#Authentic?A #New #ALS #MND #Medication: #MicroNeurotrophins - - TopicsExpress



          

#Authentic?A #New #ALS #MND #Medication: #MicroNeurotrophins - #Help bring a promising ALS/MND #medication to a #Phase 1 #Human #Clinical #Trial in just 12 to 18 months! It takes nearly 15 YEARS to bring just ONE new drug to market...ALS/MND patients cant wait 15 years! Every 90 minutes ALS/MND claims another life. There is currently no cure or effective treatment for ALS/MND. We need to transform the way scientific research is funded to speed up the process. Thats why ALS Worldwide, a nonprofit organization based in the United States, has launched this grassroots fundraising campaign to give the global community the opportunity to accelerate the preclinical testing of a new and promising ALS/MND drug compound called MicroNeurotrophins. Several prestigious universities have enthusiastically joined an international consortium convened by ALS Worldwide to study MicroNeurotrophins and quickly bring the molecules to an Investigational New Drug (IND) Trial in just 12-18 months. The ALS MicroNeurotrophin Research Consortium includes Harvard University/Massachusetts General Hospital; Virginia Commonwealth University; Penn State Hershey; The University of Sheffield’s Institute for Translational Neuroscience (SITraN) UK and the University of Crete. Experts from these leading universities believe that this ALS/MND drug is cutting edge. It could have a massive affect on ALS/MND patients - and it may well be able to help people with other neurodegenerative diseases too. The Ice Bucket Challenge showed us that people want to contribute directly to research. You want to see your money actually making a difference in treatments available to patients. There’s only one FDA-approved option right now, and it only extends life by a couple of months. TheCrowdfunding Campaign for a New ALS Medication” is a way for you to contribute directly to bring a specific treatment—vetted by experts and supported by a consortium of research teams—into a clinical trial in just 12 to 18 months. This is a concrete way for you to make a difference.Lets fund an ALS/MND drug that could save thousands of lives! About MicroNeurotrophins Developed by Dr. Achilleas Gravanis, Professor of Pharmacology at University of Crete’s School of Medicine, MicroNeurotrophins appear to mimic the beneficial properties of neurotrophins—naturally occurring proteins that help motor neurons develop, grow, function, signal, and even repair themselves—but are also small enough to pass through the blood brain barrier and reach the motor neurons affected by ALS/MND. By contrast, ALS causes motor neurons to degenerate and die and ALS/MND patients experience a profound loss of neurotrophins as the disease progresses. MicroNeurotrophins have proven highly effective and non-toxic in experimental animal models of three major human neurodegenerative diseases: Multiple Sclerosis, Parkinson’s disease and Alzheimer’s disease. This initial research indicates that MicroNeurotrophins hold great promise for treating ALS/MND because they target the multiple pathways of motor neuron degeneration that are characteristic of the disease. How do MicroNeurotrophins address the multiple pathways of ALS/MND? Anti-Apoptotic Apoptosis, programmed cell death, is responsible for motor neuron degeneration in ALS/MND. In both in vitro (cell culture) and in vivo (animal model) studies, MicroNeurotrophins have been shown to combat apoptosis by protecting and strengthening neurons. Anti-Inflammatory and Autoimmune Inhibitor Inflammation is the immune system’s defense against bacteria, viruses, and substances that are foreign and harmful. However, there is increasing evidence that inflammation accompanies the death of motor neurons in ALS/MND. MicroNeurotrophins’ powerful anti-inflammatory properties may help reduce, prevent, or even reverse this destructive autoimmune response. Glutamate Modulator ALS/MND patients are unable to properly regulate glutamate, a chemical that carries messages throughout the brain and spinal cord. Too much glutamate in the brain can be toxic and cause motor neurons to die. MicroNeurotrophins serve as an indirect protectant against excess glutamate through their ability to promote cell survival processes, increase neuronal signaling strength and as an autoimmune modulator. Neuronal Signaling In ALS/MND, loss of neurotrophic signaling causes neurons to die. MicroNeurotrophins prevent cell death by increasing neuroprotective signals that help neurons survive, differentiate, and grow. What We Need It will cost $4.5 million dollars to bring this medication to human clinical trial in just 12-18 months. ALS Worldwide has already raised $600,000 and is leading a fundraising effort to raise the additional $3.9 million needed for the FDA and EMA required efficacy and safety studies. Participating universities have generously agreed to waive their facilities and administrative fees to show their shared commitment to the research effort and to reduce the total project budget. By doing so, the partnering institutions have collectively contributed roughly $1.3 million to the overall fundraising campaign. We are asking the global community to help us raise $1.4 million in the next 2 months. That’s a little more than one-third of what’s needed. At the same time, we will continue to pursue traditional philanthropy and institutional grants to reach our overall funding goal of $3.9 million. 