news-medical.net
Recent research highlights chlorcyclizine's promising role as a treatment for erythropoietic protoporphyria, a condition that leads to severe liver damage.
This development is significant as chlorcyclizine could prevent severe liver damage, reducing the need for liver transplants, thereby offering an alternative treatment for EPP patients.
The study led by Rutgers Health and published in the journal 'Cellular and Molecular Gastroenterology and Hepatology', explores the potential of chlorcyclizine, a widely used antihistamine, to treat erythropoietic protoporphyria (EPP). Researchers utilized zebrafish larvae and mouse models to demonstrate the drug's efficacy in reducing hepatic protoporphyrin accumulation and related liver injuries, particularly in female mice. The findings suggest that chlorcyclizine could offer a less invasive treatment option for EPP patients, potentially preventing severe liver damage requiring transplantation. The research advocates for clinical trials to further explore the drug's effectiveness in human subjects.
Erythropoietic protoporphyria (EPP) is a genetic disorder characterized by extreme sensitivity to light and a risk of severe liver damage due to the accumulation of protoporphyrin. This rare condition can significantly impair quality of life and may necessitate liver transplants in severe cases. Understanding its pathophysiology is essential to develop effective treatments.
“There is an unmet need for these patients. 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.” - Bishr Omary
Researchers at Rutgers Health explored chlorcyclizine, a common antihistamine, as a potential treatment for EPP. They screened over 2,500 compounds using a zebrafish model to identify drugs that could alleviate protoporphyrin buildup. Chlorcyclizine emerged as a promising candidate, demonstrating the ability to clear hepatic protoporphyrin accumulation effectively.
Detailed studies revealed that chlorcyclizine works through several mechanisms, including reducing porphyrin accumulation in the liver and decreasing inflammation. The research demonstrated that it decreases mast cell presence, which is integral to histamine production, thus helping manage the biochemical landscape of EPP.
Considering the promising results from preclinical studies, the researchers advocate for clinical trials to test chlorcyclizine’s effectiveness in EPP patients. The established safety profile of antihistamine drugs could facilitate quicker trials, offering a potential new treatment pathway for those affected by EPP.
“Given their well-established safety, we hope to fast-track trials of chlorcyclizine either alone or in combination with cimetidine.” - Ning Kuo
A Look at the Treatment Landscape for Gastroparesis
A Look at the Treatment Landscape for Gastroparesis
