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Scleroderma Foundation Gives Voice to 'Overlooked' Disease
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In a world where potentially fatal, high-profile diseases like breast cancer and diabetes devour resources and media attention, organizations like the Scleroderma Foundation are necessary to advocate for patients with lesser known diseases.
Scleroderma impacts an estimated 300,000 Americans, with approximately one-third of those patients experiencing the systemic form of the disease, and the remainder managing local manifestations. Women outnumber men with scleroderma four to one. However, the causes of this disease are unknown, and there is no cure. It is because of this that the three-pronged mission of the Scleroderma Foundation — support for patients and their families, education and research — is so critical.
Robert J. Riggs, CEO of the Foundation, told Healio Rheumatology that there were just 62 papers published on scleroderma in 1950, but that since 2000, there have been more than 12,000. “That expansion of activity is a true achievement, but it is not satisfactory if we are to find a cure,” he said. “That number must explode, but that requires dramatically greater funding.”
Riggs explained that from the 1970s, there were actually two scleroderma-based organizations that merged to form the current iteration. The Scleroderma Foundation as it is known today is in its 21st year. “I have been here 23 years, so I have seen the growth from when it was two organizations to what we can accomplish when we pooled our resources,” he said. “One of the things that makes us unique is that we have a national presence, with 20 chapters and 160 support groups throughout the U.S. and Puerto Rico.”
Riggs noted that many of the programs and services are carried out at the chapter level. “However, certain initiatives like research and awareness are managed exclusively by the national organization because we have a pretty extensive network,” he said. “Our mission guides everything we do. We are a patient organization primarily.”
With so little still known about the genetic and environmental factors that cause the disease, Riggs stressed the research component of the Foundation’s mission. “We are the largest non-profit organization that provides peer-reviewed research to the scleroderma community,” he said. “Our grant mechanisms fund early career investigators, but also those who are well established in the field. We fund basic, translational and clinical research.”
In particular, Riggs highlighted the research grant program as the component that has made the most impact on the scleroderma community. “It is modeled on how the NIH reviews and scores grants,” he said. He added that these efforts have been ongoing for almost 4 decades as part of the two predecessor organizations, which means there are literally generations of experts who started with grants from the Scleroderma Foundation.
“We have helped launch the careers of almost all of the senior investigators working in the scleroderma field today, who are undertaking significantly larger grants or working at the NIH,” Riggs said. “The tentacles of the basic science and clinical research communities are all connected to our organization, and that leadership continues to be a point of pride for us as we look to the next generation of therapies for this disease.”
The major challenge facing the scleroderma community is that there are no on-label treatments to manage the disease, according to Riggs. “We support the researcher and the science,” he said.
Nationwide Presence
Riggs pointed out that chapters of the organization vary in size. “We have chapters that cover geographical territories such as New England, or the tristate area of New York, New Jersey and Connecticut,” he said. “There is one chapter in Texas and another in southern California but not in northern California.”
He stressed that all major metropolitan areas of the country have a Scleroderma Foundation presence in it. “All of these centers have outreach and education into the patient and medical communities through local health fairs and other such activities,” he said. “It is important for us to make sure local rheumatologists know we exist as an organization that can provide information for them and their patients.”
This presence and outreach are attempts to counteract the manpower shortage in the rheumatology community, according to Riggs. “The lack of rheumatologists is of great concern, but we are trying to view this crisis as an opportunity,” he said. “Through close relationships with the Rheumatology Research Foundation and the American College of Rheumatology, we are focused on capturing those early career researchers who may be rheumatologists, as well as those who are PhDs with interest in basic science. We want to do everything we can to help these researchers grow and expand in the field of scleroderma.”
Scleroderma stands as a rare disease in a field already marked by a shortage of clinicians, but having even a few experts who are focused exclusively on the disease for an entire career can lead to great strides and breakthroughs. “NIH and National Institute of Arthritis and Musculoskeletal and Skin Diseases funding is generally more focused on rheumatoid arthritis, lupus and other diseases,” Riggs said. “Smaller population disease states struggle to get funding. People who want to do research follow where the money is, so we try to do as much as we can to help with that.”
