Objective
The objective of this study is to carefully evaluate the safety and performance of an investigational implantable device designed to protect insulin producing cells in adults with Type 1 diabetes.
This research aims to understand how the device interacts with the human body, including how surrounding tissue responds to the implant and whether insulin producing cells can remain viable inside the device over time. The study will focus on whether the device can reduce scarring at the implant site, support healthy blood vessel growth around the implant, and shield the cells from immune attack without the use of lifelong immune suppressing medications.
A key objective is to compare how the device performs under different immune conditions. Some participants in the study will already be receiving standard immune suppressing medications as part of routine islet transplantation care, while others will receive the implant without any immune suppression. This comparison is designed to help researchers understand the role of the immune system in cell survival and device performance and to identify factors that are important for future development of immune protected cell therapies.
This study is an early stage clinical investigation. It is not designed to restore insulin independence or reduce daily insulin use at this time. Instead, the objective is to collect foundational information about safety, tissue response, immune interaction, and early biological signals related to insulin production. These data will be used to guide future studies that may explore higher cell doses, longer implant durations, or different cell sources.
By focusing on careful measurement and scientific learning, this study aims to address long standing challenges that have limited previous cell based approaches for Type 1 diabetes. The knowledge gained from this research is intended to inform the design of future clinical trials and to support the responsible development of new therapies that may one day improve quality of life for people living with Type 1 diabetes.
Background Rationale
Type 1 diabetes (T1D) is a lifelong condition caused by the immune system attacking and destroying the insulin producing cells in the pancreas. Without these cells, the body cannot regulate blood sugar on its own, and people with T1D must rely on insulin therapy every day. Although insulin delivery methods and monitoring technologies have improved significantly, they cannot fully replicate the body’s natural glucose regulation. Many individuals continue to experience blood sugar fluctuations, hypoglycemia, and long term complications despite careful management.
One approach being studied to address this challenge is cell replacement therapy. In this approach, insulin producing cells are introduced into the body with the goal of restoring natural insulin production. Clinical islet transplantation has shown that this concept can work, but it requires lifelong immune suppressing medications to prevent rejection. These medications carry significant risks and limit this option to a small number of people with severe disease who are treated at specialized centers.
To avoid the need for immune suppression, researchers have explored ways to physically protect transplanted cells using encapsulation devices. These devices are designed to allow insulin and nutrients to pass through while blocking immune cells. However, past encapsulation approaches have not succeeded in people because the body often responds by forming scar tissue around the implant. This scarring interferes with oxygen and nutrient delivery, leading to loss of cell function and survival.
Encellin is developing an implantable encapsulation device designed to address these limitations. The device uses a thin, semi permeable membrane intended to reduce scarring and support healthy blood vessel growth around the implant. It is placed under the skin and can be removed if needed, which is an important safety feature. This design is intended to create a supportive environment for insulin producing cells while limiting immune attack.
Early stage clinical research with this device has provided initial information about how it interacts with the human body. Observations from an ongoing study suggest that the device can integrate with surrounding tissue without developing dense scar tissue. Analyses of devices removed from participants have identified insulin producing cells remaining within the device. These findings are preliminary and do not demonstrate clinical benefit, but they support continued investigation of this approach.
The rationale for the proposed study is to build on these early observations by carefully evaluating safety, tissue response, immune interaction, and early biological signals in a controlled clinical setting. By studying participants with and without immune suppressing medications, the research aims to better understand how immune protection and tissue integration influence cell survival. This information is necessary to guide future studies and to responsibly advance the development of immune protected cell therapies for T1D.
Description of Project
Type 1 diabetes (T1D) is caused by the immune system mistakenly destroying the insulin producing cells in the pancreas. As a result, people with T1D must rely on lifelong insulin therapy to manage their blood sugar. While insulin and diabetes technologies have improved daily care, they do not fully replicate the body’s natural ability to regulate glucose. For this reason, researchers continue to explore new approaches to safely restore insulin producing cells in people with T1D.
One area of research involves transplanting insulin producing cells into the body. These cells have the potential to respond naturally to blood sugar levels, but the immune system typically recognizes them as foreign and attacks them. Previous efforts to protect transplanted cells using encapsulation devices have faced challenges because the body often forms scar tissue around implanted materials. This scarring can block oxygen and nutrients, which limits cell survival and function.
Encellin is developing an implantable device designed to protect insulin producing cells while allowing essential molecules, including oxygen, nutrients, and insulin, to pass through. The device is intended to reduce the scarring response that has limited earlier approaches and can be removed if needed, which is an important safety feature. The long-term goal of this approach is to support transplanted cells without the use of lifelong immune suppressing medications.
