Archive for the ‘Multiple Sclerosis Research’ Category

Eleven years ago, Megan Quinn had just gotten married and was the picture of health.

"I used to run five miles a day. All of a sudden on my third mile, I started dragging my foot and I didn't understand. I thought, I'm just getting old and I'm getting tired. I was 27 years old," she said. "Nothing ever clicked to me that something was wrong."

The diagnosis was multiple sclerosis.

Multiple sclerosis, or MS, is an autoimmune disease where the body attacks itself and damages myelin, the protective covering surrounding nerve cells. With that insulation compromised, the nerves deteriorate and can cause a wide range of symptoms including vision problems, fatigue and weakness. The disease affects as many as 350,000 Americans.

"For the past year I've had a really bad time with this disease, just with my hip not working. One night I woke up and I couldn't feel either of my legs," Quinn said.

"Right now, my biggest problem is my hamstring. I cannot get my hamstring to cooperate when I have to walk, so that's my battle right now," she said.

Current treatments only try to stop progression of the disease. Quinn is about to test a new approach: using stem cells designed to actually make MS patients better.

Stem cells can be morphed into any cell in the body. Patients like Quinn have bone marrow removed and the stem cells inside are then changed in the kind of stem cells found in the brain and spinal cord.

Those cells will then be injected directly into the spinal cord. The hope is that they will repair the insulation and perhaps even the wires underneath.

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MS patient to take part in pioneering experiment

Introduction

Over the last year, there have been over 1,500 articles published in medical journals on multiple sclerosis or animal models of the disease. The majority of these articles represent new research into MS, its causes, its mode of action or potential treatments for it. In addition, there has been an even greater amount of new research into cell biology, genetics, the immune system, other autoimmune, inflammatory and neurological diseases, virology and stem cell research - all of which give us a better understanding of the biological environment in which the disease operates.

It is impossible to sum up all of this research in a single essay and, for this reason, I intend to concentrate on those areas which seem to me to be particularly hopeful. This is, therefore, a personal view. This section will take a while to complete but I'm publishing it in installments.

There is no global authority coordinating the MS research effort. This is probably a good thing because it allows researchers to attack the problem from oblique angles and provide novel and unexpected insights into the disease. However, I intend to structure this article as if it were a progress report for just such a global research project. This project would group the research into four areas:

2. Arresting the progress of the disease

Of course, those of us who are already carrying significant deficits as a result of MS might wish to reorder these priorities. In any event, researchers are making significant progress in all these areas.

Finding the Cause of Multiple Sclerosis

Looking for the genes that convey a susceptibility to Multiple Sclerosis

Introduction

Recently, there has been a lot of interest in the genetics of complex diseases such as multiple sclerosis. The human genome has recently been mapped in its entirety and the hope is that this will allow researchers to isolate the genes for such diseases by statistical analysis of affected populations.

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Research in Multiple Sclerosis

PUBLIC RELEASE DATE:

18-Dec-2013

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (Dec. 18, 2013) Multiple sclerosis (MS), an inflammatory autoimmune disease affecting more than one million people worldwide, is caused by an immune reaction to myelin proteins, the proteins that help form the myelin insulating substance around nerves. Demyelination and MS are a consequence of this immune reaction. Bone marrow mesenchymal stem cells (MSCs) have been considered as an important source for cell therapy for autoimmune diseases such as MS because of their immunosuppressive properties.

Now, a research team in Brazil has compared MSCs isolated from MS patients and from healthy donors to determine if the MSCs from MS patients are normal or defective. The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct1131.

"The ability of MSCs to modulate the immune response suggests a possible role of these cells in tolerance induction in patients with autoimmune diseases, and also supports the rationale for MSC application in the treatment of MS," said study corresponding author Dr. Gislane Lelis Vilela de Oliveira of the Center for Cell-Based Research at the University of Sao Paulo. "We found that MS patient-derived MSCs present higher senescence, or biological aging, and decreased expression of important immune system markers as well as a different transcriptional profile when compared to their healthy counterparts."

The researchers suggested that further clinical studies should be conducted using transplanted allogenic (other-donated) MSCs derived from healthy donors to determine if the MSCs have a therapeutic effect over transplanted autologous (self-donated) MSCs from patients.

"Several reports have shown that bone marrow-derived MSCs are able to modulate innate and adaptive immunity cell responses and induce tolerance, thus supporting the rationale for their application in treating autoimmune diseases, " said the researchers.

They also noted that studies have shown that transplanted MSCs migrate to demyelinated areas as well as induce generation and expansion of regulatory T cells, important in immunity.

"We found that the transcriptional profile of patient MSCs after transplantation was closer to that of their pre-transplant MSC samples than those from their healthy counterparts, suggesting that treatment with patient self-donated MSCs does not reverse the alterations we observed in MSCs from MS patients," they concluded.

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Preferable treatment for MS found in allogenic bone marrow stem cells

AAP New research suggests a little bit of sunshine can help ease symptoms of multiple sclerosis.

Vitamin D may combat multiple sclerosis (MS) by blocking the migration of destructive immune cells to the brain, new research suggests.

The findings may help explain anecdotal reports of the "sunshine vitamin" preventing or easing symptoms, say scientists.

MS is known to be more prevalent in parts of the world furthest from the equator, where there is less sunshine to trigger production of vitamin D in the skin.

The disease is caused by the body's own immune defences damaging myelin, a fatty insulating sheath that surrounds nerve fibres and is vital to the proper transmission of nerve signals.

Destruction of myelin leads to symptoms ranging from numbness to blurred vision and paralysis.

"With this research, we learnt vitamin D might be working not by altering the function of damaging immune cells but by preventing their journey into the brain," said lead scientist Dr Anne Gocke, from Johns Hopkins University School of Medicine in the US.

"If we are right, and we can exploit Mother Nature's natural protective mechanism, an approach like this could be as effective as, and safer than, existing drugs that treat MS."

In a person with MS, immune system cells called T-cells are primed to travel out from the lymph nodes and seek and destroy myelin in the central nervous system.

Dr Gocke's team of researchers simultaneously gave mice the rodent form of MS and a high dose of vitamin D. They found that disease symptoms were suppressed in the animals.

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Vitamin D may help fight MS: study

Treatments under investigation for multiple sclerosis may improve function, curtail attacks, or limit the progression of the underlying disease. Many treatments already in clinical trials involve drugs that are used in other diseases or medications that have not been designed specifically for multiple sclerosis. There are also trials involving the combination of drugs that are already in use for multiple sclerosis. Finally, there are also many basic investigations that try to understand better the disease and in the future may help to find new treatments.

