Cytokinetics to Announce Results from BENEFIT-ALS at 66th Annual Meeting of the American Academy of Neurology

SOUTH SAN FRANCISCO, California,  April 22, 2014 — Cytokinetics, Incorporated announced today that results from BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) will be presented during a Platform Session scheduled at the 66th Annual Meeting of the American Academy of Neurology (AAN) to be held April 26 – May 3, 2014 at the Pennsylvania Convention Center in Philadelphia, PA. Cytokinetics also announced that additional poster presentations related to tirasemtiv and ALS will be presented at AAN.

Platform Session Presentation at the 66th Annual Meeting of the American Academy of Neurology

Title: The Effect of Tirasemtiv on Functional Status in Patients with ALS
Presenter: Jeremy M. Shefner, M.D., Ph.D
Date: Tuesday, April 29, 2014
Presentation Time: 4:15 PM (Eastern Time)
Session: S16.005 – 5LB.001 – Platform Session: Neuromuscular and Clinical Neurophysiology

About Tirasemtiv and BENEFIT-ALS

Tirasemtiv, a novel skeletal muscle activator, is the lead drug candidate from Cytokinetics’ skeletal muscle contractility program. Tirasemtiv selectively activates the fast skeletal muscle troponin complex by increasing its sensitivity to calcium and, in preclinical studies, demonstrated increases in skeletal muscle force in response to neuronal input and delays in the onset and reductions in the degree of muscle fatigue. In previously conducted Phase IIa clinical trials in patients with amyotrophic lateral sclerosis (ALS), tirasemtiv appeared generally well-tolerated, and demonstrated encouraging trends to improvement in patients’ functional abilities and increases in measures of respiratory and skeletal muscle strength and endurance.

BENEFIT-ALS is an international, double-blind, randomized, placebo-controlled, Phase IIb clinical trial which was designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv in patients with ALS. Patients enrolled in BENEFIT-ALS began treatment with open-label dosing of tirasemtiv at 125 mg twice daily. Patients who tolerated open-label treatment for one week were randomized to receive 12 weeks of double-blind treatment with twice-daily oral ascending doses of tirasemtiv or placebo, beginning at 125 mg twice daily and increasing weekly up to 250 mg twice daily (or a dummy dose titration with placebo). Clinical assessments occurred monthly during double-blind treatment; patients also returned for follow-up evaluations at one and four weeks after their final dose of double-blind study medication. The primary efficacy analysis of BENEFIT-ALS compares the mean change from baseline in the ALS Functional Rating Scale in its revised form (ALSFRS-R) on tirasemtiv versus placebo. Secondary endpoints include Maximum Voluntary Ventilation (MVV) and other measures of respiratory and skeletal muscle function and fatigability.

Additional Poster Presentations at the 66th Annual Meeting of the American Academy of Neurology

Title: Fast Skeletal Muscle Troponin Activator Tirasemtiv Increases Muscle Function and Performance in the B6SJL SOD1G93A ALS Mouse Model
Presenter: Fady I. Malik, M.D., Ph.D., F.A.C.C.
Date: Monday, April 28, 2014
Presentation Time: 3:00 PM (Eastern Time)
Session: P1.081 – Poster Session I: Anterior Horn Cell Disease: Pathogenesis and Pathology
Poster on Display: 3:00 PM – 6:30 PM (Eastern Time)

Title: Tirasemtiv Amplifies Skeletal Muscle Response to Nerve Activation in Humans
Presenter: Fady I. Malik, M.D., Ph.D., F.A.C.C.
Date: Wednesday, April 30, 2014
Presentation Time: 7:30 AM (Eastern Time)
Session: P4.077 – Poster Session IV: ALS: Trials and Biomarkers
Poster on Display: 7:30 AM – 11:00 AM (Eastern Time)

Title: Profile of Medical Care Costs in Patients with Amyotrophic Lateral Sclerosis in Medicare Program and Under Commercial Insurance
Presenter: Lisa Meng, Ph.D.
Date: Thursday, May 1, 2014
Presentation Time: 3:00 PM (Eastern Time)
Session: P7.102 – Poster Session VII: Neuromuscular Health Services/Outcomes Research
Poster on Display: 3:00 PM – 6:30 PM (Eastern Time)

