It’s been a tough year, to say the least. In the beginning of the year, the Center for Disease Control and Prevention (CDC) gave out the stark news that type 1 diabetes (T1D) has gone up, to nearly 1.6 million Americans, from 1.25 million people; that’s a nearly 30% increase.
And then Breakthrough T1D and The Leona M. and Harry B. Helmsley Charitable Trust published a study that found that those people living with T1D now will pay a total of approximately $813 billion over their lifetimes in medical and in lost income and productivity costs. For an individual, that works out to nearly $500,000 per person over the course of a lifetime.
Then came COVID-19.
Without a doubt, the uncertainty and unprecedented challenges of the last year have left many of us feeling anxious, stressed, and overwhelmed.
But there’s news, as well, that we can be proud of. Really proud of. Take a look below.
1. Young Adult Reversed T1D + Clinical Trial of Baricitinib
If you haven’t already heard, T1D was reversed in a young man. I’ll say it again: T1D was reversed in a young man. This was a precision medicine treatment that corrects the effect of a genetic mutation, and the young man has been off insulin treatment for more than 2 years.
The treatment is a JAK inhibitor. JAK inhibitors are a type of therapeutic that do what the name says—stop or inhibit the activity of a JAK protein in your body.
JAK inhibitors are also being researched by Breakthrough T1D-funded scientists, Thomas Kay, M.B.B.S., and Helen Thomas, Ph.D., who found that blocking the protein may work in slowing down or preventing beta cell loss. They now have a clinical trial, funded by Breakthrough T1D, to find out if a JAK inhibitor, called baricitinib, will preserve beta cell function in children and young adults with recently diagnosed T1D.
This is the first time that a JAK inhibitor is being tested in type 1.
It is a bench-to-bedside research project—that has gone from basic research at the laboratory through a clinical trial in people with T1D—that Breakthrough T1D has had a hand in for more than a decade.
2. Drug Delays T1D Onset by Nearly 3 Years
A follow-up on the clinical trial testing the drug teplizumab—a therapy that blocks the blood marker CD3, which activates immune cells—demonstrated that it could delay the onset of T1D for nearly three years in people almost certain to develop the disease—adding a year to the previous results. This was the first ever study in humans to show a delay in the onset of type 1.
A submission to the U.S. Food and Drug Administration (FDA) is complete, and we’ll know if teplizumab is approved for the delay of clinical T1D in at-risk individuals in a year. If successful, we will be moving ever closer to a world without this disease. Teplizumab is also being tested in a phase III clinical trial in people who have been recently diagnosed, headed by Provention Bio, a company with an investment from the Breakthrough T1D T1D Fund.
Teplizumab could become the first disease-modifying therapy approved for T1D.
Breakthrough T1D had a hand in the development of teplizumab from nearly the beginning, from the Career Development Award given to Kevan Herold, M.D., in 1988-1990, who showed that he could prevent autoimmune diabetes with an anti-CD3 non-activating antibody (which, later, became a humanized version, teplizumab), to clinical trials in recent-onset disease published in 2002, 2005, and 2013 and a phase III trial run by MacroGenics (it was called MGA031 at the time), who received a Breakthrough T1D Industry Discovery and Development Program grant from 2006-2011, to identifying who is likely to respond to teplizumab.
3. Special Diabetes Program
The Special Diabetes Program (SDP) has received several patches of funding this year while Congress has been working on a longer-term renewal of the program. Recently, the Breakthrough T1D Advocacy team had WONDERFUL news to share! Congress passed a 3-year, $150 million per year, renewal of the SDP—committing a total of $450 million for T1D research! This is a big, big win!
The T1D community has benefited immensely from the SDP since it was first enacted in 1997, and the program complements many of Breakthrough T1D’s research priorities. The SDP has helped scientists make significant advances, including artificial pancreas technology, beta cell replacement techniques, improvements to continuous glucose monitors (CGMs), and treatments for T1D complications like kidney and eye disease. These advancements bring us closer to cures and, in the meantime, make it easier to lead healthy lives with T1D.
We are grateful to our countless volunteers for helping us achieve this monumental accomplishment. Join us now in saying ‘Thank You’ to Congress today! And stay tuned for next steps!
4. Screening for T1D-Related Antibodies
Breakthrough T1D has launched a brand-new screening initiative for T1D, called T1Detect, designed to make early detection of T1D easier and more accessible to a broad population.
JDRF-funded scientists have discovered that having 2 or more specific autoantibodies—antibodies that are directed toward your own body—means that you have an almost 100% chance of developing T1D. We can screen for these autoantibodies, and Breakthrough T1D-funded studies have shown that screening followed by close monitoring can help decrease diabetic ketoacidosis (DKA), which can be life threatening, and expands the pool of qualified participants for future trials of disease-modifying therapies. Early detection also enables families to prepare for the onset of clinical disease.
For more information about T1Detect and how to obtain an at-home testing kit, visit www.breakthrought1d.org/t1detect.
5. Insulin Activity
In December 2017, the T1D community got a jolt after a publication that tested the insulin concentrations in 18 vials, said that “none of the vials met the minimum labeled concentration standard.” In other words, insulin was not at the recommended dose; in fact, the percentage of insulin was around 40% (as opposed to a minimum required dose of 95%). So Breakthrough T1D, the American Diabetes Association (ADA), and The Leona M. and Harry B. Helmsley Charitable Trust issued a request for proposals (RFP) to verify the findings.
