Salk Institute scientists have scored a significant gain in their quest to find a functional treatment for type 1 diabetes.
According to an article in Salk News, scientists at the Salk Institute in La Jolla, California have recently announced a significant breakthrough in type 1 diabetes treatment. The disorder affects between two to three million people in California. The majority of type 1 patients are children and teens who have difficulty managing the disease. (see Nature August 19, 2020).
Using stem cell technology and mouse models, Salk researchers developed the first insulin-producing pancreatic beta islets (cell clusters) that produce insulin and other hormones.
The Salk Institute announced that when the cells were transplanted into diabetic mouse models that had healthy immune systems, the blood glucose was kept under control.
There was no need for a follow-up with immunosuppressive drugs that carry the risk of infection. To date, lifelong immune-suppressive treatment has been required in cases of normal transplants in order to keep the immune system from attacking the transplanted tissue.
In the Salk study, the transplanted cells survived because they were undetected by the immune system.
Discovery of a Genetic Switch
The scientists were searching for a way to grow cells in an environment identical to the human pancreas. They generated cell clusters that are similar to human islet-like organoids (HILOs) that are tissue and mimic organs in the body.
In a previous study, the Salk researchers were working with beta-like cells that lacked the power to produce insulin when there is a rise in glucose levels.
A Salk Institute spokesman said that a breakthrough occurred when a team in the Ronald Evans lab discovered a genetic switch called ERR-gamma. When it is switched on the cells are “turbo-charged”.
Avoiding immune rejection presented a major challenge to the team. The scientists focused on checkpoint inhibitors that, at times, prevent T cells from destroying cancer cells.
By blocking the checkpoint inhibitors, the transplanted cells are shielded from the immune system, and the T cells are free to move forward and kill the cancer.
One Step Closer
The Salk lab stated that with their new method of transplantation that produces insulin, they are one step closer to a cure.
Scientists have been searching for alternative therapies through regenerative medicine and immune shielding. They are hoping to replace damaged cells with human islet-like cell clusters generated in the lab. The endgame is to produce cell clusters that can generate insulin-on-demand in humans.
Admittedly, more work must be undertaken before the process is safe and ready for human clinical trials.
