Here’s what you need to know about deceased donor islet cell transplants—and our plan to expand availability to them safely and responsibly. 

What Is Deceased Donor Islet Cell Transplantation? 

Deceased donor islet cell transplantation is a procedure in which insulin-producing islet cells are isolated from the pancreases of deceased donors and transplanted into a person living with type 1 diabetes (T1D). These islets are infused into the liver, where they can begin producing insulin in response to blood glucose levels.  

Deceased donor islet cell transplants are intended for, and only FDA-approved for, adults with T1D who experience: 

• Severe hypoglycemia (dangerous low blood sugar) 
• Hypoglycemia unawareness (the inability to sense when blood sugar is dropping)

These individuals often cannot safely manage their blood glucose levels with the standard therapies used to manage T1D, including continuous glucose monitors (CGMs) and automated insulin delivery (AID) systems. 

Do They Work?

Yes. For adults with T1D who experience severe hypoglycemia and hypoglycemia unawareness, deceased donor islet cell transplantation has been shown to significantly reduce—or even eliminate—the need to take external insulin.  

Do Deceased Donor Islet Cell Transplants Require Immunosuppression? 

Yes. Recipients of deceased donor islet cell transplants must take immunosuppressives to prevent the body from rejecting the transplanted cells. Due to the side effects of immunosuppressives, these transplants are only approved for adults whose risk from severe hypoglycemia outweighs the risks associated with immunosuppression. 

Breakthrough T1D is working on alternatives to traditional immunosuppression so more people can benefit. This includes our funding of the University of Chicago’s clinical trial with Eledon’s drug, tegoprubart.

Why Aren’t They Widely Available?

Deceased donor islet cells come from organ donors. A single transplant often requires multiple pancreases as isolating islet cells is a complicated process. In addition to those challenges, the availability in the U.S. is more limited than it is in other countries due to regulatory classification.  

The U.S. currently regulates deceased donor islet cells as biological drugs requiring a Biologics License Application (BLA). This is different from how whole organ transplants are regulated in the U.S. and how deceased donor islet cells are regulated in many other countries, including Canada, the United Kingdom and countries in the European Union.   

How Is Breakthrough T1D Advocating for Expanded Deceased Donor Islet Cell Transplant Availability?

Breakthrough T1D has strongly urged HHS to reclassify and expand availability of deceased donor islet cells. 

Our proposal outlines a framework to ensure safety and accessibility for the T1D community. You can read the letter here. 

We are putting 100% of our energy behind this approach, and we are doing it with the full support of leading transplant surgeons and organizations.  

Why Is Breakthrough T1D Pursuing This Route (HHS) as Opposed to Legislation (ISLET Act)?

This is our best chance of success. Passing legislation is extremely difficult at best, and HHS has existing authority over the regulatory framework governing cell therapies and organ transplantation. They can make this change. 

Additionally, the ISLET Act does not require the development of safety standards for reclassified deceased donor islet cell transplants, nor does it address insurance coverage, which could limit access to these costly treatments. 

What Are the Details of Breakthrough T1D’s Recommendations in the Letter to HHS?

Our proposal outlines a clear strategy to ensure the safety and quality of deceased donor islet cells under this reclassification. They include: 

• Designating Centers of Excellence staffed with experts in deceased donor islet cell transplants 
• Strict quality control to ensure islets are functional—and they work 
• A way to get cells from the deceased donors’ pancreases and into people with T1D
• A strategic roadmap for adding additional centers

Why Not Work with HHS and Support the ISLET Act? 

Our Breakthrough T1D legislative agenda includes several critical priorities essential to advancing our mission. This includes the ongoing renewal of the Special Diabetes Program (SDP) to fund T1D research and efforts to improve insulin affordability. These issues require legislative action, which is why we continue to focus our legislative advocacy on them. 

Not every priority is best achieved through Congress. Reclassifying deceased donor islet cells as organs, for example, can be accomplished under current law through the Department of Health and Human Services (HHS), without new legislation. This is one of the key reasons we believe the path through HHS is the best path for this issue. 

Simply put, we are pursuing each of our priorities through the channel where it has the greatest likelihood of success. And given how difficult it is to pass legislation, putting 100% of our effort toward working with HHS has the best chance of success.  

Why Doesn’t Breakthrough T1D Recommend an Amendment to the ISLET Act?

Given how difficult it is to pass legislation, we believe our effort to reclassify deceased donor islet cells as organs is much more likely to succeed focusing on HHS. 

What effect would this have on stem cell-derived islets?

Breakthrough T1D believes that stem cell-derived islets are appropriately regulated by FDA as biologic products. There is no need to change the regulatory status for manufactured islets. If adopted, our proposal would not affect them.  
 
Through Project ACT, Breakthrough T1D is working with all parties to clear the way for future, manufactured islet therapies and to ensure they are accessible. 

Cell therapies

What are cell therapies?

In people with T1D, the immune system destroys the beta cells in the pancreas, which produce insulin. One known way to cure T1D is through cell therapies, which replace destroyed beta cells with external cells that make insulin and protect them so that they can function for a very long time.

What work is Breakthrough T1D doing in this area?

117

Number of preclinical cell therapies research projects we are supporting

5

Number of cell therapies clinical trials we are supporting

We are funding research to:

1. Develop a renewable beta cell source so that every person with T1D who wants this treatment can get it.
2. Find ways to maintain these beta cells so they stay functional in the body for years—or even decades.
3. Shield these beta cells from immune attack to prevent the need for anti-rejection immunosuppressants or the recurrence of T1D.

