Breakthrough T1D joined thousands within the diabetes community at the American Diabetes Association’s (ADA) 83rd Scientific Sessions, where hundreds of experts presented significant advancements in diabetes research that may prove to be pivotal in improving lives and finding cures for T1D.

Held June 23-26 in San Diego, the ADA Scientific Sessions is one of the most prominent opportunities for researchers, healthcare professionals, and industry members to learn about the latest in diabetes research, treatment, and care. Breakthrough T1D-supported research has been highlighted at the annual event since Breakthrough T1D started funding research nearly 50 years ago. This year, scientists presented 140+ studies—with Breakthrough T1D-funding at present or in the past—to encompass breakthrough clinical trials and significant research studies that are paving the way to novel treatments and technologies for T1D.

The ADA Standards of Care for Diabetes has been updated to include Tzield™ (teplizumab-mzwv) to delay T1D in at-risk individuals. Read more about Breakthrough T1D’s work—funding discovery research to clinical development to work with regulators—that brought Tzield to FDA approval.

There were many fantastic results that came through the ADA’s Scientific Sessions. Here is Aaron J. Kowalski, Ph.D., CEO of Breakthrough T1D, with the key takeaways from the conference, and there is a written summary of Breakthrough T1D highlights below.

Cures

Stem Cell-Derived Islet Replacement Therapy: Update on Vertex’s VX-880

Breakthrough T1D Research Area: Cell Therapies

Cell replacement therapies, including stem cell-derived islet therapy, were on fire, with Vertex Pharmaceuticals leading the way in clinical trials. Vertex launched its clinical trial of VX-880, a stem cell-derived islet therapy in T1D for individuals with hypoglycemia unawareness, in combination with immunosuppressive therapy to protect the cells from rejection, in the summer of 2021. To date, six participants have received this therapy, and two are now insulin independent, have met the primary endpoint of eliminating severe hypoglycemia events, and have exceeded the ADA target time-in-range (>70%). Based on these data, the phase I/II study advanced to Part C in full target dose.

In summary: VX-880 has the potential to achieve insulin independence, as well as eliminate severe low blood-sugar events and significantly improve blood-sugar control.

Breakthrough T1D Leadership

Vertex’s phase I/II clinical trial of VX-880 was pioneered by Doug Melton, Ph.D., whose years of Breakthrough T1D-funded research led to successfully transforming stem cells into beta cells in 2014, and a catalytic investment from the T1D Fund in Semma Therapeutics—a biotech company founded by Melton to develop a stem cell-derived islet therapy for T1D, which was acquired by Vertex Pharmaceuticals.

Islet Replacement Therapy: Update on Sernova “Cell Pouch” Therapy

Breakthrough T1D Research Area: Cell Therapies

Breakthrough T1D also heard from Sernova, who provided an update on the phase I/II clinical trial of their Cell Pouch™—an implantable device designed to form a natural environment and allow the survival and function of insulin-producing (islet) cells. This device uses islets from deceased donors. In the first cohort, six individuals have received the therapy, and five have remained insulin independent, going from 6 months to more than 3 years. Observations from the first cohort led to a higher-capacity Cell Pouch, which provides up to 56% more volume to accommodate more islets, and 5 of the 7 planned participants have been enrolled in the second cohort.

Breakthrough T1D continues to support Sernova to make their Cell Pouch part of the cures portfolio.

TYK2 Inhibitors: Not for Psoriasis Anymore

Breakthrough T1D Research Area: Disease-Modifying Therapies

TYK2 is a crucial signaling pathway that has a role in autoimmune diseases, including psoriasis, Crohn’s disease, and lupus. Moreover, a TYK2 inhibitor—which works by blocking the TYK2 protein and the inflammation that it brings—was FDA-approved in September 2022 for moderate-to-severe psoriasis. But, researchers asked, would a TYK2 inhibitor have similar effects on the development of T1D?

The study showed that a TYK2 inhibitor reduced diabetes incidence by up to 80%, decreasing the immune cells that cause T1D and increasing the immune cells that signal tolerance. Taken together, their findings demonstrate that TYK2 inhibition protects against beta cell stress and T1D development, in multiple preclinical models.

Breakthrough T1D Leadership

The results were presented by Breakthrough T1D-funded researcher Farooq Syed, Ph.D., who was a Breakthrough T1D fellow working with Breakthrough T1D grantee Raghu Mirmira, M.D., Ph.D., the ADA’s 2023 Albert Renold Award recipient. Co-authors on the abstract are Breakthrough T1D-funded researchers Decio Eizirik, M.D., Ph.D. and Carmella Evans-Molina, M.D., Ph.D.—ADA’s 2023 Outstanding Scientific Achievement Award recipient.

General Population Screening: It’s Time

Breakthrough T1D Research Area: Screening and Monitoring

An ADA/Breakthrough T1D symposium, moderated by Sanjoy Dutta, Ph.D., chief scientific officer at Breakthrough T1D, featured an update on screening and monitoring in Europe (Fr1da, Diabetes Prediction and Prevention (DIPP) Study) and the United States (Autoimmunity Screening for Kids, or ASK), which are now expanding globally. The session highlighted the progress we have made, such as the benefits of screenings, including a decrease in DKA at diagnosis and lower HbA1c levels versus children who had not been screened. The gaps to move general population screening into state and federal guidelines: Cost-effectiveness of screening, at what age(s) do you screen, and how do you monitor when a person has 2+ autoantibodies. Research in all of these is under way.

2+ autoantibodies—antibodies that are directed toward your own body—means you have an almost 100% chance of developing T1D in your lifetime.

