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Weekly reads: Vertex, stem cells for MS, Athersys, 900-day paper review at Nature – The Niche

Vertex Pharmaceuticals has a lot going for it right now on the cell therapy front. For instance, they have the strongest type 1 diabetes cell therapy pipeline after some recent acquisitions. Still it’s not a simple matter to succeed in the cell therapy space even with one therapy for one targeted disease. Trials are tough to run, expensive, and groups of participants can be complex with many other health conditions or comorbidities.

Let’s start with a trial development in January for Vertex.

“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.
Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.

Vertex pause

Vertex Pauses Islet Cell Study After Patient Deaths, Medscape. I’m a little behind on this news from early January. From the article, “Vertex Pharmaceuticals, Inc. has paused a study of its investigational allogeneic stem cell–derived, fully differentiated pancreatic islet cell replacement therapy (VX-880) following two patient deaths.”

The deaths include that of Brian Shelton who was the first to get lab-grown cells for diabetes. He had been doing very well after receiving the cells. I’m not clear on the cause of death for him or the other participant who died, but the company said it was unrelated to VX-880. They are continuing another trial too.

“Vertex is continuing with a phase 1/2 clinical trial of a different product, VX-264, which encapsulates the same VX-880 cells in a device designed to eliminate the need for immunosuppression.”

Perhaps the pause has now been lifted? So far, I haven’t been able to find out. I don’t see anything on the company news page. I’m still cautiously optimistic that Vertex will ultimately get a diabetes cell therapy approved.

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