A new study has found that a drug commonly prescribed for rheumatoid arthritis can slow down the advancement of type 1 diabetes, a chronic condition in which the pancreas produces little or no insulin.
What is the drug and how does it work?
The drug is called baricitinib and it belongs to a class of medications called Janus kinase (JAK) inhibitors. These drugs work by blocking the activity of enzymes called JAKs, which are involved in inflammation and immune responses.
Baricitinib is approved by the US Food and Drug Administration (FDA) and other regulatory agencies for the treatment of moderate to severe rheumatoid arthritis, a condition in which the immune system attacks the joints and causes pain, swelling and stiffness.
The researchers hypothesized that baricitinib could also protect the insulin-producing cells in the pancreas, called beta cells, from being destroyed by the immune system in type 1 diabetes.
What did the study find?
The study, published in the New England Journal of Medicine on December 8, 2023, was conducted by researchers at St Vincent’s Institute of Medical Research (SVI) in Melbourne, Australia.
The study was a randomized, double-blind, placebo-controlled trial, which means that the participants were randomly assigned to receive either baricitinib or a placebo (an inactive substance) and neither they nor the researchers knew who received what.
The trial involved 91 participants aged between 10 and 30 years who had been diagnosed with type 1 diabetes within 100 days of starting the trial. They received either baricitinib or placebo once daily for one year, along with their usual insulin therapy.
The main outcome of the study was to measure the C-peptide level in the blood of the participants. C-peptide is a molecule that is released along with insulin from the beta cells and reflects how much insulin is being produced by the pancreas.
The study found that:
- The participants who received baricitinib had higher C-peptide levels than those who received placebo at 12 months, indicating that they had more beta cell function and less insulin requirement.
- The difference in C-peptide levels between the two groups was statistically significant, meaning that it was unlikely to be due to chance.
- The participants who received baricitinib also had lower hemoglobin A1c levels than those who received placebo at 12 months, indicating that they had better blood sugar control.
- The difference in hemoglobin A1c levels between the two groups was also statistically significant.
- The participants who received baricitinib reported fewer episodes of hypoglycemia (low blood sugar) than those who received placebo.
- The difference in hypoglycemia episodes between the two groups was not statistically significant, but it was clinically meaningful.
- The participants who received baricitinib did not experience any serious adverse events related to the drug, such as infections or blood clots. The most common side effects were mild and included upper respiratory tract infections, headache and nausea.
What are the implications of the study?
The study is the first of its kind to show that a JAK inhibitor can suppress the progression of type 1 diabetes in newly diagnosed patients. It suggests that baricitinib could be a potential disease-modifying treatment for type 1 diabetes that can be delivered as a tablet.
The study also shows that baricitinib is safe and well-tolerated in this population. However, more studies are needed to confirm its long-term safety and efficacy, as well as to determine its optimal dose and duration of treatment.
The study’s lead author, Professor Thomas Kay from SVI, said: \”It is tremendously exciting for us to be the first group anywhere in the world to test the efficacy of baricitinib as a potential type 1 diabetes treatment. Up until now, people with type 1 diabetes have been reliant on insulin delivered via injection or infusion pump. Our trial showed that, if started early enough after diagnosis, and while the participants remained on the medication, their production of insulin was maintained.\”
The study’s co-author, Professor Mark Cooper from Monash University, said: \”This is an important breakthrough for people with type 1 diabetes and their families. Management of the lifelong autoimmune disease is incredibly burdensome on those diagnosed and their families, requiring meticulous glucose monitoring and insulin administration day and night to stay alive. This trial offers hope for a simpler and more effective way of managing type 1 diabetes in the future.\”
The study was funded by the Juvenile Diabetes Research Foundation (JDRF), the National Health and Medical Research Council (NHMRC) of Australia, and Eli Lilly and Company, the manufacturer of baricitinib.