, inactivating SIRT1 can provide protection against a demyelinating injury.”
Brunet, who is also a member of the Stanford Cancer Institute, is the senior author of the research, which was published online May 5 in Nature Cell Biology. Postdoctoral scholar Victoria Rafalski, PhD, is the lead author of the study.
Blocking SIRT1 expression appears to work by promoting the development of neural stem cells in the brain into a type of cell called an oligodendrocyte precursor. These cells, in turn, become the mature oligodendrocytes that wrap the long arms of neurons with myelin — a fatty material necessary to facilitate the transmission of the electrical impulses from one nerve cell to another. In humans, most myelination occurs during infancy and adolescence.
Diseases such as multiple sclerosis wreak havoc in the central nervous system by damaging this protective myelin coating and impeding communication between nerve cells.
Because SIRT1 is more highly expressed in the brains of mice with an inducible form of multiple sclerosis, Brunet and her colleagues wondered what role the protein might play in the generation or inhibition of oligodendrocytes. To find out, they created a laboratory mouse in which the gene for SIRT1 is selectively disrupted in neural stem cells when the mouse is injected with a drug called tamoxifen. This technique allows the researchers to effectively turn SIRT1 expression off at will in neural stem cells.
The researchers found that, over time, a subset of the nerve stem cells in which SIRT1 expression had been eliminated began to make proteins indicative of oligodendrocyte precursor cells and eventually began to look like typical oligodendrocytes. Growing the neural stem cells in culture yielded similar results; genetically engineered cells lacking active SIRT1 (or unmodified cells treated with a drug that specifically inhibits the activity of the SIRT1 protein) resulted in a marked increase in the proportion of cells expressing an oligodendrocyte-specific protein marker.
When normal mice and those with inhibited SIRT1 expression were injected with a compound that causes the demyelination of nerve cells, the SIRT1-inhibited mice recovered more quickly. Furthermore, they were protected for a time from the paralysis that develops after the onset of the multiple-sclerosis-like disorder.
“Our work suggests that SIRT1 may normally limit the proliferation of oligodendrocyte precursors and that it has to be inactivated to transiently increase the number of these myelinating cells,” Brunet said.
To understand more about how SIRT1 works in the brain, the researchers identified a panel of genes that are more highly expressed when SIRT1 is absent. These genes included several involved in growth factor signaling, cell metabolism and protein production. One, called PDGFRalpha, activates a pair of signaling pathways within the cell. Blocking those pathways significantly inhibited the increase in oligodendrocyte precursor cells seen when SIRT1 is missing.
“Our study highlights the possibility of pharmacological manipulation of multiple nodes of the pathway to expand the population of oligodendrocyte precursors,” said Brunet. “Approaches such as these could have important implications for regenerative medicine.”
Other Stanford authors of the study include research assistants Peggy Ho, PhD, and Adiljan Ibrahim; undergraduate student Jamie Brett (now an MD-PhD student at Stanford); graduate student Elizabeth Pollina; postdoctoral scholar Duygu Ucar, PhD; former research associate Jason Dugas, PhD; Ben Barres, MD, PhD, professor and chair of neurobiology; and Lawrence Steinman, MD, professor of pediatrics and of neurology and neurological sciences.
The research was supported by the California Institute for Regenerative Medicine, the National Institutes of Health (grant numbers R01 AG026648, F31NS064600 and R01 NS05599705), an AFAR grant, a National Brain Tumor Society grant, the Glenn Foundation for Medical Research, the National Science Foundation, the Guthy-Jackson Charitable Foundation and the National Multiple Sclerosis Society.
Source: Health Canal (10/05/13)
Researchers make possible multiple sclerosis treatment breakthrough(10/05/13)
A potential new treatment for multiple sclerosis lies within modified adult stem cells, University of Adelaide researchers say.
The researchers are embarking on a new project which uses stem cells from fat tissue to send cells with special anti-inflammatory properties directly to the damaged site in the central nervous system.
MS is a progressive disease where the body attacks the central nervous system, causing nerve inflammation and scarring. It results in the impairment of motor, sensory and cognitive function.
Director of the Centre for Molecular Pathology, Professor Shaun McColl, said treatments for MS need to control the immune response and repair the damage caused to the fatty myelin sheaths which protect the nerves.
