Cure for type-1 diabetes closer to reality through advances in genetic engineering

About 95% percent of all diabetes in the country is type-2 marked by insulin resistance. It is very treatable with a variety of oral and injectable medications and is mostly preventable with diet and exercise even in high-risk people.

But type-1 diabetes is a very different condition, where insulin-producing “beta” cells in the pancreas are destroyed by the body’s own immune system.

Aaron Fine gives himself insulin Feb. 20 ahead of the game against Mississinewa at Noblesville High School in Noblesville, Indiana.
Aaron Fine gives himself insulin Feb. 20 ahead of the game against Mississinewa at Noblesville High School in Noblesville, Indiana.

It affects one in 500 people worldwide, and before 1922 with the first bovine insulin procured for injection into humans, the condition was universally fatal. Now, advances in genetic engineering are staring at the cure.

For 15 years genetically modified stem cells have been converted into beta cells. This was the easy part. Turning off the body’s immune system toward these cells has been the major challenge.

Two arms of the immune system have now been addressed. One is the “adaptive” arm that is involved with tissue rejection. Some proteins on the cell surface have areas that are specific to an individual. When they are not identified by the person’s immune system, killer T-cells and antibodies are called to destroy the cells as “foreign” agents.

Genetic modification has produced beta cells without these proteins. This, however, creates its own problem. In the absence of the proteins, the second arm of the immune system called the “innate” system is also activated to destroy the cells.

The innate arm can be suppressed to some degree by a protein that has also been engineered into the beta cell. Studies with monkeys show that injecting high-insulin-producing forms of these cells into the animals can “cure” type-1 diabetes for about six months. Human trials are underway.

But a better solution appears to be turning off adaptive immunity with a second cell type. These “suppressor” cells talk to killer T-cells, turning them off from reacting to the beta cells. The issue of innate immunity would become inconsequential.

Aided by advances in artificial intelligence, the field of synthetic biology is also racing forward. The potential for creating new types of cells and new forms of life carries very real risks and requires aggressive international regulation, but for someone whose entire life rotates around their chronic disease, the potential for a cure is a miracle.

William Culbert is a retired physician. He lives in Oak Ridge.

William Culbert
William Culbert

This article originally appeared on Knoxville News Sentinel: Cure for type-1 diabetes closer through genetic advances | Culbert