“Our results indicate that normal levels of a gene called C9ORF72 are crucial for the proper motor function of zebrafish, and loss of its function causes neurodegeneration,” explains Dr. Kabashi.

This is vital information, as ALS is caused by the death of motor neurons connecting the brain to the muscles. While the specific causes of ALS have eluded scientists for years, the announcement from Dr. Kabashi represents an important step forward, in hopes of finding new therapies and someday, a cure.

“Canadian researchers are recognized internationally for discoveries that further our understanding of ALS,” said Lindee David, CEO, ALS Canada. “It is a promising time for the ALS community. We are proud to support their work and are excited by Dr. Kabashi’s findings.”

First ever published animal model to study the function of gene C9ORF72

In 2011, two international consortia identified that mutations in C9ORF72 cause a higher percentage of familial and sporadic ALS than any other gene previously discovered. It was determined that the mutation makes up approximately one-third of all familial cases of ALS.

Following the important discovery, Dr. Kabashi and Dr. Pierre Drapeau, also of Université de Montréal, were co-awarded the ALS Canada-funded Bernice Ramsay Discovery Grant to create zebrafish models of reduced and mutated C9ORF72. The purpose was to determine the gene’s role in motor function and ability to cause neurodegeneration. Initial results from the research have recently been accepted for publication in the prestigious journal Annals of Neurology.

“To validate whether normal levels and/or function of C9ORF72 is essential for motor neuron health and activity, we developed an animal model to observe disease presentation in zebrafish,” said Dr. Kabashi “We observed clear motor dysfunction, swimming defects, and abnormalities at the cellular level.”

“Our hope is that these new animal models of ALS will help identify new avenues for therapy. Many more cellular and animal models of C9ORF72 are currently being studied and we look forward to continued exciting discoveries in what promises to be another breakthrough year for ALS research.” added David.

About ALS ALS is a terminal disease characterized by progressive paralysis of muscles throughout the body. Ninety percent of ALS patients die within five years of diagnosis and some in less than one. An estimated 3,000 Canadians have the disease, yet there are currently no effective treatment options. ALS is caused by death of motor neurons, which connect the brain to the muscles. While the specific cause remains unknown, promising discoveries in recent years have provided significant clues that should pave the way for new therapies and an eventual cure.

About ALS Canada The ALS Society of Canada, founded in 1977, is the only national voluntary health organization dedicated solely to the fight against ALS and support for those with ALS.  ALS Canada is the leading not-for-profit organization working nationwide to fund ALS research and, with the Provincial ALS Societies, working to improve the quality of life for Canadians affected by ALS.

SOURCE ALS Canada

The Foundation is accepting applications and the deadline is fast approaching. All entries must be postmarked by July 1, 2013!

Click on the Guidelines tab for more information and contact us through this website for scholarship information to be emailed to you.

GOOD LUCK!

This spirit of volunteerism also extends to our leadership. We have been fortunate to have some very bright and energetic people step forward to help plan how we might best serve the ALS community. With that in mind, we proudly announce that Melissa Marr has accepted the position of Marketing Director on the RASCALS Foundation Board of Directors.

Born, raised, and still residing in St. Louis, Missouri, Melissa is no stranger to the RASCALS family. She has been serving as our media maven for over a year now. It quickly became clear, however, that Melissa possessed a wealth of knowledge, fresh ideas, and commitment.

A former Media Planner and Account Executive for two local advertising agencies, Melissa brings eight years of marketing experience to her new role at RASCALS. She currently handles Marketing, Events and Business Development for a Sports and Entertainment website, InsideSTL.com and KFNS Sports Radio.

Her personal interests include traveling at every opportunity, and attending as many St. Louis Cardinals and Blues games as possible. She graduated from Southeast Missouri State University in 2005 with a Major in Mass Communications and Advertising with Minors in Graphic Design and Psychology. She was also a member of the Psi Chi Honor Society and Dean’s List.