"We've already shown that adult stem cells have great potential to both control the immune response and promote repair of the central nervous system. It also prevents further damage," he said.
"But the trick is getting the stem cells to the right location where they can perform this function."
When stem cells are injected into the blood system, very few cross the blood/brain barrier into the central nervous system.
Lead investigator Dr Iain Comerford said it was hoped the manipulated adult stem cells could cross that barrier, targeting the inflammation site and repairing the damaged myelin.
"It involves promoting stem cell migration to the central nervous system by manipulating receptors on the surface of the stem cells that control cell movement," Dr Comerford said.
"We're also modifying the stem cells to suppress the immune response by introducing molecules that regulate inflammation."
At the end of the three-year project, the researchers aim to show they can successfully modify the stem cells to effectively reach the central nervous system and inhibit inflammation.
"If it works, there is great potential for a new therapy for this debilitating disease," Dr Comerford said.
Source: news.com.au Copyright 2013 News Limited (10/05/13)
Xenetic Bioscience's MS treatment moves to phase II(10/05/13)
Xenetic Biosciences has completed the Phase I, safety trial for its multiple sclerosis (MS) treatment MyeloXen and is now treating patients to establish dosing levels. The Phase I trial passed without any adverse event on six healthy volunteers and its Russian partner, Pharmsynthez, has now started Phase II where 12 patients will receive escalating dosing levels to establish the safe dose level for MyeloXen.
The final element of the trial will test the dosing range for further safety, surrogate efficacy and mode of action analysis.
MyeloXen has been formulated using Xenetic’s ImuXen liposomal entrapment technology and is one of up to six candidates being investigated under a Co-Development Agreement between Xenetic and St Petersburg–based Pharmsynthez. The global market for MS treatments is currently estimated to be worth US$20bn.
Scott Maguire, Xenetic’s chief executive, said: "The move to dosing MS patients represents an important milestone in this clinical trial. We now have all our platform technologies as represented by their respective products at least in Phase II clinical trials.
“We are encouraged with the announcement yesterday on the commencement of the ErepoXen Phase III trial for the treatment of anaemia, where our Russian partners are now making significant progress on our collaboration. We expect to make further announcements on additional clinical candidates in due course."
Source: Proactive Investors UK Copyright © Proactiveinvestors.co.uk, 2013 (10/05/13)
Surgery and risk for MS: a systematic review and meta-analysis of case--control studies(09/05/13)
Abstract (provisional)
Background: Although the precise etiology of multiple sclerosis is largely unknown, there is some speculation that a prior history of surgery may be associated with the subsequent risk for developing the disease. Therefore, we aimed to examine surgery as a risk factor for the diagnosis of multiple sclerosis.
Methods: We searched for observational studies that evaluated the risk for developing multiple sclerosis after surgery that occurred in childhood (<= 20 years of age) or "premorbid" (> 20 years of age). We specifically included surgeries classified as: tonsillectomy, appendectomy, adenoidectomy, or "surgery". We performed a systematic review and meta-analyses and calculated odds ratios (OR) and their 95% confidence intervals (CIs) using a random effects model.
Results: We identified 33 case--control studies, involving 27,373 multiple sclerosis cases and 211,756 controls. There was a statistically significant association between tonsillectomy (OR = 1.32, 95% CI 1.08-1.61; 12 studies, I2 = 44%) and appendectomy (OR = 1.16, 95% CI 1.01-1.34; 7 studies, I2 = 0%) in individual's <= 20 years of age and the subsequent risk for developing multiple sclerosis. There was no statistically significant association between risk for multiple sclerosis and tonsillectomy occurring after age 20 (OR = 1.20, 95% CI 0.94-1.53; 9 studies, I2 = 32%), in those with appendectomy at > 20 years (OR = 1.26, 95% CI 0.92-1.72; 5 studies, I2 = 46%), and in those with adenoidectomy at <= 20 years of age (OR = 1.06, 95% CI 0.68-1.68; 3 studies, I2 = 35%). The combined OR of 15 studies (N = 2,380) looking at "surgery" before multiple sclerosis diagnosis was not statistically significant (OR = 1.19, 95% CI 0.83-1.70; I2 = 71%).