Research directions on MS treatments include investigations of MS pathogenesis and heterogeneity; research of more effective, convenient, or tolerable new treatments for RRMS; creation of therapies for the progressive subtypes; neuroprotection strategies; and the search for effective symptomatic treatments.[1]

Advances during the last decades has led to the recent approval of several oral drugs. These drugs are expected to gain in popularity and frequency of use at the expense of previously existing therapies.[2] Further oral drugs are still under investigation, the most notable example being laquinimod, which was announced in August 2012 to be the focus of a third phase III trial after mixed results in the previous ones.[3] Similarly, Other studies are aimed to improve efficacy and ease of use of already existing therapies through the use of novel preparations. Such is the case the PEGylated version of interferon--1a, that has a longer life than normal interferon and therefore it is being studied if given at less frequent doses has a similar efficacy than the existing product.[4][5] Request for approval of peginterferon beta-1a is expected during 2013.[5]

Monoclonal antibodies, which are drugs of the same family as natalizumab, have also raised high levels of interest and research. Alemtuzumab, daclizumab and CD20 monoclonal antibodies such as rituximab, ocrelizumab and ofatumumab have all shown some benefit and are under study as potential treatments for MS.[6] Nevertheless their use has also been accompanied by the appearance of potentially dangerous adverse effects, most importantly opportunistic infections.[2] Related to these investigations is the recent development of a test against JC virus antibodies which might help to predict what patients are at a greater risk of developing progressive multifocal leukoencephalopathy when taking natalizumab.[2] While monoclonal antibodies are probably going to have some role in the treatment of the disease in the future, it is believed that it will be small due to the risks associated to them.[2]

Another research strategy is to evaluate the combined effectiveness of two or more drugs.[7] The main rationale for polytherapy in MS is that the involved treatments target different mechanisms of the disease and therefore their use is not necessarily exclusive.[7] Moreover synergies, in which a drug potentiates the effect of another are also possible. Nevertheless there can also appear important drawbacks such as antagonizing mechanisms of action or potentiation of deleterious secondary effects.[7] While there have been several clinical trials of combined therapy none has shown positive enough effects to merit the consideration as a viable treatment for MS.[7]

Finally, regarding neuroprotective and specially regenerative treatments, such as stem cell therapy, while their research is considered of high importance at the moment they are only a promise of future therapeutic approaches.[8] Likewise, there are not any effective treatments for the progressive variants of the disease. Many of the newest drugs as well as those under development are probably going to be evaluated as therapies for PPMS or SPMS, and their improved effectiveness when compared with previously existing drugs may eventually lead to a positive result in these groups of patients.[2]

The main measure of evolution of symptoms, specially important as an endpoint in MS trials, is the EDSS. However, this and other measures used in clinical studies are far from perfect and suffer from insetiveness or inadequate validation.[9] In this sense there is ongoing research to improve the EDSS and other measures such as the multiple sclerosis functional composite. This is important as the greater efficacy of existing medications force functional measures in clinical trials to be highly sensitive in order to adequately measure disease changes.[9]

Advances in genetic testing techniques have led to a greater understanding of the genetics of MS. However, it is hard to predict how this future discoveries will impact clinical practice or research for new drugs and treatments.[2]

An example of a soon-to-be finished study is the Wellcome Trust case control consortium, a collaboration study including 120,000 genetic samples, of which 8000 are from individuals with MS.[10] This study may presumably identify all the common genetic variants involved in MS.[10] Further studies will probably involve full genome sequencing of large samples, or the study of structural genetic variants such as insertions, deletions or polymorphisms.[10]

Disease-modifying drugs and procedures represent possible interventions able to modify the natural course of the disease instead of targeting the symptoms or the recovery from relapses. Over a dozen clinical trials testing potential therapies are underway, and additional new treatments are being devised and tested in animal models.

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Multiple sclerosis research - Wikipedia, the free encyclopedia

Multiple Sclerosis (MS) is the most common disabling neurological disease of young adults. It most often appears when people are between 20 to 40 years old. However, it can also affect children and older people.

The course of MS is unpredictable. A small number of those with MS will have a mild course with little to no disability, while another smaller group will have a steadily worsening disease that leads to increased disability over time. Most people with MS, however, will have short periods of symptoms followed by long stretches of relative relief, with partial or full recovery. There is no way to predict, at the beginning, how an individual persons disease will progress.

Researchers have spent decades trying to understand why some people get MS and others don't, and why some individuals with MS have symptoms that progress rapidly while others do not. How does the disease begin? Why is the course of MS so different from person to person? Is there anything we can do to prevent it? Can it be cured?

This brochure includes information about why MS develops, how it progresses, and what new therapies are being used to treat its symptoms and slow its progression. New treatments can reduce long-term disability for many people with MS. However, there are still no cures and no clear ways to prevent MS from developing.

Multiple sclerosis (MS) is a neuroinflammatory disease that affects myelin , a substance that makes up the membrane (called the myelin sheath) that wraps around nerve fibers (axons). Myelinated axons are commonly called white matter. Researchers have learned that MS also damages the nerve cell bodies, which are found in the brains gray matter, as well as the axons themselves in the brain, spinal cord, and optic nerve (the nerve that transmits visual information from the eye to the brain). As the disease progresses, the brains cortex shrinks (cortical atrophy).

The term multiple sclerosis refers to the distinctive areas of scar tissue (sclerosis or plaques) that are visible in the white matter of people who have MS. Plaques can be as small as a pinhead or as large as the size of a golf ball. Doctors can see these areas by examining the brain and spinal cord using a type of brain scan called magnetic resonance imaging (MRI).

While MS sometimes causes severe disability, it is only rarely fatal and most people with MS have a normal life expectancy.

Plaques, or lesions, are the result of an inflammatory process in the brain that causes immune system cells to attack myelin. The myelin sheath helps to speed nerve impulses traveling within the nervous system. Axons are also damaged in MS, although not as extensively, or as early in the disease, as myelin.

Under normal circumstances, cells of the immune system travel in and out of the brain patrolling for infectious agents (viruses, for example) or unhealthy cells. This is called the "surveillance" function of the immune system.

Surveillance cells usually won't spring into action unless they recognize an infectious agent or unhealthy cells. When they do, they produce substances to stop the infectious agent. If they encounter unhealthy cells, they either kill them directly or clean out the dying area and produce substances that promote healing and repair among the cells that are left.

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Multiple Sclerosis: Hope Through Research: National Institute of ...

NEW YORK & PETACH TIKVAH, Israel--(BUSINESS WIRE)--

BrainStorm Cell Therapeutics (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announced that it will initiate a pre-clinical study for Multiple Sclerosis (MS) at the Hebrew University Hadassah Medical Centers SPF-grade animal laboratory in Jerusalem. The study was approved by the Institutional Animal Care and Use Committee (IACUC) of the Hebrew University.

Based on promising pre-clinical data published by the Company's Chief Scientist, Prof. Daniel Offen of Tel Aviv University, BrainStorm will conduct further studies using the Experimental Autoimmune Encephalomyelitis (EAE) animal model to evaluate MS as an additional indication for its NurOwn technology. Professor Dimitrios Karussis and Dr. Ibrahim Kassis, who have published extensively on pre-clinical research using the EAE model, will be the Principal Investigators of the study.

Prof. Karussis, a Key Opinion Leader in the field of MS, is Head of the multi-disciplinary MS Clinic and Center at Hadassah, member of the European Steering Committee for Bone Marrow Transplantation in MS, member of the Executive Board and Scientific Committee of the European School of Neuroimmunology (ESNI), and former board member of the European Council and Committee for Treatment and Research in MS. He has been the Principal Investigator of several multi-national clinical trials in MS conducted by global pharmaceutical companies.

About NurOwn

NurOwn is an autologous, adult stem cell therapy technology that induces bone marrow-derived mesenchymal stem cells (MSC) to secrete high levels of neurotrophic factors for protection of existing motor neurons, promotion of motor neuron growth, and re-establishment of nerve-muscle interaction. More information about NurOwn can be found at http://brainstorm-cell.com/index.php/science-a-technology/-nurown.

About Multiple Sclerosis (MS)

Multiple sclerosis (MS) is believed to be an autoimmune disorder that affects the central nervous system (CNS). Autoimmune means that the bodys immune system mistakenly attacks its own tissue, in this case, the tissues of the CNS. With MS, autoimmune damage to neurons disrupts the bodys ability to send and receive signals, thus causing MS-related symptoms.Symptoms may vary due to the location and extent of the damage. Worldwide, MS may affect more than 2 million individuals, including approximately 400,000 people in the United States.

