All groups with a stake in advancing research brainstorm ways to achieve that goal in groundbreaking meeting
In part one of Beyond Celiac Research Summit coverage, patients take center stage in describing what it’s like to live with celiac disease and to participate in clinical trials. Stay tuned for part two, where researchers, physicians, patient advocacy groups, the Food and Drug Administration and a representative from a health insurance company will get their say.
By Amy Ratner, Medical and Science News Analyst
Ann Horsburgh went to a clinic twice a week for five months when she participated in a clinical trial for a new treatment for celiac disease. She gave herself injections at home and consumed a watery concoction containing gluten that caused her to have symptoms.
Still, being in the trial was “a fantastic experience,” she told 50 other patients, physicians, scientists and representatives from drug companies, insurance companies, patient advocacy groups and the US Food and Drug Administration gathered at the recent Beyond Celiac research summit.
“In general, anytime you want to experiment on me, I’m here for you,” said Horsburgh, who went to a dozen doctors in three countries over 19 years before her celiac disease was diagnosed.
The research summit created a rare opportunity for all groups that play a role in advancing new treatments, and eventually a cure for celiac disease, to brainstorm about how to overcome existing barriers and accelerate clinical trials. Beyond Celiac CEO Alice Bast welcomed the diverse group of stakeholders, and Beyond Celiac Chief Scientific Officer Marie Robert, MD provided the framework and structure during the day and kept the meeting on track.
Horsburgh was on a panel of patients who had participated in clinical trials and outlined the ups and some downs of her experience. A second patient panel described the burdens of celiac disease and the gluten-free diet.
Other panels covered an update on current celiac disease treatments being studies, the issues related to including children in clinical trials, the role of patient advocacy groups, and separate perspectives from drug developers, the FDA and a health insurance company. The patient portions of the summit and the description of where celiac disease research currently stands were webcast live to an international audience.
Related: Watch a replay of the summit webcast.
Following the panel presentations, the audience at-large identified steps that could be taken to get past some of the barriers that have prevented celiac disease research from resulting in new treatment for patients. These included:
Roz Schneider, MD, moderator of the panel of patients who had participated in clinical trials, summed up the stories they shared as “the good, bad and ugly” of what it’s like to be involved in a study.
Horsburgh’s experience was largely positive. When her trial concluded she was told that she had been among patients who got the treatment under study and was given all her records from the trial.
But panelists Ellen McKinley, Jen Arters and Tina Ramos said they were not given any information when their trials ended and suggested that researchers be more open with participants once the trial concludes about what role they played and the overall results of the trial.
“I still don’t know exactly what I took or in what amount,” McKinley said of the 16-week trial in which she had to mix powder into a liquid and drink it before each meal.
Arters joined a trial very shortly after being diagnosed with celiac disease before she really understood the condition or the gluten-free diet. Throughout the trial, she took a pill before she ate, and despite her fear of needles, had blood drawn monthly for eight months.
“When the trial was over, I did not know what to do with myself because I felt like I had a cure,” she explained, noting that loss of what seemed to be an effective treatment was one of the difficult aspects of participating.
Ramos has been part of two clinical trials. The first was fairly simple, occasionally drinking a liquid and getting blood tests, and it prompted her to do another. Her second experience was much more complicated, requiring her to go to a treatment site twice a week, where she had “an awful drink.” The first time she had symptoms immediately. The next step involved injections of gluten, including those she gave herself at home. When the trial was over, Ramos was sick for about a week. Still, the most negative aspect, she said, was not knowing the results of the study.
“I’m motivated to participate because I want to help people who are like me.”
“Why do I want to do trials? I’m motivated to participate because I want to help people who are like me, who get the shock of their life [when they are diagnosed with celiac disease],” Ramos said. “I did get compensated, but money was not the motivator. Compensation for me was knowing that I am making a difference to hopefully help researchers and doctors find the information so that someday we will be able to have a cure, or at least be able to go out to dinner and not worry about being the odd person.”