Conclusions: We found a small but statistically significant and clinically important increased risk for developing multiple sclerosis, in those with tonsillectomy and appendectomy at <= 20 years of age. There was no convincing evidence to support the association of other surgeries and the risk for multiple sclerosis. Well-designed prospective etiological studies, pertaining to the risk for developing multiple sclerosis, ought to be conducted and should include the examination of various surgeries as risk factors.
Authors: Carole Lunny, Jennifer A Knopp-Sihota and Shawn N Fraser
Full Article
Source: BMC Neurology 2013, 13:41 doi:10.1186/1471-2377-13-41 © 2013 BioMed Central Ltd (09/05/13)
Mapi Pharma receives US patent for multiple sclerosis treatment(09/05/13)
Mapi Pharma, developer of complex bulk Active Pharmaceutical Ingredients (APIs), has been granted a US patent for a long acting depot formulation of glatiramer acetate, which is under the development as a once-a-month treatment for Multiple Sclerosis (MS).
The glatiramer acetate depot formulation is to be injected once-a-month under the skin or into a muscle for a prolonged release of the therapeutic agent.
Mapi Pharma president and CEO Ehud Marom said that the new patent strengthens the company's position that supports its business plan and advances the company one step closer to bringing MS patients an improved drug to be administered once-a-month.
MS is a chronic, often disabling disease that attacks the central nervous system whose symptoms include numbness in the limbs, paralysis and loss of vision that are unpredictable and differ from one person to another.
The global market for MS pharmaceuticals is estimated to be approximately $10bn, according to Mapi Pharma.
Source: PBR Drug Research Drug Delivery © PBR 2013. Part of Progressive Digital Media Group Plc (09/05/13)
Oral treatment for MS, Tecfidera, approved for use in Canada(08/05/13)
A new oral treatment for those living with multiple sclerosis has been approved for use in Canada.
Tecfidera is an oral medication recently approved by Health Canada.
It becomes just the second oral drug available in the country to treat multiple sclerosis – other treatments are done by injections.
Julie Kelndorfer has spent nine years living with MS and says it’s been a rollercoaster ride.
“It’s been an interesting journey for all of us, my whole family,” Kelndorfer.
Multiple sclerosis attacks the protective covering or myelin, of the brain and spinal cord, interrupting the flow of nerve pulses which affects vision, hearing, memory, balance and mobility.
Kelndorfer has a form of MS called relapsing-remitting.
She recently experienced an episode she’d never been through before.
“It was actually the MS attacking my brain stem so my balance was off, it was like I was having seizures, I was in a convulsion,” she said. “It was scary.”
After that episode, Kelndorfer stopped taking the daily injection medication she had been on for years.
She has since spent months researching Tecfidera to see if it will work for her.
“When something is new and exciting, at the same time you’re hesitant to be the first one out of the gate,” she said.
Kelndorfer is holding out hope that Tecfidera will mean fewer muscle spasms.
“I think it’s another arsenal in the fight against MS and I think that’s exciting,” she said.
High prevalence of MS in Alberta
Neurologist Dr. Fabrizio Giuliani says the prevalence of multiple sclerosis cases in Canada is high and those numbers are highest in Alberta.
“Alberta seems to have the highest prevalence. Some people talk of MS as the ‘Disease of Alberta’ because of the numbers,” Giuliani said.
The only other oral MS drug available in Canada comes with serious side effects that make it an option many doctors tend to avoid recommending.
That fact makes Tecfidera an attractive alternative treatment for patients.
“Every drug has side effects. We don’t have to forget that. It’s the severity of the side effects sometimes that can be different based on the mechanism of the drug,” he said. “For the Tecfidera it looks like the safety profile is quite good.”
According to the MS Society of Canada, clinical trials on hundreds of patients have shown Tecfidera reduces relapses by 53 per cent and slows the disease progression by 38 per cent.
Kelndorfer knows all therapies come with risks. She has read that Tecfidera is known to cause gastrointestinal problems but she’s willing to give the pill a try if it will – for the most part – improve her overall quality of life.
“The side effects seem fairly reasonable,” Kelndorfer said. “For the time that I’m living, I want a quality of life that let’s me be the mom, the wife, the employee.”
Kelndorfer says she was told by her doctor that she could be on the new treatment as early as next month.
These therapies cost anywhere between $20,000 to $40,000 a year.
