Vertex upbeat on 1st stem cell therapy for diabetes trial participant

It's hard in the cell therapy field not to get excited even with bits of seemingly encouraging news but with N=1 reports we have to be very cautious and that's the case with news from Vertex on their 1st stem cell therapy for diabetes trial participant.

Vertex stem cell therapy for diabetes: N=1

This need for soberness came to mind with this new piece Vertex gets much-needed win with 'extraordinary' first patient results on potential diabetes cure in ENDPOINTS.

What's the scoop here?

One trial participant with Type I diabetes reportedly had improved blood sugar stability after receiving a half-dose of the Vertex cellular therapy VX-880.

The man is around 90 days out from infusion with the experimental cell therapy.

As a reminder, Vertex is a large pharma company that acquired the small biotech Semma that was started by Doug Melton.

The current trial work has its foundation from Semma.

Vertex vs. ViaCyte

Where do we go from here on stem cell therapy for diabetes?

We should take it step by step.

It's good for patients and the field that there's competition in this space including from ViaCyte, which itself released its own similar update on 1 patient back in June.

The two companies are using somewhat similar approaches based on human pluripotent stem cells. I call this a stem cell therapy because it's based on stem cells even though stem cells themselves are not directly used.

They each differentiate the cells in distinct ways toward the pancreatic lineage, which includes beta cells that make insulin.

I believe the intended delivery systems are somewhat different but involve capsules. ViaCyte seems far ahead on the encapsulated device front.

Interestingly the first Vertex participant was given an infusion.

"While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression," said Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.

Why an infusion to start? It's a puzzling choice for a diabetes therapy. Do the cells engraft? Get rejected by the immune system? I guess one big advantage is it can be done quickly and simply as opposed to an encapsulated device.

Looking ahead on stem cells for diabetes

ViaCyte has had to make several adjustments in their clinical trial design over the past few years. For that reason I'd be surprised if Vertex also didn't have to do that kind of thing too. Still it's possible Vertex has learned from some of what ViaCyte's experience.

I'm optimistic that a cell therapy approach of some kind will be proven safe and effective for at least helping Type I diabetes within a decade. Yes, that's a long timeframe but these things always take way longer than you think. We've come a long way in the diabetes area in the last decade.

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