In a move aimed at encouraging the emerging field of regenerative medicine while also protecting patients from dangerous products, the U.S. Food and Drug Administration (FDA) on Wednesday issued guidelines for exempting certain cell and tissue treatments from the costly drug approval process.
"In the last decade, we've seen improbable advances that hold out great hope for patients," FDA Commissioner Scott Gottlieb said in a statement. "Our aim is to make sure we're being nimble when it comes to fostering innovation, while taking steps to protect the safety of patients."
The new policy sets out criteria for using biological products without formal FDA approval. The cells or tissues must come from the patient being treated; be used for the same function they perform naturally in the body; and be "minimally manipulated." The FDA believes such products pose a low risk of harm.
But the new guidance, put out as a draft in 2014, does not address the explosion of stem cell clinics offering treatments that may be ineffective or worse. Some clinics charge thousands of dollars to suck out and then inject cells taken from the patient's blood, bone marrow, or fat, saying they can fix everything from autism to Alzheimer's disease.
In the Philadelphia metropolitan area, for example, a new company founded by chiropractors called Regen America: Centers for Regenerative Medicine has been marketing bone marrow stem-cell treatments as virtual cures for arthritic knees and hips, and damaged spines. The marketing targets the elderly, even though orthopedic research shows they are least likely to benefit because the body's supply of stem cells diminishes with age.
Paul Knoepfler, a stem cell researcher at the University of California, Davis, School of Medicine and a leading voice of caution about the unbridled stem cell industry, called the new guidance "encouraging," but insufficient.
"The FDA seems serious about smoothing the path for those clinical researchers developing promising new stem-cell therapies based on rigorous data," Knoepfler said. "But it needs to do something major about the stem cell clinic problem on the other side of the coin, too."
During a press conference, FDA officials said they were not ignoring the problem of deceptive marketing, but that enforcement actions would first focus on outright dangerous clinics.
"We are going to be prioritizing where we take action," Gottlieb said, adding that top priority is products "that are not just being promoted inappropriately but are also putting patients at risk."
Peter Marks, director of the FDA's center for biologics evaluation, added, "There are literally hundreds of these clinics and we simply don't have the bandwidth to go after all of them at once."
Along with the new guidance, the FDA on Wednesday sent news outlets a consumer health alert that warned, "Stem cells have been called everything from cure-alls to miracle treatments. But don't believe the hype."
The only stem-cell therapies that the FDA has approved use cells from bone marrow or umbilical cord blood to treat blood cancers and certain immune disorders. Under rules that took effect in 2005, biologic tissues processed and marketed as therapies were supposed to go through the drug-approval process, which requires years of costly testing in humans to demonstrate safety and effectiveness.
Although there have not been many reports of serious injuries from unapproved stem-cell therapies, a handful of shocking examples led the FDA to intervene.
In August, as part of a vow to crack down on what it called "unscrupulous" practitioners, the agency announced the seizure of smallpox vaccine from a California company accused of combining the vaccine with fat-derived stem cells. The FDA also sent a warning letter to a Florida clinic that ruined the vision of three women by injecting stem cells into their eyes.
The FDA said manufacturers will have 36 months to determine whether their products qualify for exemptions from the drug-approval process, or must apply to begin that process. In general, the guidance says, low risk biological products are given by a shot rather than by intravenous infusion, inhalation or injection. Cells may be rinsed or concentrated by spinning in a centrifuge, but manipulation such as multiplying the cells in lab culture is prohibited.
Alta Charo, a professor of law and bioethics at the University of Wisconsin, praised the new guidance, but also called for better consumer information. California last month adopted a first-of-its-kind law requiring clinics to give each patient a handout disclosing that their stem-cell therapies are not approved by the FDA.
"While FDA does commit to pursuing enforcement for clinics offering the most risky interventions, this alone is not enough," Charo wrote in an email. "It is essential that patients be better informed of the regulatory status of the interventions, of the basis on which clinics have reason to believe the intervention is safe and effective, and of the fact that patients retain their legal right to complain if there has been negligence."