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Type 1 Cured in Mice

May 6, 2017

Ralph DeFronzo and his researchers at UT Health at San Antonio announced that they have cured type 1 diabetes.

Researchers think they have found a way to trick the body into curing type 1 diabetes that may also have a great impact possibly for type 2 diabetes. Even though it was only in mice, this could be very positive, even with years of testing still remaining. Doctor Ralph DeFronzo, chief of the diabetes research at the UT Health Science Center at San Antonio, says that this way of doing a gene transfer can wake up cells in the pancreas to produce insulin.


The immune system of a person with diabetes kills off useful “beta” cells, but the researchers say they have found a way to make other cells in the pancreas perform the necessary work. Their approach, announced earlier this month in the academic journal Current Pharmaceutical Biotechnology, not only would have implications for type 1, but also could help treat the far more common type 2 diabetes.

The researchers have cured mice, which are genetically similar to people but different enough that new rounds of animal testing are needed before human trials can begin. This approach is sure to attract skeptics, in part because it is a significant departure from the many other attempts at curing diabetes, which typically involve transplanting new cells and/or suppressing the immune system’s attempts to kill off useful ones.

By contrast, “we’re taking a cell that is already present in the body and programming it to secrete insulin, without changing it otherwise,” said DeFronzo.

Diabetes is a disease characterized by a person’s inability to process carbohydrates, a condition that if untreated can lead to often-catastrophic health consequences.

The core problem is insulin. Most people naturally secrete that substance when they eat something with carbohydrates, such as bread, potatoes and candy bars. Insulin acts like a concierge that escorts the sugar from the bloodstream into the cells, providing the cells with the energy to function. In most people, the body is continually monitoring blood sugar and producing insulin as needed.

In type 1 — the type the researchers studied — the body has simply stopped producing insulin. This type often manifests in children, though it can sometimes develop in adults as well.

The researchers used a “gene transfer” technique on mice, delivered via a virus that activated insulin production in cells already in the pancreas — for instance, those that produced certain enzymes. DeFronzo added that, “We’re not fundamentally changing the cell, we’re just giving it one additional task.”

The mice immune systems did not attack the new insulin-producing cells. Most important, according to the findings: The cells produced the right amount of insulin: not so much that they sent a mouse into a blood sugar free fall, not so little that blood sugar levels stayed high. The mice have shown no sign of diabetes for more than a year, according to the findings.

Quite a bit of work remains before testing will start on people. If they can raise enough money — they estimate $5 million to $10 million — they can proceed to testing on larger animals, such as pigs, dogs or primates, a next step that would be planned in conjunction with the U.S. Food and Drug Administration. They hope to start human trials in three years.

DeFronzo and Dr. Bruno Doiron said they expect skepticism but said much of it will be driven by how unconventional their work is. Doiron added that, although the technique is unconventional in the context of diabetes, using a virus to deliver a gene transfer is an established technique, having been approved dozens of times by the FDA to treat diseases.

The mice in the study have been diabetes-free for more than a year with no side effects, according to UTSA. It will take a few more years of research (and money) before testing is available for humans, but DeFronzo and Doiron received a patent in January and hope to have human trials up and going in three years.

Practice Pearls:

  • A diabetes cure could take a number of years to get FDA approval.
  • The study first has to be proven safe on larger animals than mice.
  • The cure for type 1 diabetes seems to be getting closer.