Riggs highlighted two programs that he feels represent what the Scleroderma Foundation is trying to do. “One is the early career investigators workshop, which is a 2-day program we hold every other year,” he said. “Invitees present their science and/or hypotheses to established experts in scleroderma research. As far as I know, there are not many programs like that. We have a high success rate of researchers who have gone through that program who ultimately received grants from sources like the NIH.”
The other program is the Scleroderma Foundation’s own research program. “We encourage young investigators to apply,” he said. “It helps them get initial data together, refine it and move on to more career-sustaining grants from other sources.”
Building a Network
In terms of a community of scleroderma patients and providers interacting with one another, Riggs suggested that much of this activity happens at the chapter level, as opposed to the national level. “Increasingly, online forums form our support group network,” he said. “Facebook is the most important tool our community uses to connect, but we also use Twitter and other platforms.”
The role of the regional chapter is to gather information from social media and translate it into activities for patients to attend. The organization supports anything from educational seminars to walkathons.
“We can use social media to spread information about when experts in scleroderma are coming through a particular area to hold mini-conferences,” Riggs said. “In addition to physicians and researchers, our patients can benefit from input from lifestyle experts, physical or occupational therapists, speakers discussing the benefits of exercise, or anyone who can offer insight into a holistic approach to disease management.”
At the national level, a patient education conference takes place each year. “It is the largest patient conference on scleroderma anywhere in the world,” Riggs said. “There is international participation, and a faculty of 50 or 75 experts who touch on every aspect of the disease.”
Videos of the events are available online, Riggs added. “We understand that it can be difficult for many patients to travel to these events, so we do what we can to provide them with as much information as possible,” he said.
To that point, efforts are also made to offer support to families with children who have scleroderma. “It is very rare for children to get scleroderma, particularly the systemic form of the disease,” Riggs said. “It is an overlooked disease population, particularly given the shortage of pediatric rheumatologists.”
The national meeting has a conference within a conference just to address pediatric patients. “We are proud of our efforts to help children with juvenile scleroderma,” he said. “There is a strong network of family members — mostly moms — who talk to us and teach us about how to improve the program. It is a grassroots movement with plenty of give and take between the patients, families and those of us at the Foundation.”
Advocacy and Empowerment
“If there is anything else I would like to highlight, it is our advocacy program and our ‘Patients as Partners’ program,” Riggs said. “These programs are really indicative of how we engage and help patients feel more empowered.”
The advocacy program focuses on bringing patients together with elected officials to understand the impact of this disease. Specifically, the aim is to help government leaders understand the importance of fibrosis in scleroderma, according to Riggs.
“Fibrosis, in some form or another, is responsible for over 45% of deaths in Western civilization,” he said, noting cirrhosis of the liver and the impact on the heart after a cardiac event, among other causes. “Scleroderma is a typical disease for fibrosis. If you can study fibrotic events in a disease like scleroderma that impacts all organ systems, you can potentially mitigate mortality in a number of diseases. We hope that if elected officials can understand this, more resources would come to scleroderma.”
The NIH has brought such research about, but those efforts have largely been focused on specific organ systems. “Studying scleroderma, it may be possible to improve or reverse fibrosis,” Riggs said.
The ‘Patients as Partners’ program was born from the FDA’s demand for increased input from patients in clinical trial development. “We are engaging more with industry,” Riggs said. “We are bringing patients together with scientists and clinicians who are working in the space.”
The efforts of the Foundation are finally paying off. “New research raises a whole new set of questions about the role of genetics vs. environment and offers new opportunities for study,” Riggs said. “Knowing the right questions to ask is necessary to finding answers.” – by Rob Volansky
For more information:
Robert J. Riggs can be reached at 300 Rosewood Drive, Suite 105, Danvers, MA 01923-1389; email: rriggs@scleroderma.org.
References:
Scleroderma Foundation website:
https://www.scleroderma.org/site/SPageServer/#.XML1U5NKjBI
Disclosure: Riggs reports no relevant financial disclosures.