When I’m asked about the most promising areas of stem cell clinical research, cell therapy for type I diabetes is near the top including work by a biotech called ViaCyte. Other firms are in the mix too, boosting the overall odds of success in the coming decade.
It’s really that promising even though there are no guarantees, of course.
Now a new major biotech development in this space could change things in a big way. Good? Bad? It’s more complicated than that.
Cell therapy for diabetes: ViaCyte vs. Semma
Both firms have used human embryonic stem cells (hESC) to make pancreatic cells.
One big difference has been in the types of pancreatic cells.
ViaCyte has focused on stem cell-derived pancreatic precursors, which in part mature inside a capsule once inside patients. In contrast, Semma has focused on starting with pure beta cells as their therapeutic. This beta cell approach seems to be a passion of Doug Melton, who founded Semma.
In terms of other differences, ViaCyte is arguably at least a few years ahead of Semma in terms of product testing and clinical development. For example, ViaCyte has more clinical trial data including on different capsule technologies, tested to some extent in actual patients. My impression is that Semma had made less progress on encapsulation.
Overall, there was some healthy competition between the two firms.
However, things have dramatically changed in the last three years.
Vertex acquires Semma, pushes forward with VX-880
The first big biotech development here was that Vertex Pharmaceuticals acquired Semma in 2019. Compared to stem cell biotechs, Vertex is huge. It had more than $4 billion in revenue in 2019. They have various types of products in development and aren’t just focused on cell therapies.
The experimental cell therapy for diabetes is named VX-880. From the company:
“VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. VX-880 is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production. VX-880 is delivered by an infusion into the hepatic portal vein and requires maintenance immunosuppressive therapy to protect the islet cells from immune rejection.”
From 2019 forward without Semma independently in the mix, the focus was more on Vertex and ViaCyte in this space. Both their approaches to cell therapy for diabetes have faced challenges and could have unique advantages. Again, one could consider this healthy competition.
However, perhaps the giant Vertex might be hard for the much smaller ViaCyte to compete with moving forward? The question has become moot.
ViaCyte to also become part of Vertex
Here’s the potentially game-changing headline from Fierce Biotech: Vertex absorbs ViaCyte and its stem cell-based diabetes treatment for $320M, clearing out competition.
So now Vertex will own all that was both ViaCyte and Semma.
Will this yield synergism?
Perhaps the Semma pipeline of beta cells can benefit from ViaCyte’s capsule technology. That may be a key factor in the acquisition.
Positive synergism with big resources?
Hopefully this will lead to synergism.
But what does the future hold for ViaCyte’s pancreatic precursor cell approach?
One concern is that Vertex may opt to only proceed with one of these two main cellular approaches. Since Melton, who has focused for ages on the beta cell approach, is now a Vertex leader after leaving Harvard, it’s possible that the ViaCyte cell approach could be sidelined in favor of the beta cells.
I don’t know that there’s enough data to narrow the focus at this point to only one of the two. That possibility of a narrowed focus has risks.
On the other hand, Vertex has big resources to drive stem cells for diabetes. It also seems clear to me that Vertex is incredibly engaged in this pipeline, which is great. Could Vertex pursue both approaches and trials? Probably not, but we’ll see.
Vertex recently made headlines with news of one patient infused with their cellular product becoming free of needing insulin injections. To me this infusion approach with no capsule seems like a long shot. There was also a recent FDA hold on the Vertex’s VX-880, but it has been released.
I also wonder about cost of a potential future treatment.
Current standard long-term care for diabetics is very costly so we have to think about this cellular approach in that context. A cell therapy could be a cost saver overall. A safe and effective therapy is also likely to be covered by insurance.
I can realistically imagine an exciting future where type I diabetics have fresh sets of 2-6 pancreatic cellular capsules implanted subcutaneously every five years or so, which keeps their blood sugar effectively controlled. That’s the bold vision here and it could be achieved in a decade.
Let’s hope that the cell therapy for diabetes clinical research space remains vibrant.
References and notes
- Vertex trial listing on its stem cell product for diabetes, VX-880.
- Note that some stem cell clinics are selling and certain researchers are studying other cell therapies for diabetes including especially MSCs, but to me these don’t make much sense so far.
- It’s also notable that the term “stem cell” therapies for diabetes doesn’t necessarily mean that stem cells will be directly infused into patients. Rather, stem cell-derived pancreatic cells seem the more promising way to go and are still considered “stem cell therapies”.