This Stem Cell Treatment Could One Day Make Insulin Obsolete

Type I diabetes is an exhausting full-time job. Having it means living a life full of constant care and maintenance. You’re always checking in on your blood sugar to make sure it isn’t too high or is it too low, extremes that could be easily reached with the most minor indulgence or tiniest bout of laziness.

A new treatment, as detailed in the New York Times, might change everything we know about managing type I diabetes.

Zimislecel is an experimental stem cell-based therapy that recently dropped a bomb on the diabetes world. Developed by the Boston-based Vertex Pharmaceuticals, it’s a one-time infusion that has turned 10 of 12 trial patients suffering from severe type I diabetes into people who no longer need insulin, and in less than a year.

Scientists turned stem cells into pancreatic islet cells, which are little insulin factories, and infused them into patients, where the cells made themselves at home in the liver. They immediately got to work.

Most patients were insulin-free by six months. Life-threatening glucose drops, called hypoglycemic unawareness, disappeared within three months.

This Treatment Could Free Some With Type 1 Diabetes From Insulin

The results were revealed at the American Diabetes Association’s annual conference and published in The New England Journal of Medicine, where researchers and endocrinologists collectively lost their minds. In the New York Times piece cited above, Doctor Mark Anderson of UCSF, who was not involved in the study, said a lot of nice things about the therapy, which I will summarize as if they were quotes from a movie poster: “Trailblazing!”, “Life changing!”, “The feel-good type I diabetes treatment of the year!”

I made up that last one, but it fits.

But wait. Hold on. Timeout. Let’s tamp down the excitement for a second because there is a catch. For now, patients have to take immunosuppressive drugs, possibly for life, to stop their own bodies from killing the newly implanted cells.

Those drugs come with their baggage, like infections, cancers, and the nagging sense that you may have traded one horrible thing for another. It’s also currently unclear if zimislecel will work for people whose type I diabetes isn’t as severe as those who participated in the study.

With those caveats out of the way, which are admittedly substantial, the treatment is still quite promising. Should it end up revolutionizing the way we treat diabetics, we can all thank Dr. Doug Melton, a Harvard scientist whose children were both diagnosed with type I diabetes, so he made it his life’s work to figure out a way to make their lives a little easier.

He spent two decades researching this treatment, using up over $50 million in funding, before he finally landed on a winning formula.