The Niche - Stem Cell Blog & Resources

There has been some discussion that the next-generation research on human stem cell-based embryo models (SCBEM) could someday warrant a Nobel Prize for some of those involved. At this point, however, I believe that’s unlikely. How do we evaluate the worth of and reward innovative biomedical research? Let’s start by talking a bit about Nobel Prizes. SCBEM Nobel in the future? Knoepfler image. Nobels above all else?

Readers of The Niche will recall that I’ve been skeptical of the efforts of the de-extinction firm Colossal Bio. For instance, I don’t believe they de-extincted dire wolves. They did make woolly mice, maybe their fluffiest (in a real sense) achievement. The point of that apparently was to be on the road to de-extincting woolly mammoths. After all these years, that objective is still a terrible idea. Now, oddly, the firm is teaming up with the Trump administration.

Will using AI in biology be revolutionary? If you’re a scientist, how often do you use Chat GPT or similar AI for your biomedical research? For writing? Are you more annoyed or enthralled with how AI might impact biology and the daily work of biologists? In the middle? Whatever your answers, Anthropic is hoping that in the future you will turn to its new AI tool Claude Science for many of your tasks as a life scientist. Even things like paper writing.

Recently, I predicted that RFK Jr. may stack the key pharmacy compounding committee, called PCAC, with fans of unproven peptides. Kennedy wants the unproven “pop peptides”, as I call them, to be unleashed. If the PCAC advises allowing pop peptide compounding, it gives Kennedy more leverage to overrule FDA scientists. You might recall there are literally zero good clinical trial data on these peptides such as BPC-157, Ipamorelin, etc.

The last eighteen months have been an unprecedented time of unpredictability for the FDA, but moving forward we’re likely going to see an emerging consistency: a higher amount of FDA flexibility on rare diseases. It’s already begun and is likely to accelerate with Prasad and Makary gone. How is this manifesting? FDA flexibility on rare diseases. Knoepfler image. FDA flexes flexibility I wrote last week about UniQure getting some friendly treatment from the FDA.

Are stem cells for autism promising? The answer so far is a clear “no”. Even a Duke team that was for a time very upbeat about the potential of umbilical cord cell therapy for autism seems to have lost that enthusiasm. For example, they stopped their EAP of cord cells for autism. I also don’t see that they have any active trials in this space on Clinicaltrials.gov either. In addition, the Duke Cryo-Cell deal working toward cell infusion clinics including potentially for autism is apparently finished.

I’ve been following the Huntington’s disease space carefully for about 15 years. I always hoped that some kind of promising gene therapy might emerge. Stem cell therapies or combination cell-gene therapies could have promise for Huntington’s too. Over the years, I got to know Judy Roberson, a wonderful HD patient advocate. I’m going to start this week’s wrap of regenerative medicine research with an FDA decision on HD. Patient advocates view this with hope. uniQure CEO Matt Kapusta.

I’ve often wondered why there hasn’t been more research on macrophage therapy. Macrophages are some of the coolest cells in the body. They are capable of not just eating other cells (hence their name), but also numerous other functions. Graphical abstract of paper on macrophage therapy. Cell Stem Cell, 2026, Brennan, et al. Before we dive into that, below is the latest stem cell video from my collaboration with Parmin Sedigh. Check it out and please subscribe to both of our YouTube channels.

One of the questions I get asked most often about pop peptides is why they are so popular. What puts the “pop” in pop peptides? FDA warnings on pop peptide compounding, marketing. Peptide mania After all, these peptides like Ipamorelin, BPC-157, and so on don’t have clinical trial data to support the uses for which they are being marketed. That makes them potentially unsafe and a waste of money. Yet RFK Jr. is intent on making the FDA unleash peptides on the public.

If you take a statin to improve your lipid profile, someday could you undergo LDL gene editing instead? Might that day be sooner than we think? A new paper has generated great interest in this topic including in the media. LDL gene editing paper Figure 1, NEJM 2026. LDL gene editing For example, we have: One-and-Done Heart Disease Prevention? Scientists Show It May Be Possible. A single infusion of an experimental gene-editing drug seemed to reduce LDL long-term in a small trial.