Tecfidera still needs to undergo review by the Canadian Drug Expert Committee as well as a local expert committee before determining if the medication will be funded by the province.
“Both expert committees review new medications to ensure they are safe, efficient and cost-effective, ensuring Albertans are kept safe and that public dollars are spent with the best health outcomes in mind,” Alberta Health spokesperson John Muir said in a statement to CTV News.
Source: CTV News © 2013 Bell Media (08/05/13)
Sativex® commercialisation approval in Italy(07/05/13)
GW Pharmaceuticals plc has announced that it has received commercialisation approval for its prescription medicine Sativex® in Italy. A full marketing authorization has been granted by the authorities in Italy for the treatment of moderate to severe spasticity in Multiple Sclerosis (MS) patients who have not responded adequately to other anti-spasticity medications. This license to market Sativex® in Italy comes by way of the publication of Sativex® in the Italian Official Journal (Gazzetta Ufficiale) and reflects that regulatory, pricing and reimbursement approvals are now in place for an expected commercial launch in September by GW's partner, Almirall S.A. The reimbursed price of the medicine granted by the authorities in Italy is consistent with the reimbursed Sativex® price in Spain.
The publication of Sativex® in the Italian Official Journal triggers a €250,000 milestone payment from Almirall to GW.
"The approval in Italy brings the total to 21 countries that have approved Sativex® for use in the treatment of MS spasticity," stated Justin Gover, Chief Executive Officer of GW. "In granting reimbursement approval for Sativex®, we appreciate the foresight of the Italian authorities in recognizing the value of a medicine that treats a particularly debilitating symptom of MS that is not adequately treated with currently available medications. We now look forward to working with our partners Almirall towards commercial launch in this important market in a few months' time."
Source: GW Pharmaceuticals plc (07/05/13)
Peli1 identified as pivotal actor in animal model of multiple sclerosis(07/05/13)
Scientists have identified an influential link in a chain of events that leads to autoimmune inflammation of the central nervous system in a mouse model of multiple sclerosis (MS).
An international team of researchers led by scientists in The University of Texas MD Anderson Cancer Center Department of Immunology reported their results in an advance online publication in Nature Medicine.
The researchers spell out the pivotal role of Peli1 in the activation of immune cells called microglia that promote inflammation in the central nervous system in response to tissue damage or invasion by microbes.
"The major implication of discovering a signaling role for Peli1 in this animal model is that it might also be significant in the pathogenesis of MS," said senior author Shao-Cong Sun, Ph.D., professor in MD Anderson's Department of Immunology.
Microglia cells involved in multiple sclerosis
Sun and colleagues found that Peli1 is heavily expressed in microglial cells and promotes their activation and subsequent damaging immune response. Peli1 also protects that autoimmune reaction by initiating the destruction of a protein that otherwise would inhibit inflammation.
Microglia are known to be crucial to the initiation of MS, an immune system assault on nerve fibers called axons and on myelin, the protective sheath around the axons. They also were previously known to play a similar role in experimental autoimmune encephalomyelitis (EAE), an animal model of MS.
The precise mechanism of this autoimmune-stimulating effect has been unknown. Sun and colleagues fill an important gap with their Peli1 discovery.
Microglia sense tissue damage. They secrete chemokines and inflammatory cytokines in response, drawing infection-fighting T cells into the central nervous system, leading to inflammation.
Infections genetic overreaction that inflames
The authors note that microbial infections are a known environmental trigger for the onset and maintenance of multiple sclerosis and the induction of EAE in mice. Toll-like receptors that detect pathogens play a roll in MS and EAE. They were suspected of involvement in microglial activation and inflammation.
Upon sensing microbes or cell damage, toll-like receptors launch a signaling cascade that activates a variety of genes involved in inflammation and white blood cell homing to the microbes or injury site.
Peli1 is known as a targeting agent, marking proteins with molecules called ubiquitins, ensuring they are functionally modified or found by cellular protein-destruction machinery. In this case, Sun and colleagues found that Peli1 ubiquitinates another targeting agent as a signal, which in turn marks a crucial anti-inflammatory protein for destruction.
The team found:
Mice with Peli1 knocked out were resistant to EAE. Those with Peli1 developed severe symptoms including a gradual increase in paralysis.
