Clinical Trials and the Type 1 Diabetes Cure
WRITTEN BY: Stephen Gitelman, MD
I am often asked the question, “Where is the cure for Type 1 diabetes (T1D)?” For those with long-standing diabetes, we are very close to replicating insulin producing beta cell functionality or the actual replacement of those cells — either with closed loop systems with continuous glucose sensor driving an insulin pump, or use of replacement beta cells derived from stem cells.
However, as a Pediatrician, I think the ultimate cure for T1D will be prevention. Why can’t we screen and predict who is at risk, and then prevent someone from getting Type 1 diabetes in the first place?
It turns out that T1D occurs in about 1 in 300 people in the general population, but if you already have someone in your family with diabetes, like a brother or sister, then the risk jumps to a 1 in 20 chance of developing Type 1 diabetes. This is why researchers in an NIH sponsored international research effort called TrialNet have been focusing prevention efforts on families with at least one T1D.
T1D results from both underlying genetic risk and environmental exposures, but researchers are still working to determine these specific factors. Thanks to some of this work, we now have the ability to predict who will get T1D, in some cases as long as 10-20 years before it happens (see figure). Researchers use three different pieces of information for prediction. First, we look at the immune system. This is done with a simple blood test, measuring up to five different autoantibodies that the immune system might produce against beta cells. If no abnormality is foun Continue reading