Breakthrough T1D Leadership

As a result of decades of Breakthrough T1D-funded research, we can identify those at highest risk for developing T1D—2+ autoantibodies—and we have funded screening programs since they were first introduced in the late 1980s. More recently, Breakthrough T1D launched the T1Detect screening education and monitoring program to broaden screening awareness and access to the general population. The goal: Global universal screening, which is key to developing disease-modifying therapies—like Tzield™ (teplizumab-mzwv), FDA-approved to delay T1D in at-risk individuals—and, ultimately, preventing it entirely.

Improving Lives

Lower Your HbA1c Levels + Reduce Insulin Use ≠ DKA = Success!

Breakthrough T1D Research Area: Glucose Control

SGLT therapies can lower HbA1c levels but increase the risk of diabetic ketoacidosis (DKA). So far, they are only approved for type 2 diabetes. But what if you combined an SGLT therapy with another drug that could improve blood-sugar levels AND potentially reduce ketones? A Breakthrough T1D-funded phase I/II clinical trial wanted to find out if this combo could work for people with T1D. The results? Pretty amazing.

Diabetic ketoacidosis (DKA)—a life-threatening complication, typically resulting from not enough insulin in the body—is an abundance of ketones in the blood, which typically also happens when blood sugar is very high. With SGLT therapy, however, DKA presents with blood-sugar levels at normal or near it. Because of this, there will be a need to counteract the risk if SGLT is going to be approved for T1D.

The combination therapy of an SGLT inhibitor (dapagliflozin) and a glucagon receptor inhibitor (volagidemab) reduced average blood sugars, reduced total insulin use, and increased time-in-range (by >3.8 hours a day!), and there was no DKA or severe low blood sugar. This led Schafer Boeder, M.D., presenter on the clinical trial, to conclude: “This therapeutic combination has the potential to improve glycemic control and reduce insulin use, while mitigating ketogenesis in T1D.”

Breakthrough T1D Leadership

This study involved numerous Breakthrough T1D-funded researchers: Dr. Boeder, as well as Jeremy Pettus, M.D., and Justin Gregory, M.D.—two of his authors on the abstract.

Breakthrough T1D is now funding REMD Biotherapeutics, to run a clinical trial investigating the impact of volagidemab on rescue glucagon to treat severe low blood-sugar events.

“I literally wouldn’t be here doing what I’m doing if I didn’t have funding from Breakthrough T1D. I feel like I’m making a difference, and that’s huge. So having this funding has allowed me to focus on exactly what I want to do and keep doing it thanks to their support.”

– Jeremy Pettus, M.D.
Endocrinologist and Assistant Professor of Medicine
University of California, San Diego

Technology Updates

Breakthrough T1D Research Area: Artificial Pancreas / CGMs

A ton of presentations focused on the artificial pancreas, or automated insulin delivery (AID), systems and continuous glucose monitors (CGMs). Real-world results on two artificial pancreas systems—Omnipod 5 and Medtronic 780G—demonstrated that they significantly improved time-in-range, with averages of 69% and 70% for Omnipod 5 (using the most aggressive target, internationally and in the United States) and 80% and 86% for the Medtronic 780G (for all of the United States users and those with Medtronic’s recommended settings). Time-below-range was 1% for Omnipod 5, and 2.3% for the Medtronic 780G. iLet insulin-only bionic pancreas showed that it improved blood-sugar management independent of how much users interact with the automated insulin delivery (AID) device. And Senseonics presented data from its 365-day implantable CGM, showing that it performed as well as the 180-day sensor.

Breakthrough T1D Leadership

The iLet presentation involved research from several former Breakthrough T1D-funded scientists: Ed Damiano, Ph.D., CEO of Beta Bionics; Firas El-Khatib, Ph.D., co-founder and vice president, research & innovation, at Beta Bionics; and Steven Russell, M.D., Ph.D.

The First Study to Show Correlation Between Time-in-Range and Complications

Breakthrough T1D Research Area: Complications

Continuous glucose monitoring (CGM) metrics, such as time-in-range, have been proposed as new metrics to assess diabetes management. No longitudinal studies, however, have placed time-in-range and diabetic eye disease…until now.

Viral N. Shah, M.D., in a study funded by Breakthrough T1D, analyzed up to 7 years of retrospective CGM data; 92 of people were without a diagnosis of diabetic eye disease and 71 of them were. HbA1c of the diabetic eye disease was significantly higher than those without a diagnosis. But people with diabetic eye disease had an average time-in-range of 52%, while the control group had a time-in-range of 62%—meaning that every 5% point decrease in time-in-range was associated with a 16% risk increase in diabetic eye disease. Time-in-tight target range (70-140 mg/dL) and time above range (>180 mg/dL) were also associated with the disease.

What does this mean? 1. It’s the first longitudinal study to demonstrate the association between CGM metrics and complications. 2: Time-in-range could become closer to realizing its potential application in T1D management and clinical trials.

Breakthrough T1D Leadership

Dr. Shah is an important part of the University of Colorado-Breakthrough T1D-scientist atmosphere, which goes back to the 1980s. From George Eisenbarth, M.D., Ph.D., who came up with the staging system in 1986, and Kevin Lafferty, Ph.D., who had demonstrated that a second signal is required to activate immune cells, to the next generation of researchers. Co-authors of the abstract include Gregory Forlenza, M.D., who is an expert on artificial pancreas systems in youth, Sarit Polsky, M.D., MPH, a specialist in T1D and pregnancy, and Halis Akturk, M.D., an authority on checkpoint—the “brakes” of the immune system—inhibitor-induced T1D.

