Project ACT series

This article is the last 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).

Read last month’s article about the role of Advocacy in cell therapies for T1D.

Cell therapies, which place insulin-producing cells back into the bodies of people with T1D, are making major headway in clinical trials—and people are becoming insulin independent. In past installments of this article series, we’ve explored how we got to this point at every step in the pipeline: from advocating for funding, to conducting research in laboratories, to clinical testing in humans, and to working with regulators and insurers.

But we’re not at the finish line yet. Approval of manufactured cell therapies isn’t enough to make sure that people who want these cells can get them. So, how do we fill this gap?

Medical Affairs is the backbone of clinical adoption

The answer is Medical Affairs—Breakthrough T1D’s newest mission pillar—which aims to support the clinical adoption of manufactured cell therapies. As we are soon approaching the regulatory submission of a first-generation manufactured cell therapy, Medical Affairs initiatives will be especially important in making sure that healthcare professionals (HCPs) have the tools they need to bring these therapies to clinics. By partnering with global experts and HCPs to increase knowledge and clinical competencies, the Medical Affairs team is working to ensure the future delivery of manufactured cell therapies to eligible people with T1D.

Learn more about why Medical Affairs is important for every treatment, therapy, drug, and device for T1D.

Preparation is key

Since manufactured cell therapies for T1D are not yet FDA-approved, we are at a critical moment where preparation is essential. This is why the Medical Affairs team is planning now for the eventual arrival of first-generation manufactured cell therapies. They are partnering with advanced clinical teams to determine best practices to ensure that clinics have the right resources and skillsets. They are anticipating obstacles—and finding solutions—to the adoption of manufactured cell therapies. Then, when these therapies become available, HCPs will be ready and waiting to deliver them to people with T1D.

Assembling expert clinical teams and facilities

For people with T1D to have access to manufactured cell therapies, comprehensive expert clinical care teams need to be at the ready. To make this possible, we are starting at the very beginning: in training programs.

New, updated programs and fellowships must incorporate specialized education around cell therapies (such as optimized immunosuppression regimens and pre- and post-transplant education) for the next generation of HCPs. At a time when there are too few endocrinologists and certified diabetes care and education specialists, there’s no better time than now to encourage trainees to enter the field. We need to ensure that T1D care teams have trained professionals who are up to pace with the newest cell therapy protocols.

In line with these efforts, the Medical Affairs team is also working to recognize expert multidisciplinary Centers of Reference, which are a network of T1D treatment centers that have the personnel, resources, facilities, and skills to deliver manufactured cell therapies to people with T1D with a consistent approach. These centers will act as training hubs and share outcomes to continuously improve and establish best practices as manufactured cell therapies are incorporated into clinics.

Guidelines, statements, workshops, and more

The Medical Affairs team is working with experts in the field on publications that will guide care teams to further support the standardized integration of manufactured cell therapies into clinical practice.

Clinical guidelines

Clinical guidelines are recommendations for clinicians about how to care for people with T1D. They are informed by a systemic review of evidence by experts to assess benefits versus risk of care options.

Consensus statements

Consensus statements are collective opinions of an expert panel representing the multidisciplinary care team and voice of people with T1D. These statements are developed when evidence is still being gathered.

To drive these efforts, the Medical Affairs team hosts workshops, convening international experts to collaborate and discuss ideas. Using established methodologies, these projects ensure that the final document reflects expert consensus. One of the latest workshops assembled islet cell transplantation professionals from around the world to begin developing a consensus on the clinical roadmap of cell replacement therapies—the first step of many toward clinical adoption.

Read more about the most recent Medical Affairs workshops, including one geared toward creating Centers of Reference. See a recent example of clinical guidelines for monitoring early-stage T1D.

Opportunities to learn

Through decades of research, we’ve learned a lot about T1D—and the newest therapies and treatments are as innovative and complex as ever. To keep up with the rapidly changing pace of innovation, the Medical Affairs team has put together opportunities for HCPs learn about cutting-edge advancements.

These HCP resources offer accredited, free, live or on-demand education around the latest T1D research. There is also information on upcoming conferences, published guidelines, and clinical trial opportunities, in addition to an email newsletter with regular updates. Accredited HCP education is essential to ensure that clinicians have the most up-to-date knowledge that they can share with the T1D community—an important step in clinical adoption. Cell therapy resources are currently under development and expected in the next year.

