A new therapy to prevent or delay Type 1 diabetes now available at UNC Children's Hospital

Nina Jain, MD, MBA, professor of pediatric endocrinology, and Ali Calikoglu, MD, retired professor of pediatrics, both at the UNC School of Medicine, played a pivotal role in getting this therapy to the bedside, from assisting with clinical trials to the infusion of the first pediatric patient in North Carolina at UNC Children's.

A diagnosis of Type 1 diabetes can have a profound impact on a child’s life.

This autoimmune condition occurs when the body's own immune cells attack the cells that produce insulin, a crucial hormone that regulates blood sugar in the body. To prevent life-threatening complications, the child needs multiple daily insulin injections and have a closely monitored diet and activity, which has the potential to cause significant physical, social, and financial stress for the child and their family.

To help alleviate this strain and improve the lives of their patients, pediatricians at UNC Children's are now offering infusions of Tzield, a therapy that can prevent or delay the onset of Type 1 diabetes. After 14 days of infusions, patients may be able to continue producing their own insulin, have better control over their blood glucose levels, and experience fewer medical complications for several years.

At this time, UNC Children's is the only site in the state of North Carolina that is offering this innovative care to pediatric patients.

"The cutting-edge scientists and clinicians at UNC Children's began participating in clinical trials testing of teplizumab in 2019," said Stephanie Davis, MD, the Edward C. Curnen, Jr. Distinguished Professor and Chair at the UNC Department of Pediatrics. "This new approach to prevent or delay the onset of Type 1 diabetes has been a great example of the importance of scientific research in creating new treatments to improve health. As the first treatment center in the state, UNC Children's is leading the way to provide this for the children of North Carolina."

How Does Teplizumab Work?

Teplizumab, sold under the brand name Tzield, is given through an infusion for 14 consecutive days. The drug is a targeted immunotherapy that works by "turning off" the immune cells that are attacking insulin-producing beta cells in the pancreas.

Patients may be able to continue making their own insulin for several years if the treatment effectively interferes with these autoimmune cells, ultimately reducing the need for insulin injections. Better regulation of daily blood glucose levels also means fewer long-term complications.

"Therapies that can delay the onset of diabetes will help prevent the need for frequent blood sugars checks and multiple daily injections and reduce the risk of severely low or high blood sugars,” said Jain. “Our goal is to allow these children and families more time to enjoy childhood and adjust their daily lifestyle to prevent long-term health concerns," said Jain.

The effort was a long time in the making. In 2019, Nina Jain, MD, MBA, professor of pediatric endocrinology and medical director of the Pediatric Diabetes Program at UNC Children's, and Ali Calikoglu, MD, professor of pediatrics, and the Children's Research Institute joined the PROTECT study, then led by ProventionBio, now Sanofi. The international, multi-site clinical trial centered on the safety and effectiveness of teplizumab in children and adolescents ages 8 through 17 who were recently diagnosed with type 1 diabetes.

Through the tireless work of the study coordinators and nurses at the UNC Children's Research Institute, Jain and Calikoglu successfully enrolled and completed study engagement for six pediatric patients. In November 2022, the therapy was FDA approved for clinical use.

The First Infusion in the State of North Carolina

Just two years later, in October 2024, Jain initiated the first infusion of teplizumab for a patient with Stage 2 Type 1 diabetes. During this stage, the patient already has multiple autoimmune cells which are attacking pancreatic beta cells and slightly elevated blood glucose levels but does not yet need insulin injections. Patients are expected to develop insulin dependent stage 3 Type 1 diabetes, requiring multiple doses of insulin daily, within the next 3 to 6 months.

Acknowledging that the 14-day-long infusions are intensive for both patient and parents, the clinic's infusion team, social workers, and Sanofi – the pharmaceutical company that created this treatment – jumped in to help. Despite full schedules, the infusion team and pediatric pharmacy team at UNC Children's underwent training for the new drug therapy and scheduled infusions at the UNC Children's Infusion Center as drug therapy dosing is steadily increased.

Katie Cooper, LCSW, LCAS, a social worker at the UNC Department of Pediatrics, arranged patient accommodations at the Ronald McDonald House to provide transportation and temporary housing for the family, who live 90 minutes away from Chapel Hill. After five days of infusions in clinic, the team continued with daily virtual visits with the patient and partnered with a local home health agency to continue infusions at home once dosing was stable.

"Tzield is the first therapy currently available to children 8 years and older to delay the onset of Type 1 diabetes. Research in this field is steadily growing and we at UNC Children's look forward to bringing these life-changing therapies to the children of North Carolina,” said Jain.