Repurposing a Rare Disease Treatment for the Immune Disorder Eosinophilic Esophagitis
Principal Investigator: Dr. J. Pablo Abonia
Disease: Eosinophilic esophagitis
Research Description: Eosinophilic esophagitis (EoE) is a rare disease caused when the immune system reacts inappropriately to food or other allergens, resulting in damage to the esophagus, and can affect both children and adults. Patients with EoE suffer from debilitating symptoms such as chest and abdominal pain, trouble swallowing and food getting stuck in their esophagus. This hypersensitivity is so severe that patients often must avoid six common food groups and some even require a strict amino acid‐based liquid diet. EoE has no cure, and the current therapies are difficult to tolerate or have undesirable side effects. Alpha‐1 antitrypsin (A1AT) is a protease inhibitor found in the blood, and patients who lack A1AT receive an FDA‐approved A1AT replacement therapy (Zemaira) to prevent lung and liver damage. A1AT acts similarly to the protease inhibitors lacking in EoE. The research team will investigate the ability of A1AT replacement therapy to treat EoE in 15 adult patients. Positive results from this pilot study could provide the basis for larger follow-on trials while improving the quality of life for EoE patients.
Funding Partners: Cures Within Reach
CWR funding role: Primary funder