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

In October, we reported on the remarkable success of the 1st type 1 diabetes patient in the earth to receive a transplant of laboratory-produced islet cells. The new cells secrete insulin in response to blood glucose levels, replacing the patient’s Beta cells that lost function decades ago.

Vertex released an update this month, and that 1st patient is doing even better: 270 days after receiving his transplant, he has now stopped utilizing insulin injections entirely.

The Treatment

The groundbreaking type 1 diabetes treatment, an innovation of Vertex Pharmaceuticals, is named VX-880. It 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. Because such donor cells are so scarce, the procedure is performed only rarely, in cases of extreme need. (And in the United States, it’s not performed at all.)

Patient 1

The early data—from 90 days after the surgery—showed that Patient 1, as Vertex calls him, had lowered his insulin usage by 91%.

Before the transplant, he was using 34 units of insulin per day. His A1C was 8.6%, above international glycemic targets for most adults, and he had zero detectable natural insulin production.

90 days after the procedure, Patient 1 had reduced his daily insulin usage to only 2.9 units. Despite using 91% less insulin, he 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.

When we first covered the story, Patient 1 was otherwise a mystery. Not long after, the New York instances published an exclusive interview with Patient 1. We learned that he was a 64-year-old man named Brian Shelton. He had struggled with life-threatening hypoglycemia unawareness for years.

VX-880, according to Mr. Shelton, was “like a miracle.”

The Update

Now we be aware of a little bit about how Mr. Shelton has fared 270 days into his treatment, and the news is fantastic.

According to Vertex, he is now completely insulin-independent. Just as impressively, his A1C has declined to 5.2%, well within the range for metabolically healthy, non-diabetic adults.

There’s another patient, Patient 2, who has completed over 150 days of cure. We don’t have the latest numbers, but Patient 2 is also doing well, and “has shown robust increases in fasting and stimulated C-peptide, and reductions in exogenous insulin requirements.”

Patients 1 and 2 actually achieved their improvements with only half of the dosage that Vertex hopes to use in most future recipients. (It is typical to examination the safety of lower doses of a new therapy before experimenting with higher doses.)

The first person to receive the full dosage is Patient 3. We have limited data on Patient 3, who only underwent surgery recently. In the month since, they have displayed “increasing fasting C-peptide and improving glycemic control,” but a more comprehensive examination will have to wait until day 90. More patients are lined up to receive the full dose of VX-880 treatment.

There was also some bad news, however: the FDA has asked Vertex to pause its clinical trials. Apparently, federal regulators are unsure that the full dosage treatment should be used in additional patients. A Vertex spokesperson stated that the business was “surprised by the clinical hold on the study,” and pointed to safety and efficacy data that had been validated by an independent committee.

We’re not sure why the FDA has asked Vertex to slow down, but it’s certainly a reminder of the rocky road that even the most promising of treatments have to travel on their slow journey to federal approval. While Vertex’s innovation is extraordinarily exciting, it may be wise to remember how many times the hopes of the diabetes community have been frustrated or dashed.

Is it a “Cure”?

If Patient 1 doesn’t need insulin injections to achieve healthy blood glucose levels—presuming that his new transplanted islet cells continue to work equally well for the foreseeable future—can we say that he has been cured?

If there’s a good reason for doubt, it’s this: VX-880 has not allowed Mr. Shelton to go off medication for his condition entirely. He now needs to take immunosuppressive drugs to prevent his body from rejecting the transplanted islet cells, and he will need to take them for the rest of his life.

According to the Times, Mr. Shelton reported that the anti-rejection drugs “cause him no side effects.” He may well believe that he’s been cured.

That’s extremely encouraging, but others in the diabetes research community are still reluctant to describe any solution that requires immunosuppressive drugs as a “cure.”

When we spoke to Dr. James Shapiro, the surgeon that performed the world’s first islet cell transplants, he explained that “immunosuppressive drugs are the big barrier for why we don’t do large numbers of cell transplants today. The risks include increased peril of cancers, increased risk of life-threatening infections, side effects on the kidney, and they can also be toxic to the functioning of the transplanted cells and their ability to make insulin.”

Dr. Shapiro is just one of many researchers that is working on a treatment that does not require immunosuppression. He has partnered with ViaCyte, a Vertex competitor, on a therapy using islet cells that have been gene-edited to escape detection from the immune system. ViaCyte and Vertex are also both working on physical barriers to encapsulate and protect transplanted islet cells, a “teabag” approach that would let insulin filter out but prevent immune cells from getting in.

Takeaways

VX-880, the innovative stem cell transplantation treatment for type 1 diabetes, is working better than anyone expected. The first patient to receive it is on the path towards legitimate diabetes remission—and that’s with only a half dose of new islet cells. It is getting more and more reasonable to wonder if Patient 1 has truly been “cured” of his type 1 diabetes.

However, we also learned that the FDA has asked Vertex to pause its experiment. We’re not sure what has prompted the delay, or how long it will last, but it’s a reminder that even the most promising therapies invariably confront unforeseen obstacles.

Diabetes Daily

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