ALS Lou Gehrig's ...
Follow
Find
23.0K views | +0 today
ALS Lou Gehrig's Disease
Latest research news about ALS (Lou Gehrig's Disease) from around the world
Your new post is loading...
Your new post is loading...
Scooped by ALS Research News
Scoop.it!

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

The ALS Therapy Development Institute Partners with Neurotune to Investigate Potential Treatments for MND | ALS Lou Gehrig's Disease | Scoop.it


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.

 

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

ALS clinics start implanting breathing-assist device under new FDA approval | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

ALS Untangled uses social networking to bring the #ALS community together. | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

Lou Gehrig’s disease stills man’s voice, but not his spirit, will to live | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

Lou Gehrig's Disease: Patients Take Research Into Their Own Hands | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Stephen Hawking Makes a Big Bang

Stephen Hawking Makes a Big Bang | ALS Lou Gehrig's Disease | Scoop.it

Stephen Hawking, the British physicist, has had plenty to say about the big bang theory, which made him a natural for “The Big Bang Theory.” The TV sitcom, not the beginning of the universe. “When people would ask us who a ‘dream guest star’ for the show would be, we would always joke and say Stephen Hawking,” the show’s executive producer, Bill Prady, said in a statement when Hawking’s appearance was announced in March. “In fact, we’re not exactly sure how we got him. It’s the kind of mystery that could only be understood by, say, a Stephen Hawking.”

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Exercise: stretching the limits of ALS care

Exercise: stretching the limits of ALS care | ALS Lou Gehrig's Disease | Scoop.it

In people with ALS, the motor nerves deteriorate leading to muscle weakness and ultimately paralysis. In hopes to stop this neurodegeneration in its tracks, researchers are looking towards substances called neutrophins including BDNF, GDNF, IGF-1 and VEGF to keep nerves healthy and plugged into muscles. But delivering these protective substances safely at the right place, at the right time and at the right dose has turned out to be extremely difficult to do. And, scientists remain unsure which one of these neurotrophins is the best choice for people with ALS to protect the motor nerves from further deterioration. Studies suggest that moderate aerobic exercise such as stationary bicycling or treadmilling might have the potential to help keep muscles and nerves healthy longer in people with ALS by increasing levels of many of these protective substances in the brain and spinal cord. What’s more, a moderate workout might even help fight the disease by boosting energy supplies, removing damaged proteins and reducing inflammation. Neurologists nevertheless remain reluctant to recommend specific exercise routines for their patients. There simply is not enough clinical evidence according to experts to indicate which routines are safe and offer the most benefit to people with ALS. New clinical trials promise to change that by putting exercise to the test in people with ALS.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Young Faces of ALS Campaign Announces Second Annual "National Corntoss Challenge"

Young Faces of ALS Campaign Announces Second Annual "National Corntoss Challenge" | ALS Lou Gehrig's Disease | Scoop.it

The Young Faces of ALS Campaign(YFALS) announced its second annual "National Corntoss Challenge" fundraising event occurring in multiple cities this May and June in support of research toward an effective treatment for ALS, better known as Lou Gehrig's disease. This is a charity tournament where thousands of participants in cities from coast to coast will join in a day of fundraising by playing the popular tailgate game, Corntoss (a.k.a. Cornhole, bean bag toss, baggo or bags).This year's National Corntoss Challenge will be held in the following cities: Atlanta on May 12; Boston and Rapid City on May 19; Boise, New York, Chicago, San Francisco, Seattle and Washington DC on June 23. Each city's event will be run by local volunteer "champions" who are committed to raising awareness of ALS and funding research towards a cure.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

The 2012 Annual Packard Symposium shared the latest steps toward finding a cure.

