MicroRNA Delivery as a Novel Therapeutic Strategy for Liver Cancer

Unmet need
Over 500,000 people are diagnosed with liver cancer worldwide each year. According the American Cancer Society, liver cancer resists most chemotherapy drugs and the most effective agents shrink less than 1 in 5 tumors and generally for only a short time. Hepatocellular carcinoma (HCC) is often diagnosed at an advanced stage making it difficult to cure. Further, the efficacy of current therapeutic regimen is reduced due to the presence of active drug-metabolizing pathways and multi-drug resistance transporters. There is need in art for alternative approaches for treatment of HCC.
 
Technology overview
Researchers at the Johns Hopkins University and Nationwide Children’s Hospital have developed a method to treat liver cancer comprising a method to systemically deliver therapeutic microRNAs. They have discovered that a microRNA that is normally expressed at high levels in various tissues exhibits reduced expression in liver tumors or hepatocellular carcinomas (HCC). They have developed a method to treat and suppress HCC utilizing systemic delivery of a specific microRNA in an adeno-associated virus (AAV) system which additionally simultaneously expresses eGFP for easy assessment of target tissue transduction. When the AAV-mediated microRNA is delivered systemically to mice, the microRNA is expressed at high levels in the liver without toxic effects to normal mice while suppressing tumors in tumor bearing mice. This method offers a therapeutic mechanism to treat and suppress liver cancer that minimizes toxicity to patients.
 
Stage of development
Pre-clinical

Publications
Kota J et al. Cell 137(6), 1005–17, 2009
 

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