About Cytokinetics

Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions. Cytokinetics’ lead drug candidate from its cardiac muscle contractility program, omecamtiv mecarbil, is in Phase II clinical development for the potential treatment of heart failure. Amgen Inc. holds an exclusive license worldwide to develop and commercialize omecamtiv mecarbil and related compounds, subject to Cytokinetics’ specified development and commercialization participation rights. Cytokinetics is independently developing tirasemtiv, a fast skeletal muscle activator, as a potential treatment for diseases and medical conditions associated with neuromuscular dysfunction. Tirasemtiv is currently the subject of a Phase II clinical trials program and has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency for the potential treatment of amyotrophic lateral sclerosis (ALS). Cytokinetics is collaborating with Astellas Pharma Inc. to develop CK-2127107, a skeletal muscle activator structurally distinct from tirasemtiv, for non-neuromuscular indications. All of these drug candidates have arisen from Cytokinetics’ muscle biology focused research activities and are directed towards the cytoskeleton. The cytoskeleton is a complex biological infrastructure that plays a fundamental role within every human cell. Additional information about Cytokinetics can be obtained at www.cytokinetics.com.

Forward-Looking Statements

This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements, and claims the protection of the Act’s safe harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements relating to planned presentations, and the properties and potential benefits of tirasemtiv and Cytokinetics’ other drug candidates. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to, Cytokinetics anticipates that it will be required to conduct at least one confirmatory Phase III clinical trial of tirasemtiv in ALS patients if supported by the BENEFIT-ALS data, which will require significant additional funding and it may be unable to obtain such additional funding on acceptable terms, if at all; potential difficulties or delays in the development, testing, regulatory approvals for trial commencement, progression or product sale or manufacturing, or production of Cytokinetics’ drug candidates that could slow or prevent clinical development or product approval, including risks that current and past results of clinical trials or preclinical studies may not be indicative of future clinical trials results, patient enrollment for or conduct of clinical trials may be difficult or delayed,Cytokinetics’ drug candidates may have adverse side effects or inadequate therapeutic efficacy, the U.S. Food and Drug Administration or foreign regulatory agencies may delay or limit Cytokinetics’ or its partners’ ability to conduct clinical trials, and Cytokinetics may be unable to obtain or maintain patent or trade secret protection for its intellectual property; Cytokinetics may incur unanticipated research and development and other costs or be unable to obtain additional financing necessary to conduct development of its products; Cytokinetics may be unable to enter into future collaboration agreements for its drug candidates and programs on acceptable terms, if at all; standards of care may change, rendering Cytokinetics’ drug candidates obsolete; competitive products or alternative therapies may be developed by others for the treatment of indications Cytokinetics’ drug candidates and potential drug candidates may target; and risks and uncertainties relating to the timing and receipt of payments from its partners, including milestones and royalties on future potential product sales under Cytokinetics’ collaboration agreements with such partners. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission.

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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.

Research and Markets: Global Amyotrophic Lateral Sclerosis Market Forecast 2014-2018

DUBLIN — Ireland-based reporting company Research and Markets has announced the addition of its report “Global Amyotrophic Lateral Sclerosis Market 2014-2018 with Covis Pharmaceutical, Eisai, GSK & Mitsubishi Tanabe Pharma Dominating.”

While these type reports are used for business and investment outlooks, it also offers to ALS survivors and families a window into the ALS treatment landscape of the near future.

Some of the report’s highlights:

One of the main drivers in this market is the increase in the aging population. The number of patients suffering from ALS is increasing due to its relatively high prevalence in the elderly population.

The acceptability of ALS drugs is expected to rise due to increasing public awareness of ALS and associated disorders. This is expected to result in increasing drug penetration. Rising general awareness about ALS has led to an increase in research funding and support for clinical and support services.

For instance, May is celebrated as ALS awareness month. The ALS Association strives to involve and engage patients and families in the process of increasing awareness and advocating for legislative changes. Similarly, CDMRP also organizes the ALS Research Program. The global research effort through this program has helped increase the number of scientists working on ALS, advanced new discoveries and treatments, and has shed light on the complex genetic and environmental factors involved in ALS.