We also had the opportunity to hear about the Mary Tyler Moore Vision Initiative (MTM Vision), with S. Robert Levine, M.D., the husband of the late Mary Tyler Moore and creator of MTM Vision, Jennifer Sun, M.D., MPH, and Patrice Fort, M.S., Ph.D. The first part of this initiative is a new Diabetic Retinal Disease Staging System, to update the grading system that has not been changed for 50 years, despite the advancement of our understanding the disease and its presentation.

Dr. Fort is collecting and characterizing different stages of the disease, to do in-depth analysis of those tissues to identify new potential targets to be developed. Dr. Sun presented work to identify new clinical endpoints and biomarkers for better characterization of the progression of the disease. She is spearheading clinical trials that will take place at the beginning of next year.

Breakthrough T1D Leadership

Breakthrough T1D has supported eye disease research since its beginning, and has driven discoveries that have reduced the risk of blindness by 95%, including laser therapy and anti-VEGF treatments. In 2018, Breakthrough T1D and the Mary Tyler Moore and S. Robert Levine, MD, Foundation launched MTM Vision, an ambitious initiative to reverse diabetes-related blindness and vision-threatening eye disease, which afflicts millions of individuals worldwide, held in honor of Mary Tyler Moore’s contributions to diabetes awareness and research.

Novel Interventions for the Most Vulnerable

Breakthrough T1D Research Area: Psychosocial

Many Breakthrough T1D-funded researchers presented on psychosocial issues, including the stigma associated with diabetes, telehealth and diabetes distress, depression, and anxiety, and social inequities—economically vulnerable, minorities, food insecurity—in diabetes care, including Michael Harris, Ph.D., whose Novel Interventions in Children’s Healthcare (NICH) was among the winners of the Innovation Challenge.

The inaugural ADA Innovation Challenge was designed to connect diabetes leaders with investors to move the field forward.

Funded by the Helmsley Trust, Breakthrough T1D, and other like-minded organizations, NICH is an innovative program designed to integrate medical care with behavioral therapy to support at-risk youth and their families. It deploys interventionists to address social determinants of health for the most vulnerable, highest need youth with T1D—the 1% who are responsible for 25% of cost. They had fantastic results over the past years, significantly reducing DKA and diabetes-related admissions to the hospital and a drop in HbA1c.

You can view all of the oral and poster presentations on the Diabetes journal website.

The American Diabetes Association’s 83rd Scientific Sessions is here! Scientists will present the latest type 1 diabetes (T1D) research, from beta cell replacement to regeneration, and glucose and complications trials, all with the goal of improving lives for the T1D community.

Here are Breakthrough T1D-funded researchers Drs. Sarit Polsky, Riitta Veijola, Maria Golson, and Patrice Fort to share their key takeaways from day 3, with their commentary in the video and below:


Sarit Polsky, M.D., MPH
Director, Pregnancy and Women’s Health Clinic, Barbara Davis Center for Diabetes, University of Colorado
Area: Automated Insulin Delivery Use among Pregnant Women with Type 1 Diabetes—Past, Present, and Future

Managing T1D with pregnancy is difficult, and artificial pancreas, or automated insulin delivery (AID), systems—for the most part—are not tailored to pregnancy. There are significant changes to insulin requirements over the course of pregnancy, making it difficult for pregnant women to achieve blood-sugar targets. Continuous glucose monitors (CGMs) help women increase their time spent in the optimal, pregnancy-specific time-in-range, but even with CGM some pregnant individuals do not reach their targets until the last few weeks of pregnancy.

Presenting on AID systems in pregnancy were Helen Murphy, M.D., FRACP, Carol Levy, M.D., CDCES, Sarit Polsky, M.D., MPH, and Denise Feig, M.D., M.Sc., FRCPC. They discussed the commercially available system for pregnancy (CAM APS, the only available system for pregnant women), “off-label” AID systems (like Medtronic, Tandem, Insulet, and iLet—which are not approved for pregnancy), and those in clinical trials, including one that has a special pregnancy-specific algorithm, developed by Harvard researcher (and Breakthrough T1D-funded) Eyal Dassau, Ph.D.

There are still a lot of things in the future we have to figure out—What is the optimal time-in-range? Should it be different overnight or during the day? What are the barriers to automation, such as cost and skin issues? There are currently five studies underway, with results that will be published in the next year or two.

“We’ve made a lot of progress and it’s all very exciting,” says Dr. Polsky, “so we all have a lot to look forward to.”

Riitta Veijola, M.D., Ph.D.
University of Oulu
Area: Joint ADA/Breakthrough T1D Symposium—Global Perspective on Population Level Type 1 Diabetes Screening and Monitoring

Anette-Gabriele Ziegler, M.D., Cristy Geno Rasmussen, MPH, Ph.D., and Riitta Veijola, M.D., Ph.D., presented on global screening in Europe and the United States, in a session run by Breakthrough T1D’s own Sanjoy Dutta, Ph.D., chief scientific officer at Breakthrough T1D. Dr. Veijola, who has been working on the development of T1D in children for almost 30 years, summarized the screening process. In Finland—the country with the highest rate of T1D—the most common age when islet autoantibodies appear is around 1 year, for reasons that are being explored in current studies.

2+ autoantibodies—antibodies that are directed toward your own body—means you have an almost 100 percent chance of developing T1D in your lifetime.

A screening test can tell you if you have 2+ autoantibodies. If you do, blood-sugar levels need to be monitored for the onset of type 1. You can do this in multiple ways. The traditional way is a glucometer with strips. A more modern way is continuous glucose monitoring (CGM) technology, which gives you your blood-sugar levels every 5 minutes. Another measure is to test HbA1c, which can tell you about blood-sugar levels over the past 12 weeks.

