CIRM funds many projects seeking to better understand diabetes and to translate those discoveries into new therapies.
Diabetes comes in two forms: type 1 (also known as juvenile) and type 2 (also known as adult). As many as 3 million people in the U.S. have type 1 diabetes, which is the form primarily being targeted by stem cell research.
Type 1 diabetes is an autoimmune disorder where the body’s own immune system destroys the cells in the pancreas that make insulin. Insulin normally circulates in the bloodstream after a meal and allows cells of the body to take up sugar and use it for food. Without insulin, cells starve and the sugar builds up in the bloodstream where it can damage the kidneys, blood vessels and retina.
Any potential cure for type 1 diabetes requires replacing the lost insulin-producing cells of the pancreas. Currently, the only cells that can be used for such a transplant come from donated organs, which are in short supply. Such insulin-producing cell transplants are also risky because the cells can be rejected by the recipient’s body if they don’t receive immune suppressing drugs.
To solve the first problem, groups of CIRM-funded researchers have developed methods to make replacement insulin-producing cells derived from human embryonic stem cells, which can be grown in large amounts. Implanted into mice and rats these cells are able to regulate blood sugar.
To get around the problem of rejection, CIRM-funded teams have placed cells in a device that implants under the skin and shields the cells from the immune system. Other groups are studying how to regulate the immune system to make stem cell-derived transplants safer.
Clinical Stage Programs
Caladrius is targeting the immune system as an alternative strategy for treating patients with type 1 diabetes. This disease causes the immune system to destroy the insulin-producing cells of the pancreas. The team is developing a stem cell-based therapy using the patient's own cells. They will take cells, called regulatory T cells (Tregs), from the patient’s own immune system, expand the number of those cells in the lab and enhance them to make them more effective at preventing the autoimmune attack on the insulin-producing cells.
The team has developed a way of maturing embryonic stem cells into an early form of the pancreatic cells that produce insulin. They then insert those cells into a permeable device that can be implanted under the skin. Inside the device, the cells mature into insulin-producing cells and the hope is that they will help maintain blood sugar levels at a healthy level. The team is currently testing the safety and efficacy of this device in a Phase 1 clinical trial.