Emily Mullin wrote on July 10, 2023, Wired in an article titled “A One-Time Shot for Type 2 Diabetes? A Biotech Company Is On It.” The following is a summary of her article. As in the past, I am interested in all things diabetic because #1 I am a diabetic, and #2 47% of us on dialysis are also diabetic.
Fractyl Health, a biotech company based in Massachusetts, is in the early stages of developing a one-time gene therapy treatment for type 2 diabetes and weight control. This therapy could provide an alternative to drugs like Ozempic and Wegovy, which require weekly injections to control blood sugar and body weight.
Currently prescribed medications like Ozempic and Wegovy mimic a human hormone called GLP-1, released in response to eating, which cues the pancreas to produce insulin, regulating blood sugar. These drugs also interact with parts of the brain involved in appetite, signaling fullness and thereby assisting in weight loss. However, these medications can have unpleasant side effects, and their efficacy drops off if a patient misses a dose or discontinues usage after reaching weight-loss goals.
Fractyl’s innovation seeks to solve these problems by delivering a one-time treatment that could have lasting effects for years. The therapy involves introducing an artificial gene to the pancreas that continuously produces the GLP-1 hormone, removing the need for regular injections. The delivery method involves using inactivated viruses to transport the therapeutic gene to the pancreatic cells, leveraging the viruses’ innate ability to deliver genetic material to cells.
While this therapy is currently only in the experimental stage, with tests having been conducted on animals, the initial results show promise. Using mice bred to develop type 2 diabetes, the researchers found that a single infusion of the gene therapy could reduce fasting blood sugar by 70%, compared to a 64% reduction by semaglutide injections. Separate tests also indicated that the therapy reduced body weight in mice by 23%.
The delivery method for this therapy involves an endoscopic procedure with a thin needle attached to a catheter, which is threaded down the throat and into the GI tract. Initial safety tests have been performed on pigs, which have a pancreas anatomically similar to humans, and no adverse side effects were reported.
However, this therapy raises some concerns, particularly around the potential for inflammation in the pancreas and the high cost of gene therapies. Dr. Daniel Drucker, an endocrinologist, and professor of medicine at the University of Toronto, is skeptical about directly injecting therapy into the human pancreas due to its fragility and importance. In addition, existing gene therapies on the market are priced quite high, with treatments for conditions like beta-thalassemia and hemophilia B costing millions.
Maria Escobar Vasco, an endocrinologist and diabetes expert at UT Health San Antonio, acknowledges the potential of the therapy but insists on the need for extensive testing to ascertain its safety. The first human trials are slated for the end of 2024.
In the meantime, other alternatives to injection-based therapies are being explored, including semaglutide in pill form, which may offer more convenience. Whether Fractyl’s gene therapy will result in similar side effects as current drugs is yet to be determined, but if it proves to be effective and safe, it could revolutionize the management of diabetes and weight control.
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