The Myelin Repair Foundation leaves behind a legacy whose impact will be felt for years to come.
June 22, 2015
Dear Friends and Supporters of MRF,
I hope that by now you have read that the clinical trial of guanabenz (formerly known as MRF-008) is officially underway. We are thrilled that with help from supporters and donors like you, our academic, translational and clinical research teams have been able to get this potential myelin repair therapeutic into a clinical trial.
We achieved this milestone and the others cited below on a cumulative budget of only $55 million over 11 years. Compare this to the average cost to get a single drug to clinical trial of over $400 million.
The only way this could have happened was via MRF’s Accelerated Research Collaboration (ARC) model, the business / research model that we pioneered to disrupt and accelerate the drug development landscape. It is this model that ties academic, translational and clinical research together and is what has allowed us to bring a myelin repair therapeutic closer to a reality.
But it has not been easy. Ironically even with tremendous success on the research side, we have struggled with raising enough money to keep up with our extraordinarily low research costs.
And so, as someone who has generously supported our work, I wanted to share some very painful news: Given a challenging financial position, we and our board have come to the difficult decision that we use the funds we have on hand to conduct an orderly wind down over the next couple of months. MRF will cease operation by August 31.
I know this probably comes as a surprise. I want to share with you how we have come to this point, what will happen going forward and most importantly, what we have accomplished together, and the legacy MRF will leave.
From the time of our creation in 2003 we have relied on generous donations from individuals and foundations to pursue our vital mission. Every dollar has been critical to us. While we have thousands of donors from 29 countries we have relied on a small number of donors, giving very large gifts, for most of our revenue (92% has come from only 56 donors).
Since we began funding research in 2004, our annual budget has averaged just $5M. Compare this to the $250M raised by the National MS society in 2014. With dozens of non-profits serving the MS community, it has always been a challenge to convey to potential donors how both our business model and our laser focus on myelin repair differentiate us from other organizations. Because of our innovative ARC model, which could be used to speed treatments for other diseases, we have raised half of our funds from donors with no tie to MS, but unfortunately it has not been enough.
We have always operated with the fundamental belief that MRF must greatly accelerate the entire drug discovery continuum to speed first-in-class therapeutics to patients who cannot afford to wait. With that as a parameter, and no operating income or endowment, our funds have always been immediately spoken for and put to use in the most expedient manner possible.
Recently several large donations that we had been counting on failed to materialize. As a result we do not have sufficient funds to fund our next fiscal year which begins on July 1. In order to ensure that our accomplishments to date and work in progress best serve MS patients, we will use the funds we have in hand to preserve the science and momentum we have created.
Having to stop our efforts and close our doors is the worst case for all who devoted time and / or financial support to our endeavor to help all those impacted by MS. Our consolation is that over the last eleven years we have had a dramatic impact on the myelin repair landscape. When we began, few thought myelin protection or repair was possible, and those that did thought it would be 35 to 50 years in the future.
I believe that because of MRF at least one neuroprotective or repair therapy will be available to MS patients within ten years.
Together, we can celebrate MRF’s accomplishments:
Most significantly, less than eleven years after we began funding myelin research, 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 (MRF-008) - a compound that we identified. We have an agreement with the NIH to conduct the first two phases of the trial, which will evaluate the efficacy of the compound. This trial will take several years, is fully funded, and is currently underway.
Our wind down plan includes activities to:
We have had a tremendous impact and I am confident that a myelin repair therapeutic will be available to MS patients in the future. But, it is sad and painful to me because I believe that if MRF could continue to drive myelin repair activities via our ARC model it would be available sooner.
Please know that I am forever grateful for your support of MRF and the impact, together, we have had. What we have accomplished will live on through the people we have worked with and the discoveries we have made.
“Although hundreds of thousands of scholarly scientific articles are published every year, the FDA approves less than two-dozen new drugs a year. What's missing is the translation of all that cutting-edge science into cutting-edge cures. That's where the MRF comes in.”
Scott Cook, Co-Founder, Intuit
“The Myelin Repair Foundation's model—which brings together researchers and works to ensure that their work is relevant to development of patient treatments—is a critical innovation at a time when our system of drug development is looking for new ideas.”
Elliot Gerson, Aspen Institute and Rhodes Trust
“There are many foundations funding research on different diseases, but fewer that are investing in the infrastructure to manage that research more effectively.”
