It’s been about one yr since we’ve had a significant update from Vertex Pharmaceuticals, the biotech firm developing a futuristic therapy that would represent a cure for type 1 diabetes. Today, on the primary day of the American Diabetes Association’s annual Scientific Sessions, Vertex released a fresh batch of results from its groundbreaking experiment.

Those results are remarkable: Vertex’s first two patients not need insulin injections to administer their blood sugar levels, and each other patient to receive the experimental treatment has shown impressive glycemic improvements.

Introducing VX-880

The therapy, named VX-880, is a transplant of latest pancreatic islet cells which have been grown in a laboratory from pluripotent stem cells. Those healthy latest islet cells are surgically inserted right into a patient’s portal vein, which delivers blood from the pancreas to the liver. Once in place, the brand new islet cells are in a position to sense blood glucose concentrations and secrete insulin on demand, identical to healthy, natural islet cells.

Trevor W. Reichman, MD, PhD, the Surgical Director of Pancreas and Islet Cell Transplantation on the University of Toronto’s Ajmera Transplant Center, calls VX-880 “first-of-its-kind research [that] could possibly be a gamechanger for the treatment of type 1 diabetes.”

We already knew that islet cell transplants work: Some lucky transplant recipients have enjoyed healthy blood sugar without the usage of insulin for a decade or more. But until recently, doctors were in a position to harvest viable cells only from the pancreas of a deceased organ donor. Because such donor cells are so scarce, the procedure is performed only rarely, in cases of utmost need. (And in america, it’s not performed in any respect.)

In October 2021, Vertex shared the news that its very first patient, known only as Patient 1, had lowered his insulin usage by 91 percent. Before the transplant, he was using a median of 34 units of insulin per day. His A1C was 8.6 percent, above international glycemic targets for many adults, and he had zero detectable natural insulin production.

Ninety days after the procedure, Patient 1 had reduced his day by day insulin usage to only 2.9 units. And despite using 91 percent less insulin, he also enjoyed a drop in A1C, right down to a much healthier 7.2 percent. His insulin production had rebounded to the lower end of the traditional range. The Recent York Times published an exclusive interview with Patient 1, who we learned was a 64-year-old man named Brian Shelton, stating that Shelton “will be the first person cured of type 1 diabetes.”

Though Shelton’s condition had undoubtedly improved, some people within the diabetes community bristled at the outline of a “cure,” noting that each his A1C and his use of insulin appeared to confirm that he still had diabetes.

About six months later, though, the news got even higher: Shelton had stopped using insulin injections entirely, and his A1C had improved to a wonderfully healthy 5.2 percent. At this point, it seemed accurate to say that Shelton not had type 1 diabetes, though he requires immunosuppressive drugs to guard his latest islet cells.

The Newest Results

We now have the small print on six patients who’ve undergone the VX-880 procedure. Prior to their transplants, each of those volunteers had zero detectable natural insulin secretion, and every had a history of severe hypoglycemic events and hypoglycemia unawareness. These volunteers began the study with a median A1C of 8.13 percent.

All six patients have demonstrated:

• Restored insulin secretion
• Improved glycemic control
• Improved time-in-range
• Reduced use of insulin injections
• An entire absence of severe hypoglycemic events

There are actually two patients who’ve accomplished one yr since their transplant. Shelton has now remained insulin-free for a complete of 21 months. Like Shelton, the second patient is off insulin injections entirely, and has an A1C measurement (6.0 percent) comfortably below the diagnostic threshold for diabetes. In recent months, each have achieved a time-in-range of 96 percent or greater.

Of the remaining patients, three have only accomplished 90 days, but “their trajectory is consistent” with the nice results of the primary two transplant recipients. A final patient needed to discontinue the study “attributable to non-trial-related reasons,” in keeping with Dr. Reichman.

Is It a ‘Cure’?

If VX-880 recipients don’t need insulin injections to realize healthy blood glucose levels — presuming that their latest transplanted islet cells proceed to work equally well for the foreseeable future — can we are saying that the treatment is an actual cure for type 1 diabetes?

It’s an open query. Some within the diabetes research community are still reluctant to explain any solution that requires immunosuppressive drugs as a “cure.” VX-880 recipients will still have to take immunosuppressive drugs for the remaining of their lives so as to protect their latest islet cells. In his interview with the Times, Shelton reported that the antirejection drugs “cause him no negative effects,” but the brand new data does show that a number of the patients experienced mild negative effects common to those medications.

In 2022, James Shapiro, MD, the surgeon that performed the world’s first islet cell transplants, explained to Diabetes Every day that “immunosuppressive drugs are the large barrier for why we don’t do large numbers of cell transplants today. The risks include increased risk of cancers, increased risk of life-threatening infections, negative effects on the kidney, they usually will also be toxic to the functioning of the transplanted cells and their ability to make insulin.”

Vertex is pursuing a number of therapies that aim to transcend VX-880 by completely avoiding the necessity for immunotherapy. In March, Vertex announced that it might begin experiments on VX-264, which encapsulates lab-grown islet cells “in a channel array device designed to shield the cells from the body’s immune system.” Last yr it also purchased a competitor, ViaCyte, that had begun experiments on islet cells gene-edited to flee detection from the immune system.

Whether or not you think that of it as a “cure,” VX-880’s impressive results offer hope that scientists are getting closer to a treatment that may allow some patients with type 1 diabetes to realize insulin independence. In the mean time, there’s no telling how long it’d take for VX-880 to win FDA approval, how much it might cost, or who could be eligible for the treatment, but the nice results will allow Vertex to maneuver full speed ahead with the following stage of its trials.