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

Via Scoop.it – ALS Lou Gehrig’s Disease
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.

Via www.businessweek.com

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

Via Scoop.it – ALS Lou Gehrig’s Disease
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.

Via alsn.mda.org

Hopkins Launches New ALS Research Center with $25 Million Gift

Via Scoop.it – ALS Lou Gehrig’s Disease
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.”

Via www.hopkinsmedicine.org

ALS Drug Discovery Meeting Forges Partnerships for Faster Drug Development

Via Scoop.it – ALS Lou Gehrig’s Disease
For three long and intense days in early March, The ALS Association brought together more than 120 researchers, drug developers, government officials, and others to brainstorm ways to accelerate drug discovery for the treatment of ALS. The meeting was packed with presentations, discussions, and ideas for collaboration to solve what everyone recognized is a difficult problem. Although participants didn’t minimize the difficulty of the challenge, they expressed optimism that progress is being made. “The last eight months have been the most exciting in the history of ALS genetics,” according to Don Cleveland, Ph.D., Professor of Medicine, Neurosciences, and Cellular and Molecular Medicine at the University of California at San Diego. The discovery of the C9ORF72 gene mutation, which is responsible for more than one quarter of familial ALS, has set the field abuzz with new ideas for understanding the causes of the disease. In addition, the newly discovered ubiquilin2 gene emphasizes the importance of protein aggregation as a likely mechanism in the disease. Other new findings have also been recognized for their potential importance in understanding the causes of ALS and for pointing to likely targets for drug development. These include excitotoxicity, toxic accumulation of RNA and inflammation. “The excitement is palpable among ALS researchers that we are getting closer to truly understanding the process of the disease,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., who organized the meeting. “That understanding is crucial to developing treatments that have the best hope for slowing or stopping ALS.”

Via www.alsa.org

CK-357, helping pALS live strong?

Via Scoop.it – ALS Lou Gehrig’s Disease
In people with ALS, the diaphragm and intercostal muscles gradually weaken often leading to respiratory distress and failure. In hopes to keep these muscles moving, Case Western University School of Medicine surgeon Raymond Onders MD FACS introduced a device, now approved by the FDA for people with ALS with breathing difficulties, called the NeuRX DPS which may boost the stamina of these muscles. But researchers from San Francisco’s Cytokinetics Inc. think that they might have a simpler solution: the experimental drug CK-2017357 (CK-357). Introduced in 2008, CK-357 might increase the strength of certain skeletal muscles including those needed for breathing. Now, Cytokinetics scientists reveal just how CK-357 works: the drug promises to make the most of weakening neuromuscular junctions by helping fast twitch fibers in skeletal muscles hold on to calcium, enabling more powerful contractions. This so-called fast skeletal muscle is needed in part, to maintain a healthy breathing rate. The Cytokinetics’ team anticipates that the drug could be beneficial in the treatment of number of neuromuscular diseases including ALS. Physicians are currently evaluating the safety and tolerability of multiple doses of CK-357 in people with ALS. The multi-institutional US team, led by State University of New York neurologist Jeremy Shefner MD PhD, are also checking for improvements in patients’ muscle function including breathing ability. The two 14 day placebo-controlled phase II clinical trials are expected to be completed by the end of March 2012. About 48 ALS patients are participating.
Via blogs.als.net

Cytokinetics ALS drug wins ‘orphan’ status in Europe

Via Scoop.it – ALS Lou Gehrig’s Disease
European regulators granted “orphan” status to a potential treatment for Lou Gehrig’s disease developed by South San Francisco’s Cytokinetics Inc. CK-2017357, which already has orphan drug designation in the United States, activates a muscle protein, called troponin, that could improve ALS patients’ ability to breathe as well as walk.
Via www.bizjournals.com

Cytokinetics Announces Fundamental Research in the Field of Fast Skeletal Muscle Activation Published in the Journal Nature Medicine

