Jadah: Paving the Path to Preventing Type 1 Diabetes
“All that matters to me is that I’m making a difference,” explains Jadah matter-of-factly when asked why she is part of type 1 diabetes research. Although she hasn’t been diagnosed with type 1 diabetes, 14-year-old Jadah is at high risk of developing clinical symptoms of the disease, such as high blood sugar (glucose) levels. With the goal of making a difference, she is participating in a clinical trial in the NIDDK’s Type 1 Diabetes TrialNet (TrialNet) to test a strategy for stopping the disease process at its earliest stage. People at this early stage have biological factors (autoantibodies) that characterize the beginning of the disease process and are at very high risk of developing clinical symptoms, but they still have normal blood sugar levels. Researchers are also developing therapies for individuals with diagnosed diabetes, which is marked by high blood sugar levels. Type 1 diabetes is an autoimmune disease in which a person’s immune system destroys the cells that make insulin. People with the disease (or parents of children with the disease) must carefully monitor blood sugar levels and administer insulin. It is very difficult for people with type 1 diabetes to achieve a balance between too much sugar in the blood and too little. Too much sugar could lead to serious eye, nerve, and kidney complications; too little sugar could lead to confusion, loss of consciousness, and death. While a goal of type 1 diabetes research is a cure for people who have the disease, a way to prevent the disease could spare future generations from type 1 diabetes.
“All that matters to me is that I’m making a difference,” says Jadah, of her participation in type 1 diabetes research.
The Benefits of Learning Type 1 Diabetes Risk
Type 1 diabetes has been part of Jadah’s family life since 2009, when her brother, Travis, was diagnosed. Because people with a first-degree relative with type 1 diabetes have a 15 times greater likelihood of developing the disease, scientists believe that Jadah and others like her could hold the clues for uncovering how the disease develops and identifying a prevention strategy.
Research has revealed that before type 1 diabetes symptoms appear, the immune system produces specific autoantibodies—proteins that can be detected in the blood. TrialNet’s Pathway to Prevention study screens first- and second-degree relatives of people with type 1 diabetes for these autoantibodies. From the number of different autoantibodies in a person’s blood, they can determine that person’s risk of developing type 1 diabetes. As of January 2018, TrialNet researchers had screened over 160,000 individuals. TrialNet offers yearly rescreening for children under 18 years of age, so a total of over 250,000 screenings have been conducted to date.
Jadah’s mom, Susie, had first seen TrialNet information booths at fundraising walks held by the type 1 diabetes advocacy organization JDRF. Susie was amazed that researchers could determine if there’s further risk of type 1 diabetes in her family. But she was also concerned: would she want to know if Jadah were at high risk?
Susie learned that there were several reasons to find out Jadah’s risk status. People screened by TrialNet and found to be at high risk are eligible to be monitored for the appearance of more autoantibodies and/or for an increase in their blood sugar, which would indicate development of type 1 diabetes. TrialNet’s tests are likely to detect progression to type 1 diabetes before the appearance of potentially serious clinical symptoms. For example, early detection decreases the likelihood of a life-threatening complication, called diabetic ketoacidosis (DKA), at diagnosis.
“It’s not like you are in a study. It’s like you are hanging out with your friends,” says Jadah of her experience in a TrialNet clinical trial.
Travis had DKA when he was diagnosed, and Susie didn’t want the same thing to happen to Jadah. Additionally, if Jadah were found to be at high risk, she could be eligible to participate in a TrialNet prevention trial. Susie realized she did want to know, feeling that “[it] is incredible that you could find out ahead of time if there’s a [high] risk [of type 1 diabetes].” But she and Terry, Jadah’s dad, felt strongly that it was Jadah’s decision.
From Reluctant Screener to Trial Participant
It wasn’t learning about her risk for type 1 diabetes that concerned Jadah most—it was the blood draw needed for the test. “I just hated needles when I was a kid. I mean, I was so scared of them,” she recalls. While visiting Travis at a diabetes camp in 2011, Jadah reluctantly agreed to be screened. Although she did the blood draw, the experience was too much for her. “Right after that I just wanted to leave that camp without even saying goodbye to my brother,” she remembers.
