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One of the huge advantages of a stem cell agency like CIRM (not that there is anything out there quite like us, but anyway) is our ability to support projects as they progress from a great idea to a therapy actually being tested in people.
Exhibit A on that front came via a news release from ViaCyte, a company that is developing a new approach to helping people with severe Type 1 Diabetes (T1D).
Unlike type 2 diabetes, which is largely diet & lifestyle related and develops over time, T1D is an autoimmune condition where the person’s immune system attacks and destroys the insulin-producing cells in the pancreas. Without those cells and insulin the body is not able to regulate blood sugar levels and that can lead to damage to the heart, kidneys, eyes and nerves. In severe cases it can be fatal.
ViaCyte (which has been supported with more than $72 million from CIRM) has developed a pouch that can be implanted under the skin in the back. This pouch contains stem cells that over a period of a few months turn into insulin-producing pancreatic islet cells, the kind destroyed by T1D. The goal is for these cells to monitor blood flow and when they detect blood sugar or glucose levels are high, can secrete insulin to restore them to a safe level.
They tested this approach in 15 patients in a Phase 1 clinical trial in Canada. Their findings, published in the journals Cell Stem Cell and Cell Reports Medicine, show that six months after implantation, the cells had turned into insulin-producing islet cells. They also showed a rise in C-peptide levels after patients ate a meal. C-peptides are a sign your body is producing insulin so the rise in that number was a good indication the implanted cells were boosting insulin production.
As Dr. James Shapiro, the Chair of Canada Research and one of the lead authors of the study says, that’s no small achievement: “The data from these papers represent a significant scientific advance. It is the first reported evidence that differentiated stem cells implanted in patients can generate meal-regulated insulin secretion, offering real hope for the incredible potential of this treatment.”
And that wasn’t all. The researchers say that patients spent 13 percent more time in the target range for blood sugar levels than before the treatment, and some were even able to reduce the amount of insulin they injected.
Now this is only a Phase 1 clinical trial so the goal was to test the safety of the pouch, called PEC-Direct (VC-02), to see if the body would tolerate it being implanted and to see if it is effective. The beauty of this method is that the device is implanted under the skin so it can be removed easily if any problems emerge. So far none have.
Ultimately the hope is that this approach will help patients with T1D better regulate their blood sugar levels, improve their health outcomes, and one day even achieve independence from the burden of daily insulin injections.