Gene therapy is not a new approach when it comes to treating type 1 diabetes. Scientists have been experimenting with many different options in order to stimulate the body to once again produce its own insulin and reduce or eliminate the need for insulin injections. However, some of the problems that scientists often encounter when introducing new cells into the body are that patients typically require immunosuppressant drugs which can lead to a variety of complications, the body rejects the cells over time, or the cells stop working. Finding a long-term, effective solution has been challenging.
Scientists are making strides in their efforts, though. A recent study examined the potential of using the gene-editing tool CRISPR to correct genetic mutations and create induced pluripotent stem cells that can be transformed into pancreatic beta cells. In mouse models, after the new cells were injected, mice achieved normoglycemia within a week and maintained this status for at least six months.
This approach has not yet been tested in humans, however, because it comes with its own set of challenges. First, the study was done using cells from patients with Wolfram syndrome, a condition that causes diabetes and deafness. This condition can be pinpointed to a single genetic mutation, whereas type 1 diabetes cannot. Type 1 diabetes has been tied to multiple gene mutations, as well as environmental factors. Gene-editing would have to be personalized for each individual, which could take a lot of time.
In addition, it could take billions of cells to effectively reverse diabetes in a patient, and generating this massive number of cells could take months, so it could end up being a long process to treat even one person. Plus, scientists are not entirely sure where the best place to transplant these cells is yet. They must find the spot where they will be most beneficial and able to carry out their intended purpose.
Another study using CRISPR technology is being conducted by a different group of researchers and is focused on using stem cells from the human cell line rather than from individual patients. This would make it easier to produce mass quantities of cells in a shorter period of time. It also would not require scientists to correct specific genetic mutations. CRISPR would be used to edit cells to prevent them from being attacked and destroyed by the body’s immune system.
A challenge with these approaches is that there are a lot of questions and regulations when it comes to gene-editing and using CRISPR on human subjects. Clinical trials are still in very early stages. Studies involving induced pluripotent stem cells are also relatively new in the United States. There is still a lot of work, research, and testing that needs to be done before gene-editing therapy could potentially be used on humans.
Diabetes Research Connection (DRC) will continue to follow these advancements and what they could mean for future diabetes treatment. DRC supports early-career scientists in contributing valuable discoveries and information of their own to the field by providing critical research funding. All projects funded by the DRC are focused on the prevention, treatment, and cure of type 1 diabetes, as well as minimizing complications and improving the quality of life for individuals living with the disease. Learn more and support these efforts by visiting https://diabetesresearchconnection.org.