is supported by gifts and grants from individuals, corporations and foundations. Since 2004, we have raised more than $23 million toward our initial $25 million, five-year myelin repair target discovery program. While many of our supporters have ties to multiple sclerosis, half the funds raised to date have come from individuals and foundations interested in the potential the ARC model holds for speeding research for all diseases. It is through their support and their willingness to serve as ambassadors for the MRF that our success to date has been possible and we are grateful to them.
Moving forward, in addition to continued funding for our target discovery team, we will also be supporting myelin repair target validation and drug discovery networks (as much as $1–$3 million per target). As a result, our funding requirements will increase significantly over the next five years. Though there are many opportunities to give at all levels, our supporters can feel confident that the largest percentage of their gifts will support research. In fact, over four years, in part due to the support of the Robert Wood Johnson Foundation, 90% of each gift made to the MRF has been spent exclusively on research. Most importantly though, our focused management of research is bearing fruit that will change the lives of millions suffering from MS and other diseases for which there are currently few available treatments or no cures. You are invited to join us in this extraordinary opportunity to change the way medical research is done and more rapidly deliver patient treatments to people who shouldn’t have to wait.
To donate to this worthy cause please go to: http://myelinrepair.org/donate/
If you’ve made a charitable donation to medical research or paid taxes in the last year, your dollars are part of the $40 billion spent annually on medical research at universities worldwide. While your dollars may help to support important basic scientific discoveries in those laboratories, chances are slim to none that those discoveries will ever cross the finish line for patient treatments. The system for funding and conducting medical research and translating that research into patient treatments is broken.
In the last decade, in spite of a doubling of dollars invested in academic research and commercial research and development, the number of new drugs coming to market has remained flat. As a result, for the vast majority of diseases, there continue to be few, if any, effective treatments or cures. Every American is impacted by such diseases either directly or indirectly. And the financial and quality-of-life burden this puts on those individuals, their families and the entire health care system is enormous. According to current estimates by the Milken Institute, the cost is $1 trillion a year in the U.S. and is expected to increase to $6 trillion over the next 20 years.
Promising discoveries made in university laboratories aren’t making it to patient treatments.
• Academic scientists and commercial biopharma function in different worlds with different incentives. As a result the gap between the two has grown rather than narrowed in the last 30 years.
• Understanding complex diseases requires the expertise of multiple related disciplines. The competitive reward structure of academic research prevents productive collaboration with others.
• The “product” of medical research done in university laboratories is scientific articles published in peer-reviewed scientific journals, not patient treatments. For complex diseases, to assemble, test and validate the data reported in scientific journals from multiple unrelated investigators over course of years is virtually impossible.
• Scientific data produced in university laboratories generally do not meet the necessary industry standards for commercial drug development. Without industry-standard data sets, pharmaceutical companies are unlikely to make the multimillion-dollar investment in validating the thousands of discoveries made in university laboratories. Today, the cost of bringing a single drug to market is $1 billion.
• University technology transfer offices have limited resources to protect the intellectual property developed in their laboratories. Without this protection, biotech and pharmaceutical companies will not consider these discoveries for commercial development.
• Venture capital to fund entrepreneurial academic scientists who wish to spin out their discoveries into commercially-viable therapeutics is drying up.
Promising discoveries can be identified, validated and developed in a fraction of the time.
In 2004, the Myelin Repair Foundation launched its innovative Accelerated Research Collaboration™ (ARC™) model—a new paradigm for medical research designed to speed the process of basic science and ensure that discoveries made in university laboratories were driven toward commercial development and ultimately to patient treatments for the millions living with diseases for which there are no effective treatments or cures.
To prove that basic science could be accelerated, the MRF undertook a five-year myelin repair research program that would lead to new treatments for multiple sclerosis. The goal was an aggressive one: To license for development, the first myelin repair therapeutic target within five years.
By infusing goal-oriented, best business practices and ongoing management oversight into the process of academic medical research, the MRF’s ARC model has produced extraordinary results: In just four short years, the identification of 19 potential myelin repair therapeutic pathways/targets; the development of two dozen new research tools with application for all neurological disease research; and the discovery of 18 patentable inventions. By feeding these discoveries into an MRF-funded target validation and early-stage drug discovery network, the MRF is ensuring that the necessary data sets produced by the MRF will meet the standards for commercial licensing and drug discovery.