Monitoring can help you avoid diabetic ketoacidosis (DKA)—a complication of T1D when there is too much blood sugar, that can lead to death if not caught early. Monitoring also gives you time to learn about T1D and an opportunity to take part in preventive treatment, like Tzield™ (teplizumab-mzwv), or a clinical trial. To learn more about screening and monitoring, go to our T1Detect program.

Maria L. Golson, Ph.D.
Assistant Professor, Johns Hopkins School of Medicine
Area: The Islet-Cell Interactome

Alpha cells! Beta cells! Delta cells! Oh my! Maria Golson, Ph.D., and the presenters in her session, Manami Hara, DDS, Ph.D., Danielle Dean, Ph.D., and Abdelfattah El Ouaamari, Ph.D., talked about the other cells and blood vessel architecture in T1D, and how they contribute to beta cell failure.

Dr. Golson presented on delta cells, which produce somatostatin, a hormone that inhibits insulin secretion and is also released in response to high glucose as the hormone helps regulate the gastrointestinal tract. Next up was Dr. Hara, who talked about the blood vessel system in the islets, who demonstrated that there’s more interaction than previously thought between the blood vessel tissue and islet tissue. Dr. Dean spoke about glucagon, which is secreted by the alpha cell, which is also important for regulating insulin secretion. Finally, Dr. El Ouaamari found that islets have different sensory neurons, which might explain some of the differences in diabetes incidence in males and females.

Islets were previously thought to be made up of different cells, each responsible for a singular function. Now, we see that these different cells communicate with and among each other. Additional study of these interactions could help illuminate a path to new research and therapies that act upon these intercellular actions.

Patrice Fort, M.S., Ph.D.
Associate Professor and Director, Biorepository and Resource Center, University of Michigan
Area: Creating a World without Visual Loss from Diabetes

Patrice Fort, M.S., Ph.D., presented on the Mary Tyler Moore Vision Initiative (MTM Vision), with S. Robert Levine, M.D., the husband of the late Mary Tyler Moore and creator of MTM Vision, and Jennifer Sun, M.D., MPH. The first part of this initiative, due to a workshop hosted by Breakthrough T1D in 2018, is a new Diabetic Retinal Disease Staging System, to update the grading system that has not been changed for 50 years, despite the advancement of our understanding the disease and its presentation. An article outlining the new Diabetic Retinal Disease Staging System is set for publication in the coming months.

The workshop also detailed the state of the science, and outlined scientific needs and opportunities, which helped establish a “roadmap” for next steps.

One of these, presented by Dr. Fort, is collecting and characterizing different stages of the disease, to do a very in-depth analysis of those tissues to identify new potential targets to be developed with pharma companies or other academic centers. Another one, presented by Dr. Sun, is new clinical endpoints and biomarkers for better characterization of the progression of the disease. She is spearheading clinical trials that will take place at the beginning of next year.

MTM Vision’s goal is to lead to the development of therapies that will reduce or reverse vision-threatening diabetic eye disease development and progression. Ultimately, it aims to create a world without visual loss from diabetes, honoring Mary Tyler Moore’s commitment to diabetes awareness and research.

The American Diabetes Association’s 83rd Scientific Sessions is here! Until June 26, scientists will present the latest type 1 diabetes (T1D) research, from beta cell replacement to regeneration, and glucose and complications trials, all with the goal of improving lives for the T1D community.

Here are Breakthrough T1D-funded researchers Drs. Martin Thelin and Robert Eckel to share their key takeaways from day 2, with their commentary in the video and below:


Martin Thelin, M.D., Ph.D.
Clinical Fellow–Pediatrics, University of California, San Francisco
Area: Type 1 Diabetes Through Another Lens—Lessons from Other Disease Settings

It has been known for some time that T1D is associated with increased risk of developing other autoimmune diseases, such as celiac disease, multiple sclerosis, and lupus. But cancer? It turns out that some patients on cancer immunotherapy go on to develop T1D. And there is a monogenic—meaning something that involves or is controlled by one gene—form of diabetes. It’s different from T1D or T2D, and while rare and requires a genetic test for accurate diagnosis, it can be studied for its relationship to T1D.

First up was Breakthrough T1D-funded Halis Akturk, M.D., who presented on checkpoint inhibitors—drugs that take the brakes off the immune system—in cancer and how they may lead to T1D. Next up was Martin Thelin, M.D., Ph.D., who has a Breakthrough T1D advanced postdoctoral fellowship in the laboratory of Dr. Mark Anderson, presenting on monogenic diabetes and how it can teach us about T1D. Lastly, there was Edwin Liu, M.D., who presented on celiac disease and how it correlates with T1D.

This session shone a light on how other diseases can be a resource for better understanding and managing T1D.

Robert H. Eckel, M.D.
Professor Emeritus, University of Colorado Anschutz Medical Campus
Area: Implementation of Guideline-Directed Medical Therapy for Cardiometabolic Disease—Emerging Multidisciplinary Initiatives

While he was at the University of Washington in Seattle from 1977 to 1979, Robert Eckel, M.D., was one of the first Breakthrough T1D-funded postdoctoral fellows. Now at the University of Colorado, he has been president of both the American Diabetes Association and the American Heart Association and has gone on to be an expert in cardiometabolic disorders. Cardiometabolic disorders are a cluster of interrelated factors, including high blood pressure, elevated blood-sugar levels, and high cholesterol, that put someone at high risk for having a heart attack or a stroke. Dr. Eckel also has type 1 diabetes and has had it for more than 70 years.

