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Research Fund 2007

MACPAD awards research funds to.


Dr Ray Stevens at the Scripps Research was awarded $105,000 to continue his work toward the conversion of his once a week injectable enzyme replacement therapeutic to an oral pill form. The injectable enzyme replacement drug will be entering human trials early next year. The funds currently awarded are to be used along with a National Institute of Health grant to develop a pill form of the drug. Both the injectable form and the pill form of the drug will provide a replacement for phenylalanine hydroxylase, the enzyme that digests phenylalanine in the body and does not work properly in someone with PKU.


During the last 12 years Dr. Stevens has focused on understanding the science behind PKU and its cause. In collaboration with BioMarin Pharmaceuticals and Montreal Children's Hospital, he has developed dihydrobiopterin (Kuvan), a soon to be approved oral therapeutic for some forms of PKU.


Dr. Denise Ney at the University of Wisconsin System was awarded $23,000. to continue her studies to assess the efficacy and safety of glycomacropeptide in the diet. GMP is a natural protein found in cheese whey that is uniquely suited for dietary control of PKU because it contains very low levels of phenylalanine. She has developed a variety of palatable foods and beverages made with GMP that have been well accepted by individuals with PKU. The goal is to make GMP a safe and palatable alternative protein in the PKU diet that will improve dietary compliance.


Dr Stephen Hunter of the University of Iowa Hospitals and Clinics was awarded $15, continue the pursuit of the development of a novel non-dietary therapy for maternal PKU. He hypothesizes that cells can be treated to over-express active phenylalanine hydroxylase, the enzyme required to convert phe to tyrosine. These cells can then be protected from the immune system through encapsulation, thus allowing the transplantation of these encapsulated cells without the need for immunosuppressant drugs. Therapy with these encapsulated cells during pregnancy will produce normal phe levels on a normal diet and eliminate the mental and physical anomalies imparted to the child.