Scientists Cure Diabetes In Mice With No Side Effects

A potential cure for Type 1 diabetes appeared on the horizon in San Antonio. The novel approach would also allow people who suffer from Type 2 diabetes to quit insulin shots.

Researchers at The University of Texas Health Science Center, which is now called UT Health San Antonio, made a discovery that increases the types of pancreatic cells that secrete insulin.

UT Health San Antonio researchers aim to reach human clinical trials in 3 years. However, to do so they should first test their strategy in large-animal studies, that will cost approximately $5 million.

These studies will foreshadow application to the U.S. Food and Drug Administration for Investigational New Drug (IND) approval, as Bruno Doiron, Ph.D., a co-inventor, stated.

The researchers received a U.S. patent in January, and UT Health San Antonio is spinning out a company to start commercialization.

Their strategy has cured diabetes in mice.

According to Dr. Doiron, assistant professor of medicine at UT Health, the strategy "worked perfectly. We cured mice for one year without any side effects. That's never been seen. But it's a mouse model, so caution is needed. We want to bring this to large animals that are closer to humans in physiology of the endocrine system."

As Ralph DeFronzo, M.D., professor of medicine and chief of the Division of Diabetes at UT Health, and co-inventor on the patent described the therapy:

"The pancreas has many other cell types besides beta cells, and our approach is to alter these cells so that they start to secrete insulin, but only in response to glucose [sugar]. This is basically just like beta cells."

Insulin, which reduces blood sugar, is only created by beta cells. Here's what happens in Type 1 diabetes: beta cells are destroyed by the immune system and, then, the person has no insulin. In Type 2 diabetes, beta cells fail, and insulin is lowered. At the same time in Type 2, the body doesn't use insulin in an efficient way.

The therapy is accomplished by a technique named gene transfer. A virus is used as a vector, or carrier, in order to introduce selected genes into the pancreas. The genes become incorporated and cause digestive enzymes and other cell types to make insulin.

Gene transfer using a viral vector has been approved approximately 50 times by the U.S. Food & Drug Administration to cure various diseases, Dr. DeFronzo noted. It is proven in curing rare childhood diseases, and Good Manufacturing Processes guarantee safety.

Contrary to beta cells, which the body rejects in Type 1 diabetes, the rest of cell populations of the pancreas co-exist with the body's immune defenses.

As Dr. DeFronzo said:

"If a Type 1 diabetic has been living with these cells for 30, 40 or 50 years, and all we're getting them to do is secrete insulin, we expect there to be no adverse immune response."

The second-by-second sugar control precisely regulates blood sugar in mice. This could be a great advance over traditional insulin therapy and some diabetes medications which drop blood sugar very low unless they are closely monitored.

"A major problem we have in the field of Type 1 diabetes is hypoglycemia (low blood sugar)," Dr. Doiron said. "The gene transfer we propose is remarkable because the altered cells match the characteristics of beta cells. Insulin is only released in response to glucose."

Provided by: University of Texas Health Science Center at San Antonio

Reference: Medical Express