Objective
The goal of this project is to help bring a promising new treatment for Type 1 Diabetes (T1D) one step closer to patients. T1D is a disease where the body’s immune system destroys the insulin-producing cells in the pancreas, making it impossible to regulate blood sugar levels without constant monitoring and daily insulin injections. While insulin therapy can help manage the disease, it does not fully prevent long-term complications such as heart disease, kidney damage, nerve problems, and vision loss. For many, T1D is a lifelong challenge with no cure.
Researchers have shown that replacing the lost insulin-producing cell can restore the body’s ability to control blood sugar. Some patients who received islet transplants from deceased donors have gone years without needing insulin shots. However, the number of available donor organs is very limited, and each patient typically needs cells from multiple donors. This makes islet transplantation an option for only a small number of people with severe complications, leaving most patients without access to this therapy.
To solve this, scientists at Washington University in St. Louis are developing a new way to make these insulin-producing cells in the lab using stem cells. These special cells can grow indefinitely and be directed to become the same kinds of cells found in the pancreas—including the insulin-producing beta cells destroyed in T1D. The result is a lab-grown version of a human islet, known as a stem cell-derived islet (SC-islet). These SC-islets have already been shown to produce insulin in response to sugar and can even reverse diabetes in mice.
The objective of this project is to take these early scientific breakthroughs and develop them into a real-world solution that can be scaled up and used in hospitals and clinics. Specifically, the project has two main goals:
First, the researchers want to build a manufacturing process that can reliably produce very large numbers of high-quality SC-islet cells using strict safety and quality standards required for medical products. To treat a single patient, you need about a billion cells. The team is working to create more than that in each batch, using advanced bioreactor systems that gently grow the cells in the right conditions. They are also testing several different stem cell lines to find which ones are the most reliable and productive.
Second, the researchers aim to develop methods for safely storing and shipping these cells. Right now, many advanced cell therapies must be made and used at the same location, which limits access to only a few specialized centers. This project is working on two solutions: freezing the cells for later use (called cryopreservation) and shipping them at cold temperatures (like organ transplants) while keeping them alive and functional. These strategies would allow the cells to be made at centralized facilities and shipped to clinics across the country—or even around the world.
If successful, this project could transform the way T1D is treated. Patients would no longer need to wait for a donor. Instead, they could receive a transplant of insulin-producing cells made from stem cells in a lab, potentially freeing them from the burden of daily insulin injections. Importantly, the approach is designed to be scalable, safe, and practical, making it more likely to reach patients in a wide range of healthcare settings. This work is part of a growing effort to use regenerative medicine to treat chronic diseases by restoring lost or damaged cells with lab-grown replacements. It brings together cutting-edge stem cell science, bioengineering, and clinical research to build a therapy that is not only effective but also accessible.
Background Rationale
T1D is a lifelong autoimmune disease that affects millions of people around the world. It occurs when the body’s immune system mistakenly attacks and destroys the insulin-producing beta cells in the pancreas. Without insulin, the body cannot regulate blood sugar levels, which can lead to serious and sometimes life-threatening complications. People with T1D must rely on multiple daily insulin injections or continuous insulin pumps to stay alive, constantly monitor their blood sugar, and make ongoing adjustments to food, exercise, and stress. Even with careful management, T1D remains a difficult and dangerous condition that can lead to long-term health problems.
For decades, scientists have pursued a bold idea: what if we could replace the insulin-producing cells that are lost in T1D? In fact, this approach has already shown success in a small number of patients who received pancreatic islet cells from deceased organ donors. In many cases, these patients no longer needed insulin injections and could maintain healthy blood sugar levels on their own. However, there are simply not enough donors to make this therapy widely available. Each patient often needs islets from multiple donors, and donor tissue supply is extremely limited. As a result, islet transplantation is only offered to a small number of patients with the most severe cases.
To overcome this challenge, scientists have turned to human induced pluripotent stem cells (hiPSCs). These are special cells that can be created from a person’s own tissue and have the remarkable ability to turn into nearly any cell type in the body. In the lab, researchers can guide these hiPSCs to become insulin-producing beta cells, just like those lost in T1D. When organized into small clusters that mimic natural islets, called stem cell-derived islets (SC-islets), these lab-grown cells can respond to glucose and produce insulin, offering a potential cure for T1D.
Early studies have shown that SC-islets can effectively lower blood sugar levels in diabetic animal models. Even more exciting, recent clinical trials using similar cells have demonstrated that some patients with T1D can stop taking insulin after receiving SC-islet transplants. These results offer strong proof that stem cell-based therapies for T1D are not only possible but already starting to change lives.
