The news this past week carried three reports related to developments in stem cell research. Briefly, starting with the farfetched and ending with the “nearfetched”:
First, the Wall Street Journal reported on efforts to “grow” human organs in the lab. People who try to “keep score” of therapeutic results with adult vs. embryonic stem cells may recall a case in recent years in which surgeons (in Germany, I believe) fashioned a new trachea, or windpipe, for a patient using that patient’s own adult stem cells. The current report follows this track. In its most daring application, a heart is “grown” in the lab. Well, not exactly: it’s not like researchers expect a clump of cells to magically take on the full, functional form of a human heart in a Petri dish. (I’m sorry, I should be serious, but I cannot but recall my late, lamented Weekly World News carrying the front page story that Hitler’s nose had been cloned in a Petri dish—complete with a photo of the darn thing, mustache included.) Seriously, these researches take an approach similar to the windpipe case. They obtain a donated heart from someone who has died (i.e., a cadaver heart), and strip off all the cells, leaving only the foundation of connective tissue, the scaffold on which the functional heart cells had rested. Then, they take adult stem cells—the idea is that these come from a patient whose heart is failing, who ordinarily would need a transplant—and place those on the scaffold, taking steps to induce the cells to differentiate into heart cells. It’s still very early, but in concept this approach to organ engineering looks promising. The cadaver heart need not be fresh, as it must for a transplant as currently practiced. That means that there is no urgency to harvest the heart immediately after death, and there would be no pressure on the definition of death, and no concern about over-eager transplant surgeons jumping the gun. Also, the “scaffold” is not particularly immunogenic, so the patient’s immune system would “see” the immunologic type of the patient’s own cells.
Now, there are years—10, at least—of research needed to find out whether this will work, but the approach appears promising. If it does pan out, it seems to me the principal ethical issue invoked will be justice: how much will it cost, and who will be able to get it (i.e., will we socialize the cost)? That is for another day.
Second, a federal court let stand a decision favoring the FDA over Cytori Therapeutics, in a case involving the company’s device that allows surgeons to separate out adult stem cells from a patient’s fat. The initial application has been in breast reconstructive surgery, and I believe the device is approved in at least one country outside the U.S. In the U.S., however, the FDA is going to insist that the company prove, in a large, randomized clinical trial (at least one of which is in progress) that the device is safe and effective. This places the device in a high-risk regulatory category (like, for example, cardiac defibrillators), not a lower risk category that would allow the company to get fast approval simply by showing that the device is similar to a prior, or as the FDA calls it, a “predicate” device. (There are further nuances, because in some cases, if the risk is low enough, FDA lets a new device without a predecessor serve as its own “predicate” device, but that is too far into the weeds for this post.) In any event, the court sided with the FDA, and the company was not surprised—this has been going on for a while. But it is consistent with FDA’s approach to adult stem cells in general, which is that the Agency views them as sufficiently manipulated to require full-blown development, as for new drugs—even if all a doctor does is take tissue from a patient, spin it in a centrifuge to pull out the stem cells, then re-inject the same stem cells, with no other doctoring, back into the same patient. That, in turn, is what lay behind the State of Texas trying to circumvent the FDA in recent past, to let doctors use adult stem cells in some ways for their own patients. But the FDA will win this, in the end. The net effect will be to slow down the availability of treatments using adult stem cells, and to make it necessary that deep-pocket companies bear the costs of the research. On the margin, as I have blogged in the past, I think the FDA’s maybe being a bit too conservative, but “it is what it is.”
Third, the California Institute of Regenerative Medicine (CIRM), the body that was created with several million dollars’ funding a few years ago, largely for the purpose of promoting research with embryonic stem cells—which require destruction of one or more embryos to be obtained—is establishing a stem cell bank—not with embryonic stem cells, but with induced pluripotent stem cells (iPSCs) and adult stem cells derived from them. Cells will be obtained from people with a variety of chronic diseases that are priority areas for research supported by the CIRM. Makes all the sense in the world from the standpoint of basic and translational (applied) research. The takeaways? The CIRM is following the science, and not all of the research it supports is ethically questionable (although some still is, and there are other issues with how the CIRM operates that I won’t go into here).
To close, a reminder to scientifically uninitiated social conservative types: we are not “against stem cell research.” We are against unethical research, including research that does not adequately protect human subjects (including the unborn), wherever it is proposed.