Further, the report states that one of the main challenges is the patent expiry of Rilutek. The patent expiry of Rilutek, the only marketed drug for the treatment of ALS, will result in the loss of market exclusivity.

Key vendors dominating this space include:

Covis Pharmaceutical Inc.
Eisai Co. Ltd.
GlaxoSmithKline plc
Mitsubishi Tanabe Pharma Corp.

Other vendors mentioned in the report:

Amorfix Life Sciences Ltd.
BrainStrom Cell Therapeutics Inc.
Newron Pharmaceuticals S.p.A
Neuraltus Pharmaceuticals Inc.
Nutra Pharma Corp.
Oxford BioMedica plc
Pharnext SAS
Q-Therapeutics 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.

The RASCALS Board of Directors Salutes Its Remarkable RASCALS Volunteers

AJ and Di2

Robert Stehlin and the entire Board of Directors would like to once again recognize the tremendous efforts of our RASCALS Volunteer Army.

They are the unsung heroes in this tremendous struggle.

They show up without pay, perks, or public acknowledgement, always ready to pitch in with their time and energy and unbridled enthusiasm.

They come simply to make a difference in the lives of families battling this horrid disease.

And you certainly have.

On behalf of all ALS survivors, their families, and the community we serve,

THANK YOU!!!

th

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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.

The 2014 RASCALS Foundation Higher Education Scholarships

brainy bulbThe RASCALS Foundation is pleased to announce that it is once again offering  the 2014 Higher Education Scholarship Program to assist families affected by ALS.

We would like to acknowledge and thank our newest partner Helping Hands for ALS, a student group associated with John Burroughs High School for their generous participation in making these important awards possible.

The scholarships will be available nationwide to the first 100 qualifying applicants.

To request an application packet, simply email MichaelBrand314@gmail.com  or call 314-477-8120.

You may also check the website  for guideline information to be posted in a dedicated 2014 Scholarship section.

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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.

Q Therapeutics Issues Formal Update

SALT LAKE CITY, April 21, 2014  —  Q Therapeutics, Inc. (“Q”), an emerging biotechnology company developing innovative cell therapy products for the treatment of neurodegenerative diseases, today issued a formal corporate update relating to the Company’s progress on advancing its pre-clinical research and development efforts. Q’s President and Chief Executive Officer, Deborah Eppstein, PhD, stated:

“2013 was a productive year for Q Therapeutics on many fronts — and we’ve continued to gain momentum in 2014. Of particular note is the progress we’re making in moving towards the IND filing and clinical studies using our proprietary, patented Q-Cells® as a potential therapy for Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig’s disease). In late 2012, we commenced formal GLP animal safety studies at MPI Research, and recently completed the in-life portion of those safety, biodistribution and tumorigenicity studies. We expect to complete the full data analysis of this study within the next six months. The interim data show no safety concerns attributed to Q-Cells, thereby meeting the safety milestone for the multi-year National Institutes of Health (NIH) grant that supports this work. We are optimistic that the fourth and final tranche of funding of this milestone-driven grant will be awarded to support completion of IND-enabling studies required by the FDA for clearance to begin clinical trials.

“Late in the third quarter of 2013, the FDA granted Orphan Drug Designation to Q-Cells for the treatment of ALS. This designation, given to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 patients in the United States, enables companies to benefit from certain financial incentives, including seven years of market exclusivity after product marketing clearance, access to federal grant funding opportunities to defray clinical trial costs, and assistance with clinical protocols and federal tax credits. Orphan Drug Designation represents a significant milestone for ALS patients and for our Company as we advance this potentially life-saving therapeutic. We plan to apply for similar available designations in Europe and Japan.

“We have advanced our cell manufacturing efforts in partnership with the University of Utah’s Cell Therapy and Regenerative Medicine Facility, focused on documenting Q-Cells’ manufacturing platform in accordance with FDA guidelines — documentation that also will support IND filing and clinical trials.

