Dr. Desmond Schatz
Recently, Kelly Close and Melissa Tjota, another diaTribe reporter, were fortunate to interview one of the world’s leading experts on type 1 diabetes: Dr. Desmond Schatz, medical director of the University of Florida’s Diabetes Center of Excellence. On top of leading the TrialNet center at the University of Florida, he is also a member of the expert advisory board for the SEARCH Study (diabetes in children), Stop 2 Diabetes, TODAY Studies (in type 2 children), INIT Study (in type 1 children), and DiaPep Study. His research interests focus on the prediction, natural history, prevention, and management of type 1 diabetes in children and adolescents. During our conversation, Dr. Schatz shared his thoughts on type 1 diabetes and children, TrialNet, challenges in designing clinical studies, technological advances in diabetes, and the shortage of endocrinologists and pediatric endocrinologists. Most of the discussion focused on children and type 1 diabetes.
type 1 diabetes and children
Kelly Close: Dr. Schatz, we really appreciate your taking the time to speak with diaTribe. Could you give us a broad overview on the state of the type 1 diabetes epidemic?
Desmond Schatz: Sure. The number of people with type 1 is increasing rapidly. To put some specific numbers out there, the incidence of type 1 diabetes is increasing worldwide at a rate of about 3% per year. In the United States, one in every 300 Americans has type 1 diabetes, and in Finland, one in every 123 children under the age of 16 has type 1 diabetes. In the late 1950s it was about half the current rate. Unfortunately, the increase in type 1 diabetes is predominantly seen in children that are under the age of 10 years. Clearly, we cannot afford to do nothing.
Kelly: These are astounding statistics. How would you say we are doing as a society in addressing this issue? There are several issues that need to be addressed: diabetes has an increasing incidence worldwide, it is being seen more in younger children, and intensive therapy may increase the risk of complications. The impact of intensive therapy on microvascular complications is well known, and very positive, but we still do not know about macrovascular complications. In addition, the benefits of therapy are not reaching the majority of children and people with type 1 diabetes. I don’t think that everybody has access to a pediatric diabetes team for management. I wish we could say that everybody was on basal-bolus therapy, but that is certainly not the case.
update on TrialNet
Melissa Tjota: Can you talk about your involvement in TrialNet? This is one of the most interesting areas of research for children with diabetes.
Dr. Schatz: The University of Florida is one of the 14 TrialNet centers in North America and there are another four centers internationally. We are doing studies aimed at halting the autoimmune disease and reducing beta cell destruction as well as looking at ways to prevent the disease. We are also doing research to discover the mechanisms that lead to type 1 diabetes. There are several studies that have been done in new onset cases. In a study presented at the last ADA meeting, the administration of anti-CD20 [a drug that suppresses the immune system] to new-onset patients was demonstrated to have a short term protective effect similar to anti-CD3 [another immunosuppressant] and for the GAD65 vaccine [another way to stop immune attack on the beta-cells]. Most of the effect that we are seeing in these studies is noted in the first three to six months and is preserved for 1-2 years. Editor’s note: See this issue’s Learning Curve for more information about CD-3, an immune system suppressing drug that may help to prevent type 1 diabetes.
Kelly: Do you think that the loss of effect after 1-2 years relates to dosing and that a higher dose may work longer?
Dr. Schatz: Well, let me tell you what I believe is the future of diabetes. The future will involve a combination approach, an approach that is directed against arresting the autoimmune disease process, preserving the pancreatic beta cell, and regenerating the beta cell. So, I don’t believe it is only a matter of dose. I think that you can probably refine it either by a change in dose or by re-dosing, but I honestly believe that a multi-targeted approach is the future to both prevention and cure.
Melissa: Could you give us some estimates of how close you think we are to having a solution for each of those different approaches?
Dr. Schatz: The JDRF talked about the 1990’s as the decade of the cure and that was unfortunately not true. We have made tremendous progress over the past 20 years however. We now know that type 1 diabetes is an autoimmune disease, we have learned how to predict type 1 diabetes using auto-antibodies, and we have refined the prediction process by looking at a combination of antibodies, genes, insulin measurements, and glucose tolerance. Overall, we have become pretty good at predicting the disease in both relatives and also the general population Now, when you talk about each of these components, I think that we are seeing some small effect using mono-therapies with agents like anti-CD20, anti-CD3, and the GAD65 vaccine. We are making small strides, and as I said the next step is using combinations of therapies to make even greater strides.
Melissa: Are there plans to explore more therapies?
Dr. Schatz: The goal is to continue to do more studies to explore new therapies in type 1 diabetes, determine their safety, determine their efficacy, and then take them to prevention studies. I think that several groups are going to be looking at combinations of drugs using anti-inflammatory drugs, immuno-regulatory drugs, and tolerance-inducing drugs.
diabetes devices
Melissa: TrialNet has been incredibly involved in closed loop research. Could you give us your thoughts on the progress being made toward an artificial pancreas?
Dr. Schatz: I am not the authority on the artificial pancreas. That said, I have used and prescribed pumps and continuous glucose monitoring. I think that we are certainly making progress in that field and with the technology, but we are not there yet. A few years ago we were told that we would have the artificial pancreas within a couple of years. I think that there will certainly be a closed loop system, but whether or not it is a true artificial pancreas or an improvement on the current systems will have to be seen. Hopefully we will see this in the next decade or so. I believe that we will likely see an artificial pancreas before a biological cure. The hope is that we will cure diabetes, but it is certainly not going to happen tomorrow.
how close is a cure?
Kelly: Would you say that your opinion about a cure has evolved over the last couple of years? From a patient perspective, it has been amazing how fast things have been moving recently after how slowly things were moving a few decades ago.
Dr. Schatz: We are not as close to a cure as we would like to be. Certainly, a couple of the devices are encouraging, and we have improved the technology, but we are not there yet. I think technology is moving a little faster than the biology although we have been making progress in both regards.
looking forward to the next five to ten years
Melissa: Could you tell us the top research you are looking forward to seeing in the next five to ten years?
Dr. Schatz: I don’t want to be too specific other than to say that I believe we will use combinations of drugs akin to what we do with cancer, AIDS, and tuberculosis therapies in children and adults. Combinations in smaller doses will be more efficacious, synergistic, and safer.