The 2012 Annual Packard Symposium shared the latest steps toward finding a cure. | ALS Lou Gehrig's Disease | Scoop.it

It was a meeting of the minds. On March 8th, 9th, and 10th, Packard Center grantees gathered at the Baltimore Marriott Waterfront to present and discuss their research findings from the past year. Unlike previous years, the symposium’s format integrated bench science with clinical research findings. As Packard Science Director Piera Pasinelli noted at the start of the first day, lab results and clinical trials inform each other. When each perspective learns from the other, Pasinelli said, scientists will move more quickly towards finding promising therapeutic targets to develop into drugs for ALS. "The goal of Packard has always been to bring new discoveries forward and closer to the clinic as quickly as possible,” said Pasinelli. “By encouraging these partnerships and exchange of ideas between clinicians and bench scientists, we want to take the most promising scientific ideas to the attention of the clinicians faster. Because basic and clinical science inform each other, this exchange is also beneficial to refine strategies in the lab and in the clinic." Perhaps the most significant breakthrough in ALS science in the past year has been the discovery of the repeat expansion on chromosome 9. Known as C9ORF72, it was co-discovered by Packard Scientist Bryan Traynor and is the most common genetic cause of familial ALS in North America. The identification of hundreds of copies of a repeating six DNA base pair sequence was a milestone, but like many important scientific discoveries, it raised more questions than it answered.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Stem Cell Study Aids Quest for ALS Therapies

Stem Cell Study Aids Quest for ALS Therapies | ALS Lou Gehrig's Disease | Scoop.it

An article published in the journal Proceedings of the National Academy of Sciences reports a breakthrough using cutting-edge stem cell research, which could speed up the discovery of new treatments for amyotrophic lateral sclerosis (ALS) also known as motor neuron disease (MND) outside the United States. An international research team, led by the Euan MacDonald Centre for Motor Neurone Disease Research at the University of Edinburgh in partnership with researchers from King’s College London; Columbia University, New York; and the University of San Francisco, has created motor neurons using skin cells from a person with an inherited form of ALS/MND. Researchers discovered that abnormalities of a protein called TDP-43, implicated in more than 90% of cases of ALS/MND, resulted in the death of motor neuron cells. This is the first time that scientists have been able to see the direct effect of abnormal TDP-43 on human motor neurons cultured in a dish. Professor Siddharthan Chandran from the University of Edinburgh said, “Using patient stem cells to model MND in a dish offers untold possibilities for how we study the cause of this terrible disease as well as accelerating drug discovery by providing a cost-effective way to test many thousands of potential treatments.”

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

A webinar hosted by ALS TDI reviews the biotech's development of TDI 132 (Gilenya)

A webinar hosted by ALS TDI reviews the biotech's development of TDI 132 (Gilenya) | ALS Lou Gehrig's Disease | Scoop.it

An overview of the development of TDI 132 for use in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) was the topic of a webinar hosted March 12, 2012, by the nonprofit biotech ALS Therapy Development Institute (ALS TDI) of Cambridge, Mass. TDI 132, also known as Gilenya, has received approval from the U.S. Food and Drug Administration (FDA) for use in multiple sclerosis. The drug works by modulating the immune system.ALS TDI CEO and Chief Scientific Officer Steve Perrin, and James Berry, a clinical research fellow and MDA grantee at Massachusetts General Hospital in Boston, discussed numerous aspects of TDI 132's development and advancement to clinical trials. The webinar has been archived on ALS TDI's website and is available for viewing to all who login or register.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Roger D. Kornberg Joins Prize4Life’s Scientific Advisory Board

Roger D. Kornberg Joins Prize4Life’s Scientific Advisory Board | ALS Lou Gehrig's Disease | Scoop.it

Prize4Life, a non-profit organization dedicated to accelerating the discovery of treatments and a cure for Amyotrophic Lateral Sclerosis (ALS), is pleased to announce the appointment of Roger D. Kornberg, PhD, to its Scientific Advisory Board. “Prize4Life is delighted to welcome such an accomplished and talented scientific mind,” said Avi Kremer, co-founder and Chief Executive Officer of Prize4Life. “Roger Kornberg is an excellent addition to our Scientific Advisory Board, which provides a high level of expert knowledge and wise counsel to Prize4Life and is integral to the success of our important mission.” Dr. Kornberg is currently a professor of structural biology at Stanford University School of Medicine, where his lab has continued to elucidate the complex process by which DNA is unraveled, read, and “transcribed” into RNA. Although his research is not directly related to ALS, Dr. Kornberg believes “Prize4Life’s mission of finding treatments and a cure for ALS is imperative. I look forward to joining the organization’s impressive assembly of scientific advisors. Through its innovative model, Prize4Life can produce real results for ALS patients.”

