Tag: The Myositis Association

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Patient communities

Friends Who Get It

Being with people who understand what you’re going through can be a life line for those with a rare disease.

“It’s like explaining how chocolate tastes to someone who’s never had chocolate,” Rose Mary Istre says of trying to explain dermatomyositis to her friends and family. “They don’t get it.”

When she was diagnosed more than twenty years ago, even doctors didn’t have a good understanding of this autoimmune muscle disease or how to treat it. Which made Rose Mary feel very much alone and anxious. This is also what made her want to find other people with myositis, people who had tasted this sort of chocolate and knew what it was like.

She contacted The Myositis Association who gave her a list of people in the Houston area who had some form of the disease. In those pre-internet days, Rose Mary sent everyone on the list a survey with a self-addressed, stamped envelope, asking about their interest in getting together for some sort of support group. Shortly after that, 48 of the 50 people on that list showed up at Rose Mary’s house for their first meeting, including one woman who literally crawled in the front door because she was too weak to walk.

“That’s how desperate people were to find someone else who got it,” Rose Mary says. “But the best part was, we realized we weren’t alone. There was somebody else out there struggling with the same issues we had. Even if we thought we just couldn’t cope with it, someone else was out there coping. It gave us hope. So we held hands and became friends and formed a support group, a community.”

When Lekiha Morgan was also diagnosed with dermatomyositis five or six years ago, she too realized she needed to talk to someone. Rose Mary’s group in Houston was almost an hour away from her home in Galveston, but knew she needed to get herself there.

“People around me kept saying you don’t look sick, but my body was catching hell,” she says. “It was great to be around people I could relate to. They also knew more about the disease and taught me things that I could bring home and share with my family and my kids to help them to better understand what I was facing.”

Feeling less alone and having words to express what you’re dealing with are only a few of the ways support groups help those who live with rare diseases like myositis. Simply being able to talk about your feelings and share the frustrations of living with a disease that leaves you unable to pick up your baby or walk up the stairs can be very therapeutic. Even caregivers can benefit from this form of social support, developing improved coping, lower stress levels, less anxiety, and finding hope and purpose in life.

Each support group has their own way of doing things, but they often involve opportunities to both share personal experiences and learn from expert speakers. Rose Mary, for example, has tapped contacts in the medical community, including her own treating physician, infusion nurses, a clinical psychologist, art therapists, a tai chi instructor, and even members of the group who have a special talent they can share.

Among the more popular speakers are medical professionals who are experienced in treating myositis. Members are eager for their answers to questions about everything from treatments and side effects to research and “am I ever going to get better.”

Group members don’t give medical advice, but they are able to talk about what worked for them and to guide people on how to navigate where they are with the disease. Still, this personal experience is often more informed than some physicians who have little experience working with a person with myositis.

“I believe we actually have saved lives at our support group,” Rose Mary says. “We’ve had people who wander into meetings who are going to a medical clinic or seeing a doctor who is not following what we have learned to be a really strong protocol in combating this condition. So our group is a good resource to let people know what’s out there, what’s available in the medical community, and who to contact.”

Lekiha is one of those people who was struggling with treatments that were not working well to control her symptoms and were causing complications. At the Houston support group, she learned about intravenous immune globulin (IVIG) therapy, which had been very effective for several people, including Rose Mary.

“I was having a hard time getting around, and I couldn’t work anymore,” Lekiha says. “But the doctor I was seeing at the time said I wasn’t affected to the degree that she felt I needed IVIG. After talking with people at the support group, I ended up going to see a different neurologist who said I was a perfect candidate for IVIG. She said they don’t want you to get to the point where you’re completely immobile and they can’t bring you out of it. Now IVIG is the only medication that I’m on. IVIG changed my life.”

For many people who live with rare diseases like myositis, having a support group to depend on is a life-saving experience.

“I’m so glad and grateful for this group,” Lekiha says. “It’s a scary disease to face by yourself, especially when nobody you know knows anything about it, even doctors. I’m so thankful for these support groups. I don’t think I would be here without them, honestly.”

Rose Mary, too, is grateful for the folks who have shown up at her meetings.

“I don’t know how I would have survived,” she says. “I feel like I needed them more than they needed me. I felt like I finally had purpose in helping somebody else. So many people benefited, and helping them gave me real courage to carry on. I shudder to think where I would be had I not had that support group.”

Resources

CSI Pharmacy provides a resources page with information that may be helpful for all of our patient communities.

The IVIG/SCIG Patient & Provider Support Group is a private Facebook group for those who use immune globulin therapies.

