Unapproved stem cell therapies may cause patient harm with serious and life-threatening diseases.
The FDA this week issued a warning letter to American CryoStem Corporation over its marketing of an unapproved adipose-derived stem cell product, according to a press release.
The FDA also alleges that the manufacturer had serious deviations from current good manufacturing practice requirements that may lead to safety concerns and health risks for patients.
“As part of our comprehensive policy framework for the efficient development and regulation of cell-based regenerative medicine, we’re going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,” said FDA Commissioner Scott Gottlieb, MD. “We see great promise from the field of cell based regenerative medicine, but there are also novel risks.”
Due to a recent inspection, the FDA discovered that American CryoStem was processing adipose tissue and marketing the unapproved product, called Atcell, according to the release.
Although the adipose tissue is intended to be transplanted back into the same patient from whom it was derived, the processing involves significant manipulation, the FDA reported. The alterations change the original characteristics of the tissue, which may introduce contamination and harm to patients.
Under existing laws, these altered products require FDA approval to ensure safety and efficacy. These products may also create concern regarding the way it is administered and how it is promoted to treat life-threatening conditions, according to the release.
Atcell is distributed to physicians for the treatment of patients with anoxic brain injury, Parkinson’s disease, amytrophic lateral sclerosis, stroke, and multiple sclerosis. The unapproved product may be administered intravenously, intrathecally, or by inhalation, according to the FDA.
“The use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,” said Peter Marks, MD, PhD, director of the FDA’Center for Biologics Evaluation and Research. “In addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective.”
The FDA also discovered that the company deviated from current good manufacturing practice requirements that pose additional risks, including contamination with microorganisms and other quality defects, according to the release.
Specifically, the manufacturer used unvalidated processes, a poorly controlled environment, a lack of control of production components, and a lack of validated product testing, according to the FDA.
The agency is requiring the manufacturer to provide a detailed response on how they will correct the violations, including potentially submitting an investigational new drug application.
The FDA recently released a framework for regenerative medicine that clarified the definition of minimal manipulation and homologous use. For products that are subject under the law, the FDA plans to apply a risk-based approach to enforcement; however, they will not apply this enforcement to products that pose a safety concern to patients, such as Atcell, according to the press release.
“Our goal is to implement our new policy framework in a way that helps this promising field advance, while making sure that we address issues that can put patients at risk,” Dr Gottlieb said. “It also means taking an efficient, risk-based approach to making sure that products undergoing more than minimal manipulation, which makes them subject to FDA’s review and approval, and particularly products intended to treat serious and/or life-threatening disease or conditions, must prove that they deliver the benefits they’re promising to patients.”