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

March 3, 2024 / Professor Paul Knoepfler, Ph.D. / 1 Comment

“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.

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 […]

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I found something Elon Musk & I agree on: pushing back on anti-aging bluster

February 28, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Would you guess that Elon Musk might be into using biotechnology in a high-profile way to try to live far longer than usual, say to age 150? That would have been my assumption. There are countless things about Elon Musk that I really don’t like. I also don’t see many issues on which we are

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Weekly reads: brain aging, perinatal stem cell clinics, $1M lab meat fine bill

February 18, 2024 / Professor Paul Knoepfler, Ph.D. / 1 Comment

What happens during brain aging and how can we tell if dementia is coming? Are there particular early hallmarks? There are an increasing number of medical tests for predicting or detecting dementia. Alzheimer’s disease can often be detected early. But what do patients or their doctors do with such information? Until recently there weren’t any

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Weekly reads: Xist & autoimmune disease in women, Crohn’s disease, dumb headline of the week

February 4, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Readers of The Niche have asked me many questions about stem cells for autoimmune disease but the puzzle of why women get these conditions more often than men hasn’t come up before here. For instance, why is MS so much more common in women than men? It’s remained somewhat of a mystery over the years.

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Weekly stem cell reads: cancer paper mess at DFCI, H3.3, knees, surfer keeps on posting

January 28, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Sholto David, stem cell, cancer retractions

It’s another one of those ‘double-grant’ weekends of grant writing (on brain cancer) and reviewing, but I’m still trying to find a bit of time for some other reading. There was some important news this week including a big mess of problematic papers at Dana Farber Cancer Institute (DFCI). Recommended reads Here’s the new WaPo

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Weekly reads: Vertex & CRISPR Therapeutics, Arnold Caplan death, MS genetics

January 14, 2024 / Professor Paul Knoepfler, Ph.D. / 5 Comments

The biotechs Vertex and CRISPR Therapeutics have an interesting relationship as biotechs. They are partners are multiple levels but also are very different as companies including in size. There’s been a key development in one of their partnerships. Before we jump into that, please check out the video version of my 20 stem cell and

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Dishing on biobots like xenobots & anthrobots vs. organoids

January 9, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

If you remember xenobots, mobile clusters of frog cells, now you might be interested to learn that some of the same team brings us human cell clusters called anthrobots. Both frog and human cellular clusters are considered types of biobots or robots made of cells. To me, anthrobots seem akin to human organoids or assembloids.

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Surprising new FDA remarks to AABB on cord biologics misuse & stem cell clinics

December 19, 2023 / Professor Paul Knoepfler, Ph.D. / 8 Comments

The Association for the Advancement of Blood & Biotherapies or AABB recently had a very useful Q&A with the FDA on cord blood use. There are some important new things from the FDA on the marketing and use of cord blood in there. The agency was also quite blunt in some ways about the challenges,

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Weekly reads: late Macchiarini retractions, stem cells & Lululemon

October 29, 2023 / Professor Paul Knoepfler, Ph.D. / 8 Comments

Paolo Macchiarini is one of a small group of people in the stem cell universe whose misconduct has blown up in the press. Piero Anversa, Haruko Obokata, Hwang Woo-Suk, and some operators in the unproven stem cell clinic sphere come to mind. Macchiarini published quite a few seriously problematic papers, some of which just hung

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Vivek Ramaswamy & his surprising stem cell start

September 5, 2023 / Professor Paul Knoepfler, Ph.D. / 2 Comments

Many of us first became aware of Vivek Ramaswamy as a Republican candidate for President. He was most well-known prior to that as a biotech leader. Given that biotech background, I decided to learn more about him. Surprisingly, his biomedical efforts may have started with stem cells. That caught my attention. This was way back during

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