FDA Versus Stem Cell Therapies

Who owns your cells? The FDA seems to think it does, given its lawsuit against Regenerative Sciences, a company that treats orthopedic injuries by extracting, culturing and reinjecting adult stem cells derived from a patient’s bone marrow.

The case is precedent-setting in that FDA is claiming authority to regulate a patient’s own cells as though they were chemical drugs. As one researcher describes it:

If you start to look at this product as being the patient’s own stem cell, how can the FDA claim Regenerative is manufacturing [cells] – they’re culturing them. . . . They seem to have lost perspective on using autologous stem cells. There’s just no way you could apply manufacturing standards. . . . The FDA does not come into a cardiology practice and tell doctors how to do their surgeries or how to do heart replacements. And yet they feel they can come into a stem cell clinic.

The problem with FDA “coming into a stem cell clinic” is that this could have a significantly chilling effect on this whole field of medical research. Under the burden of FDA’s regulatory intervention, the costs of developing adult stem cell treatments would explode and treatments that might have otherwise been profitable might never even make it to market—as has happened with drug development in the U.S. And while stem cell therapies are under FDA review, patients will be denied government permission to use treatments derived from their own cells.

Christopher Centeno, the director of Regenerative Sciences, described to Forbes his company’s perspective on the case:

We see this lawsuit as a 21st century civil rights issue that will define what control you have about the use of your own cells and tissue. If a loved one is dying in intensive care and a well done study shows that the patient’s own cells can be used to help, does the patient get to decide to use those cells, or is that a decision for the FDA? Will the patient still be alive while we wait on Washington to issue this decision?

The prospect of patients dying waiting for FDA to act is, unfortunately, all-too real.

Because the agency has the power to keep drugs and medical devices off the market while its regulators plod through their labyrinthine review process, doctors and patients are forbidden by law from using treatments that lack the FDA’s stamp of approval, even with informed consent. In cases where the patient has little time left and no other options, this can lead to tragic outcomes.

Consider a device designed to replace a brittle aortic valve that was recently approved by the FDA. During a four-year period of FDA’s review, the device was used safely in Europe while being denied to American patients. According to AEI’s Scott Gottlieb:

more than 15,000 patients world-wide will receive the device by the time it’s slated for approval in the U.S. Some Americans healthy enough to fly have sought the procedure in Europe. Tens of thousands of Americans unable to travel, and too sick to undergo open-heart surgery, have died during the intervening four years.

How would you feel watching a loved one die, knowing that the only thing standing between him and a possible life-saving treatment is a group of government bureaucrats who think they have the right to deny it to him?

Or consider the beta-blocker class of drugs. In the mid-70’s, these had been shown to reduce the risk of secondary heart attack and some were adopted for this purpose overseas. “But in the U.S.,” writes CEI’s Sam Kazman:

FDA imposed a moratorium on beta-blocker approvals due to the drugs’ carcinogenicity in animals. . . . In effect, FDA was denying needed cardiac drugs to people at high risk of heart attacks because of the unproven possibility that those drugs might cause cancer years in the future. Finally, in 1981 FDA approved the first such drug, boasting that it might save up to 17,000 lives per year. That meant, of course, that as many as 100,000 people may have died waiting for FDA to act.

Kazman calls these many thousands of individuals—in this and other similar cases—the FDA’s “invisible victims.”

The field of adult stem cell therapy has been slow to develop and treatments that show signs of real promise are only just beginning to emerge. It would be tragic if regulatory intervention by FDA were to stifle this field in its infancy.