Weekly reads: stem cell therapies that work, exosomes, H3.3 K27M, Joe Rogan

How close are we to having new stem cell therapies that work?

stem cell therapy, Stem cell therapies that work
Sometimes one patient in a clinical trial on cell therapies has a startlingly good response but it’s an outlier event and the therapy is never approved.

The field is nearly there on several fronts, but we shouldn’t get ahead of ourselves. Last week I noted the “soon” in a Nature News headline “Stem cells head to the clinic: treatments for cancer, diabetes and Parkinson’s disease could soon be here.” It seemed a little much to me.

We are definitely getting closer on some fronts and it’s exciting, but the final stretch often takes years longer than one might hope. In scrolling back to old posts here on The Niche, I often find the word “soon” in media pieces about stem cell therapies that I noted. Yet, in all those cases now 10 or even 14 years later, nothing has come of those investigational cell therapies in question. It’s easy to spark false hope.

Let’s start with another new piece that arguably got ahead of where things stand on stem cell therapies.

Stem cell therapies that work?

Stem-cell therapies that work: 10 Breakthrough Technologies 2025, MIT Tech Review. That definitive headline caught my eye. I would think in reading it that these therapies have FDA approval. Instead, the piece includes two still investigational stem cell therapies together as an item in a top 10 list of breakthrough technologies.

The piece highlights work from Neurona Therapeutics on its investigational epilepsy cell therapy and Vertex’s investigational diabetes cell therapy.  They are both exciting, but these therapies have not actually been proven to work yet in groups of people.

It’s crucial to be at least somewhat cautious with N=1 (or even N=2, etc.)-type reports.

All this being said, one of my predictions for 2025 for the regenerative medicine field is that the FDA will approve at least one new regenerative therapy after its recent Mesoblast approval. It’s not just the science though. Politics will play a major role.

Other stem cell and regenerative reads

Blast from the past

This is only two years ago, but fits with the risks of false hope and the N=1 trap: Overplaying N=1 in the stem cell therapy world.

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