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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ALS patients differ on treatment choices in later phases of disease

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Two new studies analyzing treatment decisions in late-stage amyotrophic lateral sclerosis (ALS) patients shed light onto treatments aimed to extend the duration and quality of life in this progressively debilitating neuromuscular disorder. Researchers from the Perelman School of Medicine at the University of Pennsylvania found that waiting until the last minute to receive one treatment resulted in not living long enough to experience the benefits. One Penn Medicine study demonstrates that ALS patients who have feeding tubes placed before an emergency situation strikes fare better. Those having surgeries in non-emergent settings were much less likely to die within one month after surgery, compared to ALS patients receiving their feeding tubes under duress. Median survival after the feeding tube surgery was 6 months overall and longer for patients undergoing non-emergent versus emergent placement (7 months vs. 4 months). In addition, mortality rates were worse for patients having procedures done at hospitals that did not regularly perform feeding tubes placement in ALS patients. In a second study, researchers found polarized treatment preferences regarding Riluzole, the first FDA approved treatment to slow ALS. Patients had sharply polarized preferences about this expensive treatment, which modestly prolongs length of life of ALS patients. In a survey of 98 patients with ALS or Motor Neuron Disease, nearly two-thirds of the patients ranked Riluzole as either the most important (30 percent) or least important (33 percent) treatment option.
“It is important to ask patients how they value their treatments, as in this case, we learned that patients who are older and looking for a high quality of life, valued this drug considerably more than people with impaired walking ability, who instead preferred supportive therapies like adaptive equipment,” said Leo McCluskey, MD, professor of Neurology and director of the Penn ALS Center. “Overall, medical care providers should work with patients to discuss treatment options throughout the progression of the disease to ensure a high quality of end-of-life care.”

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Lou Gehrig’s disease stills man’s voice, but not his spirit, will to live

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Tim Brooks was a boy when his father died young. That was only the start. Years later, the Oak Harbor resident lost two brothers within six months; one of electric shock while working, the other, like their father, in an auto accident. More recently, his mother and another brother died of Lou Gehrig’s disease, which causes progressive muscle weakness, paralysis, and ultimately death. It is more formally known as amyotrophic lateral sclerosis, or ALS. Now Mr. Brooks is living with ALS, which in his case has hindered his speech and swallowing to the point that most nourishment comes through a feeding tube. Still, he and his wife of nearly 34 years, Andrea, are enjoying life and are trying to help researchers find the abnormal gene causing ALS among relatives that could be present in more — including their two daughters and 2-year-old granddaughter. “It is hard to correct something if you don’t know what caused it,” Mr. Brooks said through an iPad, a device he uses to communicate.

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Cytokinetics Shows Hint of Effect With Drug for Lou Gehrig’s Disease

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There has never been much innovation to brag about in the treatment of the neurodegenerative disease that killed Yankees legend Lou Gehrig. But South San Francisco-based Cytokinetics is reporting today on clinical trial results that offer a hint it could be onto something for treating amyotrophic lateral sclerosis (ALS). An experimental drug from Cytokinetics met its main goal of demonstrating safety in 49 patients, while showing signs after two weeks of beating a placebo on a standard test of disease progression, according to results presented today at the American Academy of Neurology meeting in New Orleans. The result was from a small study which wasn’t designed to show a clinical benefit, and the findings were not statistically significant, meaning it could have been a fluke. But the finding is provocative enough that Cytokinetics has been emboldened to take the next step, and invest significant time and money to advance to the final phase of clinical trials normally required for FDA approval of a new drug. “The idea you can see a change after two weeks on a functional rating scale is encouraging,” says Jeremy Shefner, the chair of the Department of Neurology at the Upstate Medical University at the State University of New York, and the study’s principal investigator. “It’s something we’ve never seen in ALS before.”

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Lou Gehrig’s Disease: Patients Take Research Into Their Own Hands

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Though his body betrays him, Eric Valor’s mind is still strong. Diagnosed with Lou Gehrig’s disease in 2004, the 43-year-old relies on machines to move, talk, eat and breathe. But that hasn’t stopped him from running his own drug trial. With help, Valor has been injecting sodium chlorite, a chemical used by water treatment plants, into his paralyzed body through a feeding tube. He’s convinced it’s the active ingredient in NP001, an experimental drug made by Neuraltus Pharmaceuticals Inc. And other patients are following his lead. “My original plan was to keep it secret until I could report with confidence that it was safe and even marginally effective,” said Valor, who started taking sodium chlorite in October 2011. “But the secret got out, so I made a website to try to capture data as best I could.” From a special bed in his Santa Cruz, Calif. home, Valor uses his eyes to control a ceiling-mounted computer — a design from his days as a computer specialist. He carefully tracks his disease progression and that of more than two dozen other patients taking sodium chlorite with hopes the makeshift drug will buy them all time until NP001, if proven to work, is approved by the U.S. Food and Drug Administration. But experts say the DIY approach is dangerous. “It’s pretty frightening,” said Dr. Jonathan Glass, neurologist and director of the Emory ALS Center in Atlanta, Ga. “I think it’s a cry of desperation for these folks, using something not made to strict standards with no evidence it works.” But people with Lou Gehrig’s disease, also known as ALS, are eager to find out whether sodium chlorite works quickly and on their own terms. Frustrated with the pace of clinical research, they’ve joined forces to tackle the best leads on short order, from off-label drugs to stem cell transplants, and now makeshift NP001. It’s a far cry from a properly designed clinical trial, which has a control group to weed out the infamous placebo effect. Still, the DIY design puts patients back in control.

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Quest Diagnostics Announces C9orf72 Genetic Test for ALS (Lou Gehrig’s Disease)

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Quest Diagnostics, the world’s leading provider of diagnostic testing, information and services, today announced a new genetic testing service from its Athena Diagnostics business unit, a leader in neurology diagnostics, for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. It is the first clinically available testing service for detecting hexanucleotide repeat expansion in the C9orf72 gene. Research published in the April 2012 issue of The Lancet found that this C9orf72 mutation was present in up to 39% of familial (inherited) ALS cases examined, and between 4-8% in sporadic (no known family history) cases, in a multi-national study population. The test is offered to aid in the diagnosis of familial and sporadic ALS. “C9orf72 may turn out to be one of the most important discoveries in the history of ALS genetic research,” said Richard Bedlack, M.D, director of the Duke University ALS Clinic. “Preliminary work suggests that this is the most common identifiable cause for ALS in patients with or without a family history of the disease.” In addition, research suggests the hexanucleotide repeat expansion of the C9orf72 gene is also associated with familial and sporadic Frontotemporal dementia (FTD), the second most common form of early-onset dementia after Alzheimer’s disease.

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