FDA Crackdown on Unapproved Stem Cell Therapies Starts with American CryoStem

This week, the US Food and Drug Administration (FDA) issued a warning letter to New Jersey-based CryoStem Corporation for marketing an adipose derived stem cell product without FDA approval. The company is also alleged to have made significant deviations from good manufacturing practice requirements that raise safety concerns and put patients at risk, according to a statement from FDA Commissioner Scott Gottlieb, MD.

“We’re going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,” Gottlieb said. “We see great promise from the field of cell based regenerative medicine, but there are also novel risks.”

Gottlieb’s crackdown aims to quell a disconcerting pseudoscientific trend — one where companies seek out loopholes so they can skirt regulatory pathways and market unapproved stem-cell treatments directly to consumers.

“This loophole is…now estimated to be a $2.4 billion per year industry, involving about 60,000 patients, where patients are paying up to $40,000 out of pocket per procedure to try and fight a number of diseases,” said Thomas A Albini, MD, associate professor of Clinical Opthalmology at the Bascom Palmer Eye Institute at the University of Miami Health System.

According to Albini, companies in the space focus on creating treatments for diseases where patients are frustrated with currently available therapies. At a presentation at the annual meeting of the American Society of Retina Specialists, he pointed to a company that produced unapproved stem cell eye drops for patients with wet age related macular degeneration who weren’t keen to take anti-VEGF injections in their eye.

Albini said that there is no shortage of unapproved treatments on the market, but none of them are well studied or can demonstrate adequate safety. Gottlieb is setting his sights on reversing that trend.

“Our goal is to implement our new policy framework in a way that helps this promising field [of stem-cell therapies] advances, while making sure that we address issues that can put patients at risk,” he said. “It also means taking an efficient, risk-based approach to making sure that products undergoing more than minimal manipulation…must prove that they deliver the benefits they’re promising to patients.”

The FDA’s announcement follows an inspection of American CryoStem facilities, located in Monmouth Junction, New Jersey. The agency found that American CryoStem was receiving and processing adipose tissue into a product called Atcell, and then marketing the product without required FDA approval.

Despite the fact that the product is intended for autologous use, the manner in which it’s processed involves more than minimal manipulation of the adipose tissue. These manufacturing steps alter the original relevant characteristics of the tissue and have the potential to introduce contamination of the product, creating risks of harm to patients, according to the FDA.

Atcell’s method of administration (intravenously, intrathecally, and via aerosol inhalation), as well as its marketing promotion as a treatment for serious or life-threatening conditions were dubious, the FDA said. The product is being distributed directly to physicians for the supposed treatment diseases including anoxic brain injury, Parkinson Disease, amyotrophic lateral sclerosis (ALS), stroke, and multiple sclerosis.

Moreover, the FDA’s inspection also uncovered evidence of deviations from current good manufacturing practice requirements in the production of Atcell that pose significant additional risks. According to the statement, the manufacturing process could leave the treatment open to contamination with microorganisms and other serious product quality defects.

“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’s Center for Biologics Evaluation and Research. “In addition, this product may also cause harm to patients who put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective.”

American CryoStem was issued a list of inspectional observations at the conclusion of the FDA’s inspection. The company responded, but the FDA found that responses were inadequate. Following the issuance of the January 4 warning, the FDA has request a response within 15 days from American CryoStem. The response must detail how the violations noted in the warning letter will be corrected.

Companies that do no correct violations may be subject to enforcement action such as seizure, injunction, and/or prosecution.