Professor Grzegorz Wegrzyn's New Funding The MPS Society in UK recently committed $250,000 to fund the research of Professor Grzegorz Wegrzyn in Gdansk, Poland, "Development of Gene Expression-Targeted Isoflavone Therapy (GET IT) for Mucopolysaccharidosis Type III". Below is the statement from the UK MPS Society about this research. "As far as the MPS Society is concerned we have approved the research project 'Development of Gene Expression-Targeted Isoflavone Therapy (GET IT) for Mucopolysaccharidosis Type III' being carried out by Professor Grzegorz Wegrzyn in Gdansk and would expect this research to be carried out as per the protocol submitted. Although we sincerely hope that the research will have a therapeutic benefit for Sanfilippo patients the research is currently being carried out in the mouse model. We recognise that Isoflavone can be bought over the counter in many countries and any decision to use this product orally in the case of a child or adult suffering from Sanfilippo disease should be taken with the full agreement and understanding of that child's MPS paediatrician or in the case of an adult the relevant MPS clinician." Statement on the use of Genistein in MPS patients I have become aware that a number of families are starting their children on Genistein. The background to this development is that a team of researchers under the lead of Professor Grzegorz Wegrzyn have found that in cultures of skin cells (fibroblasts) that Genistein inhibits the build up (synthesis) of glycosaminoglycans in particular heparan sulphate the GAG that accumulates in MPS III (and also to a lesser extent in MPS I, II and VII). There is no evidence that a similar effect will occur in a patient with one of these disorders and whilst the finding is interesting it is an indication to study the effect more closely rather than an indication to support the widespread use of Genistein in MPS patients. Further research work is needed to clarify whether or not the findings in skin fibroblasts are significant or not. We are working with our Polish colleagues to try clarify this. We plan to use the product on mice with MPS I and III and will study both the pathological and clinical response to this therapy in the mice. Assuming that these studies show some evidence of efficacy it is likely that human studies would follow. Whilst I understand why many families have become excited by this work I feel it is important to avoid the indiscriminate use of Genistein as this may hamper any attempt to find out if it really is useful treatment for our children. Dr. J.E. Wraith 12/july/2005