Marie Robert, M.D., the new Chief Scientific Officer for Beyond Celiac, and Amy Ratner, Medical and Science News Analyst, recently sat down to talk about Robert’s role with the organization and how she hopes to help propel research into celiac disease. She also touches on several new Beyond Celiac initiatives, including the Young Investigator Award and the upcoming Beyond Celiac 2018 Research Symposium. Finally, Robert shares a little about her work as a pathologist at Yale University and her perhaps more unexpected interests. Here’s how their conversation went*:
Tell us a little about yourself and your interest in celiac disease.
I am a surgical pathologist and went to medical school at University of Michigan. I am from Michigan, Kalamazoo Michigan. I did my residency in Los Angeles at UCLA. Being the daughter of a scientist and spending time in my father's lab as a little girl, I gravitated toward the more scientific aspects of medicine. I ended up in pathology as a field of study and residency.
My very first project at UCLA was with a renowned clinician who studied refractory celiac disease, patients who did not respond to the gluten-free diet. From there I developed a lifelong interest in celiac disease. So, I am a surgical pathologist, a gastrointestinal liver, pancreas specialist, and I’ve spent my entire career at Yale University School of Medicine where I am today.
Tell us what a chief scientific officer does and why it's important for Beyond Celiac to have one.
The role of a chief scientific officer (CSO) in an organization is to bring the emphasis and activity of that organization, to bring scientific knowledge, to the disease and to look at the world of science and put together what the opportunities and where the needs in are research and to put the organization’s resources to work to make changes. In this case, to find treatments and a cure for celiac disease
How will Beyond Celiac having a CSO most likely impact celiac disease patients?
Beyond Celiac is going to set the research agenda in the United States, essentially, for celiac disease. The people who will serve on the scientific advisory board I am putting together are going to define the needs for research. They will determine what direction we need to move in and get the National Institutes of Health (NIH) to fund this movement so we finally get real treatments and eventually, not too far in the distance, a cure for celiac disease.
We are aware of how poorly celiac disease stacks up in NIH funding, and there is a little of the feeling that there is nothing we can do. But it seems you are saying there are things we can do to change that.
Absolutely. What attracted me to Beyond Celiac was two things; the passion for making progress in this disease and the know-how of the team. I was intrigued by the cleverness of Alice Bast, the chief executive officer, to pivot to research after making great movements in advocacy and education -- how to cook gluten-free and eat as safely as possible in restaurants -- to pivot to the next thing that is needed, which is research.
You have been CSO at Beyond Celiac for a few months, working hard behind the scenes. Have you had an opportunity to set your top priorities?
As a pathologist, I am used to working behind the scenes. I am no stranger to that. And I have set my goals. One of the first is to create a scientific advisory council that will consist of the absolute leaders in the country in celiac disease, be they pediatric or adult celiac disease clinician scientists. It wil include people from the pharmaceutical industry who are working so hard in clinical trials and moving the needle in this field. I am also looking at people who have a venture capitalist background who can help us when that great idea from a scientist comes along. When something that could really change the field dramatically comes along, we want to take that shot on goal. We need to attract that funding that will support research since the NIH hasn't quite picked it up yet.
It sounds like you want to expand the focus to figure out new ways to get new ideas from the scientists’ labs into patients’ lives.
Very soon the scientific advisory council will meet, and we will think outside the box, inspirationally. We’ll create a research agenda and put out requests for applications and proposals from the science community, the biotech industry, the innovators, the entrepreneurs who have an idea. We will review the applications and start funding them. I am excited. I think this is a moment in time in celiac disease. There is no other organization advocacy group, and certainly not the NIH, that is doing what we are doing.
What is the biggest challenge in achieving the goals you’ve set?
It's a completely surmountable challenge. As we begin this work, we need to synthesize what is known about celiac disease, putting together what is known about the immune system, how it relates to other autoimmune disorders, how it relates to viral infections -- in childhood for example. What about the microbiome? How does that fit in? Then we can start to formulate the body of work that exists already in celiac disease research and look at it from a faraway point-of-view and identify where to go next. Not to get lost and keep repeating something that's already been done, but to see how that work comes together to give us the next clue.
You’ll be looking at the big picture to explain how the pieces come together and how research can connect them. Tell us about funding of research through the new Beyond Celiac Young Investigator Award.
Beyond Celiac has already begun to fund research. We have formulated an award, let’s say it’s an early career award since age is not so much the important thing here even though it called the young investigator award. We want to encourage scientists who have a budding interest in celiac disease, the smartest people, to pursue that career by funding the early years of their work. It turns out that in the field of research, celiac disease is aging out, as hard as that is to believe, because it so difficult to get funding from NIH.
We have an award in the works in partnership with the Society for the Study of Celiac Disease, and we will continue to do this on our own with our own grant adjudicating body, the scientific advisory council.
You are saying that without the money needed to do research, you start to lose people who are interested in pursuing new treatments. Like a plant that does not get any water?
All research is funded one way or another. If you are a physician, your institution does not pay you to do research. You have to earn that money from a grant organization, either from the NIH or a foundation. There is no payment from Medicare or the insurance companies.
