Experimental drug delays type 1 diabetes onset in mid-stage trial

Breakthrough for diabetes treatment as it emerges immune system drug can delay onset of the illness

Breakthrough for diabetes treatment as it emerges immune system drug can delay onset of the illness

An experimental drug was able to slow down the progression of type 1 diabetes, an inherited disease, among high-risk patients in a small clinical trial.

Researchers gave the drug teplizumab or a placebo to a small group of people who were almost certain to develop Type 1 diabetes, based on genetics and certain symptoms. Study participants were followed until they developed Type 1 diabetes.

A drug that targets the immune system can delay the onset of Type 1 diabetes an average of two years in children and adults at high risk, according to findings presented at the American Diabetes Association meeting in San Francisco this past weekend.

During the trial, 72 per cent of the people in the control group developed clinical diabetes, compared to only 43 per cent of the teplizumab group.

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In those who did progress to a diagnosis of type 1 diabetes, development was significantly delayed in those who received teplizumab compared with placebo, with a median time to diagnosis of 48.4 months versus 24.4 months, respectively. But the findings do provide clues to potential causes of Type 1 diabetes, and suggest ways to possibly try to modify the course of the disease.

"This is the first demonstration that by doing an immunologic therapy, you can delay the clinical onset of disease", said Herold, professor of immunobiology and of medicine at Yale School of Medicine.

"The results of this trial are striking, with several caveats", they wrote. "In addition, we look forward to learning more as we observe patients during the study's follow-up period, which will also evaluate the long-term outcomes for those in whom the diagnosis of disease has been delayed to see if they will be diagnosed with T1D or are protected". These people are related to 2 types of diabetes, which related to abnormal glucose (sugar) tolerance and autoantibodies (a protein made by an immune system).

JDRF offers more facts and details about Type 1 diabetes. "The results have important implications for people, particularly youth, who have relatives with the disease, as these individuals may be at high risk and benefit from early screening and treatment".

All included individuals had dysglycemia prior to the study, marked by a fasting glucose between 110-125 mg/dL, a 2-hour post-prandial plasma glucose ≥140-200 mg/dL, or an intervening postprandial glucose levels 200 mg/dL at two occasions. It is the immune system's attack on the beta cells that characterises type 1 diabetes. This intravenous infusion was given as a 51 µgram/m of body-surface area on at day 0. This was then increased to 207 µg/m on day 2, 413 µg/m on day 3, and 826 µg/m each day from days 4-13. A total of 75% of participants who received teplizumab experienced a form of AE relating to blood or bone marrow, such as transient lymphopenia.

Dunne also noted that a delay means a child might be more mature and able to help manage the condition.

Dr. Clifford Rosen, from the Maine Medical Research Institute, and Dr. Julie Ingelfinger, from Massachusetts General Hospital, wrote an editorial in the same issue of the journal.

Dr Aaron J. Kowalski, JDRF President and global CEO, added: "These results and the potential impact to people living with type 1 diabetes and their families is exactly why JDRF funds prevention research".

The trial was supported by the NIH, the Juvenile Diabetes Research Foundation, and the American Diabetes Association.