In an ongoing early stage clinical study, Encellin is evaluating how this device interacts with the human body in people with T1D. Early findings suggest that the device can integrate with surrounding tissue and blood vessels without forming dense scar tissue. Analyses of devices removed during the study have identified insulin producing cells still present within the device. These findings are preliminary, but they provide early insight into how the device behaves in the human body and whether it can support cell survival.
The proposed research builds carefully on these observations. This study is designed to evaluate safety, tissue response, immune interaction, and early biological signals related to insulin production using blood tests. It is not designed to reduce or eliminate the need for insulin therapy at this stage. Instead, the goal is to understand whether this approach can safely support living cells over time and to gather information that will guide future studies.
This work represents an important early step in addressing long-standing biological challenges in cell based therapies for T1D. By focusing on safety, careful measurement, and scientific learning, this research aims to generate the knowledge needed to inform larger and longer term studies. While significant research remains ahead, these efforts are intended to help lay the groundwork for future therapies that could improve quality of life for people living with T1D.
Anticipated Outcome
The anticipated outcome of this study is to gain important early information about the safety and biological behavior of an investigational implantable device designed to protect insulin producing cells in people with Type 1 diabetes. Because this is an early stage clinical study, it is not expected to produce clinical benefit or reduce the need for insulin therapy. Instead, the primary outcomes relate to understanding how the device performs in the human body and whether it can support insulin producing cells in a controlled and measurable way. Key anticipated findings include confirmation that the device can be implanted and removed safely, that surrounding tissue responds in a predictable and acceptable manner, and that excessive scar tissue does not develop at the implant site.
Another anticipated outcome is improved understanding of how the immune system interacts with the device and the cells it contains. By studying participants who receive immune suppressing medications as part of standard care alongside participants who do not receive immune suppression, the study aims to clarify how immune activity influences tissue response and cell survival. These observations are expected to provide valuable insight into the conditions required for immune protected cell therapies to function safely.
The study is also expected to generate descriptive biological data related to insulin production. Measurements such as blood markers will be used to explore whether insulin producing cells remain viable and show early biological activity while inside the device. These measurements are exploratory and will be interpreted cautiously, but they are important for understanding how living cells behave within the protected environment created by the implant.
In addition, detailed examination of the device after it is removed will provide direct information about tissue integration, blood vessel growth, immune cell interaction, and cell viability. This type of analysis is rarely possible in human studies and is expected to offer insights that cannot be obtained through blood tests alone. These findings will help identify design features that support favorable tissue responses and will inform future improvements to the device.
Overall, the anticipated outcome of this research is to generate foundational knowledge needed to guide the next stages of development for immune protected cell therapies. If the study demonstrates acceptable safety and biological performance, it would support further clinical investigation using higher cell doses, longer implant durations, or different cell sources in future trials. While significant research remains ahead, the information gained from this study is intended to reduce uncertainty, inform responsible decision making, and contribute to the long term goal of improving treatment options for people living with Type 1 diabetes.
Relevance to T1D
Type 1 diabetes (T1D) is a lifelong autoimmune condition in which the body can no longer produce insulin. People with T1D must manage their blood sugar every day using insulin therapy, careful monitoring, and ongoing decision making. Even with modern technologies such as continuous glucose monitors and automated insulin delivery systems, many individuals experience unpredictable blood sugar swings, hypoglycemia, and long term health complications.
A major challenge in T1D care is that current treatments replace insulin, but they do not restore the body’s natural ability to produce insulin in response to changing glucose levels. Restoring insulin producing cells could offer a more physiologic approach to managing the disease. However, past attempts to transplant insulin producing cells have required lifelong immune suppressing medications or have failed due to immune rejection and scarring around implanted devices.
This study is directly relevant to T1D because it focuses on understanding whether insulin producing cells can be supported safely in people with T1D without the use of systemic immune suppression. The research addresses key biological barriers that have limited previous cell based approaches, including immune attack, tissue scarring, and poor integration of implanted materials with the body.
By evaluating an implantable device in adults with T1D, this study seeks to better understand how the immune system and surrounding tissue respond to a protected environment designed for insulin producing cells. The inclusion of participants both with and without immune suppressing medications allows researchers to explore how immune activity affects cell survival and tissue response. This knowledge is important for determining whether future therapies could be developed for broader use in the T1D community.
Although this early stage study is not designed to improve blood sugar control or reduce insulin use, it is highly relevant because it focuses on foundational questions that must be answered before more advanced therapies can be responsibly developed. The findings are expected to inform future clinical trials, guide device and study design, and help reduce risks associated with later stage research.
Ultimately, this research is relevant to T1D because it aims to move the field closer to therapies that address the underlying loss of insulin producing cells rather than only managing symptoms. By prioritizing safety, careful measurement, and scientific understanding, this study contributes to long term efforts to expand treatment options and improve quality of life for people living with Type 1 diabetes.