We know the pathologist plays a role in diagnosis, but he or she may be the doctor who flits in and out of your life fastest, unseen. I have heard you talk about changes pathologists have seen in biopsies related to celiac disease. Tell us about those changes.
The pathologist is the most important doctor your never see. When I teach residents at Yale, I tell them that back in the day when I was a trainee we were taught that if the little fingers in the small bowel that normally reach up are not flat then the patient does not have celiac disease. Anything that was not flat was considered not to have any chance of being celiac disease. We could not have been more wrong, though this took some time to prove.
We now know the even a biopsy with very tall fingers can still be celiac disease because there are some subtle changes that occur. The reason we figured this out was twofold. One, scientists, including Marsh in England, found there are a whole spectrum of changes in the villi. Second, the anti-tissue transglutaminase blood test (TTG) was developed. Now, it’s the test most people diagnosed with celiac disease have. When I started the test had not yet been discovered. Once it was, we were able to see that if the villi are pretty tall, yet the patient has a positive TTG test, low and behold, this patient could still have celiac disease. The was a major change for pathologists. We know this very well now, but until about 15 years ago, it was not understood at all.
When we think about managing and treating celiac, currently the gluten-free diet is still all that we have. Some patients feel we don't need any additional treatment. What would you say to them?
I would say two things. First, imagine a life where you don't have to think about gluten-free diet at all. Second, imagine that there was no treatment for heart disease and people died much earlier and restricted themselves to a very strict diet and had severely limited function. Before the invention of insulin, think about diabetics. We accept things as they are until there can be something better. There can be something, and there will be something, much better. We will look back on this era and remember it as time when there was nothing to treat celiac disease. A time when you had this cloud following you around everywhere, every time you put something in your mouth.
It seems at first, there will be a treatment that will be taken along with continuing the gluten-free diet.
Yes, that is what the clinical trials are working on. As the CSO, I am trying to make inroads and as I talk to other scientists in the United States one of the big stumbling blocks to researchers is getting celiac disease patients who think they are doing OK to sign up for clinical trials and participate in the effort to find a cure. I can’t really emphasize that need too much.
One of things that makes people reluctant to participate in clinical trials is that they don't want to consume gluten again and they recognize that trials can involve consuming some gluten.
I completely empathize with that and think that would be a concern of mine (if I had celiac disease). I would only ask that people consider that the amount of gluten exposure is for short duration over time and will certainly not lead to any long term bad effects from the disease.
It's not saying let's get gluten back in your life and see what happens. It’s a much shorter time frame. I think it’s a decision everyone has to make for themselves. But consider if you can tolerate some discomfort and some symptoms, you are playing a part in something important. You are part of the big picture of curing celiac disease and making it so that your child, who would be at increased genetic risk, might have a better life.
There are others way to participate in research, too. You can support the Young Investigator award through donations or sign up for Go Beyond Celiac. And Beyond Celiac is also getting the research gears moving through the upcoming 2018 Research Symposium.
Yes, the symposium is very exciting. It's a live meeting and a webinar which will be a discussion and a question and answer segment on Wednesday, October 10, from 6-7:30 p.m. EST. We have put together a panel of three outstanding speakers.
Ciaran Kelly, M.D. from Beth Israel Deaconess Medical Center at Harvard, one of world renowned celiac disease experts, who will be sharing the entire spectrum of what is happening with clinical trials.
Maureen Leonard, M.D., a pediatric celiac disease expert from Mass General Hospital, also at Harvard, will speak about the microbiome. This such a hot area. The microbiome is everything now. People are taking probiotics for all sorts of reasons. We think the interface between the gut and the external environment and the normal flora has a role in many diseases, diseases that have nothing to do with the gastro-intestinal tract. There is a lot of work on how the microbiome is important in celiac disease, and Maureen is going to share her expertise on that.
Finally, we have Stephen Miller, Ph.D., an immunologist at Northwestern University who works in multiple sclerosis and celiac disease and has discovered nanoparticle theory. He is going to make the link between celiac disease and other autoimmune diseases. This is also a huge, important moment in time for autoimmune disease in general. Celiac disease is the poster child of autoimmune disease because it is the only in which the trigger and response is known. We think the information we continue to learn about celiac disease will unlock the door to other autoimmune conditions.
To give people a three-dimensional picture of who you are, tell us a little about your life outside the pathology lab and Beyond Celiac.
I already said I am from a small town in Michigan. My parents are French Canadian. My husband is also a pathologist, also at Yale. We met in Michigan in medical school. We have two daughters who are 21 and 23. We like to travel and are very outdoorsy. We like to ski and do a lot of bike riding.
But I have a secret side. We all have another me, and I call this the other me. I was a musical theater actress in a former life before medical school. It was a big part of my life. In college it was a choice, this way or that way, and I made a happily choice to pursue medicine. But at one point I did nothing but singing and dancing and acting. Then, ten years ago I dusted myself off and put together a cabaret act that I preform for funding raisers in the New Haven and Yale area. But now Beyond Celiac is my new passion.