Weekly reads: stem cells for MS, good news x2, extending dog years, Neuralink updates

For about as long as I’ve been writing The Niche, people have been asking about stem cells for MS. There’s a huge need for new therapies.

While a chemo-based approach with autologous hematopoietic stem cell transplantation (HSCT) looks to work for certain cases of multiple sclerosis (although not yet approved in the US), other cell therapy approaches are being tested as well. Let’s start there.

stem cells for MS
A distinct approach to stem cells for MS involves putting neural stem cells directly into the brain.

Stem cells for MS: right into the brain?

Early-stage stem cell therapy trial shows promise for treating progressive multiple sclerosis, University of Cambridge.

My first thought on seeing this headline was “What is the rationale for putting stem cells directly into the brains of MS patients?”

Here’s the research article in Cell Stem Cell: Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis. The article suggests possible mechanisms:

“Recent preclinical data suggest that NSCs can target some of the core drivers of MS progression, including the persistent smoldering activation of myeloid cells, when delivered by intracerebroventricular injection (ICVI). NSCs reduce the pro-inflammatory activation of myeloid cells by inducing a metabolic reprogramming of myeloid cells toward oxidative phosphorylation”

From the article, “We report the analysis of 1 year of data from the first cohort of 15 patients enrolled in an open-label, first-in-human, dose-escalation phase I study (ClinicalTrials.gov: NCT03282760, EudraCT2015-004855-37) to determine the feasibility, safety, and tolerability of the transplantation of allogeneic human neural stem/progenitor cells (hNSCs) for the treatment of secondary progressive multiple sclerosis.”

No severe adverse events were reported and the dose of the NSCs seemed to correlate with parenchymal brain volume changes in an encouraging way.

There are still unknowns from this trial. As the authors conclude:

“Although our extensive animal work points to the ability of both syngeneic and xenogeneic NSC grafts to dampen inflammation and provide neuroprotection at different levels and at the same time to progress toward cell differentiation, integration, and replacement in vivo; however, the mechanism(s) of action of this novel ACT drug remains undetermined, given the impossibility to document donor cell survival, integration, and fate following transplantation in human subjects.”

Some participants did have adverse events but 100% of these were defined by the team as unrelated to the intervention. These were sick patients taking other drugs, but I do wonder how confidently clinical researchers can rule out a connection to the investigational drug and/or procedure in question.

stem cells for MS
3 stem cells for MS headlines paint very different pictures.

A tale of 3 headlines on stem cells for MS

I found three featured headlines on this story of stem cells for MS. And, yes, despite my concerns about Google Search, I still do use it sometimes.  From left to right, the first two headlines are reasonable. The Guardian headline is probably the best since it notes this is an early trial and says “encouraged.” Neuroscience News is reasonable too.

The Telegraph headline is awful. It’s irresponsible to mention a cure that way given the state of the research. We can call that one our dumb stem cell headline of the week.

Good news: many more FDA approvals & cool UC Davis trial

I went into my list of FDA-approved cell and gene therapies to update it and got a surprise.

There were a lot more to add to the list.

Some of the new ones are cell therapies too including one for Type 1 diabetes. I’m planning to cover some of these in more depth in the future.

More good news closer to home: State stem cell agency, Shriners Children’s award $15 million to UC Davis CuRe trial. It’s exciting that my colleagues are running one of the most promising stem cell clinical trials in the world.

Neuralink troubles

US Lawmakers Ask SEC to Launch Fraud Investigation Into Elon Musk, Wired. Democratic lawmakers have asked the FEC to investigate Neuralink and Musk after allegedly false statements about how the company’s animal experiments were going.  This follows on Neuralink saying a few months back that it had FDA permission to proceed with its PRIME trial of its N1 brain implant. I still see no listing for the PRIME trial on Clinicaltrials.gov, which is odd. Earlier the FDA had denied the Neuralink’s request for a trial.

Despite all these ups and downs, Neuralink recently raised $43 million USD.

Other regenerative reads

2 thoughts on “Weekly reads: stem cells for MS, good news x2, extending dog years, Neuralink updates”

  1. Per the MS trial, can the results be driven by the placebo effect? We know it is high in many clinical trials and very high in stem cell based trials especially those that are more complex. One clue can be if there is a difference between the doses. You mentioned a dose-escalation study, so was there any difference? Is the low dose working better than the higher dose?
    I’m not saying that there is no effect, or that it even needs to be compared to a placebo or a sham procedure. I actually think that in early trials and maybe in some cases in more advanced trials we should consider comparing the results to historical data and not always to placebo.

  2. Paul, thank you for all you are doing! Your site is informative and provides
    a true look into what is going on in the stem field world. Are there any updates
    with COPD/Emphesema type stem cell procedures? Where are the companies
    now, that were offering bogus procedures? Is there any company working on
    any productive product?

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