Comments From the Leading Myelin Repair Scientists
Al Sandrock, M.D., Ph.D.
Chief Medical Officer, Biogen
The MRF was the pioneer in identifying the need for and driving the importance of myelin repair, as well as bringing together top experts in the field to move groundbreaking research forward. The work of the foundation and its innovative approach to collaboration has sparked many companies to begin their own research in this important space.
Peter Calabresi, M.D.
Director of the John Hopkins Multiple Sclerosis Center
The MRF has been a visionary (with a) mission to bring basic scientists together with physician scientists to enable translation of cutting edge approaches for endogenous remyelination in people with MS. My personal experience with this group has been spectacular in that I forged new scientific collaborations with stellar scientists while at the same time helping to design a translational paradigm to facilitate testing of the many therapeutic targets identified in the OPC drug screening program. These types of interactions are critical to enhancing transfer of basic science to the bedside. Well done Scott, your vision and what you have accomplished with MRF will undoubtedly live beyond the formal structure of the group and positively impact the field for decades to come.
Daniel Reich, M.D., Ph.D.
Chief, Translational Neuroradiology Unit Neuroimmunology Branch, NINDS, NIH
The MRF has indeed had a major impact on my work over the past few years. As a member of the Clinical Advisory Board, I had the chance to interact with colleagues at the top of my field and to broaden my thinking through the experience of the MRF’s more translational advisors. The science at the annual meetings has been uniformly outstanding, and the collaborative nature of the MRF’s model sets a very high standard that should be emulated by many others.
But I’m most proud of the work we’ve done, in collaboration with MRF scientists, to develop an innovative and potentially very powerful approach to testing therapies for myelin repair in the clinic. I’m very excited to see where this takes us!
Ben Barres, M.D., Ph.D.
Over the past 10 years, MRF has had substantial impact. It galvanized the pharma and academic communities into realizing it was now time to take a new approach to making new drugs for MS patients focused on myelin repair rather than only focusing on the immune system. It created a highly innovative and new effective collaborative mechanism for greatly increasing the badly needed basic science research on myelin repair as well as a new mechanism for making sure these research advances were then converted to new drugs.
MRF greatly enhanced the ability of my lab to do myelin research over the past ten years. Thanks to MRF support and the fantastic collaboration of my MRF team mates, we made major discoveries that have lead to new insight into how myelin wrapping occurs and we identified new drug targets to promote remyelination in MS.
With the MRF drug validation unit, we were just in the process of converting these discoveries into new drugs and it is tragic that this work is now coming to an abrupt halt just as it was about to bear fruit for MS patients. Because I do not believe there is any entity, pharma included, that will fill the gap left as MRF closes down. But I am grateful to the entire MRF team and to all the many generous donors who have made the past 10 years of progress possible. MRF has left a lasting legacy in showing that it is possible to create much more effective models for how to partner academia, foundations, and pharma to greatly accelerate new drug development in MS and many other neurological diseases.
Martin Raff, CBE, FRS, FMEDSCI
University College London
The MRF was a novel experiment in accelerating disease-related research. By enticing a number of academic researchers interested in oligodendrocytes and CNS myelination to collaborate and focus part of their effort on myelin repair, the hope was to find drugs that could promote remyelination in MS patients. The experiment worked remarkably well, in that the scientists, who would have otherwise been competitors, now collaborated to understand the underlying biology required to identify effective drugs.
Over MRF’s 10-year history, it made a number of important discoveries, identified potential new drug targets, developed novel in vitro and in vivo assays, and even identified some drug candidates, some of which are in early clinical trials. Sadly, despite this impressive track record, without sustained funding, the MRF could not carry on with its ambitious program. It remains uncertain whether the therapeutic remyelination strategy will work in MS, but there is only one way to find out, which is why the bold MRF mission made sense.
Brian Popko, Ph.D.
University of Chicago
The MRF's collaborative model was extraordinarily successful. Through MRF-facilitated collaborations we were able to approach research issues using a multifaceted approach that resulted in faster, more comprehensive solutions to problems. The expertise offered by the other members of the group greatly advanced the research efforts of my lab.
The regular face-to-face meetings that the MRF sponsored provided us with timely feedback and critical input to our research efforts. This guidance from the other PIs, their lab members and the SAB was instrumental in quickly moving our projects in the most meaningful and provocative directions.
The MRF encouraged us to take chances in our research and provided us with the resources to identify new directions quickly and to eliminate false leads just as quickly.
MRFs insistence that we think about our projects from a therapeutic perspective provided the necessary encouragement and incentive to move our science into more clinically relevant areas.
Christine Stadelmann-Nessler, PROF, DR
University of Göttingen
I first met with representatives of the Myelin Repair Foundation (MRF) around two years ago, roughly ten years after the MRF had started their funding and research activities. Right from the very start, I was impressed by the precise and explicit aim of the MRF, namely the rapid advancement of therapies promoting remyelination in multiple sclerosis (MS).
For that purpose, the MRF provided a unique environment. On the one hand, the MRF possessed a state-of-the-art laboratory to perform in vitro and in vivo assays for testing potentially useful compounds for myelin repair. On the other hand, the MRF supported a consortium of highly reputed scientists from academia with expertises ranging from oligodendrocyte cell biology and in vitro and in vivo models of remyelination to immunology and human pathology. Importantly, these activities were flanked by a scientific, clinical and pharmaceutic advisory board that encompassed world-renowned leaders in their respective fields. Thus, the MRF created an extremely fruitful environment of mutual exchange and advice, focused on the goal of myelin repair.