In Dr. Eckel’s video, he talks about a clinical trial for type 2 diabetes, in which participants had bariatric surgery (surgery intended to help a person lose weight) and were followed for 12 years to see how many people went into remission. In the beginning, the participants lost weight and there was also some remission of type 2 diabetes, which is defined by an HbA1c of under 6.5% and being off diabetes-related medicines. At one year, 50% of participants had a remission of type 2 diabetes. At 12 years, however, most of them—80%—had type 2 diabetes again.

“What are the predictors of poor outcomes?” Dr. Eckel posed. “I turned to the historical literature here. It’s people who have longer durations of diabetes and also people who are on more medications to treat their diabetes.”

This has relevance for type 1 diabetes, as longer durations of diabetes often lead to an HbA1c that is 7% or higher and may lead to complications that require diabetes-related medicines, similar to type 2. Research on these metabolic measures will ultimately benefit all of those in the diabetes space, whether it’s type 2 or type 1.

The American Diabetes Association’s 83rd Scientific Sessions is here! Until June 26, scientists will present the latest type 1 diabetes (T1D) research, from beta cell replacement to regeneration, and glucose and complications trials, all with the goal of improving lives for the T1D community.

Here are Breakthrough T1D-funded researchers Drs. Cristina Nostro and Tricia Tang to share their key takeaways from day 1:

Cristina Nostro, Ph.D.
Senior Scientist, McEwen Stem Cell Institute, University Health Network
Associate Professor, University of Toronto
Toronto, Ontario, Canada
Area: Designer Beta Cells

In an all-star line-up, Cristina Nostro, Ph.D., Audrey Parent, Ph.D., Heiko Lickert, Ph.D., and Trevor Reichman, M.D., Ph.D., presented on the state-of-the-art advancements in stem cell-derived beta cells. There were talks on the use of immune-modulated cells to promote tolerance and the use of stem cell-derived receptor to produce higher levels of insulin and be sustained longer when transplanted. But the real highlight of the session was an update on Vertex’s clinical trial to test VX-880, a stem cell-derived replacement therapy for diabetes, presented by Dr. Reichman.

At the ADA’s conference last year, 3 people had been dosed with VX-880, and one of them achieved insulin independence. Now, there are 6 people treated, all of whom produced endogenous insulin and had improved blood-sugar control, while reducing or eliminating insulin use. On the latter, both participants with 1+ year of follow-up achieved insulin independence and exceeded the ADA target time-in-range, while also meeting the primary endpoint of eliminating severe low blood-sugar events.

In other words, both participants with 1+ year of follow-up no longer need to administer insulin through injections or pump therapy and have exceeded the recommended time-in-range.

Vertex’s phase I/II clinical trial of VX-880 was made possible by Doug Melton’s years of JDRF-funded research and a catalytic investment from the T1D Fund in Semma Therapeutics—a biotech company founded by Melton to develop a stem cell-derived islet therapy for T1D—which was acquired by Vertex Pharmaceuticals.

Dr. Nostro, in her video, says, “In the last 20 years, we’ve learned how to differentiate these cells, and now we’re moving them to the clinic and they’re giving us the results that we want. The future is bright. I’m super excited, and I hope you are, too.”

Yes, we are!

Tricia S. Tang, Ph.D., R.Psych.
Associate Professor, Division of Endocrinology, University of British Columbia, Vancouver, Canada
Area: Translating Psychosocial Care into Real-World Practice Settings

The presence of T1D—whether for your child, yourself, or a family member—often affects people’s social, behavioral, and emotional well-being, known collectively as psychosocial health. Psychosocial issues—such as diabetes distress, depression, and anxiety—can put people with T1D at-risk for serious complications.

The session on psychosocial care, reported by Tricia Tang, Ph.D., R.Psych., wanted to know what worked—and didn’t work—in diabetes care to reduce the psychosocial concerns in the real-world practice settings. It featured sessions on psychosocial and behavioral screening, presented by Sarah Corathers, M.D., diabetes distress in type 2 diabetes, presented by Lawrence Fisher, Ph.D., family-based intervention, presented by Jill Weissberg-Benchell, Ph.D., and peer-support models for diabetes management, presented by Dr. Tang.

The American Diabetes Association’s (ADA) 83rd Scientific Sessions is almost here! From June 23-26, scientists and healthcare professionals will gather at one of the largest diabetes conference in the world. Breakthrough T1D-funded researchers will also be there to present new study results that will improve outcomes for people with diabetes (T1D). Read what’s happening below.

ADA’s 83rd Scientific Sessions

Curing T1D

Improving Lives

Throughout the Scientific Sessions, Breakthrough T1D will be sharing updates to keep people apprised of the day-to-day happenings:

JDRF’s vision is a world without type 1 diabetes (T1D)  and in the past fiscal year, through many top type 1 diabetes advances, we’ve made incredible progress toward that goal.  

Your support of our efforts is inseparable from the top type 1 diabetes advances we’ve seen in accelerating cures, improving lives, and advocacy wins for people with T1D and their loved ones. 

As we approach the end of fiscal year 2023 (FY23), let’s highlight the many  top type 1 diabetes advances we’ve seen.

Top Type 1 Diabetes Advance 1: First T1D Disease-Modifying Therapy  

In a historic moment for T1D—and one that Breakthrough T1D had a hand in from the beginning, supporting research from the 1980s on—the U.S. Food and Drug Administration (FDA) approved Tzield™ (teplizumab-mzwv) for use in delaying the onset of clinical disease in at-risk individuals aged 8+. 

For the first time in history, Tzield will treat the autoimmune process behind T1D, not the symptoms, altering the course of the disease.  

