Scott Gottlieb, M.D., the Commissioner of the FDA, has stated that the agency is beginning to enforce stricter and more aggressive tactics against stem cell clinics who are marketing and administrating unproven therapies to patients. The administration will work toward cracking down on companies who claim to have cures or treatments for diseases that have no conclusive clinical efficacy.

In the Fall of 2017, “the FDA will advance a comprehensive policy framework that will more clearly describe the rules of the road for this new field” and will mostly be based on the administration’s existing authority, according to Gottlieb’s recent statement. Specifically, a new working group within the FDA will be directed to tackle “unscrupulous actors” within the field of stem cell therapies and regenerative medicine. These bad actors will be pursued “through whatever legally enforceable means are necessary to protect the public health.”

This new announcement comes after numerous reports that the FDA has been increasing their warnings to companies of investigational stem cell therapies. In a warning letter sent to the U.S. Stem Cell Clinic in Sunrise, Florida, the FDA stated that the clinic was selling stem cell treatments that were considered “unapproved” and “nonsterile.” The company later stated that its therapies, which involved the injection of stem cells into the spines of patients, violate no apparent laws associated with the marketing and administration of regenerative medicine. In another attempt to combat potentially dangerous stem cell therapies, the FDA also recently seized five live virus vaccines that were mixed with stem cells and injected into cancer patients’ tumors.

The use of stem cell therapies represents the most promising new fields in regenerative medicine; however, most of these technologies and therapies are in their early development stages. Clinical trials and research focused on stem cell biology has facilitated significant advancement in how these therapies might work in certain disease populations, including patients with cancer and neurological diseases.

Stem cell treatments provide hope for millions of people with hard-to-treat illnesses. Companies that claim their products provide curative potential in any form may be negatively affecting the perception of the industry as a whole. While some stem cell technologies may hold potential curative promises, their marketing as such may provide false hope to patients and place the entire field of regenerative medicine at risk, according to Gottlieb.

Discussion exists within the scientific community that the acceleration of therapy approval in the regenerative medicine space may facilitate swift improvement in patient care. Faster approval processes, however, may also translate to an increase in the number of corrupt or deceptive companies slipping through the cracks. Accelerating the approval and access of regenerative medicine comes with its own challenges, many of which the FDA is equipped to face. Katherine Hempstead, senior advisor at the Robert Wood Johnson Foundation, believes patient safety should be the top priority during the approval process of regenerative therapies. “Advancing the field of regenerative medicine is an important priority and is one of the goals of the CURES Act,” she explained, “but the issue of patient safety needs to be weighed with the accelerated approval process.”

Although some companies have taken advantage of regenerative medicine’s clinical promise by marketing products based on untested and unproven claims, most companies have continued to follow the rules and regulations set forth by the FDA. Still, the actors who exploit the clinical potential of more developed products and create false optimism among patients may be the ones contributing to the stalling of improvement within the field. According to Gottlieb’s statement, the products that are being developed based on the FDA’s regulations will become harder to advance and approve as long as “bad actors are able to make hollow claims and market unsafe science.”

Although stem cell treatments represent an exciting field of study, there is concern that this excitement may lead to challenges in the timely acknowledgement of potentially dangerous therapies that sporadically rise to the surface. This new field of regenerative technology has resulted in therapy creation in a variety of settings, according to Hempstead, but this sometimes leads to complications in both surveillance and enforcement. “The commitment to increased oversight is appropriate, but it will also be challenging,” Hempstead explained.
According to Hempstead, it’s just a matter of time before the industry can gain improved control in this field. This control relies on sound and well-designed clinical research. “Once the science is more settled,” said Hempstead, “bad actors may have more difficulty establishing a foothold. Right now they are taking advantage of uncertainty.”

There is concern that some companies in the regenerative medicine space are piggybacking on the promise the therapy holds and are marketing products that provide little to no benefit. Robert Califf, M.D., professor of Medicine at Duke University, echoed these concerns by saying he’s “afraid profit-seeking is common” within this industry.

He added that there is currently no clear definition or criteria for determining the difference between “naïve well-intended therapeutic optimism” or profit-chasing with vulnerable, at-risk patients. The recent policy efforts by the FDA initiated this fall will hopefully reduce profit-seekers, while improving the acceleration of therapies that do provide clinical potential.

This article was reprinted from Volume 21, Issue 35, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »