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Celiac symposium highlights importance of research, community partnerships
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On November 9, the patient advocacy group Beyond Celiac held its first Bold Beyond Research Symposium, where researchers, clinicians and community members gathered to discuss the current state and future trends of research in celiac disease.
An expert panel including Ritu Verma, MD, section chief of gastroenterology at The Children’s Hospital of Philadelphia, and professor of clinical pediatrics at the Perelman School of Medicine at the University of Pennsylvania, discussed these topics before a live audience at Drexel University in Philadelphia, which was webcast live to a global audience.
Healio Gastroenterology and Liver Disease recently spoke with Verma about some of the important topics discussed during the symposium, including its major theme of encouraging partnerships between researchers and the celiac disease community.
Healio: This event was designed to spur dialogue about research and community partnerships. What is so important about collaborations between researchers and celiac patients?
Verma: When we present our work in scientific forums as clinicians or researchers, we primarily only reach each other, and when we collaborate with advocacy groups, we reach out to a larger group of stakeholders who are dealing with this disease on a regular basis. If the advocacy groups reach out by themselves, perhaps the stakeholders don’t always take it as seriously, so there’s a nice combination of having the clinicians and researchers join hands with the advocacy groups to reach an audience that is living with celiac disease, while also adding legitimacy, in a sense, by including scientific perspectives.
This really does bring the group together, and I think it gets the people who are dealing with this disease, or any disease, involved in a very different way, in which they might think, “I’ve always felt this was a serious condition, and now that’s validated, because the researchers and the clinicians are joining hands with these advocacy groups.”
Healio: Describe some of the research you’ve done with your colleagues at CHOP that you shared at the symposium.
Verma: I run the Celiac Center at CHOP, and as a clinician, my first instinct has always been to provide early diagnosis, and to achieve this we need to be accessible to the community, and to the pediatricians and family practitioners in the community, so they can reach out to us when they have questions. This is one of the reasons we started the center.
We have worked to improve education about the disease and started various educational events in the community for the patients, families, nurses and physicians. We have tried to individualize and customize the educational programs for the various communities that we serve. We’ve presented at different conferences about how we aim to tailor celiac education for each individual community and this has resulted in increased diagnosis rates and better adherence to the treatment plan. Reaching out to the community has been a big priority for us, and that’s one of the things I think we do well.
We have looked at our diabetic population with celiac disease and compared to the non-diabetics with celiac disease to learn about their differences in serology, biopsies and presentations. We have also looked at a subset of patients to compare the serology to various findings in the biopsies. We have also looked at BMI and celiac disease. To summarize we have tried to look at clinical presentations, laboratory tests and biopsies and compare various populations to learn more about the clinical presentations of this disease.
We have presented some of these findings at various national and international conferences.
Our group’s focus has been educating the patient, family, community, school nurses, chefs, psychologists about the disease, how to cope with the disease and not let the disease take over someone’s life.
In addition to the physicians and practitioners, we aim to be there for the whole family, including the child, parents, grandparents, aunts, and uncles — we want to be the center that helps families deal with this disease, and we also work with schools and colleges to help children adjust well to the disease, and encourage family screening as well. We do have a strong emphasis on early diagnosis of the disease.
Healio: What recent therapeutic advances were discussed at the symposium , and do you feel any particular candidate show s the most promise?
Verma: I do believe that we are closer than ever to have a drug. We have an education day once a year at CHOP, and every year for the last several years we’ve talked about how there will be a drug for celiac disease soon and never delivered that promise. At the symposium, all of us on the panel agreed there truly will be, and I think it will be within the next 5 years. I think this time we may actually be correct! I’m going out on a limb there, but I truly think that this time it’s going to happen.
Of the various drugs that we talked about, one is a pill that patients could take for cross contamination, and I think there’s definitely a role for that. With this, a patient with celiac disease could take the pill and go out to eat, and while they would still need to eat a gluten-free diet, they would not have to be as concerned about cross contamination. So, they can’t go out and eat a regular slice of pizza, but they could have French fries without having to make sure they weren’t cross-contaminated with a small amount of gluten. This takes a huge burden off a parent’s mind and the teenager will not feel ‘different’. Currently some of the teens may ask about cross contamination and feel very different from their friends ordering food or they don’t ask and feel guilty. This pill would help all.
The parents of younger children are always concerned about medications, especially when the gluten-free diet is considered such a safe choice. However, while you can be 100% gluten-free if you’re at home, when you go out is where the fear comes in. Parents always feel that with young children, they have control over what they’re going to feed them, so they’re not very interested in medication, but then comes the teenager, and how many teenagers are going to go out to eat with their friends and ask while standing in a line, “Can you tell me how these French fries were made?” or, “Was this egg made on the same griddle as the pancakes?” So, I think this pill will help alleviate the fear that a parent gets when they have a teenager with celiac disease going out on their own, and in effect it will improve the quality of life for the parents and for the children — and I think that’s exciting.
The second candidate is the vaccine being developed by ImmusanT. I think there’s still a way to go before that becomes available but it is a very exciting concept. Will that be the next sort of revolutionizing therapy for celiac disease? In my mind, yes. The reason this will be really a great step is because it will allow patients to actually eat some gluten. For a patient and family who live with these restrictions any little step that allows them some “freedom” is huge.
Healio: Were there any discuss ions about the new monitoring tools becoming available for patients to track gluten exposure at home?
Verma: The panel did talk among ourselves prior to the event about these new tools, and I think all of us feel that they are promising. There are two important aspects of managing celiac disease: one is the quality of life, and the other is the autoimmune damage to the intestine. In terms of quality of life, some of the stool and urine tests may be particularly helpful.
What happens is that families go out to eat at what they feel is a safe restaurant, but then they may come home, and the patient develops symptoms, and they’re not sure if it is gluten or not. So, a test like this would be very helpful, because a blood test might not be helpful depending on the time it takes to get it done, and depending on how much gluten has been ingested. Conversely, a patient could do a stool or urine test quickly, and if it is abnormal, then they would know that they should not go back to that same restaurant to eat, or discuss the issue with the restaurant at their next visit. This really will help improve quality of life because part of our socialization is about going out to eat, so if a patient goes to a restaurant, has symptoms, but can confirm it’s not related to gluten, then they can feel comfortable going back.
While I do think the stool and the urine tests will help us monitor better, I don’t know whether these tests will really replace doing a second or third biopsy, although I think that’s the goal, that one would be able to make the diagnosis and then be able to correlate those stool and urine tests with serology, and not have to do a second or third biopsy to see if there is healing of the small intestine or not. That’s down the line, though, because so far, we only know that the blood test is not always equal to the amount of damage that’s in your intestine in terms of healing, and that’s why you do another biopsy for some people. We’re hoping that these stool and urine tests may eventually help us avoid some of those second or third biopsies if we can look at them on a more regular basis.
But most importantly, the burden of this disease is so high, so if the pills or tests can help reduce some of the burden by improving quality of life, that in itself is a success.
Healio: What are the barriers to federal funding of celiac disease research?
Verma: First, there has not been much excitement around medications for celiac disease because it’s generally been felt that the gluten-free diet is adequate, so if the pharmaceutical world is not excited, then you don’t get too much funding, and obviously if the pharmaceutical companies feel that there is no market for this, why would they put billions of dollars into trying to develop a medication?
Secondly, we lack consensus on therapeutic endpoints. If you want to look at the success of something, you should first determine what the end result is. Are you looking at quality of life, mucosal healing, change in blood tests, symptom improvement, biopsies? There has not been a uniform agreement of what endpoints we should be using in these trials, and I think that’s part of the reason why the funding and the seriousness of the disease is not on top of the list for federal funding.
Another reason for the lack of federal funding is that it is difficult to get celiac patients to enroll in trials because there is a fear of consuming gluten for the study and overall people are content on the diet and even if not content, they are suspicious of drugs as an alternate to the diet. When pharma and the FDA look at these issues, I think it discourages them from funding trials where enrollment will be difficult.
Healio: What was a significant question or topic that stood out to you during the event?
Verma: There was some disagreement between those in pediatrics and those who treat adults regarding family screening. In the sense of screening people who don’t have symptoms, and screening them early. In pediatrics, we strongly feel that we need to do family screening and screen early, and treat even if someone doesn’t have the classic symptoms, because in children, we’re looking at things like growth and the long-term risks of autoimmune disease. Symptoms or no symptoms, people need to be screened if they’re genetically susceptible and they’re part of a family with celiac disease.
Conversely, providers who treat adults with celiac disease differed, in that they questioned whether you really need to screen a grandparent who is 80 years old and doesn’t have any major symptoms just because his or her grandchild was diagnosed with celiac disease. So, they feel that maybe that shouldn’t happen.
Otherwise I think we all agreed on most issues, and really hope that more people will join us in doing different clinical trials for celiac disease. – by Adam Leitenberger
Disclosures: Verma reports no relevant financial disclosures.
For more information :
Watch video of the full Bold Beyond Research Symposium here. https://www.beyondceliac.org/boldbeyondsymposium/