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JDRF
and W.W. Smith Trust's $15.5 Million Gift for Type 1 Diabetes Cure
The Juvenile Diabetes Research Foundation International (JDRF) announced a major step in its continuing efforts to find a cure for Type 1 diabetes and its complications. It is awarding over $15.5 million to fund research at the JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation and the JDRF Center for Gene Therapy at Penn's School of Medicine. Over a five-year period, the JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation is receiving $8.1 million to meet the challenges of restoring normal insulin production in patients with insulin-dependent diabetes mellitus (Type 1 diabetes). The JDRF Center for Gene Therapy is receiving $7.57 million to conduct five projects focusing on gene transfer technology and gene therapy for complications of diabetes. "While our major research goals remain the same, the use of cutting-edge technology will speed up our progress toward finding a cure," said Charles J. Queenan III, JDRF's chair of research. "We rely on the generosity of funders such as the W.W. Smith Charitable Trust and thank them for their help in stamping out this insidious disease." Islet Transplantation The JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation is developing an integrated approach--three projects and two supporting core units--to deliver a successful clinical islet transplantation program. One project is using a non-invasive technique, positron emission topography (PET), to better understand the fate of islets after transplantation. A radioactive version of a drug has been developed that would bind to molecules found specifically on islets. Currently, studies are examining whether the link between the islets and the radioactive drug is strong enough to produce optimal visual images. "We are working on two important strategies. First, we want to make islets less vulnerable to attacks from the body's immune system and secondly, we are investigating new methods of observing those transplanted cells in action," commented Dr. Ali Naji, director of the JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation. In another project, Center scientists have already determined that transplanted islets have a greater chance of surviving in the thymus of rodents with diabetes. Their next step is to conduct similar studies in pre-clinical animal models and use a novel protocol of drug treatment to prevent the immune system from attacking transplanted cells. A Human Islet Transplantation Clinical Program brings the research from the lab to the patient with Type 1 diabetes. This program will identify and monitor patients with Type 1 diabetes who are suitable candidates for islet transplantation. The program will test the "Edmonton Protocol" for islet transplantation, using a combination of three steroid-free drugs to prevent rejection of the donor islets. The two core units were established to focus on procuring human pancreata and developing a standardized system to isolate high quality islets for distribution. The islet core facility will generate large quantities of highly purified and quality controlled human islets which will be used for transplantation. The Center, now also funded by the NIH as an Islet Cell Resource Center, is a comprehensive islet transplantation program, focusing on the isolation, evaluation, transplantation and imaging of high-quality human islets for treatment of Type 1 diabetes mellitus. As principal investigator, Dr. Naji is responsible for assessing the feasibility of islet transplantation in humans and evaluating new immunosupression protocols associated with the procedure. Gene Therapy The JDRF Center for Gene Therapy will use gene transfer technology and gene therapy to develop new and more effective treatments for Type 1 diabetes and its complications. At the JDRF Center for Gene Therapy scientists are making progress using gene therapy for wound healing, foot ulcers, and diabetic retinopathy--the alterations in the blood vessels of the retina caused by high levels of glucose. Growth factor genes are delivered directly to the wounded tissue, to promote healing where it is needed. Researchers are assessing whether transferring vascular endothelial growth factor (VEGF) genes to veins and arteries could promote blood vessel growth. They are also using a new technology--magnetic resonance angiography--to observe how blood vessels are formed. They are developing new vectors to deliver genes to the eye that will cause cells to produce proteins in the vitreous fluid. Abnormal blood vessel growth would be slowed down or stopped. Investigators, with Dr. James M. Wilson, director of the JDRF Center for Gene Therapy, are exploring an important challenge in gene therapy: the ability to control gene expression. When researchers transfer genes responsible for insulin production, there needs to be control because unchecked insulin leads to hypoglycemia which can be life threatening. Dr. Wilson is developing a way to turn the action of a particular gene on and off--a "biological insulin pump". Scientists are using a virus vector to deliver the insulin gene into the body. The patient would activate this transferred gene and switch on the ‘pump' by taking an oral drug. When the patient stops the drug, the insulin would switch off. The goal would be the control of blood sugar levels in a simple, patient-directed manner. "We have gathered a committed group of Penn experts with a clear objective: to develop viral vectors and use gene transfer to pursue many approaches to treatment of complications," said Dr. Wilson. "This collaboration, I believe, will produce some promising research for clinical applications."
Almanac, Vol. 48, No. 13, November 20, 2001 |
ISSUE HIGHLIGHTS: Tuesday,
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