What does the future hold for cell therapies?

Our ultimate goal is large-scale cell therapies with the fewest side effects possible, accessible by anyone with T1D who wants them. Our Project ACT initiative will get us there faster than ever. We believe that, in the future, these therapies will create a path for people with T1D to walk away from the condition for good.

Learn More About Cell Therapies

Early detection

What is early detection?

Type 1 diabetes doesn’t show up overnight (even though for many people it feels like it does). The condition develops in stages over time:

From a therapy perspective, early detection is important for identifying people who could benefit from the FDA-approved disease-modifying therapy Tzield, which can delay T1D progression for years.

From a research perspective, early detection can open doors to research opportunities, like clinical trials of other disease-modifying therapies in development.

From the clinical perspective, early detection can prevent complications at diagnosis, like diabetic ketoacidosis (DKA), and help families and individuals prepare for T1D onset.

Screening is done through a simple blood test to detect T1D autoantibodies, which signal that the immune system may be attacking the body’s insulin-producing cells.

What work is Breakthrough T1D doing in this area?

52

Number of preclinical early detection research projects we are supporting

26

Number of early detection clinical trials we are supporting

Through our Advocacy and Medical Affairs efforts, we are:

• Supporting the SCREEN for Type 1 Diabetes Act
• Leading a campaign to expand access to early detection of T1D in the United States
• Providing accredited education and resources on T1D and early detection to healthcare professionals

What does the future hold for early detection of type 1 diabetes?

We expect to improve ways to detect T1D early and prevent it from advancing by attaining a better understanding of the natural progression of T1D, which may include additional biomarkers for the condition. We will continue to expand access to early detection and find ways to improve the standard of care for early-stage T1D.

Learn More About Early Detection

Disease-modifying therapies

What are disease-modifying therapies?

T1D is a progression. At earlier stages, the immune system has begun attacking the insulin-producing beta cells, but people are not symptomatic yet. Some people at later stages have had T1D for decades and have lost nearly all insulin production. Disease-modifying therapies, or DMTs, are aimed at helping everyone with T1D—regardless of where they are on the disease continuum—to slow, halt, prevent, or reverse the condition.

What is Breakthrough T1D doing in this area?

138

Number of preclinical disease-modifying therapy research projects we are supporting

16

Number of disease-modifying therapies clinical trials we are supporting

We recently published research on the importance of C-peptide—a biomarker for insulin production—as a key measure of how effective a DMT is in clinical trials. Use of C-peptide will be essential to speeding up development and approval of new DMTs—getting potential cures to people with T1D faster.

What does the future hold for disease-modifying therapies?

As we see more DMT clinical trials advance to later-stage human testing, we expect to see additional DMTs submitted for regulatory approval—giving people with T1D more options.

Learn More About Disease-Modifying Therapies

ADA Recap Series

This article is the second of our three-part ADA Recap Series. Breakthrough T1D was on site in Chicago, IL from June 20-23 for the American Diabetes Association’s (ADA) 85th Scientific Sessions. We’re here to report on the latest-and-greatest type 1 diabetes (T1D) advancements—including many driven by Breakthrough T1D funding. Look out for tomorrow’s article for updates on Medical Affairs.

Cell therapies were front-and-center at ADA 2025. We have some exciting clinical trial updates and new ideas for optimizing islet transplantation.

Cell therapies

Autologous cell transplantation

Autologous cells are those removed from an individual and implanted back into the same individual. These cells can be modified in a laboratory before implantation. Autologous cells are still susceptible to autoimmunity in T1D, so cell protection strategies (gene-editing, encapsulation, immune modulation, etc.) are expected to be required.

Allogenic cell transplantation

Allogenic cells are those that are derived from a source other than the recipient, such as deceased donors or precursor-derived manufactured cells. Allogenic cell transplants require immunosuppression because they stimulate an immune response. Breakthrough T1D’s Cell Therapies program is focused on allogenic cells—specifically manufactured cells—because they can be generated at large scale.

One-year updates on Vertex’s manufactured cell therapy, zimislecel

• Presenter: Michael Rickels, M.D. (University of Pennsylvania)
• Zimislecel (VX-880) is a manufactured islet therapy that requires immunosuppression, infused into a vein in the liver in people with T1D who have impaired hypoglycemic awareness and severe hypoglycemic events.
• The phase 1/2 clinical trial, which is part of the pivotal phase 1/2/3 FORWARD-101 trial, is complete. Twelve participants received a single infusion of a full dose of cells and were followed for at least one year.
• All 12 participants achieved the primary endpoint, which was elimination of severe hypoglycemic events and HbA1c levels less than 7%. 10/12 (83%) participants are insulin independent.
• All 12 participants demonstrated sustained insulin production as measured by C-peptide, reduced external insulin therapy use, and achieved greater than 70% time in range.
• There were no serious adverse events. Mild to moderate adverse events were consistent with the immunosuppression regimen, infusion procedure, and complications from T1D.
• These data were published in the New England Journal of Medicine and represent further evidence of the curative potential of manufactured islet transplantation for T1D.
• Breakthrough T1D’s support for Doug Melton, Ph.D.—whose proprietary lab-created beta cells are now being advanced by Vertex—goes back decades, both via research grants and an investment from the T1D Fund: A Breakthrough T1D Venture.

