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Children's Hospitals Making Way for New Research and Advanced Pediatric Medicine

by Kathy Mahdoubi, Senior Correspondent | February 01, 2010

Dr. Paul Burn is one of those leading researchers heading up the Project. Burn is an M.D. and professor of pediatrics at the University of South Dakota Medical Center and is also senior vice president of research and development for the Juvenile Diabetes Foundation. His work with Sanford Children's Hospital and the Sanford Project is focusing on the regeneration of beta cells - the cells involved in the production of insulin - as well as the immune response that triggers the disease.

"Sanford's research into beta cell regeneration is just getting started," says Dr. Burn. "Our hope is that we can take what we learn to the patients, because we feel like we have a unique opportunity to translate that to an underutilized and relatively stable patient population."

Regeneration for the new generation
Type 1 diabetes is usually diagnosed in children from birth up to the first two decades of life. In this autoimmune disease, the immune systems of diabetes patients mistake pancreatic beta cells as a foreign threat, and begin destroying those cells. If all the beta cells are destroyed, the body is no longer able to produce insulin to regulate blood sugar levels.

Sanford Children's Hospital
became the first children's
facility in the country
to offer new CT technology
to patients.


"Injecting insulin is not really a cure for the disease," says Burn. "You are just replacing what the body can no longer provide for itself. If you want to interfere with that disease process you have to moderate the immune system so that the immune system no longer attacks those beta cells."

Dr. Burn and his growing team of researchers are getting a jump-start on the Project-and the cure-by researching a novel pharmaceutical approach to beta cell regeneration using the pill-form of already FDA-cleared drugs, namely a protein called GOP-1 and gastrin, both of which are currently used for other indications.

"Those two drugs have been shown to endogenously elevate two factors that are responsible for regenerating beta cells and may be having an effect on the immune system, as well," says Dr. Burn.

In March of 2010, Sanford Children's will prepare the investigational new drug and apply with the FDA to begin clinical trials. If successful, Sanford Children's could be on the fast track to a cure. If all goes well, the treatment might one day also be applied to type 2 diabetes sufferers.