Melissa’s other charitable efforts include Friends of Kids with Cancer, where she sits on the Communications Committee for their Young Friends Program to increase awareness and generate press for events that support the non-profit’s mission.

Congratulations, Melissa — and thank you on behalf of everyone at the RASCALS and ALS survivors everywhere.

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The Robert A. Stehlin Campaign for ALS (R.A.S.C.A.L.S.) is an all-volunteer 501(c)(3) charity. 100% of all funds raised go to building awareness, treatment research and development, plus ALS family assistance. There are no administrative costs.

Contributions are tax-deductible.

You may also be interested in visiting the RASCALS Store.

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The very next month on June 19th, his 36th birthday, the mystery was solved. Lou was diagnosed at the Mayo Clinic with amyotrophic lateral sclerosis (ALS). He never played another game, but remained with the team as their captain.

If there was ever a noble action hero worthy of emulating, it is Lou Gehrig. Lou didn’t quit. And neither will we. With that in mind, we have (respectfully) changed the name of ALS Awareness Month. This year we want to take it a step further and make May 2013:

ALS ACTION MONTH

If you’re on this Web site, chances are you already know about Lou’s heroic attitude and the disease that bears his name. You know there’s no cure and current treatment options are sadly suboptimal. So we’re asking everyone to step up and take action this month.

Make a donation to the Robert A. Stehlin Campaign for ALS (R.A.S.C.A.L.S.). Or you can help spread the word by purchasing a wrist band at the RASCALS Store. All contributions are tax-deductible.

We are a 100% all-volunteer 501(c)(3) non-profit charity. No one takes a paycheck here and there are no administrative costs. That means 100% of all funds raised by the R.A.S.C.A.L.S. Foundation go directly to fighting ALS.

The money goes to fund promising treatment research and development. It helps with ALS family assistance, such as our annual Higher Education Scholarship program.

There all are all kinds of things you can do. Simply introduce ALS to someone who’s never heard of it There are hundreds of millions of people who haven’t.

Lend a helping hand to an ALS family in your community. Offer to cut their grass, wash their windows, or tighten a doorknob. Trust us, every little bit helps when a family is trying to deal with the enormous burden of this disease.

Please do what you can. Even a little bit goes a long way. Thank you for taking action.

END ALS.

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By Kaitlynn Riely for the Pittsburgh Post-Gazette, April 26, 2013 •

A person diagnosed this year with amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig’s disease, will receive basically the same prognosis as the New York Yankee did — an average life expectancy of two to five years.

More than 70 years after Gehrig’s death, the cause of the disease that took his life and then his name remains unclear, and with the exception of one federally approved drug that has been shown to extend life a few months, there is no effective treatment and no cure. Although research into ALS continues, studies in recent years have been marked by “frustrating failures,” said Lucie Bruijn, chief scientist for the ALS Association.

But researchers at the University of Pittsburgh School of Medicine announced Thursday the results of a new study on ALS symptoms in mice that they hope may hold promise for the understanding and treatment of the disease.

The study results, published by the journal Neurobiology of Disease, showed that when mice received injections of the hormone melatonin, they developed ALS symptoms later and lived longer than mice who were given a placebo.

It’s too soon to say whether the results seen in mice will translate into results for humans with the disease, said Robert Friedlander, the study’s senior investigator and chairman of Pitt’s Neurological Surgery department. And it will be a number of years before human trials can offer any insight, he said.

In ALS research, that question — whether what works in mice can work in humans — is the big one.

“That’s the challenge in our field, really,” said Ms. Bruijn, who has a doctorate in biochemistry. “The challenge for us is to move from the mouse model and findings in the mouse model and be effective in the clinic.”

She said she was enthusiastic but cautious about melatonin as an ALS treatment, citing an earlier study that demonstrated the compound’s antioxidative effects on mice.

Dr. Friedlander, who also stressed the importance of waiting to see whether something that works in mice could work in humans, described the study’s results as a good step forward.

“There is increased knowledge that we’re gaining every day,” he said. “I don’t want to say we’re at the edge of a breakthrough.”