To learn more about how the ARC model is out-producing the traditional model of medical research and driving discoveries into commercial development, see back and visit www.myelinrepair.org.
Through its proof-of-concept myelin repair research program, the Myelin Repair Foundation has shown that a results-driven, strategic business model is as important in medical research as it is to the success of any commercial enterprise. Based on the principles of cutting edge business models, the MRF’s ARC Model, establishes new incentives for both academic scientists and commercial drug developers to accelerate the process of developing new patient treatments. The ARC model engages participants in all phases of the drug discovery process including target discovery, target validation, early-stage drug discovery, patent application and licensing deals, and the development of relevant tools and biomarkers. By bringing a comprehensive outside perspective and the necessary resources, the MRF is playing a unique role in driving forward potential therapeutic targets into commercial development.
In the target discovery phase, scientific investigators in university laboratories seek to identify the basic biological building blocks that could potentially cause or prevent a disease. The ARC model directly addresses the characteristics of traditional academic research that slow down the process of discovery, including the randomness of experiments and the scientists’ need to protect their discoveries until the results are published. To accomplish this cultural and behavior shift, the ARC model requires
• The commitment of an interdisciplinary team of principal investigators to a collaborative process in which science is shared in real time;
• The development of a focused, treatment-driven research plan with milestones and objectives;
• Cooperatively designed experiments conducted in parallel resulting in a faster rate of the discovery and application of new knowledge required to solve the research problem.
• The review and input of senior scientists to ensure high-quality science;
• The active professional management oversight of the research plan; and
• The identification and protection of relevant discoveries for future licensing to commercial development.
Validating the targets identified during the target discovery phase must be accomplished before targets can advance toward commercial development. Since this validation is outside the scope, expertise or interest of academic scientists, it can only be accomplished by commercial contract research organizations (CROs). These CROs conduct the repetitive validation studies in animal and in vitro models that will demonstrate the targets true viability. To accelerate the validation process, the ARC model assembles a network of CROs to conduct parallel target validation studies that speed the go/no go decisions for further development. The ongoing involvement and oversight by the MRF’s Drug Discovery Advisory Group ensures that the most promising targets are quickly validated to commercial standards and advanced toward early-stage drug discovery.
Drug discovery is typically accomplished in commercial biotech and pharmaceutical companies only for targets identified within their own research and development programs. Through a series of studies, drug leads are refined and evaluated in a battery of standardized pre-clinical tests. To conduct drug discovery activities on targets identified by the MRF target discovery team, the ARC model assembles a network of CROs and biopharma partners to conduct studies in parallel on multiple validated targets to ensure the rapid development of clinical drug candidates. How the ARC Model Encourages Licensing by Commercial Pharma Partners The gap between academic scientists and commercial drug developers is broad and deep. Few academic scientists have an interest in commercializing their discoveries and commercial drug developers, with competing internal research and development programs, have little interest in funding target validation and early-stage drug discovery on targets identified in academic laboratories. To gain the attention and interest of commercial drug developers in targets developed outside of their own laboratories, (1) the targets must be validated to industry standards and (2) the inventions around targets must be patented to protect the interests of all parties. Through contracts with participating universities, the ARC model ensures the funding and management of patent filing and execution for all patentable discoveries made in participating laboratories. By agreement, the universities own the patents but the MRF acts as the licensing entity. This ensures that targets are rapidly advanced toward clinical development in accordance with the MRF’s mission and goals.
Given that myelin repair is a new area of study for multiple sclerosis, existing research tools—animal models and assays—are not necessarily predictive of what will happen in humans with the disease. For that reason, the MRF supports the development of new models that will more accurately gauge the relative impact of therapeutic approaches in humans. The development of biomarkers—biological markers that indicate disease state—in parallel with target identification will not only enable earlier diagnosis of MS but also will increase the speed and reduce the cost of clinical trials. The MRF’s tool and biomarker development strategies are key to accelerating drug discovery.
