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“The Myelin Repair Foundation has had a broad impact on scientific research beyond its support of investigations that improve our understanding and treatment of multiple sclerosis. The foundation has created a new model that enhances collaboration of scientists with different expertise to accelerate scientific advance.”
 Louis Reichardt, Ph.D., Simons Foundation

Frequently Asked Questions about biomarkers and MRF

What is a biomarker?

A biomarker, or biological marker, generally refers to a measurable indicator of some biological state or condition. Biomarkers are often measured and evaluated to examine normal biological processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention.

In medicine, a biomarker can be a substance whose detection indicates a particular disease state, for example, the presence of an antibody may indicate an infection. More specifically, a biomarker indicates a change in expression or state of a protein that correlates with the risk or progression of a disease, or with the susceptibility of the disease to a given treatment.

How do biomarkers help get new treatments to patients?

Biochemical biomarkers are often used in clinical trials, where they are derived from bodily fluids that are easily available to the early phase researchers. They are also used in pre-clinical work to identify compounds that appear to modulate disease in in vivo models and therefore might be tried in human clinical trials. Disease-related biomarkers give an indication of the probable effect of treatment on patients.

How can biomarkers accelerate the availability of new myelin repair treatments?

Biomarkers of myelin repair or neuroprotection would enable vastly more rapid and accurate testing to determine if a compound truly enables repair of or protection from damage to myelin. Effective biomarkers would also likely enable smaller and faster clinical trials.

Why should MRF lead an effort to identify a myelin repair biomarker?

Biomarkers are essential tools for drug development, but are difficult to identify. They take years and millions of dollars to uncover and verify. Even for large companies with large research expenditures it is a daunting endeavor with little certainty of success. The difficulty of the task plays to MRF’s ability to build collaborative teams of the best scientists from many, often competing organizations.

Why did MRF cut back its activities?

As a non-profit, for over 11 years, MRF has relied on generous gifts from individuals, foundations and companies. MRF never had an endowment or operating income so we relied on on-going new gifts to continue our critical work. Fundraising is never easy. It has always been a challenge to raise our minimum budget, much less to increase our budget to accelerate discoveries. We do not have sufficient funds to operate as we have been. We are now solely focused on our biomarker initiate.

What happened to the MRF research consortium?

The principal investigators and scientists who were a part of our world-class research consortium have continued their research efforts. When MRF began, interest in, and funding for, myelin repair was limited. Because of MRF others are now funding neuroprotection and repair, both in academia and in biopharma companies. Members of our academic team are obtaining grants from other sources to conduct new experiments.

What about the clinical trial for guanabenz (MRF-008)?

In 2013, we were awarded an Investigational New Drug (IND) by the US Food and Drug Administration (FDA) to conduct a first-in-class clinical trial to evaluate the neuroprotective ability of guanabenz (compound MRF-008) in multiple sclerosis patients. To conduct this trial MRF entered into a Collaborative Research and Development Agreement with the U.S. National Institutes of Health (NIH).

What have been MRF’s key accomplishments over the last 12 years?

When MRF began few thought myelin repair was possible and the few that did, thought it was 50 years away. Now most think it is possible and will be the next generation of MS treatment.

  • MRF has driven programs from basic science to validation and translation, and on to the clinic.
  • MRF has transformed the myelin repair landscape: Virtually every leader in myelin repair / neuroscience has been or is associated with MRF in some way.
  • We have designed and demonstrated an innovative new model to accelerate medical research resulting in three Phase 1 clinical trials.
  • MRF created an environment where the best myelin related scientists from around the world could work collaboratively.
  • Over 135 papers have been published by our academic team.
  • We established an MRF staffed lab with unique capabilities to thoroughly examine compounds for their myelin repair potential.
  • Over 1,000 compounds have been tested in MRF developed, academically elegant but industry rigorous, state-of-the-art assays.
  • We licensed to Biogen an innovative animal model that may allow better study of progressive MS.
  • MRF established multiple partnerships with biopharma companies to help advance myelin repair.
  • The FDA awarded MRF an Investigational New Drug (IND) to test MRF-008 in humans with MS.