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We spoke with Rhonda, an IBM patient and AllStripes Ambassador, about her participation in IBM research and how other patients can get more involved.
Since her diagnosis in 2015, Rhonda has been involved in multiple efforts to advance research for IBM, which currently has no treatment. She explains that she likes being part of the solution, and hopes to encourage more IBM patients to do the same.
Rhonda described a few of the ways she, and other IBM patients, are making an impact through participating in research.
AllStripes IBM research program
AllStripes is not a traditional patient registry — it is a rare disease research platform that can complement the work a rare disease community is already doing to advance research.
Rhonda volunteered to become an AllStripes Ambassador after the IBM research program was launched in partnership with Myositis Support & Understanding and Cure IBM. By joining this AllStripes research program, IBM patients are able to receive their comprehensive medical records at no cost and contribute de-identified data from their records to be used in research to further IBM treatments. She says AllStripes helps streamline the process for IBM patients to be involved in research from home and help increase the understanding of their condition:
“Together, we can make our voices heard,” she says about the AllStripes IBM program. By being part of AllStripes, Rhonda can contribute to multiple research efforts at once and continue to multiply her impact.
Natural history study
Natural history studies are research studies that look at a group of people with a particular disease over a period of time in order to understand how the disease develops and gain insights about symptoms, and potential treatments.
In 2015, after being misdiagnosed with polymyositis for 12 years, Rhonda received a diagnosis of inclusion body myositis, or IBM. That very day, she became involved in research: the doctor who diagnosed her asked her to participate in an ongoing natural history study, and she gladly agreed.
The doctor sent Rhonda home with a stack of papers to read about the study and two weeks later she was back at UC Irvine going through what she calls a “battery of baseline tests.” She returned every month for follow-up testing for the duration of the study, and was able to learn more about her rare condition in the process.
While Rhonda participated in this study in person, AllStripes is also able to support natural history studies in a different way by enabling researchers to learn from the de-identified records of members of the condition community.
Clinical trials
Clinical trials are research studies that test a potential medical treatment to evaluate its safety and effectiveness. For a drug to get approved by the Food and Drug Administration (FDA), it must demonstrate success in clinical research.
Rhonda refers to involvement in clinical trials as both fascinating and complicated, from her experience. She explains that inclusion criteria to be involved in a clinical trial can be quite strict: “You have to be the exact round peg to fit in the round hole.”
Since IBM has no treatment, Rhonda was eager to get into a clinical trial and three years after her diagnosis she got the opportunity.
The trial she was in was a double-blind trial, so she did not know whether she was on the treatment or placebo. Regardless, she religiously took the trial pills three times a day and “treated it like a job.” Unfortunately, just a few weeks ago it was brought to light that the trial failed because phase 2 of the study did not meet its primary or secondary endpoints. Rhonda calls this news a gut punch, but tries to focus on the fact that failed trials still contribute to research because they help scientists learn more about what doesn’t work -- a statement which she first heard at a seminar a few years ago.
Patient registries
Patient registries are systems of collecting information about people with the same disease that can include patient data, patient reported information and biological samples. Data from patient registries can be used to support research.
Rhonda says there are many patient registries for IBM. As part of a number of these registries, Rhonda participates in ongoing surveys that go out to IBM patients. She thinks registries are useful in helping educate researchers and scientists about a specific disease.
While AllStripes is not a traditional registry, it can complement an existing registry. We are able to generate deep, audit-trailed insights from the community’s records that are not possible through more traditional registries
Rhonda says she is always researching new ways to be involved in IBM research. She does what she can to inform other IBM patients of research efforts they can participate in, too, and is a strong believer that it’s up to their community to continue to drive research forward.
Additionally, Rhonda has taken on a superhero role as the The Myositis Warrior, and has an advocacy group by that name on Facebook as well, to help educate and spur people in her rare community into action.