Clinical trial education and awareness

Clinical trials are a key step in ensuring that new and effective T1D treatments and cures, including manufactured cell therapies, can safely make their way to the T1D community. The Medical Affairs team is working to foster a culture of research participation by ensuring that HCPs have access to information on current clinical trials and can help to educate the T1D community on their importance.

The ultimate goal is to increase awareness about clinical trial opportunities and support shared decision-making between people with T1D and their diabetes care team about clinical trial participation. The bottom line? HCPs and the T1D community must be equipped with information to enable them to make informed choices about participating in cell therapy clinical trials.  

Medical Affairs help turn groundbreaking science into real care—by supporting doctors, educating communities, and making sure cell therapies for type 1 diabetes reach the people who need them most.”

Thomas Danne, M.D., Ph.D. Chief Medical Officer, Global

The bigger picture: Project ACT across the pipeline

In this series, we’ve explored how manufactured cell therapies are moving through every step of the pipeline. We took a deep dive into the challenges that researchers are addressing to optimize manufactured cells. We surveyed the different cell therapies that are in currently clinical testing. We reviewed how advocacy shapes funding, regulatory decisions, and access to cell therapies. Finally, we considered how to ensure the clinical adoption of manufactured cell therapies.

This is where Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) comes in. The ultimate goal is to accelerate manufactured cell therapies that do not require immunosuppression—all the way from the first discovery research in the lab to the adoption of cell therapies in clinics.

At every step of the way, we are moving cell therapies through the pipeline as fast as possible. Breakthrough T1D is preparing for the day when first generation manufactured cell therapies are available, and we are working toward a future with next generations of therapies that do not require immunosuppression. Thanks to concerted efforts by Breakthrough T1D’s Research, Advocacy, and Medical Affairs teams, each day we move closer to manufactured cell therapies becoming a reality for everyone with T1D.

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.

Type 1 diabetes (T1D) is caused by the autoimmune destruction of insulin-producing beta cells in the pancreas. People with T1D are dependent on insulin therapy to control their blood sugar levels, which is critical to prevent complications that occur when blood sugar is too high (hyperglycemia) or too low (hypoglycemia).

The best bet for T1D cures depends on cell therapies, which replace destroyed beta cells with protected, functional cells to restore insulin therapy independence and glucose control, ideally without chronic immunosuppression. Breakthrough T1D’s Cures Program has been instrumental in the incredible progress we’ve made in cell therapies research. The secret to success? Stem cells. 

What are stem cells?

Stem cells are uniquely suited for use in cell therapies because of their biological characteristics. First, they are essentially a blank slate—we can biologically engineer them to become any cell we want, including beta cells. Second, stem cells can make copies of themselves while remaining a blank slate, meaning we can generate an unlimited source of transformable cells.

Since scientists first isolated human stem cells in 1998, there has been major progress in therapies that can replace or renew damaged and diseased tissue. For example, people with blood cancer often undergo chemotherapy and radiation to destroy cancerous blood cells. Stem cells are delivered directly into the bloodstream and travel to the bone marrow, where they transform into new, non-cancerous blood cells, replacing the important cells that were lost with healthy ones. The exciting progress in stem cell research extends to T1D—researchers have harnessed the power of stem cells to generate functional beta cells and islets, rapidly accelerating the drive toward T1D cures. Breakthrough T1D has been at the forefront of stem cell research in T1D for decades. 

Stem cells are the foundation of our Cell Therapies Program 

Breakthrough T1D’s Cell Therapies Program focuses solely on making and improving stem cell-derived beta cells. For stem cell-based therapies to become a reality for everyone with T1D, there are three primary goals that we are striving to achieve:

• First, we need to generate a renewable, scalable source of beta cells so that there are enough for everyone who needs them.
• Second, we need to find a habitable site to implant beta cells so that they remain functional and healthy for very long periods of time.
• Third, we must find a way to protect implanted beta cells from immune attack so that people won’t need to take chronic anti-rejection immunosuppressants, which can come with intolerable side effects.

Breakthrough T1D has put tremendous effort into achieving these goals; we’ve seen incredible progress in recent years, pushing us closer than ever to cures for T1D.

