The Myelin Repair Foundation (MRF), which was the only non-profit organization named among the top ten biotech companies by Fast Company magazine (in 2011), has announced its entry into a Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health (NIH) to assess MRF-008, as a potential therapeutic for multiple sclerosis (MS).
The MRF is a Silicon Valley-based nonprofit research organization accelerating the development of therapeutics that repair myelin, the protective sheath surrounding nerves that is damaged in people with multiple sclerosis.
This CRADA will facilitate collaboration between the MRF and National Institute of Neurological Disorders and Stroke (NINDS) at the NIH Clinical Center to study MRF-008 — a drug identified by the MRF as a potential neuroprotective therapeutic to enhance repair in multiple sclerosis patients.
Scott Johnson, CEO, President and Founder of the Myelin Repair Foundation in a MRF release, hails the research collaboration with the NIH as an exciting achievement and powerful new beginning, that marks another major milestone to advance one of MRF’s potential compounds into clinical development.
Mr. Johnson says this compound — referred to as MRF-008 — is a generic drug the MRF’s academic research consortium has identified as a potential candidate to protect and repair myelin in MS patients. “Since MRF-008 is already FDA-approved for another disease indication,” he notes, “the MRF and the NIH see a tremendous opportunity to investigate the repurposing of this drug to protect and repair myelin in multiple sclerosis patients. Utilizing our Accelerated Research Collaboration (ARC) model, the MRF accelerated the advancement of MRF-008 from basic research through the translational gap, into clinical development. Joining forces with clinicians from the National Institute for Neurological Disorders and Stroke, we will devise an innovative clinical development strategy to investigate MRF-008 for MS patients.”
“Our partnership with the NIH will advance the clinical development of MRF-008,” says Mr. Johnson, “while addressing our overarching goal to satisfy unmet medical needs for patients with MS. We cannot disclose the name of the MRF-008 therapeutic yet, but we look forward to sharing exciting updates about this potential opportunity.”
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Through its Accelerated Research Collaboration (ARC) model, a collaborative research model designed to accelerate promising therapeutics to market, the Myelin Repair Foundation will work closely with the NIH to assess MRF-008 as a therapeutic candidate in an MS clinical trial. MRF-008 is a generic FDA-approved compound for the treatment of hypertension identified by the Myelin Repair Foundation’s academic research consortium as a novel drug repurposing candidate for neuroprotection to stimulate MS repair. Dr. Irene Cortese, M.D. and Dr. Daniel Reich, M.D., Ph.D. will lead the research study at the NIH.
There is increasing focus on the concept of remyelinization as a potentially effective treatment for MS. As fore-noted, myelin is the substance that forms a protective and insulating lining around nerve fibers, and is damaged by multiple sclerosis, leukodystrophy and the many other demyelinating disorders. On Wednesday, January 29th, 2014, The Myelin Project in a press conference at UC Davis’ Children’s Hospital in Sacramento, California introduced a historic Bill (AB1559) for mandatory adrenoleukodystrophy (ALD) newborn screening in the state of California. The bill is sponsored by state assemblyman and chair of Assembly Committee on Health Dr. Richard Pan, who is a pediatrician and understands the necessity of newborn screening to detect and and treat ALD effectively. The Myelin Project is a 501(c) international medical research organization that has been funding myelin research across the globe and inducing international scientific collaboration for over two decades.
A free access paper published online last last year by Springer Open Choice reviews the current state of knowledge of remyelination in multiple sclerosis, concentrating on advances in the understanding of the pathology and the regenerative response, and summarizes current progress on development of new therapies to enhance remyelination aimed at reducing progressive accumulation of MS disability
The paper, entitled: “Promoting Remyelination in Multiple Sclerosis – Recent Advances,” (doi: 10.1007/s40265-013-0146-8 PMCID: PMC3853368 Published online 2013 November 16) is co-authored buy E. Jolanda Münzel and Anna Williams of the MRC Centre for Regenerative Medicine, University of Edinburgh at Edinburgh, U.K. discusses key target pathways identified in experimental models, noting that while most identified targets have not yet progressed to the stage of being tested in human clinical trials, they may provide treatment strategies for demyelinating diseases in the future. Drs. Münzel and Williams also discuss some of the problems associated with testing this class of drugs, where they might fit into the therapeutic arsenal and the gaps in our knowledge.
