PROJECT TITLE Reversing diabetes with insulin produced by a patient's own stem cells
CATEGORY Diabetes, Gene Therapy
PROJECT 
DESCRIPTION
Type 1 diabetes mellitus, a disorder of complete insulin deficiency, afflicts about 1.5 million Americans. The resulting kidney, eye, nerve, and heart damage makes diabetes one of the leading causes of illness and death in the United States. Type 1 diabetics are completely dependent on external sources of insulin. 

The objective of this research project is to show that gene therapy can transform stem cells within a diabetic's own body to produce insulin. This approach could provide a permanent life-saving method of reversing diabetes by producing insulin within a patient's body without the side effects of the drugs necessary to keep transplanted donor cells alive.

This project has several objectives.  The first is to show that stem cells in the patient's own pancreatic duct can safely undergo gene transformation.  The gene changes will be produced by injecting the pancreatic duct with a harmless virus.  The virus will specifically invade the stem cells, and the viral mechanisms will create the necessary gene changes.

The second step is to determine if these gene changes will allow the cells to begin producing insulin.  The researchers will determine if the insulin produced is normal, and if the cells react be producing the insulin in response to changes in the blood sugar. It will also be critical to determine if the amount of insulin produced is sufficient to eliminate the need for external insulin.

The last step is to see if these transformed insulin producing cells can survive in the body.  Since diabetes is in part an auto-immune disease, the patient's own immune system attacks its own cells.  

Success of this study will create a human clinical trial that could find a solution for the millions of people who suffer from type I diabetes.

INVESTIGATORS Raghavendra "Raghu" G. Mirmira
CO-FUNDERS Meade Family Foundation, University of Virginia Medical School
INSTITUTION University of Virginia Medical Center
Fontaine Research
2322 Box 801407
Charlottesville, VA 22903
STATUS Project began May 2004 and just received an additional $900,000 American Diabetes Association grant.