However, significant challenges remain. Producing SC-islets at the scale needed to treat many patients while meeting the safety, quality, and regulatory standards required for clinical use is a complex task. The process must be carefully controlled, consistent from batch to batch, and capable of making billions of cells per patient. In addition, these cells must be preserved and transported in a way that keeps them alive and functional when they reach clinics across the country or around the world.
This project is designed to solve those challenges. The team at Washington University is developing a complete system for making, preserving, and shipping SC-islets for T1D therapy. They are working with clinically approved hiPSC lines and using advanced bioreactor systems that allow for large-scale growth under controlled conditions. They are also testing cryopreservation (freezing) and cold-chain shipping methods so that the cells can be stored and delivered without losing their effectiveness.
By addressing these bottlenecks in production and distribution, this project aims to make hiPSCs for T1D more practical, more reliable, and more accessible. The ultimate goal is to create a therapy that could one day be available to all people living with T1D, not just a select few. This work builds on decades of scientific discovery and brings us closer to a future where T1D can be managed not with insulin injections, but with a one-time, life-changing treatment that restores the body’s natural ability to control blood sugar.
Description of Project
Type 1 Diabetes (T1D) is a chronic condition where the body’s immune system mistakenly destroys the insulin-producing cells in the pancreas. People with T1D must take insulin for life, yet even the best treatments today cannot fully mimic the natural control of blood sugar, leading to long-term health complications. Scientists have shown that transplanting healthy insulin-producing cells, known as islets, can help some patients reduce or even eliminate the need for insulin shots. However, this approach has been limited because it relies on islets from deceased donors-a scarce and inconsistent source. This project, led by researchers at Washington University in St. Louis, aims to solve this problem by creating insulin-producing islets from human stem cells. These stem cells can grow indefinitely in the lab and be turned into many different cell types, including the insulin-producing beta cells lost in T1D. The goal is to build a renewable, high-quality, and scalable source of therapeutic cells that can be used by patients around the world. The researchers have already developed a way to grow these stem cell-derived islets (called “SC-islets”) in the lab. These SC-islets behave like natural islets. They produce insulin when exposed to sugar and can reverse diabetes in mouse models. Now, the team is taking the next big step: developing a reliable and clinically compliant manufacturing system that can make billions of SC-islet cells at once, enough to treat real patients.
The project has two main parts: 1) Large-Scale Manufacturing: The team will use advanced bioreactors—machines that can carefully control temperature, nutrients, and oxygen levels—to grow SC-islet cells in a way that meets clinical-grade standards. They will test several types of stem cells to find the most reliable and productive ones. Their goal is to consistently produce more than one billion cells per batch, roughly enough for one patient’s treatment, with all the right characteristics needed for therapy. 2) Storage and Shipping: To get these cells to patients across the country (and eventually the world), the team will also develop ways to safely freeze and ship them. This is similar to how organs are transported for transplant today, but even more delicate since these lab-grown cells must survive freezing, thawing, and transportation while still functioning correctly. The researchers will test different storage solutions and shipping conditions to ensure the cells arrive alive and ready to work.
If successful, this work will pave the way for a new kind of cell therapy for T1D—one that doesn’t depend on donor organs and can be produced at scale. It will allow insulin-producing cells to be grown in a lab, frozen for transport, and shipped to hospitals and clinics wherever they’re needed. This could dramatically expand access to cell-based treatments and help patients better manage or even eliminate their diabetes. This project also supports the larger goal of making advanced regenerative therapies more accessible and affordable. By developing scalable systems, regulatory-grade methods, and reliable distribution strategies, the team is building a foundation for future clinical trials and, eventually, commercial products. In time, these breakthroughs could lead to a world where people with T1D are no longer dependent on daily insulin and are instead offered a lasting, biologically based treatment.
Anticipated Outcome
This project aims to develop a reliable, large-scale method for producing insulin-producing cells from stem cells to treat Type 1 Diabetes (T1D). The anticipated outcome is a transformative step toward making cell therapy for T1D widely available, not just for a few patients at specialized hospitals, but for people everywhere who live with this lifelong condition.
Right now, managing T1D requires constant attention. People must regularly check their blood sugar, adjust their insulin levels, monitor food intake, and stay alert for dangerous highs and lows. Even with cutting-edge technology like continuous glucose monitors and insulin pumps, life with T1D is never easy. But what if we could go beyond managing the disease and actually replace the cells that are missing?
Scientists have already demonstrated that this is possible using donor cells from deceased individuals. In clinical trials, people who received transplants of pancreatic islet cells (the insulin-producing clusters in the pancreas) were able to stop taking insulin altogether for months or even years. Unfortunately, donor tissue is incredibly scarce, and the process is complex and expensive. That’s where stem cells come in.
