Emily Sims, MD, at a microscope in her laboratory.
Key Points:
- The FDA has now approved teplizumab, an immunotherapy drug.
- In studies, the drug was shown to delay the onset of Type 1 diabetes by almost three years, on average.
- The approval is especially critical for other with a family history of Type 1 diabetes.
The FDA has approved teplizumab, a new immunotherapy drug that delays the onset of type 1 diabetes in at-risk individuals by an average of almost three years.
Initially, 28 sites participated in the first teplizumab study conducted by TrialNet, the largest clinical trial network ever assembled to discover ways to delay and prevent type 1 diabetes. Extended follow up with participants of the trial concluded in 2019. These newest findings show high-risk individuals treated with teplizumab experienced a median delay of diagnosis by 2.7 years versus the placebo group.
“Nearly three years delay in the onset of disease is a big deal,” said Emily Sims, MD, associate professor of pediatrics and a physician scientist at IU School of Medicine. “Even if someone has the newest technology to deliver insulin, there’s still a huge financial and psychological burden that goes along with diagnosis.”
In addition to the pronounced delay of disease, those treated with teplizumab showed improved rates of insulin production, despite exhibiting insulin loss over time prior to treatment. Participants receiving a placebo continued to show a decline in insulin production consistent with disease advancement.
Study participants were relatives of people with type 1 diabetes who have two or more autoantibodies and abnormal blood sugar levels, identified by TrialNet’s Pathway to Prevention screening as having a nearly 100-percent lifetime risk of clinical diagnosis. Of the original 76 participants, 72 percent were children under age 18.
“The idea of disease prevention and disease modifying therapy is really exciting,” Sims said. “You can imagine it might really change the way we treat people with type 1 diabetes and those at high risk for developing the disease. This is just the beginning.”
In response to teplizumab’s approval, Riley Hospital for Children at Indiana University Health has opened a new clinic for individuals identified as high risk for developing type 1 diabetes.
“We’ve been able to identify people who are at risk for type 1 diabetes for decades, but until now, we haven’t had anything to mitigate that risk, which leaves families just waiting for disease to develop. To be able to discuss this as an option for delay in diagnosis is incredible,” said clinic director Jamie Felton, MD.
Information courtesy of Indiana University School of Medicine