But he said the more drugs that researchers are able to show work in mice and then move them into human trials, the closer researchers are to arriving at treatments.

“We are on the right path, and we wish we could get there faster,” said Dr. Friedlander, who has been researching ALS for 20 years. This study began five years ago.

Time is of the essence for people with ALS, which affects about 30,000 Americans, including 300 in Western Pennsylvania, at any one time. The disease leaves the mind intact while it causes the motor neurons to degenerate. A person with ALS loses the ability to control muscle movement, then experiences paralysis and trouble swallowing and breathing, causing death two to five years after diagnosis on average.

Rilutek, or riluzole, is the sole FDA-approved drug treatment available for ALS, and it extends a person’s lifetime by a few months. Melatonin emerged as a possible treatment for ALS-like symptoms in mice after Dr. Friedlander and his researchers screened 1,040 FDA-approved drugs, discovering that melatonin was one of the most effective compounds in preventing neuron death.

In mice, it delayed ALS symptoms, neurological deterioration and death by 7 percent of the mouse’s lifetime, the study showed. The importance of the study, Dr. Friedlander said, is that it identified a new potential treatment for ALS and a pathway for the disease that may help researchers better understand it.

Although melatonin is available in stores, Dr. Friedlander said he would not endorse self-treatment and cautioned that further research trials must be done. He also predicted that the eventual treatment for ALS will be a “cocktail-type approach,” involving multiple drugs, similar to HIV treatment.

“That’s what we need in a lot of these neurodegenerative diseases, including ALS, is to identify a group of drugs that will act in concert and synergistically to have an effect,” he said.

ALS is a difficult disease, Ms. Bruijn said, and many times a treatment has looked promising but didn’t work in patients. Still, she predicted news of the Pitt study would be greeted with optimism by people with ALS.

“There will be great excitement among patients, because there’s a desperation for treatment,” she said

Kaitlynn Riely: kriely@post-gazette.com or 412-263-1707.

RASCALS Note: you can read the official report on the study here from the  UPMC/University of Pittsburgh Schools of the Health Sciences or scroll down to next post.

PITTSBURGH, April 25, 2013 – Melatonin injections delayed symptom onset and reduced mortality in a mouse model of the neurodegenerative condition amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, according to a new study by researchers at the University of Pittsburgh School of Medicine. In a report published online ahead of print in the journal Neurobiology of Disease, the team revealed that receptors for melatonin are found in the nerve cells, a finding that could launch novel therapeutic approaches.

Annually about 5,000 people are diagnosed with ALS, which is characterized by progressive muscle weakness and eventual death due to the failure of respiratory muscles, said senior investigator Robert Friedlander, M.D., UPMC Endowed Professor of neurosurgery and neurobiology and chair, Department of Neurological Surgery, Pitt School of Medicine. But the causes of the condition are not well understood, thwarting development of a cure or even effective treatments.

Melatonin is a naturally occurring hormone that is best known for its role in sleep regulation. After screening more than a thousand FDA-approved drugs several years ago, the research team determined that melatonin is a powerful antioxidant that blocks the release of enzymes that activate apoptosis, or programmed cell death.

“Our experiments show for the first time that a lack of melatonin and melatonin receptor 1, or MT1, is associated with the progression of ALS,” Dr. Friedlander said. “We saw similar results in a Huntington’s disease model in an earlier project, suggesting similar biochemical pathways are disrupted in these challenging neurologic diseases.”

Hoping to stop neuron death in ALS just as they did in Huntington’s, the research team treated mice bred to have an ALS-like disease with injections of melatonin or with a placebo. Compared to untreated animals, the melatonin group developed symptoms later, survived longer, and had less degeneration of motor neurons in the spinal cord.

“Much more work has to be done to unravel these mechanisms before human trials of melatonin or a drug akin to it can be conducted to determine its usefulness as an ALS treatment,” Dr. Friedlander said. “I suspect that a combination of agents that act on these pathways will be needed to make headway with this devastating disease.”