Not long after, Susie and Terry received a call from TrialNet telling them that Jadah was at high risk, but that another blood draw was needed to confirm the result. This time Jadah said no. Susie and Terry tried everything they could think of to change Jadah’s mind, but she held her ground for 4 years. In the meantime, Susie was constantly worried, wondering if every time Jadah seemed sick she was really showing early signs of type 1 diabetes. Eventually, it was Jadah’s grandmother who changed her mind about the rescreening. “I just thought that I should take her advice,” says Jadah, “…I thought this was something that I could do for her, that she would want me to do.”
In April 2015, Jadah went to her local University of Minnesota TrialNet site for her second screening and shortly after learned the result, which confirmed the earlier screening: she was at high risk for developing type 1 diabetes. Because she was at high risk for the disease, but had not yet developed it, that September Jadah was offered a unique opportunity to join a clinical trial testing a drug for prevention of type 1 diabetes.
Whether or not to enroll in the trial wasn’t a difficult decision for Jadah. She had made friends with the staff at the University of Minnesota TrialNet site and enjoyed spending time with them. “I loved these nurses so much that I wanted to be in the study right away, just so I could be with these nurses,” says Jadah. Susie shared her daughter’s enthusiasm for the trial, and they enrolled her.
Taking Part in the Abatacept Prevention Trial
Jadah is participating in a trial testing the agent CTLA-4Ig, also known as abatacept, for its ability to delay or prevent type 1 diabetes in people at high risk for the disease. In 2011, TrialNet investigators found that over a 2-year period, abatacept slowed disease progression for 6 to 9 months, compared to placebo, in people newly diagnosed with type 1 diabetes. This resulted in higher insulin production in the people who had been treated with abatacept. Moreover, this improvement persisted for 1 year after the drug was discontinued. These results suggested that abatacept could be used in combination with other agents for a possibly more robust and prolonged effect in people newly diagnosed with type 1 diabetes, and that it should be tested for its ability to prevent the disease in those at high risk. This led to the trial in which Jadah is participating.
“Being able to have the opportunity to be part of this [research] is amazing,” Susie remarks. “We want to stop type 1 diabetes in this generation, and we are excited to be a part of this important research.”
Jadah’s experience in the trial required her to make three visits to the University of Minnesota TrialNet site in the first 4 weeks, then one visit a month for 11 months. Each visit took about 2 hours, including a 30-minute infusion of the drug or a placebo, followed by 1 hour of observation to make sure there were no side effects or complications, like an allergic reaction. Jadah and Susie turned the visits into a bonding experience, going out to breakfast or lunch afterwards before returning Jadah to school.
A Bond Inspires the Future
Now that Jadah has finished her treatment course, she only returns to TrialNet for blood tests every 3 months and has her blood sugar levels tested every 6 months. She could even do the blood tests at her local clinic, but Jadah quickly said no to that. “Even if you’re in there for 5 minutes,” she explains, “it’s worth it because they [the nurses at the University of Minnesota TrialNet site] take their time and they make you feel comfortable… It’s not like you are in a study. It’s like you are hanging out with your friends.” The TrialNet staff have even inspired her to work toward becoming a pediatrician in the future, as long as she can still have time to visit the lake, camp, and ski.
As for the possibility that she might develop type 1 diabetes, Jadah says, “I know that, even if I do get diagnosed, I’ll still have my family, and they’re going to know what to do. I’m going to know what to do because I see Travis doing it.”
Susie is so grateful that Jadah has had this wonderful experience and what she views as the chance to be part of changing history. “Being able to have the opportunity to be part of this [research] is amazing,” Susie remarks. “We want to stop type 1 diabetes in this generation, and we are excited to be a part of this important research.”
For now, Jadah and her family will continue to raise funds for type 1 diabetes research—with JDRF walks and, for Susie this past summer, through JDRF’s Ride to Cure Diabetes—while they eagerly await the results of the abatacept prevention trial. Does Jadah wonder whether she received the drug or placebo? “Nope, I don’t wonder at all,” she answers. “I just want to find a cure for all the other people out there who have type 1 diabetes.” Jadah is ready to participate again, if needed, in another prevention trial or in a trial for people who are newly diagnosed, if she develops type 1 diabetes.
Jadah and her family encourage anyone who can to get screened by TrialNet and to consider participating in research. “The sooner people get involved, the better we are going to be in the future,” says Susie. “We can’t wait… All we need right now is participants [in clinical trials]. If we get participants, we’ll get a prevention and a cure.”
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. The 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 the NIDDK is carefully reviewed by NIDDK scientists and other experts.