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With Gene Therapy for Diabetes, San Antonio Researcher Eyes Funding

Xconomy Texas — 

San Antonio — Most diabetes treatments work by giving the body the insulin it needs to break down sugar. But that approach deals with the symptoms of diabetes. In recent years, scientists and companies have taken aim at the root cause of the condition by attempting to stimulate or replace the cells in the pancreas responsible for producing insulin in the first place. One of them is a San Antonio researcher hoping to use gene therapy—a potentially one-time, long lasting treatment—to do the trick.

When cells in the pancreas, known as beta cells, either get destroyed by the immune system or stop producing enough insulin, the result is type 1 or type 2 diabetes. Companies large and small—-from European diabetes drug giant Novo Nordisk to privately held startups ViaCyte, of San Diego, and Semma Therapeutics, of Cambridge, MA—want to engineer stem cells that develop into pancreatic beta cells to help a patient produce insulin.

Other researchers, such as Bruno Doiron, a scientist and assistant professor at the University of Texas Health Science Center at San Antonio, have different ideas. Doiron has developed an injectible treatment consisting of three molecules— glucokinase, a second that targets a protein known as PTP1B, and a third that targets a protein called Pdx-1, a so-called transcription factor that regulates genes—that, when infused into the body, are meant to help stimulate the formation of new beta cells. Doiron has tried the method on mice, and based on some encouraging early results, intends to move the work forward through a startup company.

“You have to prove you can translate that to a large animal model,” he says.

The San Antonio company, Syner-III, got its name because of the “synergistic” use of three molecules to generate the beta cells, he says. Those molecules are administered via a gene therapy procedure: they’re stuffed into a modified virus and injected directly into the pancreas in a one-time treatment, where they are meant to stimulate beta cell production. The work was published in the peer-reviewed journal Current Pharmaceutical Biotechnology in 2016.

Doiron hopes to raise as much as $10 million to complete preclinical testing.

Others, including Novartis, are considering different ways of boosting beta cell production. Researchers from the Swiss company published findings in Nature Communications that showed a group of compounds called aminopyrazines could be packed into a pill and similarly lead to more beta cells, and more insulin, in mice. Such attempts are fraught with failure, however. In an article on its own website, Novartis notes that researchers have succeeded in producing beta cells in mice many times, but haven’t been able to reproduce those results in humans.

The potential payoff, however, is huge. Some 29.1 million Americans have diabetes, and 1.25 million of them have type 1 diabetes, according to the American Diabetes Association. Doiron believes the therapy may be able to help both types. While stem cell research has had its share of failures and competition continues to increase in insulin therapy—such as pumps that automatically deliver the treatment—Doiron says a gene therapy, if successful, could result in a longer-lasting, more effective treatment.

“When I use your own body to produce medicine, that drastically changes the field,” he says.