6-month update on Sana Biotechnology’s immune-evasive islets

• Presenter: Per-Ola Carlsson, M.D., Ph.D. (Uppsala University)
• Sana’s donor-derived islet therapy engineered with Hypoimmune (HIP) technology can evade the immune system without immunosuppression.
• These cells were implanted intramuscularly in a first-in-human study into a person with T1D with no measurable insulin production.
• Six months post-transplant, this person is consistently making their own insulin, as measured by C-peptide levels. Yet, they still require external insulin therapy because they received a smaller dose of cells than the dose that would be required to achieve insulin independence. They did not experience any serious side effects, so the cells and procedure are safe and well-tolerated.
• A Mixed Meal Tolerance Test (MMTT) confirmed that these cells are not only surviving but also responding to changes in blood glucose levels.
• This is a promising first step toward a functional cure for T1D that does not require immunosuppression. Sana Biotechnology is planning on applying this technology to manufactured islets.
• Sana has received support from the T1D Fund to advance their HIP technology in islets, and Breakthrough T1D continues to work closely with them.

A new transplantation site for autologous manufactured islets

• Presenter: Hongkui Deng, M.D. (Peking University)
• Cells derived from adipose tissue (fat) can be removed from a person and chemically induced in the laboratory to become islet cells.
• Implantation of autologous manufactured islets into the sub-anterior rectus sheath in preclinical models of T1D improves glycemic control.
• In humans, this implantation site is easily accessible by an ultrasound-guided needle.
• In a first-in-human study, autologous manufactured islets were implanted into this site in a person with T1D. This person no longer needs external insulin therapy and has greatly improved blood glucose control. This person had also received a liver transplant and was taking immunosuppressants.

A new encapsulation device for immune protection of transplanted islets

• Presenter: Nicolas Laurent, Ph.D. (Adocia)
• Adoshell® is a novel islet cell encapsulation device that can shield islets from the immune system, meaning that immunosuppressants are not needed.
• The hydrogel-based device is non-degradable, easily retrievable, and allows the exchange of glucose and insulin from the vasculature surrounding the device while excluding immune cells from encapsulated islets based on pore size.
• This device showed promise in animal models, and human clinical testing is next.

Disease-modifying therapies

A major focus at ADA 2025 was addressing the underlying immune mechanisms of T1D—including alterations in immune cells that facilitate beta cell destruction and other factors that contribute to autoimmunity onset. Read on for some highlights.

The role of B cells in T1D autoimmunity

• Presenter: Mia Smith, Ph.D., DVM (University of Colorado)
• B cells are a type of immune cell that can activate destructive immune cells that facilitate autoimmunity in T1D.
• B cells can become wrongly activated against insulin-producing beta cells due to converging dysregulation of factors that regulate immunity.
• These cells represent another potential target for disease-modifying therapies in T1D.

Early detection

A key focus at ADA 2025 was the growing recognition of the heterogeneity of T1D, including autoantibody-negative disease onset, genetic variation, and the frequent misdiagnosis of T1D in adults, underscoring the need for greater diversity and inclusion in research and care. The expanded role of continuous glucose monitoring (CGM) and continuous ketone monitoring (CKM) was also highlighted, not only for daily management but as essential tools for understanding disease progression.

Using genetics to predict T1D risk

• Presenters: Richard Oram, M.D., Ph.D. (University of Exeter), Leslie Lange, Ph.D. (University of Colorado), Aaron Deutsch, M.D. (Massachusetts General Hospital), Josep Mercader, Ph.D.(Massachusetts General Hospital) and Eimear Kenny, Ph.D. (Icahn School of Medicine at Mount Sinai)
• Polygenic risk scores (PRS) estimate the risk a person has for developing a disease like T1D based on variations in different genes.
• Ancestry is a major influence on PRS, particularly based on differences in genes that regulate whether the immune system can distinguish between “self” and “non-self.”
• Most PRS models have been developed using data from European populations and have a limited ability to accurately determine risk in other ethnic groups, such as individuals of African and East Asian descent.  
• Potential applications of PRS include incorporation into screening to better understand T1D risk, ensure accuracy in diagnostic tests, and develop precision medicine-based therapeutic approaches.

Understanding how genetic diversity contributes to T1D

• Presenters: Suna Onengut-Gumuscu, Ph.D. (University of Virginia), Dominika A. Michalek, MS (University of Virginia), Aaron Deutsch, M.D. (Massachusetts General Hospital), and Stephen I Stone, M.D. (Washington University School of Medicine), among others.
• These talks highlighted several studies conducted in diverse populations to better understand the pathophysiology of T1D.
• Work presented from Consortia, such as RADIANT, focused on rare and atypical forms of diabetes.

Controversies in CGM and benefits for early detection

• Presenters: Peter Calhoun, Ph.D. (Jaeb Center for Health Research), Michael Kohn, M.D., MPP (University of California San Francisco), Nicole Ehrhardt, M.D. (University of Washington) and Tadej Battelino, M.D., Ph.D. (University of Ljubljana)
• CGM use holds value in identifying progression in early stages of T1D prior to symptomatic onset.
• There was a call to update the clinical guidelines so that the benefits of CGM can be maximized within the T1D community—including at early and later stages of T1D.
• Integrating newer measures of blood glucose, like the glucose management indicator (GMI) and time in tight range (TITR), will be essential.

Contributions of CKM to early detection

• Presenters: Ketan Dhatariya, MBBS, M.D., Ph.D. (Norfolk and Norwich University Hospitals), Lori Laffel, M.D., MPH (Harvard University), Jennifer Sherr, M.D., Ph.D. (Yale University), and Richard Bergenstal, M.D. (HealthPartners Institute).
• It will be critical to explore whether ketone monitoring could help reduce the incidence of diabetic ketoacidosis (DKA) at stage 3 clinical T1D onset.
• Early detection of rising ketones will be important for people with T1D to take action before DKA occurs.