Mice with intact Peli1 had high levels of microglial activation after EAE began and low levels of resting microglia. Mice with Peli1 knocked out had high levels of resting microglia.
Expression of proinflammatory chemokines and cytokines was impaired in microglia taken from Peli1 knockout mice. Peli1 sends signal to destroy Traf3
Sun and colleagues then tracked down the role Peli1 plays in protecting one of the molecular networks that is set off when toll-like receptors detect microbes or injury. The MAPK pathway activates a variety of genes involved in inflammation and T cell response.
MAPK is kept in check by a protein called Traf3. The team found that Peli1 signals another ubiquitin ligase that in turn marks Traf3 for destruction, liberating the MAPK network.
After EAE is induced, mice with intact Peli1 have a gradual depletion of Traf3 in their microglia. Traf3 accumulated in the microglia of Peli1 knockout mice. EAE was restored in Peli1 knockout mice when Traf3 was inhibited.
Sun said the team is studying the pathway in human multiple sclerosis to replicate their findings and explore the possibilities for potentially treating MS.
Source: Medical News Today © MediLexicon International Ltd 2004-2013 (07/05/13)
Multiple Sclerosis more common in black women than white(07/05/13)
Multiple sclerosis (MS) is more common in black women than in white women, according a new study.
The research was conducted by Kaiser Permanente and was published in the journal Neurology. The results contradict the widely believed notion that black people are less vulnerable to the disease.
The electronic health records of over 3.5 million members of Kaiser Permanente Southern California were analyzed from the beginning of 2008 to the end 2011. A total of 496 patients newly diagnosed with MS were identified.
Of these new cases, black patients had a 47% greater likelihood of MS compared to white patients. Results also showed that Hispanics had a 50% reduced risk compared to white patients, and Asians had a 80% lower chance than whites.
Seventy percent of MS cases occurred among females, the researchers said. However, "this preponderance of females diagnosed was more pronounced among black patients than white patients."
Black females had an increased prevalence of multiple sclerosis compared to both white males and females, the authors pointed out. Black men, on the other hand, had a comparable probability of MS riskto white men.
Additionally, the reduced risk among Hispanic and Asian patients was true for both males and females.
Leading author Annette Langer-Gould, MD, PhD, of the Kaiser Permanente Southern California Department of Research & Evaluation, said:
"Our findings do not support the widely held belief that blacks have a lower risk of MS than whites, but that MS risk is determined by complex interactions between race, ethnicity, sex, environmental factors and genotypes.
Although additional research is needed, possible explanations for the higher incidence of MS in black women include a greater prevalence of hormonal, genetic, or environmental risk factors such as smoking, compared to patients from other racial or ethnic groups."
Approximately 19,000 Americans are diagnosed with MS each year, or 250 each week, according to the report. The mean age of MS diagnosis among the subjects was 41.6 years, however, onset can occur between 8.6 and 78.3 years.
Although the average time from the start of symptoms to MS diagnosis was 4 months, it could be as long as 40 years, the authors explained.
Overall, Hispanic and Asian patients were younger at the time of their diagnosis than whites and African-Americans.
Worldwide prevalence reports as well as a single study of Korean War veterans in the 1950s, which showed that white males had a two times higher chance of receiving disability compensation for MS than blacks, are responsible for the idea that MS is not common in black people.
Dr. Langer-Gould concluded:
"A possible explanation for our findings is that people with darker skin tones have lower vitamin D levels and therefore an increased risk of MS. However, this does not explain why Hispanics and Asians have a lower risk of MS than whites, or why only black women but not black men are at a higher risk of MS.
Our findings indicate that including persons from different racial and ethnic groups in future studies of MS susceptibility and prognosis will likely reveal important insights into the causes of this often debilitating disease."
Source: Medical News Today © MediLexicon International Ltd 2004-2013 (07/05/13)
Acclaimed Brambles MS respite care centre to close(02/05/13)
Acclaimed Brambles Respite Care Centre for people with Multiple Sclerosis (MS) has announced it is to close this Saturday (May 4).
The sudden announcement on the centre's website from MS Respite and Care Services, which runs the Brambles, stated the move was being made “with great regret,”staff and guests had been met with and told the position, and those who had made imminent bookings had been contacted.
Unfortunately, the closure announcement coincides with this year's MS Awareness Week, which seeks to raise the profile of the debilitating condition and the search for a cure.