Additionally, Ramos got some personal health benefits out of participating, including biopsy results that gave her information about the condition of her intestine, blood work that helped monitor her thyroid and diabetes and an EKG to detect heart abnormalities.
Schneider, who previously led global patient affairs for Pfizer, noted that across the pharmaceutical industry there is discussion about how to make studies more patient-centered so that participation is less disruptive to daily life.
In addition to more information about the trial and its results, patients agreed that being able to get some study assessments done closer to home would be helpful, particularly because travel sometimes necessitates taking a day off from work.
“Take a moment to look at patients and what we are experiencing,” Arters said. “How far do we have to go? How long to we have to be there? Give us Uber because nobody wants to have to drive after a lengthy injection. Make it easy for us so we can help you in the best way.”
They all agreed with Horsburgh that when a gluten challenge is part of a study, researchers should try to offer something that tastes good instead of the slurry of gluten and water used in some trials.
A panel of patients who addressed the overall difficulty of living with celiac disease noted that one of the first issues is that, unlike other conditions, the burden is not easily recognized by the outside word. But often it includes an impact on quality of life by limiting social activities and educational and employment opportunities.
The constant focus of finding safe food on the gluten-free diet reduces time and energy for other aspects of life. Additionally, the whole family, not just the person who has celiac disease, is affected. Diagnosis is still often delayed and celiac disease education is still limited among healthcare providers.
Julie Kennedy and her oldest daughter have celiac disease and for their family the psychological and social impact is the most difficult. “Every time we go out to eat, every time, without fail, we worry,” Kennedy said.
She described guilt she feels about the six years her daughter went before being diagnosed. “Every time we fed her bread, we might as well have given her a cigarette because of the damage it was doing to her body,” Kennedy explained.
Natalie Dabrowski’s nine-year-old daughter, the oldest of Dabrowski’s four children, lost all of her hair very quickly in the months before her celiac disease diagnosis. When she was tested for celiac disease, her blood test results were off the charts, Dabrowski said.
In retrospect, Dabrowski said hair loss was not the only symptom. Her daughter had severe constipation and irrational anger.
“Of course, when you have multiple children, you wonder if they might have celiac disease as well,” Dabrowski said, noting that her household is gluten-free. “It’s very stressful because you feel like gluten is poison even though it might not be for your other children.”
But the fact that the gluten-free diet could prevent the other children, who were initially screened from being able to be tested and properly diagnosed in the future is another worry. “That’s something that weighs heavily on me,” Dabrowski said.
Katie Kenyon saw doctors for more than a decade during which she was diagnosed with irritable bowel syndrome, depression and anxiety despite the fact that she had classic celiac disease symptoms. Often, she was told stress was the culprit, especially since she worked in with victims of domestic violence. Finally, a new primary care doctor, within minutes of seeing Kenyon in his office, said he thought she probably had celiac disease. After diagnosis, Kenyon had several miscarriages before adopting two sons.
Priyanka Chugh, MD, was diagnosed through family screening when she was 12 after her brother was found to have celiac disease. “I was tall, never had any weight loss issues, never had any belly pain that I could think of,” said Chugh, currently a general surgery resident, said. She explained that it is hard for some who has no symptoms to accept that they need to change their whole life and change their diet
Another challenge for those who are asymptomatic, she said, is that the gluten-free diet does not make them feel better as it does for those who are sick.
“I often go hungry.”
Through medical school and her residency, Chugh found few gluten-free options even in hospitals. In one of her rotations she lost 10 pounds because often the only safe food available was salad. “I often go hungry,” she said. “I think it does impact your ability to think and to function and to be able to just live a normal day, when so much of your brain is preoccupied with what you are going to be able to eat.”
The two patient panels at the summit were organized to give people with celiac disease and parents of children with celiac disease a forum for speaking directly to those who treat patients, design clinical trials, oversee drug approval and will eventually decide whether to offer insurance coverage for an drug that is approved for patient use.