Researchers identify special protein that could treat metabolic diseases

Researchers at the Hebrew University of Jerusalem's Institute for Drug Research, Israel, led by Professor Amiram Goldblum, have discovered 27 new molecules that all activate a special protein called PPAR-delta, which have the potential to treat fatty liver disease, obesity, diabetic nephrotoxicity and to heal wounds.

The findings, ‘PPAR-delta agonists by ligand based machine learning and structural modeling’, published in Scientific Reports, a Nature journal, and was made possible thanks to an award-winning algorithm that Goldblum's team developed. This algorithm sifted through a database of 1.56 million molecules and picked out 27 with a strong therapeutic potential, as determined by biologists at the Novartis Genomic (GNF) Institute in San Diego, CA,

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Amiram Goldblum

To date, these new molecules are undergoing pharmaceutical evaluations to treat two main health conditions. The first is non-alcoholic steatoheptatis (NASH). This disease currently has no cure and is a leading cause of liver cancer in the Western world. The second is obesity. PPAR-delta activation has the potential to increase physical endurance and trim waistlines by getting muscle cells to burn more fat. Future evaluations will hopefully include testing treatments for improved wound healing, and to prevent kidney toxicity in diabetics.

"With such a large group of highly active molecules, there is a high probability to find treatments for several common diseases. However, we should wait till all the experiments are done before we get our hopes up too high," explained Goldblum.

To date, there is much pharmaceutical interest in Goldblum's new molecules. Integra Holdings, Hebrew University's biotech company, determined that 21 of the 27 have the potential to reach pharmaceutical success, especially as a possible cure for Fatty Liver disease. Additionally, Israel's Heller Institute of Medical Research is currently testing PPAR-delta's physical endurance properties on mice. Goldblum predicts that in a few years we will hopefully be seeing several of these molecules in the pipeline for clinical studies on humans.

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