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Myelin Repair: The Science

ARC Model Clinical Development Translational Medicine Platform Discovery Biology

Our myelin repair research program is the proof-of-concept project for our Accelerated Research Collaboration™ model (ARC™). The ARC model is a wholly integrated strategy that considers all elements of the basic research, drug discovery and drug development value chain.

Our work is guided by senior scientists and advisors from academia and the pharmaceutical industry. All research is overseen and managed by our staff of experienced industry professionals.

Many believe the ARC model has the potential to shorten the time to market for new medicines for all diseases. Proving the model’s effectiveness through our myelin repair research program is the first step in demonstrating the model’s effectiveness and replicability for other disease indications.

Clinical Development

In preparation for seeking FDA approval for a myelin repair clinical trial, we established our Clinical Advisory Board led by Dr. Henry McFarland, Former Chief of the Neuroimmunology Branch and Director of Clinical Neuroscience at the National Institute for Neurological Disorders and Stroke at the National Institutes of Health. The Clinical Advisory Board is ensuring that the appropriate steps are taken to facilitate the development of effective trials. In 2011, we launched a biomarker development initiative to establish systematic, measurable endpoints for myelin repair. The availability of such biomarkers will help to accelerate the pace of clinical trials.

Translational Medicine Platform

The success of our discovery biology program has provided us with a plethora of opportunities for further validation of the more than 100 myelin repair targets identified in our laboratories. At this time we have two development paths before us:

  • Validate the most promising novel myelin repair therapeutic targets through contract research organizations.
  • Investigate the potential for repositioning of more than 40 targets that are in clinical development by various companies for other indications.

Designed to improve the odds of successful clinical trials, our translational medicine platform will ensure that to the extent possible there is consistent and concordant data in cell cultures, multiple animal models and assays, and in human tissue.

An analysis (Figure 1) of publicly available data conducted by our Drug Discovery Advisory Board of biopharmaceutical experts is helping to prioritize the overwhelming number of targets generated in our funded discovery biology laboratories. The blue area shows those discoveries that are entirely novel. The dark green area shows the overlap of those discoveries with those in the Validated Targets and Pathways Database (VTPD) that are in development, often at a number of competing companies. Source: Validated Targets and Pathways Database

(Figure 2) These are documented programs under investigation by various companies on a subset of Myelin Repair Foundation targets.

Discovery Biology

Our discovery biology program is a managed collaboration that brings together world-class investigators with complementary knowledge in neurobiology, immunology, and myelin research. Their research is organized in a detailed research plan that addresses seven key areas that are believed to hold the answers to myelin repair. The graphic below shows each of the seven key areas and how the expertise of the funded laboratories crosses over from one area to another.

The plan is reviewed and revised annually by a Scientific Advisory Board of senior neuroscientists widely recognized as experts in their fields. The experimental design is informed by our Drug Discovery Advisory Board of experienced “drug hunters” who ensure the clinical relevance of the experiments and help set priorities for further development of the most promising therapeutic targets.

Our discovery biology program funds more than 35 full-time scientists in laboratories at Stanford University, Case Western Reserve University, the University of Chicago, Northwestern University, the University of California, San Francisco and the University of Melbourne and the National Institutes of Health.

Roll over to read more about each area of the research plan.

View Our Progress

Understanding how oligodendrocytes are normally generated from neural stem cells and how MS perturbs this process Understanding the underlying mechanism of myelination and how it is perturbed in MS Understanding how nodes of Ranvier and paranodes are normally formed and how they are perturbed in MS Understanding how the Blood Brain Barrier is affected in MS and its role in the disease Understanding the immune response in MS and how inflammation affects myelin repair Development of better animal models for study of MS and remyelination Biomarkers

More about myelin…

What is myelin?

Myelin is the fatty protective coating surrounding nerve fibers of the central nervous system. Because of its role in aiding the conduction of electrical signals among and between neurons in the brain and spinal cord, it is often compared to the insulating material around an electrical wire. Myelin rich “white matter,” constitutes nearly one-half of the human brain.

What is multiple sclerosis?

Multiple sclerosis (MS) is a neurological disease that affects more than 400,000 people in the U.S. and 2.5 million worldwide. In MS, the body’s immune system attacks and destroys myelin in the brain and spinal cord. Though healthy human systems have the natural ability to repair myelin damage, in MS where damage occurs, scars of hardened sclerotic tissue inhibit and/or prevent that process from taking place. Loss of myelin, referred to as demyelination, interferes with the transmission of nerve signals and triggers a wide range of unpredictable symptoms, including blurred vision, loss of balance, poor coordination, slurred speech, tremors, numbness, extreme fatigue, problems with memory and concentration, paralysis, stiffness, bladder problems and blindness. There is no cure for MS. Myelin loss has also been linked to a number of other neurological diseases including Parkinson’s disease, Alzheimer’s disease, and depression.

The promise of myelin repair

Today’s approved therapies for Multiple Sclerosis work by suppressing the entire body’s immune system to slow or prevent the immune system from attacking. If successful, the treatments can slow the progression of the disease and severity of the attacks. Immunosuppressant treatments do not repair the myelin damage caused by MS and therefore play no role in restoring the lost function associated with the disease. A treatment that can restore and protect myelin holds promise to restore that lost function.


Accelerate Treatments to MS Patients

Your support will help us deliver a first-in-class myelin repair treatment to multiple sclerosis patients as rapidly as possible. Donations at all levels are critical to achieving this mission.

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