October 22, 2020

The Niche

Knoepfler lab stem cell blog

ACT patent filing for MSCs: of Dancing Mice & Multiple Sclerosis

Is Advanced Cell Technology (ACT) on the road to cure Multiple Sclerosis (MS) using stem cells and specifically MSCs?

dancing mice stem cells

Some folks seem to think it’s quite possible, but I remain skeptical.

Still there have been some interesting developments.

ACT has a relatively new patent filing for a mesenchymal stromal cell (MSC) product that was just recently published.

The filing has 125 claims. Wow.

Mentioned prominently in the patent filing is MS as a target disease. In fact, there are rumors that ACT has already “cured” MS in mice leading to “dancing mice” that if not for a stem cell treatment should be paralyzed.

Talk of dancing mice in the stem cell field (as opposed to say the movie, Cinderella) should be viewed cautiously.

Still, one wonders what the back story is here and it turns out to be intriguing.

advanced cell technology

A member of the Investor Stem Cell Site named Patti apparently transcribed a recent talk by ACT CSO Bob Lanza in Boston, which contained the following section that seems relevant here:

We’re starting to now look in various animal models. So one of the first that we looked in was the experimental autoimmune encephalitis, the DAA model. A mouse is one of the best models we have right now for multiple sclerosis. So when you induce the equivalent of MS in these animals, you have a clinical score. So when you get to 2, these animals are unable to use their forward limbs, they’re paralyzed. By the time they’re up to 4, they’re completely paralyzed, all of their limbs are paralyzed. So what we were able to show is that we can go in with MSCs. In this particular model we’re using is very aggressive, severe clinical symptoms, when you go in with bone marrow MSCs, we see minimal impact on the course of the disease. By contrast, when you go in with the MSCs derived from the embryonic stem cells, completely knocks it out. The animals have clinical scores of less than 1; in many cases almost completely normal at 0. So these animals are jumping around, you know currently with no symptoms.

This is a surprisingly definitive statement, but I’ll wait to see the data before making any conclusions.

I would also note that recently some serious questions have been raised about how effective mouse models of inflammatory human diseases  (the so-called EAE (experimental autoimmune encephalitis) mouse model would fit into this category) actually are given the big differences in how humans and mice react to these kinds of diseases.

Getting back to the MSC patent, an important question stands out: how is this patent filing unique and hence would not infringe on the scads of existing MSC-related patents?

The title of the filing gives a potential clue (emphasis mine):

METHODS OF GENERATING MESENCHYMAL STROMAL CELLS USING HEMANGIOBLASTS

It would seem that the sourcing of the MSCs from hemangioblasts is a key distinguishing feature.

Another question that then logically follows is that if MSCs are already so readily available from fat, bone marrow, and other sources, why should anyone get particularly excited about yet another source of MSCs?

In addition, since ACT’s way of making MSCs clearly requires more than minimal manipulation, their product is definitely a biological drug subject to relatively more lengthy FDA regulatory oversight. While I think that oversight is appropriate, it is important to mention this reality in the context of competitors using MSC products doing everything under the sun and moon to avoid such FDA regulation.

This makes for a difficult potential future market for ACT’s MSC product should this patent be awarded down the road.

I’m not trying to be a wet blanket here, but it’s important to place this in the appropriate context.

In the patent filing, ACT states (emphasis mine):

The hemangioblast-derived mesenchymal stromal cells of the instant invention retain a novel, youthful phenotype as defined by expression or lack thereof of specific markers.

This would seem to be the key element: ACT’s MSCs stay young for a long time, whereas generic run of the mill MSCs say taken from a liposuction aspirate do not.

Let’s see how this develops, but it seems to me that no dancing in the streets (especially on Wall Street) by humans or mice seems appropriate based on this.

Not yet at least.

Disclosure: I do not have stock or any financial interest in ACT. 

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