The End of Diabetes: The Eat to Live Plan to Prevent and Reverse Diabetes

Potentially massive news from biotech firm Vertex: a brand new stem cell remedy for type 1 diabetes posted incredible results, lowering insulin utilization by 91% while delivering huge simultaneous improvements in A1C.

How the cure Works

The procedure, which is called VX-880, uses new pancreatic islet cells that have been grown from pluripotent stem cells in a laboratory. Those healthy new islet cells are then transplanted into a patient’s portal vein, which delivers blood from the pancreas to the liver. Once in place, the new islet cells are capable to sense blood glucose concentrations and secrete insulin on demand, just like healthy natural islet cells.

We already knew that islet cell transplants work: some lucky transplant recipients have enjoyed healthy blood sugar without the use of insulin for a decade or more. But until now, doctors were only able to harvest viable cells from the pancreas of a deceased organ donor.

Such donor cells are scarce, and the procedure has generally been limited to patients with a dire need—for example, those with extreme hypoglycemia unawareness or advanced kidney disease. In those rare cases, islet transplantation can be a lifesaver. (In the United States, however, a quirk in the regulations has brought the use of the procedure to a stop.)

The treatment could have a game-changing effect. Islet cell transplantations could become significantly more common with an abundant source of healthy, effective, and safe islet cells. The innovator behind the treatment, Dr. Doug Melton, has set himself the explicit public goal of curing type 1 diabetes. In the past, he has stated that he is “convinced” that stem cells “will cure the disease.”

The Results

The new outcomes are from a single patient, the 1st to receive the new treatment. “Patient 1” is an adult that had been diagnosed with type 1 diabetes some 40 years ago. Before the transplant, they were using 34 units of insulin per day, and had recently experienced multiple life-threatening hypoglycemic episodes. Their A1C was 8.6%, above international glycemic targets for most adults, and they had zero detectable natural insulin production.

90 days after the procedure, Patient 1 had reduced their daily insulin usage to only 2.9 units. Despite using 91% less insulin, they also enjoyed a drop in A1c, down to a much-healthier 7.2%. Measures of insulin production were on the lower end of the ordinary range.

Remarkably, Patient 1 only received half of what Vertex expects to be the full clinical dose, suggesting that they might have enjoyed even better results had they received the full dose.

Immune System Suppression

If there’s a catch, it’s the fact that VX-880 recipients will also require to take immunosuppressive drugs to prevent their bodies from rejecting the transplanted islet cells. Patient 1 used a “standard regimen of immunosuppressive agents,” and will need to keep up with that regimen as long as they hope to keep their new islet cells alive.

It’s unclear how significant the side effects of immunosuppression will be for VX-880, but patient 1 evidently tolerated that their treatment well, with no serious side effects reported.

Vertex is already working on an improved transplantation solution that would not require immunosuppression. The firm is exploring ways to encapsulate the islet cells in a kind of “teabag” or pouch that would allow blood and insulin to filter through, but keep immune cells out. This vision, which may still be many years from fruition, would arguably represent a true cure for type 1 diabetes.

What’s Next?

More than one patient has received the treatment – approximately 16 more are involved in Vertex’s ongoing Phase 1/2 clinical trial, and more will be enrolled. The trial is designed to evaluate results one year after transplantation, with the hope that most patients will experience improved blood glucose control, reduced insulin usage, and fewer hypoglycemic events. Investigators will continue to follow patients and evaluate safety for up to five years.

At the moment, the clinical trials of VX-880 are limited to adult patients with hypoglycemia unawareness and a history of severe hypoglycemia. There’s no telling if or when Vertex might try to test the novel treatment in others.

Patient 1’s great results are incredibly encouraging. Although much study is still required before we can realistically talk about VX-880 becoming available outside of a clinical trial, the recent results are a fantastic first step in Vertex’s efforts.

Diabetes Daily

The End of Diabetes: The Eat to Live Plan to Prevent and Reverse Diabetes