About BrainStorm Cell Therapeutics, Inc.

BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. For more information, visit the companys website at http://www.brainstorm-cell.com.

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BrainStorm to Initiate Study for Multiple Sclerosis at Hadassah Medical Center

"We want all of our supporters and event participants to know that the National MS Society is the very best investment to find MS solutions."

Instead of taking a country hike, do it by bike. The National Multiple Sclerosis Society: Louisiana will hold their 30th annual Bike MS: Louisiana on October 5-6.

The two-day, 150-mile ride takes place between Hammond, La., and McComb, Miss..

Multiple Sclerosis, or MS, is usually diagnosed between the ages of 20 and 50, although it also can affect children. Women are diagnosed two to three times more often than men. MS is a chronic disease that attacks the brain, spinal cord and optic nerve. As the disease attacks the central nervous system, it damages or destroys myelin and scar tissue (sclerosis) is formed. Each person has different symptoms of MS and a different degree of severity.

We want all of our supporters and event participants to know that the National MS Society is the very best investment to find MS solutions, said Rebecca Pennington, Louisiana vice president for development. We fund more MS research than any patient group in the world; have paved the way for 13 FDA-approved MS therapies; have led global efforts in gender, genetics and nervous system repair, including myelin repair and progressive MS; have utilized the best scientific review and management process; have set the standards in diagnosis, symptom management, stem cell research, clinical trial strategies, complementary and alternative medicine, pediatric MS and rehabilitation research; and have added more than 800 scientists to the MS research field.

Cyclists can accept the challenge and join the movement to eliminate MS. The NMSS: Louisiana has a goal of raising $800,000 through race sponsorships.

The statewide Louisiana Ride will begin and end at Southeastern Louisiana Universitys University Center. Breakfast and registration will begin at 6 a.m. on October 5, with the ride starting at 8 a.m. Rest stops will be located every 10 to 12 miles along the route and Support and Gear vehicles will be present between the stops. There will be a party and awards ceremony at Percy Quin State Park in McComb that night, with awards presented for Top Individual Fundraiser, Top Fundraising Team, Top Fundraising Average by a Team, Best Team Spirit, Best Decorated Team Tent and Best Team Jersey.

The registration fee for Bike MS: Louisiana is $40 per person, or $50 per person on race day. Cyclists must raise a minimum of $275 from sponsors. To register, go online to http://www.bikemslouisiana.org, or call 800.344.4867. Online registration will end Sept. 27 at 7 p.m. On-site registration can be done on Oct. 4 between 5-7 p.m., at the kick-off celebration, or on Oct. 5 from 6-8 a.m. Those who are ages 12-16 need a notarized waiver and parental supervision to participate.

The cycling event also needs volunteers to assist in both Hammond and McComb each day, as well as at rest stops along the cycling route. To volunteer, call 504.322.3779 for information or to sign up.

Transportation will be provided for those cyclists who decide to stay at a hotel in McComb and overnight storage will be provided for their bicycles. Transportation also will be available for those who only want to take part in one day of the event.

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Bike 150 miles in two days to raise money for Multiple Sclerosis research

Featured Article Main Category: Multiple Sclerosis Also Included In: Stem Cell Research Article Date: 17 Aug 2013 - 0:00 PDT

Current ratings for: Stem cell spine injections for MS - trial approved

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The US Food and Drug Administration (FDA) has approved a new clinical trial of a groundbreaking strategy using stem cells for the treatment of MS (multiple sclerosis).

Researchers from the Tisch MS Research Center of New York say the FDA has granted approval to begin early clinical investigation (phase 1 trial) of autologous neural stem cells in the treatment of MS.

Multiple sclerosis is a chronic disease that attacks the central nervous system (the spinal cord, optic nerves and brain). Common symptoms are numbness of the limbs, but more severe cases can lead to paralysis and blindness.

According to the Multiple Sclerosis Foundation, there are currently between 350,000 to 500,000 people in the US who have been diagnosed with MS, and 200 people are diagnosed with the disease every week.

The new regenerative strategy will involve using autologous, mesenchymal stem cell-derived neural progenitor cells (MSC-NPs), which will be harvested from the bone marrow of 20 MS patients who meet the criteria for the trial.

The stem cells will then be injected into the cerebrospinal fluid that surrounds the spinal cords of the patients.

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Stem cell spine injections for MS - trial approved

NEW YORK, Aug. 14, 2013 /PRNewswire/ --The Tisch MS Research Center of New York announced today that it has received Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to commence a Phase 1 trial using autologous neural stem cells in the treatment of multiple sclerosis (MS). MS is a chronic human autoimmune disease of the central nervous system that leads to myelin damage and neurodegeneration and affects approximately 2.1 million people worldwide.

"To my knowledge, this is the first FDA-approved stem cell trial in the United States to investigate direct injection of stem cells into the cerebrospinal fluid of MS patients, and represents an exciting advance in MS research and treatment," said Dr. Saud A. Sadiq, Senior Research Scientist at Tisch MS Research Center of New York and the study's principal investigator.

The groundbreaking study will investigate a regenerative strategy using stem cells harvested from the patient's own bone marrow. These stem cells will be injected intrathecally (into the cerebrospinal fluid surrounding the spinal cord) in 20 participants who meet the inclusion criteria for the trial. This will be an open label safety and tolerability study. All study activities will be conducted at the Tisch MS Research Center and affiliated International Multiple Sclerosis Management Practice (IMSMP).

The clinical application of autologous neural progenitors in MS is the culmination of a decade of stem cell research conducted by a dedicated team of scientists headed by Dr. Sadiq and by Dr. Violaine Harris, Research Scientist at Tisch MS Research Center.

Preclinical testing found that the injection of these cells may decrease brain inflammation and promote myelin repair and/or neuroprotection. "This study exemplifies the Tisch MS Research Center's dedication to translational research and provides a hope that established disability may be reversed in MS," Dr. Sadiq noted.

Participants will undergo a single bone marrow collection procedure, from which mesenchymal stem cell-derived neural progenitor cells (MSC-NPs) will be isolated, expanded and tested prior to injection.Participants will receive three rounds of injections at three month intervals. Safety and efficacy parameters will be evaluated in all participants through regular follow-up visits.

For more information on this study visit: http://www.tischms.org

ABOUT TISCH MS RESEARCH CENTER OF NEW YORK For over twenty years, Dr. Saud A. Sadiq has believed that combining excellence in clinical care with innovative research targeted at finding the cure for multiple sclerosis would set an exemplary standard in the treatment of MS patients. Today, the Tisch MS Research Center of New York embodies this new model of healthcare, in which your doctor is also your researcher. Dr. Sadiq helps those with MS by conducting cutting-edge, patient-based research to ensure unparalleled care. The close relationship of the non-profit research center and its affiliated clinical practice (International Multiple Sclerosis Management Practice) enables the testing of new MS treatments and accelerates the pace at which research discoveries move from lab bench to bedside. The Tisch MS Research Center of New York aims to identify the disease trigger, optimize treatments for patients, and repair the damage caused by multiple sclerosis.

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FDA Approves Stem Cell Clinical Trial For Multiple Sclerosis

Public release date: 18-Jul-2013 [ | E-mail | Share ]

Contact: Tara Womersley tara.womersley@ed.ac.uk 44-131-650-9836 University of Edinburgh

Research into multiple sclerosis and motor neurone disease is to be boosted with an international collaboration to further understanding of these illnesses.