Look out for tomorrow’s article for an update on Medical Affairs presented at ADA 2025!

We’ve made major progress in the development of cell replacement therapies for type 1 diabetes (T1D) over the past few decades. We know that manufactured islets can be safely implanted into people and produce insulin. Yet, there is more work to do to advance cell therapy research and bring these therapies to the larger T1D community. Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) will make manufactured cell replacement therapies that do not require broad immunosuppression a reality, faster.

The Breakthrough T1D x Stem Cell Network Partnership

To drive innovation in manufactured islet therapies, Breakthrough T1D, Breakthrough T1D Canada, and the Stem Cell Network (SCN) have partnered to support four new projects led by Canadian researchers. The organizations issued a joint Request for Applications and together will maximize resources to drive high-impact research into manufactured cells. This partnership is a novel and meaningful part of Breakthrough T1D’s global Project ACT effort to power high-impact cell therapies research.

“Accelerating cell therapies is a central focus of Breakthrough T1D’s research strategy to drive toward cures for type 1 diabetes,” said Breakthrough T1D Vice President of Research Esther Latres, Ph.D. “We’re excited to join Stem Cell Network and Breakthrough T1D Canada in funding these outstanding cell therapy researchers and projects that can build on the current momentum to overcome barriers and advance cell therapies that can benefit all those who live with type 1 diabetes.”

These projects will receive support from May 2025 to April 2027. They are a part of a broader SCN investment totaling more than $33 million to support 36 regenerative medicine research projects and clinical trials.

Read on to learn more about the exciting, newly funded projects.

 A closer look at the projects

Current cell therapies for T1D, while often effective, are hampered by reliance upon donor-derived cells and poor cell survival after transplant, necessitating large doses of cells and repeat procedures.  This ambitious new project will address both the source of islet cells and the low cell survival rates associated with islet transplantation by accelerating Lunar Therapeutics’ preclinical development of a manufactured islet replacement product. Takebe’s lab describes it as “complex miniature organs” for T1D.  

This product will consist not only of insulin-producing cells, but also endothelial cells, which line blood vessels. Endothelial cells will support islet cell survival and engraftment upon transplantation.  

Organoid

An organoid is a three-dimensional tissue grown in the lab that resembles an organ.

To accomplish this objective, Lunar Therapeutics will bring together Canadian expertise in manufactured islets and clinical islet transplantation led by Drs. Timothy Kieffer and James Shapiro. The team will also include U.S.-based Dr. Takanori Takebe, who specializes in designing complex organoids composed of various cell types. Using technologies developed across each laboratory, this multidisciplinary team will work to address challenges in islet cell transplantation.  

The implantation of manufactured islets into people with T1D can restore insulin production, eliminating the for external insulin and improving quality of life. However, after islet cells are derived from donors or manufactured in the lab, they must be stored before being used to treat a person with T1D. Currently, the storage and transportation of islet cells is difficult, and the only storage method is freezing at low temperatures in the presence of chemical solutions that help with the freezing process. However, these solutions cause cell death during thawing and may also cause allergic reactions in people after transplantation.   

This project will address this gap in the field by aiming to develop non-toxic, naturally derived gels to optimize islet freezing and storage. The gel-based products will be evaluated for large-scale commercial production. The success of this project will provide new intellectual property that will be of interest to researchers and companies in regenerative medicine in Canada and across the globe. 

Manufactured islets offer a potentially unlimited source of islets for transplantation. Since manufactured islets carry unique risks compared to donor-derived islets, containment within a device could allow retrieval if off-target growth ever occurs. However, encapsulation devices that have been tested in clinical trials so far and have shown minimal success, mainly because blood supply to the cells is limited by the device barrier. In this project, the team proposes to develop a device where the manufactured islets are placed around pre-established vessels that can improve islet cell survival and speed of insulin responses via improved blood supply. In this project, they will optimize their device design and conduct advanced preclinical studies. 
 
This project could lead to better survival and function of manufactured islets, so they can keep producing insulin. The project may also pave the way for other engineered human-scale encapsulation devices, also sometimes called bioartificial organs.

There are many new treatments on the horizon for T1D, including those that block autoimmunity or replace insulin-producing cells. However, a major barrier to these therapies is the lack of an easy-to-use model in which their effects on human cells can be tested before advancing to human trials. The standard preclinical model is to test therapies in small animal models of T1D, but this has significant limitations since it is nearly impossible to replicate the human immune system. In fact, diabetes has been “cured” hundreds of times in a mouse model, which has not translated to humans. 
 
To overcome this barrier, Dr. Levings and her team will establish a new a model that recreates human T1D autoimmunity in the lab. The model will use manufactured cells to create the three types of cells that are involved in the disease: insulin-producing cells and two different types of immune cells. Using the model, cells can then be combined in different ways to recreate what usually happens during autoimmunity. 
 
A model of human T1D that can be generated in the lab will help test potential treatments and prompt new questions about why T1D develops, and how to prevent it. Thus, this research has the potential to support the further development of innovative therapies that may offer new approaches to prevent or treat people with T1D. 

Putting it all together

Curing T1D is the north star of Breakthrough T1D. These partnerships will help us work together toward our shared goal of a world without T1D—through innovation, forward-thinking cell therapy research, and the best and brightest scientists.

A meeting of the minds

This past week, Breakthrough T1D helped organize an event hosted by Member of European Parliament Tomislav Sokol, Ph.D., titled “Accelerating Breakthroughs to Address Unmet Needs in Type 1 Diabetes.” This meeting, a significant coming-together of Breakthrough T1D and European policymakers, focused on the role of the EU in addressing the needs of the T1D community and accelerating T1D breakthroughs.