Cure JM Foundation seeks to find a cure and better treatments for Juvenile Myositis (JM) and improve the lives of families affected by JM.

The Myositis Association (TMA) is the leading international organization committed to the global community of people living with myositis, their care partners, family members, and the medical community.

Myositis Support and Understanding (MSU) is a patient-centered, all-volunteer, 501(c)(3) nonprofit organization empowering the myositis community through education, support, awareness, advocacy, access to research, and need-based financial assistance.

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Patient communities

Finding Hope through Community

Larry and JoAnn smiling at CSI Pharmacy success

Larry Leisher had no idea what inclusion body myositis (IBM) was when he was diagnosed with this slowly progressive muscle disease 16 years ago. He’d been having trouble going up and down the stairs for a while before that. His legs were looking very thin, and he was falling too often for it to be normal. When the diagnosis came, it threw Larry and his wife JoAnn for a loop.

“The biggest problem was not having any idea where it came from or how it started,” Larry says. “I’d never really been a sick person, so to have this come upon me the way it did was really devastating for both JoAnn and me.”

The Leishers are not alone in feeling confused and overwhelmed. Being diagnosed with a rare, chronic condition, especially one like IBM that has no treatments, can be one of the most difficult and stressful life experiences, for both the patient and her or his partner. Suddenly, a spouse becomes a caregiver and the life they thought they were living has been upended. Plans for the future, work and social life, and relationships all have to be retooled to include medical interventions and disability. None of that is easy.

About a year into his diagnosis, Larry ran across an article in the local paper written by Lee Thames, the first person Larry ever heard of who had IBM. So he called him.

“Lee and I got together and decided to start a support group,” Larry says. “At first it was just he and I. Our first meeting was in February of 2007, and it’s been going ever since.”

Getting together with other people who also live with myositis can be a life saver. It’s a way to learn more about this condition that most people have never heard of before. And because no one else in your life really knows what you’re going through, sharing the story of your journey with someone else who understands the challenges of living with this life-changing disease can lighten the load, whether you’re the patient or the caregiver.

Over the years, the myositis support group Larry helped to start in Jacksonville has grown to include 20-25 people, both patients and their caregivers. Eventually they connected with The Myositis Association (TMA) and became an official part of the organization’s national network of support groups.

Before the pandemic ended in-person meetings, they would try to get together quarterly. Larry arranged for speakers to come in and talk with the group about different aspects of their disease. 

“A support group is a good thing to have,” Larry says. “It gives hope to those who have none. It gives people a chance to talk with others who have the same difficulties. We talk about things that get us through the day, things that you can’t just turn on the computer and find.”

But it’s not just about the meetings. Larry works hard to make sure members of his group are taken care of. He calls everyone every few weeks, just to check in. And pre-pandemic, he and another member, Jim Farrlley, would also get in the car and drive as far as 70 miles to visit members who were no longer able to make it to meetings.

These efforts have paid off. Before the pandemic, the group was so connected that, even after their loved one passed away, a couple of caregivers still attended in-person meetings. And more than one member continued with the group even after their doctor decided they didn’t have myositis after all. 

Like everything in our lives over the last year or so, however, the social distancing has made it difficult for members to be there for each other. While many events have moved online through video meeting apps like Zoom, that hasn’t been an option for the Jacksonville group. Too many of the members are unfamiliar and uncomfortable with this technology, and some don’t have access to a computer.

So Larry has been on the phone a lot. He calls members weekly or biweekly, just to let them know they’re still part of a supportive community that cares about them and to keep them abreast of what’s going on at TMA and in the myositis research world.

“It’s been hard since we can’t get out to visit people,” Larry says. “So I just try to keep the conversations going. A lot of people are disappointed because we’re no longer getting together. Things are getting more and more difficult for folks…problems at home and such.”

More than anything, Larry has an unwavering positive attitude. He is committed to his mission of passing on hope to folks who may be feeling even more lost and alone than in normal times.

Recently, for example, a report on arimoclomol, a new medication being tested for IBM, showed the drug had failed to meet research expectations in clinical trials. It was devastating news for IBM patients who had pinned all their hopes on this, the only drug anywhere close to being a possible therapy for their disease.

“I contacted our members who have IBM and let them know about the results,” Larry says. “But I also let them know that, although it failed, this was not a time to give up. We still have to have hope. We still have to support the research and TMA.”

In the end, this optimistic attitude and sharing it with others keeps him going too. He refuses to let this disease take him down.

“I don’t want to lose the group,” Larry says. “Even if it’s shrinking in size, I still feel that, between our group and TMA, there’s a lot of hope that can be passed around.”

