At the 2024 International Society for Pediatric and Adolescent Diabetes (ISPAD) Conference in Lisbon, Portugal, the world’s leading diabetes researchers, academics, and members of industry gathered to share the latest and greatest in diabetes research. Breakthrough T1D leaders and many of our funded researchers and collaborators were on hand —for ISPAD’s 50th birthday—to share new science, breakthroughs, and a glimpse at what the next generation of T1D therapies will look like.
Let’s look at a few highlights!
The T1D Index Levels Up
Breakthrough T1D Chief Scientific Officer Sanjoy Dutta, Ph.D., and Thomas Danne, MD, Chief Medical Officer, International presented a much-awaited update on the T1D Index at ISPAD.
The T1D Index is a first-of-its-kind data simulation tool that measures the human and public health impact of the T1D crisis in every country across the globe. Until the creation of the Index, there have been wide gaps in the data about the incidence and impact of T1D. Leveraging data and insights from the T1D Index can help change the lives of people living with T1D by identifying attainable country-by-country interventions, including timely diagnosis, accessible care, and funding research that could lead to cures.
First , Dr. Dutta gave a refresher on our recent brand evolution, which includes why this change was necessary and how our research strategy has not changed.
Dr. Danne followed with a reintroduction of the Index, its place in our mission, and how we improved it. This includes:
- An enhanced dashboard and user interface designed to improve user experience
- A plan to develop Index 2.0
- An invitation and plea for all countries and existing registries to collaborate with data to enhance and develop Index 2.0
- Advanced simulation capabilities allowing for scenario analysis.
- Validation of data against available national registry data.
- Performance upgrades, including improved platform speed and minor adjustments to further refine accuracy.
In the future, the Index aims to have even more detailed data on a regional level.
Key takeaway:
Breakthrough T1D is focused on improving the lives of people with T1D all around the world—that includes every country in the world. The Index is key to defining the global scope of T1D and the country-by-country solutions to bring better outcomes to everyone affected by T1D.
Is metabolic control enough for young people with T1D?
We have known for decades that T1D is accompanied by the risk of developing complications. The conditions that are most associated with T1D include diabetic eye disease (retinopathy), kidney disease (nephropathy), and heart disease (cardiovascular disease). This is not an exhaustive list—there are many others, including mental health disorders. These complications most commonly occur in adults living with T1D, but new research suggests there’s more we can do to monitor and intervene with these complications in youths with T1D in addition to striving for ideal glycemic control.
At ISPAD, Breakthrough T1D-funded researcher Risa Wolf, MD, discussed long-term microvascular and macrovascular complications in T1D, including the complications mentioned above, and the data is striking. There is a low percentage of complications 4-7 years after diagnosis but after 12 years:
- 24% will have a decline in eGFR (a biomarker for kidney function)
- 13% will have neuropathy
- 52% will have retinopathy
- 28% will have dyslipidemia (abnormal levels of lipids in the blood)
Thankfully, leaps in care management, including automated insulin delivery systems and continuous glucose monitors, have led to improvements in A1c and, in turn, the rate at which people develop complications. But more can be done.
Through regular screening for these more common complications for all youths with T1D, we can better identify, diagnose, and monitor individuals as they develop these complications—especially because many of these screenings are low-cost.
Breakthrough T1D, for example, is funding Dr. Wolf to use the autonomous AI system (IDx-DR, Digital Diagnostics), which is FDA-approved for adults 21+ to detect and diagnose diabetic retinopathy in children. Ideally, this will lead to FDA approval and a better tool for monitoring this complication in kids.
Drs. M. Loredana Marcovecchio – another Breakthrough T1D funded investigator and Didac Mauricio took a similar look at vascular complications. Their presentations had related themes—it is critical to develop ways to identify complications like cardiovascular disease earlier. If they’re identified earlier, individuals can take advantage of the approved therapies.
Key Takeaway:
Like most areas of T1D management: there are still significant unmet needs.
We need to have a long view of these complications and their pathogenesis, which can start in youth. Glycemic control is critical to preventing the onset of these complications, but it is not always enough. Cardiovascular disease, retinopathy, dyslipidemia, and more can all be detected at a young age, which gives more time for interventions.
Breakthrough T1D is working on all of this in our Improving Lives program.
ISPAD / Breakthrough T1D Sessions
Breakthrough T1D Chief Scientific Officer Sanjoy Dutta, Ph.D. joined several collaborators for two sessions.
