Objective
To develop a manufacturing process that allows the large-scale generation of high-quality islet-like cells from a clinically relevant source of stem cells.
Background Rationale
We have developed a method to efficiently produce high numbers of islet-like cells from stem cells (SC-islets), which contain the insulin-producing β-cells found in the pancreas. To expand the use of these cells in both research and clinical settings, we plan to develop manufacturing process to scale up the production of these cells. The project aims to identify clinically relevant stem cell lines and establish a large-scale production process for SC-islets compatible with current Good Manufacturing Practice (GMP) standards, ensuring that the cells meet high quality and safety criteria and are ready for clinical translation in the near future.
Description of Project
Early clinical trials using islet-like cells derived from stem cells have shown encouraging results, with some patients achieving insulin independence. However, generating stem cell-derived islets (SC-islets) is complex, costly and inefficient. Access to a consistent product, could enable more research using SC-islets to develop better solutions for type 1 diabetes treatment. We have recently optimized a protocol to produce high-frequency, high-yield SC-islets. To facilitate the broader deployment of these cells in research and clinical studies, we plan to enhance the manufacturing process for SC-islets from unique cell sources, ensuring scalability, reproducibility, and accessibility.
The goal of our project is to scale up this process so we can produce large quantities of these vital cells safely and efficiently. We plan to adapt the existing method to work with advanced equipment, which allow cells to grow in large volumes under carefully controlled conditions. This will involve developing new techniques to expand the cells, monitor their quality without interfering with their growth, and harvest them in a way that meets all regulatory standards for clinical treatments.
By the end of our project, we aim to have a manufacturing process that can reliably produce high-quality, insulin-producing cells at a scale suitable for clinical applications. This means we could provide a consistent and safe supply of these cells for potential therapies, bringing us one step closer to offering a new and effective treatment option for people living with diabetes. By establishing a reliable supply of SC-islets for the research community, this project will support advancements in type 1 diabetes research and potential therapies. Our work could significantly improve the quality of life for millions of patients and represents a major advancement in the field of regenerative medicine.
Anticipated Outcome
The collaborative activities outlined in this project will create a platform to manufacture SC-islets for both research and commercial use at scale, helping to speed up the translation of discoveries into clinical applications and establish a Beta Cell Manufacturing Facility.
Relevance to T1D
Type 1 diabetes (T1D) is a chronic autoimmune condition that irreversibly damages the insulin-producing beta cells in the pancreas. Without treatment, or when individuals with T1D struggle to keep their blood sugar levels within a normal range, they face elevated risks of developing serious complications, such as heart disease, nerve damage, eye problems, and kidney disease. The primary approach for managing T1D involves regular insulin injections to help keep blood glucose levels as close to normal as possible. While this insulin therapy is the standard treatment, it only addresses the symptoms—specifically, high blood sugar levels—without offering any chance for a cure. Additionally, those relying on insulin replacement therapy can sometimes miscalculate their dosage, causing their blood sugar to drop to dangerously low levels. These challenges underscore the critical need for treatments that go beyond symptom management.
Recent advancements in generating insulin-producing cells from human pluripotent stem cells, combined with innovations in genetic and bioengineering technologies, hold promising potential to address the current treatment gap for T1D. These methods could provide an unlimited supply of cells for transplantation, potentially eliminating the need for immunosuppressive drugs. However, the high production costs for these cells pose a significant barrier to their widespread use of these cells for research and pre-clinical studies. To accelerate progress in T1D research, it is crucial to refine the manufacturing process, making these cells more affordable and accessible for research and future therapeutic use.