“We continue to work closely with our academic collaborators. For our initial clinical trials in ALS, these include Nicholas Maragakis, MD at The Johns Hopkins University School of Medicine and Jonathan Glass, MD, and Nicholas Boulis, MD at Emory University School of Medicine, who are all working to further define and develop clinical protocols for the ALS trials. Dr. Boulis is also working with us to design and conduct a short-term safety study of Q-Cells in mini-pigs, using the injection platform for cell delivery that we intend to employ in clinical trials. This important safety study is being conducted by MPI Research to document compatibility between Q-Cells and the injection platform, and its effectiveness in implanting those cells. Studies led by Dr. Piotr Walczak at The Johns Hopkins University continue to demonstrate and validate the benefits of Q-Cells in myelination deficiency models. We expect Dr. Walczak’s work to support our future studies in other indications, including transverse myelitis, multiple sclerosis and stroke.

“I’m pleased to report that we have also advanced the preclinical study of Q-Cells as a regenerative therapy for the treatment of traumatic spinal cord injuries. Dr. Itzhak Fischer’s laboratory at Drexel University is engaged in demonstrating the benefits of Q-Cells for this therapeutic application, and in June 2013 published positive study results in the peer-reviewed Journal of Neurotrauma.

“We also formed exciting new scientific collaborations with XCell Science, Inc. to develop manufacturing technologies for new neural cell products derived from induced pluripotent stem cell (iPSC) lines. We expect that will enhance our proprietary product portfolio with new tissue sources and allow us to identify novel and impactful new therapeutic targets in the neurodegeneration area.

“Finally, we continued to prosecute our intellectual property portfolio, bringing the number of issued patents to 18 covering neural lineage cells, in addition to two in-licensed patents concerning the spinal injection platform.

“On the corporate side of our business, we have continued to dedicate effort to securing growth capital necessary to fund Q’s ongoing R&D efforts. Since March 7, 2014, we have closed on $4.4 million with several strategic and financial investors. We expect to seek additional funding to support further development of our product candidates, including initiation of our clinical trial plans for those candidates.

“In closing, I’d like to acknowledge that our leadership team has been enhanced notably with the return of Q’s Scientific Founder, Mahendra Rao, MD, PhD, in the roles of Chief Strategy Officer and Scientific Advisory Board Chair. Q Therapeutics remains committed to optimizing and realizing the value we have created — and continue to enhance — in our Company and in our Q-Cells platform. We are highly optimistic about being in position to submit an IND to the FDA later this year and proceeding to clinical trials in relatively short order,” concluded Dr. Eppstein.

About Q Therapeutics, Inc.
Headquartered in Salt Lake City, Utah, Q Therapeutics, Inc. is a fully reporting, non-trading company engaged in developing adult stem cell therapies to treat debilitating diseases of the central nervous system. The Company’s first product, Q-Cells®, is a cell-based therapeutic intended to restore or preserve normal activity of neurons by providing essential support functions that occur in healthy central nervous system tissues. Q-Cells may be applicable to a wide range of central nervous system diseases, including demyelinating conditions such as multiple sclerosis, transverse myelitis, cerebral palsy and stroke; as well as other neurodegenerative diseases and injuries, such as ALS (Lou Gehrig’s disease), Huntington’s disease, spinal cord injury, traumatic brain injury, Parkinson’s disease and Alzheimer’s disease. Q Therapeutics’ initial clinical target is ALS, with a first IND submission expected in 2014. The Company’s proprietary product pipeline also encompasses neural cell products derived from induced pluripotent stem cells (iPSC). For more information, visit www.qthera.com.

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 Q Therapeutics’ 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 its 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 results and other risks and uncertainties are detailed from time to time in Q Therapeutics’ periodic reports, including the Company’s Annual Report on Form 10-K for the year ended December 31, 2013.

FOR MORE INFORMATION:
Hanover|Elite
Erin Palmer
Director of Client Relations
407-585-1080
Email Contact

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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.

Mayo Clinic Researchers Place ALS Hope in Stem Cells

Posted by Jeff Hansel, for the PostBulletin, April 19, 2014 •

Seventy-five years after baseball player Lou Gehrig was diagnosed with ALS, Mayo Clinic researchers have begun a human study hoping to slow progression of the devastating ailment.

ALS, or amyotrophic lateral sclerosis, has become widely known as Lou Gehrig’s disease.

