July 13, 2020

The Niche

Knoepfler lab stem cell blog

Advanced Cell Technology (ACTC) announces plan to make iPS cell-derived platelets: some thoughts

ACTCAdvanced Cell Technology (ACT) is well into clinical trials for macular degeneration (the leading cause of blindness) using human embryonic stem cell (hESC)-based retinal pigmented epithelial cells (RPE). To date, the trials suggest the products are safe. Efficacy? We don’t know. I am cautiously hopeful, but it is frustrating to know that most clinical trials fail.

Lest you think that ACT is a one trick pony, it is doing a bunch of other interesting stuff too including using induced pluripotent stem (iPS) cells. Over the years ACT has had a mixed attitude toward iPS cells, at one point suggesting that they age too fast, while at other times seeming more enthusiastic.

Well, that enthusiasm was on display today in a WSJ article by Jonathan Rockoff (cool name) that was strangely entitled “Stem-Cell Trial Without Embryo Destruction”, making me wonder if ACT had finally gotten through the thick heads of the conservatives about their blastomere technology for making hESC, but that wasn’t it. The article was focused on ACT’s plans to use iPS cells to make a platelet product for treating people with various disorders. Given that this was the uber-conservative, Karl Rove-employing WSJ, perhaps it is not so surprising that they obsessively focused on the embryo part, but really the most exciting element is the hope that unlimited patient-specific platelets could become a reality, saving a lot of people’s lives.

In theory this might also make ACT and its investors bundles of money. ACT’s stock has not had a good year so they could use some holiday cheer.

Of course the platelet therapy might be a long way down the road, but apparently ACT is pitching the idea of trials to the FDA at this point, which is encouraging.

One of the benefits of a platelet-based product for ACT and for patients is that platelets have no nuclei. Thus, they cannot divide nor carry genetic information. As a result, while nothing is absolutely safe, iPS-derived platelets would seem to be intrinsically far safer than say RPEs made from hESC or iPS cells, which already seem relatively safe (so far at least).

There still could be some risk from residual iPS cells, but one can imagine a number of strategies for lowering such risk given the unique nature of platelets. For example, even though iPS cells are smaller, platelets are super tiny compare to iPS cells, perhaps allowing for a size exclusion step.

The billion dollar question is whether the iPS cell-derived platelets would behave like normal platelets. If so, this is a revolutionary therapy for many conditions. The WSJ piece, which by the way (errantly?) called ACT a Massachusetts company rather than a California one, ended:

If the stem-cell derived platelets worked like normal platelets, “the potential impact would be great,” said Cynthia Dunbar, a stem-cell researcher at the National Heart, Lung and Blood Institute and editor of the journal Blood.

Reason for excitement, but as usual coupled to a good dose of patience. Still, I’d predict that most likely in 5-10 years ACT is going to be a household name. I know….that’s still a long long time.

Note: I do not own stock in ACT nor do I give financial advice. Financial decisions should be made with a qualified financial advisor. Investing in biotech stocks is inherently extremely risky.