The goal of this project is to turn stem cells into insulin-producing cells, called stem cell-derived islets (SC-islets), and make them at a scale that could eventually treat thousands, or even millions, of people. The research team at Washington University is building a high-tech, highly controlled manufacturing process that can produce billions of these cells at once, using clinical-grade materials and methods that meet the strict safety standards required for human therapies.
If successful, the team will achieve several key outcomes:
1) The team will demonstrate that it’s possible to grow large batches of SC-islets, enough for a single patient or more, in a way that is safe, consistent, and suitable for future clinical trials. They aim to use multiple lines of stem cells that comply with FDA guidelines and produce high-quality insulin-producing cells with the characteristics needed for therapeutic use.
2) Another major goal is to create standardized methods for preserving and transporting the cells. This includes freezing the cells so they can be stored long-term (called cryopreservation) and shipping them live at cold temperatures. These systems will allow SC-islets to be made in one place, like a central manufacturing facility, and delivered safely to hospitals and clinics across the country or even internationally.
3) By solving these production and logistics challenges, the project sets the stage for making this therapy accessible to more people—not just those near a major medical center. This opens the door for a more equitable future in which children, adults, and families affected by T1D can benefit from cell therapy regardless of where they live.
4) The work completed through this project will also provide the foundational data needed to support future human trials. This includes establishing benchmarks for cell quality, potency, and safety. The results will help shape regulatory strategies and pave the way for the therapy to move from the lab to the clinic and eventually to the marketplace.
Ultimately, the anticipated outcome is not just a new therapy, but a new model for how life-changing regenerative medicine can be delivered at scale. This project aims to reduce dependence on donor organs, minimize the burden of daily insulin therapy, and bring new hope to millions of people living with Type 1 Diabetes. It represents a bold step toward a future where diabetes can be treated not just by managing symptoms, but by restoring the body’s own ability to control blood sugar—with the potential for long-term independence from insulin.
Relevance to T1D
T1D is a serious autoimmune disease that affects people of all ages, often beginning in childhood. In T1D, the immune system mistakenly attacks and destroys the beta cells in the pancreas that produce insulin, a hormone needed to control blood sugar levels. Without insulin, the body cannot regulate blood sugar, leading to dangerously high or low glucose levels and long-term complications like heart disease, nerve damage, blindness, and kidney failure.
For over a century, people with T1D have relied on insulin injections to survive. While modern tools such as continuous glucose monitors and insulin pumps have improved care, they still fall short of providing natural blood sugar regulation. Managing T1D remains a 24/7 job that includes frequent blood checks, dietary planning, and constant vigilance. Despite their best efforts, many people still face risks of emergency situations and serious health problems over time.
This is where the research described in this project becomes especially relevant. The ultimate goal is to restore the body’s natural insulin production by replacing the cells that were destroyed—something that would represent a true cure rather than ongoing treatment. One promising strategy is to transplant insulin-producing cells, called islets, into people with T1D. In some clinical studies using donor islets from deceased individuals, patients have been able to stop taking insulin altogether. However, this therapy is limited to only a few people with severe complications due to the scarcity of donor organs.
The innovation at the heart of this research is the ability to create insulin-producing cells from stem cells in the lab. These stem cells can be programmed to develop into “stem cell-derived islets” (SC-islets), which function like the pancreas’s own insulin-producing beta cells. SC-islets respond to sugar in the blood by releasing insulin, just like healthy islets would. In early studies, these lab-grown cells have already been shown to reverse diabetes in animal models and produce insulin in clinical trial participants.
But creating these cells in the lab is only part of the challenge. The big question now is: can we make enough of them, safely and reliably, to treat many patients, not just a handful?
This project directly tackles that question. The research team is working to build a robust and scalable system for producing SC-islets in large quantities using clinical-grade materials and processes that meet the strict safety standards set by the FDA. Each person with T1D may need a billion or more of these cells, so being able to produce them at this scale is essential. Just as important, the team is developing methods to freeze, store, and ship the cells so they can be delivered safely to clinics far from where they were made.
The relevance of this work to people with T1D is profound. By creating a renewable source of insulin-producing cells and solving the manufacturing and distribution challenges, this research lays the foundation for making SC-islet therapy a practical and accessible option. If successful, it would mean a future where T1D could be treated with a one-time or occasional cell transplant, freeing people from the burdens of daily insulin therapy and improving their long-term health outcomes.
In short, this project offers real hope to the millions of people and families affected by T1D. It brings together years of cutting-edge research, bioengineering, and clinical experience to build a therapy that not only works in the lab but could soon reach those who need it most. By addressing both the scientific and logistical barriers, this work is helping transform the promise of stem cell science into a real, scalable solution for curing T1D.