The purpose of this meeting was to raise awareness of T1D and the urgent need for the accelerated development and approval of breakthrough therapies in the EU. Conversations between Breakthrough T1D and European policymakers homed in on barriers and opportunities to advancing cures—including cell therapies and disease-modifying therapies—in the EU to get them into the hands of people with T1D, faster. This was an important step in establishing an open dialogue between Breakthrough T1D and the EU Institutions about working together to address T1D globally.

“This event in the EU Parliament allows us to engage with important decision-makers to ensure that the research and policy environments are oriented in a way to accelerate development of T1D breakthrough therapies in the EU as we also do in other countries,” explained Campbell Hutton, Senior Vice President of Global Advocacy at Breakthrough T1D.

Driving toward T1D cures in the EU and beyond

T1D is on the rise around the world, including in Europe. Recent publications by Breakthrough T1D staff and leadership brought attention to the rise in incidence and global T1D burden. We need to act now in conjunction with governments around the world—like the EU—to address the unmet needs of everyone around the world affected by T1D.

T1D cures, including cell therapies, are advancing through the clinical pipeline. Bringing awareness of T1D to the forefront—and educating key people on the progress we’re making toward cures—will be incredibly important for driving T1D research forward in the EU. This represents a critical opportunity for the EU to accelerate cell therapies faster than ever. As a global organization, Breakthrough T1D is collaborating with the EU government to help make this possible.

As the largest global funder of T1D research, Breakthrough T1D has provided funding to researchers across the world in addition to the EU. Right now, Breakthrough T1D is supporting €56 million in European initiatives, including 31 clinical trials—representing 19% of our funded research (including the U.K.). Breakthrough T1D has expert teams in research, medical, regulatory, and advocacy in Europe, meaning we are uniquely positioned to provide guidance on how the EU can strengthen its T1D efforts and collaborate on a global scale to drive T1D breakthroughs, especially in cell therapies.

Type 1 diabetes is a critical disease in Europe, and I was pleased to host an event for the T1D community and my colleagues in the European Parliament to learn about the unmet needs in T1D and how we can work together to accelerate breakthrough therapies in Europe to address those needs.”

Tomislav Sokol, Ph.D. Member of the European Parliament

What Breakthrough T1D leadership is saying

“This meeting is critically important to bringing the unmet needs of the T1D community into the spotlight in the EU. Global advocacy for curative T1D research is essential to achieving Breakthrough T1D’s mission, and continued collaboration with EU policymakers will get us there faster.”

Lynn Starr

Chief Global Advocacy Officer

“International efforts to accelerate global adoption of T1D cures will become increasingly important as newer, emerging cell therapies become available to people with T1D. Conversations like these with EU policymakers is bringing this urgent need to the forefront.”

Thomas Danne, M.D., Ph.D.

Chief Medical Officer, Global

“Cell therapies are accelerating through the clinical pipeline faster than ever. We need to act now on a global scale to ensure that people with T1D around the world can access these transformative therapies. This meeting is a significant step toward that goal.”

Sanjoy Dutta, Ph.D.

Chief Scientific Officer

Accelerating global action is paramount to our mission

Global problems require global solutions. This meeting served as a critical launching point for a continued partnership with the EU to fill gaps and address unmet needs for the T1D community. Building relationships and fostering long-term partnerships is critically important for reaching our common goal of bringing cures to people with T1D as soon as possible.

These important conversations between Breakthrough T1D and the EU government align with our Project ACT (Accelerate Cell Therapies) initiative to accelerate the development of cell therapies that do not require immunosuppression—for everyone with T1D in every country. In addition, through international Centers of Reference, Breakthrough T1D’s Medical Affairs team is developing expert clinical care centers that will be trained and ready to provide cell therapies to people with T1D once they become available. We are at the forefront of global action to prepare the world for curative cell therapies.

We are driving toward a future in which everyone with T1D—no matter where they are—has access to therapies, treatments, and care, bringing us closer to achieving our mission of a world without T1D. The more people we have working toward our mission, the faster we will get there.

Outside the Cammett family’s Michigan home sat a new toy truck. John, around six years old, admired its sturdy metal frame, fresh rubber wheels, and bright yellow decals. But it would not stay that way for long. 

John and his two brothers were athletic, boisterous, and yes, at times, destructive. After the truck was thoroughly battered, John’s mother, Barbara, began her work on the piece. She was creative and artistic and knew just what to do. With paint brushes in hand, she covered the damaged truck in a collage of color and something new emerged. It was no longer a wreck, it was a work of art.

John Cammett, now 62, says he’ll always remember that day and the special lesson that came from it. 

“Even with all that destruction, she could make something look beautiful,” he said.

Like mother, like son 

John’s mom was diagnosed with type 1 diabetes (T1D) in her 30s and passed away in 2021 at age 89. She inspired John, who also lives with T1D, to become a champion for others with the condition. John has since become a longtime volunteer, leader, and advocate for Breakthrough T1D—providing transformational support of our mission. 

In recognition of his deep commitment, the Breakthrough T1D Center of Excellence in New England was recently named for his mother. The center will now be known as the Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England. John helped establish the center and provided foundational support for Project ACT (Accelerate Cell Therapies), Breakthrough T1D’s initiative to make cell therapies as cures for T1D a reality. 

My mom was the strongest person I’ve ever known—a real warrior. Even back then, with limited technology, she never let T1D set her back.”