Among our top type 1 diabetes advances, this is the first disease-modifying therapy—treatments that can slow, halt, or reverse the course of the disease—for T1D to be approved, but it won’t be the last.  

Additionally, months after Tzield’s FDA approval, Sanofi acquired Provention Bio, the manufacturer of Tzield.  

The acquisition brings the first T1D disease-modifying therapy available in the U.S. into the portfolio of a global leading pharmaceutical company, representing an endorsement of the potential of these types of therapies and, we hope, the opportunity to bring this life-changing therapy and others in the pipeline to more people faster.  

Tzield and breakthroughs like it put us on the pathway to finding cures and, one day, preventing T1D entirely. 

Top Type 1 Diabetes Advance 2: A Blood Pressure Drug Preserves Beta Cell Function  

A Breakthrough T1D-funded study found that children and teens newly diagnosed with T1D who took verapamil—a drug already approved to treat high blood pressure—were making more insulin one year after diagnosis than those on placebo. In other words, in the children and teens who took verapamil, more beta cells were healthier one year post T1D diagnosis than those in the children and teens who took the placebo. 

This was the second trial that found the drug can preserve beta cells in the newly onset period.  

Additional studies may be needed to further validate the results, as well as identify all benefits and potential side effects of the drug. Breakthrough T1D has the strategy to answer these and other questions. 

The finding brings us closer to our goal of having numerous disease-modifying therapies widely available for people with type 1 diabetes. 

Top Type 1 Diabetes Advance 3: Affordable Insulins for Everyone 

Breakthrough T1D and partnering organizations are supporting nonprofit pharmaceutical manufacturer Civica Rx to produce biosimilar insulin that will cost no more than $30 a vial/$55 a box of five pens, regardless of insurance status.  

One year after the Civica announcement, Eli Lilly, Novo Nordisk, and Sanofi all announced reductions to the prices of their insulins—including the most used insulins, such as Humalog, NovoLog, and Lantus.  

Another big win for insulin affordability was the $35 monthly out-of-pocket co-pay cap for those on Medicare included in the Inflation Reduction Act that Breakthrough T1D fought hard to secure.  

In April, the Senate Diabetes Caucus Co-Chairs, Jeanne Shaheen (D-NH) and Susan Collins (R-ME), introduced the INSULIN Act of 2023, another key step toward achieving affordable insulin for all who need it.  

The bill seeks to limit out-of-pocket insulin costs by ensuring that people with commercial insurance pay no more than $35 or 25 percent of the net price per month for at least one insulin of each type and dosage form, and includes other important provisions to help make insulin more affordable and accessible.  

You can contact your members of Congress and encourage them to support the INSULIN Act of 2023.  

Top Type 1 Diabetes Advance 4: Turbo Boosting Cell Therapies  

Breakthrough T1D is working to develop and deliver life-changing therapies that place healthy, insulin-producing beta cells back into the bodies of people with T1D. There was a lot of progress in FY23.  

Vertex, which previously acquired Semma Therapeutics, also acquired ViaCyte, bringing together the leading companies developing stem cell-based therapies for diabetes.  

Vertex is advancing a stem cell-derived islet replacement therapy for T1D. It’s in human clinical trials and showing amazing results, with one participant being off insulin entirely.  

Vertex also started a trial with a new product using encapsulated stem cell-derived islets as replacement therapy, and is exploring gene-edited stem cell-based therapies—both  with the goal of eliminating the need for immunosuppressive drugs. 

Just this past April, Aspect Biosystems—an industry leader in 3D bioprinting technology—and Novo Nordisk announced a partnership to expand the development of a new class of treatments for diabetes and obesity, using Aspect’s bioprinting technology and Novo Nordisk’s expertise in stem cell and cell therapy development. 

The Aspect-Novo Nordisk partnership’s initial focus will be on developing bioprinted therapies for transplant that would be designed to maintain normal blood-sugar levels without the need for immunosuppression. This could represent a transformative treatment for people living with T1D. 

Additionally, the U.S. Food and Drug Administration (FDA) approved CellTrans’s Lantidra™, the first cell therapy to be authorized in the United States, for use in adults unable to approach average blood glucose levels due to current, repeated episodes of severe low blood sugar. This therapy, which requires the use of immunosuppressive drugs, takes deceased donor islets and places them into people with T1D suffering from repeated severe low blood-sugar, called hypoglycemia, events. This is an exciting first. 

Approved! Numerous T1D Management Technologies

Breakthrough T1D funds research to facilitate the development of new therapies and technologies to make day-to-day life with T1D easier, safer, and healthier. In the past year, we saw: 

Newly-Approved Artificial Pancreas (AP) Systems and Algorithms 

 Newly-Approved Continuous Glucose Monitoring (CGM) Systems  

A New Tool to Accurately Diagnose Type 1 in Adults 

Misdiagnosing adults with T1D as having T2D is an all-too-common problem that can have tragic consequences. Breakthrough T1D and IQVIA teamed up to develop an algorithm using artificial intelligence to examine medical records and identify individuals who were diagnosed with T2D but actually have T1D. This could be used in real time to correct misdiagnoses, offering the potential for future development into a clinical decision support tool. 

A First-of-its-Kind Lifesaving Tool: The T1D Index  

Breakthrough T1D and other T1D-related organizations launched the T1D Index, a first-of-its-kind data simulation tool that offers the most accurate estimate of T1D ever created. The Index measures and maps how many people live with this condition in every country, the healthy years of life it takes from people living with T1D, the number of people who would still be alive today if they hadn’t died prematurely from T1D complications, and our global strategy to reduce the impact of T1D. 

