Mikayla: Contributing to Research Leading to the First Preventive Therapy for Type 1 Diabetes
In 2016, Mikayla was told that she was about 6 months away from having clinical type 1 diabetes and being dependent on insulin. Seven years later, at age 21, she is still free of clinical disease and is insulin independent, a happy result she attributes to her participation in a clinical trial through Type 1 Diabetes TrialNet.
A Sibling’s Harrowing Type 1 Diabetes Diagnosis
Mikayla’s experiences with type 1 diabetes began in 2016, when her younger sister, Mia, was diagnosed with the disease at age 9 while the family was living in Colorado. Mikayla was 14 at the time and recalls the harrowing experience. “We went to the doctor for me because I sprained my knee,” Mikayla remembers. At the same time, her sister was not feeling well. When the family got home, Mikayla’s mother quickly realized that Mia needed emergency care and rushed her to the hospital. Mikayla’s father was in Florida for work, so Mikayla and her younger brother went to a neighbor’s house. When Mikayla called her mother to check on Mia, the first thing her mother said was that her sister was being airlifted to another hospital.
Mia had diabetic ketoacidosis (DKA), a lifethreatening condition that is sometimes the first sign of type 1 diabetes in people who have not yet been diagnosed. The helicopter medical transport took Mia to the Barbara Davis Center for Childhood Diabetes, where she was in the intensive care unit for several days with kidney failure and other life-threatening complications. “It was very scary because my sister is a very bubbly personality, and during that time she was not, so you knew something was wrong,” recalls Mikayla. Thankfully, Mia received the medical care she needed, and the family began managing her newly diagnosed type 1 diabetes at home after she was released from the hospital. Mikayla says that Mia is doing great today.
Participating in Type 1 Diabetes Trialnet
Mikayla enrolled in the teplizumab TrialNet clinical trial “to prevent what happened to [my sister] from happening to other people.”
Shortly after Mia’s diagnosis, staff at the Barbara Davis Center—one of 22 clinical research centers participating in TrialNet—suggested that Mikayla, her parents, and her brother be screened for early signs of type 1 diabetes, “to make sure that nobody else was at risk,” Mikayla says. Because type 1 diabetes has a genetic component, relatives of people with the disease have an increased risk of developing it. The screening involved a blood test to look for the presence of five autoantibodies that are early markers of type 1 diabetes. When the screening results came back a few days before the family was scheduled to move to Florida, Mikayla found out that “everyone was clear but me…. I had four of the five markers.” She had stage 2 type 1 diabetes, putting her at high risk of developing clinical (or stage 3) disease. At that time, the scientists told her that it would be about 6 months before she needed to start taking insulin.
Mikayla and her parents also learned that she was eligible to enroll in a new TrialNet clinical trial testing a drug targeting the immune system, called teplizumab, to see if it could delay progression to clinical type 1 diabetes. Because of their move, they were referred to the University of Florida (UF) TrialNet center, where Mikayla enrolled in the trial “to prevent what happened to [my sister] from happening to other people.” She didn’t worry about the possibility of getting randomly selected to the placebo arm of the trial and not receiving teplizumab. She remembers thinking that getting the placebo would be fine, but “if I get the drug, awesome.”
“During the study, the doctors and the nurses were very nice… They made it very welcoming,” says Mikayla, speaking about her experiences with TrialNet scientists and staff.
Mikayla and her mom stayed at a hotel and went to the UF TrialNet clinic each day for the 14-day intervention. “Every day for those 2 weeks, I would sit in the hospital bed,” says Mikayla, where the staff would give her an intravenous infusion treatment that she said lasted 4-5 hours. After the treatment, Mikayla and her mom went to lunch and then rested in the hotel, as sometimes Mikayla had side effects from the treatment. Mikayla remembers the TrialNet staff warmly. “During the study, the doctors and the nurses were very nice.… They made it very welcoming.”
After the intervention part of the trial, Mikayla went to follow-up visits every few months so the TrialNet scientists could measure her blood sugar levels and assess how well her body was producing insulin. (She continues to have follow-up visits yearly.) After a couple years, she was told by the researchers that she had received teplizumab as part of the trial.
Teplizumab Trial Results and FDA Approval
In 2019, TrialNet announced that the trial Mikayla participated in showed that teplizumab could delay diagnosis of clinical type 1 diabetes by 2 or more years among people who were at high risk. The results have since been extended to an average 3-year delay in clinical diabetes onset.
However, Mikayla has that number beat. Nearly 7 years after receiving teplizumab infusions, she is still free of clinical disease and remains insulin independent. “I don’t know how long that’s going to last, but I hope it lasts a little bit longer,” she says. She is grateful that she has been able to navigate her high school and college years without needing to manage type 1 diabetes, knowing first-hand the burden that disease management places on individuals and their families.
For Mikayla, the FDA approval of teplizumab “means that people can at least prolong not having to take insulin, not having to check blood sugar…. I’m very happy about that.”
More exciting news came in 2022, when the U.S. Food and Drug Administration (FDA) announced that it approved teplizumab as the first drug to delay the onset of clinical type 1 diabetes in adults and children 8 years and older who have stage 2 type 1 diabetes. Mikayla says that for her, the approval “means that people can at least prolong not having to take insulin, not having to check blood sugar…. I’m very happy about that.” She’s particularly excited about what a preventive therapy means for children and families. “It gives parents, especially, a sense of relief because managing type 1 diabetes in a kid is very stressful.” When asked if she would encourage others to participate in a clinical trial, she replies, “Yes, I would. If you have the requirements to meet it and you’re available, I would recommend it.”
Now a college senior, Mikayla is pursuing her career goal of becoming a medical illustrator— combining her love of drawing with her passion for anatomy. She is an avid reader and a huge Harry Potter fan. During college, she has served as a resource for friends diagnosed with type 1 diabetes by giving them information about the disease and its management. “They say ‘how do you know all this’ and I say, ‘it’s a long story,’” she recalls with a laugh.
Mikayla also recognizes that she is likely to develop clinical type 1 diabetes in the future. “I do know I’m going to get it eventually. I don’t know when, but I’m more mentally prepared and more physically prepared… I’m just happy it hasn’t happened yet.” Through her dedication to research, Mikayla has not only benefitted from her participation in the trial, but has achieved her goal of helping other people at risk for developing type 1 diabetes.
This content is provided as a service of the National Institute of Diabetes and Digestive and Kidney Diseases
(NIDDK), part of the National Institutes of Health. NIDDK translates and disseminates research findings to increase knowledge and understanding about health and disease among patients, health professionals, and the public. Content produced by NIDDK is carefully reviewed by NIDDK scientists and other experts.