Stem Cells, FDA, and the Edge of Science: Three Expert …

Last week I wrote about a pending lawsuit by the FDA against Regenerative Sciences,a medical company thatdevelopeda stem cell treatment for orthopedic injuries. Whileadult stem cell therapies have been fraught with concerns about safetyand efficacy, this case is particularly important not only because it is unprecedented the FDA is attempting toclassify non-embryonicstem cellsas drugs but also because itbrings into focusthe disconnect between the pace of scientific progressand the struggle ofregulatorsto keep up withrapidly evolving technologies.

I spoke withthree people with aunique perspective onwhats at stake a lawyer who specializes in FDA regulation, a lab director of an in-vitro fertilization clinic, and a bioethicist to geta sense of how thecase is viewed intheir industries. One common threadthat emergedfrom these conversations is that the FDA is conflicted, even confused,about how to regulate stem cell-based products.Theprolonged legal battle with Regenerative(it dates back to at least 2008) is duelargely to the fact that the agency recognizes the absurdity of coming down with a one-size-fits-all regulatory approach thatwould apply to every clinicoffering such treatments.

To be sure, stem cell medicine has a terrible reputation.Stem cell therapies thatpurport to treateverything fromshot knees and slipped disks toautism, multiple sclerosis,and Parkinsons diseaseare rightlyregarded by many as modern-day quackery. That is because unscrupulous doctors have seized on the promise of this exciting new science and used it to peddle unproventreatments to desperateand ill-informedpatients.

In 2010, CBS aired a 60 Minutes special titled 21st Century Snake Oil,in whichDoug Sipp, a researcher at the RIKEN Center for Developmental Biology in Japan and a critic of stem cell fraud,who also runs the watchdog blog Stem Cell Treatment Monitor, warned of the dangers of blossoming stem cell medical tourism. That same year, NPR aired a story called Offshore Stem Cell Clinics Sell Hope, Not Science.Andin 2011, the journal Naturereported on Texas governor Rick Perrys crusadetosoften the FDAsposition on stem cellclinics,whose number in the U.S.is estimated to be around a dozen.The International Societyfor Stem Cell Research (ISSCR), a non-profit organization of medical doctors and scholars,alsofunctions as amyth busterfor clinically untestedtreatments. On its website, the group writes that themesenchymal stem cell, which is at the heart of the FDA lawsuit andis used byRegenerative in its procedures,has become a cell of intense interestbut has not yet been shown to have a clear-cut advantage over existing therapies, is not considered a standard of care for any condition and does not have regulatory approval for the routine treatment of any disease.

Against this background, the FDA is grappling with a difficult decision how to curb the proliferation of unproven stem cell treatmentshawked by unconscientious doctors, while recognizingthatthis branch ofmedicine holds alegitimatepromise whose full potential is only beginning to be discovered.

Areta Kupchyk, a partner at the law firm Nixon Peabody who counsels clients on FDA regulation and development of new biotechnologies, including human tissue and stem cells, says cell-basedproductsare tough to regulate because they are individualized and manipulated to different degrees for usesin individual patients.

The FDA has been trying to figure out how to regulate stem cell product development because it is so difficult individual hospitals, individual doctors are performing individual procedures. It has hada very difficult time over the years trying to regulate something thats not a big manufacturing facility. Its very hard to get your arms around something like that as a regulatory agency, Kupchyk told me ina telephone interview.

Shenoted that where minimal manipulation is concerned, the FDA may face a special challenge. One of the arguments in the agencys case for regulating Regenexx, a procedure in which adult stem cells are extracted, cultured in a special serum, and reinjected back into the same patient, is thatthe cells are more thanminimally manipulated.

I can understand why the FDA is focusing on minimal manipulation that is something they have been focusing on for a number of years. But they havent been able to articulate what minimal manipulation means. Theyre regulating on a case-by-case basis and theyre not informing the public well on where they draw the line,Kupchyk said.One good reason theyre not coming down is that this area is rapidly evolving and there are products created out of cells that have never been used or created before. So theyre struggling with new products, they are not sure how they are going to work in the body. This case could be very important in forcing the FDA to define it more clearly.

The number ofscientists and clinicsdeveloping or providing adultstem cell treatmentsis another reason the FDA may beat a disadvantage trying toestablish hard-and-fast new rules, Kupchyk added. If the FDA is successful in this case, its going to raise a lot of questions for everyone out there whos trying to develop new uses for stem cells for patients. There may be a chilling effect, certainly, and its very expensive to test new products [through the formal regulatory framework]. If every single physician or clinic who was doing something similar applied, the FDA would not be able to handle it. Thats another reason theyre not coming down on every single case.

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Stem Cells, FDA, and the Edge of Science: Three Expert ...