John Cammett, Co-Chairman and Founding Partner of Realterm and Passionate Seed Funder of The Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England

Honoring her positive spirit 

Since its inception, the Breakthrough T1D Barbara Dewey Cammett Center of Excellence in New England has made significant progress advancing islet cell-based T1D research, enhancing the understanding of the immune response following islet transplantation to prevent rejection, and creating genetically modified islet cells that could withstand the immune attack after transplantation into people living with T1D.  

The breakthrough research happening at the center—one of five Breakthrough T1D Centers of Excellence around the world powering advances to deliver cures and life-improving breakthroughs for T1D—is particularly inspiring to John. He takes pride in knowing that the center he helped establish will not only advance this important work but also honor his mother’s positive spirit and enduring legacy. 

“You can’t be a researcher without optimism—every breakthrough stands on the back of countless failures. My mom lived the same way. She was the most optimistic person I’ve ever known, never said a bad word about anyone, and kept going no matter how hard things got. Just like the researchers pushing forward every day, she stayed focused, kind, and hopeful through it all.” 

A beacon of strength

John remembers his mom as a woman whose generous heart, zest for life, and unwavering optimism inspired everyone she met.  

She managed T1D for nearly 60 years with grace, determination, and a smile. Known for her vibrant personality, Barbara embraced life fully. You could often find her cheering on her Wisconsin Badgers, painting beautiful art, volunteering in her community, and effortlessly outdriving her husband on the golf course.

Her kindness, resilience, and passion for connecting with others made her a beloved friend, devoted wife, and beacon of strength to those navigating life with T1D.  

“I wish I could have done this while she was still with me,” John said. “But I know she’d look back and smile. She wanted to help everyone she could. That spirit lives on.”  

By: Adam Baker

Breakthrough T1D’s newest mission pillar, Medical Affairs, is bridging the gap between access to and adoption of T1D therapies. The establishment of this program is essential to Project ACT (Accelerate Cell Therapies): Breakthrough T1D’s initiative to accelerate the development of manufactured islet cell replacement therapies that do not require immunosuppression. The goal is to make sure that people with type 1 diabetes (T1D) can get these therapies as soon as they hit the market.

The field is moving quickly: people are becoming insulin-independent in cell therapy clinical trials. We are advancing towards the submission of the first-generation manufactured islet cell therapy that requires immunosuppression, Vertex’s zimislecel (VX-880). We are at a critical moment and need to act now to ensure that healthcare providers (HCPs) are ready to bring manufactured islet cell therapies into clinical settings.

Enter Medical Affairs

This is where Medical Affairs comes in. The team, led by Thomas Danne, M.D., Chief Medical Officer International, is working with the medical community to anticipate obstacles to getting manufactured islet therapies into clinics and find ways to overcome them now.

To accomplish this, Breakthrough T1D recently hosted two cell therapy workshops, convening multidisciplinary, international experts in islet cell transplantation to discuss a clinical roadmap for manufactured islet cell therapies—and how to ensure that clinical teams are in place and prepared to provide these therapies to people with T1D who qualify. By preparing now, we can get this first-generation therapy into the hands of people with T1D as soon as possible after regulatory approval.

The cell therapy workshops were hosted by Breakthrough T1D’s Thomas Danne, M.D., Chief Medical Officer International, and Anastasia Albanese-O’Neill, Ph.D., APRN, CDCES, Vice President of Medical Affairs.

Read on to learn more about the cell therapy workshops, the attendees, and what each accomplished.

Workshop #1: Who may benefit most from manufactured cell replacement therapies

The purpose of this workshop was to start developing a five-year roadmap to help guide the T1D care community to support the adoption of manufactured islet cell therapies in clinical care. The evidence-based recommendations will be vetted by a larger group of clinical experts, diabetes organizations, and people with lived experience with T1D to ensure there is broad agreement. The consensus document will ultimately be published to expand its reach.

This process will summarize the essential evidence that will help HCPs decide who may benefit the most from manufactured islet cell replacement therapies. These decisions will take into account the perspective of people with T1D and differences in age, hypoglycemia unawareness, or kidney health, to name a few. This can help HCPs better understand the benefits versus risks for manufactured cell therapies on a person-by-person basis—making sure that each clinical decision is made jointly to prioritize long-term health.

Workshop #2: Pilot workshop to develop international Centers of Reference for T1D cell therapy

The objective of this workshop was to take the first step toward creating Centers of Reference for T1D manufactured cell therapies. “The initial purpose is to accelerate readiness of healthcare professionals to deliver manufactured islet cell therapies once they become available,” explained Dr. Danne. “…making such a treatment a success needs teamwork. Accredited Centers of Reference will not only deliver advanced T1D treatments but also serve as a training hub for professionals aspiring to become experts.”

This workshop focused on better understanding what potential Centers of Reference need to be successful. The attendees covered a range of topics: what an ideal T1D care team might look like, the education and training required for experts in T1D manufactured islet cell therapy, and career development for early-stage T1D professionals.

The goal is to prepare expert clinicians—who are already doing islet cell transplants—to bring manufactured islet cell therapies into clinical practice at their institutions and others, once they have regulatory approval. These centers will serve as a benchmark for best practices in T1D manufactured cell therapy, establishing a network of expert teams to make sure that everyone who can benefit from manufactured cell replacement therapy is given the opportunity to consider it.

What the experts are saying

“We need to build consensus and teamwork. When manufactured cell therapies exist, it’s going to take significant coordination between endocrinologists, transplant surgeons, and people with T1D to ensure as many people as possible are benefiting from these therapies.”

