Empowering People with ALS to be 'Research Ambassadors'

by Nancy West on Sun, 2012-01-01 12:00

New training teaches individuals and families how to advocate for ALS research

Article Highlights:
  • The ALS Clinical Research Learning Institute (CRLI) was designed to help those affected by ALS and their families become "research ambassadors" —  advocates who can help raise awareness about ALS research and encourage participation in clinical trials.
  • Faculty and staff from 10 ALS research institutions provided participants with a training session on the fundamentals of research, as well as clinical trials, news about the ALS research pipeline and ALS advocacy.
  • ALS research ambassadors can help bring ALS research into the forefront by lobbying in Washington, speaking at support groups and educating others about the benefits of enrolling in clinical trials.

Fight smart, not just hard. That’s the motto of Richard Bedlack, a world-renowned ALS research warrior who directs the ALS Clinic at Duke University, Durham, N.C. “Everyone I’ve ever met who is affected by ALS wants to roll up their sleeves and do something about it,” he says.

Now individuals with ALS and their families have a new way to join the fight to find a cure for ALS. Last October, almost two dozen people attended the first annual ALS Clinical Research Learning Institute (CRLI), organized and led by Bedlack and Merit Cudkowicz, director of the MDA/ALS Center at Massachusetts General Hospital (Boston) and professor of neurology at Harvard Medical School. Cudkowicz also serves on MDA’s Medical Advisory Committee (MAC).

Learning research fundamentals

Designed to help those affected by ALS become “research ambassadors,” the CRLI featured faculty and staff from 10 ALS research institutions. This distinguished group outlined for participants the fundamentals of research, including trial design, ethics, informed consent, funding, regulatory affairs and enrollment barriers, as well as news about the ALS research pipeline and advocacy opportunities.

“We had open discussion of therapy development in ALS, what are the hopes, the challenges, and how we can work together to efficiently develop new treatments for people with ALS today,” says Cudkowicz.

A segment of the CRLI was devoted to understanding scholarly ALS research papers. Working in small groups, participants learned to critique a study. People with ALS want to keep up with the latest research articles “as much as we investigators do,” says Bedlack, but they don’t always have the background and training that allows for critical appraisal and interpretation.

“We want to teach them to find the hypothesis, dissect the methods used to investigate it, and be able to accurately assess the results and conclusions,” he says. “With this knowledge, our new research ambassadors are better prepared to go out and make contacts to help optimize ALS research in many ways, whether lobbying in Washington, speaking at support groups, or educating others with ALS about enrollment in clinical trials through blogs and online social networks.”

Addressing low enrollment

The idea for the CRLI grew from the discovery that only a small number of patients in ALS clinics have been enrolling in clinical research studies. “Yet, the only way to develop better therapies is through research,” observes Bedlack.

A study conducted by Bedlack and colleagues found that two of the biggest barriers to research enrollment are lack of awareness of research opportunities and misconceptions about the research process. Low enrollment in studies is a huge detriment to research because, as a result, trials take longer, cost more, and the results may not be generalized to the majority of people with ALS.

It’s very common for individuals with ALS to turn down participation in a clinical trial because they don’t want to be “guinea pigs,” says Bedlack.

Such ethical concerns were addressed at the CLRI training by James Russell, director of the neurology residency program at the Lahey Clinic, who discussed the protections now afforded to patients who enroll in research.

In settings such as ALS support groups, “We are counting on our new research ambassadors to help dispel the fears that patients have about research,” Bedlack continues. “By explaining the research process and discussing misconceptions, we can help them argue against those and other issues that are slowing our progress in finding a cure for ALS.”

Breaking down barriers with education and advocacy

Other barriers to optimal ALS research include funding limitations and government regulations, which Bedlack and Cudkowicz believe might be reduced through education and advocacy efforts.

“These efforts would be most effective if undertaken by large groups — not just scientists and clinicians, but also patients and caregivers who have the most at stake,” says Bedlack.

In developing the CRLI, Bedlack and Cudkowicz modeled it after a similar initiative that has been very successful in the Parkinson’s disease community. “We learned of their efforts to train patients and caregivers who are innately talented and charismatic — people who make others sit up and listen when they talk, and we sought out the same kind of people in the ALS community.” CRLI faculty members nominated patients and caregivers from within their practices to participate.

“So many people in the ALS community have so many diverse talents,” says Annie Kennedy, MDA senior vice president of Advocacy. “Many are professionals who can no longer work because of their disease progression or because they are caregivers. But armed with knowledge from the CRLI, they can use their talent and ability to make powerful connections with people and advocate very effectively, in some cases without ever leaving home.”

Empowering people as advocates

One CRLI participant was Mary Murray, 66, of Braintree, Mass., who received an ALS diagnosis in January 2010.

“I feel empowered by the fact that the research community is looking to us to help spread the word about clinical trials and encourage participation,” she says. “ALS is often a fast-acting disease. So many patients get ‘too sick too soon’ to become active in the community. Those of us who have slower-developing symptoms must try to encourage new research to find new treatments for this disease. We also can encourage other patients with ALS to participate in clinical trials by helping them to understand what is involved.”

