North Carolina teen among first in U.S. to receive new diabetes drug at Children’s Hospital of Richmond at VCU
February 13, 2023
High school sophomore, William Troutman, is enjoying a run outside. (Children's Hospital of Richmond at VCU)
RICHMOND, Va., (February 13, 2023) — High school sophomore and avid runner William Troutman has never had any symptoms of diabetes, yet he’s spent the last two weeks receiving a newly FDA-approved drug, Tzield™ (tepluzimab), to delay its onset. William and his mother, Amanda, traveled nearly 300 miles from their home in North Carolina to Children’s Hospital of Richmond at VCU – the closest TrialNet affiliate – so he could be among the first in the county to receive it.
In November, tepluzimab gained FDA approval for use in people ages 8 and older with stage 2 type 1 diabetes, which means they have two positive diabetes related autoantibodies and elevated, pre-diabetes range blood sugars. It has proved effective at delaying onset of stage 3 diabetes by an average of two years and up by helping to prevent the auto-immune attack of the beta cells in the pancreas responsible for making the hormone insulin. The challenge, however, is that diabetes symptoms do not typically appear, and the disease is not diagnosed until it reaches stage 3, which most people think of as classic type 1 diabetes and requires intensive insulin and blood glucose management.
“This is the first preventative treatment for type 1 diabetes and first novel advancement in diabetes care outside of insulin,” said Dr. Bryce Nelson, chief of endocrinology at CHoR. “This is a really exciting opportunity, but it’s going to require a completely new way of monitoring for and diagnosing type 1 diabetes. Collaboration with pediatricians and general practice providers will be essential in getting kids in for treatment right away.”
William’s risk was identified after his younger sister was diagnosed with type 1 diabetes in September 2020. Eager to know everything she could about her daughter’s newly diagnosed condition, Amanda Troutman began listening to a podcast by another parent of a child with type 1 diabetes. That’s where she first heard about tepluzimab, which had been in clinical study for nearly 10 years, and the importance of identifying disease risk early. She immediately ordered at-home test kits from TrialNet for herself, her husband and William. William tested positive for two of the hormones associated with diabetes. His mom and dad had none.
“Siblings carry a 5 to 7 percent lifetime risk of getting type 1 diabetes, which is 15-fold higher than the general population, so it’s important for families to test when a member has been diagnosed,” said Dr. Nelson. “Research tells us that the disease starts many years before blood sugars become abnormal. When people develop two autoantibodies, like William, it’s not a question of if but when they will develop type 1 diabetes. Younger kids tend to develop it faster, older people slower.”
Amanda found CHoR and Dr. Nelson through TrialNet. The two remained in contact throughout the remainder of the drug’s study.
“We did the tests in November 2020. At that point tepluzimab was still under investigation, and I worried it might be too late to get it for William by the time it got approved,” said Amanda.
Right before Thanksgiving 2022, the great news came that it had been approved – and William was still in stage 2 type 1 diabetes, which meant he was a candidate.
“We already had the specialty medical team, trained nurses and infusion capabilities in our Children’s Pavilion to begin offering the tepluzimab infusions. At that point we simply needed to order the medication, get William on the schedule and allow time for his family to travel from out of state for a couple weeks,” said Dr. Nelson.
Tepluzimab is given through a once daily infusion for 14 consecutive days, with escalating doses for the first five days.
Postponement of symptomatic diabetes has many benefits including:
- Minimized short term risks, such as hypoglycemia
- Decreased long-term complications and health care costs related to diabetes
- Onset of symptoms occurring at older ages when they’re more easily managed
- Improvements in school attendance and performance
- Fewer concentration issues related to high or low blood sugar
This is great news for the approximately 100 kids in Virginia – and thousands across the country – who would qualify for the medication.
“Dr. Nelson is a leader in the field of pediatric endocrinology, and we brought him to CHoR to help ensure that kids in Richmond and throughout our region have access to clinical advancements that will improve the course of their lives,” said Elias Neujahr, CHoR president. “This is an exciting milestone as our teams continue to help kids stay has healthy as possible through promising new treatments.”
Diagnoses of type 1 diabetes have increased by 3-5 percent annually in recent years, indicating that more and more people may be eligible for tepluzimab. If there is a family history of type 1 diabetes, a test kit can be ordered from TrialNet. Family history of other autoimmune diseases, such as multiple sclerosis, Graves disease, lupus, rheumatoid arthritis and others, can also signal increased risk for diabetes. Families are encouraged to talk with their primary care providers about risk assessment.
