Weekly reads: upbeat stem cell clinical trials update, CRISPR ups & downs, story of 1st marrow transplant survivor

It’s always great to read about stem cell clinical trials that are looking encouraging.

Stem cell clinical trials

Proving a new therapy is safe and effective is so challenging. Remarkably, there are more than 9,300 stem cell clinical trials listed for a “stem cell” search on Clinicaltrials.gov. Of course, not all of these are actual active interventional studies transplanting cells, but hundreds fit the bill.

If you limited the Clinicaltrials.gov search to actively recruiting interventional stem cell trials, the search still yields more than 1,300 listings. I’ve captured a map of these listings, which I’ve included below. Today I’m going to start off by talking about one specific trial that is groundbreaking.

stem cell clinical trials
Stem cell clinical trials map. Pic from Clinicaltrials.gov.

I’m not involved in it, but a spina bifida stem cell clinical trial here at UC Davis has had my attention for years.

Now our local paper, The Sac Bee, reports on encouraging early results from the spina bifida trial. Some initial participants have done unexpectedly well, which is encouraging. There’s a long road ahead with the trial, which uses a cell therapy patch to protect the spinal cord during development. The leaders of the trial are my colleagues Drs. Diana Farmer and Aijun Wang. CIRM has provided key funding for this clinical work.

I’ve written before about this spina bifida trial and its preclinical work in bulldogs. You can see a pic of one of the dogs below. Bulldogs have a high rate of spontaneous spina bifida.

arthur the dog helps test stem cells for spina bifida
Arthur the dog helps test stem cells for spina bifida.

Some more recommended reads

The role of Notch signaling in endometrial mesenchymal stromal/stem-like cells maintenance, Comm Bio.

CRY2 isoform selectivity of a circadian clock modulator with antiglioblastoma efficacy.”, PNAS. Inhibiting glioblastoma stem cells could be a major step toward an effective therapy.

A human iPSC-array-based GWAS identifies a virus susceptibility locus in the NDUFA4 gene and functional variants, Cell Stem Cell.

Aplastic Anemia: Nancy Lowry – Longest Living Transplant Survivor, BMT Infonet. I didn’t know this, but Nancy was the recipient of the first successful bone marrow transplant and she’s still alive and doing well to this day. What a story.

Gene editing accuracy and challenges

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells, Cell Stem Cell.

Pervasive donor DNA integration defies precision gene editing of hematopoietic stem cells, Cell Stem Cell.

1 thought on “Weekly reads: upbeat stem cell clinical trials update, CRISPR ups & downs, story of 1st marrow transplant survivor”

  1. Hello I know you’re extremely skeptical when it comes to mesenchymal stem cells but…..

    This clinical trial took place at UC Davis where you work correct Dr. Knoepfler? Well guess what they used to cure these babies of their spina bifida? Placenta derived mesenchymal stem cells!!!!!! Now I know we don’t know if these MSCs actually cured their spina bifida but the fact that these babies came out of the womb kicking their legs and wiggling their toes is a very good sign according to the doctor who ran the trial.

    I’m hoping you respond to me I’d like to talk to you?

    This is how I originally learned about MSCs, thanks to Joe Rogan, so this Joe Rogan interview is how I first came to know about MSCs, this is just the first ten minutes the whole interview is on Spotify, watch? I think you’re familiar with Neil Riordan aren’t you?

    So I’ve been studying MSCs since 2018 now and I’ve also learned about exosomes as well. Now I know you’re extremely skeptical about MSCs but in my opinion, umbilical cord derived MSCs are revolutionary, Neil Riordan claims that umbilical cord MSCs can cure congestive heart failure. I think it’s just a matter of time til umbilical cord MSCs successfully pass a placebo controlled phase 3 trial, it’s just a matter of time til the FDA approves MSCs.

    There have been some companies that failed phase 3 trials recently like Mesoblast, but guess what they were using bone marrow MSCs, Neil Riordan says in his book that umbilical cord MSCs are the most efficacious so that’s why he uses UC-MSCs at his clinic in Panama. Btw quite a few hollywood celebrities have been to Neil Riordan’s clinic in Panama. One of these days UC-MSCs are going to pass a placebo controlled phase 3 trial with flying colors, it’s coming!

    Please publish this comment thanks.

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