The ALS Therapy Development Institute Partners with Neurotune to Investigate Potential Treatments for MND

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CAMBRIDGE, Mass. and SCHLIEREN-ZURICH, Switzerland. Partnership Aimed at Neuromuscular Junction Strength against Disease Course The ALS Therapy Development Institute (ALS TDI) announced today that it has entered into collaboration with Neurotune to investigate a potential treatment for ALS (aka Motor Neuron or Lou Gehrig’s disease). “Maintaining the health of motor neurons and their connections to muscles will be central in the effort to combat ALS. We are very pleased to be partnered with Neurotune on this important project and are eager for results,” said Steve Perrin, Ph.D., CEO & CSO of ALS TDI. In ALS, motor neurons and their axons become disconnected from the muscle. Maintaining connectivity at this “neuromuscular junction” is crucial to a person’s ability to freely move, eat and breathe independently, all functions that are gradually lost as ALS progresses. Neurotune has developed a novel class of compounds to maintain neuromuscular junction strength and stability. Under the terms of the agreement, ALS TDI will use one of those compounds in a preclinical model of ALS to determine if the treatment has an effect on disease course. “The collaboration with ALS TDI will allow Neurotune to have one of its most promising compounds developed for neuromuscular diseases tested in the ALS disease model. This might open new approaches in the treatment of ALS,” emphasizes Armin Mader, Ph.D., CEO of Neurotune.

 

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Neuralstem Updates ALS Stem Cell Trial Progress – FDA Permits Additional Dosing of Return Patients

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Neuralstem, Inc. announced that the Federal Drug Administration (FDA) has approved the return of three patients from earlier cohorts in its ongoing Phase I safety trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells (HSSC’s). These patients will be permitted to return to the trial for second treatments as the next cohort of patients, provided they meet inclusion requirements at the scheduled time. They will be the first to receive stem cell transplantation along the length of the spinal cord. The first twelve patients in the trial, which is taking place at Emory University Hospital in Atlanta, Georgia, received stem cell transplants in the lumbar (lower back) region of the spinal cord only. The last cohort of three, completed in April, received transplants in the cervical (upper back) region of the spinal cord, where stem cell transplantation could help support breathing, a key function that is lost as ALS progresses. The next cohort of three patients is designed to receive 10 HSSC injections in the lumbar region and 5 in the cervical, for a total of 15 injections along the length of the spinal cord. In the case of the returning patients, who have already received 10 lumbar injections, they will receive five cervical injections. These patients are between 15-17 months out from their first dosing and appear to have tolerated the first procedure well. Additionally, Neuralstem has submitted a trial amendment to the FDA to increase both the number of patients treated as well as the dose in future cohorts. The amendment would also expand the trial to include certain efficacy endpoints. The trial was initially designed as a safety trial to treat 18 patients. “The return of these patients to the trial for second treatments is a continuing validation of the trial’s safety. Typically, Phase I trials do not bring study subjects back, as that could increase their exposure to potentially harmful treatments,” said Karl Johe, PhD, Neuralstem Chairman and Chief Scientific Officer. “Treating these patients who have already received injections in one part of their spine allows us to both increase the overall dosage for each patient as well as transplant them in regions of the spine where they have not been treated,” Dr. Johe continued. “This next cohort of patients will be the first in the world to receive stem cell transplants in both cervical and lumbar regions of their spinal cord. With cervical injections of the lumbar patients, for example, we could also potentially support their breathing function, which is vital for preserving quality of life.”

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ALS clinics start implanting breathing-assist device under new FDA approval

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Cedars-Sinai Medical Center has become the first West Coast site — and one of only three nationwide — to implant a device that stimulates the respiratory muscle in the chest and draws air into the lungs of patients suffering from amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease) under recently approved Food and Drug Administration guidelines. The progressive disease attacks neurons in the brain and spinal cord that control muscles throughout the body; most patients with ALS die from lung complications and respiratory failure. “We currently have no cure for ALS, which often causes patients to become completely paralyzed in the later stages, but this respiratory-assist system is one way we can attempt to improve our patients’ quality of life, helping them remain comfortable and possibly delaying the need for a ventilator,” said Dr. Robert H. Baloh, director of the Neuromuscular Division in the Department of Neurology at Cedars-Sinai Medical Center and a top ALS clinician and research scientist.

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New research may help explain why there are so few ALS treatments available.

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Despite hundreds of potential targets and nearly as many trials, only one drug–riluzole–remains FDA-approved for treating ALS. The array of treatment approaches combined with their promise in pre-clinical trials has made researchers ask what might be hindering their progress. Scientists have identified one of the hurdles that may make so many drugs ineffective against ALS and other neurological disorders. Some of the proteins in the protective blood-brain barrier that act as de facto bouncers by pumping foreign chemicals out of the cell are over-active in ALS patients, according to a new study published this month in Neurobiology of Disease. This increased activity removes potential therapeutic drugs out of the central nervous system before they have a chance to work. Developing a way to inhibit this process may one day improve the efficacy of potential ALS drugs. “We identified two particular drug efflux transporters that are upregulated in the spinal cord in ALS. The increased activity of these transporters could potentially affect the bioavailability and efficacy of ALS-treating compounds,” said Mike Jablonski, a neuroscience PhD student working in the laboratory of Davide Trotti at Thomas Jefferson University in Philadelphia, and first author of the study.

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ALS Untangled uses social networking to bring the #ALS community together.

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Richard Bedlack wanted to give his patients answers. As a Packard scientist and the director of the ALS Clinic at Duke University, his patients frequently asked him about a variety of treatments for their disease. For some of these treatments, like riluzole, Bedlack could respond swiftly and accurately. For a growing number of treatments about which patients were asking, however, Bedlack could only answer, “I don’t know.”

So Bedlack did what every good physician does: he turned to the clinical research literature and to other online data. The process was lengthy and time-consuming, but it gave the neurologist a better understanding of what patients were looking for and what was being offered. Soon, Bedlack found himself looking up the same things over and over again, which got him thinking about the need for a central database about non-mainstream ALS treatments. Physicians and patients needed to know what worked and what didn’t. For that, researchers would have to investigate the different possibilities brought to them by patients and see whether these treatments were plausible and might help, or whether they would simply be a waste of patients’ time, hope, and money. “We want to put our scientific tools to work for patients, and help them understand if this treatment makes any sense, what data is out there, what the potential risks are,” Bedlack said. “This is not obvious- these aren’t easy questions to answer.” The project is a collaboration between Bedlack and 80 other scientists from around the world that attempts to unsnarl the complicated array of non-traditional ALS treatments. Anyone can suggest an idea or treatment for the researchers to investigate. The best way to contact the ALSUntangled researchers is via email or Twitter.com/@ALSUntangled. (For a free Twitter account, sign up at twitter.com) The researchers then begin the lengthy process of investigating these ideas, and then they publish their findings as an open access article (which means everyone can read it, free of charge) in the peer-reviewed research journal Amyotrophic Lateral Sclerosis.

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