Diabetic Gene Therapy Proves Promising in Mice

Diabetic Gene Therapy Proves Promising in Mice: Research can be a protracted effort with baby steps resulting in new findings that simply ask new questions. One recent discovery indicates the potential to control diabetes independent of weight. As with most research this is preliminary with more research needed.

According to ScienceDaily.com, “Researchers have found that even a very little bit of the fat hormone leptin goes a long way when it comes to correcting diabetes. The hormone controls the activity of a gene known as IGFBP2 in the liver, which has antidiabetic effects in animals and could have similar therapeutic effect in humans, according to a report published by Cell Press in the January issue of Cell Metabolism.”

Jeffrey Friedman of Rockefeller University is quoted by the online resource as saying, “It was surprising to me how potent leptin was in treating diabetes. It had a highly significant impact at plasma levels that were undetectable.”

Leptin provides an interesting yet helpful side effect, “Leptin also causes marked weight loss, which by itself can improve diabetes. To get around that issue in the new study, Friedman and his colleagues first identified the lowest dose of leptin that could correct insulin resistance and diabetes without leading animals to eat less or lose weight,” according to ScienceDaily.com.


In essence researchers were already aware of the fact that this hormone causes weight loss, but by significantly reducing the dose in research subjects (animals) they further found that even the small doses had a profoundly positive effect on diabetes. The report further declared, “Earlier studies had shown that leptin treatment effectively corrects high blood sugar and insulin levels in leptin-deficient mice and humans. Leptin’s usefulness as a therapy has also been shown in some clinical settings, in people with rare metabolic disorders. But it wasn’t clear exactly how the hormone produced in fat tissue acts to improve diabetes.”

In a practical application researcher were led to the IGFBP2 gene. The ScienceDaily.com report states, “Treatments designed to increase IGFBP2 expression in obese and diabetic mice reversed their diabetes. Further study showed that animals treated with the protein responded to insulin three times better than untreated ones.

“They also found that leptin-deficient patients do indeed have lower blood levels of IGFBP2 at baseline and that those levels can be raised with low-dose leptin treatment.”

As mentioned earlier this discovery may sound amazing on the surface, but in order for it to eventually lead to a medical therapy further clinical research will be needed to answer the new questions raised from this initial research.

To immediately begin marketing this as a cure-all for diabetes may be irresponsible because while all indicators point to effective control, the research has yet to extend to humans and side effects have not been noted in long-term use. In fact, “Friedman said that future experiments in mice lacking IGFBP2 altogether are needed to confirm that the protein is required for leptin’s antidiabetic influence. Now that they know that very high levels of IGFBP2 can act to improve diabetes, they’ll also need to explore the effects of normal physiologic levels,” said ScienceDaily.com.

In other words there will still be rodent research before it ever gets to human clinical trials. The most important ‘next’ finding will simply be to replicate the findings of this initial, and compelling research and answer as many new questions as possible in the process.