Experts from the University of Edinburgh and the Massachusetts-based biotechnology company Biogen Idec will work together to seek greater insight into the cell processes behind these debilitating conditions.

This will include identifying drug compounds that could potentially be used as treatments.

The three-year collaboration will combine the University's expertise in translational medicine which develops laboratory discoveries into treatments for patients with Biogen Idec's strength in drug discovery and development.

Siddharthan Chandran, Professor of Neurology at the University of Edinburgh's College of Medicine and Veterinary Medicine, said: "This landmark partnership is a brilliant example of academic-industrial collaboration in the field of discovery science. Only by better understanding the biological processes behind these devastating diseases can we hope to discover new and effective therapies."

Clinicians and scientists, based at Edinburgh BioQuarter Scotland's flagship lifesciences project will be involved in the project, which will draw on the University's strength in neuroscience, stem cell research and regeneration.

The initiative is being funded by Biogen Idec, which is known for its strength in developing therapies for neurological disorders, particularly its portfolio of treatments for patients with multiple sclerosis.

"We have embraced academic collaborations as a part of our strategy to maintain a vibrant and innovative research organization and better understand the underlying biology of neurodegenerative disease. Our research partnership with the University of Edinburgh is an excellent example of this strategy," said Ken Rhodes, Vice President of Neurology Research at Biogen Idec. "We are committed to continuing to improve the treatment of people with MS and motor neuron diseases, and this collaboration is expected to provide an in-depth portrait of their pathophysiology, and identify important new targets for potential therapies."

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Transatlantic partnership to tackle neurodegenerative disease

Albany, New York (PRWEB) July 14, 2013

Multiple sclerosis (MS) is an inflammation of fatty myelin sheaths around the axons of the brain and spinal cord. MS tends to affect the ability of nerve cells to communicate with each other in the brain and spinal cord. MS affects people with random symptoms based on the location and severity of attack. One event can last for days, weeks or months and can affect any part of the body. Symptoms include imbalance, loss of motor function, spasms, numbness, incontinence, and difficulty in swallowing.

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There is no known cure for the disease currently and the only drugs available in the market are for delaying the progression of the disease. MS is estimated to affect 2.5 million people worldwide with around 5000 new cases developing each year. The National Multiple Sclerosis Society, Multiple Sclerosis Foundation, Multiple Sclerosis Association of America and several others are organizations which are determined to ensure improvement in the lives of affected people to make them more educated and spread awareness of this disease.

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Until the last year, the MS drugs market was led primarily by injectables, yet this trend is changing very quickly to give way to oral MS drugs which is expected to grow strongly beginning from 2013. Gilenya and Aubagio are the top current oral drugs in this segment and Biogens oral BG-12 and Teva Laquinimod are two other drugs in the pipeline for approval with a significantly higher efficacy to be launched in the next five years. Although oral MS drugs seem to be growing quickly, it is not expected that they would unsettle injectables anytime soon.

Several fatal side effects have been linked to use of Gilenya among those who have a heart disease and thus shows that there is still a long way to go for oral MS drugs. Top contenders in this segment till now are Copaxone, Avonex, Rebif, Betaseron, and Tysabri. Most of these drugs work actively to control MS relapses. Other treatments such as stem-cell transplantation are currently under investigation. The process includes the injection of stem cells which will replace the affected inflamed and diseased cells, greatly improving patients quality of life.

Browse Bolg : Pharmaceutical Market Research Reports http://pharmaceutical-market-reports.blogspot.com/

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include North America Asia Pacific Europe Rest of the World This report provides comprehensive analysis of Market growth drivers Factors limiting market growth Current market trends Market structure Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

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Multiple Sclerosis Drugs Market - New Industry Research Report is Now Available for Pre-Order at Transparency Market ...

The first technology innovations inspired by Cambridge researchers from a new European-leading bioscience campus in the UK have emerged.

The programme gives university researchers access to the drug development expertise of GSK and other pharmaceutical companies, while giving industry access to Cambridge research and know-how to accelerate the development of new medicines.

There are now two University research projects in place at SBC. Professor Peter McNaughton of the Department of Pharmacology is working on a novel new approach to the pain associated with heat.

Billions of dollars are spent each year on the treatment of pain, but there is currently no effective treatment for the extreme pain associated with hypersensitivity to heat.

Professor Robin Franklin of the Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute is developing a new regenerative therapy for multiple sclerosis (MS). MS affects almost 100,000 people in the UK, 400,000 in the United States and several million worldwide.

This is a groundbreaking approach to early-stage drug discovery, which is typically enormously time-consuming and expensive, said Professor McNaughton.

The exchange of scientific ideas and overall atmosphere of collaboration at SBC can help us as researchers, as well as our industrial colleagues, become more efficient in developing new ideas which will lead to better drugs and improved clinical treatments.

Cambridge is the first university to establish this type of arrangement. Recently, the university, in a bid led by University College London, was awarded a share of 50 million in funding from the Higher Education Funding Council for England (HEFCE) to enable the two universities to work together at SBC, in part to establish a range of collaborative training programmes to develop the next generation of entrepreneurial researchers, particularly in drug discovery.

The collaboration is directly supported by the National Institute for Health Research University College London Hospitals' Biomedical Research Centre, and will be further expanded through UCLPartners.

Professor Sir Leszek Borysiewicz, the Universitys Vice-Chancellor, said: Cambridge generates world-leading medical research and clinical insight, and in order to develop that research to the point where it can benefit patients, we work in partnership with industry.

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Revolution starts at bioscience campus

India, May 26 -- The All India Institute of Medical Sciences (AIIMS) is set to advance stem cell research for Multiple Sclerosis (MS), a disease that attacks the central nervous system.

"A proposal will soon be sent to the Stem Cell Ethics Committee at the hospital to conduct both epidemiological and therapeutic study using conventional drugs and studies related to stem cell therapy for the disease," said Dr Rohit Bhatia, additional professor of neurology at AIIMS.

A proposal for hematopoietic stem cell transplant has already been submitted for ethics clearance and for mesenchymal stem cell therapy. The proposal will be soon sent for clearance and funding.

"We have proposed a therapeutic study for multiple sclerosis which involves removing the cells with stem cells that repopulate the body to treat the abnormality due to expansion of autoreactive cells," said Dr Kameshwar Prasad, Professor of Neurology, AIIMS.

Though it is know that multiple sclerosis is common among people from temperate or colder climes, a study conducted by AIIMS showed that such cases were increasing in India as well.

According to the study, of 101 patients at the hospital between June 2011 to December 2012, it was proved that ratio of female patients to male was 6:4 - 61 female patients and 40 male. It was also proved that 30% of the patients were moderately disabled and about 60% had episodic relapses with the rest of the cases being progressive in nature.

The patients with extreme cases were between the age group 20-40.

Published by HT Syndication with permission from Hindustan Times.

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Advanced Sclerosis research at AIIMS soon

Researchers from the Stanford University School of Medicine discovered that blocking the expression of a single protein in the brains of mice with a form of multiple sclerosis can delay its effects, including paralysis.

The protein, SIRT1, helps to produce the cells that make the protective myelin coating needed to transmit nerve signals in the brain. Autoimmune diseases such as multiple sclerosis damage this coating, impeding communication between nerve cells.

"We are excited by the potential implications our study has on demyelinating diseases and injuries," said Anne Brunet, associate professor of genetics at Stanford and the senior author of the research. "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."

