Liberate the world from type 1 diabetes
The ultimate goal of Cure One is to realize a life without type 1 diabetes. Our aim is to enable individuals living with the condition to achieve lasting insulin independence, so they can live a life free from the daily burdens of disease management. Our mission is to develop, accelerate and implement regenerative solutions towards curing and preventing type 1 diabetes.
Structure of Cure One
Cure One is an international pre-clinical discovery and clinical research center focused on type 1 diabetes (T1D). It is an interdisciplinary center with researchers from different backgrounds that bring together various expertise that is needed to achieve our aim. Pre-clinical research is performed to answer fundamental questions about the biology of T1D. This will aid the development of solutions that are relevant for translation to the clinic. Clinical studies are aimed at protecting islets, monitoring islet function, and functionally curing T1D by performing investigator-initiated clinical and sponsor (companies)-initiated studies within an (inter)national network of healthcare providers.
The center is supported by an operational team, which includes support for valorisation of the research findings. Discoveries made by the center’s researchers will be filed for patents, and options for licensing and commercialization of the patents will be explored.
"We combine fundamental and translational research in a clear and ambitious roadmap."
Here, we work with a team that understands the importance of speed.
We spare no effort to accelerate the journey toward a treatment.
Ambitious roadmap
Our goal is to develop solutions for type 1 diabetes that are ready for the first clinical application in humans within five years. To achieve this, we have created a detailed roadmap outlining our research plan. This roadmap serves as a strategic guide, describing the key objectives, priorities, milestones, and timelines of our research initiatives. It helps coordinate our efforts, align stakeholders, and focus resources on critical questions and challenges. By providing a clear path, the roadmap promotes efficient progress, fosters collaboration, and ensures that research activities stay on track to achieve our goals.
Cure One focuses on four main research lines:
Research aim 1: Autologous stem cell islet transplantation
Problem
Transplantations with islets derived from a foreign source (non-self, allogeneic), such as donor islets, require long-term treatment with immunosuppressive drugs. These drugs prevent the immune system from attacking the transplanted islets, which could lead to rejection and failure of the transplant. However, immunosuppressive drugs are associated with several problems in islet transplantation. They are toxic to the islets and increase the risk of systemic side effects, such infections and malignancies.
Strategy
Our ambition is to develop a platform for generating stem cell-derived islets from patient’s own cells . Transplantations with such patient-specific islets (autologous) are anticipated to not require immunosuppression. This will eliminate the risks associated with immunosuppressive drugs.
Research aim 2: Next-generation hypo-immunogenic iPSC line
Problem
The cell surface is lined with proteins that create a unique identification pattern, the HLA haplotype. This pattern helps the immune system distinguish between your own cells and foreign cells. For transplantations with islets from a foreign source, the HLA haplotype from the transplant needs to match as closely as possible with that of the patient. Mismatches in HLA haplotype between donor and recipient significantly increase the risk of rejection.
Strategy
We want to generate SC-islets with reprogrammed HLA haplotypes, so they are not recognized by the immune system as being foreign (hypoimmunogenic). This will lessen the activation of the immune system and reduce the risk of rejection of transplanted islets. Such a “stealth” cellular product would be suitable for transplantation in all patients.
Research aim 3: Autologous CAR-Treg cell product
Problem
Type 1 diabetes is an autoimmune disease. Insulin-producing beta cells in the Islets of Langerhans are destroyed by thepatients’ own immune system. An error in the immune system causes it to attack the body’s own healthy beta cells.
Strategy
To avoid the destruction of beta cells, this specific autoimmune reaction needs to be suppressed, without lowering the function of the entire immune system. We will develop targeted and personalized immuno-therapeutics that intervene in islet autoimmunity and destruction. This includes the development of autologous CAR-Tregs; patient-specific T regulatory cells that are engineered to suppress specific immune reactions.
Research aim 4: In-vitro platform for Beta cell – Immune cell interaction
Problem
The local environment (microenvironment) surrounding islets in the pancreas is very important for beta cell function. This includes blood flow that facilitates a proper balance of blood glucose/insulin levels and the immune system. In type 1 diabetes, immune cells are responsible for the inflammatory conditions that lead to beta cell destruction. Currently, there is no model to study the complex interplay between the pancreatic cell types with the presence of cells lining the blood vessel walls (endothelial cells ) and immune cells.
Strategy
We will set up a dynamic 3D cell culture system containing pancreatic tissue with endothelial and immune cells to allow evaluation of beta cell function and communication with other tissues. This platform will also serve as a screening platform for islet-immune cell interaction of genetically modified (e.g. hypoimmunogenic) stem cell islets.
