New oral therapy for type 1 diabetes can delay disease progression and lower insulin requirements for at least 2 years

A small clinical trial has highlighted a new possible oral therapy to delay type 1 diabetes progression, as well as a potential new biological marker to monitor disease progression. In type 1 diabetes, the immune system launches a misguided attack against the insulin-producing β (beta) cells in the pancreas. Novel therapies to protect β-cells from this attack are urgently needed. Similarly, easily monitored markers of β-cell health are needed to warn of the earliest stages of type 1 diabetes and to predict and track disease severity.

Previous NIDDK-supported research suggested that verapamil might slow diabetes progression. Verapamil is an oral medication approved by the U.S. Food and Drug Administration (FDA) over 30 years ago to treat high blood pressure. In a small clinical trial, 24 male and female volunteers recently diagnosed with type 1 diabetes were treated with insulin and either verapamil or an inactive placebo. In 2018, scientists reported that people taking verapamil for 1 year had better insulin responses, used less insulin, and had fewer incidents of low blood glucose (sugar) than did people taking a placebo, all with no adverse effects. These results suggested that verapamil could safely improve overall β-cell function and might prevent β-cell loss.

Now, new research has detailed a year-long extension of this trial. In the trial’s second year, the verapamil-treated group was split into two subgroups: one continued taking verapamil, while the other stopped. Those who used verapamil for 2 years maintained their β-cell health and continued to need reduced levels of insulin compared to the group that never took verapamil. However, those who stopped the drug after 1 year saw their β-cell health decline and their insulin needs rise, demonstrating that verapamil’s benefits required continuous use. Researchers also found that levels of a protein called chromogranin A (CHGA) in the blood may be a promising biological marker for poor β-cell health. Specifically, they found that CHGA blood levels were elevated in people with type 1 diabetes compared to people without the disease. CHGA levels dropped substantially in people who took verapamil, but remained high in those who took the placebo, suggesting that CHGA levels reflect changes in β-cell health in response to verapamil treatment. Thus, CHGA levels might be a simple way to track type 1 diabetes initiation, progression, and/or response to treatment.

These findings may lead to new options to diagnose, treat, and monitor type 1 diabetes. Future studies will be needed to better characterize both verapamil’s safety and effectiveness, as well as CHGA’s usefulness as a marker of β-cell health.