By Amy Ratner, Beyond Celiac Medical and Science News Analyst
For many in the celiac disease community, Robert J. Beall, Ph.D. is a new name and face as he joins the Beyond Celiac Board of Directors.
But he is familiar to Abby Schwartz, the mother of a 20-year-old daughter who has both celiac disease and cystic fibrosis.
Schwartz knows about all the work Dr. Beall did in his 35 years with the Cystic Fibrosis Foundation, where he was president and CEO for 21 years before retiring in 2015. And she is excited about the role he will play in moving celiac disease forward.
“He was a dedicated and passionate leader who was visionary in how he approached research and treatment,” Schwartz says. “He took cystic fibrosis to groundbreaking places.”
Dr. Beall’s recent addition to the board dovetails with the Beyond Celiac commitment to accelerating research to find new treatment options and an eventual cure for celiac disease. Although cystic fibrosis and celiac disease are different in many ways, they share a need for new treatments, and the Cystic Fibrosis Foundation has paved a way to propel research.
“Beyond Celiac has always looked to best practices with other diseases to be able to further our work,” noted Alice Bast, Beyond Celiac CEO. “Dr. Beall will bring his brand of innovation and leadership to help us move the bar and hopefully see similar success in the field of celiac disease.”
As a self-described risk taker, Dr. Beall looked to the business model of venture capital for innovative ways to speed cystic fibrosis research. He pioneered venture philanthropy by investing a half billion philanthropic dollars in early stage research conducted by pharmaceutical companies. Venture philanthropy has been a game changer in drug research and development and has been adopted by many patient advocacy organizations as a result of Dr. Beall’s groundbreaking work.
Dr. Beall’s long list of accomplishments include the approval of two drugs that address the underlying cause of cystic fibrosis, a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. He’s also credited with helping to build a pipeline of nearly 30 drugs in development to treat all aspects of the disease.
During his tenure, the life expectancy of those with cystic fibrosis more than doubled. For the first time, more than 50 percent of those with cystic fibrosis are older than 18 years of age. With affected patients living beyond childhood years, the condition is no longer classified as a pediatric disease.
Under Dr. Beall’s leadership, the foundation became one of the most respected voluntary health organizations in the country and is recognized for its innovative approaches to bringing new therapies to patients with the disease. Dr. Beall inspired the foundation’s Research Development Program, a dynamic, interdisciplinary research network that laid the groundwork for the discovery of the cystic fibrosis gene in 1989. In 1997, he established the foundation’s Therapeutics Development Program, a unique coalition that has advanced cystic fibrosis drug development through a centralized network of clinical trials and alliances with biotech companies.
Building patient relationships
But he also measures his success in his relationship with patients. He’s most proud of the fact that they are now living long enough to go to college, embark on careers and have families of their own — none of which they could have imagined even a short time ago. When he retired, the parent of one patient arranged for Wayne Gretsky, the hockey star whom Dr. Beall often quotes, to give him a prized, signed jersey. The mother of another has sent him cookies for decades.
At Beyond Celiac, we know that the quest for treatments in addition to the gluten-free diet is a new frontier for the celiac disease patient community. So, we sat down with Dr. Beall to talk about his background and enthusiasm for using his experience to help those with celiac disease push research forward by becoming actively engaged. Here’s what he had to say.
Your background is in science. Did you always want to be a scientist?
When I grew up I always thought I was going to be a dentist. Then I went to dental school for two days, and I decided that was not for me. Much to my father’s chagrin, I went back and finished my bachelor’s degree at Albright College. Then I got my doctoral degree from the State University of New York in the biological sciences. After several years on the faculty of Case Western Reserve medical school, I decided I did not like bench research. I wanted to become a health scientist administrator and so I went to the National Institutes of Health (NIH) where I got involved in diabetes research.
What got you interested in cystic fibrosis?
My director at NIH asked me if I would be interested in working on cystic fibrosis. I told him I could not even spell “cystic fibrosis.” While he knew I was involved in the implementation of the diabetes program, he was quite persuasive. He suggested I go to a cystic fibrosis meeting in San Diego before making my decision. San Diego in February sounded good, so I said yes.
I was so moved by these parents and patients with cystic fibrosis, that I came back and told the director I wanted to run the institute’s cystic fibrosis program. I got involved and worked on the disease at NIH for about five years. Then the foundation recruited me to become their executive vice president of medical affairs.
