Last month, FDA commissioner Scott Gottlieb and Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, coauthored a special report in the New England Journal of Medicine entitled “Balancing Safety and Innovation for Cell-Based Regenerative Medicine,” in which they outline new regulations designed to expedite approval of stem-cell therapies to treat various diseases and disorders. Encouraging innovation is consistent with the direction that the FDA has taken under Gottlieb. These policies, however, especially concerning stem cells, represent an unfortunate entrenchment of the status quo.
Stem cells have numerous therapeutic uses, some of which have been controversial. One of the simpler treatments involves autologous stem cell transplants: a doctor extracts stem cells from a patient’s body and uses them to treat a medical problem in the same patient. The procedure can be done in a day or over the course of several visits if the doctor chooses to multiply the patient’s cells before reinjecting them—a process dubbed “cell culturing.” One key objection to unregulated autologous stem cell transplants is that transplanted cells may multiply, migrate, or metastasize in unanticipated ways. You get treated for a bum knee, the argument goes, and two years later, you have a tumor in your lung. In other words, like prescription drugs, stem-cell transplants come with the potential for unforeseeable, harmful side-effects, and as such need to be studied, approved, and fitted with an FDA warning label before being used.
But as Manhattan Institute senior fellow Peter Huber has noted, there’s no way to make one-size-fits-all determinations about the safety and efficacy of treatments engineered using a patient’s own cells, which are ultimately “controlled entirely by the patient’s own unique code.” A standard hip-to-knee stem-cell transplant may work wonders for one patient but have a nasty side-effect for another. As with any procedure, autologous stem-cell treatment is a matter of informed choice.
The procedure was relatively uncontroversial until the FDA filed suit against Regenerative Sciences, a group of physicians in Colorado that had been treating orthopedic injuries with autologous stem-cell transplants. At issue was the cell culturing: according to the FDA, once a patient’s cells had been multiplied by a doctor, they now constituted a drug, subject to FDA regulation, even though the altered cells were being returned to the patient from whom they were taken. After an extended legal battle, the U.S. District Court for the District of Columbia ruled against Regenerative Sciences in 2012, effectively upholding the FDA’s sweeping definition of what constitutes a drug.
Many objected to the decision, claiming that it allowed the FDA to meddle in the practice of medicine. In the Wall Street Journal, a onetime FDA official wrote that the FDA had “stretched that definition” of a drug “to the point where a reasonable limit no longer exists,” and that the consequences could be “deadly.” The official was Scott Gottlieb, later to rejoin the agency when President Trump appointed him to head it.
When the FDA began regulating autologous stem-cell transplants like a drug, it set a dangerous precedent for federal overreach in the practice of medicine. To put it in perspective, consider the fact that the FDA does not regulate organ transplants, which involve a second party (and a host of potential complications). By furthering this precedent in his capacity as FDA commissioner, Gottlieb has come close to embracing the “purely oppositional attitude” he once rightly criticized as an impediment to medical innovation.
Gottlieb remains a huge improvement on his predecessors, who dragged their feet on approving stem-cell treatments, but this is a crucial moment in the new era of FDA regulation. Time will tell whether we see more of Scott Gottlieb, forward-thinking and unapologetic innovator.
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