In February 2014, the Huntington’s Disease Society of America officially endorsed the Enroll-HD study—the first time that the national nonprofit patient-advocacy organization has signed on to any research project. “There aren’t a lot of times in science that you can call something a potential game-changer, but this is one of those times,” says Louise Vetter, Chief Executive Officer of the HDSA.
Because the organization’s mission is to support the entire HD community, HDSA hasn’t put its weight behind studies of individual drugs or treatments before, says Vetter. But because Enroll-HD is inclusive and has such broad goals, this is a different situation. “This is more than your average study,” she says. “It’s a platform from which future HD science will be made possible.” The HDSA membership will have the chance to learn more about the study this month at the annual convention in Louisville, Kentucky.
HDSA plans to help raise awareness about the study with an educational campaign through its 54 local chapters and affiliated organizations nationwide. The idea is to make sure that everyone who might be interested in participating hears about it, and to demystify how the study works and what it involves. “We want there to be broad community awareness so that people can decide whether or not to get involved,” says Vetter.
A careful decision
The HDSA board spent a long time talking over the decision, says board member Jang-Ho Cha, MD, PhD, who has been involved in HD research at universities and pharmaceutical companies for decades. Ultimately, they agreed that Enroll-HD’s aim of accelerating all HD research was worth endorsing. “This study is so important for laying the groundwork for the clinical trials that we anticipate will be coming down the road,” he says.
“There aren’t a lot of times in science that you can call something a potential game-changer, but this is one of those times.” — Louise Vetter
There are roughly 7,000 “orphan diseases” like HD that affect a relatively small number of people. Pharmaceutical companies that want to test new drugs for these diseases often struggle to find enough study participants. Without enough people, “a lot of companies will walk away from that disease, even if there’s a good scientific rationale and unmet medical need,” says Cha. Enroll-HD is helping to establish many clinical centers with trained experts who know how to assess people with HD, and a registry of potential volunteers who may be willing to participate in drug trials. These tools will catch the attention of pharmaceutical companies and help bump HD to the top of their priority list.
Both Cha and Vetter emphasize that getting involved in research is a personal decision. Not everyone may want to participate. But everyone who wants to do so should have the opportunity. “Everyone asks what they can do,” says George Yohrling, PhD, Director of Medical and Scientific Affairs at the HDSA. “The greatest thing they can do is get involved in the research process. You don’t have to sit on the sidelines waiting— you can be an active participant in the fight against HD.”
The potential of Enroll-HD to speed up how drugs are tested is valuable, says Vetter. It allows the research community to find out quickly whether something works, and use that information to move to the next step. “Enroll-HD provides a concrete way to get involved without having to get tested [for the HD gene] or make decisions about therapies,” says Vetter. “It’s a simple way to do something, and that’s important.”
This story was originally published in the June 2014 issue of Enroll!