Jon Odorico, M.D.

Professor of Surgery and Director of Pancreas and Islet Transplantation at University of Wisconsin Health Transplant Center

“We’re trying to solve access and awareness. There’s a definite gap between primary care endocrine diabetes specialists and transplant specialists…there’s so much more that we have to fill in for [people with T1D].”

Helen Nelson, BSN, RN, CCTC, CPTC

Program Manager, Organ Allocation/Clinical Triage and Pancreas Transplant Program at University of Wisconsin Health Transplant Center

“It’s going to be a significant problem if we have a cure but no one has access to it because no one can deliver it. We must work together—transplant surgeons, endocrinologists, researchers, everyone. It’s like building Cape Canaveral in anticipation of sending rockets into space.”

Peter Senior, MBBS, Ph.D.

Islet Transplant Endocrinologist, Professor in the Department of Medicine, and Director of the Alberta Diabetes Research Institute at the University of Alberta, Canada

“There is amazing excitement around creating cell therapies. People are excited about it. We must ensure that organizations like Breakthrough T1D bridge the gap between research and the T1D population so that there is no difference between an individual’s reality and what therapies are available.”

James Shaw, M.D., Ph.D.

Transplant Endocrinologist and Professor of Regenerative Medicine for Diabetes at the Institute of Transplantation, Newcastle upon Tyne, United Kingdom

This is just the beginning

Manufactured islet cell therapies are coming. We need teamwork to get these therapies into clinics so people with T1D don’t have to wait years to get them. This is why Breakthrough T1D is acting now: when the first manufactured islet cell therapy hits the market, multidisciplinary care teams around the world will be ready. These workshops—the first of many—will help accelerate the safe and effective integration of manufactured islet cell therapies into clinics.

“This way we will ensure that the medical community is ready to deliver manufactured cell therapies once they become more widely available.,” Dr. Danne said. Thanks to the hard work of the Medical Affairs team at Breakthrough T1D, this goal is in sight.

Project ACT series

This article is part of a series exploring the different ways that Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) is shaping the future of cell therapies for type 1 diabetes (T1D). The next article in the series will focus on Project ACT’s advocacy efforts to ensure there is a regulatory pathway to approval for these therapies and that they will be covered by payers.

Read last month’s article about challenges and solutions of T1D cell therapies.

Despite significant advances in treatments for T1D, our community still has significant unmet needs. Breakthrough T1D believes that novel cell therapies will transform T1D management, and Project ACT is how we’re going to make them a reality.

First-generation cell therapies, including FDA-approved, donor-derived Lantidra® and Vertex’s manufactured islet therapy in phase 1/2/3 clinical trials, VX-880 (Zimislecel), are incredibly promising. They have some limitations, including:

• There are not enough donor-derived islets to meet the needs of everyone with T1D.
• These therapies are only available to people with severe hypoglycemia unawareness and hypoglycemic events.
• The number of people who can receive these therapies is further limited in that they must be able to tolerate chronic, broad immunosuppression.

Current research efforts at the preclinical, clinical, and manufacturing levels are working to address these challenges. The ultimate goal is a future in which manufactured islet therapies exist in large supply, survive and produce insulin in the body after implantation, and remain protected from the immune system. Learn more about Breakthrough T1D’s Cell Therapies Program and take a closer look at what researchers are doing to turn these ideas into a reality.

Clinical trials to keep an eye on

Up-and-coming cell therapies for T1D are in the clinical pipeline and working their way towards the market, including many that Breakthrough T1D has contributed to. There are some highly anticipated (and currently enrolling!) trials that we have our eyes on right now, and we hope to see data soon. Read about each study in detail below or scroll down to see a summary table.

Recruiting cell therapies clinical trials

Where do we go from here?

The emergence of cell therapies for T1D in clinical trials is incredibly exciting for the T1D community. Advancements using deceased donor-derived islets are paving regulatory pathways for manufactured islet therapies—which are curing people with T1D in clinical trials—to make their way towards the market.

“One of the greatest effects that manufactured islet cell therapies will have for the T1D community is being able to think about their type 1 diabetes less,” explained Nicholas Mamrak, Ph.D., a scientist at Breakthrough T1D. This may soon be a reality for a subset of people with T1D as we drive towards the approval of a first-generation manufactured islet replacement therapy, reducing the day-to-day burden of managing blood glucose and insulin dosing.

The ultimate goal is to make sure manufactured islet therapies are available to everyone with T1D—ideally without the need for immunosuppression. This is the objective of Breakthrough T1D’s Project ACT.

Before cell therapies can become available for everyone, they must first receive regulatory approval. A key part of Project ACT is streamlining and advancing regulatory pathways for the treatment of T1D. To help achieve this, Advocacy and Regulatory leadership teams at Breakthrough T1D are working on a new publication capturing how day-to-day lives have changed for people with T1D who have received cell therapies. By using personal experiences to share what benefits of cell therapies matter most to patients, we can help regulators understand the powerful impact that islet replacement therapies can have.

None of this would have been possible without the T1D community’s continued generosity and support, as we all work together to move the needle forward.

Have an impact by participating in clinical trials

Without clinical trials, we would never know if new therapies developed by scientists in the lab could make a difference in people’s lives. This is where the T1D community comes in—by volunteering to participate in clinical trials, you become uniquely positioned to help drive biomedical research forward. Moreover, by participating, you help not only yourself, but everyone with T1D. Find a clinical trial near you and see if you are eligible to participate. Connect with a Clinical Trial Education Volunteer in your area, who can answer any questions you may have.