The catastrophic diagnosis carries a crushing prognosis for both the patient and those around him.

Gehrig, for instance, only lived two years after he was diagnosed. But the new treatment offers the hope of extending the life of those with ALS.

New York Yankees first baseman Lou Gehrig, the "Iron Horse," wipes away a tear while speaking during a sold-out tribute at Yankee Stadium on July 4, 1939. Gehrig's record breaking career was cut short by neuromuscular disease. (Photo: AP/Murray Becker)

New York Yankees first baseman Lou Gehrig, the “Iron Horse,” wipes away a tear while speaking during a sold-out tribute at Yankee Stadium on July 4, 1939. Gehrig’s record breaking career was cut short by neuromuscular disease. (Photo: AP/Murray Becker)

“Lifespan after diagnosis is roughly two to three years, but there’s a huge range,” said Mayo neurologist Dr. Nathan Staff. Affected individuals can survive more than a decade after diagnosis, or die within six months.

Brian McCoy, a Rochester carpenter, is one of the first 25 people ina Phase I ALS stem-cell clinical trial at Mayo in Rochester.

“I have nothing to lose,” said McCoy, who was diagnosed in October, 2012.”I don’t think it will help me. But, down the road, it might help somebody.”

The trial is a safety study to determine if it’s safe to give ALS patients injections of stem cells grown from samples of their own fat tissue.

If the process is indeed safe, the study will be expanded to more patients. That will allow researchers to further study both safety and effectiveness in a Phase II randomized, double-blind, placebo-controlled clinical trial.

“We don’t know if it will work or not,” said Dr. Anthony Windebank, deputy director for discovery in the Center for Regenerative Medicine at Mayo. “It may even do harm.”

If the treatment is both safe and effective and has a marked impact on survival, it would be the first body-heal-thyself treatment designed specifically to slow ALS progression.

More than 700 people with ALS have expressed interest in joining one of the Mayo studies, illustrating the tragic reach of the disease.

Currently there’s only one main drug that extends the life of ALS patients. But on average, it only adds about two months to lifespan.

And that short time, McCoy says, just isn’t enough.

Mayo researchers dream of adding a new treatment tool to fight the disease directly, instead of fighting symptoms.

The ALS Association says “progressive degeneration of the motor neurons in ALS eventually leads to their death.”

When the neurons die, “the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed.”

The chance to actually make a difference in survival and quality of life for people who otherwise have no options, Windebank said, is “what really make one want to get up in the morning and go to work — you’re working on something that has the potential to make a world of difference.”

Health reporter Jeff Hansel writes the Pulse on Health column every Monday. Follow him on Twitter @JeffHansel.

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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.

Final Results of ALS Phase I Stem Cell Trial Published in Annals of Neurology

NEURALSTEM, INC. LOGO

A Press Release from Neuralstem Inc.

ROCKVILLE, Maryland, March 17, 2014 (PRNewswire) — Neuralstem, Inc. announced that the final results from the Phase I safety trial using NSI-566 spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) were published in the peer-reviewed journal, Annals of Neurology. In “Intraspinal Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis: Phase I Trial Outcomes,” results were updated from Phase I interim data, reported earlier, to include data from the last six patients in the trial. These six patients were the first to receive cervical stem cell transplants. Three of them were also the first to be transplanted along the length of their spines, in both the lumbar and the cervical regions.

The results showed that NSI-566 human spinal cord stem cells can be safely transplanted in both the lumbar and cervical spinal cord segments, did not accelerate disease progression, and warrant further study on dosing and therapeutic efficacy. Furthermore, the researchers were able to identify potential therapeutic windows, suggesting that more injections, as well as multiple injections, are better and may increase both the length and the magnitude of the potential benefits. This is consistent with the hypothesized neuroprotective mechanism-of-action for this cell therapy.

Since concluding Phase I, the trial has progressed to Phase II at three centers, Emory University Hospital in Atlanta, Georgia, the ALS Clinic at the University of Michigan Health System, in Ann Arbor, Michigan, and Massachusetts General Hospital in Boston, Massachusetts, which treated its first patient in February. Treatment of three of the five Phase II cohorts has been completed.

