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FDA Cracks Down on Stem Cell Treatments
Takes action against two clinics, promises more guidance and scrutiny for burgeoning industry
The FDA is cracking down on unapproved stem cell therapies, kicking off their initiative by seizing products at one California stem cell company and issuing a warning letter to a Florida clinic.In a statement, FDA Commissioner Scott Gottlieb, MD, said the agency is "stepping up our enforcement activity" regarding clinics selling unapproved stem cell therapies to patients.
On Friday, U.S. marshals seized five vials of live Vaccinia virus vaccine from San Diego-based StemImmune, which was being used in combination with fat-derived stem cells to treat cancer at the California Stem Cell Treatment Centers, the agency said.
FDA was particularly concerned about how the company acquired the vaccine; it's not commercially available and is usually reserved for those at high risk of smallpox, such as members of the military. The agency is currently investigating how the company obtained the product.
And in a warning letter to U.S. Stem Cell Clinic of Sunrise, Fla., the agency slammed the company for marketing stem cell products without FDA approval and for deviating from current good manufacturing practices that could put patients at risk.
That clinic was using fat-derived stem cells to treat a wide swath of conditions, including Parkinson's disease, amyotrophic lateral sclerosis, chronic obstructive pulmonary disease, heart disease, and pulmonary fibrosis.
"Unfortunately, these are examples of a larger pool of actors who claim that their unproven and unsafe products will address a serious disease, but instead put patients at significant risk," Gottlieb said in the statement.
"These dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products as a way of deceiving patients and preying on the optimism of patients facing bad illnesses. This puts the entire field at risk," Gottlieb continued. "Products that are reliably and carefully developed will be harder to advance if bad actors are able to make hollow claims and market unsafe science."
To curb the problem, Gottlieb said the agency will be issuing a regulatory framework for cell therapies and regenerative medicine. It will determine which procedures will be subject to approval, and which will not.
An agency spokesperson did not have an estimate as to how many of the stem cell clinics currently operating around the U.S. would be affected by that guidance.
Recent inspections at the California locations confirmed that the vaccine was used to create an unapproved stem cell product -- a combination of large doses of the vaccine plus fat-derived stem cells. It was then given to cancer patients -- either intravenously or directly into their tumors -- who potentially have compromised immune systems, posing the potential for harm such as myocarditis. The clinics are located in Rancho Mirage and Beverly Hills, Calif.
FDA said one of the five vials of Vaccinia virus vaccine was partially used; the other four remained intact. Each vial held 100 doses of the vaccine.
In an emailed statement, StemImmune said it is “fully cooperating with the FDA about the development of its stem cell-based investigational cancer therapy.”
A recent inspection of the Florida clinic found the clinic was creating adipose tissue-derived stem cells and giving the product back to patients either intravenously or injected into the spinal cord in order to treat several conditions, which would make it a drug and subject to FDA regulation.
The agency also found other "significant deviations" from current good manufacturing practice requirements, including a lack of established and written protocols to prevent microbial contamination, which could impact the sterility of the product and put patients at risk, FDA said.
The Florida clinic was also said to have impeded the FDA's investigation during the most recent inspection by refusing to allow entry except by appointment and denying FDA investigators access to employees.
In a video statement, U.S. Stem Cell Clinic chief science officer Kristin Comella said the company has offered the FDA unrestricted access to its clinic since 2014, and it will abide by all of FDA’s requirements.
Earlier this year, three elderly women treated at the Florida clinic went blind after having the adipose tissue-derived stem cells injected in an attempt to slow macular degeneration.
In his statement, Gottlieb emphasized that the FDA will draw "bright lines" between new treatments that are subject to FDA regulation, and the therapies that are "individualized by surgeons in such a way that they are not subject to FDA regulation."
Therapies that would require premarket review would include instances where cells undergo significant "manufacturing" or when cells aren't intended to perform their same basic functions before being returned to the body, Gottlieb said.
Therapies that wouldn't be subject to FDA review include cells or tissues taken from and given back to the patient; when they don't undergo significant "manufacturing;" or when they're intended to perform the same basic function and aren't combined with another drug or device.
A regulatory framework that will be "minimally burdensome and less costly" will be released this fall, Gottlieb said. It will build on a series of public meetings and work that FDA has already done, and will serve to implement provisions of the 21st Century Cures Act related to regenerative medicine.
Gottlieb also emphasized the agency's support of its Regenerative Medicine Advanced Therapy designation that industry is already familiar with, and said it is working on a new approach to aid small product developers. The framework will be accompanied by a compliance policy that will lay out a "reasonable" time frame for working with FDA to determine if a marketing authorization application is needed.
Finally, FDA formed a new working group to pursue "unscrupulous clinics through whatever legally enforceable means are necessary to protect the public health" and said it will seek to take additional actions in the coming months.
"Our aim is to provide an efficient route to market for promising technologies," Gottlieb said in the statement. "But at the same time, we will take a firm stance against those that prey on the medical promise of regenerative cell therapies to market treatments potentially unsafe or unproven so-called cures."
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