In the first, titled “Joint-Symposium Breakthrough T1D (formerly JDRF)-ISPAD: Type 1 Diabetes (T1D) Treatments: Hope and Promise Ahead,” two recipients of ISPAD/Breakthrough T1D fellowships, which allow care providers to travel to and receive training at world-class diabetes centers, shared their experiences and how the fellowship enabled them to improve the care they can provide to their communities.
After a presentation from Chantal Matthieu, a longtime Breakthrough T1D investigator, partner and reviewer, Dr. Dutta presented an overview of our work, our vision, and how the next generation of researchers in the room can apply for funding and join us in our work.
In the second session, the Joint ISPAD – Breakthrough T1D symposium, Dr. Dutta and others provided an update on our progress in treating this disease and what’s on the horizon. They touched on the many ways we can slow down disease progression, the exciting state of stem cell-derived cell therapies (they’re in human clinical trials and they’re producing insulin), and how artificial intelligence can play a role in reducing or eliminating T1D misdiagnosis in adults which can be as high as 40%.
Key takeaway:
It’s an exciting time in T1D research, and Breakthrough T1D’s strategy has mapped out what it takes to get to cures—including the importance of bringing in the next generation of researchers to contribute.
Revolutionizing Diabetes Care: Cutting-Edge Therapies
This session, which was a personal favorite of the author, gave updates on the biggest priorities we have at Breakthrough T1D: cures and how close we are to realizing them.
Spoiler alert: we’re not that far away.
It featured presentations from several Breakthrough T1D-funded researchers, including Colin Dayan, MD, Lori Laffel, MD, and Kimberly Simmons, MD.
First up: Dr. Simmons spoke about Tzield, the first disease-modifying therapy approved for T1D, and its use in the real world. A quick summary of the data:
- 322 people have received the drug to date
- The side effects reported in the clinical trial are consistent with what has been seen at the Barbara Davis center
- “Most of our patients have told us that they would do this treatment again.”
- Long term data for how people are tolerating the therapy and progression to T1D is not available yet.
As a reminder, Tzield was FDA-approved in November 2022 for individuals with stage 2 T1D to delay onset by an average of two years. Learn more in this comprehensive story on how it came to be and the role Breakthrough T1D played in getting it to market.
Dr. Michael Rickels then explored cell replacement therapies: where we are and where we’re going. His presentation covered the current landscape: We can use cadaveric islets to restore insulin production in people with T1D. However, the requirement for chronic immunosuppression and scarcity of donor islets mean this is not a viable option for the vast majority of people with T1D.
The next step is using an unlimited supply of stem cell-derived islets and discovering methods to keep them safe without the use of chronic immunosuppression, which is consistent with our strategy in this area.
Dr. Rickels also touched on the study that used autologous cells to restore insulin production in a person with T1D.
Lastly, Dr. Colin Dayan gave an update on combination therapies to preserve beta cell function in people with T1D.
Combination therapies utilize several different therapies that work by discrete mechanisms to target a singular problem. An example is tuberculosis or HIV, which both relied on complicated treatments composed of different pills that had to be taken at different times of day but are now treated with singular pills.
As we have shown, there are many drugs that can preserve insulin production. Now we’re learning which drugs can be taken together to preserve that insulin production longer. Dr. Dayan discussed the T1D Plus study (which builds on the Ver-A-T1D trial), which is funded by Breakthrough T1D, JDRF Australia, INNODIA, and the Helmsley Charitable Trust. This study will test several different drug combinations to see their effectiveness, including Tzield, verapamil, antithymocyte globulin (ATG), and golimumab.
Not only will this trial test multiple drugs—the innovative design of the study will speed up the length of the study, shaving years of the time it will take to see what combinations work best.
Key takeaway:
Breakthrough T1D is at the center of cell therapies and disease-modifying therapies. The clinical trials being run in humans today are very exciting and are a glimpse into how this disease could be treated in the not-too-distant future. It’s possible there will be approved combination therapies to slow progression and cell replacement therapies that restore insulin production entirely in the not-too-distant future.
Until next year!
These are just a few highlights from three days of sessions and presentations that cover our entire research portfolio. For example, Dr. Danne presented on the importance of early detection due to its numerous, clearly defined benefits and strategies for identifying these youths with stage 1 and stage 2 T1D. There were also sessions on the impact of AID systems, specific areas of complications, and much, much more.
We’re already looking forward to next year!
