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Invention Summary:
Erythropoietic protoporphyria (EPP) is a severe genetic disorder caused by defects in heme biosynthesis, leading to the accumulation of protoporphyrin IX (PP-IX) in the liver. This accumulation results in cholestasis and, in the most severe cases, end-stage liver failure, necessitating liver transplantation or leading to death. Currently, there are no effective treatments available to counter the progression of liver disease in EPP, leaving patients with limited options and a significant unmet medical need.
Researchers at Rutgers have identified H1 and H2 antihistamines, such as chlorcyclizine (CCZ) and cimetidine, as potential therapeutic agents for EPP. In zebrafish, mouse hepatocytes, and an in vivo mouse model, antihistamines significantly reduced hepatic PP-IX accumulation and associated liver injury. Data show that treatment decreased PP-IX levels in the liver and increased excretion, while reducing liver injury markers and oxidative stress, suggesting that repurposing antihistamines offer a promising new approach for EPP, potentially reducing the need for liver transplantation and improving patient outcomes.
Market Applications:
- Treatment for erythropoietic protoporphyria
Advantages:
- Current treatments for erythropoietic protoporphyria are invasive and costly, but repurposed antihistamines could enable effective treatment that is neither invasive nor costly.
Publications: • Manuscript in-review.
Intellectual Property & Development Status: Provisional application filed. Patent pending. Available for licensing and/or research collaboration. For any business development and other collaborative partnerships, contact: marketingbd@research.rutgers.edu