They note that the pathological hallmarks of all MS subtypes are focal areas or ‘plaques’ of demyelination in the Central Nervous System (CNS), with surrounding inflammation and neurodegeneration. MS, classically thought of as an autoimmune disease, is the most common cause of non-traumatic disability in young people, affecting approximately 2.5 million people worldwide, and that despite much research effort, the aetiology is not fully understood, and may include genetic susceptibility, epigenetic regulation and environmental factors, and that different phases of the disease reflect the underlying pathology, which is initially (in the relapsing and remitting phase) more inflammatory and demyelinating in nature, then (in the progressive phase) progressing to become more neurodegenerative. The researchers observe that all current medications used to treat MS in clinical practice act primarily on modulating or suppressing the immune system to prevent further relapses, but do not delay or slow disease progression, or affect remyelination. These currently licensed disease-modifying drugs for MS are anti-inflammatory; ie: suppress the immune system and reduce relapse rates, but are ineffective in progressive disease, and there are as yet no progressive disease-modifying drugs to slow, stop or reverse neurodegeneration.
Drs. Münzel and Williams suggest that rather than an autoimmune disorder, MS might be instead a primarily neurodegenerative disease. They note that neuroprotective drugs have been sought for many other neurological diseases, including stroke, spinal cord injury and Alzheimer’s disease, with limited success, but an alternative strategy is available in MS, where neuroprotection may be enhanced through myelin repair – ergo: remyelination. In their review, Drs. Münzel and Williams outline recent advances made in current understanding of how myelin is lost in MS, what prevents remyelination in humans from being efficient, and how it might be possible to stimulate and manipulate remyelination to enhance it to treat progressive MS. They note that currently several drugs are under review as potential myelin repair and regeneration treatments including Alemtuzumab. Dimethyl Fumarate. Anti-LINGO-1 Antibodies, and Human Monoclonal IgM Antibody 22.
“As a non-profit organization beholden to patients, not profits, we are uniquely positioned to advance MRF-008, a generic drug identified by the MRF academic consortium, forward as a novel therapeutic candidate to stimulate repair for multiple sclerosis,” says Mr. Johnson. “With the NIH’s eminent expertise in MS clinical trials, we have found an exemplary partner to conduct the research necessary to assess MRF-008. With world-class advisors, academic scientists, industry partners and this opportunity to collaborate with NIH scientists, we remain on track to develop and deliver the next generation of MS therapeutics for patients.”
The Myelin Repair Foundation’s ARC model leverages its nonprofit status to advance promising compounds to the clinic, and ultimately to patients. Promising scientific discoveries from the MRF academic research consortium are accelerated into clinical development through the ARC model. The Myelin Repair Foundation’s goal-oriented, collaborative approach is designed to facilitate partnerships between top academic scientists, pharmaceutical partners, the NIH and other key players throughout the entire therapeutics development continuum to speed effective treatments to patients.
The MRF is demonstrating on myelin repair for MS, a new and comprehensive model for medical research and drug development that significantly shortens the time to market for new medicines for all diseases. By recognizing the incentives and limitations of academic scientists, commercial biopharma and government regulators, the ARC Model provides a clear pathway from bench to bedside by leveraging the MRF’s position as a non-partisan, non-profit organization with only one goal in mind: shortening the time to market for new medicines for patients who can’t afford to wait. The Foundation’s Accelerated Research Collaboration model is bridging the gaps from academic research to FDA approval to develop the first myelin repair treatment for MS by mid-2019 — within 15 years of its founding. MRF is committed to demonstrating the applicability of its model to speed up the development of treatments for all diseases.
Since 2004, the MRF has funded basic research leading to the publication of more than 120 peer-reviewed scientific articles, the identification of more than 100 novel potential myelin repair treatment targets and the discovery of multiple new research tools – animal models and assays – that may help to accelerate research on all neurological diseases. Since 2004, the organization has gained increasing attention and interest from a broad array of individuals, organizations and the media, both for its achievements in myelin repair research and for the potential of its Accelerated Research Collaboration (ARC) model to change the way all medical research is done.
MRF’s Scott Johnson has been a guest speaker at industry conferences and forums where the ARC model has been showcased for its best business practices. In 2010, he was recognized as a Northern California Ernst and Young Entrepreneur of the Year. In 2011, the Myelin Repair Foundation was the only non-profit organization named among the top ten biotech companies by Fast Company magazine.
The Myelin Repair Foundation is supported by the gifts of individuals, foundations and corporations. Since 2004, the Foundation has raised $45 million to support its myelin repair research program. For more information, visit:
The Myelin Repair Foundation
Springer Open Choice Free Access Article “Promoting Remyelination in Multiple Sclerosis – Recent Advances”
The Myelin Project
The Myelin Repair Foundation