The process works by developing neural stem cells in the brain into another type of cell, called an oligodendrocyte precursors. Mature oligodendrocytes wrap neurons' arms with protective myelin, helping transmit electrical impulses from one cell to another.

Brunet and her team began by injecting a lab mouse with a drug called tamoxifen, which can effectively turn the SIRT1 gene off in neural stem cells.

Over time, nerve stem cells in which SIRT1 expression had been blocked began to make proteins that looked like typical oligodendrocyte precursor cells. The lack of SIRT1 activity increased the production of cells that express oligodendrocyte-specific protein makers.

When the researchers injected normal mice and those with blocked SIRT1 expression with a compound that causes the demyelination of nerve cells just as multiple sclerosis does, the SIRT1-blocked mice had quicker recoveries than the normal mice. They were also protected for a time from the paralysis that comes after the onset of multiple sclerosis.

"Our study highlights the possibility of pharmacological manipulation of multiple nodes of the pathway to expand the population of oligodendrocyte precursors," Brunet said. "Approaches such as these could have important implications for regenerative medicine."

The research was published online May 5 in Nature Cell Biology, a journal that publishes cell-related research and papers.

The research was supported by the California Institute for Regenerative Medicine, the National Institutes of Health, a grant from the American Federation for Aging Research, 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.

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Stanford: treatment delays multiple sclerosis onset in mice

April 15, 2013

Lawrence LeBlond for redOrbit.com Your Universe Online

The painful, debilitating symptoms associated with myelin disorders, such as multiple sclerosis and cerebral palsy, may one day be avoided if new breakthroughs in medical science have anything to say about it. One such breakthrough by a team of scientists at Case Western Reserve School of Medicine (CaseMed) has discovered a technique that directly converts skin cells into a type of brain cells destroyed in patients with these myelin disorders.

The breakthrough, revealed in a paper published in todays issue of the journal Nature Biotechnology, enables on demand production of myelinating cells, which insulate and protect neurons and also enables delivery of brain impulses to the body. In patients with multiple sclerosis, cerebral palsy and other similar disorders, these myelinating cells are destroyed and cannot be replaced.

In the research lab, Paul Tesar, PhD, assistant professor of genetics and genome sciences at CaseMed, and his colleagues used a new technique involving converting fibroblasts an abundant structural cell in the skin and most organs into oligodendrocytes, the type of cell responsible for myelinating the neurons of the brain.

This is alchemy on the cellular level, Tesar noted. We are taking a readily accessible and abundant cell and completely switching its identity to become a highly valuable cell for therapy.

Using cellular reprogramming, the team manipulated the levels of three naturally occurring proteins to induce fibroblast cells to become precursors of oligodendrocytes (oligodendrocyte progenitor cells, or OPCs). With the new method, the team was able to rapidly generate billions of these induced OPCs, and then show that they could regenerate new myelin coatings around nerves after being transplanted in mice.

Tesar and his colleagues, co-first authors Fadi Najm and Angela Lager, report that this new technique, effectively conducted in mice, could hopefully be used someday to treat human myelin disorders. Currently, cures require the myelin coating to be regenerated by replacement oligodendrocytes. But previously, OPCs and oligodendrocytes could only be obtained from fetal tissue or pluripotent stem cells, which were costly procedures and only offered limited benefits.

The myelin repair field has been hampered by an inability to rapidly generate safe and effective sources of functional oligodendrocytes, explained study co-author and myelin expert Robert Miller, PhD, professor of neurosciences at CaseMed and the universitys vice president for research. The new technique may overcome all of these issues by providing a rapid and streamlined way to directly generate functional myelin producing cells.

The next critical step in the research will be to effectively demonstrate the efficacy and safety of using human cells in the lab setting for myelin research. If the technique can prove successful, it will undoubtedly have widespread consequences for those suffering from debilitating myelin disorders. Therapeutic applications could be far-reaching.

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Turning Stem Cells Into Brain Cells To Cure Multiple Sclerosis And Cerebral Palsy

Public release date: 26-Mar-2013 [ | E-mail | Share ]

Contact: Anita Kar anita.kar@mcgill.ca 514-398-3376 McGill University

A new study by Multiple Sclerosis researchers at three leading Canadian centres addresses why bone marrow transplantation (BMT) has positive results in patients with particularly aggressive forms of MS. The transplantation treatment, which is performed as part of a clinical trial and carries potentially serious risks, virtually stops all new relapsing activity as observed upon clinical examination and brain MRI scans. The study reveals how the immune system changes as a result of the transplantation. Specifically, a sub-set of T cells in the immune system known as Th17 cells, have a substantially diminished function following the treatment. The finding to be published in the upcoming issue of Annals of Neurology and currently in the early online version, provides important insight into how and why BMT treatment works as well as how relapses may develop in MS.

"Our study examined why patients essentially stop having relapses and new brain lesions after the bone marrow transplant treatment, which involves ablative chemotherapy followed by stem cell transplantation using the patient's own cells," said Prof. Amit Bar-Or, the principle investigator of the study, who is a neurologist and MS researcher at The Montreal Neurological Institute and Hospital -The Neuro, McGill University, and Director of The Neuro's Experimental Therapeutics Program. "We discovered differences between the immune responses of these patients before and after treatment, which point to a particular type of immune response as the potential perpetrator of relapses in MS."

"Although the immune system that re-emerges in these patients from their stem cells is generally intact, we identified a selectively diminished capacity of their Th17 immune responses following therapy - which could explain the lack of new MS disease activity. In untreated patients, these Th17 cells may be particularly important in breaching the blood-brain-barrier, which normally protects the central nervous system. This interaction of Th17 cells with the blood-brain barrier can facilitate subsequent invasion of other immune cells such as Th1 cells, which are thought to also contribute to brain cell injury.

Twenty-four patients participated in the overall clinical trial as part of the 'Canadian MS BMT' clinical trial, coordinated by Drs. Mark Freedman and Harry Atkins at the Ottawa General Hospital. The new discovery, made in a subset of patients participating in the clinical trial, was based on immunological studies carried out jointly in laboratories at The Neuro and the Universit de Montral. Results of this study not only show the clinical benefits of BMT treatment, but also open a unique window into the immunological mechanisms underlying relapses in MS. Th17 cells could be the immune cells associated with the initiation of new relapsing disease activity in this group of patients with aggressive MS. This finding deepens our understanding of MS and could guide the development of personalized medicine with a more favourable risk/benefit profile.

Among the patients treated in the Canadian MS BMT clinical trial, was Dr. Alexander Normandin, a family doctor, who was a third- year McGill medical student getting ready for his surgery exams when he first learned he had MS, "I was so engrossed in my studies that I didn't pay attention to the first sign but within a few days of waking up with a numb temple, my face felt frozen. I learned that I had a very aggressive form of MS and would probably be in a wheelchair within a year. It was a brutal blow. I became patient #19 of only 24 for this experimental treatment. My immune system was knocked out and then rebooted with my stem cells. Today, my MS has stabilized. I now have this disease under control and I take it one day at a time."

###

Both the clinical and biological studies were supported by the Research Foundation of the Multiple Sclerosis Society of Canada. Link to study: http://onlinelibrary.wiley.com/doi/10.1002/ana.23784/abstract

Multiple Sclerosis

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Major advance in understanding risky but effective multiple sclerosis treatment

London, Feb. 9 (ANI): A patient's own skin could be used to repair the damage caused by multiple sclerosis, which is currently incurable, researchers have claimed.

