They shared new developments and applauded the speed of scientific advancements. At this year’s Strategic Research Session, the tone was dynamic as Breakthrough T1D’s internal team of scientists, leading type 1 diabetes (T1D) researchers, innovative strategic advisors and volunteers from the T1D community came together to chart the direction of Breakthrough T1D research for the year ahead.
“This is a truly exciting time in T1D research,” Breakthrough T1D President and CEO Aaron Kowalski, Ph.D., said at the start of the two-day session. “We are laser focused on curing type 1 diabetes while also driving advances to improve the lives of those living with T1D today.”
Dr. Kowalski and Breakthrough T1D’s team of researchers set scientific priorities and funding strategies. They outlined work in two areas—Curing T1D and Improving Lives—by explaining the critical potential in key research programs and sharing perspectives, ideas and proposals with experts across the T1D research space.
One key message emerged: there is no single path to curing T1D. More than one way exists to end a reliance on insulin; different options will likely be needed at different stages of T1D. As a result, several options in the categories of drug therapies and beta cell therapies will continue to be fully explored and supported.
To ensure that development proceeds on multiple promising avenues to cures, each research area is working off a roadmap that details the steps and tools necessary to advance therapies already in the pipeline.
At the gathering, researchers focused on the importance of discovering and validating biomarkers, as well as the need to attract more people into clinical trials to move research forward faster. Important as well is the Breakthrough T1D T1D Fund’s ability to attract private capital and partner with multiple companies to move assets from the discovery stage through to clinical trials. There was also a discussion about developing greater support to help people deal with the psychological effects of T1D.
Research programs were shared, then reviewed, and voted on—all to chart the course of a new year of investing in T1D research.
Key details included:
Curing T1D:
Beta Cell Replacement
To replace insulin-producing beta cells in a person, Breakthrough T1D is funding research that will tackle three main factors necessary for success. First, create abundant, reliable and scalable sources of cells. Second, determine how to protect those cells from an immune system attack and help them thrive. Third, determine the most effective ways to swiftly translate this research into clinical trials.
Beta Cell Regeneration
Improving the function and health of a person’s remaining beta cells is a crucial step toward insulin independence. To that end, Breakthrough T1D is analyzing how drugs approved for other diseases could promote the survival of beta cells during the honeymoon phase, the time period right after diagnosis when the pancreas still produces enough insulin to mostly maintain glucose control. Breakthrough T1D is also working to advance novel drug candidates, and exploring how to deliver drugs selectively to the beta cells to avoid side effects in other organs. The program is also determining the most effective ways to test new therapies alone, or in combination with immune therapies.
Immune Therapies
T1D is an autoimmune disease, and tackling the immune attack on the beta cells is central to developing cures for T1D. To bring us to the first approved immune therapy for T1D, Breakthrough T1D is working to better understand immune pathways, establish biomarkers that measure a positive response in trials, and develop trial designs that make clinical testing easier. In addition to studying the T1D immune system, Breakthrough T1D is studying how progress in immune therapies for cancer and other autoimmune diseases can advance new therapeutic approaches for T1D.
Prevention
Identifying people at the highest risk for developing T1D is the first step towards preventing T1D. Currently, pilot programs are screening for T1D risk in certain segments of the population. Breakthrough T1D is focused on expanding T1D screening to the whole population. Not only will widespread testing allow us to identify individuals who are in a position to test specific preventive therapies, but this testing can also prevent diabetic ketoacidosis (DKA) at diagnosis. DKA is a life-threatening complication of diabetes and preventing DKA at diagnosis can improve brain cognition in the short term and glucose control in the long term, including lowering future HbA1c. Another vital step forward is Breakthrough T1D’s support to develop of an anti-enteroviral vaccine. There is increased evidence that alterations in the microbiome, and viral infections in particular, may trigger T1D in certain people.
Improving Lives
Artificial Pancreas
Breakthrough T1D is focused on leading industry to transition from “hybrid” artificial pancreas systems to our goal of fully automated insulin delivery systems, where people with T1D wouldn’t need to check their continuous glucose monitors frequently, or self-administer insulin for meals in order to achieve glucose targets. We are also continuing to drive innovations that will make devices smaller, easier to use, personalized, and interoperable with other devices. All of these factors are expected to improve outcomes and reduce the daily burden of T1D, thereby increasing adoption and successful use of these life-improving devices among the community.
Metabolic Control
To help improve metabolic outcomes for people with T1D, Breakthrough T1D is funding research to develop next-generation insulins and therapies adjunctive to insulin that will work together to provide more holistic and healthy metabolic control than the current standard of care. New insulins will be responsive to glucose, target the liver so they will work more efficiently, and work very quickly. We are prioritizing development efforts to co-formulate insulin and the hormone amylin, an innovation that has the potential to meaningfully improve metabolic balance without any increase in burden. We are also helping to study whether particular drugs used for type 2 diabetes can be repurposed to help people with T1D as well.
Complications
Too often, people with T1D are not meeting glycemic targets despite advances in treatments. As a result, eyes, kidneys and other organs remain at risk for serious complications. Currently, Breakthrough T1D’s focus is on identifying biomarkers for earlier stages of T1D kidney failure and eye disease, and using genetic screening to identify genes related to these T1D complications. Breakthrough T1D supports complications research across demographic data points and works to advance the translation of promising generic drugs that may delay or prevent complications.
Behavioral Health and Psychosocial Support
Breakthrough T1D is committed to expanding the understanding of how mental, emotional and behavioral outlooks for people with T1D affect their overall health. This under-developed aspect of managing the disease is critically important. For instance, suicide rates are 6% higher for people with T1D when compared to people with other chronic diseases. Breakthrough T1D is supporting intervention-based research and training for health care professionals and families to be support systems for those with the disease.
Thanks for all your support, as we turn type 1 into type none.
Updated Research Storyline
To increase engagement and understanding of their work, Breakthrough T1D researchers—supported by the International Board of Directors and leading T1D scientists—outlined a more focused approach to telling the Breakthrough T1D research story.
At Breakthrough T1D’s annual Research Strategy Session last week, researchers said they would center the research story around two key pillars:
- Curing T1D
- Improving Lives
“Our aim is to ensure that Breakthrough T1D supporters understand our focus and our work, and how together we are striving to develop cures for T1D while also improving the lives of those living with T1D today,” said Breakthrough T1D President and CEO Aaron Kowalski, Ph.D.
Researchers also explained that cures are advancing on multiple pathways. As a result, Breakthrough T1D will now talk about cures for T1D — using the plural when discussing its pursuit of curing the disease.
“Lastly, to ensure that we are telling a more holistic Breakthrough T1D story, we will talk about Breakthrough T1D’s work to find cures and improve lives through an interlocking of our three key areas: research, supported by advocacy, and community outreach,” Dr. Kowalski said.
The changes more clearly communicate how Breakthrough T1D’s work brings its mission to life— with both today and tomorrow in clear focus.
Breakthrough T1D will find cures and improve lives through an interlocking of research, advocacy and outreach.
