Innovator Spotlight

Q: Not too long ago the association created a "research institute without walls." What prompted that decision, and can you describe how it operates?
Research on chronic fatigue syndrome (CFS) has produced some 5,000 peer-reviewed publications over the past 25 years. Abnormalities in the central and autonomic nervous, endocrine, and immune systems have been documented; however, most of the published studies were conducted on small numbers of subjects, and many still await replication attempts by other labs. To overcome this "one and done" approach to research, we sought to create a supportive infrastructure to foster collaboration, cross-disciplinary engagement, and centralized repositories for data and samples from well-characterized subjects – both patients and controls. We recognized that this kind of coordinated support would be necessary to move CFS research from the bench to the bedside; in 25 years of study there have been few advances in treating CFS.

Building a bricks and mortar institute would have consumed time and money that we didn't have. We learned from models pioneered by organizations including the Myelin Repair Foundation, Dr. Susan Love Army of Women, Sage Bionetworks, and the Michael J. Fox Foundation. On Feb. 23, 2012, we launched the Research Institute Without Walls (RIWW) with eight tightly integrated research projects organized around a biobank and data-sharing platform core. We've leveraged leading experts at top institutions through our ability to provide financial support and, more importantly, actively coordinate studies and exercise continuous quality improvement through collaborative refinement. This is also the first time that a set of studies aimed at identifying disease-modifying treatment for CFS was initiated at once.

Q: What are some of the tools you're creating and using to help facilitate collaboration?
In 2008, we issued a broad request for applications (RFA). In addition to posting the RFA publicly, our scientific director, Dr. Suzanne Vernon, directly contacted researchers working in areas where there had been promising pilot studies conducted in CFS. These investigators told her they were interested in testing those findings in their own labs but lacked access to appropriate clinical populations. Researchers we involved in the review of applications received in response to the RFA said the same thing: they had ideas for studies they might conduct, but no subjects to study. This was a stumbling block we could clear away.

In March 2010, we launched the SolveCFS BioBank under the integrated registry and biobanking infrastructure provided by the Genetic Alliance BioBank. We now have more than 500 consented participants – well-characterized CFS patients and controls – and a growing inventory of clinical data and biological samples. Five of the projects under our Research Institute Without Walls will make use of these assets and three others will expand enrollment through the clinical populations they study.

Another important tool we've leveraged is the Research Electronic Data Capture (REDCap) application developed by Vanderbilt University. REDCap is our secure data-sharing platform. It is customizable for each investigator but also facilitates agreement on standard information and instruments that will be used consistently across sites. This level of collaboration and standardization has not occurred before in CFS, but we see tremendous value in developing a system like the one the National Institutes of Health (NIH) has supported for autism through the National Database for Autism Research.

Q: You're also engaging in some novel projects that involve data mining and drug repurposing. Can you talk about those briefly and why they're important to you?
We're very fortunate that Suzanne trained as a virologist yet embraced a systems biology approach to the study of disease. She was ahead of the "big data" boom in understanding the value of using informatics tools to harness complex biological data. In a field that has chased a series of "in vogue" theories, we saw a critical need to deploy objective, data-driven approaches to complement investigator-initiated projects.

2011 article in The Scientist alerted Suzanne to companies applying informatics to drug repurposing. One of them,Biovista, applied to our 2011 funding opportunity and its proposal to identify non-obvious drug therapies for CFS scored well for scientific and strategic merit. This project is now part of our Research Institute Without Walls and will utilize data from the SolveCFS BioBank in addition to its proprietary COSS platform to develop a target list of evidence-based drug repurposing candidates.

The other data mining project is a collaboration with startup LogosOmix. The project began with a database of 200 CFS-related journal publications and has grown to include parts or full text of 500,000 articles about CFS or related conditions. Integrated with this database are other publicly available libraries including PubMed and Chemical Entities of Biological Interest. Using proprietary software, these data sources are being broken down into a Unified Medical Language System, and then querying tools will generate organized, relevant data on possible linkages between various biological components of CFS. The key deliverable will be a prioritized list of potential biomarkers for CFS to drive future research.

Q: What do you consider the association's greatest accomplishments to date?
We are obviously very enthusiastic about the Research Institute Without Walls, its component parts and the collective change it marks for the way CFS is studied, and promise it offers to people living with CFS. It's worth noting – especially for the benefit of other nonprofits with modest budgets – that we launched the RIWW with about $2 million. Many of the organizations that benefit from FasterCures' TRAIN collaborative are large foundations with household names and impressive budgets. But we were able to adapt a lean, mean model within our available resources. That's an accomplishment in itself!

Another important contribution we've made is seeding a lot of the research that forms the existing foundation for understanding CFS. In fact, our last round of sponsored research studies has secured follow-on funding from other institutions (including the NIH and Department of Defense) totaling more than seven times the sum of our awards, and we anticipate that multiplier will continue to grow.

Finally, for 25 years this organization has sustained hope. People with CFS experience significant losses as a result of the disabling nature of the condition. Because the disease is not well understood and is saddled with a trivializing name, diagnosis is often delayed and is accompanied by stigma and isolation. Financial losses are common, and family relationships and friendships change and often erode. Awareness is improving, and medical and social acceptance are, too. For a quarter of a century, this organization has been a buttress in shifting scientific, medical, and cultural tides.

Q: What are your top research goals over the next year, and what will it take to reach those goals?
Over the next year we will carefully steward the projects underway, continue expanding the SolveCFS BioBank, and establish new collaborations to make use of its resources. We will seek partners to advance the outcomes of the projects being conducted with Biovista and LogosOmix. We hope that successes from these approaches will provide revenue-generating opportunities, possibly by licensing the LogosOmix approach to other institutions. We will host an international consortium of investigators focused on designing the next generation of clinical trials for CFS. We will be the leading force for identifying effective treatment for CFS.

To accomplish these goals, we must attract more financial support to the cause by mobilizing the resources of the patient community, venture philanthropy, pharma, and biotech. CFS precipitates huge unmet medical needs that result in annual economic losses of about $30 billion a year. With no approved diagnostic or therapy, it also represents a substantial new market. Our communications and development activities must evolve to support our dynamic research program.

Our governance model will also have to evolve. Hands-off granting mechanisms have been the traditional mechanism used by nonprofits to support research, but shifting from a passive "benefactor" role to active partner warrants experimentation with novel funding mechanisms that yield a greater stake in the outcomes. We're learning from organizations like the Multiple Myeloma Research Foundation and Parent Project Muscular Dystrophy at the leading edge of this paradigm shift. It's an exciting time for our organization and others driving change for their communities!