A team of researchers at the University of Rochester Medical Centre used advances in stem-cell research to attempt to repair the myelin, a protein that nerves insulate themselves with, the BBC reported.

They took a human skin cell sample and converted it into stem cells -capable of becoming any other type of cell in the body.

The next step was to transform the stem cells into immature versions of brain cells, which produce myelin.

According to the researchers, when these cells were injected into mice born without any myelin it had a significant effect.

However, patients suffering from multiple sclerosis are still going to have the problem of their immune system attacking their myelin.

Another treatment would need to be used alongside other therapies for taming the immune system - or would need to be repeatedly performed.

The animal tests have been published in the journal Cell Stem Cell. (ANI)

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New skin cell treatment offers new hope to MS patients

Public release date: 7-Feb-2013 [ | E-mail | Share ]

Contact: Mark Michaud mark_michaud@urmc.rochester.edu 585-273-4790 University of Rochester Medical Center

A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

The study is the first successful attempt to employ human induced pluripotent stem cells (hiPSC) to produce a population of cells that are critical to neural signaling in the brain. In this instance, the researchers utilized cells crafted from human skin and transplanted them into animal models of myelin disease.

"This study strongly supports the utility of hiPSCs as a feasible and effective source of cells to treat myelin disorders," said University of Rochester Medical Center (URMC) neurologist Steven Goldman, M.D., Ph.D., lead author of the study. "In fact, it appears that cells derived from this source are at least as effective as those created using embryonic or tissue-specific stem cells."

The discovery opens the door to potential new treatments using hiPSC-derived cells for a range of neurological diseases characterized by the loss of a specific cell population in the central nervous system called myelin. Like the insulation found on electrical wires, myelin is a fatty tissue that ensheathes the connections between nerve cells and ensures the crisp transmission of signals from one cell to another. When myelin tissue is damaged, communication between cells can be disrupted or even lost.

The most common myelin disorder is multiple sclerosis, a condition in which the body's own immune system attacks and destroys myelin. The loss of myelin is also the hallmark of a family of serious and often fatal diseases known as pediatric leukodystrophies. While individually very rare, collectively several thousand children are born in the U.S. with some form of leukodystrophy every year.

The source of the myelin cells in the brain and spinal cord is cell type called the oligodendrocyte. Oligodendrocytes are, in turn, the offspring of another cell called the oligodendrocyte progenitor cell, or OPC. Myelin disorders have long been considered a potential target for cell-based therapies. Scientists have theorized that if healthy OPCs could be successfully transplanted into the diseased or injured brain, then these cells might be able to produce new oligodendrocytes capable of restoring lost myelin, thereby reversing the damage caused by these diseases.

However, several obstacles have thwarted scientists. One of the key challenges is that OPCs are a mature cell in the central nervous system and appear late in development.

"Compared to neurons, which are among the first cells formed in human development, there are more stages and many more steps required to create glial cells such as OPCs," said Goldman. "This process requires that we understand the basic biology and the normal development of these cells and then reproduce this precise sequence in the lab."

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Cells forged from human skin show promise in treating MS, myelin disorders

Feb. 7, 2013 A study out February 7 in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

The study is the first successful attempt to employ human induced pluripotent stem cells (hiPSC) to produce a population of cells that are critical to neural signaling in the brain. In this instance, the researchers utilized cells crafted from human skin and transplanted them into animal models of myelin disease.

"This study strongly supports the utility of hiPSCs as a feasible and effective source of cells to treat myelin disorders," said University of Rochester Medical Center (URMC) neurologist Steven Goldman, M.D., Ph.D., lead author of the study. "In fact, it appears that cells derived from this source are at least as effective as those created using embryonic or tissue-specific stem cells."

The discovery opens the door to potential new treatments using hiPSC-derived cells for a range of neurological diseases characterized by the loss of a specific cell population in the central nervous system called myelin. Like the insulation found on electrical wires, myelin is a fatty tissue that ensheathes the connections between nerve cells and ensures the crisp transmission of signals from one cell to another. When myelin tissue is damaged, communication between cells can be disrupted or even lost.

The most common myelin disorder is multiple sclerosis, a condition in which the body's own immune system attacks and destroys myelin. The loss of myelin is also the hallmark of a family of serious and often fatal diseases known as pediatric leukodystrophies. While individually very rare, collectively several thousand children are born in the U.S. with some form of leukodystrophy every year.

The source of the myelin cells in the brain and spinal cord is cell type called the oligodendrocyte. Oligodendrocytes are, in turn, the offspring of another cell called the oligodendrocyte progenitor cell, or OPC. Myelin disorders have long been considered a potential target for cell-based therapies. Scientists have theorized that if healthy OPCs could be successfully transplanted into the diseased or injured brain, then these cells might be able to produce new oligodendrocytes capable of restoring lost myelin, thereby reversing the damage caused by these diseases.

However, several obstacles have thwarted scientists. One of the key challenges is that OPCs are a mature cell in the central nervous system and appear late in development.

"Compared to neurons, which are among the first cells formed in human development, there are more stages and many more steps required to create glial cells such as OPCs," said Goldman. "This process requires that we understand the basic biology and the normal development of these cells and then reproduce this precise sequence in the lab."

Another challenge has been identifying the ideal source of these cells. Much of the research in the field has focused on cells derived from tissue-specific and embryonic stem cells. While research using these cells has yielded critical insight into the biology of stem cells, these sources are not considered ideal to meet demand once stem cell-based therapies become more common.

The discovery in 2007 that human skin cells could be "reprogrammed" to the point where they returned to a biological state equivalent of an embryonic stem cell, called induced pluripotent stem cells, represented a new path forward for scientists. Because these cells -- created by using the recipient's own skin -- would be a genetic match, the likelihood of rejection upon transplantation is significantly diminished. These cells also promised an abundant source of material from which to fashion the cells necessary for therapies.

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Cells forged from human skin show promise in treating multiple sclerosis, myelin disorders

British Israel Research and Academic Exchange Partnership (Birax) will grant NIS 25 million over five years for seven joint Israel-UK stem cell research programs.

Prime Minister Ehud Olmert and Prime Minister Gordon Brown launched Birax in 2008 through the Ministry of Science and Technology and the British Council. The British Embassy in Israel and the UK-Israel Life Sciences Council decided that Birax would focus on regenerative medicine.

Israel and the UK are global biotechnology leaders, with expertise in stem cells. Stem cells are cells which have not completed their speciation and have unique reproductive, renewal, and regenerative capabilities for organs. The ultimate dream is to use stem cells to build new tissue and organs, but in the meantime, stem cells are being developed for drug delivery and tissue regeneration. The first stem cell-based drugs were approved in 2012.

Under Birax, joint projects have already been established by Cambridge University, Oxford University, the University of Edinburgh, and the University of Nottingham in the UK and the Weizmann Institute of Science and the Hebrew University of Jerusalem. The projects are in multiple sclerosis, Parkinson's disease, and type 1 diabetes, as well as basic research on ways to prevent the immune system from attacking stem cells.

The grants will be financed by the Israeli and UK governments, and Britain's Pears Foundation, Britain's Bonita Trust, the Charles Wolfson Charitable Trust, the Rosetrees Trust, Lord Fink, the United Jewish Israel Appeal (UJIA), the Rothschild family's Yad Hanadiv, Morris Kahn's Aurum Ventures MKI Ltd.

