A cure for Type 1 diabetes? For one man, it seems so
New treatment based on infusion of insulin-producing cells surprises experts, offers hope to the 1.5 million sufferers in the U.S.
Brian Shelton’s life was ruled by Type 1 diabetes. When his blood sugar plummeted, he would lose consciousness without warning. He crashed his motorcycle into a wall. He passed out in a customer’s yard while delivering mail. Following that episode, his supervisor told him to retire, after a quarter-century in the Postal Service. He was 57.
His ex-wife, Cindy Shelton, took him into her home in Elyria, Ohio. “I was afraid to leave him alone all day,” she said.
Early this year, she spotted a call for people with Type 1 diabetes to participate in a clinical trial by Vertex Pharmaceuticals. The company was testing a treatment developed over decades by a scientist who vowed to find a cure after his baby son and then his teenage daughter got the devastating disease.
Brian Shelton was the first patient. On June 29, he got an infusion of cells, grown from stem cells but just like the insulin-producing pancreas cells his body lacked.
Now his body automatically controls its insulin and blood sugar levels.
Shelton, now 64, may be the first person cured of the disease with a new treatment that has experts daring to hope that help may be coming for many of the 1.5 million Americans suffering from Type 1 diabetes.
“It’s a whole new life,” Shelton said. “It’s like a miracle.”
Diabetes experts were astonished but urged caution. The study is continuing and will take five years, involving 17 people with severe cases of Type 1 diabetes. It is not intended as a treatment for the more common Type 2 diabetes.
“We’ve been looking for something like this to happen literally for decades,” said Dr. Irl Hirsch, a diabetes expert at the University of Washington who was not involved in the research. He wants to see the result, not yet published in a peer-reviewed journal, replicated in many more people. He also wants to know if there will be unanticipated adverse effects and if the cells will last for a lifetime or if the treatment would have to be repeated. But, he said, “bottom line, it is an amazing result.”
Dr. Peter Butler, a diabetes expert at UCLA who also was not involved with the research, agreed while offering the same caveats.
“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.”
And it all started with the 30-year quest of a Harvard University biologist, Doug Melton. He had never thought much about diabetes until 1991 when his 6-month-old baby boy, Sam, began shaking, vomiting and panting.
“He was so sick, and the pediatrician didn’t know what it was,” Melton said. He and his wife, Gail O’Keefe, rushed their baby to Boston Children’s Hospital. Sam’s urine was brimming with sugar — a sign of diabetes.
The disease, which occurs when the body’s immune system destroys the insulin-secreting islet cells of the pancreas, often starts around age 13 or 14. Unlike the more common and milder Type 2 diabetes, Type 1 is quickly lethal unless patients get injections of insulin. No one spontaneously gets better.
The only cure that has ever worked is a pancreas transplant or a transplant of the insulin-producing cell clusters of the pancreas, known as islet cells, from an organ donor’s pancreas. But a shortage of organs makes such an approach an impossibility for the vast majority with the disease.
For Melton and O’Keefe, caring for an infant with the disease was terrifying. O’Keefe had to prick Sam’s fingers and feet to check his blood sugar four times a day. Then she had to inject him with insulin. For a baby that young, insulin was not even sold in the proper dose. His parents had to dilute it.
“Gail said to me, ‘If I’m doing this, you have to figure out this damn disease,” Melton recalled. In time, their daughter Emma, four years older than Sam, would develop the disease, too, when she was 14.
Melton had been studying frog development but abandoned that work, determined to find a cure for diabetes. He turned to embryonic stem cells, which have the potential to become any cell in the body. His goal was to turn them into islet cells to treat patients.
One problem was the source of the cells — they came from unused fertilized eggs from a fertility clinic. But in August 2001, President George W. Bush barred using federal money for research with human embryos. Melton had to sever his stem cell lab from everything else at Harvard. He got private funding from the Howard Hughes Medical Institute, Harvard and philanthropists to set up a completely separate lab with an accountant who kept all its expenses separate, down to the light bulbs.
Over the 20 years it took the lab of 15 or so people to successfully convert stem cells into islet cells, Melton estimates the project cost about $50 million.
Melton then set up a company to make a drug that could get to market. It was acquired by Vertex Pharmaceuticals in 2019. Their challenge was to make sure the production process worked every time and that the cells would be safe if injected into patients. Two years later the FDA allowed Vertex to begin a clinical trial with Shelton as its initial patient.
Like patients who get pancreas transplants, Shelton has to take drugs that suppress his immune system. He says they cause him no side effects, and he finds them far less onerous or risky than constantly monitoring his blood sugar and taking insulin.
Last month, Vertex was ready to reveal the results to Melton. He did not expect much.
“I was prepared to give them a pep talk,” he said.
Melton, normally a calm man, was jittery during what felt like a moment of truth. He had spent decades and all of his passion on this project. By the end of the Vertex team’s presentation, a huge smile broke out on his face; the data were for real.
He left Vertex and went home for dinner with Sam, Emma and O’Keefe. When they sat down to eat, Melton told them the results.
“Let’s just say there were a lot of tears and hugs.”