By Brian Shelton, a retired Post Office worker, may be the first person to be cured of Type 1 diabetes using a new treatment that helps the body regulate its own insulin levels. The treatment is part of an ongoing clinical trial that will take another five years to complete, but it’s welcome news for some 1.5 million people living with the disease nationwide.
A New Lease on Life
Diabetes dominated Shelton’s life for years. He would lose consciousness without warning when his blood sugar levels plummeted, leading him to crash his motorcycle. He also passed out in someone’s yard while delivering mail. His declining health led to an early retirement at age 57. His condition got so bad that his ex-wife Cindy Shelton decided to stay with him. “I was afraid to leave him alone all day,” she said.
Earlier this year, she saw a listing for a clinical trial for those with severe Type 1 diabetes. It was conducted by Vertex Pharmaceuticals, whose chief scientist has been working on a cure for the disease for years ever since his baby son and teenage daughter were diagnosed.
Shelton had the privilege of being the first patient. Doctors gave him an infusion of cells created from stem cells designed to mimic the ones that produce insulin in the pancreas. His body can now regulate insulin levels on its own, making him the first person to be cured of the disease via cell infusion.
“It’s a whole new life,” Shelton said. “It’s like a miracle.”
The scientists were enthusiastic but urged caution. The trial is ongoing with another 17 participants awaiting treatment. The results need to be replicated in more people before seeking regulatory approval. The researchers also have yet to publish their findings in a peer-reviewed journal.
Many experts were quick to praise the results.
Dr. Irl Hirsch, a diabetes expert at the University of Washington who was not involved in the research, said, “We’ve been looking for something like this to happen literally for decades.”
It’s not clear how long the results will last or if there are adverse side-effects, but “bottom line, it is an amazing result,” said Hirsch.
Dr. Peter Butler, a diabetes specialist at UCLA who also was not involved with the research, took the news in stride. “It is a remarkable result,” Butler said. “To be able to reverse diabetes by giving them back the cells they are missing is comparable to the miracle when insulin was first available 100 years ago.”
The disease can be fatal unless patients receive a dose of insulin right away.
“It’s a terrible, terrible disease,” Butler added.
Type 1 diabetes is also the leading cause of blindness and kidney failure in the U.S. A lack of insulin can also lead patients to having their legs amputated. It also increases the risk of heart attack and stroke.
The price of insulin continues to rise year after year, making it a costly disease to maintain. The only other known cure for Type 1 diabetes is a pancreas transplant or a transplant of the insulin-producing cell clusters of the pancreas from a donor, but there just aren’t enough transplant organs to go around.
Dr. Ali Naji, a transplant surgeon at the University of Pennsylvania and principal investigator on the clinical trial, said, “Even if we were in utopia, we would never have enough pancreases.”
Looking for a Cure
The clinical trial was a passion project for Dr. Doug Melton, a Harvard biologist that’s been searching for a cure for over 30 years.
Melton started focusing on the disease in 1991 after his six-month-old son started shaking and vomiting.
“He was so sick, and the pediatrician didn’t know what it was,” Dr. Melton said. He and his wife Gail O’Keefe took him to Boston Children’s Hospital where they found high levels of glucose in his urine, a sign of diabetes.
Experts say the disease typically starts at around ages 13 or 14, making his son a rare case. Melton and his wife quickly realized how difficult caring for a child with diabetes can be. O’Keefe had to prick the child’s fingers to test his blood sugar levels four times a day. They also had to inject him with insulin, which isn’t readily available in children’s doses, so they had to dilute it.
“Gail said to me, ‘If I’m doing this you have to figure out this damn disease,’” Dr. Melton recalled. A few years later, their daughter was diagnosed with disease when she was 14.
Melton quit his work studying frogs to pursue his goal of finding a cure. He quickly turned his attention to embryonic stem cells, which can turn into any type of cell in the body. With a team of 15 scientists, he was able to turn these cells into islet cells, the insulin-producing cells in the pancreas, after around 20 years of work. The project cost around $50 million overall, according to Melton.
The scientists first had to study how islet cells are made in pancreas. They used blue dye to track the stages of the stem cells as they develop. Once they had the formula, they could design islet cells from scratch. The team celebrated by making blue hats with different colors for each stage of the development process.
As the company continues studying the treatment, it will need to find a way to bring it to market. Vertex Pharmaceuticals says it won’t announce a price until it has been approved, but the company has a history of inflating prices for its drugs, often to the chagrin of patients.
Melton says he was ecstatic when he discovered the treatment was a success. “Let’s just say there were a lot of tears and hugs.” He later had dinner with his wife, son, and daughter.