The British Embassy also supports the life sciences through the UK-Israel Tech Hub. The Hub Biomed Manager Dr. Iris Geffen Gloor is becoming a major power in brokering Israeli-UK academic and business ties. The Hub's recent important projects include creating access to binational foundations, establishing ties between academic institutions and new companies and British pharmaceutical commercialization companies, and establishing ties between Israeli parties and the UK's National Organization for Clinical Research Infrastructure (NOCRI), a powerhouse for clinical studies.

Published by Globes [online], Israel business news - http://www.globes-online.com - on January 20, 2013

Copyright of Globes Publisher Itonut (1983) Ltd. 2013

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Birax grants NIS 25m for Israel-UK stem cell projects

Jan. 9, 2013 After uncovering a mechanism that promotes chronic intestinal inflammation and the development of colorectal cancer, scientists from Virginia Commonwealth University Massey Cancer Center have found that fingolimod, a drug currently approved for the treatment of multiple sclerosis, could potentially eliminate or reduce the progression of colitis-associated cancer (CAC).

The study, published online in the journal Cancer Cell, was led by Sarah Spiegel, Ph.D., Mann T. and Sara D. Lowry Chair in Oncology, co-leader of the Cancer Cell Signaling program at VCU Massey Cancer Center and chair of the Biochemistry and Molecular Biology Department at the VCU School of Medicine. Spiegel's team discovered that increased production of an enzyme known as sphingosine kinase 1 (SphK1) causes cells lining the intestine to produce more of a signaling molecule known as sphingosine-1-phosphate (S1P), which activates a variety of biological mechanisms that lead to chronic intestinal inflammation and the development and progression of CAC. The researchers then used animal models to demonstrate that the drug fingolimod decreased expression of SphK1 and S1P's receptor, S1PR1, which subsequently interfered with the development and progression of CAC, even after tumors were established.

"Perhaps the most significant aspect of this study is the therapeutic potential of fingolimod in the treatment of colitis-associated cancer," says Spiegel. "Since this drug is already approved for clinical use, we're hoping to initiate a clinical trial to study its efficacy in patients with CAC in combination with approved therapies."

Essentially, the researchers discovered a self-feeding loop that results in chronic intestinal inflammation and increases the progression of CAC. The team showed that increased production of SphK1 and S1P lead to sustained activation of NF-kB and Stat3, which are both proteins called transcription factors that control the way DNA is transcribed in a cell's nucleus in order to respond to environmental stimuli. This increased activation of NF-kB and Stat3 led to an increased production of TNF-a and IL-6, which are small pro-inflammatory molecules secreted by immune system cells. The increased inflammation, in turn, led to increased production of SphK1 and S1P, which continued the malicious cycle.

This is the first time that SphK1 and S1P have been linked to NF-kB, Stat3, chronic inflammation and CAC.

"Because one of the consequences of inflammatory bowel diseases is an increased risk of developing colorectal cancer, the next step in our research is to examine blood samples from patients with irritable bowel syndrome and colitis-associated cancer to measure levels of S1P," says Spiegel. "Colorectal cancer is one of the leading causes of cancer-related deaths, and we're hopeful that this research will lead to more effective treatments."

Spiegel collaborated on this study with Kazuaki Takabe, M.D., Ph.D., and Tomasz Kordula, Ph.D., both members of the Cancer Cell Signaling program at VCU Massey; and Jie Liang; Masayuki Nagahashi, M.D., Ph.D.; Eugene Y. Kim, Ph.D.; Kuzhuvelil B. Harikumar, Ph.D.; Akimitsu Yamada, M.D.; Wei-Ching Huang; Nitai C. Hait, Ph.D.; Jeremy C. Allegood, Ph.D.; Megan M. Price; Dorit Avni, Ph.D.; and Sheldon Milstien, Ph.D., all from VCU Massey Cancer Center and the Department of Biochemistry and Molecular Biology at VCU School of Medicine.

This study was supported by NIH grants R37GM043880, RO1CA61774, U19AIO77435, T32HL094290, P30CA16059 and K12HD055881, a Susan G. Komen for the Cure Research Foundation grant and National Institute of Neurological Disorders and Stroke core grant 5P30NS047463.

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Multiple sclerosis drug may one day treat colorectal cancer

Cecelia Johnson was an artist, cellist, tap dancer, and 22-year-old college student when she was diagnosed with multiple sclerosis in 2001. The disease, in which the bodys immune system attacks the tissue protecting nerves, proceeds at its own pace: Sometimes the deterioration is halting, sometimes it can be delayed, but there is no cure. Johnsons decline was swift.

Six years after her diagnosis, undone by fatigue and pain and often unable to walk, Johnson gave up on conventional medicine. In the spring of 2007 she traveled from her home in Houston to Mexico, where an American doctor gave her an infusion of adult stem cells that were supposed to regenerate her damaged tissue. I thought this guy might be peddling snake oil, says Johnson. But I would have taken snake oil. The procedure cost her $14,000.

Within a few months, she began to feel better. The effects werent lasting, though, and she returned to Mexico every year until the Federal Bureau of Investigation arrested her doctor in December2011. Francisco Morales was accused of conspiracy and fraud: He wasnt a licensed doctor, and he was using umbilical-cord stem cells he bought in the U.S. for treatments that the Food and Drug Administration had not approved. He pleaded guilty in September 2012 and awaits sentencing.

By then, Johnson had learned she could receive infusions of her own stem cells in Houston. An orthopedic surgeon there, Stanley Jones, had recently co-founded Celltex Therapeutics, a company that multiplied and stored adult stem cells. It took Johnson and her mother much of the spring and summer of 2012 to raise the $30,000 fee for the treatment, which was part of a clinical study. A study I have to pay $30,000 for? Sure, Im skeptical, says Johnson. The point is that stem cells are available, I desperately need them, and I will pay for them. In August, Johnson had several hundred thousand stem cells harvested from her abdominal fat.

Photograph by Thomas Prior for Bloomberg BusinessweekEller and Jones founded Celltex in 2011

Jones was not just a doctor, he was also a satisfied customer. He had been treated for autoimmune arthritis with his own adult stem cells through a South Korean company, RNLBio. In March2011, he and Houston businessman David Eller founded Celltex, one of the first commercial stem cell laboratories in the country. They had RNLs technology and eventually some 200 paying patients desperate for relief. One of them was Texas Governor Rick Perry, who suffered from back problems. Together they encouraged the state medical authority to let doctors provide stem cell treatments under its supervision.

Then the FDA got involved: The agency inspected Celltexs lab, found 14 major manufacturing problems, and later warned the company it was illegally marketing an unlicensed drug. Celltex shut down the lab in early October 2012, four days before Johnson was to receive her first batch of cells. It hasnt yet resumed processing stem cells for Johnson or anyone else. In a December letter to patients the company stated: Celltex remains fully committed to advance the most promising new field in human health in decadesregenerative medicine.

Celltexs venture raises some of the most vexing, emotional issues in the business of medicine. Stem cells hold enormous promise, but promise isnt proof, and anecdotal evidence isnt science. Small companies often cant do the research required by the FDA and make money at the same time. Some patients will pay to be part of an experiment, but many doctors and regulators dont think they should. In Texas the science of stem cells has collided with a governors ambitions, a businessmans optimism, a doctors faith, and patients hopes. It seemed too good to be true, Johnson says, and it was. Stem cells, often thought of as the bodys master cells, help form and repair tissue, organs, and blood. There are different types of stem cells, each with their own capabilities. Embryonic stem cells, potentially the most powerful, are the most controversial; George W. Bush restricted federal funding for embryonic stem cell research when he was in office. Induced pluripotent stem cells are adult cells that have been genetically reprogrammed to have some attributes of embryonic stem cells. Adult stem cells are believed to exist in tissue throughout the body. The main function of mesenchymal adult stem cells, the type Celltex works with, is to repair tissue damaged by daily use. They also have anti-inflammatory properties. The cells can be found in special niches in bone marrow, umbilical-cord blood, muscle, and fat. When the body is injured, the cells leave their niche and become more specialized, but they are not, like embryonic stem cells, able to transform into any kind of cell. A blood-forming cell can become a red blood cell; it cant become a brain cell.

