Promising New Drug Did Not Prove in Trial

A new drug to break down gluten and render it harmless did not prove effective in the latest Phase 2 trial. To win approval, the FDA requires that a drug be both safe and effective. This process is divided into two Phases. In Phase 1, safety is assessed.The drug is given to a small group of test subjects to determine safety as well as dosage levels and side effects.  If successful, the drug moves on to Phase 2. In this Phase, there is a larger group of subjects receiving the drug and there is usually a group receiving a dummy pill (placebo). This Phase determines the effectiveness of the medication. A drug named Latiglutenase and produced by Immunogenx looked very promising after Phase 1 studies.  It is a combination of enzymes to break apart gluten and render it harmless. The body produces enzymes all the time. For example, these chemical helpers break down food so that the body can reassemble the nutrients in it into things useful to the body.  In the Phase 2 trial, the company recruited 500 subjects with Celiac who had been on a gluten-free diet for one year. Some were given varying doses of Latiglutenase and some were given placebos.  All subjects were asked to complete a standardized questionnaire dealing with symptoms. All subjects, whether receiving the drug or a placebo, reported improvement in symptoms. This perplexing result may be due to the "trial effect" All the subjects knew they were part of a study. Since they were taking the pill three times a day, there was a constant reminder to adhere to the gluten-free diet. They were then more careful to avoid exposure and their symptoms improved.  It was noted though that subjects with the higher Celiac disease antibodies reported the greated improvement. So there may be some drug effect. The company feels motivated to design further studies.