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Patient stories

Rolling with It

Bill and Linda Mathews smiling together.

When Linda Matthews started having trouble standing up out of a chair, she wasn’t surprised. She was 70 years old, about the same age as her mother when she was diagnosed with inclusion body myositis (IBM).

“I was her caretaker, so I knew all about IBM,” Linda says. “As soon as I started showing symptoms, I went to my doctor and I said I’ve got IBM. They did the muscle biopsy and the bloodwork and the EMG. Within a week, it was definitely diagnosed.”

Often when someone is diagnosed with a disabling condition like IBM, they rely on their partner or a family member to care for them as the tasks of daily living become more difficult to manage alone. Linda, however, was already serving as caregiver for her husband Bill, who had been recently diagnosed with a different rare, autoimmune muscle disease: myasthenia gravis (MG).

In March of 2012, Bill had heart bypass surgery. About a month or so later during a routine eye exam, his ophthalmologist noticed his eyes looked a bit droopy. Linda’s brother, who had a friend with MG suggested he see a neurologist. When he did, it didn’t take long for the diagnosis to be confirmed. Bill says his neurologist thought it was the surgery that set it off.

Despite their disabling diagnoses, the Matthews do not despair. In fact, sharing similar diseases makes them more sensitive to each other’s needs. They have many of the same symptoms—muscle weakness, fatigue, mobility challenges—so they each understand, for example, when the other says they need to rest.

“Nothing is as important as getting the rest you need and taking care of yourself,” Linda says. “And it’s good that I know he’s got my back. I can say, ‘No, not right now,’ and I don’t have to justify it.”

“Sometimes you can tell when the care partner doesn’t understand that,” says Bill of other couples they’ve met. “And you can see how difficult it makes it for the person who’s got the disease. In a way, it’s to our advantage that we understand firsthand what the disease is doing to each other. We don’t have to have that fight.”

Having her mother pave the way for her has also made Linda’s journey just a bit easier. She knows what to expect, and she can plan accordingly as her muscle weakness progresses.

When she saw a Hoyer lift at an estate sale, for example, she scooped it up, knowing that one day it may come in handy if she starts having trouble moving from the bed to the chair. She has a motorized wheelchair that somebody gave her. It’s parked in the garage, though, since she still gets around fine with a rollator. The couple also recently traded their car for a wheelchair accessible van.

When they downsized their home earlier this year, Linda and Bill moved to a one-story (stairs had become a challenge) duplex next to their daughter’s family. Before they moved in, they remodeled the house to accommodate their future needs, adding a roll-in shower, raised toilets, grab bars in the bathroom, and wider doors that will accommodate a wheelchair.

The one thing that couldn’t be retrofitted for accessibility, though, was the kitchen. But Linda just winks and says, “Maybe by then I’ll teach Bill to cook, because I’m the one in the wheelchair.”

That’s not a problem for Bill. “We’ve been married for 56 years. We know how it’s done,” he says.

Linda and Bill feel very lucky to have the support of their family. They love living next to their daughter, son-in-law, and nine-year-old grandson and call on them when they need a hand. When they want to give their son-in-law a break—like when Bill needed to get to the hospital recently for cataract surgery—one of their two sons is always willing to step in and help out too.

Being part of a patient support community helps the couple in other ways. They belong to MG Texas, an MG support group in the Dallas/Fort Worth area. And they are active in Northeast Texas support group of The Myositis Association as well as on Facebook forums through Myositis Support and Understanding.

Getting together with others who share their disease helps Linda and Bill learn the medical ins and outs of both diseases, so they are better able to care for themselves and each other. It also gives them access to a whole pool of practical information that’s not readily available elsewhere: things like who is the best neurologist in a certain area, or tips and tricks people have used for managing certain challenges.

“The myositis Facebook site is wonderful, because people ask questions, and a lot of times it’s a question I have too,” Linda says. “And then in the MG group, we love it when we are able to go to the meetings. They’re always interesting, with new speakers and new ideas. It’s also nice getting to know other members who are in the same situation as we are.”

While living with conditions they know can cause significant health concerns, Linda and Bill remain upbeat.

“My mother was a wonderful example for me how to live my life with this disease,” Linda says. “You just roll with it and try to figure out another way of accomplishing your tasks.”

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Patient communities

Connecting with Patients is the Reward

Huub Kreuwel

As a research scientist, immunologist Huub Kreuwel, PhD never really worked with patients. He spent most of his time in an academic lab, trying to understand the basic biology of certain diseases and identifying molecules that could serve as targets for new therapies. He never got to see what happened in the later stages of drug development—that part where patients got better because of the discoveries he’d made.

