The first interim report on the NORD Research Grant to Dr. Diva DeLeon of the Children’s Hospital of Philadelphia for her work on exendin (9-39) has been submitted. NORD has graciously permitted us to share the public portion of this interim report. This groundbreaking research is funded by the Mario Batali Foundation. Dr. Deleon receives high marks from the NORD Medical Advisory Group for this work: Here is the text available to the public:
“The most common and severe form of congenital hyperinsulinism is due to mutations of the KATP channels. Children with this form of hyperinsulinism have severe fasting and protein-induced hypoglycemia. Most of these children are unresponsive to medical therapy and require pancreatectomy shortly after birth to ameliorate the hypoglycemia. Thus, there is an urgent, unmet need for safer and more effective medical treatments for this condition.
We have strong preclinical and clinical data supporting a novel therapeutic approach employing the GLP-1 receptor antagonist, exendin-(9-39). The purpose of this proposal is to examine the effect of exendin-(9-39) on protein-induced hypoglycemia using a mouse model of congenital hyperinsulinism and pancreatic islets isolated from children with this disease. The results from the studies supported by NORD in combination with ongoing clinical studies with exendin-(9-39) will provide critical data for the design of a pivotal clinical trial. Our goal is to ultimately develop exendin-(9-39) as a subcutaneous drug to treat children with congenital hyperinsulinism.”
