A common antihistamine may offer hope for patients with a rare genetic disease that can lead to severe liver damage and ultimately require transplantation, according to new research from Rutgers Health. The study in Cellular and Molecular Gastroenterology and Hepatology found that chlorcyclizine, a decades-old allergy medication, could potentially treat erythropoietic protoporphyria (EPP), a condition that creates extreme skin light sensitivity and can produce toxic levels of protoporphyrin in the liver, bone marrow, red cells, and plasma.
“There is an unmet need for these patients,” said Bishr Omary, senior vice chancellor for academic affairs and research at Rutgers Health and senior author of the study. “The primary treatment for patients with severe liver damage is liver transplantation, which is a major and life-saving surgery that depends on available donor organs.” EPP is a rare condition, affecting an estimated 4,000 people in the U.S. Of those, only a small percentage suffer enough liver damage to require transplantation. It is unlikely, therefore, that any company will develop a drug to treat the condition, which is why Omary and his colleagues tested existing medications.
The research team screened more than 2,500 compounds, including many FDA-approved drugs, in a zebrafish larvae EPP experimental system previously described by Omary’s lab. The tiny fish allows researchers to easily visualize the buildup of toxic compounds and test potential treatments. To read the full story.