The website statement explained: “We advise, with great regret, that it is necessary for MS Respite and Care Services Limited to close the existing MS Respite care service at Brambles Respite Care Centre.
“We continue to believe there is demand for an MS service in this area, but only one that is allied to a larger care home, providing a wider range of services.
“We've met with both staff on site and guests who were at the home and explained the position.”
The statement continued: “The staff conducted themselves with great professionalism and I believe will continue to provide an excellent service for guests who continue to use the service up until closure on May 4.”
It added: “Although an independent company, St Cloud Care, is offering to assist any guests with alternative facilities elsewhere, for those who wish to consider that option.”
The facilities highlighted included Priory Court, a large nursing home at Ewell, near Epsom.
The statement concluded: “We would like to thank those who have supported the Brambles service over the years.”
The Brambles Respite Care Centre in Suffolk Close is widely regarded as one of the best of its kind in the country.
The state-of-the-art complex offered 24-hour care for people with MS and a much-needed break for their carers.
Over more than 20 years, its staff helped thousands of people with MS and their carers and families.
However, in June 2010, the MS Society, which used to run the Brambles, withdrew its funding.
The society said it was to move away from directly providing respite care services at its four centres, to allow it to focus on enabling people across the UK to access respite and short breaks that are right for them wherever they live. The society made efforts to find an alternative care provider to take over the running of the Brambles, but announced in autumn 2011 that its talks with the main hope to take over the centre, MS Respite and Care Services Limited, had fallen through. Arrangements were being made to close the centre with redundancy talks taking place and letters being sent out to the people who used it, when the MS Society announced it had reached a last minute agreement with MS Respite and Care Services Limited. The initial agreement, known as ‘Heads of Terms,’ stated that MS Respite and Care Services Limited would continue services for people with MS and transfer existing staff into their employment. The centre did close its doors temporarily in November 2011, but reopened the following February under MS Respite and Care Services Limited, an associated company of St Cloud Care plc.
Source: This Is Local London © Copyright 2001-2013 Newsquest (London) Ltd (02/05/13)
Multiple sclerosis patients are missing out on drugs(29/04/13)
Only 40% of people eligible for drugs to combat multiple sclerosis in the UK are actually taking them, says a report from the MS Society.
A survey of more than 10,000 adults with MS showed that many were missing out on the seven licensed medicines approved for use.
The charity said a lack of information and access to specialists was to blame.
It is calling for the government to provide a personalised care plan to every person with MS.
The MS Society's survey and accompanying report showed that there were differences in access to disease-modifying treatments (DMTs) across the four nations of the UK.
These are medicines that can reduce the frequency and severity of MS attacks, and in some cases can slow the progression of the disabling condition.
Someone living in Northern Ireland with MS was twice as likely to be taking a DMT (68%) than someone with the condition in Wales (30%), for example.
Access to treatment in Scotland and England was only a little higher at 36% and 40%.
In Europe, additional research shows that only Poland and Romania have a smaller proportion of people with MS taking licensed medicines.
Routine assessment
The charity's report said that being well informed about the medicines available was crucial.
Those who felt they had enough information about medicines were 32% more likely to be taking a DMT, the survey found, and those with access to a specialist MS nurse or neurologist were more than twice as likely to be taking the appropriate drugs.
Northern Ireland is the only place in the UK where most people with MS are routinely invited every six months to see a neurologist or MS nurse for a review.
This means that people with MS are constantly having their treatment options assessed, the report says.
As a result, they are more likely to get the information they need and discuss issues such as side-effects.
Yet this may not be the only solution. Forty-one per cent of those who said they did have enough information about drug treatments still did not take a disease-modifying treatment.
The report concluded: "This could be due to barriers to accessing medicines; because individuals make an informed decision not to take them; or because they don't know what information is out there that they could have access to, such as around new treatments or new evidence of efficacy."
New policy
Nick Rijke, director for policy and research at the MS Society, said people with multiple sclerosis were facing a lottery.
"These findings worryingly suggest that the likelihood of someone receiving a life-changing treatment is often based on luck - like where they live or how helpful their healthcare professional is - rather than their genuine clinical need.
"When it comes to prescription rates, the UK ranks 25th out of 27 European countries. Given the relative wealth of the UK this is simply unacceptable."