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Hood Meadows Hosts New Ski to Defeat ALS Event

Hood Meadows Hosts New Ski to Defeat ALS Event | ALS Lou Gehrig's Disease | Scoop.it

The goal is $100,000, and a former CMH heli guide is one of the driving forces behind the fundraiser to be held at Mt. Hood Meadows in Oregon. The fundraiser on April 14, 2012 aims raise money for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease. Seventy-four-year-old Fred Noble, known to friends and the Portland ski community as The Fredinator, was diagnosed with ALS in December 2010. The life-long skier who worked with Canadian Mountain Holidays for 38 years suddenly needed to adapt everything in his life to accommodate living with ALS, and the local chapter of The ALS Association helped out. In repayment, Noble is leading the fundraising charge, having set a personal goal of $10,000. Noble credits the local chapter of The ALS Association with helping him with loans of adaptive equipment, advice on remodeling his house to accommodate his new needs, and dealing with medical insurance. Even with ALS, he has not given up skiing, but has converted to skiing with adaptive gear.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

Neuralstem Updates ALS Stem Cell Trial Progress - FDA Permits Additional Dosing of Return Patients | ALS Lou Gehrig's Disease | Scoop.it

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."

more...
Susan Waugaman's comment, February 4, 2013 8:11 PM
I believe theFDA needs to speed up this process
Laurie Orellana San Andres's comment, May 20, 2013 8:44 AM
This should be available to more patients and it does need to be faster. The word has to get out there.
Scooped by ALS Research News
Scoop.it!

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

New research may help explain why there are so few ALS treatments available. | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

ALS patients differ on treatment choices in later phases of disease

ALS patients differ on treatment choices in later phases of disease | ALS Lou Gehrig's Disease | Scoop.it

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."

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

Cytokinetics Shows Hint of Effect With Drug for Lou Gehrig’s Disease | ALS Lou Gehrig's Disease | Scoop.it

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.”

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

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

Quest Diagnostics Announces C9orf72 Genetic Test for ALS (Lou Gehrig's Disease) | ALS Lou Gehrig's Disease | Scoop.it

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.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Brain Scanner Being Used To Give Stephen Hawking A New Voice

Brain Scanner Being Used To Give Stephen Hawking A New Voice | ALS Lou Gehrig's Disease | Scoop.it

World renowned scientist Stephen Hawking has given so much to the world of physics. Now he’s doing what he can for biology – and perhaps he will benefit from the experience himself in a big way. Hawking is testing the iBrain, a user-friendly brain scanner that can be worn all the time. At the very least the tests will help to improve the iBrain, but if things go exceedingly well, the device could help Hawking to speak. Hawking suffers from amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, a condition in which neurons involved in controlling movement are impaired or dead. As is the case with Hawking, the disease leaves many of its victims severely handicapped. Already having difficulties communicating, a tracheotomy in 1985 removed Hawking’s ability to speak altogether. Right now, in order talk Hawking relies on a tiny IR sensor that detects twitches in his cheek muscle. To formulate words, let alone sentences, is time-consuming and tedious task. Last summer the founder of NeuroVigil, Philip Low, brought one of his company’s brain scanners to Hawking’s house in Cambridge, England. Low hopes that the iBrain will be able to read the 70-year-old physicist’s brain signals and send them to a computer where they would be translated into speech. They’re only getting started, however, and it is really way too early to know if the iBrain will be effective, but Low recently announced some encouraging, albeit very preliminary results. During his visit, Low asked Hawking to think about making a fist. Even though he is incapable of actually making a fist, just by thinking of it, the neurons in his motor cortex still “commanded” his hand to do it. The iBrain was able to see the change in brain activity that corresponded to the command. Like I said, pretty preliminary. Detecting a change from a clenched fist is a far cry from translating brain activity into words and sentences. Low plans on returning to Hawking’s house this summer to continue the study.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Lou Gehrig’s Disease May Bring Biogen $1 Billion Payoff

Lou Gehrig’s Disease May Bring Biogen $1 Billion Payoff | ALS Lou Gehrig's Disease | Scoop.it