Dr. Gary D. Gaugler/PhototakeA blood-forming adult stem cell, found in bone marrow

Hundreds of clinical studies are under way in the U.S. to test the safety and efficacy of stem cells for all sorts of disorders: Alzheimers, Crohns, Epstein-Barr, lymphoma, diabetes, multiple sclerosis, infertility. Scientists say stem cells could help repair the heart or spine, regenerate cartilage, and improve brain function after a stroke. Were formed from stem cells, everything about our body is a stem cell product, says James Willerson, president of the Texas Heart Institute, where about half of the research conducted involves stem cells. I believe the right cells in the right place in the right person will do amazing things. Arnold Caplan, a professor of biology at Case Western Reserve University and founder of two stem cell companies, says: It sounds like stem cells could be the magic elixir for every malady. The answer is that, on a scientific basis, they could be.

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Stem Cell Showdown: Celltex vs. the FDA

SAN DIEGO--(BUSINESS WIRE)--

ACADIA Pharmaceuticals Inc. (ACAD), a biopharmaceutical company focused on innovative treatments that address unmet medical needs in neurological and related central nervous system disorders, today announced that it will receive funding from Fast Forward, LLC, a not-for-profit organization established by the National Multiple Sclerosis Society, and EMD Serono, a subsidiary of Merck KGaA, Darmstadt, Germany. This funding will support ACADIA research, to be conducted in collaboration with Dr. Rhonda Voskuhl of UCLA, directed at using AC-186, ACADIAs proprietary and selective estrogen receptor (ER)-beta agonist, as a new approach to the treatment of multiple sclerosis (MS).

We are grateful for the commitment by Fast Forward and EMD Serono, which will enable us to expand on promising research in our ER-beta program and broaden its application to MS, said Uli Hacksell, Ph.D., ACADIAs Chief Executive Officer. We also are excited to collaborate on this research with Dr. Voskuhl, Professor and Program Director at the UCLA Department of Neurology, who is a recognized expert in MS and neuroprotection.

Currently, there are multiple immunology-based, disease-modifying drugs approved for the treatment of relapsing forms of MS. In contrast, no drug is currently approved for the treatment of progressive forms of MS and no currently available drugs were developed to specifically target neurodegeneration in MS. A new MS drug with neuroprotective properties would fill this major unmet medical need. Studies in animal models of MS suggest that selective ER-beta receptor agonists provide neuroprotective effects while avoiding stimulation of ER-alpha receptors, which are believed to mediate toxicity. In this new program, AC-186, a selective ER-beta agonist discovered by ACADIA, will be evaluated to further test this hypothesis.

Fast Forward and EMD Serono will provide up to $545,000 to support preclinical studies with AC-186 designed to further evaluate pharmacokinetics and its therapeutic potential in preclinical models of MS. This funding will be provided by the parties Accelerating Commercial Development Fund, which is allocated to for-profit entities and is designed to accelerate the development of research discoveries into new or improved therapies for people with MS.

We are pleased to partner with ACADIA and UCLA on this innovative approach to targeting MS neurodegeneration, said Dr. Timothy Coetzee, Chief Research Officer at the National MS Society and Fast Forward. This is another example of our steadfast commitment to seek out and support promising new therapeutic approaches that address critical unmet needs and could improve the lives of patients with MS.

About Multiple Sclerosis

Multiple sclerosis (MS) is a chronic, inflammatory condition of the central nervous system and is the most common, non-traumatic, disabling neurological disease in young adults. It is estimated that approximately two million people have MS worldwide. While symptoms can vary, the most common symptoms of MS include blurred vision, numbness or tingling in the limbs and problems with strength and coordination. The relapsing forms of MS are the most common.

About Fast Forward, LLC

Fast Forward, LLC, established by the National Multiple Sclerosis Society as part of a comprehensive approach to MS research and treatment, focuses on speeding promising research discoveries towards commercial drug development. Fast Forward accelerates the development of treatments for MS by connecting university-based MS research with private-sector drug development and by funding small biotechnology/pharmaceutical companies to develop innovative new MS therapies and repurpose FDA-approved drugs as new treatments for MS. For more information, please visit http://www.fastforward.org.

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ACADIA Pharmaceuticals to Receive Funding from Fast Forward and EMD Serono for Multiple Sclerosis Program

ROCKLAND, Mass. and NEW YORK, Dec. 20, 2012 /PRNewswire/ --EMD Serono, Inc., a subsidiary of Merck KGaA, Darmstadt, Germany, and Fast Forward LLC, a wholly-owned subsidiary of the National Multiple Sclerosis Society, today announced the third group of recipients to receive funding through their collaboration, which is designed to accelerate innovation and commercial development of multiple sclerosis (MS) therapies.

(Logo: http://photos.prnewswire.com/prnh/20121220/SF32295LOGO-a)

(Logo: http://photos.prnewswire.com/prnh/20121220/SF32295LOGO-b)

The awards total approximately $1.4 million and will be distributed from the Accelerating Commercial Development Fund created by EMD Serono and Fast Forward to encourage early-stage drug discovery for MS. The Accelerating Commercial Development Fund is open to early-stage for-profit commercial organizations that have achieved Series A or comparable investment funding. The other fund in the collaboration, the Accelerating Innovation Fund, is open to academic institutions, non-profit research organizations, and seed-stage for-profit commercial organizations.

EMD Serono and Fast Forward distributed a call for proposals to fund projects directed towards the development of therapies to prevent, treat or reverse nervous system damage in MS. These priority research areas were determined by a joint steering committee comprised of Fast Forward staff and representatives from EMD Serono and Merck KGaA.

The following organizations will receive funding:

Under the Accelerating Commercial Development Program:

"We are pleased to announce the 2012 funding recipients whose work has the potential to broaden our knowledge and understanding of MS, and hopefully, result in new treatment options for people living with this disease," said Bernhard Kirschbaum, PhD, Executive Vice President, Global Research and Development at Merck Serono, a division of Merck KGaA, Darmstadt, Germany. "Our ongoing collaboration with Fast Forward reflects our sustained commitment to leveraging internal as well as external expertise in furthering scientific excellence in MS."

EMD Serono and Fast Forward entered into an initial two-year, worldwide agreement in March 2009, and recently extended the collaboration. As part of the up to $19 million collaborative agreement with Fast Forward, EMD Serono provides the majority of funding for the research awards, with Fast Forward contributing 10 percent of the total financing of the awards disseminated from each of the two funds.

"Advancing new treatments for people with MS requires continuing research and discovery in order to find new and better treatments," said Dr. Timothy Coetzee, Chief Research Officer at the National MS Society and Fast Forward. "We are pleased to have the opportunity to advance research through the continued collaboration between Fast Forward and EMD Serono. We remain committed to being a driving force of research and treatment options to stop MS, restore function, and end MS forever, and we look forward to learning more from the results of these innovative projects."

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EMD Serono, Inc. and Fast Forward, LLC Announce Recipients of Funding for Multiple Sclerosis Research