Breakthrough T1D has driven more than $156 million to cell therapies research in the past decade and has spearheaded several initiatives to drive this effort forward. In 2013, the Beta Cell Replacement Consortium was launched, bringing together 50+ of the brightest scientists and key industry players to integrate expertise in bioengineering, animal models, transplant medicine, and other key research areas.

Similarly, Breakthrough T1D established several Centers of Excellence, a collection of institutions driving exceptional advancements in immunology, stem cell biology, and gene editing, all of which are critically important to cell therapies research. The ultimate goal of these initiatives is to foster collaboration, exchange resources and data, and accelerate the development of stem cell-derived islet therapies. 

Looking forward

With Breakthrough T1D’s commitment to driving cell therapies forward, we have made significant headway in the development of life-changing therapies that can place healthy, insulin-producing beta cells back into people with T1D. “Even with today’s fantastic automated insulin delivery systems and advanced algorithms, those living with T1D still spend a significant amount of time interacting with their devices in order to maintain blood sugar control,” said Nicholas Mamrak, a scientist at Breakthrough T1D. “The prospect of cell therapies lies in the ability to take off these pumps and spend less time managing and worrying about their diabetes.”

The drive toward stem cell-based therapies becoming a reality for everyone with T1D continues—despite how far we’ve come, we still have more work to do.

This article is the first 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). Stay tuned for the next article, which will explore the present and future of cell therapies for T1D.

Today, Sana Biotechnology released significant clinical data: the first person with type 1 diabetes (T1D) who received deceased donor islets engineered to evade the immune system is producing insulin without immunosuppression.

The details

This is a big step for cell-based therapies for T1D. Sana’s first-in-human study consists of allogeneic islets, meaning they are derived from an external source, which in this case is the pancreases of deceased donors. These islets were engineered to avoid recognition by the immune system (hypoimmune) and were implanted intramuscularly into a person with T1D. After four weeks, circulating C-peptide increased, meaning that the beta cells are alive, healthy, and producing insulin—all without the need for immunosuppression and no safety issue. This is the first evidence of engineered islets successfully avoiding immune destruction.

What this means for the T1D community

While this is an incredibly promising step forward for the T1D community, currently available cell therapies that rely on deceased donor islets (Lantidra®) are only accessible to a small portion of the T1D population because there are very few donor cells available. They also require broad anti-rejection immunosuppressants, which can come with serious side effects that may not be manageable for everyone with T1D. Engineering cells to evade immune attack is a new path forward to protect the insulin-producing beta cells and avoid the use of immunosuppressants. Most importantly, this technology can now be applied to stem cell-based therapies, which is a scalable solution for many people with T1D.

What’s next: lots to look forward to

Breakthrough T1D believes that the best bet for type 1 diabetes cures lies in stem cell-based therapies since deceased donor islets are in short supply, while stem cell-derived islets can be produced at scale. We have now opened the doors to apply hypoimmune technology to stem cell-derived islets, moving us closer to the possibility of having enough immune-evading cells for everyone with T1D. While this will take significant time, effort, and money, every day we take another step toward a possible life-changing T1D cure. 

Breakthrough T1D’s role

The primary objective of Breakthrough T1D’s beta cell replacement efforts is to place insulin-producing cells into people with T1D without the use of immunosuppressants. Breakthrough T1D strongly supports the development of stem cell-based therapies that do not require broad immunosuppression and recently launched an initiative to accelerate this faster than ever (Project ACT – Accelerate Cell Therapies). To contribute to the advancement of these game-changing therapies, the T1D Fund: A Breakthrough T1D Venture invested in Sana recognizing that their hypoimmune engineering technology held significant promise for type 1 diabetes cell therapies. We look forward to seeing how the trial progresses.

While we look back on 2024, we can reflect upon the incredible progress we’ve made in advancing breakthroughs toward cures and improving everyday life with T1D.  

This wouldn’t have been possible without each and every one of you and your continued support of our mission as we drive toward cures for T1D.  

Here are the top 11 advances that together, we made happen in 2024: 

It’s certainly been an exciting year! While we still have more work to do, it’s crucial to celebrate our wins, both big and small, to see how far we’ve come in our push to make T1D a thing of the past.

Together, we’re accelerating breakthroughs for people with T1D, and the support of the T1D community drives our mission forward every single day, leading the way to lifechanging therapies and cures. Let’s see what 2025 has in store!