Murray has been participating in a clinical trial of dexpramipexole since April 2011.

One of the most powerful ways to advocate is to reach out to elected officials and tell your story. MDA provides a variety of advocacy opportunities, such as its yearly MDA Fly Out, which schedules visits to legislators when they return to their home districts from Washington D.C.; and its “Take 5” alerts about pending legislation of interest. (For more information about advocacy activities — both formal and informal — see "Use Your Talents to Advance ALS Research and Build Awareness" below.)

“The power of patients is amazing,” says Bedlack, and Laura Tuttle, another CRLI participant, agrees. Tuttle, 49, of Raynham, Mass., learned in December 2009 that she has ALS. “It’s important for patients to give their point of view as well as clinicians. You can really have an impact when you tell your story,” she says.

Future opportunities

In the future, ALS research ambassadors may have opportunities to serve on federal committees such as the National Institutes of Health Advisory Councils that review research and grants, or U.S. Food and Drug Administration ad hoc committees convened for a drug review.

“When you sit on a committee as a layperson, you’re not expected to be a scientist, but you need to have an appreciation of the scientific process,” says Kennedy. “By providing that knowledge to our research ambassadors, the CRLI has helped to improve the likelihood that we will have people who meet the criteria to fill these committee openings as they become available.”

Bedlack also hopes to see CRLI participants blogging on the new blog space recently created on the Northeast ALS (NEALS) website.

“Attending a program like CRLI, targeting support groups, talking to Congress, participating in studies — to me, that’s a combination of fighting hard and smart that will help us move research toward a cure for ALS faster,” emphasizes Bedlack.

“The more information we each have about the disease and the newest work being done, the better,” adds Murray. “We cannot afford to let the new and exciting momentum in research suffer from lack of support — either the support of a knowledgeable and active community or financial support.”

Nancy West is a freelance writer based in Fort Washington, Pa. She is a staunch advocate in behalf of her husband, Tim, who is affected by Pompe disease (acid maltase deficiency).

Individuals with ALS and their families are a dedicated and talented group! There are many ways to use these diverse talents and skills to advance ALS research and build awareness of the needs of the ALS community.

No matter what your professional background or available free time, there’s a role for everyone within MDA’s ALS advocacy community; please consider becoming more involved in 2012.

Become an MDA Advocate to receive timely updates on policy issues that impact the ALS community and to become involved in current advocacy campaigns. It’s not necessary to have an ALS diagnosis to be an MDA Advocate — it’s open to all. Register as an MDA Advocate and ask your friends, neighbors, coworkers and family members to do so too. Let’s ensure that the ALS community’s voice is heard loud and clear by policymakers!

Stay informed about current research and clinical trials via the MDA/ALS Newsmagazine, MDA website and your MDA clinic. Sign up to receive monthly email summaries of online research news.

Participate in the MDA Fly Out. Many organizations have "fly-ins," in which individuals and families travel to Washington, D.C., to meet with their federal representatives. The MDA Fly Out provides an effective, accessible and less-costly alternative, in which meetings are arranged in local congressional offices when the representatives "fly out" to visit their home districts, both during the August recess each year and during critical legislative efforts being conducted by MDA. To learn more about participating in the MDA Fly Out, contact your local MDA office.

Participate in MDA support groups and local outreach events, and take advantage of opportunities to inform others about research and advocacy news. Contact your local office to learn more.

Help plan an MDA fundraising or social event. MDA is proud to be the world leader in ALS research and services, and grateful to the hundreds of thousands of Americans who contribute year-round, helping to raise the vital funds that make these programs possible. Contact your local MDA office to learn more about getting involved.

MDA’s National Task Force on Public Awareness is an advisory body composed of individuals with neuromuscular disease who are leaders in their communities and achievers in their professional fields. The group serves as consultants to MDA on issues of concern to people with disabilities. Please notify your local MDA Health Care Services Coordinator if you’d like to be considered to serve on MDA’s National Task Force.

MDA’s annual campaigns such as Anyone’s Life Story (May) and MDA Salutes Caregivers (November) help raise awareness of the challenges faced by people with ALS and their loved ones. Support these campaigns through your participation and by spreading the word about them to others.

Federal Advisory Councils: Individuals with ALS and their family members can participate on federal advisory councils and ad-hoc committees as consumer representatives at the National Institutes of Health, the Food and Drug Administration, and other federal agencies.

National ALS Registry: A working group of scientific and clinical leaders in the field of ALS, representatives from MDA and the ALS Association, and members of the ALS community serve as advisers to the Centers for Disease Control’s oversight of the National ALS Registry. Input from the ALS community is the critical component in this collaboration.

Speak up: Opportunities for ALS advocacy can be found through letters to the editor; letters, calls and emails to your legislators; blogs; Facebook and other social media; and online communities.

The strength of the MDA ALS community depends on educated and empowered advocates. There’s a critical role for everyone in the fight against ALS!

To learn more, contact MDA’s Advocacy office at advocacy@mdausa.org or call your local MDA office at (800) 572-1717.

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