There are many unique challenges that need to be addressed to cure type 1 diabetes (T1D). For example, it’s incredible that, thanks to years of Breakthrough T1D funding, stem cell-derived insulin-producing beta cells created in a lab are in human clinical trials. However, this won’t translate into a cure for everyone with T1D until those cells can thrive inside the body to fully regulate blood-glucose levels without the use of broad immunosuppression. It’s an incredibly complex engineering and immune challenge, which is why Breakthrough T1D and the National Institutes of Health (NIH) brought together the top minds in these fields to discuss new technologies, approaches, and strategies.
Over two days, from July 27-28, nearly fifty scientists from a variety of scientific disciplines, including transplant immunology and bioengineering, assessed the scientific landscape, identified knowledge gaps and barriers, and outlined a roadmap to achieving localized immune modulation and immune tolerance key to implanted beta cells thriving in the body.
“We are very excited about the potential for cutting-edge research at the intersection of immunology and bioengineering,” said Jaime Giraldo, Ph.D., Breakthrough T1D Associate Director, Research. “It’s critical that Breakthrough T1D provides the opportunity to explore unique approaches for protecting transplanted cells, allowing for the next generation of beta cell replacement therapies to benefit a broad patient population. While we remain enthusiastic about the progress made with encapsulation technologies and their potential to address the challenges we face in creating beta cell replacement therapies, we are also encouraged at the prospect of next-generation technologies that can enable cell survival and function, without the need for physical barriers to protect the cells.”
Different Approaches, Same Goal
In order to protect the beta cells put into people to cure their T1D, they must be kept safe from the immune system. This can be done through localized immune modulation and induced immune tolerance.
Localized immune modulation creates small areas in the body that are immune privileged, which means they can tolerate the introduction of antigens—the alarm sirens secreted by beta cells—without eliciting an inflammatory response. This could be accomplished by:
- Using special drug delivery systems that release drugs locally in the vicinity of implanted cells; or
- Enabling beta cells to secrete biological signals that block the activity of attacking immune cells.
Inducing immune tolerance means teaching the body’s immune system to ignore and not mount an attack against certain stimuli. In T1D, people are missing immune tolerance to some of their beta cell proteins, which allows the body’s immune system to destroy their beta cells, giving them T1D.
These approaches have the potential to create an environment that allows a transplanted beta cell to flourish in an individual with T1D—safe from the autoimmune attack that created T1D in the first place.
JDRF’s Strategy: Get the Top Minds Together
JDRF’s approach to solving complex problems involves bringing a diverse set of experts to explore myriad solutions. This workshop featured immunologists, material scientists, biomedical engineers, and transplant specialists, among others, to brainstorm potential solutions and collaborations that have not been possible before.
“It was a great pleasure to work with Dr. Giraldo and the Breakthrough T1D team in the organization of this successful workshop that attracted an outstanding group of speakers, moderators and participants who discussed current therapeutic barriers and how to tackle them with immuno-engineering solutions enabling more effective and durable interventions in the context of cell replacement therapies for immune mediated diabetes,” said Dr. Guillermo Arreaza-Rubín, M.D. Director, Clinical Immunology and Diabetes Technology Program. “Workshops like this one allow our agencies to better coordinate and catalyze efforts to promote cutting edge and translational research in key priority areas”
This group came to the consensus that, while this technology has come a long way in recent years, there is still some work to be done before it becomes a commercially available therapy. Breakthrough T1D plans to take this information and create funding opportunities aimed at addressing fundamental gaps in knowledge needed to move forward, while continuing to push on other key approaches in cell replacement therapy such as encapsulation, oxygenation, gene modification, clinical development and others.
Not only was the workshop a productive two days of information sharing and brainstorming, but it was also a return to normalcy for Breakthrough T1D research. Interdisciplinary workshops have been key in forming the relationships and partnerships that produce solutions to complex problems which have brought T1D science to where it is today. This workshop was the first Breakthrough T1D hosted since the pandemic began in 2020.
Learn more about Breakthrough T1D’s work in cell therapies here.