100% of all donations will go directly to the efforts of the ALS MicroNeurotrophin Research Consortium. Contribute today. Please donate whatever amount you can most afford. Share, Share Share! Please share our campaign through email and on Facebook and Twitter (Check out Indiegogos easy to use share tools above) Be a Fundraiser! Please ask your family, friends, and co-workers to donate and spread the word about our Indiegogo campaign. Stay connected. Check back often as our story and campaign unfolds! Together, we can make something truly historic happen! What You Get This grassroots fundraising campaign is led by ALS Worldwide, a small nonprofit organization based in the United States, so there is no fancy website, no motivational wristband and no rock concert. We have lined up some amazing perks from having your name published in the research papers to an invitation to one of the consortium partners labs to meet the scientists and receive an exclusive tour and presentation. Everyone who donates will receive the benefit of knowing youre helping to bring us one step closer to an effective ALS/MND treatment. About the Consortium Stephen and Barbara Byer co-founded ALS Worldwide in 2008 to honor the memory of their son Ben who died that year from ALS/MND at the age of 37. In 2013, their daughter, Sarah Byer, and her husband Barry Wein, whose grandfather also died from ALS/MND, joined the nonprofit organization to contribute their professional expertise and personal experience to ALS Worldwide’s mission. Today, ALS Worldwide advances scientific breakthroughs by fostering collaboration and innovation among leaders from all sectors of medical research. ALS Worldwide provides free personalized support via videoconference, email, phone and in-person visits to ALS/MND patients and their loved ones in more than 85 countries. To learn more, please visit alsworldwide.org or connect with us on Facebook and Twitter. Dr. Achilleas Gravanis After rigorous testing in my laboratory, our team discovered that MicroNeurotrophins hold great promise for treating ALS because they target the multiple pathways of motor neuron degeneration that are characteristic of the disease. However, without scientific collaborators with expertise in ALS, I knew that my discovery would never advance to a clinical trial. That’s why I am grateful to ALS Worldwide for assembling the consortium and leading the fundraising effort to underwrite our collective research. -- Dr. Achilleas Gravanis, November 2014 Dr. Achilleas Gravanis, Professor of Pharmacology at University of Crete’s School of Medicine will continue his work as principal investigator of MicroNeurotrophins, leading his team of scientists while providing technical assistance to the other members of the consortium. Dr. Gravanis has served as vice-president of the Hellenic Pharmacological Society, the Hellenic Biochemical Society, and as a member of the steering committee of the European Pharmacology Network. He is a member of the Board of the Hellenic Quality Assurance Agency for Higher Education and serves as the Chairman of the Biosciences Committee of the Hellenic Research and Technology Council. He is a member of the Hellenic Council of Public Health. Dr. Gravanis has published 120 papers in peer-reviewed journals, cited in PubMed. He is the co-founder of Bionature, established in 2003, as a spin-off of the University of Crete. Dr. Ghazaleh Sadri-Vakili I believe that MicroNeurotrophins are one of several new exciting avenues in ALS research. Specifically, MicroNeurotrophins show considerable promise as they readily enter the brain and target multiple pathogenic pathways that lead to neuronal death in ALS/MND. I am excited to be a part of the ALS MicroNeurotrophin Research Consortium, which includes my colleagues at Harvard University and teams from Virginia Commonwealth University, Penn State Hershey, The University of Sheffield’s Institute for Translational Neuroscience (SITraN), and University of Crete. The Consortium provides us with a rare and wonderful opportunity to work closely with other experts from all across the world, each with numerous years of experience. It is an honor to be a part of this dynamic team working collaboratively in order to determine the therapeutic potential of MicroNeurotrophins. I am grateful to ALS Worldwide for assembling the Consortium and leading the fundraising effort to underwrite our collective research. Dr. Ghazaleh Sadri-Vakili, the Director of the NeuroEpigenetics Laboratory at Massachusetts General Hospital and Assistant Professor of Neurology at Harvard Medical School will oversee the testing of MicroNeurotrophins using an ALS/MND mouse model. Dr. James Bennett Jr., Bemiss Professor of Neurology, Psychiatry and Physiology/Biophysics at Virginia Commonwealth University’s School of Medicine will lead the testing of MicroNeurotrophins in stem cells genetically modified and used to generate motor neurons in a dish. Professor Dame Pamela Shaw, Professor of Neurology at the University of Sheffield and Director of the Sheffield Institute for Translational Neurosciences (SITraN), one of the world leading centers for ALS/MND research, will oversee testing of MicroNeurotrophins in both ALS/MND mouse and zebrafish animal models. Glenn S. Gerhard, M.D. is a Professor in the Departments of Biochemistry and Molecular Biology and Pathology and Laboratory Medicine at the Penn State College of Medicine and Co-Director of the Institute for Personalized Medicine. Professor Gerhard will oversee testing of MicroNeurotrophins in ALS/MND zebrafish animal models and in tissue samples from ALS/MND patients. About ALS/MND In the United States, Amyotrophic Lateral Sclerosis (ALS) is commonly known as Lou Gehrig’s disease, named after the legendary Yankees baseball player who died from ALS in 1941. In the United Kingdom and other parts of the world, ALS is often called Motor Neurone Disease (MND). ALS/MND is a neurodegenerative disease that causes the neurons in the brain, brain stem and spinal cord to degenerate and die, which leads to muscle weakness, paralysis, and death. ALS can strike anyone and is typically fatal within 2-5 years of diagnosis, but about 10 percent of individuals survive for 10 or more years.
Posted on: Tue, 18 Nov 2014 00:42:20 +0000

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