Objective
Our goal is to bring a new implantable device, called NICHE, from the laboratory into its first human study for people with Type 1 Diabetes (T1D). T1D occurs when the immune system destroys insulin-producing cells in the pancreas, forcing lifelong dependence on insulin injections. Transplanting healthy insulin-producing cells offers a potential cure, but current methods require lifelong systemic immunosuppressive drugs, which can cause serious side effects such as infections and kidney damage.
We developed NICHE to overcome these challenges. The device creates a small, vascularized “home” for transplanted insulin-producing cells under the skin. It allows new blood vessels to grow into the device before the cells are added, ensuring that oxygen and nutrients reach them. It also contains a separate reservoir that can deliver immunosuppressive drugs locally, only where they are needed, instead of throughout the body. This reservoir can be safely refilled through the skin, avoiding further surgery. The first human study will test whether the NICHE device can be used safely and whether it provides a supportive environment for the transplanted cells. We will enroll adults with long-standing T1D who have already received a kidney transplant and are therefore already taking systemic immunosuppressive drugs. This approach allows us to evaluate the implant’s safety and biological performance in a controlled setting.
If the study confirms that the NICHE device can safely integrate and support the survival of insulin-producing cells, future trials will evaluate the same device with localized immunosuppressive delivery only. These studies could lead to a new treatment that restores natural insulin production while avoiding the harmful side effects of systemic immunosuppression.
Background Rationale
Type 1 Diabetes (T1D) develops when the immune system mistakenly destroys the body’s insulin-producing cells. People with T1D must rely on insulin injections and constant blood-glucose monitoring to control their condition. While current technologies like insulin pumps and continuous glucose monitoring help manage the disease, they do not fully replicate the body’s natural ability to regulate blood sugar. Transplanting new insulin-producing cells could restore natural insulin production, but this procedure requires systemic immunosuppressive drugs to prevent rejection, drugs that can cause significant side effects over time.
We designed the NICHE device to overcome these limitations. NICHE is a small implant placed under the skin that provides a vascularized environment where transplanted cells can survive and function. The device has two compartments: one that holds the therapeutic cells and another that delivers small amounts of immunosuppressive drugs directly where they are needed. Because the drugs act locally rather than throughout the body, this approach aims to protect transplanted cells while minimizing toxicity. The device can be refilled noninvasively, allowing long-term use without repeated surgeries.
In this first human study, we will focus on safety and feasibility rather than treatment. The trial will include participants who already take immunosuppressive medication due to prior kidney transplantation, making them ideal for early evaluation of the device. This design allows us to assess vascularization, cell survival, and local immune response without adding systemic drug exposure.
By carefully testing each component of the NICHE system step by step, we aim to build a foundation for future trials that combine local immunosuppression with fully functional insulin-producing cells. If successful, NICHE could make cell replacement therapy for T1D safer, more durable, and accessible to more patients.
Description of Project
Type 1 diabetes (T1D) is a chronic disease in which the body’s immune system destroys insulin-producing cells in the pancreas, forcing patients to rely on lifelong insulin injections to control blood sugar. While insulin therapy helps, it cannot perfectly replicate the body’s natural regulation of glucose, leaving patients vulnerable to serious complications such as nerve damage, kidney failure, and heart disease. One promising approach is pancreatic islet transplantation, in which healthy insulin-producing cells are introduced into the body to restore insulin production. However, this approach has been limited by immune rejection and the need for toxic systemic immunosuppressive drugs.
The NICHE (Neovascularized Implantable Cell Homing and Encapsulation) technology is designed to overcome these challenges. NICHE is a small, implantable device that provides a nurturing environment for transplanted insulin-producing cells while delivering immune-protecting drugs locally, only where needed. This targeted drug delivery minimizes the need for whole-body immunosuppression and its side effects. The device can also be refilled with medication through a minimally invasive procedure, allowing long-term maintenance without additional surgeries.
Our team has demonstrated the safety and effectiveness of NICHE in preclinical studies, showing that it can support healthy islet cells, restore insulin production, and remain stable over time in animal models. The next step is to translate this innovation into clinical use.
In this project, we will begin with sentinel NICHE devices, smaller versions of the full NICHE system. These smaller devices will be used to test the key functional aspects and feasibility of the NICHE concept in a minimally invasive way. The data obtained from these sentinel implants will provide essential knowledge about tissue integration, vascularization, and drug distribution, which will guide and finalize the design of the full-size therapeutic NICHE product intended for long-term insulin replacement.
This milestone-driven program will include advanced preclinical testing and a first-in-human clinical study in adults with T1D. By integrating engineering, drug delivery, and cell therapy, NICHE has the potential to transform diabetes care, offering a safer, more durable, and physiologically relevant treatment that could dramatically improve the quality of life for individuals with T1D.
Anticipated Outcome
We expect this first-in-human study to demonstrate that the NICHE Sentinel implant can be safely used and can support early survival of transplanted insulin-producing cells under the skin. Our main focus is to verify that the device integrates well with surrounding tissues, becomes naturally vascularized, and provides a healthy environment for the cells. The goal is not to achieve a cure at this stage, but to confirm that the design functions safely in people.
Each participant will receive three small NICHE implants. One will be removed soon after implantation to confirm successful blood vessel growth, and the other two will be loaded with donor-derived islet cells and removed at approximately three and six months. We will analyze these samples to assess vascularization, immune response, and islet viability. We will also monitor healing, local tissue reactions, and any side effects or complications during follow-up. If the results confirm safety and successful vascularization, the next study will evaluate NICHE combined with localized immunosuppressive drug delivery. This would allow us to test whether transplanted islets can survive long-term using only local immune protection, eliminating the need for systemic drugs.
The anticipated outcome of this program is to establish a retrievable, refillable implant that allows safe and durable islet transplantation for people with Type 1 Diabetes. The data obtained will guide future clinical trials, refine device design, and advance our goal of restoring natural insulin production while reducing the risks of systemic immunosuppression.
Relevance to T1D
For individuals living with Type 1 diabetes (T1D), pancreatic islet transplantation offers the possibility of restoring the body’s own ability to produce insulin and maintain normal blood glucose levels. However, this approach has been limited by the need for lifelong systemic immunosuppression, which exposes the entire body to immune-suppressing drugs that can cause infections, kidney damage, and other serious side effects. As a result, only a very small number of people with the most severe forms of T1D currently qualify for transplantation.
The NICHE technology aims to overcome this long-standing barrier by introducing a completely new way to protect transplanted insulin-producing cells. Instead of relying on systemic drug exposure, NICHE delivers immunosuppressive medications locally within a small, vascularized space under the skin where the islet cells are placed. This localized immunomodulation is designed to protect the transplanted cells from immune attack while minimizing the risk of harmful side effects elsewhere in the body. The NICHE device can also be refilled in a simple, minimally invasive procedure, supporting long-term treatment without repeated surgeries or systemic drug administration. As part of this project, sentinel NICHE devices, smaller versions of the full therapeutic implant, will be tested to study how the body responds to the implant, how blood vessels form around it, and how effectively local drug delivery protects the transplanted tissue. These studies will generate critical data to finalize the design of the full-scale therapeutic NICHE for future clinical use.
If successful, NICHE could represent a transformative step forward for people with Type 1 diabetes. It would make islet transplantation safer, less invasive, and accessible to a much broader group of patients—not just those eligible for intensive systemic therapy. By removing the burden of lifelong immunosuppressive drugs, NICHE has the potential to restore natural insulin production, free patients from daily injections or pumps, and dramatically improve quality of life.