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Promising Findings: Common Antihistamine May Alleviate Liver Complications in Rare Disease
- Common antihistamine, chlorcyclizine shows promise for treating erythropoietic protoporphyria (EPP), a rare genetic disorder that can lead to severe liver damage and may necessitate a liver transplant.
- EPP affects an estimated 4,000 individuals in the US, and current treatment protocols predominantly involve managing symptoms and providing supportive care, leaving a substantial unmet need for effective interventions.
- Researchers at Rutgers Health screened over 2,500 compounds, particularly focusing on FDA-approved medications to identify potential treatments for EPP.
- Researchers found a noticeable reduction of hepatic protoporphyrin levels and corresponding liver injury in mice models with EPP upon applying chlorcyclizine.
- Chlorcyclizine appears to operate through a multifaceted mechanism to enhance the liver's ability to expel toxic protoporphyrin and reducing the overall inflammatory response associated with liver damage.
- The historical safety profile of antihistamines provides a solid foundation for their application in new therapeutic contexts, enabling potential streamlined development of chlorcyclizine for EPP.
- Clinical trials are being proposed and researchers aim to secure funding and support to evaluate the efficacy of chlorcyclizine in EPP patients and explore combined use with other antihistamines.
- The findings hold promise for a brighter future for individuals battling the challenges of erythropoietic protoporphyria, and the ongoing research may very well signal a turning point in their journey toward better health and diminished suffering.
- The study serves as a compelling reminder of the value of rigorous scientific inquiry and innovation in the face of unmet medical needs, and highlights the collaborative spirit essential for advancing medical research towards tangible patient benefits.
- The potential for chlorcyclizine to provide a safe, effective therapy holds promise for the entire field of rare disease therapeutics.
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