JDRF-funded researchers tested 9 insulin formulations, purchased at 4 pharmacies within 5 geographic locations in the U.S., and the results are out: All of the insulins purchased contained the expected quantity of active insulin. (Can we all say, “Phew!”) This study brings welcomed news to the T1D community and should ease any remaining doubt about the quality of our insulin supply.
6. Regulatory Approval of Several T1D Therapies and Technologies
We fund research to enable the development of new therapies and technologies to make day-to-day life with T1D easier, safer, and healthier, until we can find cures or prevent this disease. In 2020, we got a lot to be thankful for.
Artificial Pancreas Systems
- The second artificial pancreas system—the Tandem t:slim X2™ insulin pump with Control-IQ™ technology—ushered us into the New Year, but it was only available for people 14 years and older. In June, however, the FDA expanded the device for children ages 6 and older.
- The Medtronic MiniMed™ 780G artificial pancreas system was approved in Europe for people ages 7-80 years old. This is an update on the 670G, which was approved in 2016. In the pivotal clinical trial, the time-in-range was 75%, helping those with T1D maintain more consistent and healthier glucose levels. (In the United States, the MiniMed™ 780G system not approved for sale, yet.)
- The Medtronic MiniMed™ 770G artificial pancreas system—a Bluetooth-enabled version of the 670G—was approved by the FDA for children ages 2 to 6 in the United States. It is the first marketed device that can automatically adjust insulin delivery based on the continuous glucose monitoring (CGM) values for children in this age range.
CGM
- The FDA authorized the Abbott FreeStyle® Libre 2 System, a 14-day CGM that transmits data every minute, for adults and children age 4 and up in the United States. (It was approved in Europe in late 2018.)
Insulin
- Mylan and partner Biocon obtained FDA approval for Semglee™, a long-acting insulin, for adults and children with T1D (and type 2 diabetes), based on clinical trial data of Semglee versus Lantus. (Semglee has the same structure as Lantus.) The approval brings an affordable treatment option for people with diabetes.
- The FDA has approved Lyumjev™ in adults with T1 and T2 diabetes. As a rapid-acting mealtime insulin, Lyumjev controls high blood-sugar levels after meals in adults with diabetes.
7. Predicting Which Children Will Develop T1D
What if there were a way to more precisely estimate future T1D risk? Breakthrough T1D-funded scientists in The Environmental Determinants of Diabetes in the Young (TEDDY) study developed a model that can help predict which children will develop T1D during the first 10 years of life.
This can guide families regarding the risk of impending onset of T1D in their child, which can, among other things, prevent diabetic ketoacidosis (DKA)—averting the treatment costs and distress that it brings. It can also greatly improve the costs of early clinical trials, such as those testing expensive vaccines, by reducing the number of participants needed.
8. A Potential First-in-Class Adjunct Therapy
A JDRF-funded clinical trial by vTv Therapeutics provided evidence that treatment with TTP399—an oral therapy to be used in conjunction with insulin—resulted in significant improvements in HbA1c with reduction in insulin dose and, importantly, without increasing the risk of life-threatening events associated with T1D: diabetic ketoacidosis (DKA) and severe low blood sugar.
TTP399 is a glucokinase (or GK) activator. GK acts as a key regulator of sugar levels in the body. If blood-sugar levels are deemed too high, activation of GK has been shown to increase glucose utilization, which in turn lowers glucose levels in the blood.
The next step: A phase III clinical trial.
9. England to Provide Pregnant Women with CGM Technology
It’s a milestone for pregnant women with T1D and Breakthrough T1D: the National Health Service (NHS) England and the National Institute Health and Care Excellence (NICE) will provide CGM devices to pregnant women with T1D.
This is something that Breakthrough T1D can be proud of—evidence for it came from the JDRF-funded CONCEPTT study, which proved that CGM use during pregnancy improves the health outcomes for both mothers and babies and reduces the cost for neonatal hospitalization, an otherwise frequent occurrence.
10. Europe Supports Autoantibodies
It was 2015—five years ago—that three organizations (JDRF, the Endocrine Society, and the American Diabetes Association) published a statement urging the adoption of a staging system of T1D that uses islet autoantibodies as biomarkers. (Biomarkers, in this sense, are indicators that a person is developing T1D but is still without symptoms.)
In the spring of 2020, the European Medicines Agency (EMA) presented a letter of support to the Critical Path Institute’s T1D Consortium—who Breakthrough T1D and like-minded organizations have partnered with—to facilitate the development and validation of autoantibodies for T1D for regulatory use. This regulatory endorsement will help provide sponsors with the confidence to use autoantibodies in clinical trials evaluating novel therapies focused on the delay and/or prevention of T1D.
One Additional Accomplishment: Breakthrough T1D Turned 50!
It was 1970 and Lee Ducat’s son had just been diagnosed with a disease she had never heard of: juvenile diabetes (now known as type 1 diabetes or T1D; it was called “juvenile diabetes” because it was believed it only affected children).
After a doctor told her that with enough money, scientists could cure the disease impacting her son. So, starting with one cocktail party on May 21, 1970, she aimed to raise enough money to support T1D research—to help her son and every child affected.
“It was on that evening that the Juvenile Diabetes Foundation was born,” says Lee.
At the same time, in New York, another family was following a parallel course. Carol Lurie was seeking out families who also were struggling with a diagnosis they were told would last a lifetime. Her son was 10.
Together, the two became an unstoppable force. They were two moms who stood up, joined forces, and brought others together into what is now a growing, supporting community. They wanted to make the world see how T1D affects those impacted by the disease. They wanted to form a community that would improve diabetes management and support research that could cure T1D.
