ALTERNATIVE DIETARY THERAPIES FOR GLYCOGEN STORAGE DISEASE TYPE 1

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The glycogen storage diseases (GSD) are a set of genetic disorders in which the phenotype is severe hypoglycemia because of the inability to produce glucose from glycogen in the liver and kidney 1-3. Type I glycogen storage disease (GSDI) is caused by a deficiency of hepatic glucose-6-phosphatase, which is an integral part of both glycogenolysis and gluconeogenesis 3. Lack of this enzyme results in the inability to maintain normal glucose homeostasis and severe fasting hypoglycemia. Patients presently are treated with an average of 6.2 doses of cornstarch per day. There is a need for a digestible starch that can provide a source of glucose for longer duration after meals in GSD patients. The purpose of this pilot study is to explore a modified cornstarch with properties that suggest a better glycemic index and the ability to provide prolonged glucose absorption compared to the traditional cornstarch therapy used for these patients. The main goals of this study will be the tolerance and safety of the glucose sources. This study is not designed to demonstrate significance, but rather to identify the best agent for future studies.