The MS Society is now calling on all four governments in the UK to ensure every person with MS has a personalised treatment, care and support plan, with two comprehensive reviews each year.
Ed Holloway, head of care and services research at the MS Society, said that because some MS drugs were costly, they were often not offered when they should be because of restricted NHS budgets.
'Speak to doctor'
A spokesman for NHS England, which has recently taken on the commissioning of treatment for MS from primary care trusts, said a new policy from 1 April would mean that people across England would have the same access to treatment.
"By making decisions nationally about specialist treatments, we are confident that patients will now be able to receive the treatment they need, irrespective of where they live.
"As with all policies, we will continue to collect and review the outcome of treatments for patients and consider them when our policy is reviewed.
"If a patient has concerns about the treatment they are receiving we would urge them to speak to their GP or consultant."
Source: BBC News © British Broadcasting Corporation 2013 (29/04/13)
Aubagio® delays onset of clinically definite multiple sclerosis, study(25/04/13)
Genzyme, a Sanofi company, announced today positive top-line results from the TOPIC trial for Aubagio (teriflunomide). The trial was designed to assess whether early initiation of Aubagio (teriflunomide) in patients who experienced their first neurological symptoms consistent with Clinically Isolated Syndrome (CIS) can prevent or delay conversion to clinically definite multiple sclerosis (CDMS).
Clinically isolated syndrome (CIS) is defined as a first clinical attack with features suggestive of MS. It typically occurs in young adults and is often a prelude to CDMS.
In the TOPIC trial, patients receiving Aubagio 14 mg and 7 mg were significantly less likely to develop CDMS, defined as occurrence of a second clinical attack, the primary endpoint, as compared to placebo. Additional results, including key secondary and tertiary objectives, will be presented at a forthcoming scientific meeting.
“Clinically Isolated Syndrome (CIS) is often a prelude to clinically definite multiple sclerosis, and early treatment has proved beneficial,” said Dr. Aaron E. Miller, Medical Director, The Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Mount Sinai Medical Center. “These findings are important as there is an unmet need for an efficacious oral option for patients at this stage of disease.”
The average duration of Aubagio exposure in TOPIC was approximately 16 months. Adverse events observed in the trial were consistent with previous clinical trials with Aubagio in MS. The most common types of adverse events reported more frequently in the Aubagio arms were ALT (Alanine transaminase) elevations, nasopharyngitis, headache, hair thinning, diarrhea and paresthesia. There were no deaths reported in either teriflunomide group over the course of the study. There was one death due to suicide in the placebo arm. The rate of treatment discontinuation due to adverse events was similar across treatment arms (9.1 percent in placebo arm, compared to 12.1 percent in 7 mg teriflunomide arm and 8.3 percent in 14 mg teriflunomide arm).
“Aubagio is the only oral marketed MS therapy to successfully be studied in CIS and we are very pleased by these topline results which further reinforce the consistent efficacy and potential for treating a broad spectrum of MS patients,” said Genzyme President and CEO, David Meeker, MD. “We remain committed to addressing unmet needs in MS through new areas of research and delivery of differentiated therapies for this devastating disease.”
The trial compared treatment with either 14 mg or 7 mg once-daily, oral Aubagio against placebo. This double-blind, multi-center trial enrolled 618 patients who had experienced a first acute or sub-acute, well-defined neurological event consistent with demyelination, as well as onset of MS symptoms within 90 days of randomization, and MRI scan showing two or more T2 lesions characteristic of MS.
Aubagio is approved in the U.S., Australia and Argentina for the treatment of relapsing forms of MS. Marketing applications for Aubagio are also under review by regulatory authorities globally.
The ongoing Aubagio clinical development program, involving more than 5,000 patients in 36 countries, is amongst the largest of any MS therapy. Some patients in extension trials have been treated for up to 10 years.
Source: Fort Mill Times © Copyright 2013, Fort Mill Times (25/04/13)
Ingredient in new MS drug, Tecfidera, linked to PML(25/04/13)
The active ingredient in a drug that’s expected to become a popular treatment for multiple sclerosis has been linked to four European cases of a rare but sometimes fatal brain disease called progressive multifocal leukoencephalopathy (PML).
