Dear Doctors: As someone who was born with Type 1 diabetes, I’m very interested in any new advances that make it easier to live with this disease. I’ve recently heard on the news that someone has actually been cured. Is that possible? Is there going to be a treatment soon?
Dear Reader: You’re referring to the release of preliminary data from a clinical trial that is testing a potential cure for Type 1 diabetes. The trial is quite small — just 17 participants — and the initial result concerns only one person. However, the outcome is so remarkable, it’s making international news. According to the Massachusetts-based pharmaceutical company conducting the trial, the first participant has not only decreased his daily insulin use by 91%, but for the first time in his life, his body is producing insulin in response to blood sugar fluctuation.
For those who are not familiar with Type 1 diabetes, it’s an autoimmune disease. This occurs when the immune system malfunctions and mistakenly attacks and damages the body’s own tissues. In Type 1 diabetes, certain white blood cells attack and destroy the clusters of specialized cells in the pancreas known as islets, which produce insulin. Without insulin, the cells of the body can’t access blood sugar, which is one of the products of digestion. Not only does this leave cells without their main source of energy, but it results in an uncontrolled buildup of glucose in the blood, which is extremely dangerous.
Keeping blood sugar within a healthful range when you have Type 1 diabetes requires constant testing, daily insulin and a carefully controlled diet. Even so, people living with the disease are at increased risk of a range of adverse health effects, including heart attack, stroke and nerve impairment. The disease is the leading cause of blindness and kidney failure in the United States.
The only known cure for Type 1 diabetes is either a pancreas transplant or a transplant of the specialized pancreatic cells that produce insulin. But with a shortage of available organs and 1.6 million people in the U.S. living with the disease, a cure for the vast majority is not possible. That’s what makes the positive results in this clinical trial such big news. The first participant, who is 64, has been living with Type 1 diabetes for nearly 50 years. His disease is so severe, he regularly experienced sudden drops in blood sugar that caused him to lose consciousness. A few days after receiving an infusion of stem cells that have been “taught” how to behave like islets, everything changed. His blood sugar readings were in the normal range. Even after eating a meal, which requires the body to secrete insulin in order to manage blood glucose, his readings remained perfect.
If this first participant continues to respond to ongoing treatment, his will be the first-ever functional cure of diabetes. It’s a thrilling — but also very early — result. There are 16 more participants in the clinical trial, and five years left to go. Whether or not the man continues to respond to treatment, and whether or not the results can be duplicated in other patients, remains to be seen.
Eve Glazier, M.D., MBA, is an internist and associate professor of medicine at UCLA Health. Elizabeth Ko, M.D., is an internist and assistant professor of medicine at UCLA Health. Send your questions to firstname.lastname@example.org, or write: Ask the Doctors, c/o UCLA Health Sciences Media Relations, 10960 Wilshire Blvd., Suite 1955, Los Angeles, CA, 90024. Owing to the volume of mail, personal replies cannot be provided.