Go Forward 

Your partnership has been crucial to these advances and many more. On behalf of our community, thank you for moving us forward and ever closer to a world without T1D.  

We are excited for the top type 1 diabetes advances that fiscal year 2024 (FY24) will bring! 

Read Past Blogs about Top Type 1 Diabetes Advances: 

What We Can Be Proud of in 2022 

Celebrating the Best of 2021 

What We Can Be Proud Of in 2020 

Top 10 T1D Breakthroughs of 2019 

The U.S. Food and Drug Administration (FDA) cleared the iLet® Insulin-Only Bionic Pancreas System, which is designed to autonomously determine and deliver insulin doses to control blood-sugar levels, for people 6 years of age and older with type 1 diabetes (T1D). It includes an algorithm and an integrated infusion pump, which communicates directly with a compatible FDA-cleared integrated continuous glucose monitor (iCGM), enabling it to be an artificial pancreas, or automated insulin dosing (AID), system.

What’s new about this system? Ease-of-use. The iLet system is designed to have users enter only their weight for the iLet to initialize therapy. Immediately thereafter, the iLet begins controlling blood-sugar levels automatically, without requiring the user to count carbohydrates, set insulin delivery rates, or deliver additional insulin for meals or corrections. (Users do have to say whether the amount of carbs in a meal is small, medium, or large, but the algorithm learns over time in response to their individual insulin needs.)

The submission was based on a multi-center randomized insulin-only iLet Bionic Pancreas pivotal trial, which tested the insulin-only configuration in 440 adults and children 6 years and older with T1D. The trial met all key endpoints, demonstrating improved outcomes over standard of care for people living with T1D:

There are now multiple artificial pancreas systems on the market: The Medtronic 670G (2016), Tandem Control-IQ™ (2019), Medtronic 770G (2020), Insulet Omnipod 5 (2022), Medtronic 780G (2023), and, now, the iLet® Insulin-Only Bionic Pancreas System. (Tidepool Loop, an app that contains an algorithm that automates insulin dosing, has also been approved, but it has not yet announced its insulin pump manufacturer.)

Breakthrough T1D Impact

Breakthrough T1D started the Artificial Pancreas Project over 15 years ago to ensure people with T1D have better, more innovative ways to manage their type 1 diabetes until there are cures. Our goal was to ensure life-changing options for people with T1D and a competitive ecosystem that drove continuous innovation. To date, Breakthrough T1D has funded more than $140 million in artificial pancreas research.

Through these grants, Breakthrough T1D supported the development of the algorithm and preclinical and early clinical research—in partnership with the Helmsley Charitable Trust—through grants to:

This is a win for the T1D community and provides people with T1D another option to improve daily blood-sugar management, until cures are found.

Clinical trials are essential for delivering new devices and treatments to people with type 1 diabetes (T1D). There are currently over 100 actively recruiting studies for T1D in the U.S.

Participating in a clinical trial allows you to make an important contribution to the advancement of medicine. Below, meet two people who are helping improve the lives of people with type 1 diabetes and getting us closer to cures, one study at a time.

Meet Phyllis Kaplan

Type 1 diabetes clinical trial participant Phyllis Kaplan

Phyllis Kaplan has been part of Breakthrough T1D for so long, it was before the “R.” “Back then it was just JDF,” she recalled.

Phyllis has lived with type 1 diabetes for more than 40 years. After several decades of volunteering in outreach and One Walks, she joined Breakthrough T1D professionally as a Community Engagement Manager for the Greater New England chapter in 2022.

She also supports the T1D community by participating in clinical trials—she’s currently in her eighth one! Phyllis has taken part in studies on the impact of T1D on the liver, insulin pump infusion sets, and using glucagon in a bionic pancreas, to name a few.

“I consider clinical trial participation as my ultimate form of diabetes advocacy,” she said. “Without clinical trial participants there can’t be clinical trials.  Without clinical trials, there can’t be new treatment options.”

Phyllis partners with her chapter’s Clinical Trial Education Volunteer (CTEV), Amanda Gilchrist, to build a culture of research participation in the T1D community. “Being in a clinical trial is like being at the forefront of research,” she said. “It isn’t always easy, but it’s always important.”

Meet Amanda Gilchrist

Type 1 diabetes clinical trial participant Amanda Gilchrist with her family

When second-grade teacher Amanda Gilchrist received a T1D diagnosis, she was still adjusting to her daughter Emmie’s T1D diagnosis just a year earlier. She spent months learning about T1D so she could be a better caretaker for Emmie, but her diagnosis still came as a complete surprise.

Clinical trials sparked Amanda’s interest after she heard that researchers often struggle to find participants. “It shocked me to learn some trials don’t complete due to a lack of people signing up,” she said. “We need clinical trials to move forward with research, new devices, or a cure.”

She immediately got Emmie involved in clinical trials, including testing a new continuous glucose monitor (CGM) and artificial pancreas (AP) system. “Being in type 1 diabetes clinical trials makes Emmie feel like she’s a scientist working to help others with T1D,” she said.

Amanda has taken part in clinical trials, too. One aimed to reduce parental concerns about low blood sugar. Another explored the potential of plasmid therapy to retrain the immune system. She’s currently in a clinical trial for an automated insulin delivery system where it isn’t necessary to count carbs.

Amanda is also a Clinical Trial Education Volunteer (CTEV). She works with Phyllis to provide members of her local T1D community with information on clinical trials in their area. She loves helping families find a clinical trial that works for them. “You can be a hero like my daughter and move T1D science forward!”

Learn more about clinical trials and find local opportunities in your area.