Biogen Idec Inc. Chief Executive Officer George Scangos was skeptical when he learned company researchers were pursuing a new treatment for Lou Gehrig’s disease. No one knows the cause of the illness that killed the famous baseball player more than 70 years ago, and no medicine for it has been shown to slow its advance for long. Scangos changed his mind after he took charge two years ago and reviewed data about dexpramipexole. The compound, which may slow disease progression, is now in the final stages of clinical trials required for U.S. Food and Drug Administration approval, with results expected this year. “I don’t know any disease that’s in more need of therapy than ALS,” said Scangos, whose company is the world’s largest maker of drugs for multiple sclerosis, in a telephone interview. “It’s certainly risky, but the data speaks for itself. So we made a calculated bet.” There’s one drug on the market, Paris-based Sanofi’s Rilutek, and it provides only a modest benefit in reducing ALS’s progression. Rilutek has U.S. sales of about $50 million, according to Eric Schmidt, an analyst with Cowen & Co. in New York. The market for dexpramipexole, if it’s approved, may top $1 billion a year, Schmidt estimates. The company paid $80 million in cash and stock for a licensing deal in August 2010 with closely held Knopp Biosciences, which first developed the drug, and will pay an additional $265 million if certain regulatory and sales goals are met. The compound looked promising enough in earlier trials by Knopp to attract the attention of Al Sandrock, Weston, Massachusetts-based Biogen’s head of neurology research and a former physician who worked with ALS patients. It’s designed to improve functioning of the mitochondria, the energy producers in cells, and to provide protection to neurons under stress.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

The Missing Link? Two new studies take a fresh look at the relationship between SOD1 and ALS.

The Missing Link? Two new studies take a fresh look at the relationship between SOD1 and ALS. | ALS Lou Gehrig's Disease | Scoop.it

Researchers first associated mutations in the SOD1 gene with familial ALS in 1993, one of the first times that a specific gene had been associated with the illness. Since this discovery nearly twenty years ago, scientists have been hard at work trying to figure out exactly how changes in the SOD1 protein can lead to ALS. Two new studies published in the Proceedings of the National Academy of Sciences by Packard scientists in March have revealed some important clues in the relationship between SOD1 and ALS. Only a small percentage of ALS patients carry an inherited mutation in SOD1, yet some studies revealed that the SOD1 protein doesn’t work properly even in sporadic ALS patients without a known mutation in the gene. Packard Center Science Director Piera Pasinelli showed that SOD1 is over-oxidized in certain disease-fighting white blood cells in sporadic ALS patients with bulbar onset. When further stressed, this over-oxidized SOD1 acquires toxic properties similar to those seen in mutant SOD1, linking mechanisms of toxicity between a subset of sporadic and familial patients respectively. In a separate study, Packard researchers Jonathan Glass, a neuroscientist at Emory University in Atlanta, and Jean Pierre Julien, a neuroscientist at Laval University in Quebec, developed antibodies to recognize misfolded SOD1 in motor neurons in familial ALS patients. Although mutant SOD1 is found in every cell in the body, the antibodies only recognized the misfolded SOD1 in dying motor neurons. This indicates, Glass said, that specific changes are happening to SOD1 in motor neurons during the course of disease.“We still don’t know the cause of ALS. We know that a mutation in certain genes is associated with ALS, but we don’t know why these mutations kill cells,” Glass said. These studies are helping researchers figure out this association.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Neuralstem ALS Stem Cell Trial Interim Results Reported in the Journal, STEM CELLS

Neuralstem ALS Stem Cell Trial Interim Results Reported in the Journal, STEM CELLS | ALS Lou Gehrig's Disease | Scoop.it

Neuralstem, Inc. announced that safety results from the first 12 patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) to receive its stem cells were reported online in the peer-reviewed publication, STEM CELLS, on March 13th. "Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS: Results of a Phase I Trial in 12 Patients" reports that one patient has shown improvement in his clinical status, even though researchers caution that the study was not designed to show efficacy. Additionally, there was no evidence of accelerated disease progression due to the intervention in any of the 12 patients, who were followed from 6-18 months after they were transplanted with the cells. All of the patients, who received transplants in the lumbar (lower back) region, tolerated the treatment without any long-term complications related to either the surgery or the cells. The 12 patients, part of the ongoing Phase I trial to evaluate the safety of Neuralstem's stem cells and transplantation procedure in patients with ALS, were the first in the world to receive intraspinal stem cell injections. "For these first 12 patients, we have met the objective of the Phase I trial, demonstrating safety for both the procedure of intraspinal injection and the presence of the neural stem cells in the spinal cords of ALS patients," said Jonathan Glass, MD, lead author of the publication. "We are encouraged by these results and have now advanced our trial to injections into the cervical spinal cord, targeting the motor neurons that control respiratory function." Dr. Glass is Professor of Neurology and Pathology at Emory University School of Medicine, as well as the Director of the Emory ALS Center. "As this article points out, our experience in the lumbar spinal cord has been overwhelmingly positive," commented Karl Johe, PhD, study author and Neuralstem Chairman and Chief Scientific Officer. "We have already transplanted two patients in the cervical spinal cord, where we believe we can affect patients' lives the most by improving their breathing. We are in active discussions with the FDA to increase the number of cells and the number of injections as well."...

