High school sophomore and avid runner William Troutman has never had any symptoms of diabetes, yet he’s spent the last two weeks in our infusion clinic receiving a medication to delay its onset. You could say he’s one of the lucky ones to be identified as a candidate for a new FDA-approved drug, TzieldTM (teplizumab), and now he’s among the first in the country to receive it after traveling from his home in North Carolina to Virginia for this life-changing infusion at CHoR.
“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.
In November, teplizumab 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. The challenge, however, is that typically symptoms don’t appear, and the disease isn’t diagnosed until it reaches stage 3, which is what we know as classic type 1 diabetes and requires intensive insulin and blood glucose management.
“This is a really exciting opportunity, but it’s going to require a completely new way of monitoring for and diagnosing type 1 diabetes,” added Dr. Nelson. “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. She had been excessively thirsty, using the restroom more than normal, napping all the time and losing weight, but things became especially scary for the family when she began experiencing labored breathing. They took her to the emergency room where they found out she was in diabetic ketoacidosis – a life-threatening complication of diabetes where the body doesn’t have enough insulin and produces acids called ketones that build up to dangerous levels. About a third of people who get to stage 3 diabetes without treatment will experience diabetic ketoacidosis.
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 teplizumab, 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. Sure enough, William tested positive for two of the hormones associated with diabetes. His mom and dad had none.
“Siblings of people with type 1 diabetes 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.”
“We did the tests in November 2020. At that point teplizumab 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.
Amanda found CHoR and Dr. Nelson through TrialNet. We’re the only TrialNet affiliate in the region and Amanda was ready and willing to travel the nearly 300 miles to Richmond from her family’s home in North Carolina if it meant getting this life-changing medication for her son. She and Dr. Nelson remained in contact throughout the remainder of the drug’s study. Right before Thanksgiving 2022, they got the great news 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, infusion capabilities in our Children’s Pavilion and supportive leadership in place to begin offering the teplizumab infusions. At that point we simply needed to order the medication, get William on the schedule and allow his family time to travel from out of state for a couple weeks,” said Dr. Nelson.
Teplizumab is given through a once daily infusion for 14 consecutive days, with escalating doses for the first five days. William started his infusions on January 30.
While teplizumab hasn’t shown to prevent type 1 diabetes altogether and is not for use in patients with type 2 diabetes, it has proved effective at delaying onset of stage 3 by an average of two years and up. It does this by helping to prevent the auto-immune attack of the beta cells in the pancreas responsible for making the hormone insulin. This postponement has many benefits including:
This is great news for hundreds of kids in Virginia – and thousands across the country – who would qualify for the medication. While William’s sister is doing quite well these days and her diabetes is under control, the entire Troutman family is thrilled that her big brother has this opportunity to delay onset and symptoms – and tremendously grateful for Dr. Nelson’s guidance and care.
Dr. Nelson and his team provide ongoing care for 850 children with type 1 diabetes and approximately 1,100 with types 1 and 2 diabetes combined.
“We’re your patients for good now,” Amanda said to Dr. Nelson with a laugh from the CHoR infusion clinic overlooking downtown Richmond.
Once William’s infusions are complete, he’ll only need to check in with Dr. Nelson for follow-ups every six months, or earlier if symptoms develop. In the meantime, he’ll be running 20 miles a week, mountain biking, snowboarding or out driving, basking in his new 16-year-old independence.
“I’ve been on this journey of diabetes care since 1995. I’m overjoyed and excited that we’ve gotten to this exciting milestone of a new and promising treatment,” said Dr. Nelson. “This is one of the reasons I came to CHoR – the opportunity to be part of clinical advancements that will change the lives of kids like William.”
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 teplizumab.
If there’s a family history of type 1 diabetes, you can request a test kit from TrialNet to determine your risk. Family history of other autoimmune diseases, such as multiple sclerosis, Graves disease, lupus, rheumatoid arthritis and others, can also signal increased risk for type 1 diabetes. Talk with your family’s pediatrician or primary care provider about screening and a referral to our pediatric endocrinology program.
){kind=link}
){kind=link}
){kind=link}