Are Enzymes Safe for the Celiac Disease Community? Researchers Set the Record Straight
August 11, 2015
Experts Joseph A. Murray, MD, Mayo Clinic and Daniel A. Leffler, MD, MS, BIDMC weigh in on the misconceptions that enzymes can treat or aid in managing celiac disease.
While the gluten-free diet may still be the only treatment for celiac disease, there are many challenges to strictly maintaining a 100% gluten-free lifestyle.
It’s common for people with celiac disease to continue experiencing symptoms while on a gluten-free diet and sometimes these symptoms can come and go over time. Also, the gluten-free diet requires significant ongoing education and motivation because it’s a self-administered treatment. As a result, patients carry the burden of this self-managed disease. What’s more, it can take more than two years for some adults with celiac disease to heal their small intestine and it is also possible for some people to still have intestinal damage, despite having a normal celiac disease blood test.
Fortunately, researchers are actively studying new ways to help patients better manage and treat this genetic immune-mediated disease. Every day, scientists and physicians are working together to identify and develop new treatment options that meet the needs of celiac disease patients. Most of these new treatment options are still being studied, with some in clinical trials, and none have been submitted for approval to any regulatory agency anywhere in the world for the treatment of celiac disease.
When looking to develop new treatments, researchers pay close attention to the specific points in a disease process where they can change the course of action. For celiac disease, this can include pills or enzymes that can help to breakdown or bind to gluten so that patients can safely eat foods or drink beverages that contain gluten.
The National Foundation for Celiac Awareness (NFCA) applauds the scientific community for working to advance the celiac disease field and is excited that the media takes such a strong interest in the topic. However, by drawing attention to very early-stage developments, the media can sometimes provide misleading and overly optimistic expectations for patients, leading to disappointment. One recent example has been coverage of pills and enzymes that may one day help to treat celiac disease.
Because having a safe and effective treatment is a top priority for our community, it’s important to NFCA that patients know that at this time, there are no medical treatments on the market that have been approved by the FDA as safe for the management of celiac disease . A strict lifelong gluten-free diet remains the only treatment for celiac disease today.
“Until there is a safe and effective pharmaceutical treatment for celiac disease, it is very important that patients adhere to the only treatment known to be currently effective (the gluten-free diet) until and if an effective alternative treatment is developed,” stated Joseph A. Murray, MD, Mayo Clinic.
NFCA strongly encourages patients and their families and caregivers to carefully read the fine print of any pills or enzymes currently on the market and available in stores that promote easier digestion of gluten-containing foods. For example, some products do include a disclaimer noting that it is not intended to treat or prevent celiac disease, so it’s very important that patients and caregivers read all information on the label or website.
Because celiac disease is a genetic immune-mediated disease that must be taken seriously and managed carefully, NFCA turned to its scientific and medical advisors, Dr. Joseph Murray and Dr. Daniel Leffler, for an update on the facts.
Q. First and foremost, what is the most important message people with celiac disease should hear about pharmaceutical or enzyme therapies?
A. “It is vitally important that patients with celiac disease do not use any of these preparations that are being touted for reducing gluten. They have no proven benefit for patients with celiac disease and, indeed, it would be illegal for them to claim so in the U.S. However, sometimes disclaimers are hidden or not at all obvious to people who could be reading this,” Joseph A. Murray, MD, Mayo Clinic
Q. Alvine Pharmaceuticals, Inc. has developed the medication
for people with celiac disease who continue to experience symptoms despite a strict gluten-free diet. Can you give an example of an everyday scenario where this medication might improve their lives?
If approved, would it be taken everyday or only in certain situations?
What is the status of these clinical trials and, if approved, when do you think ALV-003 would be available on the market?
A. “I think both are reasonable possibilities. There are some people who are either highly sensitive to gluten or have diets that are difficult to fully control, for instance college students on a meal plan or frequent business travelers. For these individuals, this type of a medication would be most effective if taken routinely. On the other hand, there are people who can achieve adequate dietary control most of the time, but will need some extra protection on weekends when they eat out or on vacation, for example,” Daniel A. Leffler, MD, MS, BIDMC.
“As of right now, all we know is that the ALV-003 phase IIb trial is closed to enrollment and until there is data released, it will be hard to speculate when it would be available on the market,” Daniel A. Leffler, MD, MS, BIDMC
a group from University of Alberta in Canada
reported creating a supplement from the yolks of chicken eggs that would bind to wheat proteins in the stomach. Would this be suitable for people with celiac disease since they also need to avoid barley and rye, two other gluten-containing grains? How would this work and would patients with celiac disease avoid intestinal damage as well as have symptom relief? Has this been approved in Canada or the U.S. yet?
A. “There is no published data on this. This has not been approved in neither Canada nor the U.S. as a treatment for celiac disease or for any other disease. This is a great example of media hyping something at the very early stage of development,” Joseph A. Murray
Q. BioLineRx Ltd. has developed a potential treatment,
, that also binds to gliadins, or the parts of the gluten protein that cause damage to people with celiac disease. BioLineRx’s Drug Development Director, Yotam Nisemblat, presented data on their phase I/II safety study results at the International Celiac Disease Symposium (ICDS) this past June. What does it mean to complete a phase I/II safety study and what do the results from this study mean for patients?
A. “A phase I/II safety study basically tells the sponsors (for example, a company who is developing a new drug) if there are safety issues relating to the use of their medication or device in healthy individuals, as well as patients with the disease that is being targeted,” Joseph A. Murray, MD, Mayo Clinic
Q. Earlier this summer a small neutraceutical company introduced
Tolerase G (AN-PEP)
, a product some patients might think of as the “gluten version” of a lactaid pill. Has this product been approved by the FDA? What type of research was involved in developing AN-PEP?
A. “This has not been approved by the FDA for treatment of celiac disease or management of any disease or diagnosis. The research that has been developed is not sufficient in order to make any claim as a drug in the U.S. or elsewhere. The public should be aware that dietary supplements are very loosely regulated by the federal government and there is no requirement that safety or efficacy data be presented to the FDA. In fact, it is really only regulated in response to complaints or labeling issues, for example where a manufacturer has placed a label that could imply that it could be used to manage a disease,” Joseph A. Murray, MD, Mayo Clinic
Q. Would a patient with non-celiac gluten sensitivity (‘gluten sensitivity’ or NCGS) benefit from taking any of these currently available over the counter supplements?
A. “The use of these enzyme preparations for patients with celiac disease has never been proven and, indeed, there is no data to support the use of those enzymes for non-celiac gluten sensitivity either. Additionally, there is no evidence that any agent in development for celiac disease, or for example Tolerase G, has benefit for patients with non-celiac gluten sensitivity, as no studies have been performed,” Joseph A. Murray, MD, Mayo Clinic
Q. What is the current overall landscape for new celiac disease treatments?
“The targets for treatment of celiac disease are many, largely because we understand a lot about the process that results in celiac disease damage in patients with the condition. This provides many different potential targets for intervening to prevent or reverse the damage, and these can be anything from altering the gluten in foods, trying to detoxify or breakdown the gluten in the gut, bind the gluten in the gut so it is not absorbed, or interrupting the immediate or delayed effects of gluten on the mucosa that the lining cells of the intestine, such as permeability, or interrupting the immune reactions that occur. Other approaches may include the possibility of generating tolerance in patients with celiac disease to gluten. All of these are being approached in various development stages. People with celiac disease in the general population need to be patient as these can often take many years to put together all of the evidence to show they are both safe and effective,” Joseph A. Murray, MD, Mayo Clinic