ISSCR Responds to FDA Announcement of Enforcement Direction

The International Society for Stem Cell Research (ISSCR) commends the U.S. Food and Drug Administration (FDA) for its policy direction and enforcement efforts, announced today, to provide “bright lines and appropriate oversight” when regulating stem cell treatments and regenerative medicine. The agency reported its enforcement actions against clinics in California and Florida offering unproven stem cell therapies, and announced pending policy and regulatory efforts against these practices.

“The FDA plays a critical role in ensuring the American public that stem cell medicines are safe and effective,” said ISSCR President Hans Clevers. “We have been very concerned about reports of patients using unproven medical therapies whose safety and efficacy have not been tested in systematic clinical trials,” he said. “Many of these patients have suffered great harm, and even death as a result of using unproven stem cell therapies. We are hopeful that increased regulatory enforcement against clinics offering unproven treatments will deter this practice and help protect patients,” he said.

Currently, stem cell therapies are known to be effective for only a small subset of diseases, mostly diseases that involve the blood-forming system. While stem cell research has enormous potential to yield new therapies for other diseases, in most cases, years of research will be required to identify the diseases that can benefit from stem cell treatments and to figure out how stem cells can be used safely and effectively in those contexts. Nonetheless, scores of clinics have sprung up throughout the United States selling stem cell therapies whose safety and efficacy have not been proven in clinical trials. In some cases, the “therapies” are not even scientifically or medically plausible. These clinics are preying on the hopes of desperate patients, selling what amounts to snake oil.

The ISSCR believes that the safety and efficacy of all stem cell and regenerative medicine therapies should be proven in systematic clinical trials, reviewed by the FDA, before they are sold to patients. In its 2016 Guidelines for Stem Cell Research and Clinical Translation (Section3), the ISSCR outlines clinical standards, including the requirement of persuasive evidence of clinical promise, as well as the need for independent review and oversight as potential treatments move through clinical testing toward the market.

“We agree with the FDA that regenerative medicine therapies have tremendous potential to address human health,” said Clevers. “We also believe that patient welfare is primary, and that risky, unproven therapies should be regulated more aggressively and should not be directly marketed to consumers,” he said.

According to its announcements, the FDA plans to develop a working group to use legal enforcement to pursue “unscrupulous clinics,” and will introduce a new policy framework this fall to address stem cell -and regenerative medicine treatments. The agency directs the public to report any stem cell clinic problems through its MedWatch Adverse Event Reporting program.