“Although this was a Phase I trial, and functional outcome data were collected for the purpose of assessing safety, we performed secondary analyses of these data as a means to gain insight into how cellular transplantation affected disease progression rates and to inform outcome assessment approaches in future trial phases,” said Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute, Director of Research of the ALS Clinic at the University of Michigan Health System, principle investigator for the NSI-566/ALS trial and lead author. Dr. Feldman is also an unpaid consultant to Neuralstem.

“Pre-surgical disease progression rates for the various functional outcome measures were calculated to create slopes for each patient, so that we could determine if post-surgical data points, at 6, 9, 12 and 15 months, improved relative to predicted points. We also did analyses to determine which, if any, functional outcome assessment most closely correlated with the overall ALSFRS-R scores,” said Dr. Feldman. “Comparison of the outcome data to predicted outcome points in group E (patients who received both lumbar and cervical injections) revealed improvements in a significant number of measures at 6, 9, 12 and 15 months post-surgery. Overall, 50% of the patients in the trial showed improvement across multiple clinical measures at the same time points. We also found that a measure of grip strength correlated most closely with the overall ALSFRS-R scores.

Dr. Feldman added, “Finally, we conducted an analysis to identify the most biologically active period of the injected cells for the patients receiving both lumbar and cervical injections. This analysis reveals that the maximal periods of benefit correlate with the two surgical interventions. Importantly, as the ‘bell-shaped curve’ associated with each intervention is likely due to disease progression, increasing the total cell dose, and applying multiple applications of these stem cells, may increase both the length and magnitude of the potential benefit. We are of course exploring this very dosing regimen in our ongoing Phase II trial.”

“The completion of this Phase I study is a major milestone for the testing of intraspinal stem cell therapy for ALS,” said Jonathan Glass, MD, Professor of Neurology and Pathology, Emory University School of Medicine and Director of the Emory ALS Center, site principal investigator and a senior study author. “We have now shown that the procedure is safe for both lumbar and cervical injections, allowing us to move forward with an aggressive program to test whether this treatment will improve the course of disease for patients with ALS.”

“This peer-reviewed article is the first such report of cervical and dual-targeted intraspinal transplantation of neural stem cells in ALS subjects,” said Karl Johe, PhD, Neuralstem’s Chairman of the Board and Chief Scientific Officer. “We believe our cells offer a means to replace lost cells, provide neurotrophic support, and improve the diseased microenvironment.  This study demonstrates these factors, and that the cells and the novel surgical route of administration are safe and well-tolerated. Our ability to directly inject cells into the cervical regions of the spinal cord represents a significant advance in the field of cell therapy.

“We would like to thank the incredible teams at both Michigan and Emory who made this study possible, and who continue working with us today in our ongoing Phase II trial. We’d like to give special thanks to Dr. Jonathan Glass, Director of the Emory ALS Center, the Emory site principle investigator, and Dr. Nick Boulis, Associate Professor of Neurosurgery at Emory School of Medicine, the surgeon for all of the Phase I surgeries, and the inventor of the spinal-mounted stabilization and injection platform and floating cannula surgical devices used to deliver the cells,” concluded Dr. Johe.

About Neuralstem

Neuralstem’s patented technology enables the production of neural stem cells of the 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 glial cells. Neuralstem’s NSI-566 spinal cord-derived stem cell therapy is in Phase II clinical trials for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig’s disease. 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 and ischemic stroke. The company has received FDA approval to commence a Phase I safety trial in chronic spinal cord injury.

Neuralstem also maintains the ability to generate stable human neural stem cell lines suitable for 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 neurons, possibly reversing pathologies associated with certain central nervous system conditions.  The company has completed a Phase I safety trial evaluating NSI-189, its first neurogenic small molecule product candidate, for the treatment of major depressive disorder (MDD). Additional indications might include traumatic brain injury (TBI), Alzheimer’s disease, and post-traumatic stress disorder (PTSD).

For more information, please visit www.neuralstem.com or connect  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, 2013.

SOURCE: Neuralstem, Inc.

For further information: Planet Communications – Media Relations: Deanne Eagle 917.837.5866; MDC Group – Investor Relations: Susan Roush 747.222.7012, David Castaneda 414.351.9758

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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.

