Recent scientific advances in treatment have led to the first FDA-approved therapy for eosinophilic esophagitis. This is an exciting development for those living with EoE, an inflammatory disease that affects the esophagus and gastrointestinal tract.
EoE is associated with symptoms such as narrowing of the esophagus (strictures), food getting stuck in the throat (food impaction), and abdominal or chest pain. Research studies show that EoE also largely affects the mental health of those living with the condition. This finding highlights the need for more research and development of treatments that improve quality of life.
Keeping up to date on the newest studies can help you stay informed on new treatments and findings in the field you can discuss with your doctor. As research continues to look into new therapies, there may be opportunities to try these treatments for yourself.
EoE is an immune system disorder in which specialized white blood cells, known as eosinophils, collect in the esophagus tissue. This causes inflammation and swelling, making it difficult to swallow (known as dysphagia). Researchers are not quite sure what causes EoE, but they believe it may be related to food allergies or a response to an allergen in the environment.
Traditionally, EoE treatment has focused on reducing the symptoms of the condition rather than targeting its cause. Proton pump inhibitors (PPIs) are primarily used to treat heartburn caused by gastroesophageal reflux disease (GERD). PPIs are often the first therapy tried to help people with EoE.
Topical corticosteroids can also be used if PPIs do not work. Two treatment options are budesonide or fluticasone, which come in a liquid form and are swallowed to help treat inflammation in the esophagus. Dietary therapy is also used to help treat EoE by addressing the potential food allergy component. One example is following the elimination diet, in which you stop eating foods that commonly cause allergies to see if your symptoms improve. A procedure called esophageal dilation is sometimes used to help ease the symptoms of EoE.
Although traditional treatments can be effective for managing symptoms, they do not help address the cause of EoE. Researchers and health care providers continue to investigate new therapies to treat inflammation at its source.
On May 20, the FDA approved the medication dupilumab (Dupixent) for treating pediatric EoE in children and adolescents 12 years and older and adults who weigh at least 88 pounds. This is the first-ever treatment FDA approved for EoE specifically, highlighting an exciting advancement in the field. Dupilumab was originally approved by the FDA in 2017 for treating moderate to severe atopic dermatitis (eczema) in children and adults.
Because EoE is an allergic condition involving the immune system, researchers have found that directly targeting the source of inflammation helps treat it. Dupilumab is a treatment known as a biologic. It uses human-made antibodies (known as monoclonal antibodies) that act on the immune system to dampen inflammation. Specifically, dupilumab works by blocking the effects of proteins called cytokines that are released by eosinophils.
Dupilumab was studied in a randomized phase 3 clinical trial for 24 weeks. Study participants were divided into parts A and B and received either 300 milligrams of dupilumab or a placebo every week during the trial. Researchers measured how well the treatments worked by looking at eosinophil levels in the esophagus under a microscope. They also had participants fill out the Dysphagia Symptom Questionnaire (DSQ) to measure if their dysphagia improved throughout the study.
Overall, 60 percent of participants from part A who received dupilumab met the study goal of reduced eosinophil levels in the esophagus, compared to only 5 percent from the placebo group. The dupilumab group also improved their DSQ score by 22 points, compared to only 10 points in the placebo group.
Participants from part B saw similar results — 59 percent of those who received dupilumab met the study goal of reduced eosinophil levels, compared to 6 percent with placebo. The dupilumab group also improved their DSQ scores by 24 points, compared to only 14 points from the placebo group.
By far, the most common side effect of dupilumab is a reaction at the injection site. Upper respiratory tract infections may also occur. Other, more rare side effects (experienced by less than 3 percent) noted during the research included joint pain and viral infections with a herpes virus.
Other treatments currently being studied in clinical trials for EoE include tezepelumab, benralizumab, and cendakimab. Tools for better diagnosis are also being studied, such as improved ultrasound for detecting EoE.
Tezepelumab is another monoclonal antibody drug that targets a different part of the immune system than dupilumab. It blocks a specific cytokine known as thymic stromal lymphopoietin, which plays a role in different inflammatory conditions such as chronic obstructive pulmonary disease and asthma.
Tezepelumab was given Orphan Drug Designation (ODD) status by the FDA in 2021. The name ODD is given to drugs being researched for treating rare diseases (those that affect fewer than 200,000 people in the U.S.). There are currently no clinical trials that study tezepelumab for treating EoE specifically, but the drug has completed trials for treating asthma.
Benralizumab (Fasenra) is another monoclonal antibody drug that was given ODD status by the FDA in 2019. Cytokines, which send signals for inflammation in the body, work by binding to receptors on the outside of cells. When they bind, they create more inflammation signals to send to other cells. Benralizumab works by attaching itself to the receptor, which blocks the cytokine from binding and sending signals. This process dampens inflammation, which is useful for treating conditions like EoE. Benralizumab blocks signals from the cytokine called interleukin-5.
Benralizumab is already FDA approved as an injectable pen for treating asthma — however, it is not yet approved for treating EoE. One active phase 3 clinical trial is currently studying this medication for treating EoE, and it is set to end in mid-2024. If the results of the trial are positive, the drug may be approved.
Cendakimab is a third antibody drug that also blocks cytokines binding to its receptor. The FDA gave cendakimab ODD status in 2015, and it has since been studied for treating EoE. Some early clinical trials show that this drug reduces EoE and helps some people reach remission (a decrease in or disappearance of signs and symptoms). Researchers are currently recruiting for a phase 3 clinical trial studying cendakimab as maintenance therapy in people with EoE.
Researchers are also interested in coming up with new ways to diagnose gastrointestinal diseases like EoE. Currently, EoE is diagnosed with endoscopy and biopsy, which can be expensive and invasive. With this in mind, many clinical trials have investigated the amount of nitric oxide exhaled by people with EoE and those without. Specialized medical devices are used to measure the concentration of nitric oxide that a person breathes out, which can be used to diagnose EoE.
The Mayo Clinic is also hosting a clinical trial using a special type of ultrasound imaging to look at the esophagus and blood vessels in people with EoE. This would be a less invasive method than endoscopy for diagnosis and could potentially have a significant impact on diagnosing pediatric EoE.
If you are interested in trying a newly approved treatment like dupilumab or want to join a clinical trial, talk to your doctor. They will likely be up to date on studies investigating treatments for gastrointestinal disorders like EoE and can give you recommendations.
On myEoEcenter, the site for people with eosinophilic esophagitis and their loved ones, people come together to gain a new understanding of EoE and share their stories with others who understand life with EoE.
Have you tried dupilumab to treat your EoE? Has your doctor recommended trying an investigational drug? Share your experiences in the comments below.
I just started Dupixent 4 weeks ago. I have been experiencing a lot of nausea. Has anyone else had this reaction? Does your EOE ever make you nauseous? I'm not sure what to think here.