Substrate Reduction Therapy Sanfilippo A,B,C,D (Genistein Derivatives)
University of Gdansk Department of Molecular Biology
Dr. Grzegorz Wegrzyn wegrzyn@biotech.ug.gda.pl
Dr. Wegrzyn has tested many synthetic, artificial derivatives of genistein. Based on his evaluation there are a few that are promising (ability to inhibit GAG synthesis, low cytotoxicity and high potential to cross the blood-brain-barrier). Until now, we have performed only experiments on cell cultures, thus, the next step should be tests on mice. The promissing molecules are about 50% more active than genistein, are 2-3 times less toxic than genistein (though genistein is still fine in these tests), and have several times higher LogP index (calculated theoretically on the basis of chemical formulas) suggesting their more efficient crossing the blood brain barrier. We are now negotiating with a company which is interested in patenting these molecules.