RASCALS 5K POSTPONED :(

Due to unforeseen scheduling conflicts, the RASCALS Foundation regrets to announce that its 3rd Annual 5K Walk/Run on May 4th has been postponed to a later date.

We are, of course, very disappointed and sincerely apologize for any inconvenience to our volunteer race staff and participants.

The decision was not as easy one.

Reimbursements will be processed as soon as possible, and a new date for the 5K will be announced in the near future.

Thank you for your understanding and continued support.

Above and Beyond: Jon Imber

Film Review: ‘Jon Imber’s Left Hand’ an Extraordinary Documentary

By Daniel Kany for the Portland Press Herald, April 5, 2014 •

Jon Imber, who is fighting amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, was photographed in his studio last fall for a profile by Telegram arts writer Bob Keyes. Telegram file photos by Gregory Rec/Staff Photographer

Jon Imber, who is fighting ALS, was photographed in his studio last fall for a profile by Telegram arts writer Bob Keyes.
Telegram file photos by Gregory Rec/Staff Photographer

“Jon Imber’s Left Hand” opens with the 63-year-old artist standing in front of a canvas with two helpers. He can hardly move and barely hold a brush. His speech has withered to grunts. The scene was shot on Feb. 10, 2014.

In the fall of 2012, Imber was diagnosed with amyotrophic lateral sclerosis, or ALS, the fatal degenerative condition also known as Lou Gehrig’s disease.

The next scene shows a robust and articulate Imber talking about art. He is friendly, forceful and witty. Fully aware of his prognosis but not yet seriously limited by the disease, Imber reveals his destiny: “I have just months.”

Months to live? Or months to paint?

This is the question underlying director Richard Kane’s bittersweet and deeply moving film, which premiered last week during the Maine Jewish Film Festival and is the latest in the superb Maine Masters series produced by the Union of Maine Visual Artists.

“Jon Imber’s Left Hand” picks up at the start of what Imber calls “my ALS summer.” It is a story about the artist and his wife, the painter Jill Hoy. The pair for decades have been key members of the Stonington and Maine painting communities. While Imber is the object of the film, Hoy is its true subject. We see Imber lean more and more on his loving wife. And while we watch him become a prisoner of his own failing body, it is Hoy’s heart that we feel breaking.

For Imber, the diagnosis is an urgent challenge: It motivates him to spend every last moment living.

And for Imber, art is life.

The film features a painting session in which ALS has forced Imber to switch to his left hand. Almost defiantly, Imber’s two-inch house brush glides with drippy-wet paint, boldly swishing and diving over a tall, slender canvas. Within minutes, the painting is beautiful – too beautiful.

So Imber flips over the canvas and goes back to work.

A recent portrait by Stonington painter Jon Imber.

A recent portrait by Stonington painter Jon Imber.

This reveals Imber’s goal, not to accurately paint the vase of flowers before him, but to make a good painting. For him, a satisfying outcome needs to resolve tension. Anything facile is too easy to be interesting, and this is why Imber’s ALS-forced switch leads to such surprisingly strong work.

While many painters are not used to people looking over their shoulder as they work, Imber, an extroverted teacher, is accustomed to an audience and to thinking out loud. This makes “Jon Imber’s Left Hand” an extraordinary document about both ALS and the artistic process.

Imber is a natural narrator, so Kane lets his voice set the pace of the film. Gallery reception conversations are buoyant and jaunty. Imber’s comments to crowds are funny and forceful but punctuated with time to look. And his intimate conversations are generous and patient.

The film exudes the artist’s credo: To paint is to live

Despite the insightful comments from an impressive cadre of colleagues, critics and curators, Kane lets the key questions and conclusions come from Hoy and Imber. Deep into his ALS, Imber is more dedicated to being a parent than painting (their son is a student at Bates College). But Hoy is persuasive when she finally lets the tears flow, saying “painting is his life.” She believes that, when he can no longer paint, Imber will depart. So Hoy does everything possible to keep him painting.

Imber knows his legacy will live on through his family and among the students he taught over 27 years at Harvard. And it will ride the ages in the scores of paintings we see throughout the film. Moreover, Imber has the measure of his post-diagnosis work: His newest abstract landscapes are as strong as anything he ever painted. And he knows it.