The twice-yearly meetings of the MRF enabled intense interactions between leaders in pharmaceutical industry, basic science, and clinical science and promoted a highly cooperative spirit and commitment. The MRF never deviated from the central goal of supporting and performing research while always having its clinical application in mind. Thus, the MRF has advocated a bench-to-bedside strategy long before this concept entered mainstream thinking.
I want to thank Scott Johnson for his impressive leadership and long lasting commitment to the cause. I also want to thank the extremely motivated and highly qualified executive, research and administrative teams of the MRF who were extremely supportive in all aspects. I am fully convinced that the spirit created by the very special environment of the MRF over the last years will further prevail in the myelin research community and hopefully lead to a substantial improvement in the treatment and care of patients with MS.
Louis Reichardt, Ph.D.
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. Inclusion of pre-doctoral and postdoctoral trainees in its meetings has broadened their education and importantly also permitted establishment and maintenance of collaborations between them that might easily have been overlooked by their over-committed laboratory heads.
A number of other foundations focused on other biomedical problems have adopted features of the Myelin Repair Foundation’s scientific model for research acceleration. Thus the foundation’s impact is important and will endure long into the future.
The Myelin Repair Foundation’s direct impact on scientific studies of multiple sclerosis has also been important. The foundation has supported scientific studies with high promise for overcoming the challenge posed by auto-immune disorders, including multiple sclerosis. The foundation’s support has enabled us to understand the factors that control remyelination (ensheathing of nerves by oligodendrocytes) to repair the damage created in the brain as a result of multiple sclerosis.
Its recent discovery of the important role of cellular stress in creating the damage to cells observed in multiple sclerosis has made it possible to identify drugs that reduce cell stress as candidates for pharmaceutical intervention in multiple sclerosis. Clinical trials are underway to test the benefit of such drugs for humans afflicted with multiple sclerosis.
In summary, the activities of the Myelin Repair Foundation over the past decade have illustrated the benefits of enhanced collaboration for advancing scientific research in all areas of biomedical science as well as making possible discoveries with potential to make a significant positive impact on the lives of individuals suffering with multiple sclerosis. The foundation will long be remembered and appreciated for these important contributions.
Samuel Ludwin, M.D.
I have served on the Scientific Advisory board of the Myelin Repair Foundation for 6 years, and I observe with sadness that that it will cease to function.
In looking back at my time on this Board, I feel that the Foundation has accomplished many things of which it, and especially the founder and chief architect, Scott Johnson, can be justifiably proud. Although the original goal, to find a therapeutic cure for remyelination in Multiple Sclerosis has not yet been achieved, nevertheless, the scientific results produced under its sponsorship and guidance have been outstanding, and will doubtless be of immense value as the search for a cure progresses.
The Foundation has funded the research of four outstanding research laboratories in the USA, as well as starting collaborations with centers in Germany. However, of as much significance, the individual efforts of these laboratories have been greatly enhanced by the enormous inter-lab collaborations fostered by the MRF. Although collaborative projects are not unique to this Foundation, the close interactions formed not only between the principal investigators, but also their staff and students, has the potential to continue well into the future.
Through its annual and working meetings, and with the input of some outstanding members of the Advisory Committee and staff, the transmission and dissemination of ideas has contributed greatly to the success of the scientific investigations. These will all be sadly missed.
Finally, but by no means less importantly, the Foundation has conveyed a patient--centered feeling of urgency to all who have been involved. Although the pace of science may be slow, and cannot be forced, the Foundation’s central raison d’etre, of providing a model for rapid drug and therapeutic development, will stimulate others to build on this to achieve the goals we all await - a cure for MS.
Stephen D. Miller, Ph.D.
Working with the other investigators in the MRF, as well as with the distinguished members of the scientific advisory board, was a fantastic experience. My lab is now exploring questions related to the intersection of the underlying immune pathology of MS and glial/myelin biology that would have not been possible without the funding, knowledge gained, advice, guidance and collaborations made possible by the generous funding provided by MRF.
This new research avenue has proven to be extremely fruitful. Several examples of the advances in my laboratory made possible by my association with MRF are:
- MRF funding, in part, facilitated the completion of a phase I clinical trial illustrating the safety and preliminary effectiveness of an immune tolerance based strategy utilizing myelin antigen-coupled patient peripheral blood mononuclear cells (PBMCs) to regulate autoimmune T cells in MS patients. This provided the first proof-of-principle that tolerance-based strategies can be successfully employed in human autoimmune diseases;
- MRF funding, in part, led to the development of a second generation tolerance strategy using antigen-coupled biodegradable nanoparticles which has generated significant interest from large biopharmas for translation of the treatment not only of MS, but other autoimmune and allergic diseases;
- we have more recently paired our long-standing knowledge of immune regulation using myelin antigen-specific tolerance-based strategies with knowledge gained about the processes stimulating myelin repair and the identification of myelin repair enhancing drugs to develop a combinatorial approach for the reversal of clinical disease in MS animal models which hopefully will soon be translatable to the treatment of the human disease.
Our collaborations with the other MRF investigators will continue using other funding streams and I am confident will continue to lead to major advances in our understanding of the processes underlying immunopathology of MS and to improved methods for the diagnosis, prevention and treatment of this devastating human disease.
Read about the clinical trial of a potential MS drug to protect and repair myelin.