Via Scoop.it – ALS Lou Gehrig’s Disease
Cytokinetics, Incorporated announced today the publication of preclinical research regarding the activation of the troponin complex of fast skeletal muscle by its drug candidate CK-2017357 and the potential therapeutic role that this novel mechanism may play for patients with neuromuscular disorders. This publication in the March 2012 issue of the journal Nature Medicine reveals the mechanism of action for CK-2017357 and the scientific rationale for directly modulating fast skeletal muscle contractility as an innovative therapeutic strategy for improving physical activity in diseases in which neuromuscular function is compromised. “We are honored to have Cytokinetics’ novel scientific research into direct modulators of the skeletal muscle contractile apparatus published in this prestigious journal,” stated Fady I. Malik, MD, PhD, FACC, Cytokinetics’ Vice President of Biology and Therapeutics and senior author of this report. “This publication summarizes pioneering work performed by our dedicated research team that has supported the progression of CK-2017357 into Phase II clinical development for the potential treatment of patients with neuromuscular diseases.” The publication, titled “Activation of Fast Skeletal Muscle Troponin as a Potential Therapeutic Approach for Treating Neuromuscular Diseases,” discusses the potential clinical role for therapies that directly activate troponin in fast skeletal muscle. CK-2017357 is currently the subject of a Phase II clinical development program and has been granted orphan-drug designation by the U.S. Food and Drug Administration for the potential treatment of ALS, a debilitating disease of neuromuscular impairment.
Via www.cytokinetics.com

The ALS Association and the Packard Center Partner to Develop Animal Model Systems for Most Common Cause of Familial ALS

Via Scoop.it – ALS Lou Gehrig’s Disease
The ALS Association and the Robert Packard Center for ALS Research at Johns Hopkinshave entered into a partnership to expedite the development of animal model systems to expand the knowledge about the C9ORF72 gene, which has been identified as the most common cause of inherited amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease) and Frontotemporal dementia (FTD).“The Association is very pleased to partner with the Packard Center to expedite these important studies,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D. “The Association along with the Packard Center have both invested significant funds into the identification of this new gene, and we are pleased to be able to work together to support the critical next steps to ensure that possible discoveries from these projects are translated as rapidly as possible into therapies for ALS. In October, 2011, a large expansion of a hexanucleotide GGGGCC repeat was discovered in the C9ORF72 gene, but how the expansion causes malfunction of the nerve cells in ALS and FTD remains unknown. It is thought that the messenger RNA (mRNA) derived from this large repeat aberrantly accumulates. This scenario is reminiscent of what is known in other diseases caused by expanded repeats, especially myotonic dystrophy. Building on that example and in partnership with Isis Pharmaceuticals, the Cleveland Laboratory in San Diego, Calif., has designed a gene silencing approach to develop a drug called an antisense oligonucleotide (ASO) that will selectively destroy the ALS-causing mRNA with the expanded repeat. Essential for drug development is a mouse model expressing the expanded human C9ORF72 mRNA. The investigators will build these models and use them to validate efficacy of the ASO drug.
Via www.alscenter.org

ALS Clinical Research Update – February 2012

Via Scoop.it – ALS Lou Gehrig’s Disease

The pace of (ALS) discovery is increasing. While Riluzole is currently the only approved drug for ALS, many other treatments are being investigated for their potential roles in ALS treatment. Researchers are improving upon clinical trial design and dosage; they are learning from past trials and using this information to design trials that are faster, more precise, and more appealing to participants. What is coming down the ALS research pipeline?
Via campaign.r20.constantcontact.com

Disappointing news from the UK based lithium clinical trial

Via Scoop.it – ALS Lou Gehrig’s Disease
Results from a recent clinical trial into motor neurone disease (MND), involving people with MND in England have been announced and show that lithium carbonate is ineffective at treating the disease. The trial, known as the ‘LiCALS trial’, was co-funded by the Department of Health and the MND Association. It tested for safety and effectiveness (efficacy) of lithium in 215 patients with ALS* across England. While it has been shown to be safe and well tolerated, it did not demonstrate a significant increase in survival, compared to patients receiving the placebo (dummy drug) together with riluzole. The lithium clinical trial principal investigator, Prof Ammar Al-Chalabi from King’s College London said: We are most grateful to all of the participants for taking part in the LiCALS study. Although the results are disappointing, it is thanks to the dedication and commitment of those that took part in LiCALS that the trial has allowed the development of a trials network of ten centres in the UK, which will be of great benefit for rapidly testing future therapies. For that we are extremely grateful.
Via www.mndassociation.org