Objective
Our goal is to engineer transplantable insulin-producing cell clusters, called islet organoids, that function like natural islets but are made entirely from stem cells. These organoids will include cells that help form blood vessels and support immune protection in the body. We also add a built-in safety switch to remove hazardous dividing cells. This project will test whether these engineered islet organoids can survive, release insulin, and avoid immune rejection when implanted in animal models. If successful, this work could lead to a better treatment for people with type 1 diabetes.
Background Rationale
Type 1 diabetes is caused by the immune system destroying the body’s insulin-producing cells. People with this disease must take insulin for life, but such treatments cannot perfectly control blood sugar. This leads to serious health problems and daily challenges. Transplanting healthy insulin-producing cells can help, but this approach faces major hurdles, including a lack of available donors and the lifelong need for drugs that suppress the immune system. Although recent advances in stem cell technologies allow us to create insulin-producing cells in the lab, making them survive and work in the body remains a challenge.
Description of Project
We are developing a new form of cell therapy that could help people with type 1 diabetes live without needing insulin injections or chronic immunosuppression. Our strategy uses stem cells to create mini-organs, called islet organoids, that can sense sugar and release insulin. These organoids also include supporting cells to help them survive and connect with the body’s blood supply. To avoid rejection by the immune system, we use advanced gene-editing techniques to make the cells invisible to immune attack. This project brings together science and technology to create a safer, longer-lasting treatment option for people with diabetes.
Anticipated Outcome
We expect to develop a reliable and safe way to produce functional islet organoids from stem cells. These organoids should be able to rapidly connect to the body’s blood supply, release insulin in response to elevated sugar levels, and avoid being attacked by the immune system. Our lab tests and animal studies will show whether this approach can control blood sugar without the need for daily insulin or immune-suppressing drugs. The knowledge gained from this work could not only inform new treatments for type 1 diabetes but also have the potential to expand to other metabolic disorders like type 2 diabetes or obesity.
Relevance to T1D
This project is focused on finding a better solution for people with type 1 diabetes. Instead of relying on insulin injections, which are difficult to manage and not always effective, we aim to provide a functional cure by replacing the lost insulin-producing cells using stem cells. Our approach addresses the key challenges of cell survival, immune rejection, and safety. By developing a stem cell-based therapy that can be used broadly and safely, this work has the potential to change the lives of millions of people living with type 1 diabetes.