These resources are made possible in part through the support of
Dompe and Vertex Pharmaceuticals.
Dompe

VERTEX

Cell replacement therapies aim to provide insulin on demand from cells implanted in the body, but, today, the shortage of donor beta cells and the need for chronic immunosuppression limit its widespread clinical adoption.

Breakthrough T1D is funding researchers around the world working on every aspect of cell replacement technologies to make them a reality—a cure—for type 1 diabetes (T1D). There’s a lot to figure out an many obstacles to overcome until these therapies realize their potential. These include:

Several approaches, however, are aiming to make these obstacles a thing of the past. Read more below.

Blood vessel growth + Local immunosuppression = A win for islet cell therapy

A way to combine islets—the group of pancreatic cells that produce insulin and glucagon—with blood vessels before transplantation would give them a virtually infinite supply of oxygen and nutrients, but it exposes the islets to the immune system, requiring systemic immunosuppression to prevent rejection. A novel encapsulation approach that integrates blood vessel growth for islets with effective immune evasion to prevent rejection could work.

Encapsulation: An advanced form of transplantation where a material is designed to keep cells protected from immune attack, while letting insulin out and letting oxygen and other nutrients in.

Enter the NICHE. It stands for Neovascularized Implantable Cell Homing and Encapsulation device. It is a quarter-sized device with two reservoirs—one for islets which have undergone the process of developing blood vessels and one for local immunosuppressant delivery—for the transplantation of islets to treat T1D. It is the first platform that integrates both blood vessel growth plus local immunosuppression into a single, implantable device.

In animal models, this device restored healthy glucose levels and eliminated T1D symptoms for more than 150 days while avoiding the adverse effects of anti-rejection therapy by administering immunosuppressive drugs only where the transplanted islet cells were located.

The results pave a path for the continued translational development of the NICHE technology, which has the potential to transform the field of islet transplantation.

Engineering a pancreas-like organ for transplantation

An unlimited source of less immunogenic islets? Check. Blood vessel growth prior to transplantation? Check. A novel scaffold to put these into? Check.

What does this mean?

You have an immune-protected, functional pancreatic organ—the first bioengineered device aimed to treat T1D.

What’s more? Results showed immediate function upon transplantation, preserving normal blood-sugar levels for up to 18 weeks.

Eventually, with further testing, this could be the next phase of cell replacement therapy, overcoming the current limitations in islet transplantation to generate a bioengineered device for the treatment of T1D.

Transplantation without the need for any immunosuppression

Vertex Pharmaceuticals—which acquired Semma Therapeutics in 2019 and ViaCyte in 2022, both of which had Breakthrough T1D or Breakthrough T1D T1D Fund support, with the goal of developing stem cell-derived replacement therapies for T1D—has a new first: No immunosuppression.

VX-264 takes the stem cell-derived therapy VX-880—which is being used to try to restore the body’s ability to produce insulin combined with immunosuppression—and encapsulates it with an immunoprotected device. The trial will begin to recruit later this year.

In Vertex’s phase I/II clinical trial, the first person to receive VX-880 is 100% insulin independent 270 days after receiving the therapy.

“Advancing research in cell replacement is a core pillar of Breakthrough T1D’s research strategy and we have been a significant supporter of these and other promising approaches,” said Jaime Giraldo, Ph.D., Associate Director of Research at Breakthrough T1D. “There is a revolution in cell therapy technologies and approaches, which will bring us one day to finding cures for type 1 diabetes.”

To one day cure type 1 diabetes (T1D), we must halt the destruction of beta cells that produce insulin. A new Breakthrough T1D-funded study suggests a potential path to keeping beta cells healthier for longer—meaning their body will still make insulin for more time, known as the “honeymoon” phase—for newly diagnosed youth.

The clinical trial looked at whether the effects of a hybrid closed loop system (also known as an artificial pancreas system or automated insulin delivery system) and/or verapamil preserved beta cell function one year after diagnosis in children and teens with T1D.

The study found that newly diagnosed individuals on verapamil were making more insulin one year after diagnosis than those on placebo, with the average C-peptide, which is used to measure insulin, being 30% higher for the verapamil group compared to placebo. HbA1c was 6.6% in the verapamil group versus 6.9% in the placebo group, at one year. (There was no change, however, in the hybrid closed loop system arm.)

“Safe, effective therapies are urgently needed to delay disease progression in people recently diagnosed with type 1 diabetes, an area of high priority for Breakthrough T1D,” said Sanjoy Dutta, Ph.D., Breakthrough T1D chief scientific officer. “The CLVer study is the second trial showing that verapamil, an inexpensive and widely used blood pressure medication, can preserve beta cells in the new onset period, making us one step closer to our goal of having disease-modifying therapies widely available for people with type 1 diabetes.”

But you don’t have to rely on what the blog said; from the clinical study authors: “oral verapamil was well-tolerated and slowed the rate of beta cell decline in youth with newly-diagnosed type 1 diabetes….In view of the favorable safety profile…once-a-day oral administration, and low cost, initiation of verapamil therapy should be considered for newly-diagnosed type 1 diabetes.”

What Does It Mean for the T1D Community?

Today, verapamil is not an approved therapy for newly diagnosed people with T1D, and it will not be in the very near future. There are additional studies that may need to be conducted to validate the results and learn so that all the benefits of the drug are known, as well as all of the potential side effects. Breakthrough T1D has a strategic road map to answer all these questions. These include:

There’s a lot more we must learn!

What Comes Next?

Breakthrough T1D will gather longer-term evidence of verapamil’s effectiveness, while in the near term sharing these data with the clinical community and other health care leaders to facilitate future access: