Non-alcoholic fatty liver disease (NAFLD) and associated diseases Non-alcoholic steatohepatitis (NASH), Cirrhosis, and Hepatocellular carcinoma (HCC) cost the US economy an estimated at $103 billion per year. Lipotoxicity is a major reason for NAFLD progression.
To date, there has not been any approved anti-lipotoxic treatment of NAFLD. We discovered that the effect of a combination of specific small soluble compounds synergistically activates defense pathways in hepatocytes against lipotoxicity.
According to our preliminary data, such an approach may lead to novel drug therapy for liver metabolic diseases. So far we have demonstrated a synergistic activation of large number of defense genes. Preliminary in vitro results show that liver cells exposed to a high level of saturated fatty acids that are treated with the proprietary combination of specific small compounds prevent cell death when compared to untreated cells, of which an average 25% of the cells die.
There is no doubt that there is a crucial need to develop a drug that directly addresses the toxicity of saturated fatty acids accumulating in the liver cells and provides a protective mechanism for these cells.
The researcher is seeking research funding for beginning in vivo experiments to test the protective treatment in mice models fed with high fat diets. Additionally the researcher wishes chemically to conjugate small compounds for injection and or oral administration.
This treatment will not only target NAFLD and associated diseases, but also meet an unmet need for drug development for metabolic diseases like atherosclerosis, congestive heart failure beta-cell dysfunction, kidney diseases, and diabetes.