When he left academia to serve as medical science liaison at Johnson and Johnson, however, he discovered a whole new experience. Now, years later, as Vice President for Scientific and Medical Affairs in the United States for Octapharma, talking to patients and providers about the plasma-based products his company produces is the best part of his job.

“When I came out of academia, I found it was very satisfying to actually talk to a patient who had tried our drug and had good results,” he says. “As an immunologist, it made sense to work on a lot of these rare diseases like primary immune deficiency and dermatomyositis. And it’s gotten more and more interesting over the years.”

Working in the medical affairs department also offers the opportunity to get involved with a wide variety of projects. Huub and his team work with regulatory agencies when the company is seeking approval for new products. They help set up clinical trials to test new therapies and answer physicians’ questions about how those therapies work. Best of all, he meets the people who benefit from Octapharma’s treatments, such as immune globulin (IG) therapies, and helps them enroll as research subjects in the company’s clinical trials.

Recently, the company completed a trial testing intravenous immune globulin (IVIG) therapy in patients with dermatomyositis (DM). While the results have not yet been made public, Huub says the trial did meet its primary endpoints, so it looks very promising that Octagam 10% will eventually become one of the few FDA-approved treatments for this disabling disease that affects the skin and muscles.

Part of what made this trial so successful was the feedback Huub and his team received from patients. In the process of developing the clinical trial, they worked with patient organizations, including The Myositis Association and Myositis Support and Understanding, to understand how patients experienced the disease so they could improve the study protocol and to help recruit participants for the trial.

“We work on a lot of orphan drugs,” Huub says. “And there aren’t that many patients sometimes, so we need everybody to help us to finish these trials. It worked quite well in the DM trial. Those were very productive relationships.”

The success Octapharma had with this phase III clinical trial with DM will also pave the way for future clinical trials for this indication. When rare diseases have few previous clinical trials, researchers often fumble to find tests that will tell them whether a particular drug is working or not. Octapharma’s trial in DM not only proved that the treatment was effective, it also showed that their measures of effectiveness worked in this patient population.

Huub is now developing protocols to test Octapharma products with other diseases. Among these are pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS for short—a disease in which psychiatric symptoms such as obsessive-compulsive disorder appear suddenly after a strep infection) and secondary immune deficiency (SID—a problem that occurs when immune system deficiencies occur because of something other than genetics, such as HIV or chemotherapy).

As they did with the DM study, he and his team are talking to patients to get input that will improve these studies. One way they do this is by recruiting an advisory board of about a dozen patients who spend the day with company representatives sharing their experiences and suggestions. These open-ended discussions provide insights into all manner of ideas: how to better explain data, ideas for new trials, how patients need to be supported during a trial, and more.

“Those discussions are really good for the company, and usually they’re very productive,” Huub says. “Often patients have ideas for new products or practical solutions that might make our products better. And a lot of times it actually has led to either different products or different marketing material or revamping our website or providing patient education sessions.”

These days the thing that has captured Huub’s interest is COVID-19. Healthcare providers on the front lines of the pandemic are finding success in treating the virus with IG. In fact, recent events have made Octapharma a leader in exploring new therapies for COVID-19.

The company is currently supporting two investigator-initiated projects—one testing IVIG as a treatment for COVID-related respiratory failure, the other using IVIG and steroids to treat COVID-19 patients who are developing heart problems. Octapharma is also conducting their own phase III clinical trial to see if high-dose IVIG can be used to improve severe COVID-19 symptoms. Initial results from the investigator-initiated study with COVID-related respiratory failure are very promising.

“Of course COVID is horrible,” Huub says. “But it also became an opportunity for us to delve deeper into IVIG and how it can potentially work in that disease. It’s very satisfying for me personally and for my team to try and come up with other drugs that could help COVID. So overall, it’s been a very interesting ride.”

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Patient stories

When You Need a Little Help from Your Friends

Maddie Cure, a young female advocate.

Madison Davis had been planning her career in the military since she was seven or eight years old. Living near the Naval Academy in Annapolis, Maryland, she joined the Naval Sea Cadets and was intent on moving up through the ranks. She strategized her high school resume to help her get accepted into the Coast Guard Academy, as a soccer player, if possible.

“She was obsessed with the Naval Academy,” says her mom, Christine Davis. “She always wanted to visit there and wear the uniform and work on advancing her ranks. She was a big creator, too, a lego girl, up until she was 13 or so. She always talked about wanting to work on Lear Jets, fixing planes. She always knew what she wanted to do, and she was excited.”

