Thanks to the efforts of our donors and volunteers, we have made a series of top advances in 2019 type 1 diabetes research. We will continue our fight to cure type 1 diabetes (T1D) and keep our community healthy, until we reach a day when all of this is history. Read on to discover some of the most exciting and innovative progress for our community.
Approval of Two Drugs in Europe
Do you think that just providing optimal insulin is the answer for people with T1D? Think again. Millions of people living with the disease are still at risk for dangerous blood-sugar highs and lows. A couple of years ago, Breakthrough T1D funded a clinical trial that showed that a drug called Zynquista™ could help diminish the risk of blood-sugar swings and, this year, we got some amazing news: Zynquista™ and Forxiga®—both SGLT inhibitors—were approved by the European Commission for T1D, as an adjunct to insulin. Their approval provides another option to improve daily blood-sugar management and is a big win for the T1D community in Europe.
Immune Therapy Delays T1D for More than Two Years
For the first time ever, an immune therapy treatment was able to significantly delay—for over two years—the onset of T1D in participants with a high risk of developing the disease. The drug, called teplizumab, targets CD3, a blood marker that activates the immune system’s T cells, to suppress the autoimmune response. Breakthrough T1D funded one of the first clinical trials of this drug, results of which were integral to the teplizumab prevention trial, and funds TrialNet, who conducted the study. Teplizumab is now being tested in a phase 3 clinical trial in people recently diagnosed with T1D. If successful, this could become the first immune therapy approved for this disease.
Powerful Proof-of-Concept
A clinical trial in newly diagnosed adults showed that a drug called verapamil helps to slow the progression of T1D. This provides a powerful proof-of-concept that controlling beta cell stress can slow down—or even stop—the progression of the disease. The investigator was able to take her research from the lab to the clinic so quickly because verapamil was approved by the FDA in the 80s, and is widely available to treat high blood pressure. This repurposing of a drug for T1D provides an accelerated path to clinical testing and to people with the disease.
Stem Cells to Insulin-Producing Beta Cells
Beta cell replacement therapies aim to provide insulin on demand from cells implanted in the body. Today, this can be done using islet cells from donors, but only a small fraction of people with T1D benefit. One potential method is to produce an unlimited supply of beta cells from other cell types, and Breakthrough T1D-funded scientists have introduced a new step in the process, which results in beta cells responding to blood sugar more like human beta cells. This discovery sets the stage for probing unexplored aspects of beta cell function and will, hopefully, lead to a breakthrough for people with the disease.
Cancer + T1D = An Extraordinary Collaboration
A subset of people with cancer who receive certain immune therapies go on to develop T1D or other autoimmune diseases. We want to know why. Breakthrough T1D and The Leona M. and Harry B. Helmsley Charitable Trust have teamed up with the Parker Institute for Cancer Immunotherapy to better understand this phenomenon. They will follow 1,600 patients receiving cancer immune therapy to better understand who is at risk and why some people go on to develop autoimmunity—with the ultimate goal to develop preventive therapies for the disease.
Making Open Protocol Mainstream
The Breakthrough T1D artificial pancreas open protocol initiative—which allows people to connect to their preferred devices, even if made by different manufacturers—is working. Breakthrough T1D introduced the open protocol initiative in October 2017, and, now, there are nearly 10 organizations that are pursuing open protocol approaches. We are thrilled to see manufacturers moving to allow communications between different diabetes devices, which will provide people with diabetes more options, accelerate innovation and, most importantly, improve diabetes outcomes. The FDA has also authorized the first interoperable insulin pump that can digitally interact with devices made by other manufacturers—a big win for our open protocol initiative, on the heels of approving an interoperable continuous glucose monitor (CGM) last year.
A Target for Kidney Disease
Diabetic kidney disease is responsible for more than half of all new cases of end-stage kidney disease, and there has been little in terms of biomarkers to tell who will get this disease. Breakthrough T1D-funded scientists, though, found 17 inflammatory proteins that were strongly associated with progression to end-stage kidney disease. Many of the inhibitors affecting these pathways are currently being tested in clinical trials for other chronic inflammatory disorders. Further testing these compounds in diabetic kidney disease may offer hope to people living with T1D that they may thwart end-stage kidney disease altogether.
As a community, we want to grow stronger and healthier together. As an organization, we will push harder, faster and with dedicated focus to get us to cures. Because we are, with you, part of the T1D community. A cure for T1D is a cure for all of us.