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Lowen and Navarro's Eric Lowen dies of ALS at 60

Lowen and Navarro's Eric Lowen dies of ALS at 60 | ALS Lou Gehrig's Disease | Scoop.it

Eric Lowen, 60, a singer, songwriter, guitarist and half of the Lowen and Navarro folk group who with his songwriting partner Dan Navarro penned “We Belong” for Pat Benatar, died Friday at Kaiser Permanente Panorama City Hospital, the band announced. Lowen had been diagnosed with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease, in 2004. The degenerative disease affects nerve cells in the brain and spinal cord and eventually weakens muscles throughout the body. Born Oct. 23, 1951, in Utica, N.Y., Lowen met Navarro in Los Angeles in the early 1980s when they worked as singing waiters at the Great American Food and Beverage Co. After they discovered they harmonized together well, they began performing and writing songs as a duo. The pair wrote “We Belong” for Benatar in 1984 and it became a top-five hit for her. Lowen, who was tall and blond, and the dark-haired Navarro regularly sang their acoustic versionin concert. The two also wrote for such pop acts as the Bangles, Dave Edmunds and David Lee Roth, and they composed jingles for commercials and TV shows. Beginning in 1988 Lowen and Navarro played a regular acoustic gig at the Breakaway in Venice, and they released their first album, “Walking on a Wire,” in 1990. They released a handful of studio albums and toured extensively until 2009, when Lowen’s condition worsened.

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Hopkins Launches New ALS Research Center with $25 Million Gift

Hopkins Launches New ALS Research Center with $25 Million Gift | ALS Lou Gehrig's Disease | Scoop.it

A $25 million gift has enabled Johns Hopkins to establish a new center to develop novel therapies for the neurodegenerative disease known as amyotrophic lateral sclerosis, Lou Gehrig’s disease, or ALS. Much of the center’s research will focus on using stem cells individually derived from ALS patients to develop new model systems to investigate how nerve cells degenerate, as tools to screen new drug therapies, and to develop stem cell therapies as transplants to potentially slow or reverse the disease. The new center, dedicated March 21 and formally known as the Michael S. and Karen G. Ansari ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins, is named for its benefactors: Michael and Karen Ansari. Michael Ansari is the founder, chairman and CEO of M.I.C. Industries. The gift, representing a five-year commitment, will fund a variety of efforts that aim to eventually cure ALS. The disease targets motor neurons, a type of nerve cell that controls muscle movement, and affects about three out of every 100,000 individuals. “Despite knowing about this disease for decades and the large number of clinical trials that have been completed, we still have little in our arsenal to treat it,” says Nicholas J. Maragakis, M.D., an associate professor of neurology at the Johns Hopkins University School of Medicine, co-medical director of the ALS Clinic and director of the new center. “We are now able to think out of the box about this disease. The goals of a center will focus on the use of stem cells as tools to foster aggressive programs in discovering the underlying mechanisms behind what causes ALS and rapidly translating these discoveries to the patients in our clinic.”

more...
No comment yet.
Scooped by ALS Research News
Scoop.it!

Samsung Develops Low-Cost “Eye Mouse”

Samsung Develops Low-Cost “Eye Mouse” | ALS Lou Gehrig's Disease | Scoop.it

A group of engineers at Samsung Electronics have come up with a low-cost eye-controlled computer mouse that they hope will enable a wider number of severely disabled people to use computers. Samsung’s “eyeCan” device builds on the functions of the eyeWriter, allowing users to enter text by blinking while looking at an on-screen keyboard and includes a navigation function for users to do things like exploring streets on a map. While the product isn’t unique, similar devices already in the market have hefty price tags of up to around $10,000. Samsung isn’t actually selling the eyeCan; it provides the software and instructions on how to make the device from readily available components costing around $50.

more...
No comment yet.