Multiple Sclerosis (MS) and Transverse Myelitis (TM) are members of a group of neuroimmunologic disorders in which the immune system attacks and injures the nervous system. Although each of these disorders is rare, the worldwide prevalence of the group exceeds 2.5 million people. Since they typically afflict young people, the personal and economic impact of these disorders is staggering. Recent studies have suggested that the total societal costs for MS in the United States are 5.1 billion dollars annually, with the bulk of that cost due to sick leave, premature retirement and loss of income. However, despite the economic importance of these disorders, their rarity makes them difficult to study since few centers can identify and study enough patients. Even within institutions, researchers often study these disorders individually rather than striving to identify common mechanisms and identifying shared treatments. As a result of this, research in these disorders has been slow. Researchers cannot coordinate efforts and cannot rapidly develop new ideas. Perhaps most importantly, there has been little translation of new ideas into clinical therapies.

The implications are that money and effort are wasted, brilliant research is not optimized, and progress is slower than it needs to be. Every day, patients are becoming more disabled, going into or staying in wheelchairs. The solution to this problem is the development of a new way to study these disorders. Researchers must have money to rapidly test ideas, they must collaborate in order to accelerate developments, they must develop model systems to test ideas, and they must have the ability to carry out clinical trials as a prelude to new therapeutic strategies. The Johns Hopkins Department of Neurology is launching a new initiative that will achieve these goals.

Termed the Johns Hopkins Project RESTORE, this multidisciplinary research and clinical collaboration is emerging from The Johns Hopkins TM and MS Centers, and will develop new diagnostic and therapeutic strategies in the treatment of neuroimmunologic disorders, such as multiple sclerosis (MS) and transverse myelitis (TM).

 The Johns Hopkins Project Restore: Overview

The Johns Hopkins Project RESTORE is a five year effort that will result in new therapies for neuroimmunologic disorders. The Project will not be a physical space at Johns Hopkins, but rather will be a “new way of doing business.” The Project will fund researchers to do collaborative research, will develop novel biomarkers in neuroimmunologic diseases, will develop novel imaging strategies, and will carry out clinical trials to test ideas. At the end of five years, we expect to have dramatically improved our understanding of neuroimmunologic disorders and to have initiated clinical trials testing novel treatments based on this understanding. Funding this sort of project is not within the scope of the National Institutes of Health (NIH) or the pharmaceutical industry at present. The NIH funds established ideas that represent incremental advances from previous work. Funding from the NIH often takes two years to obtain and does not support infrastructure. Further, project funding is finite and a project must cease if the funding lapses. The result of such funding is that researchers spend a great deal of time trying to secure additional funds to support their research. We hope that Project RESTORE will be funded principally by philanthropic support for the first five years for researchers to develop novel, groundbreaking approaches. This funding will allow the rapid testing of ideas and will support the infrastructure to rapidly develop these ideas into clinical therapies. Endowment support, in particular, will protect researchers’ time to allow sustained focus on research.

We expect that the Johns Hopkins Project RESTORE will continue efforts at the end of five years, but will have made such substantial advances that a greater share of the funding will be from the National Institutes of Health and pharmaceutical companies. Furthermore, by that point, we believe that Project RESTORE will have marketable and patentable therapies in development, and could share in the royalties from these products.

Project Restore has three principal goals:

RECOVER

STOP

REGENERATE

By developing new treatments for TM and MS, we hope to Restore Hope, Restore Function, and Restore Lives to patients and families with these disorders.

Project Restore: Mission And Vision

Although advances have been made in the treatment of MS and TM, current therapies are only partially effective and many patients continue to develop disability despite the therapies. Our mission is as follows:

We are poised to make dramatic advances in all of these areas over the first five years of Project RESTORE. In the first two years, we will develop novel imaging strategies that define the pathologic features of MS and TM in order to direct appropriate treatments to these patients. We will also develop novel immunologic and neuroprotective strategies using animal models and cell culture models that will then lead to clinical trials in years three to five. We will further define the ability of stem cells to “rewire” the nervous system and will prepare for neurorestorative clinical trials using stem cells in years four to five. Perhaps, most importantly, we will initiate and carry out clinical trials of novel compounds currently in development.

Conclusion

We believe that we are poised to make major changes in how we treat neuroimmunologic disorders, and have established a formal mechanism for how to achieve this goal. We have leading experts in these diseases. However, the Johns Hopkins Project RESTORE will allow progress to be made at a much faster pace. New discoveries will result in new therapies and will diminish the suffering and disability of millions of patients.

http://www.hopkinsneuro.org/restore/