Co-authors of the paper include other scientists from the University of Pittsburgh School of Medicine; Harvard Medical School; Ohio State University; Weifang Medical University; Bedford VA Medical System, Boston; St. Joseph’s Hospital and Medical Center, Phoenix; University of Texas Medical School at Houston; and VA Pittsburgh Health Care System.

The project was funded by grants NS051756, NS039324, and NS055072 of the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health; the U.S. Department of Defense; and the Muscular Dystrophy Association.

ROCKVILLE, Maryland, April 29, 2013  — Neuralstem, Inc. announced that final data on the intraspinal delivery method employed in its NSI-566 Phase I trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) was presented today at the American Association of Neurological Surgeons Annual Meeting. In a presentation called “Intraspinal Stem Cell Transplantation in ALS, A Phase I Trial: Cervical Microinjection Safety Outcomes,” Jonathan Patrick Riley, MD, of the Department of Neurological Surgery at Emory University, in Atlanta, GA, presented data from all 18 procedures, in 15 patients, treated in Phase I. (Three of the patients returned to the trial for subsequent treatments in a different region of the spinal cord.) The study found that none of the patients experienced neurological worsening from injections into either the upper (cervical) or lower (lumbar) region of the back, nor was there any evidence of spinal cord injury. The cells also appeared to be safe with no evidence of toxicity. Researchers further noted that, even in the vulnerable ALS spinal cord, serial injections were well-tolerated. They concluded that the approach of starting in the lower region of the spine, where the potential risk to the patient was less, and then progressing upwards, might not be required in future trials, and that this intraspinal delivery technique is an option for treating other neurodegenerative and traumatic spinal cord disorders.

“This is an extremely important confirmation of the safety and tolerability of both the route of administration and our cells,” said Karl Johe, PhD, Neuralstem Chairman and Chief Scientific Officer and a study author. “These patients are in many ways more fragile, and at greater risk from spinal surgery, than patients with other types of diseases or injuries where our cell therapy may be applicable. That our cells and method of delivery are safe in ALS patients bodes very well for expanding to other indications. We expect to commence our FDA-approved Phase I trial in chronic spinal cord injury later this year using the same methodology. We want to thank the surgeons at Emory, who developed these techniques, as well as the patients and their families who have taken part in the trial.”

About Neuralstem

Neuralstem’s patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem completed an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig’s disease, in February 2013, and has received FDA approval to begin Phase II. Neuralstem has been awarded orphan status designation by the FDA for its ALS cell therapy.

In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company received approval to commence a Phase I safety trial in chronic spinal cord injury in January 2013.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain’s capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions.  The company is in the last cohort of a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include traumatic brain injury (TBI), Alzheimer’s disease, and post-traumatic stress disorder (PTSD).

For more information, please visit www.neuralstem.com or connect with us on Twitter, Facebook and LinkedIn

Cautionary Statement Regarding Forward Looking Information

This news release may contain forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem’s technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem’s periodic reports, including the annual report on Form 10-K for the year ended December 31, 2012.

SOURCE: Neuralstem, Inc.

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The Robert A. Stehlin Campaign for ALS (R.A.S.C.A.L.S.) is an all-volunteer 501(c)(3) charity. 100% of all funds raised go to building awareness, treatment research and development, plus ALS family assistance. There are no administrative costs.

Contributions are tax-deductible.

You may also be interested in visiting the RASCALS Store.

The material presented here is for informational purposes only and should not be construed as medical advice, or relied upon as a substitute for medical advice from a health care provider.

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That means no salaries, payments, or perks for anyone. Out-of-pocket expenses truly means out of our own pockets. There are no fancy cars or credits for gas or mileage. No big offices to write off. In fact, we don’t even have an office. Everyone works from home—and from the heart.

As a result, 100% of all funds raised by the R.A.S.C.A.L.S. Foundation go directly to fighting ALS. The money goes to things like maintaining this informative Web site to educate and build awareness. It helps fund promising treatment research and development. And it goes to ALS family assistance, such as our annual Higher Education Scholarship program.

