Team cures diabetes in mice without side effects

May 5, 2017 by Will Sansom
Bruno Doiron, Ph.D., left, and Ralph DeFronzo, M.D., of UT Health San Antonio co-invented a patented technique that has cured diabetes in mice for one year without side effects. Credit: UT Health San Antonio

A potential cure for Type 1 diabetes looms on the horizon in San Antonio, and the novel approach would also allow Type 2 diabetics to stop insulin shots.

The discovery, made at The University of Texas Health Science Center, now called UT Health San Antonio, increases the types of that secrete .

UT Health San Antonio researchers have a goal to reach human clinical trials in three years, but to do so they must first test the strategy in large-animal studies, which will cost an estimated $5 million.

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

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

The strategy has cured diabetes in mice.

"It worked perfectly," Dr. Doiron, assistant professor of medicine at UT Health, said. "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."

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

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

Insulin, which lowers , is only made by beta cells. In Type 1 diabetes, beta cells are destroyed by the immune system and the person has no insulin. In Type 2 diabetes, beta cells fail and insulin decreases. At the same time in Type 2, the body doesn't use insulin efficiently.

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

Gene transfer using a viral vector has been approved nearly 50 times by the U.S. Food & Drug Administration to treat various diseases, Dr. DeFronzo said. It is proven in treating rare childhood diseases, and Good Manufacturing Processes ensure safety.

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

"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," Dr. DeFronzo said.

Second-by-second sugar control

The therapy precisely regulates blood sugar in mice. This could be a major advance over traditional insulin therapy and some diabetes medications that drop blood sugar too low if not closely monitored.

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

People don't have symptoms of until they have lost at least 80 percent of their beta cells, Dr. Doiron said.

"We don't need to replicate all of the insulin-making function of beta cells," he said. "Only 20 percent restoration of this capacity is sufficient for a cure of Type 1."

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1 / 5 (1) May 06, 2017
$5 million seems ridiculously expensive, and three years an excessively long time for animal trials. Surely a few transfers to see the technique work in apes would be sufficient to support a clinical trial in human beings without such a commitment of time and treasure?
The cure for diabetes might be human skin surface pheromones, to decommission the body's rejection. Transferring pheromones is done all the time in kissing, and there are no infections, no surgery necessary. Extreme care must be taken to prevent side effects in collection and administration staff, because these are, after all, human pheromones the most potent of drugs and capable of dramatic artificial emotional "damage" which can last decades. Bitterness, jealousy, arrogance, superstition, suspicion, and even stupidity/astonishment can be permanent poison difficult to digest, surely, by any scientist.

not rated yet May 08, 2017
3 years is not that long at all for large animal trials, and the price tag is not unusual either, for the researches, equipment, food animals and the space to do it all in. And pheromones are not going to cut it. (although the jealousy and arrogance are certainly inherent dangers in this field)

They are talking about transitting a genetic fix to the pancreas, and to do that they have to infect it with some form of a controlled disease that will transcribe the gene into the pancreas cells and then be easily cured. I am interested in seeing how this plays out, it is about Time we actually started Curing things rather than just Treating them forever.
1 / 5 (1) May 09, 2017
I do not dispute the commonness of the waste, just the simplicity that requires it. The cause of diabetes is a human pheromone coupled w/ pheromone receptivity from lacrimation. This is why pheromone pathways are involved, why the similarities with animal pheromone reception, etc. About half the time, the diabetic is rebuffed for some infraction at the beginning of disease. Measurable quantities of the pheromone should be obtainable at that time. Then find emitters and get them to stop, perhaps by hygiene changes or some other intervention or alternatively, find the poisonous pheromone receptive and try to diminish their receptivity, e.g. prevent emotional lacrimation, alter pheromone receptor proteins &/or ions in emotional lacrimal fluid to alter pheromone receptivity. Or just advise them to stay away from poisonous emissions somehow. Pheromone detectors might be fashioned to warn people at least or even to automatically turn on oscillating fans to break pheromone plumes.

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