Teplizumab: Medication for Type 1 Diabetes

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First in Kentucky, Indiana or Ohio to Offer, Administer Teplizumab

Wendy Novak Diabetes Institute became the first program in Kentucky to offer and administer teplizumab.

In April 2023, 15-year-old Kylie Smith received the diabetes medicine and became the first patient in Kentucky, Indiana or Ohio to receive the treatment. Six months after receiving the treatment, she showed positive signs the drug was working.

If you have a close family member with Type 1 diabetes, you are at risk for the disease and may benefit from teplizumab, a recently approved Type 1 diabetes medication that can delay the disease’s onset and slow its progress.

Teplizumab (pronounced tep-LIZ-oo-mab) is available for children ages 8 and older and adults who are developing the condition, but don’t yet have Type 1 diabetes and have no symptoms. A screening is available at no cost if you meet eligibility requirements, which include being between ages 2 and 45 and having a relative with Type 1 diabetes.

The screening, based on a simple blood test, checks for the presence of diabetes autoantibodies that are at the root of developing Type 1 diabetes. The autoantibodies trigger the immune system’s T-cells to attack insulin-producing beta cells in the pancreas. With beta cells under attack, the pancreas doesn’t produce enough insulin. Without sufficient insulin, blood sugar, also called blood glucose, can’t pass through cell membranes, where the sugar fuels energy.

If You Think You or Your Child Is Eligible For a Screening

Talk with your child’s pediatrician or call Shannon Hall, clinical research nurse, Norton Children’s Research Institute, affiliated with the UofL School of Medicine, at (502) 588-8910 for more information on a screening for Type 1 diabetes.

Reduced beta cell function and the accompanying reduced levels of insulin leave excess blood sugar flowing through the blood stream. As the sugar builds up, it begins to cause symptoms of Type 1 diabetes, which can come on quickly and become severe without treatment.

Teplizumab has been shown to delay development of Type 1 diabetes by about two years. About half the patients who take the medication get a longer delay and half get a shorter delay. Wendy Novak Diabetes Institute, a part of Norton Healthcare and Norton Children’s, offers teplizumab screening through the TrialNet Pathway to Prevention Study.

Wendy Novak Diabetes Institute is focused on caring for all individuals with diabetes to help them live healthy and active lives. We are continuously ranked as a top pediatric endocrinology and diabetes program in the country. Our team is passionate about research for Type 1 and Type 2 diabetes, and giving our families access to cutting-edge technology, therapies and treatments.

Our specialists are at the forefront of new diabetes treatments and conduct clinical trials to investigate innovative therapies. This means our patients have access to potential breakthrough treatments, and our providers have extensive experience with treatments once they are approved for wide use.

Why Delaying Type 1 Diabetes Is Important

Type 1 diabetes is different from Type 2 diabetes. Both result in a high blood sugar level in the blood stream as the sugar can’t make its way into cells to provide fuel. Type 1 diabetes, a lifelong autoimmune condition, is related to reduced beta cell function and low insulin production. Type 2 diabetes, on the other hand, happens when the body builds up insulin resistance from continued high levels of blood sugar. Gestational diabetes happens when insulin produced by the body during pregnancy doesn’t effectively lower blood sugar levels.

Most people with diabetes have Type 2 diabetes mellitus. About 5% of people have Type 1 diabetes, according to the American Diabetes Association.

Managing Type 1 diabetes with blood glucose level monitoring and insulin therapy can prevent complications and improve day-to-day life. While some patients rely on an insulin injection, many can use an insulin pump or inhaled insulin. Inhaled rapid-acting insulin is approved for adults, and a clinical trial at Wendy Novak Diabetes Institute is evaluating its use in children.

As the disease progresses and blood sugar takes a toll on the body, risks increase for cardiovascular disease, blood vessel damage, neurological complications, vision loss and kidney disease.

Managing the condition to keep blood sugar levels down reduces the risk for complications. Delaying the disease’s onset also can delay the development of any complications.

How Teplizumab Works

Teplizumab, sold under the brand name Tzield, is given to eligible patients with Stage 2 Type 1 diabetes. Stage 2 describes the point at which patients have lost enough insulin-producing beta cells to result in high blood sugar levels (dysglycemia), but haven’t developed diabetes. Patients in Stage 2 do not have any symptoms of diabetes.

  • Stage 1 Type 1 diabetes: At this point, the immune system’s T-cells begin to attack beta cells in the pancreas that produce insulin. There are no symptoms at this stage, and blood sugar levels aren’t affected.
  • Stage 2: The disease has progressed and some beta cells have been destroyed. The result is high blood sugar levels, but there are still no symptoms. As T-cells continue attacking beta cells, the disease progresses.
  • Stage 3: Because nearly all beta cells have been destroyed, insulin production effectively shuts down, and Type 1 diabetes symptoms begin.

Teplizumab is effective at Stage 2. The drug binds to a specific protein on the T-cells, disrupting their ability to attack beta cells. This helps the cells survive and continue making insulin to control blood sugar. While it slows the progression of Type 1 diabetes, it does not cure or prevent it.

Pediatric patients who qualify can receive this therapy in the Norton Children’s Infusion Center. Each infusion takes approximately 30 minutes once daily for 14 straight days. Doctors will order labs before to ensure the medication is safe for the patient and will prescribe medicines before the course of treatment to reduce the chance of adverse effects.

Current Clinical Trials at Wendy Novak Diabetes Institute and Norton Children’s Endocrinology

Norton Children’s and Wendy Novak Diabetes Institute Help You and Your Child Manage Diabetes

USNWR best hospitals for diabetes & endocrinology and DNV certification for glycemic management are shown
  • Norton Children’s is recognized as a top pediatric diabetes and endocrinology program in the U.S. News & World Report Best Children’s Hospitals rankings.
  • Once your child’s pediatrician makes a referral, you’ll have quick access to a team of board-certified endocrinologists and advanced practice providers offering comprehensive diabetes care.
  • Next-day, urgent appointments are available.
  • Diabetes educators, nurses, dietitians, psychologists, social workers, child life specialists, exercise physiologists and nurse practitioners help children and families manage diabetes.
  • Our nutrition program gives families hands-on experience planning and cooking diabetes-friendly meals and snacks.
  • Christensen Family Sports & Activity Program focuses on improving the health, safety and athletic performance of children and young adults with diabetes.
  • As your child grows and gains more independence, our providers help prepare them for managing diabetes while at college or living on their own. This program is available to teenagers and young adults from ages 16 to 26.
  • The Novak Center for Children’s Health is equipped to provide imaging, labs and appointments all in the same location.
  • Medicaid and most major commercial insurance plans are accepted.
  • Keep up with your child’s lab results, communicate with your provider, schedule appointments and get alerted if an earlier appointment becomes available with Norton MyChart.

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