Project ACT series

This article is part of a series exploring the different ways that Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) will shape the future of cell therapies for type 1 diabetes (T1D). The next article in the series will discuss the progress of cell therapies in clinical trials.

We’ve made major progress in the development of cell replacement therapies for type 1 diabetes (T1D) over the past couple of decades. We know that manufactured islets, such as Vertex’s VX-880 (now Zimislecel), can restore insulin therapy independence and glucose control when implanted into people with T1D.

However, there is more work to do.

We need to make sure the cells survive and function, ideally without immunosuppression, and ensure that these therapies are accessible to everyone with T1D. Read on to learn more about where there’s room for improvement and what we’re doing about it.

Optimizing manufactured islets

Priority #1: Cell source

Priority #2: Cell survival

Omentum

The omentum is a fatty tissue layer that surrounds and protects the organs in your abdomen. Researchers are testing it as a new implantation site for manufactured islets.

Priority #3: Cell protection

The future of manufactured islets and T1D cures

There’s a lot of promising solutions in the pipeline. What’s next? Unlocking access.

“We recognize that approval of cures is not a life-changing breakthrough if people do not have access to the therapy itself,” said Aaron Turner-Phifer, Senior Director of Health Policy at Breakthrough T1D. “Building on the experience gained from past breakthroughs, we are working now, across our Mission teams, to identify and remove any potential barriers to people accessing cell therapies.”

“While our work is just beginning, we’ve already conducted market analysis to identify clinical and payer barriers to give us clarity on where to start. We are directly engaging policymakers and health plans to educate them on T1D cell therapies. We’ve also launched data projects to begin to generate the types of data required to positively inform future policy and coverage decisions.”

Aaron Turner-Phifer

In the coming years, it’s likely that first-generation manufactured islet therapies will be available to people with T1D with severe hypoglycemia unawareness and will require broad immunosuppression.

Later, advancements in cell survival and immune protection—combined with the advent of more tolerable immune suppression approaches—will open the doors for more people with T1D to access these life-changing therapies.

Without continuous support from the T1D community and its supporters, we would never have gotten this far. Breakthrough T1D looks forward to a future where manufactured islet therapies are a reality for everyone with T1D, and we will not stop until we get there.

Disease-modifying therapies

Also "DMTs" for short, these therapies prevent, slow, halt, or reverse T1D progression.

With the establishment of our Medical Affairs team, we are reaffirming our organization’s commitment to creating a world where every individual with type 1 diabetes has access to life-changing therapies. By addressing systemic barriers and fostering clinical readiness, Breakthrough T1D will be pivotal in driving the timely adoption of emerging therapies and transforming care.” 

Thomas Danne, M.D. Breakthrough T1D Chief Medical Officer, International

Our goal is to make this education as accessible as possible.”

Julianne Lally, DMSc, MHS, PA-C Breakthrough T1D Associate Director of Community Screening and Clinical Trial Education

Detecting T1D before symptoms present

A simple blood test can detect T1D in the earlier stages—before obvious symptoms develop. The biggest challenge is educating clinicians and the general population about it.  

“Endocrinologists, Pediatricians, and some other specialty physicians learn about T1D screening and monitoring during their residencies, but it’s not a part of the general curriculum of the first four years of medical school,” said Lally, who built the learning management system for the new resources and is organizing the virtual offerings. “We’re working to advance that knowledge to yes, doctors, but also other clinicians whose patients could benefit.”

Many clinicians hesitate to order unfamiliar tests—especially if they are unsure what to do with the results. Most people who see any kind of healthcare provider could benefit from screening for T1D—according to a paper published in the journal US Endocrinology, roughly 85 percent of people diagnosed with type 1 do not have a blood relative with the autoimmune disease. 

“Clinicians need to learn about the stages of type 1 and the specific autoantibody tests that identify type 1 versus type 2 and identifying type 1 in individuals at risk before they need insulin,” said Colleen Buggs-Saxton, M.D. Ph.D.

Buggs-Saxton, a Pediatric Endocrinologist at Wayne Pediatrics in Michigan, is the clinical leader of a Breakthrough T1D Early Detection pilot clinic. Using the new resources, she and Albanese-O’Neill are going to lead a grand rounds about T1D early detection at her institution, which is affiliated with the Wayne State University School of Medicine and healthcare system. 

Clinicians should consider autoantibody testing for adults who have been diagnosed with type 2 but don’t have typical clinical features and require insulin to manage their blood sugars.” 

Colleen Buggs-Saxton, M.D., Ph.D. Pediatric Endocrinologist

Grand rounds

Grand rounds are educational meetings and presentations for clinical teams at a given institution or healthcare facility to provide a summary of updates to the standards of care.

I think of research, advocacy, and medical affairs as three legs of a stool—in terms of clinical adoption, each helps answer a different question. Research: Does it work? Advocacy: Will the regulators approve it and will insurance companies cover it? Medical Affairs: Do clinical teams have the competency and readiness to prescribe the treatment and educate and support people with T1D?” 

Anastasia Albanese-O’Neill, Ph.D., APRN, CDCES Breakthrough T1D Associate Vice President of Community Screening and Clinical Trial Education

The most challenging part is helping patients understand what a clinical trial is, what it involves, and how previous scientific advances were only possible because of clinical trials. Healthcare providers often don’t have enough time in their busy clinics to discuss this with patients and families.”

Laura Jacobsen, M.D. Pediatric Endocrinologist

Cell Therapies

Also called islet cell therapies, these therapies replace destroyed beta cells so that people with T1D can again produce their own insulin.