The development of a new drug to cure Type 1 diabetes is big news, a triumph of medicine. The process of developing the medicine and bringing it to market interests me for several reasons. One is that Doug Melton spent 20 years of effort on it. His passion was due to having skin in the game: he has two children with the disease. Another is that it took so many years “of painstaking, repetitive, frustrating work.” I emphasize the common importance of trial-and-error in many major medical discoveries. Another is that the trial-and-error was to develop a “chemical cocktail to turn stem cells into islet cells.” Several major medical advances have required nimble and persistent trial-and-error to adjust drug cocktails, in terms of components and doses. Examples include HIV, Hodgkin’s lymphoma, and childhood leukemia.
A final reason I am interested in the case is that Melton selected the Vertex company to bring the drug to market. Vertex is an interesting case of a large firm struggling to keep the innovative culture of its startup roots. I read a book about its struggles called The Antidote. I intend to read an earlier book about its early years called The Billion Dollar Molecule.
(p. 17) A single infusion of a stem cell-based treatment may have cured 10 out of 12 people with the most severe form of type 1 diabetes. One year later, these 10 patients no longer need insulin. The other two patients need much lower doses.
The experimental treatment, called zimislecel and made by Vertex Pharmaceuticals of Boston, involves stem cells that scientists prodded to turn into pancreatic islet cells, which regulate blood glucose levels. The new islet cells were infused and reached the liver, where they took up residence.
The study was presented Friday evening [June 20, 2025] at the annual meeting of the American Diabetes Association and published online by The New England Journal of Medicine.
“It’s trailblazing work,” said Dr. Mark Anderson, professor and director of the diabetes center at the University of California in San Francisco. “Being free of insulin is life changing,” added Dr. Anderson, who was not involved in the study.
. . .
The treatment is the culmination of work that began more than 25 years ago when a Harvard researcher, Doug Melton, vowed to find a cure for type 1 diabetes. His 6-month-old baby boy developed the disease and, then, so did his adolescent daughter. His passion was to find a way to help them and other patients.
He began, he said, with an “unwavering belief that science can solve the most difficult problems.”
It took 20 years of painstaking, repetitive, frustrating work by Dr. Melton and a team of about 15 people to find the right chemical cocktail to turn stem cells into islet cells. He estimated that Harvard and others spent $50 million on the research.
Dr. Peter Butler, a professor of medicine at the University of California, Los Angeles and a consultant to Vertex, said he was awed by the achievement of the Harvard team.
“The fact that it worked at all is just freaking amazing to me,” he said. “I can guarantee there were a thousand negative experiments for every positive one.”
When Dr. Melton finally succeeded, he needed a company to take the discovery into the clinic. He joined Vertex, which took up the challenge.
For the full story see:
(Note: ellipsis, and bracketed date, added.)
(Note: the online version of the story was updated June 21, 2025, and has the title “People With Severe Diabetes Are Cured in Small Trial of New Drug.” The online version says that the article appeared on page 24 of the New York edition of the print version. But the article appeared on page 17 of my National edition.)
The NEJM academic article co-authored by Melton and mentioned above is:
The books that I mentioned about Vertex are:
Werth, Barry. The Antidote: Inside the World of New Pharma. New York: Simon & Schuster, 2014.
Werth, Barry. The Billion-Dollar Molecule: One Company’s Quest for the Perfect Drug. New York: Simon & Schuster, 1994.