Experimental Drug Protects Animals from Type 1 Diabetes by Reducing Beta Cell Stress

Experimental Drug Protects Animals from Type 1 Diabetes by Reducing Beta Cell Stress

–JDRF-funded study confirms the role of role of beta cell stress in type 1 diabetes and discovers that an experimental drug can protect animals from type 1 diabetes by preventing beta cell stress–

JDRF Media Contact:     
Tara Wilcox-Ghanoonparvar           


New York, NY, June 11, 2012-JDRF-funded researchers at Harvard School of Public Health have found that an experimental drug, called TUDCA, can dramatically reduce the occurrence of type 1 diabetes (T1D) in mice specially bred to develop the disease. Presented at the American Diabetes Association’s (ADA) 72nd Scientific Sessions in Philadelphia, they reported that TUDCA protects beta cells from a stress response that occurs in beta cells under diabetic or pre-diabetic conditions. The primary function of the pancreatic beta cell is to synthesize and secrete insulin in response to glucose. Under normal conditions, insulin is produced by the beta cell in a highly controlled manner. But excess glucose or other stresses in the body can alter the ability of the beta cell to secrete sufficient insulin, and beta cells may become overwhelmed and die. This pathological stress response is increasingly thought to be a contributing factor in T1D as well as type 2 diabetes. TUDCA is currently in clinical study as a potential treatment for people with signs or symptoms of type 2 diabetes. 

“Based on earlier work, we expected this might be a useful approach for reducing the incidence of type 1 diabetes, but we were surprised by the magnitude of the effect,” said Gokhan Hotamisligil, Ph.D. the principal investigator of the study. “Given that this mechanism of beta cell destruction and early onset diabetes is shared between experimental rodent models and humans, the potential for translating these findings to humans is clear. While TUDCA may not be the optimal therapy due to its chemical properties, these findings hold great promise for guiding the development of an improved therapy for the prevention or treatment of recent onset type 1 diabetes.”

In the work presented at the ADA meeting by Dr. Hotamisligil’s post-doctoral fellow, Feyza Engin, Ph.D., mice bred to get T1D were treated with TUDCA for four weeks. At the end of the treatment period, the treated mice experienced significantly lower inflammation in the pancreas compared to untreated mice. In another experiment, T1D-prone mice were treated daily with TUDCA for 30 weeks and blood glucose levels were monitored daily to detect the onset of T1D. As a result of this treatment, only 10 percent of the TUDCA-treated mice developed T1D compared to almost half of the untreated mice. Dr. Engin also went on to show  that biochemical changes in the stress response in beta cells from the animals correlate with similar changes in isolated human beta cells obtained from people with T1D.

“Curing type 1 diabetes requires restoring and maintaining a person’s functional beta cells,” said Patricia Kilian, Ph.D. director of JDRF’s beta cell regeneration therapies program. “A major goal of JDRF’s regeneration program is to develop therapies to promote beta cell health and prevent beta cell loss. Dysfunction of the beta cell from beta cell stress appears to be a component of type 1 diabetes, and this research suggests that therapies targeting it may have the potential to prevent the disease or to preserve beta cell function in recent onset type 1 diabetes.”


About T1D
In T1D, a person’s pancreas stops producing insulin, a hormone that enables people to get energy from food. People with T1D need to test their blood sugar and give themselves insulin (with injections or an insulin pump) multiple times every day, and carefully balance insulin doses with eating and daily activities throughout the day and night. However, insulin is not a cure for diabetes, and even with that intensive care, a significant portion of the day is still spent with either high or low blood sugar, placing people with T1D at risk for devastating complications such as heart attack, stroke, blindness, and amputation.

About JDRF

JDRF is the leading global organization focused on type 1 diabetes (T1D) research. Driven by passionate, grassroots volunteers connected to children, adolescents, and adults with this disease, JDRF is now the largest charitable supporter of T1D research. The goal of JDRF research is to improve the lives of all people affected by T1D by accelerating progress on the most promising opportunities for curing, better treating, and preventing T1D. JDRF collaborates with a wide spectrum of partners who share this goal.

Since its founding in 1970, JDRF has awarded more than $1.7 billion to diabetes research. Past JDRF efforts have helped to significantly advance the care of people with this disease, and have expanded the critical scientific understanding of T1D. JDRF will not rest until T1D is fully conquered. More than 80 percent of JDRF’s expenditures directly support research and research-related education.