Diabetes Research Institute 2003 Research Update
On Saturday, October 25, 2003, researchers from the Diabetes Research Institute in Miami, Florida, along with other colleagues, spoke to hundreds of guests about the progress being made in the quest for the cure for type 1 diabetes. Master of Ceremonies and DRIF National Development Director Tom Karlya opened the conference and welcomed everyone. Dr. Denise Stern, Research Update Chairperson, then introduced the first scientist, Dr. Alberto Pugliese, who spoke about type 1 diabetes and the immune system.
Dr. Pugliese described how the thymus is supposed to eliminate immune system cells that attack the body, including the cells that would attack beta cells. Somehow that process of elimination doesn't happen correctly in people with type 1 diabetes, and those auto-reactive immune cells survive. If they then become activated, those cells lead the destruction of the beta cells and the person develops type 1 diabetes. Dr. Pugliese and his associates are working on ways to restore self tolerance to those otherwise damaging immune system cells in the hope that the underlying autoimmune destruction of beta cells that defines type 1 diabetes can be halted.
Dr. Camillo Ricordi followed Dr. Pugliese and spoke about the status of islet cell transplantation. He recounted the many years of difficulty and poor success rates that preceded the Edmonton Protocol process, which for the first time led to substantial success in patients achieving normal HbA1c readings following islet cell transplantation. Dr. Ricordi also spoke about the efforts of the Immune Tolerance Network to establish new islet transplantation centers so as to better meet the needs of people who want and need islet transplants.
The next speaker was Dr. David Scharp, Chief Scientific Officer and Executive Vice President for Research and Medical Affairs from Novocell, Inc., a company that is working to develop and market encapsulated islets to cure type 1 diabetes. Novocell has developed a conformal coating process that encapsulates individual islets and has been shown to be effective in restoring normal glucose levels in pigs and non-human primates. The encapsulation technology eliminates the need for immunosuppressive drugs since the coating prevents immune system components from reaching the islets. Dr. Scharp said that the next step is to work with the FDA to begin trials in humans.
Dr. Elizabeth Fenjves spoke about her work involving islet regeneration and protection using genetic therapy. Her efforts focused on ways to get transplanted islet cells to produce erythropoietin (EPO), a chemical which protects cells, including beta cells, from cytokine-induced apoptosis. Apoptosis means programmed cell death, and it is through apoptosis that the immune system destroys beta cells in people with type 1 diabetes. Since this underlying autoimmunity remains in place for people who receive islet cell transplants, Dr. Fenjves hopes that her work will help transplanted islets survive better, which could perhaps reduce the amount of islets needed for a successful transplant. For more information, see Human, nonhuman primate, and rat pancreatic islets express erythropoietin receptors.
Dr. Sarah Ferber from the Sheba Medical Center in Israel then spoke about her efforts to induce liver cells to sense and respond to glucose levels in the blood. Her team uses a modified virus that contains PDX-1 to coax liver cells to begin expressing genes that turn them, in effect, into pancreatic beta cells. This process has proven to be very successful in animal models that are made diabetic through drugs, but only 60% effective in the NOD mouse autoimmune model of type 1 diabetes. For more information, see Functional, persistent, and extended liver to pancreas transdifferentiation.
Dr. Pugliese spoke about type 1 diabetes and the immune system
Dr. Ricordi brought us up-to-date on the status of islet cell transplantation
Islet transplant recipient Gary Kleiman spoke at lunch
October 26, 2003
Last Updated: Thursday February 27, 2014 19:28:21
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