The diagnosis of celiac disease forces patients to make a number of sacrifices. Anyone who could develop a drug that would free the sick from the rigors of the diet and unpleasant symptoms for breaking it, would earn a fortune. No wonder that more pharmaceutical companies are joining the research.
Like many people with gluten intolerance, Kristen Sweet avoids wheat proteins, which can harm her health. However, when she dines at a restaurant or at friends’ houses, she can never be sure whether what she eats does not contain a substance that is dangerous to her. “Whenever you go out and put your health in someone else’s hands, you are taking a risk,” notes the 29-year-old with celiac disease. “Once I get sick, I collapse in pain for a couple of days and there is nothing to be done about it. There are no medications to help me.
Pharmaceutical companies are doing their best to be the first to develop effective treatments for celiac disease, which scientists now say is much more common than previously thought. The first drugs would not hit the market earlier than 2018, but some of them have turned out promising in small clinical trials and could soon enter the final testing phase. With this in mind, the US Food and Drug Administration (FDA) organized a debate some time ago on an unprecedented question: how to evaluate the effectiveness of drugs for celiac disease in clinical trials?
The fact is that most of the specifics currently being developed will not allow patients to abandon a gluten-free diet. Their action will be limited to reducing the symptoms of the disease in people who have ingested some wheat protein. For now, small companies are working on the development of drugs, although larger ones would like to join the research. (…)
Celiac disease is now considered a disease of the immune system in which the body attacks its own tissues, especially the lining of the small intestine, through which nutrients are absorbed. This autoimmunity occurs in people who are intolerant to gluten, the protein found in wheat, barley and life, which cannot be properly digested by patients. “We’re talking about the first autoimmune disease in which we’ve identified antigens,” notes Francisco Leon, co-founder of Celimmune, a company working on a cure for celiac disease. Leon says celiac disease will best verify the value of autoimmune disease testing companies, as their effectiveness in this case is easy to test by giving people gluten.
Some people suspect that medications for other immune system diseases could also work for celiac disease. Celimmune has approved amgen for degenerative inflammation, a drug that is now being tested in patients with extremely refractory gluten intolerance.
Developing a gluten-free diet brought celiac disease a lot of relief, but it didn’t do the trick. For example, one study showed that in two-thirds of adults, the mucosa of the small intestine was damaged after two years of using a safe diet. This could indicate that the treatment of celiac disease takes a long time. Or that the subjects were still exposed to small doses of gluten penetrating their bodies. Gluten is found in lipsticks, in some prescription medications and in many other surprising products. But strictly following a diet can be difficult for some people, especially teenagers who would like to go out for a pizza with their friends from time to time. “It comes with a high level of anxiety and concern about social isolation,” admits Daniel A. Leffler, research director at the Beth Israel Deaconess Medical Center’s Celiac Disease Center in Boston and a consultant on drugs under development.
Pharmaceutical companies have different approaches to it. And so the agent tested by Alvine, called ALV003, consists of two enzymes designed to neutralize gluten before it enters the small intestine and causes an unpleasant reaction. The medicine is in the form of a powder dissolved in water and would be taken before a meal. For research purposes, volunteers agreed to eat breadcrumbs daily for six weeks. The intestines of people taking ALV003 were not damaged, unlike the participants in the study taking a placebo. However, no statistically significant difference was noticed in terms of the severity of the disease symptoms.
Later this fall, Alvine, a privately held company based in San Carlos, California, expects to get results from a wider trial involving 500 patients – the most comprehensive trial ever tested.
Christina Buettner, a participant in study ALV003, said she did not notice a difference in how she felt, though she did not know whether she was being given the drug or a placebo. This nurse from the Boston area said she was a bit bothered by drinking 230 ml of a fruity drink, but she would like to do so if it could remove the long-term effects of her illness in the future. She declares that she would be happy to take a drug like ALV003 before a meal that she couldn’t say with certainty is gluten-free.
Already, there are supplements on the market whose manufacturers claim to crack down on gluten. These measures, however, have not been approved by the FDA, and specialists in celiac disease do not feel very positive about them.
At least one company, ImmusanT, awakens patients’ hopes to free themselves from a gluten-free diet and announces that people with celiac disease in the future will be able to eat whatever they want. Injecting patients for many weeks with small doses of gluten would lead to an immune response that would eventually lead to better tolerance of the substance – a procedure borrowed from allergy treatment. Some experts estimate that the chances of success with this method are remote.
According to the recent debate on the FDA forum, assessing the effectiveness of drugs for celiac disease is all the more difficult as the disease itself affects people to a varying degree. There is also no clear link between symptoms and damage to the intestinal mucosa. Ideally, the new drug should affect the symptom sphere and repair the damage done, and at the same time prevent the long-term effects of the disease, such as osteoporosis (…).
So far, gluten-free products have gained popularity even among people without diagnosed celiac disease or gluten sensitivity. Some patients feel angry and claim that their affliction is trivialized as a result.
Alice Bast, founder and head of the National Foundation for Celiac Awareness, an organization working for patients with celiac disease, believes that the introduction of drugs will help to take patients more seriously, both in society and in healthcare settings where more doctors will direct people to tests. Bast, diagnosed with celiac disease 21 years ago, first spent eight years investigating the cause of her numerous health problems, migraines, hair loss, brittle teeth, debilitating diarrhea and three miscarriages. Seeking help, the woman visited 22 specialists. – Work on drugs will lead to increasing awareness of this disease among doctors – he says about celiac disease. – From now on, we will control the disease, not just control what we eat.