In May 2017, however, gazing down at her daughter lying limply in a hospital bed as doctors gave their diagnosis, a strange, rare disease that seven weeks earlier had weakened Maddie’s muscles, given her a rash over large portions of her body, and paralyzed her into the worst pain she’d ever experienced, Chris cried. Not only was her daughter suffering, but she knew all Maddie’s dreams had just been evaporated by an autoimmune disease they couldn’t even pronounce: juvenile dermatomyositis (JDM).

“I was so numb to everything in the hospital. It really didn’t hit me,” Maddie says. “I wasn’t actually facing all these things I had just lost. I was just on to the next thing. Like, okay, so what do I do now? What schools do I apply to now? I didn’t really give myself a lot of time to deal with it in the moment. And believe it or not, I felt like I was handling everything very well.”

The next few years were difficult for Maddie. She is fortunate that she lives near one of the world’s leading Myositis Centers at Johns Hopkins University, so the medical treatment she received, including prednisone, methotrexate, and intravenous venous immune globulin (IVIG) therapy, was excellent and effective. Her mental health, however, was a different story.

Maddie missed out on much of the excitement of her senior year of high school because she was too weak to participate in many of the activities. Not only was her athletic strength gone, but the prednisone, a steroid medication, made her gain weight and gave her what she calls “’roid rage,” making her emotionally erratic. She lost friendships, because she missed a lot of school and couldn’t go out on weekends, and her friends just didn’t understand why she was behaving so strangely.

“She suffered a lot in many different ways,” Chris says. “We were just trying to survive it every day and trying to seek answers to make it better.”

Maddie and Chris did find answers, mostly from others who live with rare diseases and know what Maddie was going through. On her first outing after spending three weeks in the hospital followed by two weeks in rehab to relearn how to walk, Maddie and Chris attended a local support group meeting of The Myositis Association (TMA). The speakers at that meeting happened to be three members of the CSI Pharmacy team, talking about immune globulin (IG) therapy and home infusion.

During the presentation, a representative from CSI Pharmacy shared her own journey with myasthenia gravis—another rare autoimmune disease—and IG therapy. In her talk, she described how her disease had ended her career as a Navy nurse, and how hard that was to deal with.

Maddie remembers glancing at her mother and whispering, incredulous, “Did she just say she was in the Navy?”

After the talk, Maddie and Chris approached the speaker and shared Maddie’s similar regrets about losing her own dream career in the military. After that, The two not only became good friends, but she also became Maddie’s home infusion nurse, allowing Maddie to receive her IVIG treatments at home rather than in the hospital infusion center. Later, when Maddie went off to York College of Pennsylvania, her new friend helped her learn how to give her own IG treatments subcutaneously (SCIG, under the skin, rather than in the vein).

The Davises also found out about Cure JM Foundation, a group more focused on children with myositis. They attended the Cure JM annual patient conference, and there Maddie befriended a group of other teens who also lived with myositis. It didn’t take long for Maddie to become a leader in this organization, one of the “older kids” who, the following year, helped lead the panels she had learned so much from the previous year.

Maddie discovered a passion for helping others through the rough spots of living with JDM. She had a Facebook page and website, Maddie Strong, where she talked about her challenges with her disease. Once she even posted a video demonstrating how she gives herself SCIG treatments.

Maddie may have powered through at the beginning of her disease, but eventually the walls started crumbling. During the summer before college, she started looking around and realizing how much had been taken away from her.

“That’s when I started dealing with depression,” she says. “I was thrown so much off course. It was really, really hard. I know everything happens for a reason and I’m really I glad to be where I am. I’m glad I’m at York. I’m actually really happy I’m not playing soccer. But it took a lot out of me.”

It’s the friends she met along the way, the other kids who share this disease and understand the journey, who have helped her get through that dark time.

“They have always been the first families that we’ve reached out to,” Chris says. “If she was feeling really low or like life isn’t worth living anymore or the medicine is just too much, she always had people to turn to who could walk her through it, because they’ve already been there.”

Now as she prepares to start her junior year of college, Maddie is strong. She’s an education major, making the dean’s list, works with K through eighth graders at the York City schools, and is an education technology entrepreneur with a fun teaching app for kids she’s hoping to market. Best of all, her disease is in remission.

Still, kids who are struggling with coming to terms with the life-altering experience of a disabling rare disease diagnosis like JDM are never far from this young advocate’s mind.

“The one thing that I wish I could tell parents is, for kids who are going through this, the mental health aspects are a really big deal,” Maddie says. “I would say focus your attention more on the fact that your kid is going through a lot of mental stuff right now. They are confused and they don’t understand and its really scary. Just talk about what’s going on.”