The FDA announced that it was ramping up oversight of companies involved in regenerative medicine. Commissioner Scott Gottlieb remarked that as the agency "takes new steps to advance an efficient, modern approach to the regulation of cell-based regenerative medicine, at the same time we will be stepping up our enforcement actions against clinics that abuse the trust of patients and…endanger their health."
Specifically, the FDA said it plans to create a comprehensive policy framework more clearly describing its rules for regenerative medicine, adding that "this comprehensive policy is based on our existing authority." The agency also stated that it will provide "responsible product developers" a pathway for more efficiently attaining FDA approval through a "minimally burdensome and less costly" process.
"Ultimately, the agency's goal is to make sure that the potential of regenerative medicine can continue to advance to benefit the patients who need new and innovative options for their medical problems," commented Gottlieb, adding "these technologies hold out the potential to significantly alter the course of a broad range of diseases."
Stem cells for ocular indications have been gaining attention in recent years. Some companies are exploring the potential of using stem cells to restore vision in people with AMD, retinitis pigmentosa, and Stargardt disease.
In June 2016 the American Academy of Ophthalmology issued clinical guidance that covers the appropriate use of stem cell therapy in eye care. An article co-authored by the Academy’s chief executive officer in the November issue of the Academy’s journal Ophthalmology highlights the dangers of unapproved trials, the need for appropriate oversight and the risk to patients looking for a transformative cure.
The Academy has called on both the National Institutes of Health and the U.S. Food and Drug Administration to take steps to better ensure public safety associated with treatments outside of FDA-approved clinical trials. Good research is being performed under FDA-approved trials by innovative clinician-scientists; tragedy associated with non-approved used of stem cells threatens the conduct and impact of this high-quality science.
The move coincides with an FDA warning letter issued to US Stem Cell Clinic for marketing stem cell products for unapproved uses, as well as for significant deviations from current good manufacturing practice (cGMP) regulations. The agency said that following an inspection, it found that US Stem Cell Clinic was processing adipose tissue into stromal vascular fraction and administering the product intravenously or directly into the spinal cord of patients to treat various conditions such as amyotrophic lateral sclerosis, heart disease, Parkinson's disease, chronic obstructive pulmonary disease and pulmonary fibrosis. The FDA noted that it has not reviewed or approved any biological products developed by the firm for any use.
Commenting on the news, US Stem Cell Clinic said it abides by the FDA's requirements and "will switch to 510(k) approved systems to process tissue in-clinic such as bone marrow and/or fat systems while we work with the FDA to establish protocols going forward." The company, which earlier this year applied for a regenerative advanced therapy designation from the FDA, added that it also intends to respect specific stem cell guidelines that the agency is slated to release by December 13. Further, US Stem Cell Clinic noted that it is "not manufacturing a drug and therefore medical clinics are not required to follow cGMP."
Meanwhile, US authorities also raided the offices of the California Stem Cell Treatment Centers and seized five vials of StemImmune's live Vaccinia Virus Vaccine, which is typically reserved for people at high risk of smallpox. According to the FDA, the vaccine was being used to create an unapproved stem cell product that was then injected intravenously and directly into the tumours of cancer patients. "As the vaccine is not commercially available, the FDA has serious concerns about how StemImmune obtained the product for use as part of an unapproved and potentially dangerous treatment," the agency said, adding it is actively investigating the matter.