What prompted you to get involved with celiac disease and the Beyond Celiac board?
I got involved with Beyond Celiac because my best friends have a granddaughter who has celiac disease. My decision was based on that friendship. At the organization’s request, I presented some thoughts to the Board of Directors, and they seemed to appreciate what I said. I’m excited to see that the organization will be placing a bigger emphasis on research in its work moving forward. I hope I can contribute in some small way from my experiences. If I can help my friends’ granddaughter and others with the disease, I am going to do it. I have met a few people with celiac disease, and I certainly understand the consequences of the having this autoimmune condition.
What factors led to the Cystic Fibrosis Foundation’s successes under your leadership?
One of the things I thought very early in my work at the Cystic Fibrosis Foundation was that if we really wanted to have an impact on this disease, we had to understand the basic defect. We were treating the symptoms, and that was important, but it had limitations.
When I got to the foundation in 1980, the first thing we did was set up our own research center network, eventually with about 12 sites. In a very short time these truly became centers of excellence in cystic fibrosis research. Groups from different disciplines were working together. In 1989 with major support from the foundation, we discovered the cystic fibrosis gene.
By 1998 we had a good idea what was going wrong, but we were not getting the pharmaceutical industry involved with us in research. We started our therapeutics development program to encourage industry to enter the field of cystic fibrosis. We embraced industry as part of our ecosystem and started a clinical trials network to help industry conduct clinical trials. In addition, we provided financial support. Since that time, we have committed nearly $500 million to industry. One of our early investments led to the development of two drugs, Kalydeco and Orkambi, which can help 60 percent of cystic fibrosis patients. These drugs treat the basic defect and are proving to be life changing for many patients.
What needs to be done to similarly propel celiac disease research?
I would like to see Beyond Celiac expand its work with industry. Biopharmaceutical companies can be great partners in the celiac disease effort. We certainly have a better understanding of the underlying mechanism of celiac disease than we did when I started work with cystic fibrosis. If industry needs money, access to patients or access to knowledge, I’d like Beyond Celiac be the place to go, like the Cystic Fibrosis Foundation is the place to go for cystic fibrosis.
What would you say to those in the celiac disease community who hesitate to support drug development because the gluten-free diet was seen for a long time as the only treatment needed?
I got involved with Beyond Celiac because of my friends’ granddaughter. She is 10 years old. I have seen the consequences of celiac disease and the impact on her life. Her needs go beyond following the gluten-free diet. The continued burdens of pancreatitis and migraine headaches can be tough for a child. I have also met some adults with celiac disease who have osteoporosis or have had stillborn children. There is a spectrum of how celiac disease impacts people.
We have to look at patients who have a more profound impact. I think many physicians would say those are the patients we need to learn more about to better understand the disease and treat it. We need to measure the profound impacts and learn more about the consequences of the disease from all angles, not just ongoing symptoms.
For too many, a gluten-free diet is not enough.
Based on what you know so far about what is going on in celiac disease research, what do you think is the opportunity?
I’ve looked at some research. I am glad to see there are clinical trials already underway. This is a great starting point. But are there other opportunities for clinical intervention? Who can do the studies and who will fund them?
How would you approach new research about celiac disease treatments? Should the first step be a treatment to supplement the gluten-free diet and then move onto a cure, or do they work simultaneously?
There is a continuum. First, let’s get an effective medical treatment so that people are not suffering because they have celiac disease. Then let’s go from there to a cure, if it can possibly be done. I think anyone would accept a daily pill or a monthly injection if they could live a normal life, but even that is a treatment not a cure.
Do you have a primary goal in working with Beyond Celiac?
I hope that I can share my experiences with some of the success we had at the Cystic Fibrosis Foundation. Hopefully I can help steer the organization in a way that it can staff appropriately for identifying scientific opportunities and be able to make industry aware of the opportunities that Beyond Celiac can provide.
Then I hope we can focus on research efforts specifically driving for a cure. At the Cystic Fibrosis Foundation, we pulled out a segment of our fundraising efforts really focused on getting major gifts to push for the cure.
Are there other steps that you think would translate well for Beyond Celiac?
At the foundation, we shared the drug development pipeline to visually show the community where the research is and what clinical trials are underway. Patients can go to the website, put in their zip code and see the clinical trials that are being conducted in their area. They can see where a drug is in development, what phase it’s in and whether it’s been approved. But most of all they can see that there are a lot of drugs being developed that could impact their disease.
