I’ve always said that IF there is a cure for Type 1 diabetes, it will be on the front page of The New York Times. Well, here it is, from today’s (Saturday, 27 November 2021) front section of the digital version of the NYTimes, in bold print …
A Cure for Type 1 Diabetes? For One Man, It Seems to Have Worked.
As reported by Gina Kolata in the Health Section: A new treatment using stem cells that produce insulin has surprised experts and given them hope for the 1.5 million Americans living with the disease. I’ve been posting about Doug Melton’s work at Harvard and then at Semma Therapeutics, which was recently bought by Vertex, which was in my 25 October 2021 post … and NOW, blasted in The NYTimes!
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.
Mr. 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. Mr. 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,” Mr. 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 U.C.L.A. who also was not involved with the research, agreed while offering the same caveats. “It is a remarkable result,” Dr. 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.
Dr. Melton had been studying frog development but abandoned that work, determined to find a cure for diabetes, after his infant was diagnosed with Type 1 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. Dr. 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, Dr. Melton estimates the project cost about $50 million. The challenge was to figure out what sequence of chemical messages would turn stem cells into insulin-secreting islet cells. The work involved unraveling normal pancreatic development, figuring out how islets are made in the pancreas and conducting endless experiments to steer embryonic stem cells to becoming islets. It was slow going.
After years when nothing worked, a small team of researchers, including Felicia Pagliuca, a postdoctoral researcher, was in the lab one night in 2014, doing one more experiment. “We weren’t very optimistic,” she said. They had put a dye into the liquid where the stem cells were growing. The liquid would turn blue if the cells made insulin. Her husband had already called asking when was she coming home. Then she saw a faint blue tinge that got darker and darker. She and the others were ecstatic. For the first time, they had made functioning pancreatic islet cells from embryonic stem cells.
His company Semma was founded in 2014 … and in 2019, Semma was acquired by Vertex Pharmaceuticals for $950 million.
CAVEAT: Like patients who get pancreas transplants, Mr. 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. He will have to continue taking them to prevent his body from rejecting the infused cells.