Should the US Food and Drug Administration (FDA) have the authority to regulate cells derived from-and later put back into-a person?
A recent opinion-editorial published in The Wall Street Journal by former FDA Associate Commissioner Scott Gottlieb and Coleen Klasmeier, head of FDA practice at the law firm Sidley Austin LLP, argues FDA's efforts to increase regulatory enforcement against stem cell manufacturers has the potential to backfire against the nascent and potentially promising industry.
At issue, write Gottlieb and Klasmeier-both of whom have an admitted financial interest in stem cell therapies-is a recent court case decided in favor of FDA by the US District Court in Washington, DC. In US v. Regenerative Sciences LLC, Judge Rosemary Collyer decided FDA had correctly interpreted its regulations by classifying certain stem cells manufactured by Regenerative Sciences as drugs and biological products.
Tissue or a Drug?
According to FDA, the company had subjected itself to FDA regulations by extracting mesenchymal stem cells, which were then cultured and grown before being injected back into a patient for therapeutic purposes. Collyer backed up FDA's assertions in her 23 July 2012 ruling against Regenerative Sciences, saying FDA's definition of "drug" was met in this particular case because of its intended use and violation of the "minimal manipulation" standard required to avoid FDA regulation.
Further, because it is a drug, it is regulated by FDA as opposed to the Colorado state medical board, which would ordinarily have jurisdiction over the matter. A similar April 2012 case in Texas ended with the Texas State Medical Board approving the ability of doctors to use adult stem cells on patients, potentially setting up a challenge between it and FDA.
The Regenerative case could have other, immediate effects on companies in the enforcement sights of FDA. A May 2012 letter sent to Pennsylvania-based Young Medical Spa cites the company for similar violations after FDA said it has altered the "relevant characteristics of the adipose tissue relating to the tissue's utility for reconstruction, repair or replacement" by extracting adult stem cells from the tissue.
"Therefore," continued FDA, "the processing would not meet the definition of minimal manipulation for structural tissue such as adipose tissue," and the tissue would be classified as a "biological drug product."
'A Reasonable Limit no Longer Exists'
Gottlieb and Klasmeier said the decision gets it wrong: The re-injection of a patient's own stem cells is a medical procedure-not an article like a drug or device.
"Federal regulators have stretched that definition to the point where a reasonable limit no longer exists," the authors explain. "The constraint that a drug needed to be a "thing" has been read out of the law by FDA, and the district court appears to have accepted that position."
Though Regenerative Sciences has appealed the decision, it could have an enormous impact on the sector, write Gottlieb and Klasmeier. "If the FDA's victory is upheld on appeal, then conceivably nothing done as part of clinical practice is beyond the agency's reach … there may be little to prevent the FDA from imposing its drug-approval requirements on any cell-based procedure."
Future Regulatory Challenges
This represents novel regulatory challenges, they note. In this case, personalized medicine is just that-personalized. Each person's cells are unique to their own genetic code, so how would clinical trials on a procedure be conducted, asks Gottlieb and Klasmeier. "The FDA requirements, designed for products manufactured and sold on a mass scale, can't be readily satisfied when it comes to treatments that are personalized to individual patients," they conclude.
Ultimately, Gottlieb and Klasmeier say, companies may not wait around for FDA or the courts to come to what they perceive to be a more amicable solution. Regenerative Sciences, they note, has already moved some of its laboratories to the Cayman Islands, and more seem likely to follow in light of additional enforcement actions by FDA.