Each day, my scientific team runs through dozens of scientific studies on Type 1 diabetes. It’s important to deduce what hasn’t panned out and why. We recently reviewed a Bench-to-Clinic Symposium on many of the immunotherapy trials used in man for the treatment of Type 1 diabetes; the goal of such an examination is to figure out the steps needed to take a new treatment from the experimental stage to everyday use. The publication we reviewed listed 18 different studies, all of which reversed diabetes in mice, but failed to do so in man. We have carefully looked at each study listed, and many more, to evaluate what we can learn.
This is not just an academic exercise to us. As we hear from so many patients who live with Type 1 diabetes and are working so hard to maintain their glucose levels, we know the gravity of our mission. I just saw one of my long-term patients who experienced a hypoglycemic seizure; she broke her ankle while her blood sugar levels were low. Another of my patients is now in Intensive Care when he developed a kink in his pump insertion cannula, which prevented insulin delivery. I hear from the parents of young children with Type 1 diabetes, who haven’t slept through the night in years for fear of their children going low. I have promised a young patient I will make him insulin independent before he is an adult, and the only way to do that is to learn from what hasn’t worked.
In a way, when we sift through these past studies we find that we’re dealing with physiological apples and oranges. Type 1 diabetes in mice is far different from diabetes in humans, and the Islets of Langerhans, which contain insulin-producing beta cells, are much more structurally complex in humans than in mice. The human islet contains a higher percentages of cell types other than beta cells; these are there to sustain man through times of feast and famine and maintain glucose levels constantly within a normal range. For example, performance artist David Blaine was able to suspend himself in a glass box over the city of London without eating for 44 days, and he had a perfect blood sugar before and after his fast. Not so true for the mouse who eats constantly and has a greater percentage of beta cells that turn over fast.
Many diabetes clinician researchers have come to the conclusion that Type 1 diabetes in humans may not be simply a disease of autoimmunity, like it may be in rodents; even with immune treatment we find that beta cells don’t increase in numbers. Human studies that used only immune tolerance agents have been proven to slow decline in beta cell mass, but patients who are treated still end up producing insulin at a rate that is 5 times lower than average.
Many researchers have concluded that insulin independence among Type 1 patients will require both an immune agent to block the attack on beta cells and a beta regeneration therapy.1 To date, this combination hasn’t been tried, but our team at Perle Bioscience plans to launch the first study later this year, pending FDA approval.
Our mission is clear and commitment unwavering to our patients. We are learning from all of the successes and lack of successes in Type 1 diabetes and believe that insulin independence will happen sooner than believed possible.