Kane shows Imber talking through his relationships with powerful figures such as his father and his teacher Philip Guston. (Despite Guston’s oversized role in Imber’s life, the key unspoken comparison of the film is with Willem deKooning both because of his approach to painting and the famous effect of Alzheimer’s on his career.)

Chatting over family photos, Imber claims his Judaism had little to do with his life and work, yet again the insightful Hoy persuasively disagrees. While this poignant scene binds the film brightly to the Maine Jewish Film Festival, it’s a family moment with which everyone can identify: It’s often easier, after all, for others to see our family in us.

The film closes with a return to the scene shot last month in Somerville with Imber heroically struggling against his failing body to paint – to live.

A masterpiece of intimacy in the face of tragedy, “Jon Imber’s Left Hand” is an extraordinary accomplishment in film. It is the eulogizing of the creative force and artistic life of one of America’s leading painters – in his own vibrant voice.

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Freelance writer Daniel Kany is an art historian who lives in Cumberland. He can be contacted at: dankany@gmail.com

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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.

Why is Lou Gehrig’s Disease Closely Related to Veterans?

gulf-war-syndrome

By S. E. Smith at Care2, April 11, 2014 •

Something is eating away at the nervous systems of military veterans like Thomas Corbett, Jeff Long, and Mike White. It started with some tingling and numbness, difficulty walking and poor hand coordination, and it progressed to stumbling and an increasingly limited range of motion.

It’s called amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, after the baseball player who made it famous (though he’s not the only famous patient — Stephen Hawking also has the condition). ALS attacks the motor neurons in the brain and spinal cord responsible for controlling muscle movement, slowly paralyzing patients over time.

As the disease progresses, they start to need assistance walking, finally needing a wheelchair for mobility. Eventually, patients need assistance with even basic daily tasks, including grooming, using the bathroom and eating. Many patients wind up on ventilators because they are no longer able to breathe independently, with pneumonia and other lung infections being a common cause of death as their immune systems decline and their limited mobility makes it challenging to fight off infections.

Depending on the progression of the disease and the care the patient receives, it can move quickly and ruthlessly, or more slowly. And while a very small percentage of cases appear to have a genetic component, others appear out of the blue.

Still, none of this explain why military veterans, particularly those who served in the First Gulf War, are twice as likely to get it when compared to the rest of the population. Since 2008, the Veterans Administration has recognized that there’s a clear link between ALS and military service, and the agency considers it to be a service-connected disability, meaning that service either causes or exacerbates it. This makes veterans eligible for comprehensive assistance from the VA, but it still doesn’t address the root question: where are all these cases of ALS coming from?

An estimated 2,000 veterans live with ALS today, while as many as 30,000 people overall have the disease. It’s an extremely common motor neuron disease, making it a prime subject for research and development, yet scientists have been unable to pin down specific environmental causes. One study found a possible link between ALS and recreational athletics — the condition is seen in soccer and NFL players, for example, and Iraq veterans tend to have injuries similar to those observed in athletes. It could also be environmental, related to chemical exposure or other factors.

Most likely, it’s a combination of factors that causes proteins to go rogue and start attacking motor neurons. Therapy for ALS is focused on developing treatments that target these proteins, or take another tack, using stem cells to replace damaged motor neurons. While these treatments in development are good news for current patients, everyone is still waiting for the answer to the more fundamental question about the origins of ALS, and whether it could be prevented or mitigated.

Numerous illnesses appear to have mysterious connections to service in the Gulf, including Chronic Fatigue Syndrome and so-called “Gulf War Syndrome.” This could be due to chemical weapons and other toxins that may have been present in the environment, or it could have been caused by the military’s own materials. It’s possible that even the vaccines used might have created problems for servicemembers. Whatever’s causing the increase in neurological diseases, among others, hasn’t been narrowed down, but it’s very real for the veterans struggling with these conditions.

Can researchers and the VA pin down the origins of ALS? It might be important not just for veterans but for civilians, especially those in war zones who may be exposed to the same environmental factors that veterans interact with during their service.

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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.