Diabetes Drug May Prevent Autoimmune Disorders
Thanks to a breakthrough drug that delays the progression of type 1 diabetes, Mikayla Olsten has, so far, been able to avoid the complications of the chronic disease.
Mikayla witnessed her younger sister struggle with type 1 diabetes. In 2016 when Mikayla was 14 years old, her sister, Mia, was taken to intensive care with failing kidneys and fluid in her lungs. Mia was diagnosed with ketoacidosis, a life-threatening complication of type 1 diabetes that can lead to diabetic coma or death.
Type 1 diabetes, once known as juvenile diabetes or insulin-dependent diabetes, usually develops in children or young adults but the condition can occur at any age. In Mikayla’s case, her family had no previous history of type 1 diabetes before Mia’s health scare. Nonetheless, Mikayla was tested for signs of diabetes.
As it turned out, Mikayla had four of the five markers that doctors look for in people who could be at risk for developing the disease. Clinicians estimated that Mikayla’s odds of making it through secondary school without developing diabetes was about one in three.
Mikayla was enrolled in a clinical study for teplizumab (pronounced “tep-liz-oo-mab”), a drug designed to delay the onset of diabetes as long as possible. In July 2016, Mikayla received a two-week treatment of teplizumab. Now, Mikayla is a 21-year-old student studying exercise physiology at Brigham Young University–Idaho in Rexburg, and has been diabetes-free for 6.5 years.
“At this point, I’m just grateful that I don’t have diabetes yet,” Mikayla said in an article published in the journal, Nature. “I know that I’m going to eventually have it, and I’m prepared for that. But right now, I’m more just loving my life.”
Claire Wirt, a 16-year-old who also participated in the clinical trial, said she has been free of type 1 diabetes for seven years.
“It gets you so many childhood experiences without having to deal with diabetes 24/7,” Claire, a resident from Rochester, New York, said in an article published in the journal, Nature.
Most individuals who develop type 1 diabetes might not know they are at risk for its diagnosis and they may not have a family member with the disease, according to researchers involved in the study.
“Therefore, most patients arrive at a physician’s office or emergency room with stage 3 type 1 diabetes in which the classic symptoms of the disease, including diabetic ketoacidosis—a serious diabetes-related complication—have already appeared,” Dr. Kevan Herold, a Yale Medicine endocrinologist who was involved in the teplizumab trials, said in an article for Yale Medicine.
Type I Diabetes Symptoms
It can take months or years before type 1 diabetes symptoms are noticed, according to the U.S. Centers for Disease Control and Prevention. Type 1 diabetes symptoms can develop in just a few weeks or months, and once they appear, they can be severe. Symptoms of this chronic condition include:
- Blurry vision
- Feeling very hungry
- Feeling very thirsty
- Feeling very tired
- Frequent urination
- Losing weight without trying
- Having numb or tingling hands or feet
- Having very dry skin
- Having more infections than usual
People with type 1 diabetes need to take insulin shots or wear an insulin pump every day to survive.
Diabetes Drug Receives FDA Approval
The promising results of the clinical trial were instrumental in the U.S. Food and Drug Administration (FDA) approving teplizumab to delay the onset of Stage 3 type 1 diabetes in adults, and pediatric patients 8 years of age and older with Stage 2 type 1 diabetes.
In announcing the approval of Tzield (pronounced TEE-zeeld), the brand name of teplizumab, Dr. John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research, said the therapy adds an important new treatment option for certain at-risk patients.
“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease,” Dr. Sharretts said in a press release.
Type 1 diabetes develops when the pancreas makes little to no insulin, a hormone that helps blood sugar enter the body’s cells so it can be used for energy. In the first stage of the disease, T cells—which help protect the body from infection—mistakenly attack beta cells in the pancreas that produce insulin.
In the second stage, some beta cells are destroyed, leading to abnormal blood sugar. In Stage 3, nearly all beta cells are destroyed, leading to type 1 diabetes symptoms. Teplizumab slows T-cell destruction of beta cells, delaying the onset of Stage 3 type 1 diabetes.
Teplizumab’s Trial Results
The clinical trial in which Mikayla participated involved 76 people with stage 2 type 1 diabetes and ran from 2011 to 2018. Of the 76 participants, 55 were under age 18 and all had a relative with type 1 diabetes. Study participants were randomly assigned to a placebo group and the teplizumab group.
Researchers found that 72 percent of participants who received the placebo developed diabetes symptoms, compared to 43 percent of the teplizumab group. The average time for people in the placebo group to develop clinical diabetes was just 24 months, while those in the teplizumab group had an average of 48 months before progressing to a diagnosis, according to the study.
While teplizumab delays the onset of stage 3 diabetes, the effects are only temporary, which is why Dr. Herold said he is interested in combining the drug with other treatments that may maximize its therapeutic benefits.
“I could envision starting patients out with teplizumab when they’re first diagnosed, and then perhaps intermittently give them another immune suppressant that maintains the effect,” Dr Herold said. “That’s another area we’re quite interested in pursuing.”
Dr. Jay Skyler, deputy director for clinical research and academic programs at the Diabetes Research Institute (DRI) at the University of Miami Miller School of Medicine in Florida, said the DRI participated in the teplizumab studies. While there was celebration over the approval of teplizumab, “it doesn’t mean we’re done,” Dr. Skyler said in an article published in the journal, Nature. “It means we’re just beginning.”
Source Links:
https://www.nature.com/articles/d41586-023-00400-x
https://medicine.yale.edu/news-article/for-type-1-diabetes-teplizumab-tzield-preserves-beta-cell-function-and-shows-significant-other-benefits/
https://www.prnewswire.com/news-releases/fda-approves-first-drug-that-can-delay-onset-of-type-1-diabetes-301682219.html
https://www.niaid.nih.gov/research/drug-delaying-type-1-diabetes
https://www.cdc.gov/diabetes/basics/what-is-type-1-diabetes.html
https://www.trialnet.org/events-news/blog/teplizumab-first-drug-delay-diagnosis-t1d-median-two-years