The ingredient, dimethyl fumarate, is used in a drug called Fumaderm that was approved in Germany in 1994 to treat the skin condition psoriasis. It is also in a different but closely related medication called Tecfidera, which was just approved by the U.S. Food and Drug Administration in March for the treatment of multiple sclerosis (MS). It is known as a fumaric acid ester, which is commonly used as a food additive and has been used to treat psoriasis in Germany for 30 years.
According to reports published in the April 25 issue of the New England Journal of Medicine, however, four patients who were taking Fumaderm to treat their psoriasis developed PML.
In a letter responding to the reports, Biogen, the company that makes both drugs, said Tecfidera may be safer because it contains only dimethyl fumarate, while Fumaderm also contains three other fumaric acid esters.
The company also noted that none of the patients taking Tecfidera during clinical trials (then known as BG-12) developed PML. Since Tecfidera is a pill rather than an injection, and was effective and well-tolerated by patients in clinical trials, analysts have predicted it would soon become the top-selling multiple sclerosis treatment.
But the German doctor who treated one of the psoriasis patients who got PML thinks there is still cause for concern.
Dr. Jorg Schulz, a neurologist at Rheinisch-Westfaelische Technische Hochschule Aachen, a research university in Aachen, said the two drugs are virtually identical once they are broken down in the body.
“The problem is that the studies with BG-12 covered a two-year period, but no longer periods,” Schulz said, and he believes it may take prolonged treatment with the drug for PML to surface.
“With the publication of our case, we wish to create awareness that treatment with any form of fumaric acid may bear the risk of developing PML,” Schulz said.
PML is caused by the JC virus, which normally lies dormant in the body and causes no harm. About half of multiple sclerosis patients have antibodies to the JC virus in their blood, suggesting a current or former infection. When the immune system is depleted by illnesses like cancer or AIDS or suppressed by certain medications, the virus can flare and destroy nerve cells in the brain.
Ironically, PML is a lot like multiple sclerosis, but it progresses more rapidly as it causes weakness, paralysis, confusion, memory loss and loss of vision or speech. Quick treatment can stop the damage, although patients may be permanently disabled.
PML is rare, but it is so serious that Genentech pulled its psoriasis treatment Raptiva off the market in 2009 after reports of four cases in patients who were taking the biologic medication. Biologic drugs are medications derived from living organisms that are used to prevent certain diseases, including ones where the immune system malfunctions.
Another biologic, Tysabri, a treatment for multiple sclerosis and Crohn’s disease, was shelved in 2005 for about a year after three patients involved in clinical trials of the drug developed PML. Tysabri returned to the market in 2006 with strict new safeguards in place. Before starting the drug, for example, patients must get an MRI of their brains. They also may get blood tests to check for antibodies to the JC virus. They also are monitored by doctors every three to six months while taking the medication, which is also made by Biogen.
No such precautions are currently recommended for Tecfidera, which has been hailed as a less toxic alternative to other treatments. Other multiple sclerosis drugs are known to cause flu-like symptoms, chest pain, and heart, liver and eye problems.
In clinical trials, the main side effects reported with Tecfidera were comparatively mild, and included facial flushing, stomach upset and low white blood cell counts.
“The main reason why our [psoriasis] patient developed PML after three years of treatment with Fumaderm is prolonged lymphocytopenia [low white blood cell counts],” Schulz said. “In both BG-12 trials published in the New England Journal of Medicine in 2012, 4 percent to 5 percent of patients developed this kind of severe lymphocytopenia and are in my view at risk to also develop PML.”
Another expert noted another caveat.
About 3 percent of patients in the Tecfidera trials were taken off the medication when their white blood cell counts dropped too low, said Dr. Robert Fox, a staff neurologist at the Mellen Center for Multiple Sclerosis at the Cleveland Clinic in Ohio.
“The intensive monitoring of the clinical trial may have been what helped to prevent PML,” said Fox, who was on the steering committee for one of the studies that tested the medication and who has been a paid consultant for Biogen.
Fox added that the PML case reports are “very important additions to our understanding of this class of therapy.”
“Although we haven’t seen any PML cases in patients treated with Tecfidera, I think there’s good reason to apply the lessons learned here to Tecfidera,” he said.
Source: Health Copyright © 2013 Health Media Ventures, Inc (25/04/13)
