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(Article) Patient advocacy groups take research quest to innovative open-access publication

PLoS CurrentsPat Furlong says she has a “microwave mentality.” When she wants something, she wants it instantly.

In her case, that something is to accelerate treatments and cure for Duchenne muscular dystrophy (DMD) – a disease that claimed the lives of her two teenage sons in 1995. Furlong, a nurse, and her physician husband, Tom, who also have two grown daughters, tried to become experts on the condition as they cared for Christopher and Patrick. They have since pushed the agenda forward on behalf of all families affected by Duchenne.

Furlong founded Parent Project Muscular Dystrophy (PPMD) in 1995 to help improve the treatment, quality of life, and long-term outlook for all individuals affected by the disease. The organization works to raise awareness, fund promising research, and has been an active advocate of Open Access to encourage the sharing of scientific progress on Duchenne.

Now, Furlong has blazed a new trail in the fight to end the disease. She has spearheaded a partnership between PPMD and the Public Library of Science (PLoS) to launch an open-access publication, PLoS Currents: Muscular Dystrophy, this fall. The new forum will promote the rapid exchange of information, hypotheses and experimental results related to the rare disease.

“Currents is about encouraging the rapid communication of research in a particular area,” says Mark Patterson, director of publishing for PLoS. “It’s about accelerating the dissemination of new data and also expanding the sharing of new information.”

PLoS Currents is organized in sections and PLoS Currents: Muscular Dystrophy will be its sixth focus area. In 2009, PLoS launched PLoS Currents in response to the H1N1 influenza pandemic and the pressing need for a very rapid communication channel focused on influenza research. It also has a section on Huntington Disease, produced in support with CHDI, a private, not-for-profit research organization working to discover drugs that slow the progression of disease.

Furlong has long been frustrated with the issue of access – having to wait for a journal paper to go through the many steps prior to being published and then, if you don’t have a subscription, having to pay to read the findings. So, she is a big fan of Open Access.
“Open Access helps everyone,” says Furlong. “Families can navigate, learn, read and re-read the most important information because they are connected with the scientific environment and then can make the most informed choices for their children.” And for scientists, it will enhance research, hopefully leading to new discoveries.

About four years ago, PPMD had established a registry, DuchenneConnect (, linking families to genetic counseling and research. But Furlong was looking globally at the community for other ways to share information. There were a number of new industry trials studying Duchenne and she wanted a more robust system to distribute their results.

In early 2010, Furlong consulted her board of directors and then spoke with Liz Allen, Director of Marketing and Business Development at PLoS in San Francisco, about starting a project together. “It made sense to me to pursue an online journal because I think that’s the future,” she says. “PLoS is able to quickly turn around credible information that is peer-reviewed.”
Allen says patient advocacy groups often have sophisticated Web sites, but PLoS can help advance their causes further. “Foundations and groups are eager for a way to quickly communicate the work that they and others fund in their field of interest,” says Allen.

Furlong wants to see all research data related to Duchenne available freely – even negative findings.
“I think we waste too much time, effort and money not learning from studies that didn’t produce positive results. Maybe it was technique or methodology and someone else can learn from that change,” she says. “We have an opportunity because of the connectedness of the community. I think this really will help the community and our goal for Duchenne is to accelerate wherever and whenever we can. We felt PLoS offered significant opportunity to accelerate.”

Compared to a traditional journal, the PLoS Currents publication process is compressed. Authors write their contributions online and when they are satisfied with the way the text, figures, tables and references look, they hit a button and submit it to an editorial process, says Patterson. Then editors do a quick quality control check. Once the material passes that step, the editors choose people from a board of reviewers to identify obvious problems or mistakes. After any necessary revisions, the work can then be immediately published.

Once published, the works from Currents are also archived in Pub Med Central. There is no author fee to publish.

“The idea is to make it very streamlined, quick, and hassle free,” says Patterson. “This publication does all the essential jobs of a journal – including peer review and archiving – but it has the potential to do that very fast and, also, much more cheaply.”

Before PPMD signed on to the partnership, which required a financial commitment, Furlong says she checked with many investigators in the field of Duchenne to make sure they would use the online platform. “Without exception, they said it was time, they would participate, and think it would move the field forward,” she says.  

Furlong and Sharon Hesterlee, the director of research for PPMD, worked with Allen at PLoS on developing the publication, suggesting editors and scientific reviewers.

Allen is working on outreach through ads and social media, to make sure the community has heard of the site and encourage those involved in MD research to submit their work.

In July, there was a soft launch at the PPMD conference and the response was enthusiastic. The official launch is slated for this fall. Furlong says PPMD is committed to the project for two years and then will look at contributions and consult with scientists about moving forward.

“I hope we see more and more data,” says Furlong. “I hope we are able to be much more selective about the targets, methodology, trial design and outcomes. I hope we will continue to see novel approaches to research and I believe we will learn a great deal in the process. The goal is to ensure the pipeline remains full with possibilities and that, one day soon, all individuals diagnosed with Duchenne muscular dystrophy will have access to treatments.”

Patterson says labs are selective in what they choose to publish, but there is more information out there that is worth sharing. “Some data is not being shared because it does not form a coherent story, but it is important to share that data too,” says Patterson. “Combining these findings with other similar studies can generate more meaningful conclusions.”

Allen says the submission doesn’t have to be a full-length article. They can be preliminary findings or ideas that are works in progress. “This is quite different from the current way of doing things,” she says. “It can take people awhile to understand the full potential of this new way of communicating.”

Furlong thinks other patient advocacy groups will likely follow the lead of PPMD and look to online journals to exchange information more quickly.

PLoS Currents

"Pat was quick to grasp the possibilities that an outlet like PLoS Currents presents to share all kinds of credible research results more quickly than ever," said Heather Joseph, Executive Director of SPARC. "The ability to give a targeted community of researchers fast access to the combination of articles, data, and even negative results presents important new opportunities to make new connections and speed the pace of discovery."

When Furlong first got involved in the field, there was a base of knowledge about Duchenne but the investment wasn’t there. In recent years, research on Duchenne has evolved and the community is building partnerships. Centers of excellence have been established at UCLA and the University of Pittsburgh to study aspects of the disease. “We have reached out to industry and induced them to work with us,” says Furlong. “We seeded a great deal of work, more advocacy organizations came on board, and I think we are now at this really critical mass.”

PPMD has grown from 20 parents when it was established to a robust organization with 3,000 families and a database with 30,000 extended families today. It has advocated for research and helped get the MD Care Act passed in 2001 and reauthorized in 2008. Furlong says families used to have little hope when their children were diagnosed, but more advances are being made and there is a shift in attitude about the prospects for a breakthrough.

“For investigators, I hope this enhances what they do and makes their proposals look more interesting and the NIH stand up and take notice,” says Furlong. “This is a useful opportunity to move rare disease research forward.”

-by Caralee Adams