Nancy Barrand, Special Advisor for Program Development, Robert Wood Johnson Foundation
“Innovation is a decidedly social process encompassing diverse individuals, corporations, communities, networks, and regions. The work of the Myelin Repair Foundation is an excellent demonstration of these principles.”
John Hagel, Co-Chair, Deloitte LLC, Center for Edge Innovation
“I am drawn to new ideas that have the potential to change a market. I am inspired by the MRF's business model and advanced medical research concepts. MRF is a non-profit, but it breaks every rule and barrier to the speed of a start-up with the agility of a successful business.”
Samantha Fein, Managing Director, Threxy
“Unlike a lot of other organizations, the Myelin Repair Foundation really understands the intersection between academia and pharma. And if you're going to make a difference in MS, myelin repair is where you're going to have to put your efforts. We are putting efforts there.”
Craig Sorensen, Vice President, Vertex Pharmaceuticals
“Working with MRF is an unmistakable path for me to bring effective therapeutics — and new hope — for all patients in need.”
Beatrice Perotti, Ph.D., M.B.A., President and CEO, Beatrice Perotti, Inc.
“Breaking down barriers between academic research and commercial drug development will be the centerpiece of the Myelin Repair Foundation's legacy.”
William K. Bowes. Jr., Founder Amgen, U.S. Venture Partners
“Through our funding, we look for ways to make medical research more relevant to health improvement. MRF's leadership in transforming the research paradigm is very compelling to us.”
Lynne Garner, Trustee & President, Donaghue Foundation
“We view the Myelin Repair Foundation's Accelerated Research Collaboration model as just that, a transformative idea with the potential to pioneer a new approach to medical research that can speed the discovery process and lead to the development of new treatments.”
Carl Schramm, President and CEO, Ewing Marion Kauffman Foundation
“Not enough progress has been made, fast enough, toward effective treatments for MS. I am involved with the Myelin Repair Foundation because I really believe in the methodology: Getting the best people together to solve a really tough problem collaboratively. I truly believe that the Myelin Repair Foundation offers real hope.”
Julie Wainwright, Founder and CEO, SmartNow.com
“MRF's collaborative model is … definitely accelerating results. The scientists have made important advances that, if the labs had been working on their own, would have been much less likely.”
Brian Popko, Ph.D., University of Chicago, Director, Jack Miller Center for Peripheral Neuropathy, Associate Chair for Research, Department of Neurology
“I support the MRF for two reasons. Personally, I have a connection to MS: My husband has MS. But even beyond that, the vision of Myelin Repair Foundation and the method that they are proving out to get drugs to people faster is a really big vision that is important for a lot of unmet medical needs and it is exciting to be a part of it.”
Sharon Wienbar, Managing Partner, Scale Venture Partners
“If you just leave it to basic scientists working alone in their own labs, converting basic scientific discoveries into drugs almost never happens. Without the ARC model there is no infrastructure for accelerating drug development.”
Ben Barres, M.D., Ph.D., Stanford University School of Medicine, Chair, Neurobiology Department
“Disease foundations play an important role in funding early stages of research when other research and investment dollars are scarce. I am impressed by what the Myelin Repair Foundation is doing to encourage collaboration. MRF's model is novel and spot-on in terms of moving innovative research forward.”
Gail Maderis, President and CEO, BayBio: Northern California's Life Science Association
“The Myelin Repair Foundation has identified the best labs in a defined area and brought them together with excellent and independent minds from the pharmaceutical industry to advance the most promising ideas for novel therapeutics.”
Martin Raff, M.D., Emeritus Professor, University College London
“Collaborative innovation — bringing together people from different disciplines, with complementary skills — is a powerful strategy for solving complex problems. The MRF's model provides important lessons for the pharmaceutical industry, which faces a crisis of innovation in developing treatments for complex diseases.”
Karim R. Lakhani, Harvard Business School
“Not enough progress has been made, fast enough, toward effective treatments for MS. I am involved with the MRF because I really believe in the methodology: Getting the best people together to solve a really tough problem collaboratively. I truly believe that the MRF offers real hope.”
Ted Yednock, Executive Vice President, Head of Global Research, Elan Pharmaceuticals
“I believe the most striking accomplishment has been the success of the model. The thought that one could get several excellent basic scientists to work in a united effort with a clinical target in mind is really impressive... Not only has the group worked together, but there are now products of this effort. Very impressive!
Henry F. McFarland, M.D. (Ret.), National Institutes of Health