What do you see as the biggest challenge the gluten-free community faces in terms of research?
Money is always a challenge. Researchers can be at a point where they have a good idea, but their own academic institution or company has not invested in it yet. This period between having the idea for a drug and moving it forward in development in science is called “the valley of death” because so many drugs never make it out of this stage. Beyond Celiac could have a role like the one taken by the Cystic Fibrosis Foundation, to provide the financial support to get potential drugs through this valley.
At the Cystic Fibrosis Foundation, we committed $3 million to one company and got them through the valley of death. The company was able to independently raise $120 million just because of our good housekeeping seal of approval. Eventually the project was sold to another company for $600 million.
Ultimately, we have to look at ways that we can accelerate the translation from the test tube to the bedside.
The cystic fibrosis patient community went out and raised millions of dollars. How did they raise that much money and what motivated them?
The foundation has been working to raise money since 1955. The first grant was for $10,000 in 1958. Now the medical budget is $175 million to support care centers, research, training and more. The community is very engaged because they are informed. There has been a lot of progress. Two drugs have been approved and patients are living longer. The life expectancy when the foundation was founded was 6 years of age and now the median survival age is close to 40.
The Cystic Fibrosis Foundation was a pioneer in venture philanthropy. How do you define it?
Venture philanthropy is taking philanthropic dollars and providing support to private industry. It’s similar to venture capital, where investment is made in start-up companies that have potential for long-term growth. In venture philanthropy, donations are invested in companies to get them involved in research, be it for cystic fibrosis or celiac disease. It’s been adopted by many voluntary health organizations. Faster Cures, a Washington, D.C.-based center that works to speed up and improve the medical research system, was founded around venture philanthropy. Beyond Celiac has participated in Faster Cures for four years.
As companies are working to develop drugs to treat celiac disease, what do patients need to understand?
They need to know it is a time-consuming process. There are going to be disappointments, and drugs will never be approved unless we can do clinical trials. Participation in clinical trials is imperative. No drug in the United States is approved unless there are clinical trials.
For cystic fibrosis, it took 32 years, from 1980 to 2012, to launch treatments directed at treating the basic defect. Meanwhile, there was a lot of progress in treating the symptoms of the disease.
How can the urgency of the need for new treatments for celiac disease be best communicated?
The gluten-free diet is not a cure, and it’s not a control. That message needs to be promoted. We now know it’s not a complete control. We know that there are underlying mechanisms causing something to go on in the body, and it is destructive.
Are those who want to see change the key to accomplishing change?
Absolutely. It’s the power of passion. If people are passionately committed to seeing something change, they will make it happen. There is no room for sitting on the sidelines by scientists, researchers, patients, their families and other volunteers. It takes a village.
Fundraising is very important, but an engaged patient community can also create the momentum for research advances. When patients are well-informed by advocacy groups, including through social media, they become more powerful. They can also effect change by participating in clinical trials and helping to create a body of patient data for research use.
You accomplished a lot for cystic fibrosis, including billions of dollars for research and an increase in the chances that a child with the disease will get to grow up. Do you feel pressure from high expectations about what you might be able to do for celiac disease?
Anytime you can impact the quality of life for people with a disease and the quantity of life, that is a worthy goal. I want my friends’ granddaughter, Brooke, to be able to grow up in a way that her celiac disease is not an obstacle to what she wants to be.
Is there an incident that set the path for your approach in your work for cystic fibrosis and the way you will approach your role on the Beyond Celiac board?
It goes back to my introduction to patients in 1976 when I was working in diabetes at NIH, and I met the desperate families with children who had cystic fibrosis. There was no research. They were looking for answers. The Cystic Fibrosis Foundation was so small at that time that the families at the meetings were running their own projector and bringing their own food. My introduction to them had a profound impact on me.
I came back from that meeting and said to my wife, "I can be a small fish in the big diabetes area or maybe I could really have an opportunity with this." I am still in touch with some of those people. One woman still sends me chocolate chip cookies. Her son, who at that time had a life expectancy of 18 years, is now in his 40s.
What is the most important thing celiac disease patients should know about you as you join the board?
I’m impatient and I am willing to take risks on behalf of the patients. My philosophy can be summed up in a quote from Wayne Gretsky. “You miss 100 percent of the shots you don’t take.”
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