Together with our generous donors, it is our volunteers who make all of this possible, selflessly giving it 110% whenever called upon, through their time, energy, and compassion.  I couldn’t be more proud—or grateful.

On behalf of ALS survivors and families everywhere, I thank you from the bottom my heart. Your efforts are top of mind each and every day.

                                                                            — Bob Stehlin

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For more information on volunteer opportunities, contact the RASCALS founder and president, Bob Stehlin, at 636-464-6704.

You may also send him an email at stl.rascals@yahoo.com.

(Bob is an ALS survivor, diagnosed September 11, 2009.)

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BOSTON, Massachusetts, March 29, 2013 — Published online today in Lancet Neurology, an SOD1-related familial ALS trial under the direction of NEALS researchers Timothy Miller, MD, PhD (Washington University School of Medicine) and Merit Cudkowicz (Massachusetts General Hospital) indicates that antisense oligonucleotide delivery to the central nervous system may be a feasible therapeutic strategy in treating ALS.  Mutations in SOD-1 cause 13% of familial ALS cases.

The placebo controlled, double-blind, randomized, dose escalation Phase I clinical trial sought to evaluate safety, tolerability, and pharmacokinetics of ISIS 333611, an antisense oligonucleotide against SOD1.  Participants with ALS caused by mutations in SOD1 were enrolled. ISIS 333611 was well-tolerated.  There were no safety concerns.  ISIS 333611 was delivered by intrathecal infusion using an external pump over 11.5 hours at increasing doses to four cohorts of eight subjects with SOD1 mutation (randomized 6 drug: 2 placebo/cohort).  Participants could enroll in more than one cohort.  The four consecutive dose cohorts were 0.15 mg, 0.5 mg, 1.5 mg, and 3 mg.  Pharmacokinetic levels were consistent with levels predicted from preclinical studies.

Supported by Isis Pharmaceuticals, the Muscular Dystrophy Association, and the ALS Association, the Isis trial is the first time antisense oligonucleotides were delivered directly to the cerebrospinal fluid (CSF) of patients with SOD1 ALS.  It was also the first antisense oligonucleotide trial for a central nervous system disorder. The concept of “turning off” SOD1 in patients with this mutation led to the groundbreaking Phase I trial.  “What we developed is a way to turn off harmful genes, erase them essentially by deleting a small part of the RNA which then no longer makes this abnormal protein,” Miller states.  This approach will also likely be applicable to other diseases of the central nervous system.

The Phase I trial took place at four NEALS clinical research sites: Washington University in St. Louis, Massachusetts General Hospital, Johns Hopkins University, and Methodist Neurological Institute.  Study Coordination and Data Management was led by the MGH Neurological Clinical Research Institute (NCRI). A total of 21 participants were enrolled in the trial.  Further development of SOD1 antisense oligos for SOD1 familial ALS is ongoing.

About the Northeast ALS Consortium (NEALS)
The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. Their mission is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible.  NEALS has over 100 member sites in the United States, Canada, Ireland, and Israel.
www.alsconsortium.org

About the Neurological Clinical Research Insitute (NCRI) at Massachusetts General Hospital
The Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital accelerates translational research in neurological disorders through initiating and testing novel therapies.  The NCRI has an extensive history in leading clinical research to find new treatments for neurological diseases including Amyotrophic Lateral Sclerosis (ALS), myasthenia gravis, diabetic neuropathy, stroke, multiple sclerosis, Parkinson’s disease, and Huntington’s disease.
www.NCRInstitute.org

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The Robert A. Stehlin Campaign for ALS (R.A.S.C.A.L.S.) is an all-volunteer 501(c)(3) charity. 100% of all funds raised go to building awareness, treatment research and development, plus ALS family assistance. There are no administrative costs.

Contributions are tax-deductible.

You may also be interested in visiting the RASCALS Store.

The material presented here is for informational purposes only and should not be construed as medical advice, or relied upon as a substitute for medical advice from a health care provider.

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