Q: What was the impetus of starting MRF?
A: In 1976, at the age of 20, I was diagnosed with multiple sclerosis. Unlike many who are diagnosed with MS, I was fortunate to be able to pursue an active business career, including leading three start-up companies. In 2001, I read a news brief in BusinessWeek that described work being done at Yale on myelin repair in MS. (Myelin is the insulating substance coating the nerves in the brain and spinal cord. It is also the substance that is damaged in MS. This damage is what causes the disease’s unpredictable symptoms). Up to that point, everything I had read about MS had focused on how to cure the disease or how to slow the immune reaction. So I picked up the phone, tracked the scientist down and started asking questions.
I learned two important lessons from my conversation with this scientist and others. First, scientists were starting to believe that regeneration or repair in the central nervous system was possible, and that it probably happens naturally in healthy people. Second, and perhaps the real lesson, was that it seemed that if you did things slightly differently throughout the entire continuum of medical research and drug discovery that the process of developing new medicines could be greatly accelerated. That was really the genesis of the Myelin Repair Foundation.
Q: What is the Accelerated Research Collaboration model and how is it different from traditional approaches to basic research and drug discovery?
A: The traditional model for medical research and drug discovery typically divides into several parts: basic biology, often conducted in an academic laboratory; pharmaceutical drug development; clinical trials; and FDA approval. For a variety of reasons, over the past several decades, these entities have grown farther and farther apart. The result has been a failure, essentially, to connect the vital dots that would lead to bringing a new medicine to market. That is why, in spite of a doubling of investment in research from 1995-2005, the number of new drug approvals has remained flat.
The Accelerated Research Collaboration model optimizes and integrates the contributions of all those who participate in the value chain of making new medicines. As a result, it provides a larger number of therapeutically relevant treatment targets in the pipeline, it translates discoveries made in animal models to the human system, it builds partnerships with pharmaceutical companies to develop and bring new treatments to market, and, ultimately, improves quality of life for millions living with diseases for which there are currently no effective treatment or cures.
Q: What are some of the exciting, new initiatives the MRF will be launching in the next 1-5 years?
A: The success of our discovery biology program has brought us many high-quality therapeutic targets and several new research tools that may be used more broadly in research for other neurological diseases.
At this time, we have two development paths before us: to validate the most promising novel myelin repair therapeutic targets through contract research organizations and to investigate the potential for repositioning of more than 40 targets that are in clinical development by various companies for other indications. We expect to pursue these paths in parallel and have developed a robust translational medicine platform to accomplish this.
We are also investing in the development of myelin repair biomarkers. Establishing one or more clearly measurable biomarkers will help us select the most promising of our targets for development.
Q: How are you measuring success in the near and long term?
A: Near-term success is already being measured by the number of targets identified in our funded laboratories as well as the number of new research tools that are enabling this research. In the longer term, of course, the only real measure of success will be one or more successful clinical trials for a myelin repair therapy. We are currently aiming for a myelin repair clinical trial for one of our targets in 2014.
Q: What is your fundraising strategy?
A: Though many think of us as a biotech company, we are in fact a non-profit organization supported by generous individuals, corporations and foundations with either an interest in finding a cure for MS or an interest in improving the outcomes of all medical research. In six and a half years, we have raised gifts and pledges totaling $43 million. We anticipate the next phase of our work will require as much as $80 million to complete.
Q: What is your partnership strategy?
A: There has been considerable interest from several companies in our both our portfolio of targets and our research tools. At this time, we are seeking partnerships with pharmaceutical companies who have an interest in myelin repair or who have a compound in clinical development for another disease indication that from our research appears to affect the same mechanisms for myelin repair. We are reaching gout to those companies now.
CHI-Advancing California biomedical research and innovation